COMMISSION STAFF WORKING DOCUMENT IMPACT ASSESSMENT Strengthening of the EU Cooperation on Health Technology Assessment (HTA) Accompanying the document Proposal for a Regulation of the European Parliament and of the Council on health technology assessment and amending Directive 2011/24/EU
Date: 2018-01-31
Institutions: Directorate-General for Health and Food Safety, European Commission
Subjects: cross-border cooperation, exchange of information, health care, innovation, medical device, medical science, patient's rights, public health, scientific cooperation, technology assessment

EN 

  EN 

   EUROPEAN COMMISSION  Brussels, 31.1.2018  SWD(2018) 41 final  COMMISSION STAFF WORKING DOCUMENT  IMPACT ASSESSMENT  Strengthening of the EU Cooperation on Health Technology Assessment (HTA) Accompanying the document Proposal for a Regulation of the European Parliament and of the Council on health technology assessment and amending Directive 2011/24/EU {COM(2018) 51 final} - {SWD(2018) 42 final}  
 
 
1. 

Introduction _________________________________________________________ 10 

1.1. 

Context _________________________________________________________________  10 

1.2.  What is HTA?  ____________________________________________________________  11 

1.3. 

The role of HTA  __________________________________________________________  12 

1.4. 

State of play _____________________________________________________________  15 
HTA in the Member States  ___________________________________________________ 15 
HTA at EU level _____________________________________________________________ 21 

1.4.1. 
1.4.2. 

1.5. 

Political context __________________________________________________________  24 

1.6. 

International outlook ______________________________________________________  26 

2. 

Problem definition ____________________________________________________ 27 

Problem 1. Impeded and distorted market access _____________________________________  27 

Problem 2. Duplication of work for national HTA bodies  _______________________________  32 

Problem 3. Unsustainability of HTA cooperation ______________________________________  39 

3.  Why should the EU act? ________________________________________________ 41 

4. 

5. 

Policy objectives ______________________________________________________ 42 

Policy options ________________________________________________________ 43 

5.1.  Discarded policy option ____________________________________________________  44 

5.2. 

Key characteristics of the policy options  ______________________________________  44 

5.3.  Description of the policy options  ____________________________________________  46 
5.3.1. 
Policy option 1 (Baseline scenario). No Joint Actions after 2020 ___________________ 46 
5.3.2. 
Policy option 2. Project-based cooperation on HTA activities ______________________ 49 
5.3.3. 
Policy option 3. Permanent cooperation on common tools, procedures and early 
                dialogues  __________________________________________________________________ 51 
5.3.4. 
Policy option 4. Permanent cooperation on common tools, procedures, early  
                dialogues and joint REA______________________________________________________ 53 

6. 

Impacts of the policy options  ___________________________________________ 56 

Policy option 2. Project-based cooperation on HTA activities  ___________________________  57 
Economic impacts ___________________________________________________________ 57 
Social impacts ______________________________________________________________ 60 

6.1.1. 
6.1.2. 

Policy option 3. Permanent cooperation on common tools, procedures and early 
6.2. 
             dialogues ________________________________________________________________  62 
Economic impacts ___________________________________________________________ 62 
Social impacts ______________________________________________________________ 65 

6.2.1. 
6.2.2. 

6.3. 
Policy option 4. Permanent cooperation on common tools and procedures, early 
             dialogues and joint REA ____________________________________________________  66 
Economic impacts ___________________________________________________________ 67 

6.3.1. 

1 

 
 
6.3.2. 

Social impacts ______________________________________________________________ 71 

6.4. 

Analysis of the governance structure and financing system _______________________  72 
Description of the governance arrangements  ___________________________________ 72 
Feasibility and efficiency of the governance arrangements ________________________ 76 
Input from studies and stakeholders on the feasibility and efficiency of the  

6.4.1. 
6.4.2. 
6.4.3. 
                governance arrangements  ___________________________________________________ 80 
Costs related to the governance arrangements __________________________________ 81 
6.4.4. 

7. 

8. 

Comparing policy options ______________________________________________ 84 

Preferred policy option  ________________________________________________ 91 

8.1.  Description of the preferred option __________________________________________  91 
Joint outputs ________________________________________________________________ 91 
Technology scope ___________________________________________________________ 91 
Instrument __________________________________________________________________ 92 
Governance and financing ____________________________________________________ 95 

8.1.1. 
8.1.2. 
8.1.3. 
8.1.4. 

8.2. 

Justification of the preferred option __________________________________________  97 

8.3. 

Implications for Member States and other stakeholders  ________________________  101 

9. 

Monitoring and Evaluation ____________________________________________ 105 

2 

 
 
 
 
 
ANNEXES TO THE IMPACT ASSESSMENT: 

Annex I. Procedural Information 

Annex II. Stakeholder Consultation: Synopsis Report 

Annex III. Who is Affected by the Initiative 

Annex IV. Analytical Models 

Annex V. Health Technology Sectors 

Annex VI. European Cooperation on HTA  

Annex VII. International Outlook  

Annex VIII. Earlier Market Access Calculations 

Annex IX. Implementation Mechanisms and Policy Options  

Annex X. Costs of Joint Outputs and Overall Costs per Policy Option and Implementation 
Mechanisms 

Annex XI. Implementation Tables for Preferred Policy Option 

STUDIES CONDUCTED TO SUPPORT THE IMPACT ASSESSMENT 

Accessible online at: https://ec.europa.eu/health/technology_assessment/policy_en 

Mapping of HTA National Organisations, Programmes and Processes in EU and Norway. 
2017, Contract nr. 17010402/2016/734820, ISBN: 978-92-79-77080-7 

Mapping of HTA Methodologies in EU and Norway. 2017, DG SANTE Contract nr. 
17010402/2016/736040, ISBN: 978-92-79-77074-6 

Study on Impact Analysis of Policy Options for Strengthened EU Cooperation on HTA. 2017. 
Sogeti, Austrian Public Health Institute, London School of Economics. 
(CHAFEA/2016/Health/16), ISBN: 978-92-79-73966-8 

3 

 
 
 
 
 
 
 
 
 
 
ABBREVIATIONS 

CHAFEA 

Consumers, Health, Agriculture and Food Executive Agency 

ED 

EFPIA 

EMA 

Early dialogue 

European Federation of Pharmaceutical Industries and Associations 

European Medicines Agency 

EUnetHTA 

European Network for Health Technology Assessment 

EURORDIS 

Rare Diseases Europe 

GÖG  

HTA 

ICT 

IVD 

JA 

LSE  

OECD 

POP 

RCT  

REA 

SEED 

SMEs 

WHO 

Austrian  Public  Health  Institute  /  Gesundheit  Österreich  Forschungs- 
und Planungs GmbH  

Health Technology Assessment 

Information and Communication Technology 

In Vitro Diagnostics 

Joint Action 

London School of Economics 

Organisation for Economic Co-operation and Development 

EUnetHTA Planned and Ongoing Projects database 

Randomised controlled clinical trials 

Relative Effectiveness Assessment 

Shaping European Early Dialogues for health technologies 

Small and Medium Enterprises 

World Health Organization 

4 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
GLOSSARY1 

Term or acronym 

Meaning or definition 

Additional data generation 

Refers to the generation of additional clinical evidence in the course of 
an  HTA  process  and  includes  all  studies  and  provision  of  data  in 
addition to the clinical studies carried out for the purpose of obtaining 
marketing authorisation. 

Appraisal 

Refers to the drawing of conclusions on added value on the basis of the 
scientific  evidence  presented  in  the  HTA  report,  in  order  to  inform 
pricing and reimbursement decisions. 

Clinical Trials 

Clinical trials as defined in Regulation (EU) 536/2014 

Clinical assessment  

See 'REA' below 

Domains 

Early dialogue (see also 'Parallel 
scientific advice/early dialogue' 
below) 

EUnetHTA  

Refer to the areas of assessments covered by the HTA Core Model®. 
Four  are  clinical  domains  (i.e.  health  problem  and  current  use  of 
technology; description and technical characteristics of the technology; 
safety;  clinical  effectiveness)  and  five  are  non-clinical  (cost  and 
economic  evaluation;  ethical  analysis;  organisational  aspects;  patient 
and social aspects; legal aspects). (EUnetHTA) 

An early dialogue allows input from HTA bodies on the development 
of the health technology. It focuses on development strategies and not 
on pre-evaluation of data. The advice is prospective in nature (advice 
on on-going trials is out of scope). Early dialogues can be requested 
during the initial clinical development phase of the technology. For 
pharmaceuticals, it should ideally be requested at the end of the phase 
II to discuss the content of the planned Phase III i.e. planned 
confirmatory trial(s) and the economic rationale. The objective of an 
early dialogue is to reduce the risk of inadequate data when products 
are presented for evaluation with the aim of reimbursement by national 
health insurance. 
(SEED project) 

The European Network for Health Technology Assessment is a Joint 
Action, co–funded by the Health Programme of the European 
Commission (DG SANTE) and other participating actors. It gathers 
mainly national and regional HTA bodies and also organisations using 
HTA to support decision making. Its scope of activities is on scientific 
and technical issues. (See Annex VI for more details on EUnetHTA's 
activities)  

EUnetHTA joint work 

Activities  in  which  countries  and/or  organisations  work  together  in 
order  to  prepare  shared  products  or  agreed  outcomes.  These  may 
include,  for  example,  literature  reviews,  structured  information  for 

1 The purpose of this glossary is to provide the reader with better understanding of the terms used in this IA. It 
should in no way prejudge the terminology defined in the legal proposal.  

5 

 
 
 
                                                            
 
Economic assessment 

Efficacy 

Effectiveness 

Emerging health technology 

Full HTA 

Health technologies 

Health technology assessment 
(HTA) 

HTA Core Model 

rapid  or  full  HTAs,  early  dialogues  or  scientific  advice  on  R&D 
planning  and  study  design.  Joint  work  aims  at  supporting  Member 
States in providing objective, reliable, timely, transparent, comparable 
and transferable information and enables an effective exchange of this 
information. (HTA Network) 

The comparative analysis of the costs and consequences of two or more 
possible options. Depending on whether the consequences are 
expressed as monetary, physical or qualitative variables, the analysis 
may be a cost-benefit, cost-effectiveness or cost-utility analysis.  
(HTA Glossary.net) 

The extent to which an intervention does more good than harm under 
ideal circumstances, e.g. in a controlled clinical trial. (High Level 
Pharmaceutical Forum, 2005 – 2008. Final Report) 

The extent to which an intervention does more good than harm when 
provided under the usual circumstances of health care practice. (High 
Level Pharmaceutical Forum, 2005 – 2008. Final Report) 

A (new) health technology that has not yet been adopted within the 
healthcare system.  
Note: pharmaceuticals in the Phase II or III clinical trial, or pre-launch 
stage; medical devices are in the pre-marketing stage.  

A health technology assessment covering not only the clinical domains 
(i.e. REA), but also other non-clinical domains: cost and economic 
analysis, ethical analysis, organisational aspects, patient and social 
aspects, as well as legal considerations. 

Health technologies refer to a pharmaceutical, a medical technology or 
medical and surgical/radiation procedures as well as measures for 
disease prevention, diagnosis or treatment used in healthcare (Directive 
2011/24/EU). 

Health technology assessment (HTA) is a multidisciplinary process that 
summarises information about the medical, social, economic and 
ethical issues related to the use of a health technology in a systematic, 
transparent, unbiased, robust manner. Its aim is to inform the 
formulation of safe, effective, health policies that are patient focused 
and seek to achieve best value. (EUnetHTA) 

The HTA Core Model is a methodological framework for collaborative 
production and sharing of HTA information.  It consists of three main 
components: 

  The HTA ontology contains an extensive list of generic 

questions that can be asked in an HTA. The ontology also 
identifies relations between the questions 

  Methodological guidance helps researchers in finding answers 
to the questions defined by the ontology. It recommends the 
use of already existing, generally recognised guidance and 
guidelines along with other methodological recommendations 
and requires transparency on the methods used when applying 
the HTA Core Model 

6 

 
 
HTA methodologies 

HTA Network  

Horizon scanning  

Joint actions 

Joint output 

Joint reports 

Market launch 

  A common reporting structure for presenting findings in a 

standardised "question-answer pair" format  

The Guiding Principles on the HTA Core Model Use provides the basic 
principles on the Model's utilisation in various settings (EUnetHTA). 

Should be understood as scientific and technical methodologies applied 
by HTA institutions or groups of HTA researchers in the collection, 
analysis and synthesis of evidence and information on health 
technologies and their use in healthcare to inform decision making. 

It is a voluntary Network set up under Article 15 of Directive 2011/24. 
It gathers mainly Ministries of Health or competent authorities 
responsible for HTA, appointed by Member States. Its scope of 
activities is on strategic issues. The HTA Network works in synergy 
and complementarity with the Joint Action(s) EUnetHTA, which 
provides to the Network the technical and scientific expertise to foster 
EU cooperation on HTA. 

The systematic identification of health technologies that are new, 
emerging or becoming obsolete and that have the potential to effect 
health, health services and/or society.  
(HTA Glossary.net) 

Are collaborative projects specific to the Health Programme aiming to 
develop / share / refine / test tools, methods and approaches to specific 
issues or activities, and engage in capacity building in key areas of 
interest for the Member States and countries participating to the 
Programme. They are co-financed by the European Commission and 
authorities of the Member States. This type of project was introduced 
during the 2nd Health Programme (2008-2013) and continues under the 
current one (2014-2020). 

In this Impact Assessment, the term "Joint output" is used as an 
umbrella term to cover any result of joint work in the context of the EU 
cooperation. In particular it includes:  
(1) “Technology Specific Reports" produced through cooperation (Joint 
Early Dialogues, Joint Rapid Relative Effectiveness Assessments, Joint 
Full Health Technology Assessments);  
(2) “Common tools and procedures” essential for the cooperation, 
including IT tools enabling exchanges and data gathering 
(methodologies (e.g. EUnetHTA Core Model and Standard Operating 
Procedures/SOP), horizon Scanning, submission templates and 
templates for other key documents, training materials and other 
capacity building activities). 

"Joint reports" refer to REA and/or full HTA reports carried out jointly 
by Member States HTA bodies according to jointly agreed HTA 
methodologies and procedures.   

Occurs for medicinal products after a market authorisation has been 
granted (either at EU level or national level) and, for medical 
technologies (i.e. medical devices, in vitro diagnostics), once the CE 
marking is in place. It normally happens at Member State-level 
following the conclusion of pricing and reimbursement negotiations or 
when these are at an advanced stage. Market launch can subsequently 

7 

 
 
 
occur in other Member States. 

Medical Technologies  

Medical devices and in vitro diagnostics as defined by Regulation (EU) 
2017/745 and Regulation (EU) 2017/746 respectively  

Multiple Technology Assessments 
(MTA) 

Multiple Technology Assessments are assessments which cover more 
than one technology, or one technology for more than one indication 
(cf. NICE technology appraisal guidance) 

National uptake  

Other health technologies 

"National uptake" means that the joint output is used in national 
decision making process (i.e. in the same way as an output carried out 
at national level) and the joint activity is not duplicated (i.e. re-done) by 
HTA bodies at national/regional level.  

Currently the term is mainly used in the context of EUnetHTA output 
(i.e. joint assessments, submission templates, guidelines, POP 
Database, HTA Core Model®, etc.) where it refers to the general 
implementation of a joint output in a local/national HTA setting. 

Other health technologies refer to interventions that typically involve 
the use of pharmaceuticals, medical devices or diagnostics, but are 
characterised by additional layers of complexity (e.g. use of one or 
more technologies in the context of a medical procedure, or a 
vaccination or screening programme).  

Parallel scientific advice/early 
dialogue (see also 'early dialogue' 
above) 

It refers to the parallel/simultaneous scientific advice given by 
regulators and HTA bodies to medicine developers on the appropriate 
tests and studies to be carried out during the development of a new 
medicine. It started as a pilot project in 2010 by the EMA.   

As of July 2017 EMA offers consultations in parallel with EUnetHTA 
in order to allow medicine developers to obtain feedback from 
regulators and HTA bodies on their evidence-generation plans to 
support decision-making on marketing authorisation and 
reimbursement of new medicines at the same time. 

Priority setting 

The assignment of an order of priority based on explicit or implicit 
criteria for selection of health technologies for assessment. (HTA 
Glossary.net) 

Planned and Ongoing Projects 
(POP) Database 

Relative Effectiveness  

The POP database was set up by the EUnetHTA Joint Action and 
allows HTA agencies to share information with each other on planned 
and on-going projects conducted at the individual agency. The aim of 
the database is to reduce duplication and facilitate collaboration among 
HTA agencies. 

Relative effectiveness can be defined as the extent to which an 
intervention does more good than harm compared to one or more 
intervention alternatives for achieving the desired results when 
provided under the usual circumstances of health care practice (High 
Level Pharmaceutical Forum 2005-2008, European Commission DG 
Enterprise & Industry and DG Health & Consumers)   

Rapid Relative Effectiveness 

The Rapid Relative Effectiveness Assessment (REA) covers and is 
limited to the clinical domains and measures the medical/therapeutic 

8 

 
 
 
 
Assessment (REA) 

added value of a technology. It is also called clinical assessment.  

SEED  

"Shaping European Early Dialogues for health technologies" was a 
project running from 2013 to 2015, financed by the European 
Commission for conducting pilots on early dialogues with health 
technology developers (pharmaceuticals and medical devices) by 
participating HTA bodies. The work was carried out based on 
experience from and in synergy with the EUnetHTA Joint Action 2. In 
total, eleven early dialogues were carried out are planned with an aim 
to conduct 7 on medicinal products and 3 on medical devices. 

Single Technology Assessment 
(STA) 

Single Technology Assessment is an assessment of a single technology 
for a single indication (NICE technology appraisal guidance) 

9 

 
 
 
 
 
1.  Introduction 

1.1.  Context 

The rapidly evolving health technology2 market provides important opportunities to improve 
public  health  by  delivering  better  outcomes  for  patients  and  society  as  a  whole.  The  health 
technology  market  is  also  a  key  driver  of  economic  growth  and  innovation  in  the  Union. 
Pharmaceuticals  and  medical  technologies3  are  two  large  sectors  of  the  Union’s  health 
technology market, contributing significantly and steadily to growth and job creation, even in 
years  of  slower  economic  development.  (For  more  information  on  these  sectors,  see  Annex 
V).  

At the same time, in the EU, the total (public and private) health care expenditure amounts to 
around EUR 1 300 billion per annum4 (including EUR 220 billion for pharmaceuticals5 and 
EUR 110 billion for medical technologies).6 Health care expenditure thus accounts on average 
for  approximately  10%  of  EU  GDP.7  This  expenditure  is  likely  to  increase  in  the  coming 
years, given inter alia Europe’s ageing population, the increase of chronic diseases, and the 
expected  introduction  of  complex  new  technologies.8,9  Simultaneously,  Member  States  are 
increasingly confronted with budgetary constraints which will require them to further improve 
the  efficiency  of  their  health  care  systems  in  order  to  ensure  the  maximum  benefit  for 
individual patients and public health in general.10  

In order to address the above mentioned challenges and opportunities and to balance various 
interests,  health  technology  assessment  (HTA)  has  become  an  increasingly  important  tool 
used to assist Member States in creating and maintaining sustainable health care systems and 
to stimulate innovation that delivers better outcomes for patients11 and cooperation is ongoing 
at EU level. 

This  Impact  Assessment  has  aimed  to  investigate  opportunities  for  strengthening  EU 
cooperation on HTA, building on its achievements and addressing its shortcomings. 

2 See section 1.2 for further details on the scope of health technologies.  
3 For the purpose of this Impact Assessment, the term medical technologies comprises medical devices and in 
vitro  diagnostics  as  defined  by  Regulation  (EU)  2017/745  and  Regulation  (EU)  2017/746  respectively  (see 
Annex V for further details). 
4  Eurostat  -  expenditure  of  providers  of  health  care  using  data  from  2012  or  latest  data  entry  for  all  Member 
States available. The figure is complemented by WHO Health data for the following countries: IE, IT, MT and 
UK (ECB annual exchange rate).  
5  Eurostat  data,  see  DG  GROW  SWP  2014.  Pharmaceutical  industry:  A  Strategic  Sector  for  the  European 
Economy. 
6 Communication on Safe, effective and innovative medical devices and in vitro diagnostic medical devices for 
the  benefit  of  patients,  consumers  and  healthcare  professionals.  COM(2012)  540  final.  World  Bank,  EDMA, 
Espicom and Eucomed calculations.  
7 European Commission. European Semester Thematic Fiche: Health and Health systems, 2015. 
8 DG ECFIN. The 2015 Ageing report, 2015. and DG ECFIN- EPC 2016. Joint Report on Health Care and Long-
Term Care Systems & Fiscal Sustainability, Institutional Paper 37. 7 October 2016 
9 OECD. 2015. Pharmaceutical expenditure and policies: past trends and future challenges. 
10 DG ECFIN. Cost-containment policies in public pharmaceutical spending in the EU, 2012. 
11  Council  conclusions  on  strengthening  the  balance  in  the  pharmaceutical  systems  in  the  EU  and  its  Member 
States (2016/C 269/06). 

10 

 
 
 
                                                            
1.2.  What is HTA? 

In  this  Impact  Assessment  report,  HTA  is  defined  as  "a  multidisciplinary  process  that 
summarises information about the medical, social, economic and ethical issues related to the 
use of a health technology in a systematic, transparent, unbiased, robust manner. Its aim is to 
inform the formulation of  safe, effective health policies that are  patient focused  and seek to 
achieve  best  value".12  The  term  ”health  technology”  is  to  be  understood  in  a  broad  sense 
in  vitro 
comprising  pharmaceuticals,  medical 
diagnostics)13 and other technology-based tools for disease prevention, diagnosis or treatment 
used in healthcare.14 

(medical  devices  and 

technologies 

HTA is thus an evidence-based process that independently and objectively assesses a (new or 
existing)  technology  and  compares  it  to  other/existing  ones.  A  HTA  can  cover  different 
aspects  (domains)  ranging  from  clinical  domains  (e.g.  safety,  clinical  effectiveness)  to  non-
clinical domains (e.g. economic, ethical, organisational etc.) (see Figure 1). Broadly speaking 
two  types  of  assessments  can  be  distinguished:  (1)  the  Rapid  Relative  Effectiveness 
Assessment (REA)15 which covers the clinical domains and evaluates the medical/therapeutic 
added value of a technology; and (2) the full HTA, which also includes other domains (e.g. 
cost-effectiveness).  

Whilst  clinical  assessments  (REA)  are  often  based  on  global  evidence  (e.g.  global  clinical 
trials  in  the  case  of  pharmaceuticals),  full  HTA  assessments  include  domains  that  are  more 
sensitive to national/regional contexts. 

Figure 1. HTA domains (based on EUnetHTA HTA Core Model) 

12 EUnetHTA Joint Action definition. 
13 Medical devices and in vitro diagnostics as defined by Regulation (EU) 2017/745 and Regulation (EU) 
2017/746 respectively 
14 This includes more complex health interventions that involve the use of pharmaceuticals, medical devices or 
diagnostics (e.g. in the context of a medical procedure, or a vaccination or screening programme). 
15 The generally accepted term is Relative Effectiveness Assessment. If REA is done at the time of marketing 
authorisation, it is usually based on efficacy data from clinical trials. For re-assessments, the availability of data 
on  effectiveness  is  more  frequent.  Efficacy:  is  the  extent  to  which  an  intervention  does  more  good  than  harm 
under ideal circumstances. Effectiveness is the extent to which an intervention does more good than harm when 
provided under the usual circumstances of health care practice (High Level Pharmaceutical Forum, 2005 – 2008. 
Final Report). 

11 

 
 
 
                                                            
1.3.  The role of HTA 

Before new health technologies are placed on the market, they are evaluated for their quality, 
safety  and  efficacy  (marketing  authorisation  of  pharmaceuticals)  or  safety  and  performance 
(CE  marking  of  medical  devices).  While  HTA  builds  on  the  evidence  used  for  these 
assessments,  it  focuses  specifically  on  the  potential  benefits  of  a  new  health  technology  in 
comparison to the existing standard of care in the health system. The box below discusses the 
differences  between  marketing  authorisation  and  HTA  in  more  detail,  including  the  added 
value  of  HTA.  Market  access  pathways  for  health  technologies  and  relevant  EU  legislation 
are described in detail in Annex V. 

Differences  between  marketing  authorisation  and  the  clinical  part  of  HTA  (example  of 
pharmaceuticals) 

Marketing  authorisation  and  HTA  have  different  remits  and  answer  different  questions,  even  if  they 
base their answers on some common evidence (e.g. pivotal clinical trials, typically phase III trials). 

Marketing authorisation assesses the quality, safety and efficacy of an individual product. A marketing 
authorisation  is  granted  if  a  new  product  has  a  positive  benefit-risk  ratio  in  the  sense  that  it  is 
efficacious and its safety profile is acceptable. It is not within the remit of the marketing authorisation 
to  determine  the  existing  standard  of  care  and  to  conduct  a  comparative  assessment  of  the  new 
product against alternative products reflecting the standard of care. By contrast, the clinical part of 
HTA (REA) assesses the  added clinical value of a product, i.e.  its  relative effectiveness and  relative 
safety  compared  to  one  or  more  existing  products  (or  other  health  interventions)  reflecting  the 
standard  of  care.  HTA  therefore  reviews  and  uses  a  broader  evidence  base  than  the  assessment  for 
marketing authorisation: First of all, the evidence base on existing products/interventions needs to be 
reviewed  in  order  to  determine  the  current  standard  of  care.  Subsequent  steps  of  the  HTA  process 
analyse in how far the pivotal clinical trials submitted for marketing authorisation purposes cover the 
full  spectrum  of  the  standard  of  care.  Frequently,  these  trials  include  one  comparator,  while  the 
standard of care includes more than one alternative pharmaceutical/intervention. HTA will therefore 
review additional studies on other relevant pharmaceuticals/interventions and consider whether and 
how this additional evidence can be assessed (e.g. via indirect comparisons or network meta-analysis 
approaches). 

Moreover,  HTA  aims  to  understand  relative  effectiveness  under  the  conditions  of  usual  clinical 
practice (rather than under the ideal conditions of a controlled trial). Therefore, relevant data sources 
for the clinical part of HTA go beyond the initial pivotal clinical trials and also include observational 
(“real  world”)  data  from  clinical  practice  (e.g.  disease-specific  patient  registries,  health  data 
recorded  by  health  services  and  insurances).  When  HTA  is  conducted  around  the  time  of  or  shortly 
after  marketing  authorisation,  some  limited  effectiveness  data  may  already  be  available  (e.g.  from 
early access schemes in some EU Member States, or from another jurisdiction such as the U.S. where 
the product was licensed earlier). Even if relative effectiveness data from clinical practice are not yet 
available, HTA may use modelling approaches to predict relative effectiveness based on efficacy data 
from pivotal trials. At later points in time (HTA re-assessments), relative effectiveness assessments can 
typically draw on increasing sources and amounts of data from clinical practice. 

Finally, the clinical  part of HTA differs from the assessment for marketing authorisation in the way 
that patient subgroups are considered and analysed. HTA aims to consider all patient subgroups that 
are  relevant  for  clinical  practice  (whether  or  not  they  may  have  been  included  and  analysed  in  the 
pivotal clinical trials for marketing authorisation purposes). To this end, HTA first considers whether 
the  existing  standard  of  care  differs  for  different  patient  subgroups  within  a  particular  therapeutic 
indication. HTA then assesses whether and how the added value of the new pharmaceutical differs by 
patient subgroup. 

12 

 
 
It  is  also  important  to  underline  that  HTA  does  not  comprise  pricing  and  reimbursement 
decisions.  However,  HTA  can  substantially  contribute  to  the  sustainability  of  health 
systems16 by providing scientific evidence/input for national decision-making on pricing and 
reimbursement.17  The  link  between  HTA  and  the  decision  on  pricing  and  reimbursement  is 
currently  more  pronounced  in  the  case  of  pharmaceuticals  than  for  medical  technologies 
(medical  devices  and  in  vitro  diagnostics)  and  other  health  technologies  (e.g.  complex 
interventions). For pharmaceuticals, pricing and reimbursement decisions are typically taken 
at national/central level at times of market launch (or shortly thereafter), whereas for medical 
and other health technologies such decisions are often taken in a more decentralised manner, 
e.g. through local (hospital level) decisions or procurement processes, with more limited input 
from  HTA  reports.18  However,  there  is  a  growing  trend  towards  applying  HTA  to  support 
decision-making  also  for  medical  technologies  and  other  health  technologies,  including  the 
development  of  HTA  methods  and  processes  adapted  to  the  specificities  of  these 
technologies.19,20 

Figure  2  shows  a  schematic  overview  of  the  HTA  step  in  the  market  access  pathways  for 
pharmaceuticals and medical technologies. For further details, see Annex V. 

Pharmaceuticals 

Medical devices and in vitro diagnostics 

Figure 2. HTA in the context of market access pathways for pharmaceuticals and medical 
technologies21 

It should also be noted that in addition to informing national/local pricing and reimbursement 
decision, HTA supports the development of evidence-based clinical guidelines (for diagnosis 

16  European  Commission  (DG  ECFIN-  EPC),  Joint  Report  on  Health  Care  and  Long-Term  Care  Systems  & 
Fiscal Sustainability – Volume 1, Institutional Paper 037 | October 2016.  
17 A distinction also needs to be made between health technology “assessment” and “appraisal”. Assessment is 
defined  as  compilation  and  critical  evaluation  of  the  available  scientific  evidence  on  all  or  selected  domains, 
whereas  appraisal  means  that  conclusions  are  drawn  on  the  basis  of  the  assessment  results  which  are  used  to 
support national or regional decision-making, typically on pricing and reimbursement. The scientific process of 
assessment demonstrates potential for convergence and EU cooperation, whereas appraisals are not in line with 
the explanation above. 
18 See Annex V. Health Technology Sectors 
19  Tarricone  R.,  Torbica  A.,  Drummond  M.  and  MedtecHTA  Project  Group:  Key  Recommendations  from  the 
MedtecHTA Project. Health Econ. 2017 Feb; 26, Suppl 1:145-152. 
20  Lysdahl  KB.,  Mozygemba  K.,  Burns  J., Brönneke  JB.,  Chilcott  JB.,  Ward  S.,  Hofmann  B..  Comprehensive 
assessment of complex technologies: Integrating various aspects in health technology assessment. Int J Technol 
Assess Health Care. 2017 Aug 7:1-7. 
21 R&D: Research and development. P&R: pricing and reimbursement. 

13 

 
 
 
  
                                                            
and  treatment  of  patients  in  particular  therapeutic  areas)  and  thus  promotes  evidence-based 
healthcare.  Finally,  HTA  is  applied  to  both  new  technologies  coming  to  the  market  and  to 
technologies  which  have  been  in  use  in  healthcare  for  some  time,  i.e.  it  informs 
(dis)investment  decisions  for  new  and  existing  health  technologies.  HTA  thus  helps  to 
prioritise health technologies with high added value and to de-prioritise technologies with no 
or  limited  additional  benefits  (lower  prices,  disinvestment,  and  discontinued  use).  In 
summary,  HTA  can  facilitate  evidence-based  decision-making  and  efficient  allocation  of 
resources in healthcare, ultimately supporting the optimisation of national healthcare systems. 

Some individual studies offer a certain level of insight on the potential role of HTA in terms 
of economic benefits.22,23,24 A recent study from the UK,25 focusing on 10 HTAs reached the 
conclusion that a potential benefit of approximately GBP 3.0 billion/year could be achieved in 
the UK if the recommendations from HTA reports were fully followed. Another study from 
Austria  found  that  for  medical  technologies  in  hospitals,  HTA  led  to  more  reasonable 
investments and saved several million euros at the level of a single hospital association.26 In 
the lower income countries, HTA is particularly important as these Member States have more 
limited financial resources and the health status of their populations also tends to be lower.27  

A  well-functioning  HTA  system  also  improves  business  predictability  for  industry  and 
creates and maintains a stimulus for innovation.28,29 A predictable HTA system which rewards 
innovations  with  added  value  for  patients  can  influence  longer-term  R&D  investment 
decisions  by  industry  and  thus  play  an  important  role  in  incentivising  innovation  for  the 
benefit  of  patients.  In  particular,  it  can  help  to  steer  industry  resources  towards  the 
development of products that address unmet medical needs and significantly improve health 
outcomes for patients.30  

HTA  can  also  contribute  to  greater  transparency  and  has  the  potential  to  improve  the 
involvement of key stakeholders, such as patients and health professionals. Patients' input is 

22 Schumacher I, Zechmeister I: Assessing the impact of health technology assessment on the Austrian healthcare 
system. Int J Technol Assess Health Care 2013, 29:84–91.   
23 Ognyanova, D, Zentner, A, and Busse, R. Pharmaceutical reform 2010 in Germany: striking a balance between 
innovation and affordability. Eurohealth (Lond). 2011; 17: 11–13   
24  Rosen,  M.  and  S.  Werko  (2014).  "Does  health  technology  assessment  affect  policy-making  and  clinical 
practice in Sweden?" International Journal of Technology Assessment in Health Care 30(3): 265-272. 
25  Guthrie  S,  Hafner  M,  Bienkowska-Gibbs  T  and  Wooding  S,  Returns  on  research  funded  under  the  NIHR. 
Health Technology Assessment (HTA) Programme: Economic analysis and case studies. RAND Report RR-666-
DH, 2015. Estimated using assuming that recommendations of 10 HTA reports were followed in the UK during 
the course of 1 year. Figure report the potential net-benefit including possible savings and health gains in terms 
of QALYS using a value of £20,000 per QALY.  
26 Schumacher I, Zechmeister I: Assessing the impact of health technology assessment on the Austrian healthcare 
system. Int J Technol Assess Health Care 2013, 29:84–91.   
27 Kaló, Z., Gheorghe, A., Huic, M., Csanádi, M., and Kristensen, F. B. (2016) HTA Implementation Roadmap 
in Central and Eastern European Countries. Health Econ., 25: 179–192.  
28 Kanavos, P., Manning M., Taylor D., Schurer W., Checch K., (2010): Implementing value-based pricing for 
pharmaceuticals in the UK. London 
29  OECD  (2004):  Increasing  value  for  money  in  health  systems:  the  quest  for  efficiency.  In:  Towards  High-
Performing Health systems. 
30 Rovira, J. et al, (2015) Health technology assessment and the Incentives to Innovation in the life Cycle of a 
Health technology. In. Health Technology Assessment and Health Policy Today: A Multifaceted View of their 
Unstable Crossroads.   

14 

 
 
                                                            
particularly  relevant  for  assessing  which  treatment  options  improve  their  health-related 
quality of life.31 

In  the  Open  Public  Consultation  carried  out  by  the  Commission  addressed  to  citizens,  the 
majority  of  the  respondents  (98%)  indicated  that  they  consider  HTA  useful  for  decision 
making (see Annex II). 

1.4.  State of play 

1.4.1.  HTA in the Member States 

In the last 20 years, all Member States have started to introduce HTA processes at national or 
regional level (i.e. with 51 HTA bodies established in 26 Member States).32 There are national 
legal frameworks for HTA in place in 26 Member States and Norway; some Member States 
are only at the initial phase of establishing HTA systems and/or have dedicated only limited 
resources to HTA. Whilst there is some convergence in national HTA systems there are also 
significant  discrepancies.  A  summary  of  these  discrepancies  is  set  out  below.  For  further 
information see Annexes VIII, IX and X.33  

(a) Main differences in the procedural framework 

1)  The  national  HTA  systems  differ  in  the  scope  of  health  technologies  that  are  being 
assessed. Whilst the majority of Member States and Norway report national HTA activity for 
pharmaceuticals,  two  Member  States  have  no  such  activities,  but  are  in  the  process  of 
developing  their  national  HTA  systems.  20  Member  States  and  Norway  indicate  having  an 
HTA  system  for  medical  devices  (with  five  of  these  countries  stating  that  it  has  not  been 
formalised  yet).  17  Member  States  and  Norway  indicate  that  they  have  an  HTA  system  for 
other technologies, whilst the remaining countries do not carry out such assessments.  

2) National HTA organisations also differ in terms of tasks allocated. The main role of most 
HTA  organisations  is  to  carry  out  assessments  and  provide  recommendations  for  decision 
making (i.e. pricing and reimbursement decisions). In addition to this main role, some HTA 
bodies develop quality standards (12 Member States and Norway) and/or clinical guidelines 
(14  Member  States  and  Norway),  perform  horizon  scanning34  (10  Member  States  and 
(11  Member  States  and  Norway),  or  offer  early 
Norway),  manage 
dialogues/scientific  advice  to  health  technologies  developers  (12  Member  States  and 
Norway). 

registries 

3) Concerning the resources available in the national HTA organisations, the study mapping 
on HTA processes across the EU reveals that there are significant differences between MS. In 
particular,  the  number  of  staff  ranges  from  no  human  resources  being  dedicated  to  HTA 

31  Patient  Involvement  in  Health  Technology  Assessment  in  Europe.  Results  of  the  European  Patients'  Forum 
Survey. 2013. 
32  Mapping of HTA national organisations, programmes and processes in EU and Norway. 2017. Contract nr. 
17010402/2016/734820, ISBN: 978-92-79-77080-7. 
33 A short summary of such differences is also found in the ECP-ECFIN report on ageing. Getting more value for 
money: the example of Health Technology Assessment (HTA). p. 100.  
34 Horizon scanning refers to the systematic identification of emerging technologies that could have significant 
effects on health care, and which might be considered for health technology assessment (WHO definition). 

15 

 
 
 
 
                                                            
activities (one Member State), to departments with less than 10 full-time equivalents (FTEs) 
(seven Member States), to countries with more than 100 FTEs (four Member States) with a 
maximum  of  600  FTEs  (one  Member  State).  The  available  expertise  of  the  core  staff  also 
varies between HTA organisations.35 36 As regards financing, HTA organisations from 26 EU 
Member  States  and  Norway37  are  public  bodies,  most  of  them  being  financed  from  public 
money  (annual  budget  allocated  from  governments).  A  combination  of  budget  and  service 
fees directly received from industry is reported by five Member States. The data provided by 
26 HTA bodies show that the annual budget allocated to HTA varies from no specific budget 
(one  Member  State)  or  Member  States  with  up  to  EUR  100  000  (four  Member  States)  to 
Member  States  with  more  than  EUR  1  million  (nine  Member  States  and  Norway),  with  a 
maximum of EUR 70 million (one Member State). It has to be noted that the cost of one HTA 
report varies considerably. In the survey conducted by the GÖG-LSE study38, the cost of an 
HTA  (single  technology  assessment)  reported  by  HTA  bodies  ranged  from  EUR  4  000  to 
EUR 135 000. This reflects differences in the resources available to different HTA bodies, but 
also  factors  such  as  the  scope  and  depth  of  the  assessment  (e.g.  how  much  an  HTA  body 
invests into conducting its own analysis to evaluate and contextualise the evidence generated 
by industry)39.  

4) In many  Member States, HTA bodies consider a  dossier submitted by industry in their 
assessments. For pharmaceuticals, 20 Member States reported that they carry out a review of 
an industry submission of evidence. The extent of this review varies among HTA bodies and 
can  cover  aspects  such  as  missing  evidence,  errors  in  submitted  evidence,  internal  and 
external  validity,  as  well  as  additional  evidence  analyses  produced  by  the  HTA  body  itself 
(e.g.  based  on  the  scientific  literature  or  clinical  study  registers).  For  medical  technologies 
only  9  out  21  Member  States  reported  that  they  review  industry  submissions,  i.e.  a  greater 
proportion of Member States carry out their own standalone assessments.40 

35  Gulacsi,  L.,  Rotar  A.,  Niewada,  M.,  Loblova  O.,  Rencz  F.,  Petrova  G.,  Boncz  I.,  Klazinga,  N.S.  Health 
technology  assessment  in  Poland,  the  Czech  Republic,  Hungary,  Romania  and  Bulgaria.  Eur  J  Health  Econ 
(2014) 15 (Suppl 1):S13–S25   
36 WHO, 2015 Global Survey on Health Technology Assessment by National Authorities - Main findings 
37  Information  from  two  Member  States,  Greece  and  Cyprus,  which  are  currently  in  the  process  of  setting  up 
their national HTA systems is not available. 
38 Study on impact analysis of policy options for strengthened EU cooperation on HTA. 2017. Sogeti, Austrian 
Public Health Institute, London School of Economics. CHAFEA/2016/Health/16. ISBN: 978-92-79-73966-8. 
The GÖG-LSE Study is the main study supporting the Impact Assessment process by collecting evidence and 
providing  an  in-depth  analysis  on  the  potential  impacts  of  identified  policy  options  for  cooperation  of  the 
European  Commission  (including  the  baseline  scenario),  and  providing  the  relevant  literature  on  HTA,  with  a 
specific  focus  on  the  European  Union.  The  study  was  carried  out  by  a  consortium  consisting  of  Gesundheit 
Österreich  Forschungs-  und  Planungsgesellschaft  (Austria),  London  School  of  Economics  -  LSE  Health  (UK) 
and  SOGETI  (Luxembourg).  For  establishing  the  baseline  scenario,  a  relevant  sample  of  health  technologies 
which  included  20  pharmaceuticals,  15  medical  devices  and 5  “other  technologies”  (including  complex  health 
interventions)  was  analysed  (i.e.  HTA-process  per  type  of  technology  and  Member  State,  costs  incurred  by 
technology  developers/industry  and  HTA  bodies  per  technology,  influence  of  the  legislative  framework  on 
technology  developers).  The  analysis  of  the  impacts  included  a  survey  concerning  the  opinions  of  industry, 
public  administrations  and  other  stakeholders  on  the  potential  economic  and  social  impacts  of  the  identified 
policy options, complemented by focus groups, interviews and findings from literature review. A description of 
the implementation mechanisms and an estimation of their costs were also provided. The study has been peer-
reviewed by leading experts in the field. 
39 EUnetHTA WP7 draft report and study "Mapping of HTA national organisations, programmes and processes 
in EU Member States and Norway"  
40 EUnetHTA WP7 draft report 

16 

 
 
                                                            
5)  As  regards  the  type  of  assessments,  all  Member  States  carry  out  single  technology 
assessments (STA) (i.e. an assessment of a single technology compared with the standard of 
care)  and  13  Member  States  perform  multiple  technology  assessments  (MTA)  (e.g.  an 
assessment  of  several  technologies  in  use  for  a  particular  clinical  indication).41  For 
pharmaceuticals most of the Member States apply a single technology assessment (STA). Six 
Member  States  and  Norway  indicate  performing  both  STAs  and  MTAs.  For  medical 
technologies,  seven  Member  States  and  Norway  indicate  carrying  out  STAs  and  MTAs, 
whilst only six Member States report carrying out only STAs for medical devices. 

6)  The  number  of  assessments  produced  varies  considerably  between  countries.  The 
mapping  study  of  HTA  procedures  across  Europe  showed  that  the  number  of  assessments 
carried  out  by  HTA  bodies  (single technology assessments  of  pharmaceuticals,  medical  and 
other health technologies) ranged from about 5 HTA per year to up to 390 HTA per year. For 
pharmaceuticals, some HTA bodies assess all new products (including generics, biosimilars) 
and all licence extensions (including minor variations) of existing products. However, given 
current national working practices, pharmaceutical topics that are most likely to be assessed 
by  many  Member  States  across  the  EU  are  products  with  new  active  substances  requiring 
central  marketing  authorisation  and  major  licence  extensions  of  existing  products.42  For 
medical  devices,  the  number  of  assessments  performed  annually  is  lower  than  for 
pharmaceuticals across all EU countries.43 Innovative medical technologies with potentials to 
transform the organisation of care (“transformative technologies”) and  medical technologies 
subject to the scrutiny mechanism44 are most likely to be covered in national assessments. 

7) According to a survey of HTA bodies, the  time needed to complete a health technology 
assessment  process  (single  technology  assessment,  from  topic  selection/identification  to 
delivery of HTA results) ranges from a few weeks to more than a year.45 For pharmaceuticals 
assessments based on industry submissions, the time from industry submission to completion 
of the review ranges from less than 10 days to up to 200 days across HTA bodies, although 
the  majority  of  HTA  bodies  complete  their  review  within  2-3  months.46  In  general  the 
timeframes  for  the  assessment  of  medical  technologies  are  longer  than  those  for 
pharmaceuticals across Member States. 47 

41 For example, the assessment of a particular anti-cancer drug for the treatment of a specific type of cancer falls 
into the category of STAs. The assessment of several anti-cancer drugs available for the treatment of a specific 
type of cancer represents a MTA. 
42 Recent EMA annual reports give an idea of the number of new active substances and new therapeutic 
indications for existing products licensed per year. For example, in 2015, 39 new active substances and 54 
extensions reflecting a new therapeutic indication of an existing product were licensed. The number of medical 
devices receiving CE marking in 2015 is estimated to be around 4,500  (2015, MedTech Europe data) 
43 Study "Mapping of HTA national organisations, programmes and processes in EU Member States and 
Norway". 
44 Mechanism for scrutiny of conformity assessments of certain class III and class IIb devices (as defined in 
Regulation (EU) 2017/745, Article 55) and Mechanism for scrutiny of conformity assessments of class D 
devices (as defined in Regulation (EU) 2017/746, Article 50) 
45 Study "Mapping of HTA national organisations, programmes and processes in EU Member States and 
Norway". Note that HTA bodies in some MS produce assessments which do not inform pricing and 
reimbursement decisions (but are rather e.g. clinical guidelines). 
46 EUnetHTA WP7 draft report (unpublished) 
47 EUnetHTA WP7 draft report and study "Mapping of HTA national organisations, programmes and processes 
in EU Member States and Norway" 

17 

 
 
                                                            
8)  There  are  also  differences  across  EU  Member  States  in  the  starting  point  of  the  HTA 
process.  Figure  3  illustrates  HTA  timelines  for  pharmaceuticals  in  different  EU  Member 
States compared with the EU marketing authorisation timeline (EMA process)48.  

Figure  3.  Stylised  comparison  between  EUnetHTA  and  several  national  HTA  timelines 
(EFPIA/CRA Study) 

The figure shows that depending on the Member State, HTA reports can be published after or 
at the time of the last step of the marketing authorisation, while the HTA preparatory process 
can already start in parallel49. However, in reality, HTA often takes place later, because HTA 
submissions  by  industry  are  typically  not  initiated  simultaneously  or  at  the  earliest  possible 
dates in all countries. 

9)  Another  important  aspect  refers  to  stakeholders'  involvement  (patients,  healthcare 
providers, payers, etc.) throughout the HTA process which varies from country to country (for 
further details, see Mapping study on HTA processes). It should be noted that even within the 
Member  States  indicating  that  they  engage  stakeholders,  there  are  significant  differences  in 
the level of involvement, with stakeholders being consulted in one or more or all of the steps 
of their HTA processes. In general for pharmaceutical assessments stakeholder involvement is 
greatest  towards  the  end  of  the  assessment  process  when  the  assessment  is  reviewed  and 
advice/decision is made. For medical technologies there is greater stakeholder involvement in 
the earlier stages  (e.g. scoping, production  of the assessment)  than for pharmaceuticals, and 
less involvement in the advice and decision making steps. 

48 Note that the majority of new innovative medicines are authorised at EU level, via the centralised procedure. 
For further details on the market access pathway for pharmaceutical, see Annex V. 
49  EFPIA/Charles  River Associates.  2017.  Assessing the wider benefits of  the  EU’s proposal  on  strengthening 
cooperation on health technology assessment from the industry perspective. 

18 

 
 
 
                                                            
(b) Main differences in methodologies 

There  are  also  divergences  in  the  methodologies  used  by  different  HTA  bodies  when 
assessing the evidence produced by technology developers.50 For example, HTA bodies can 
take different methodological approaches when assessing the acceptability of particular types 
of studies and study design issues such as the comparator used, endpoints measured, the type 
of patients enrolled and the duration of the study.  
1)  The  choice  of  a  comparator51  is  decisive  in  any  health  technology  assessment.  A  recent 
study  mapping  HTA  methodologies  in  EU  Member  States  and  Norway52  showed  that  there 
are  commonalities  but  also  differences  in  the  criteria  used  for  choosing  a  comparator.  For 
example, when assessing pharmaceuticals:  

-  25  HTA  bodies  consider  both  whether  the  comparator  reflects  current  healthcare  practice 
and whether its use is supported by evidence on its efficacy and safety, while 7 HTA bodies 
consider only healthcare practice and 4 only the evidence base. 

 -  27  HTA  bodies  accept  different  technologies  (i.e.  also  medical  technologies  and  other 
technologies) as possible comparators in a pharmaceuticals assessment, whilst 10 HTA bodies 
compare pharmaceuticals only to other pharmaceuticals. 
-  6  HTA  bodies  do  not  accept  indirect  comparisons53,  whilst  a  large  majority  (i.e.  40  HTA 
bodies) accepts such comparisons. 

The  process  for  choosing  the  comparator  may  also  differ,  for  example  the  extent  to  which 
proposals  by  the  manufacturer  or  input  from  medical  societies/healthcare  professional 
organisations are considered. 

A similarly heterogeneous picture can be described for the selection of the comparator when 
assessing medical and other technologies. In this regard, 20 HTA bodies reported using as a 
comparator a  technology likely  to be replaced  by the assessed technology if proven inferior 
but  also  the  comparator  supported  by  evidence  of  its  efficacy  and  safety  profile.  Six  HTA 
bodies  reported  using  only  the  first  type  of  comparator,  whilst  4  other  HTA  bodies  stated 
using only the second one. Two HTA bodies informed that other criteria area also used when 
choosing the comparator for assessing these categories of health technologies 52.  

However, there is scope for cooperation; in the GÖG-LSE case studies, in 68% of the cases, 
the  comparator  included  was  the  same  across  HTA  bodies.  Moreover,  it  should  also  be 

50  References:  Mapping  study  methods,  GÖG-LSE  study,  Nicod  2017  (Eur  J  Health  Econ),  Nicod  2016 
(International  Journal  of  Technology  Assessment  in  Health  Care),  Akehurst  2016  (Value  in  Health),  Kleijnen 
2016 (Annals of Oncology) 
51  In  the  context  of  relative  effectiveness  assessment,  a  comparator  is  a  health  care  intervention  with  which  a 
pharmaceutical  is  compared  in  order  to  establish  if  it  has  an  added  therapeutic  benefit  (in  terms  of  clinical 
effectiveness and/or safety). Such a comparator could be another pharmaceutical, a medical device, a procedure 
or psychological approach, surgery or, if appropriate, providing advice, for example advice on diet or smoking, 
any combination of these, or “watchful waiting” (no intervention). 
52  Mapping  of  HTA  methodologies 
17010402/2016/736040. ISBN: 978-92-79-77074-6. 
53 The need for indirect comparisons arises when comparing treatments A and B, but the only available evidence 
comes from studies comparing A with C and B with C. By using a common comparator, in this case treatment C, 
it  is  possible  to  generate  an  indirect  comparison  with  treatments  A  and  B.  For  a  variety  of  reasons,  placebo-
controlled trials are commonly conducted in preference to head-to-head trials giving rise to the need for indirect 
comparisons (EUnetHTA guidelines for comparators and comparisons). 

the  EU  and  Norway.  2017.  DG  SANTE  Contract  nr. 

in 

19 

 
 
                                                            
highlighted that, as noted in the study mapping HTA methodologies, HTA is closely linked to 
the  broader  field  of  evidence-based  medicine  (EBM).  Many  developments  in  EBM  are 
already taking place at European level (e.g. development of evidence-based clinical guidelines 
by European-level medical/scientific societies) or international level54. HTA bodies will thus 
be  increasingly  able  to  draw  on  European-level  or  even  international  guidance  when 
considering the evidence-based standard of care. 

2) Furthermore, HTA bodies have different methodological approaches with regard to certain 
health outcomes and outcome measures. For example, a minority of HTA bodies does not 
accept  surrogate  endpoints55  (one  HTA  body),  composite  endpoints56  (nine  HTA  bodies)  or 
patient-reported  outcomes  (e.g.  questionnaires  on  health-related  quality  of  life)  (10  HTA 
bodies).44 

3)  Another  important  difference  in  methodology  relates  to  the  type  of  evidence/studies 
accepted  by  HTA  bodies.  Whilst  the  gold  standard  for  all  HTA  bodies  is  randomised 
controlled clinical trials (RCTs) and a small minority of HTA bodies (4) accept only RCTs, 
the  large  majority  of  HTA  bodies  accept  also  other  types  of  studies  (e.g.  observational 
studies) 44. 

Case studies conducted in the context of the GÖG-LSE Study confirmed the differences but 
also highlighted a tendency towards common methods in assessing products, which shows the 
scope for cooperation. In particular the primary clinical trials considered were  generally the 
same type. 57, 58, 59 

The  diversity  of  approaches  related  to  HTA  methodology  in  the  EU  Member  States  is 
confirmed  by  the  input  to  the  public  consultation  from  both  pharmaceutical  and  medical 
technologies industry and described in section 2. 

Regional cooperation on HTA 

While  this  Impact  assessment  is  being  prepared  some  groups  of  countries  have  started  to 
develop  stronger  regional  cooperation.  Typically,  this  type  of  cooperation  brings  together 
neighbouring Member States with similar socioeconomic situation, with the overall objective 
of  addressing  the  challenge  of  ensuring  access  to  innovative  technologies  through  possible 
joint  procurement.  The 
joint  economic  assessments, 

joint  price  negotiations  and 

54 Examples include the work of Cochrane (http://www.cochrane.org/) and the Guidelines International)  
55  In  clinical  trials,  a  surrogate  endpoint  (or  marker)  is  a  measure  of  effect  of  a  specific  treatment  that  may 
correlate with a real clinical endpoint but does not necessarily have a guaranteed relationship. For example, the 
serum  cholesterol  concentration  may  be  considered  a  surrogate  endpoint  when  assessing  pharmaceuticals 
aiming  to  prevent  complications  of  cardiovascular  disease.  However,  only  about  10%  of  those  with  serum 
cholesterol concentration above the reference range are going to have a stroke or heart attack. Therefore more 
relevant clinical endpoints could be the number of nonfatal myocardial infarction or stroke cases.  
56 Composite or combined endpoints are defined as the combination of component (singleton) endpoints, each 
of which has clinical significance in its own right. For example, a heart attack study may combine in  a single 
endpoint the number of patients who present with at least one clinical endpoint, either chest pain or myocardial 
infarction, or death. The alternative would be to conduct RCTs with distinct clinical endpoints such as death and 
nonfatal acute myocardial infarction (AMI). 
57 GÖG-LSE Study, Section 7.1.9 
58  Tarricone  R,  Torbica  A,  Drummond  M,  Schreyögg  J.  Assessment  of  medical  devices:  challenges  and 
solutions. Health Economics 2017; (S1):1-152.  
59 Tarricone R, Boscolo P.R, Armeni P. What type of clinical evidence is needed to assess health technologies? 
European Respiratory Review, 2016;25:259-265 

20 

 
 
                                                            
BENELUXA60, la Valletta cooperation61, the Nordic countries and the Visegrad cooperation 
are examples of such regional cooperation recently set up (the first one about 18 months ago, 
the most recent formalised its terms of references only a few weeks ago). The objectives of 
the  regional  cooperation  are  different  from  the  EU  cooperation  on  HTA,  which  excludes 
pricing and reimbursement. However, the tools and procedures developed by EUnetHTA are 
being  used  to  perform  joint  assessments.  To  date  only  BeNeLuxA  has  produced  few  Joint 
assessments and La Valletta is in the process of identifying the technologies to assess in the 
coming months.  

Regional initiatives are also referred to in section(s) 5.2.1 and 6.1.1. 

1.4.2.   HTA at EU level 

At EU level, the value of HTA and the fact that joint work could facilitate the implementation 
of HTA processes and reduce redundancies regarding the assessment of technologies has been 
recognised. Already in the 1980s, the Health Services Research Committee of the European 
Commission  began  to  assign  contracts  for  economic  appraisals  and  mechanisms  for  the 
regulation  of  expensive  health  technologies  in  different  countries.  Between  1993  and  2002 
three  projects  were  funded  by  the  European  Commission  to  support  collaboration  on  HTA 
between  Member  States.  In  2004,  the  European  Commission  and  Council  of  Minsters 
requested the establishment of a sustainable European network on HTA. This was initiated in 
2005  when  a  group  of  35  organisations  started  the  EUnetHTA  project,  which  explored 
possibilities and key challenges for an enhanced transnational collaboration for the following 
years. 

Since then, to support cooperation between HTA bodies, the European Commission has made 
substantial  investments.  Two  Joint  Actions  (EUnetHTA  JA)  have  been  carried  out  together 
with a number of projects62: (1) EUnetHTA 1 from 2010-2012 (budget EUR 6 million) and 
(2) EUnetHTA 2 from 2012-2015 (budget EUR 9.5 million). A third Joint Action (EUnetHTA 
3 - budget EUR 20 million) was launched in June 2016 and runs until 2020. Participation in 
the Joint Actions has been very high and the latest Joint Action has more than 80 members 
from  all  Member  States  and  a  number  of  observers  from  Member  States,  Norway  and 
Switzerland 63 

The  primary  objective  of  the  Joint  Actions  is  scientific  and  technical  cooperation,  more 
precisely  to  develop  common  methodologies,  pilote  and  produce  joint  REA  and  full  HTA 
reports,  and  to  develop common  ICT  tools.  The  Joint  Action  partners  also  piloted  so  called 
early dialogues  (i.e.  a mechanism  via which HTA bodies provide scientific advice to  health 
technology developers on the design of clinical trials  – typically phase III or pivotal trials  - 
with  a  view  to  encouraging  the  generation  of  evidence  that  better  meets  the  needs  of  HTA 

60 BeNeLuxA is an initiative started by Belgium and the Netherlands (2015), later joined by Luxembourg  and 
Austria  (2016).  This  group  of  countries  intends  to  collaborate  more  closely  across  a  range  of  areas:  health 
technology  assessment;  horizon  scanning;  exchange  of  information  on  pharmaceutical  markets;  prices  and 
disease-specific cross-border registries; and pricing and reimbursement, including joint negotiation. 
61 Round table meeting for European Health Ministers and Heads of pharmaceutical companies (Malta, 9 May 
2017) 
62  Public  Health  Programme:  EUR-ASSES  1994-1997;  ECHTA/ECHAI  1999-2001;  EUnetHTA  2006-2008. 
Research Programme: AdHopHTA; INTEGRATE-HTA; MedtechHTA; Advance HTA. 
63 Switzerland is an affiliated partner. 

21 

 
 
                                                            
agencies). All these activities in which HTA bodies prepare shared products/agreed outcomes 
are referred to as “joint output”64.  

More precisely, EUnetHTA 1 and 2: 

- developed the HTA Core Model as a methodological framework for assessments, databases 
for exchanging information (e.g. Planned and On-going Projects (POP) database allowing for 
sharing of information on planned and on-going assessments in the Member States) and 

- delivered a number of outputs, including 13 clinical assessments/REAs (7 on pharmaceutical 
and 6 on non-pharmaceutical technologies such as medical devices), 5 Full HTA reports, 11 
Early  dialogues,  14  methodological  guidelines,  as  well  as  common  evidence  submission 
templates for pharmaceuticals and medical devices.  

The ongoing Joint Action EUnetHTA 3 has planned to scale up the joint outputs, and aims to 
deliver by the end of 2020 approximately 80 joint assessments and up to 35 early dialogues. 
However, as the Joint Action 3 has so far only produced a small number of assessments, it is 
currently uncertain if project will be able to deliver the target number of 80 joint assessments. 

In  addition,  the  importance  of  HTA  cooperation  at  EU  level  is  emphasised  in  Directive 
2011/24/EU on the application of patients’ rights in cross-border healthcare, which states that 
'cooperation in the evaluation of new health technologies can support Member States through 
economies  of  scale  and  avoid  duplication  of  effort,  and  provide  a  better  evidence  base  for 
optimal  use  of  new  technologies  to  ensure  safe,  high-quality  and  efficient  healthcare'.65  To 
further strengthen the technical cooperation between HTA bodies, the Directive provides for 
the establishment of a network connecting national authorities or bodies responsible for HTA 
(HTA Network). Such a Network was set up in 2013 to provide strategic guidance and policy 
orientation  for  the  scientific  and  technical  cooperation.  66,67  While  participation  in  the 
Network  is  voluntary,  all  Member  States  have  applied  for  membership  and  participate.  The 
HTA  Network  develops  policy  papers68  and  discusses  areas  of  potential  collaboration69, 
which are then implemented by the Joint Action, in accordance with its work plan. 

The  current  cooperation  model  described  above,  in  particular  the  Joint  Actions  as  an 
instrument  to  implement  cooperation  on  HTA  at  scientific  and  technical  level,  is  meant  to 
develop/share/refine/test  tools,  methods  and  approaches  for  specific  issues  or  activities  and 
involve  a  degree  of  capacity-building70.  In  this  respect  the  EUnetHTA  Joint  Actions  have 
been successful, as outlined by the Mid-term evaluation of the Public Health programme71.  

Their  usefulness  has  mainly  been  associated  with  an  increased  level  of  trust  between  HTA 
bodies  and  stakeholders  involved;  increased  knowledge  of  working  procedures  and 

64 Or joint work in some EUnetHTA and HTA Network documents 
65 Cross-border Healthcare Directive 2011/24/EU, RECITAL 58 
66 Cross-border Healthcare Directive 2011/24/EU, Article 15. 
67 Commission Implementing Decision of 26 June 2013 providing the rules for the establishment, management 
and  transparent  functioning  of  the  Network of national  authorities  or  bodies  responsible  for health  technology 
assessment 
68 HTA Network. Strategy for EU Cooperation on Health Technology Assessment. 2014  
69  HTA  Network  Reflection  Paper  on  “Synergies  Between  Regulatory  and  HTA  Issues  on  Pharmaceuticals. 
2016. 
70 Extract from the evaluation report on the second Public health programme 
71 It should be noted that Joint Action EUnetHTA have not be subject to a specific evaluation in the context of 
the Report, but only used as an example of growing interest in a specific policy area. 

22 

 
 
                                                            
methodologies  in  Member  States  and  capacity  building  and  sharing  of  best  practices  (see 
report on the public consultation).   

On the other hand, the current  cooperation model has demonstrated important  shortcomings 
including:  

  Delays. The current Joint Action has started with significant delays, due to a complex 
and long negotiation process between the high number of beneficiaries and the funding 
Agency. The process from the evaluation to the signature of the contract lasted nearly 
one  year.  The  late  start  was  further  aggravated  by  implementation  challenges  due  to 
non-delivery by certain beneficiaries and/or misunderstandings between beneficiaries 
of the task(s) at stake. One important example is the delay in securing an efficient and 
reliable IT infrastructure which would enable the cooperation to function. 

  Changes  in  Human  Resources.  During  and  after  the  negotiation  process  some 
beneficiaries  which  had  agreed  to  take  up  important  responsibilities  and  had  been 
allocated corresponding resources, have undergone reorganisations leading to changes 
in priorities. This has caused dramatic decrease in the expertise and human resources 
which  were  expected  to  be  available  from  that  beneficiary.  While  funding  was 
available  to  recruit  the  necessary  staff,  changes  in  priorities  of  the  beneficiary 
organisation prevented the recruitment. To date, after more than 12 months from the 
start of the Joint Action, organisations leading key workplaces have not yet a full team 
with  the relevant  expertise  in  place,  with  important  consequences  on the  progress  of 
the activities.   

  High  number  of  beneficiaries  and  heterogeneous  profile/roles  in  national  HTA 
activities. In the Joint Action model the Member States appoint organisations  which 
have  an  interest  in  the  subject  of  the  Action.  Due  to  the  high  interest  which 
cooperation on HTA has generated in the EU the EUnetHTA Joint Action included at 
its start 79 beneficiaries and at the time this report is written the number increased to 
82.  The  large  number  also  implies  very  heterogeneous  profiles  between  the 
beneficiaries, ranging from national HTA bodies with a statutory function in informing 
decision makers for pricing and reimbursement decisions, to HTA bodies which has a 
remit  oriented  toward  development  of  clinical  guidelines,  to  regional  HTA  agencies, 
and also academic institutions with an interest in HTA but with no (official) role in the 
national  HTA/decision-making  process.  The  high  number  of  beneficiaries  and  their 
heterogeneous  profile  and  role  in  the  decision-making  process  in  the  respective 
countries  increases  significantly  overheads  to  the  coordinating  agency,  leading  to 
inefficiencies  in  the  use  of  resources  and  creates  challenges  in  identifying  relevant 
tasks for the appropriate profiles.    

  Uncertain  delivery.  To  produce  a  high  quality  and  useful  joint  assessments  the 
involvement of technology developers has proven to be extremely important.  Under 
the current cooperation model such involvement is often seen as an add-on activity for 
technology  developers,  which  have  to  prepare  submissions  for  national  HTA 
assessments and for joint assessments carried out under the Joint Action. This situation 
is likely to persist as long as the uptake by national HTA bodies of Joint Assessments 
is not happening to the desired extent. Both in Joint Action 2 and Joint Action 3, the 
Commission  in  cooperation  with  the  Joint  Action  secretariat  and  relevant  trade 
associations  had  to  organise  activities  to  trigger  interest  and  commitment  from 
technology  developers  to  submit  technologies  for  Joint  Assessments.  While  the 
activities were successful, the engagement is not certain as it will depend on the ability 

23 

 
 
of  the  Joint  Action  to  secure  uptake  of  joint  assessments  in  national  processes.  This 
situation brings an additional element of uncertainty to the ability of the Joint Action 
to meet the planned target(s) within the necessary timeline.  

  Inconsistency of quality and timely delivery. The examples outlined above result in 
very  uneven  progress  in  the  activities  of  the  Joint  Action,  which  in  turn  affects  the 
ability to deliver both in terms of time and quality of the output. The Joint Action is 
highly dependent on the organisation responsible for the delivery of the task and even 
on the technology developer whose technology is being assessed.  While this issue can 
be handled in an action which aims at developing and testing a proof of concept, it is 
not acceptable when the objective is to timely deliver high quality output to be used in 
national decision-making processes.  

1.5.  Political context 

In recent years, many key players have called for reinforced EU cooperation in the area of 
HTA.  As regards Member States, a clear orientation was contained in the “Strategy for EU 
Cooperation  on  HTA”72,  which  was  adopted  by  the  Member  States  representatives  in  the 
HTA  Network  in  October  2014.  In  this  document,  the  HTA  Network  called  upon  the 
Commission to explore how to secure support for the joint work in the long-term. Moreover, 
the  Council,  in  its  conclusions  on  “Innovation  for  the  benefits  of  patients”73  adopted  in 
December  2014,  acknowledged  the  key  role  of  HTA  and  called  on  the  Commission  to 
continue  to  support  sustainable  cooperation.  Furthermore,  in  the  Council  conclusions  on 
“Personalised  medicines  for  patients”  of  December  201574,  the  Member  States  and  the 
Commission  were  invited  to  reinforce  HTA  methodologies  applicable  to  personalised 
medicine.  The  Council  conclusions  on  “Strengthening  the  balance  in  the  pharmaceutical 
systems” in June 201675 confirmed again that Member States see a clear added value of EU 
HTA cooperation. The joint report of DG ECFIN and the Economic Policy Committee calls 
for further developing European cooperation on HTA.76 

The  European  Parliament  has  also  asked  for  a  reinforcement  of  HTA  cooperation  at  EU 
level.  In  its  joint  motion  for  a  resolution  on  the  Commission  Work  Programme  2016,  the 
Parliament  called  for  "a  step  forward  towards  a  common  European  Health  Technology 
Assessment"77.  Moreover,  in  its  resolution  of  2  March  2017  on  EU  options  for  improving 
access  to  medicines78,  the  Parliament  calls  on  the  Commission  to  "propose  legislation  on  a 
European  system  for  health  technology  assessment  as  soon  as  possible,  to  harmonise 

72 HTA Network. Strategy for EU Cooperation on Health Technology Assessment. 2014 
73 Council conclusions on innovation for the benefit of patients (2014/C 438/06) . 
74 Council conclusions on Personalised medicine for patients (2015/C 421/03),  
75 Council conclusions on strengthening the balance in the pharmaceutical systems in the EU and its Member 
States (2016/C 269/06)  
76 DG ECFIN- EPC 2016. Joint Report on Health Care and Long-Term Care Systems & Fiscal Sustainability, 
Institutional Paper 37. 7 October 2016 
77 European Parliament resolution on the Commission Work Programme for 2016 (2015/2729(RSP) 
78  European  Parliament  resolution  of  2  March  2017  on  EU  options  for  improving  access  to  medicines 
(2016/2057(INI) 

24 

 
 
                                                            
transparent  HTA  criteria  in  order  to  assess  the  added  therapeutic  value  of  medicines".  The 
Parliament has also commissioned a study on HTA, highlighting its interest in the subject.79  

The  Commission  has  on  several  occasions  referred  to  HTA,  including  as  a  key  part  of 
supporting  other  important  Commission/EU  initiatives.  For  example,  the  Commission 
Communication on effective, resilient and accessible health systems80 suggested HTA as one 
way  to  build  resilience.  In  a  recent  Staff  Working  Document,  the  lack  of  “binding 
mechanisms for mutual recognition of joint assessments” was identified as one of the major 
shortcomings of the current HTA system.81 The Staff Working Document “Better Regulation 
for  innovation  driven  investment  at  EU  level”  pointed  out  that  the  fragmentation  of  HTA 
systems  in  the  EU  is  currently  “very  high”  and  that  the rise  of  personalised  medicine82  will 
accelerate  the  concerns  of  fragmentation.83  The  recent  Commission  Communication 
“Upgrading  the  Single  Market:  more  opportunities  for  people  and  business”  contained  a 
commitment  that  the  Commission  will  introduce  an  initiative  on  HTA  with  a  view  to 
improving the functioning of the Single Market of health technologies, in particular in order 
to avoid duplication of efforts for Member States and industry.84  

Stakeholder views 

A  thorough  stakeholder  consultation  has  been  carried  out  in  the  context  of  this  Impact 
Assessment in order to collect stakeholders’ views on EU cooperation on HTA beyond 2020 
(see synopsis report, Annex II).  

The  usefulness  of  EU  cooperation  on  HTA  was  underlined  by  the  input  provided  by 
stakeholders in response to the public consultation. Most of the contributors (69%) consider 
EU  cooperation  on  HTA  useful  or  to  some  extent  useful,  with  most  benefit  seen  by  public 
administrations, payers and academia (100%) and less benefits typically seen by the medical 
technologies industry (for more information, see Annex II). The most cited benefits of the EU 
cooperation on HTA were the opportunity to share knowledge and best practices, contribute 
to  HTA  capacity  building  in  the  Member  States,  contribute  to  building  trust  between 
participating organisations and increase awareness (Figure 4). 

79  Van  Wilder,  P.  (Vrije  Universiteit  Brussel  and  SMART&BI),  Mabilia  V.  (Milieu  Ltd.),  Kuipers  CavacoY. 
(Milieu Ltd.) and McGuinn J.(Milieu Ltd.), "Towards a Harmonised EU Assessment of the Added Therapeutic 
Value of Medicines", Report commissioned by the Committee of Environment, Public Health and Food Safety at 
the European Parliament, 2015. 
80 Commission Communication "On effective, accessible and resilient health systems", COM (2014) 215 final 
81  Commission  Staff  Working  Document,  "A  Single  Market  Strategy  for  Europe  –  Analysis  and  Evidence", 
SWD(2015) 202 final 
82 Personalised medicine refers to a medical model using characterisation of individuals' phenotypes and 
genotypes (e.g. molecular profiling, medical imaging, lifestyle data) for tailoring the right therapeutic strategy 
for the right person at the right time, and/or to determine the predisposition to disease and/or to deliver timely 
and targeted prevention (see Council conclusions on personalised medicine for patients, 2015/C 421/03). 
83 Commission Staff Working Document "Better Regulation for innovation driven investment at EU level" SWD 
(2015) 298 final. 
84  Commission  Communication  “Upgrading  the  Single  Market:  more  opportunities  for  people  and  business” 
(COM (2015) 550 final. 

25 

 
 
 
                                                            
Figure 4. Benefits of EU cooperation on HTA as reported by the contributors to the public 
consultation (Total number of replies = 186) 

In general, Member States’ public authorities are in favour of continuing EU cooperation on 
HTA  beyond  2020.  Some  Member  States  have  indicated  a  preference  for  voluntary 
cooperation,  while  others  support  a  system  with  mandatory  elements.  Most  contributors 
highlighted that in case of a mandatory framework, uptake of joint work should be limited to 
clinical matters, whereas assessment of non-clinical domains (e.g. economic, ethical) should 
be carried out individually or jointly by interested Member States on a voluntary basis. The 
idea of a phase-in approach was also raised by some contributors. 

1.6.  International outlook 

There is a growing world-wide recognition of the significant benefits of HTA.85 The World 
Health Organization (WHO) suggested that a global cooperation of HTA bodies would seem 
useful, in particular on the clinical domains86 and the World Bank supports the development 
of national HTA programmes around the globe.   A recent OECD report87 assesses the state of 
play and gives recommendations for the use of HTA. The International Monetary Fund also 
promotes the development of national HTA systems.88 A number of countries outside the EU 
have developed well established HTA systems. The Canadian example of centralisation or the 
regular review of the HTA system in Australia, are interesting examples (see Annex VII). It is 
also interesting to note the setting up of networks between HTA bodies in other parts of the 
world, following the model of EUnetHTA89. 

85  Ciani  O,  Wilcher  B,  Blankart  CR,  et  al.  Health  Technology  Assessment  of  Medical  Devices:  A  Survey  of 
Non-European  Union  Agencies.  International  Journal  of  Technology  Assessment 
in  Health  Care. 
2015;31(3):154-165.  
86 WHO resolution on Health intervention and technology assessment in support of universal health coverage. 
87  OECD  (2017),  New  Health  Technologies:  Managing  Access,  Value  and  Sustainability,  OECD  Publishing, 
Paris. 
88 Examples include the Memoranda of Understanding for Greece, Portugal, Cyprus or Romania. 
89 Regional Network for HTA for the Americas RedETSA.  

26 

 
 
 
                                                            
2.  Problem definition 

While  HTA  is  considered  a  valuable  tool  for  ensuring  sustainability  of  health  systems  and 
stimulating  innovation  and  cooperation  at  EU  level,  evidence  shows  that  a  series  of 
shortcomings  affect  the  exploitation  of  the  benefits  for  Member  States  and  economic 
operators,  with  subsequent  negative  consequences  also  for  EU  patients  and  healthcare 
professionals.  

Figure 5. Main problems, their drivers and consequences 

Problem 1. Impeded and distorted market access  

The different national processes and methodologies of national and regional HTA bodies 
illustrated  in  section  1.4.1  (and  Annex  V)  mean  that  economic  operators  who  want  to 
introduce  a  health  technology  in  multiple  Member  States  are  confronted  with  various  data 
requests. This in turn contributes to an impeded and distorted market access, leading to lack of 
business  predictability,  higher  costs,  and  in  the  long  run  negative  effects  on  innovation. 
Differences in national processes and methodologies also lead to differences in how evidence 
is  considered  in  assessments  and  to  potentially  different  HTA  conclusions,  which  can 
contribute to delays and inequalities in patient access to technologies90.  

The differences in HTA processes and their effects were underlined by various stakeholders in 
the  public  consultation.  The  most  significant  impact  was  reported  by  representatives  of  the 
pharmaceutical industry who pointed out that this diversity constitutes a hurdle for companies, 
as  they  have  to  adapt  to  multiple  and  various  national  requirements  (e.g.  regarding  the 
starting moment of the assessment, data requirements, length of the procedures, scope of HTA 
and  type  of  assessment  carried  out).  With  regard  to  differences  in  HTA  methodologies, 

90  Valzania  C,  Torbica  A,  Tarricone  R,  Leyva  F,  Boriani  G.  Implant  rates  of  cardiac  implantable  electrical 
devices in Europe: a systematic literature review. Health Policy 2016;120(1):1-15 

27 

 
 
 
 
                                                            
representatives  of  the  pharmaceutical  industry  emphasised  (among  others)  differences  with 
respect  to  the  acceptability  of  particular  comparators,  endpoints  and  data  other  than 
randomised  clinical  trials,  which  contribute  to  the  lack  of  business  predictability  due  to 
different outcomes of national HTA clinical reports91.  

Duplication of assessments also increases costs for industry, which needs to prepare dossiers 
for  multiple  national  systems  with  potentially  different  data  requirements.  Requirements  for 
additional  evidence  are  a  key  cost  component,  with  potential  delays/risks  in  market  access. 
Costs  related  to  additional  evidence  requirements  are  particularly  high  if  they  necessitate 
carrying  out  new  trials.92  The  GÖG-LSE  study  provides  an  indication  of  current  costs  for 
industry related to the meeting current national HTA requirements (see text box below). 

Current costs for industry related to the preparation of national HTA dossiers 
On the basis of the figures reported in the survey carried out by the GÖG-LSE study93, the costs for 
industry related to national pharmaceutical HTA processes are summarised in the table below. 

The  survey  carried  out  by  the  GÖG-LSE  Study  indicates  that  earmarking  the  costs  for  one  single 
assessment  is  difficult  as  manufacturers  prepare  a  global  value  dossier  for  each  product,  which  is 
usually the main source of input for their HTA departments/teams. Moreover, certain costs can pertain 
also  to  the  regulatory  (e.g.  related  to  evidence)  or  pricing  and  reimbursement  processes  (e.g.  in-
country staff costs).  

HTA  costs  are  particularly  relevant  for  SMEs  as  they  typically  do  not  have  structures  or  resources 
dedicated to HTA, or the in-country capacities needed to adapt to multiple national requirements and 
formats. In the open public consultation carried out by the Commission, a higher proportion of SMEs 
indicated that differences among EU Member States regarding HTA procedures and/or methodologies 
contributed to high costs/expenses for their organisation. In the dedicated SME consultation, 75% of 
pharmaceutical  and  71%  of  medical  technologies  companies  indicated  that  differences  among  EU 

91 Nicod E, Why do health technology assessment coverage recommendations for the same drugs differ across 
settings?  Applying  a  mixed  methods  framework  to  systematically  compare  orphan  drug  decisions  in  four 
European countries. Eur J Health Econ. 2017 Jul;18(6):715-730.  
92 Martin L, Hutchens M, Radnoy A, How much do clinical trials cost?, Nature Reviews Drug Discovery, 16, 
381–382 (2017). Note that this study estimated the average cost of a phase III clinical trial at USD 21.4 million. 
93 The GÖG-LSE survey asked respondents to (1) indicate their current HTA related costs and (2) express their 
expectations regarding the impacts in a number of areas of the 5 policy options described in the Inception Impact 
Assessment on strengthening EU cooperation on HTA. These areas cover economic and social aspects and are 
defined by Better Regulation Guidelines of the European Commission; for each area, the study defined specific 
indicators. 120 stakeholders of the medical technologies industry, 20 stakeholders of the pharmaceutical industry 
and 37 stakeholders representing public administration and other organisations (total 177) participated.  

28 

 
 
 
 
                                                            
to  high 
Member  States  regarding  HTA  procedures  and/or  methodologies  may  contribute 
costs/expenses  for  their  organisation.  The  same  figure  was  44%  for  non-SMEs  in  the  public 
consultation, confirming the expectation that differences are more difficult to handle by SMEs.  

With regard to additional data generation, it should be noted that it is mainly the requirements of the 
largest  markets  that  companies  take  into  account  when  designing  clinical  trials  or  generating 
additional evidence.  

In their input to the online public consultation it was highlighted that while most of the larger 
pharmaceutical companies resort to national affiliates to engage with national authorities and 
ensure  adequate  preparation  of  the  documentation  requested  by  HTA  bodies,  smaller 
companies  with  limited  resources  may  face  difficulties  in  putting  in  place  such  a 
mechanism, which could create a discriminatory environment and discrepancies in the speed 
of market access. This situation is confirmed by a recent report from EuropaBio showing that 
SMEs have limited experience in working with HTA bodies and may not have staff dedicated 
to  HTA  work.  According  to  EuropaBio,  this  has  led  to  some  products  getting  marketing 
authorisation  but  not  being  recommended  by  the  HTA  bodies  because  the  data  was  not 
sufficient to establish the required added clinical and/or economic value.94 

The variety of HTA processes and methodologies is also reflected in the  HTA spending by 
industry. For example, the HTA spending by the pharmaceutical sector ranges from EUR 73 
000 and EUR 1 700 000 per HTA submission95 (depending also on the type of assessment). 
Extra evidence generation is responsible for additional expenditure (between EUR 50 000 and 
EUR  20  000  000/submission),  with  85%  of  companies  reporting  such  costs.96  Furthermore, 
the  pharmaceutical  sector  actively  engages  in  early  dialogues  with  HTA  bodies  (69%  of 
responses)  with  an  average  cost  of  EUR  55  750  per  case,  but  which  have  the  potential  to 
alleviate costs in the next phases of development/approval/access to market. With regards to 
the medical technology industry, HTA submission dossiers range from EUR 1 000 to EUR 3 
400 00097. Additional evidence generation in the context of a HTA submission dossier had a 
range of EUR 17 000 - EUR 12 800 000, with 37% of companies reporting such costs98. Early 
dialogues  are  not  a  routine  procedure  in  the  sector.99  While  the  costs  related  to  HTA 
submissions may not always be significant (seen in the context of overall industry spending) it 
is  important  to  note  that  the  parallel  submissions  and  assessments  by  national  HTA  bodies, 
entail  a  significant  risk  of  divergent  outcomes  in  different  Member  States,  which  has  a 
negative  impact  on  business  predictability.  Insufficiently  predictable,  fragmented  and 

94  Deerfield  Institute  -  EuropaBio  Report  on  regulatory  and  HTA  scientific  advice  for  small  and  medium 
enterprises, 2015. 
95 These values represent a sum of average costs per HTA submission per company as reported to the GÖG-LSE 
survey. Companies reported staff costs, consultant costs, in-house model costs, external model costs, and other 
costs. Section 7.1.12. 
96  Other  calculations  confirm  this  range:  the  Ecorys  report  on  European  Cooperation  on  Health  Technology 
Assessment:  Economic  and  governance  analysis  of  the  establishment  of  a  permanent  secretariat,  (2013) 
estimates the total costs for industry at 200.000 €. These cost figures do not capture the full costs, as they only 
refer to the human resources needed to prepare the submission dossiers (for industry)  
97  These  values  represent  a  sum  of  average  costs  per  HTA  submission  per  company  (comprising  staff  costs, 
consultant costs, in-house model cost, external model costs, and other costs) through the survey carried out by 
the GÖG-LSE Study.  
98  It  should  be  noted  that  obligations  for  additional  evidence  generation  for  medical  technology  is  much  less 
frequent than for pharmaceuticals, due to the different regulatory process.  
99 GÖG-LSE Study Section 7.1.12 

29 

 
 
                                                            
delayed  market  access  is  therefore  the  most  important  shortcoming  resulting  from  the  HTA 
fragmentation across the EU (see impact estimate in chapter 6.4.1.1).  

As  confirmed  in  interviews  and  focus  groups  with  pharmaceutical  industry  representatives, 
poor business predictability and high fragmentation of HTA systems across Europe constitute 
barriers  to  investment  by  industry  in  development  programmes  for  innovative 
technologies.100  

Finally,  as  described  in  section  1.4.1  (and  GÖG-LSE  Study  Section  7.1.7  and  7.1.14)  the  high 
variability in the timing (in terms of both the starting point and the duration) of assessments 
contributes  to  differences  in  the  availability  of  health  technologies  for  EU  patients.101 
Moreover, divergences in the conclusions of HTA reports on added value, which are due to 
different  approaches  taken  by  HTA  bodies  as  described  above,  contribute  to  differences  in 
availability of medicines to EU patients102. As illustrated by Table 1 for a sample of cancer 
drugs, there are common trends but also discrepancies in the conclusions reached by different 
HTA bodies on the same product. The observed divergences between HTA bodies are due to 
differences in the clinical  part  of HTA (REA) and/or  the economic part  of  HTA. In  several 
examples,  divergences  in  HTA  conclusions  were  influenced  by  how  different  HTA  bodies 
assessed  effects  seen  for  particular  clinical  endpoints  (when  assessing  the  new  drug  against 
the  same  comparator).  The  authors  note  the  potential  for  European  consensus-building  on 
these  clinical  aspects,  which  may  be  informed  by  ongoing  scientific  initiatives  of 
European/international  medical  societies.  Other  case  studies  for  other  pharmaceutical 
products and therapeutic areas show similar results, confirming both current HTA divergences 
but also the potential for improved scientific consensus-building on the clinical part of HTA 
103,104. 

100 GÖG-LSE Study, Section 7.1.13 
101 GÖG-LSE Study, Mapping HTA procedures, Akehurst 2016 (Value in Health)  
102  References:  Impacts  study,  Nicod  2017  (Eur  J  Health  Econ),  Nicod  2016  (International  Journal  of 
Technology Assessment in Health Care), Akehurst 2016 (Value in Health), Kleijnen 2016 (Annals of Oncology) 
103  Impacts  study,  Nicod  2017  (Eur  J  Health  Econ),  Nicod  2016  (International  Journal  of  Technology 
Assessment in Health Care), Akehurst 2016 (Value in Health) 
104 Spinner DS, Birt J, Walter JW, Bowman L, Mauskopf J, Drummond MF, Copley-Merriman C, Do different 
clinical  evidence  bases  lead  to  discordant  health-technology  assessment  decisions?  An  in-depth  case  series 
across three jurisdictions, Clinicoecon Outcomes Res. 2013; 5: 69–85. 

30 

 
 
                                                            
Table 1. Conclusions of HTA reports across a sample of cancer drugs105  

For  medical  technologies,  the  case  studies  conducted  by  the  GÖG-LSE  study  indicate  an 
overall  lower  number  of  assessments  produced  by  HTA  bodies  across  Europe  than  for 
pharmaceuticals  (see  Table  2).  However,  the  case  studies  also  showed  several  examples 
where different HTA bodies reached divergent conclusions on the same medical technology 
due to differences in the clinical and/or economic parts of their assessments (see GÖG-LSE 
study  for  further  details106).  Increased  HTA  activities  in  Member  States  on  medical 
technologies  (see  problem  2)  have  the  potential  to  increase  further  the  number  of  parallel 
assessments  on  the  same  technologies.  Considering  the  different  national  processes  and 
methodologies,  this  is  likely  to  lead  to  increased  divergence  across  the  EU  and  thus 
fragmentation of the market. 

The considerations above are confirmed by the overall input received from the online public 
consultation. According to the respondents, the main consequences of the differences in HTA 
processes and methodologies across the EU are the diverging outcomes of HTA reports which 
may  affect  patients'  access  to  new  technologies  (e.g.  delays,  restricted  access)  (81%  of 
contributions), followed by duplication of work for both HTA bodies and industry (54%), 
decrease  in  business  predictability  (53%),  higher  costs  for  the  actors  (38%)  and  negative 
effect  on  innovation  (37%)(Figure  6).  Further  details  on  the  public  consultation,  including 
results by stakeholder group, are presented in Annex II.  

105  Figure  courtesy  of  W.  Goettsch  (Presentation  to  EHFG  2016).  Based  on  data  published  in  "Relative 
effectiveness  assessments  of  oncology  medicines  for  pricing  and  reimbursement  decisions  in  European 
countries.", Kleijnen S, Lipska I,,Leonardo Alves T, Meijboom K, Elsada A, Vervölgyi V, D’Andon A, Timoney 
A, Leufkens HG, de Boer A, Goettsch WG. Ann Oncol (2016) 27 (9): 1768-1775 (see Supplementary Table 4 to 
the publication for further details on comparators and clinical endpoints considered). 
106 GÖG-LSE Study (Table 26 and Annexes to the study) 

31 

 
 
 
                                                            
Figure 6. Consequences of differences in HTA process and methodologies across the EU as 
identified by public administrations, organisations and associations responding to the online 
public consultation (Total number of replies = 186) 

Problem 2. Duplication of work for national HTA bodies  

The  duplication  of  work  refers  to  assessments  of  the  same  technology  being  conducted  in 
parallel or within a similar time frame by HTA bodies in different Member States. The text 
box  below  illustrates  current  costs  for  HTA  bodies  related  to  the  production  of  REAs  at 
national level, as reported by the GÖG-LSE study. 

Current costs for HTA bodies related to the production of REAs at national level  
According  to  the  figures  reported  in  the  survey  carried  out  by  the  GÖG-LSE  study,  costs  for  HTA 
bodies range from an average of EUR 35 000 for a REA produced mainly by an HTA body and EUR 
20 000 per REA produced by industry and reviewed by an HTA body to EUR 95 000 for a full HTA 
produced  by  an  HTA  body  and  EUR  40  000  for  the  cases  in  which  the  full  HTA  is  produced  by 
industry  and  reviewed  by  HTA  body.  Further  research  and  benchmarking  of  these  figures  with 
additional  HTA  bodies  not  included  in  the  survey  indicate  that  these  costs  may  be  underestimated. 
This means that the duplications expected to continue under this scenario would have an impact on the 
spending/budget of Member States and public administrations 

In  addition,  the  current  low  uptake  of  joint  REA  undertaken  by  the  EUnetHTA  results  in 
duplication  and  incurs  additional  work  and  costs.  The  duplication  may  be  associated  with 
different  outcomes/conclusions  (depending  on 
type  of  assessment  and  applied 
methodology),  which  negatively  affects  business  predictability  and  contributes  to  delays 
and inequalities in patient access.  
The  GÖG-LSE  Study107  found  that  in  a  sample  of  20  pharmaceuticals,  8-15  HTA  reports 
were conducted by different Member States for each individual product.108 This indicates that 

the 

107 Section 7.1.2 
108 The study aimed to build a representative sample; however in order to have sufficient data for analysis, one of 
the criteria for inclusion for a pharmaceutical was to have been assessed by at least 5 MS between 2012-2016. 
Therefore  the  result  cannot  be  generalised  to  conclude  that  all  pharmaceuticals  undergo  on  average  X  HTA 

32 

 
 
 
                                                            
 
there is a group of pharmaceuticals (typically innovative, centrally authorised, approximately 
40 new molecules/year) where there is a significant duplication of work for HTA bodies and 
industry.  At  the  same  time,  the  report  concludes  that  not  all  Member  States  have  adequate 
capacities to produce HTAs on all relevant products. 

Although  a  lower  number  of  Member  States  assess  medical  and  other  technologies  than 
pharmaceuticals109  (Table  2)  there  is  still  considerable  duplication  of  efforts  also  in  these 
sectors;  albeit  to  a  more  limited  extent  than  in  the  case  of  pharmaceuticals  (3-8  reports  per 
technology in the sample).110 111 112   

processes,  but  it  can  be  concluded  that  there  is  a  group  of  pharmaceuticals  (typically  innovative,  centrally 
authorised) where there is an important duplication of HTA. 
109  Study  "Mapping  of  HTA  national  organisations,  programmes  and  processes  in  EU  Member  States  and 
Norway":  23  MS  indicated  assessing  pharmaceuticals,  20  MS  for  medical  technologies  and  17  MS  for  other 
technologies.  Moreover  while  all  23  MS  conducting  pharmaceuticals  use  it  for  pricing  and  reimbursement 
decision, only 19 do so for medical devices and 9 for other technologies. 
110 Section 7.1.2 Just as in the case of pharmaceuticals, having sufficient number of HTA reports was a criterion 
for selection. Therefore it should not be concluded that this figure is representative for all medical technologies 
but that there is a group of medical technologies where important duplications take place. 
111 Tarricone R, Callea G, Ogorevc M, Prevolnik Rupel V. Improving the methods for the economic evaluation 
of medical devices. Health Economics 2017;26(Suppl S1):70-92 
112  Rummel,  P,  Hawlik,  K,  Wild,  C.  Health  Technology  Assessments  on  Medical  Devices  in  Europe.  Final 
Report. LBI-HTA Rapid Assessment Nr. 12; 2016. Vienna: Ludwig Boltzmann Institute for Health Technology 
Assessment. 

33 

 
 
                                                                                                                                                                                          
Table 2. Number of assessments carried out by HTA bodies in the Member States reflecting 
duplication of assessments - case studies  

In  the  public  consultation,  MedTech  Europe  argued  that  the  duplication  of  efforts  is  not  so 
prominent  for  medical  technologies.  However,  recent  publications  have  shown  increasing 
duplication  of  assessments  also  for  medical  technologies.113,  114  This  is  in  line  with  current 
trends  for  increased  HTA  activities  on  medical  technologies  in  Member  States  (see  box 
below). 

113 Tarricone R, Callea G, Ogorevc M, Prevolnik Rupel V. Improving the methods for the economic evaluation 
of medical devices. Health Economics 2017;26(Suppl S1):70-92. 
114  Hawlik  K,  Rummel  P,  Wild  C,  Analysis  of  duplication  and  timing  of  health  technology  assessments  of 
medical  devices  in  Europe,  International  Journal  of  Technology  Assessment  in  Health  Care  (accepted  for 
publication in October 2017) 

34 

Pharmaceuticals IncludedTOTAL ASSESSEDMedical Devices IncludedTOTAL ASSESSEDOther Technologies IncludedTOTAL ASSESSEDAbiraterone13Endovascular stents6HPV Vaccination8Aclidinium Bromidium12Home haemodialysis device5Colorectal Cancer Screening10Alemtuzumab12Transcatheter implantable devices6Pneumococcal Vaccination4Apremilast10Balloon Eustachian Tuboplasty4Rotavirus Vaccination3Ataluren10Oscillometric blood pressure monitor4Cervical cancer screening6Canagliflozin14High intensity focused ultrasound (HIFU)8average6,2Dapagliflozin13Self-monitoring coagulometers7Defibrotide8Positron emission tomography (PET)6Ivacaftor11Cochlear implants8Mirabegron10Left ventricular assist devices6Nivolumab12LASER KTP8Nintedanib13Gene expression profiling diagnostics7Ocriplasmin11Nucleic acid amplification tests (NAATs)3Ofatumumab12Duodenal-jejunal bypass sleeve7Omalizumab10In-vitro fertilisation (IVF) 5Pasireotide8average6Ramucirumab14Rilpivirine10Riociguat12Sofosbuvir15average11,5 
 
 
 
                                                            
Examples  of  Member  States  confirming  the  growing  trend  towards  applying  HTA  to  support 
decision-making for medical technologies 

Spain.  The  HTA  Spanish  Network  (which  includes  the  regional  HTA  agencies  of  seven  autonomous 
regions and representatives from the regional health-care administrations of the remaining regions) is 
fully operational since few years and relies on regional HTA agencies to perform and coordinate HTA 
work  with  a  focus  on  medical  technologies.  The  network  has  been  instrumental  to  increase  the 
evidence-based  information  available  to  local  health  authorities  to  take  decision  on  access.  It  has 
contributed  to  build  capacity,  increase  consistency  and  quality  of  HTA  within  Spain  and  avoid 
duplication of assessments on medical technologies115.  

Italy. A recently adopted "national programme for HTA on medical technologies" is the response to 
the growing need of HTA for medical technologies in Italy. The programme has established a Steering 
Committee,  coordinated  by  the  Ministry  of  Health  and  gathering  key  national  agencies  and  the 
regional  HTA  bodies  which  have  expertise  and  perform  HTA  and  foresees  an  active  engagement  of 
stakeholders’ organisations. The final objective of the Programme, similarly to the one of the Spanish 
HTA Network, is to increase the availability of HTA for medical technologies, to provide guidance to 
decision  makers,  increase  consistency  and  avoid  duplication  of  assessments  for  better  use  of 
resources116.  

United Kingdom. While the National Institute for Health and Care Excellence/NICE has traditionally 
focused on pharmaceuticals, in the last years it established a dedicated programme for assessments of 
medical technologies117. 

The  issue  of  duplication  was  confirmed  by  the  input  received  in  response  to  the  public 
consultation,  where  57%  of  all  respondents  and  53%  of  participating  public  administrations 
indicated that differences in HTA processes and methodologies also resulted in duplication of 
work for their organisation. 

Differences  in  methodologies  and  procedures  are  considered  significant  obstacles  for  EU 
cooperation on HTA, limiting also the possibility of pooling resources and of a full benefit 
from the potential efficiency gains at EU level118.  

The uptake of joint EU outputs (i.e. joint tools – EUnetHTA Core Model, guidelines, joint 
early dialogues, joint REA, joint full HTA) at national level has remained low. Despite the 
fact  that  a  joint  European  report  was  prepared,  most  Member  States  still  performed 
assessments  of  the  same  technology.  The  low  uptake  of  joint  reports  was  confirmed  by  the 
evaluation  report  of  EUnetHTA  Joint  Action  2.  Looking  at  the  11  joint  assessments  carried 
out under this second Joint Action, on average each European assessment was used in the EU 
Member  States  6.4  times:  twice  related  to  direct  decision-making;  3.3  times  for  cross-
checking evidence or as a source of information; 0.7 times related to the category 'other' and 
for 0.4 times no data was indicated.119 

115  L.  Sanpietro-Colon,  J.  Martin  Eds,  Hospital-Based  Health  Technology  Assessment.  The  Next  Frontier  for 
Health Technology Assessment. Springer International Publishing 2016, pg.78 
116 http://www.salute.gov.it/portale/temi/p2_6.jsp?id=1202&area=dispositivi-medici&menu=tecnologie  
117 HTA National Programme for medical technologies  -  Strategy Document 19 September 2017 
https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-medical-technologies-guidance  
118 Kleijnen S, Toenders W, de Groot F, Huic M, George E, Wieseler B, Pavlovic M, Bucsics A, Siviero PD, van 
der  Graaff  M,  Rdzany  R,  Kristensen  FB,  Goettsch  W.  European  collaboration  on  relative  effectiveness 
assessments: What is needed to be successful? Health Policy. 2015 May;119(5):569-76. 
119http://www.eunethta.eu/national-uptake  

35 

 
 
                                                            
The low uptake of joint outputs is confirmed by the HTA GÖG-LSE Study which points out 
that most national HTA bodies, despite taking part in the joint assessments, did not adequately 
make use of the resulting output.  

Barriers  to  uptake  have  been  analysed  in  several  documents,  including  the  HTA  Network 
reflection paper on “Reuse of Joint Work in National HTA Activities”120, a survey carried out 
by EUnetHTA Joint Action 2121 a study commissioned by EFPIA122, and publications in peer-
reviewed journals118, 123,124. Barriers to uptake were also discussed by national organisations in 
their replies to the public consultation. 

Across the above-mentioned sources, the following main hurdles to uptake were identified: 

  Legal  uncertainty:  Uncertainty  around  the  status/relevance  of  the  joint  outputs  in  the 
context  of  national  HTA  frameworks  constitutes  a  major  reason  for  the  current  low 
uptake. As discussed in section 1.4.1., there are national legal/procedural frameworks for 
HTA in place in 26 Member States (also see Annexes VIII and IX for further details). As 
part  of  these  national  frameworks,  the  preparation  and  uptake  of  e.g.  national  clinical 
assessments  is  regulated  at  national  level.  While  there  is  some  diversity  due  to  the 
different legal systems, in general, key provisions related to the roles and responsibilities 
of the HTA bodies and the HTA assessments are  outlined in national law, while  further 
details are elaborated in administrative provisions (e.g. in procedural rules). By contrast, 
the  legal  status  of  joint  outputs  stemming  from  the  EUnetHTA  Joint  Action  and  their 
relevance for national HTA processes is not defined, making difficult for national decision 
makers to adapt their national legal framework to joint outputs.  

  Concerns  around  timeliness:  The  timely  availability  of  the  joint  output  (e.g.  joint  REA 
report)  for  national  decision  making  process  has  been  underlined  as  another  important 
limitation leading to low uptake. While in national HTA systems, timelines are enforced 
as defined in respective legal/procedural HTA frameworks, the EUnetHTA Joint Actions 
have so far not been able to ensure timeliness of joint outputs (e.g. joint REA reports) to 
meet Member States needs and feed into national decision-making processes. 

  Concerns  around  quality:  As  was  highlighted in  the  public  consultation,  Member  States 
will only  use a joint REA report if it is  of high quality. Some respondents in the public 
consultation considered that the first reports prepared under the first two EUnetHTA Joint 
Actions  were  of  sub-optimal  quality.  While  national  HTA  systems  have  established 
standard  operating  procedures  (SOP)  and  dedicated  quality  assurance  mechanisms  for 
national work, it has so far not been possible to ensure comparable procedural and quality 
standards  for  the  production  of  joint  work  under  the  EUnetHTA  Joint  Actions. 
Furthermore, there continue to be a number of methodological differences between HTA 

120 HTA Network. 2015. Reflection paper on “Reuse of Joint Work in National HTA Activities”. 
121 EUnetHTA JA2. WP3 DELIVERABLE Report on evaluation of project completion including assessment of 
impact on secondary users of HTA information p. 23. 
122  Charles  Rivers  Associates.  2017.  EU  REA  –  A  discussion  of  barriers  for  adoption  and  possible  actions  to 
overcome them Main findings. 
123 Lo Scalzo A, Vicari N, Corio M, Perrini MR, Jefferson T, Gillespie F, Cerbo M. Collaborative models for the 
joint  production  of  core  health  technology  assessments:  negative  and  positive  aspects  for  the  joint  work  of 
different European agencies. Int J Technol Assess Health Care. 2014 Nov;30(5):536-43. 
124 Vondeling H, Sandvei M. Is the planned and ongoing project (POP) database a suitable tool to reduce 
duplication in the process of assessing new health technologies in the European Union? Preliminary experience 
in the context of the EUnetHTA Joint Action Project Framework (2010-2012). Int J Technol Assess Health Care. 
2014 Nov;30(5):504-7. 

36 

 
 
                                                            
bodies (described in detail in section 1.4.1). Some HTA bodies have therefore expressed 
concerns  that  joint  work  may  not  be  fully  in  line  with  their  national  methodologies  (as 
defined in national legal/procedural frameworks and methods papers), impeding national 
uptake. 

  Topic prioritisation for joint work: The relevance of the topics selected for joint work (in 
particular  for  joint  REAs)  to  national  work  plans  and  priorities  is  another  important 
element to ensure national uptake. A number of Member States have noted that there have 
been  insufficient  mechanisms  for  topic  prioritisation  in  the  EUnetHTA  Joint  Actions  so 
far.  Some  topics  may  have  been  relevant  to  the  authors  or  other  partners  involved  in 
particular  Joint  Action  work  packages,  but  have  not  met  the  needs  and  priorities  of  all 
HTA bodies. 

  Other issues: Language barriers have been identified as a hurdle to uptake of joint outputs. 
In some HTA systems, use of the national language (e.g. in national HTA reports or early 
dialogues)  is  currently  defined  by  legal/procedural  frameworks.  Some  HTA  bodies  also 
noted that they would need to adapt their current human resources, to have staff with the 
right  profile  (e.g.  language  skills)  for  facilitating  uptake  of  joint  outputs.  Moreover,  a 
number of HTA bodies expressed the need for more training related to joint outputs (e.g. 
on the process and methods underlying the production of joint REAs), in order to facilitate 
incorporation in national work. 

While the points above refer to specific elements hindering the uptake of joint work, all 
of  them  are  related  to  the  first  point  on  the  legal  uncertainty  of  the  status  of  joint 
outputs. The current cooperation model via the Joint Action EUnetHTA is trying to address 
some  of  the  challenges  outlined  above  on  an  ad  hoc  basis  to  improve  uptake.  For  example, 
with support from the European Commission and EMA, arrangement have been put in place 
to facilitate contacts between CHMP125 rapporteurs of the dossier for marketing authorisations 
(once finalised) and authors of the Joint REA to allow an early start of the joint REA work 
which  could  facilitate  more  timely  availability  of  the  joint  assessment  report.  Criteria  for 
selection of authors and co-authors are also being developed by the Joint Action to contribute 
to  secure  quality  output;  quality  management  procedures  are  being  developed  and  stricter 
commitment  requirements  are  being  explored  to  engage  national  HTA  bodies  in  delivering 
what  was  promised.    However,  from  the  input  received  so  far  in  the  context  of  EUnetHTA 
and the HTA Network  discussions, it is  clear that as  long  as  the issue of the legal status  of 
joint outputs is not resolved, it is very challenging for national HTA bodies to ensure uptake 
of joint output in a systematic and continuous manner. 

In addition to the above, it should also be noted that low uptake decreases the readiness of 
industry to submit new technologies for a joint assessment. The preparation of a submission 
file costs financial and human resources. Such investments are not worthwhile and will not be 
done if the joint assessments have no relevance for national procedures. The current model of 
cooperation mainly relies on industry voluntary submission of joint assessments. Without the 
certainty  that  such  assessments  will  be  used  for  national  decision  making  (i.e.  uptake)  the 
willingness of industry to continue submission for joint assessments is expected to decrease.  
Also from industry's perspective the key problems with the joint reports are the same as those 
referred  to  above,  i.e.  inappropriate  timing,  insufficient  assurance  of  consistent  quality  and 
lack  of  uptake  in  national/regional  decision-making.  In  addition,  from  the  medical 

125 The Committee for Medicinal Products for Human Use (CHMP) is the European Medicines Agency's (EMA) 
committee responsible for human medicines. 

37 

 
 
                                                            
technologies perspective, not sufficiently addressing the specificities of the sector is also seen 
as a problem.  

Duplication  of  efforts  and  low  uptake  contribute  to  increased  work  and  costs  for  HTA 
bodies  and  to  sub-optimal  use  of  their  resources,  as  they  repeat  identical/similar 
assessments.  By  contrast,  current  experience  suggests  that  sharing  the  work  can  lower  the 
costs for HTA bodies significantly (in one case where only two agencies agreed to cooperate 
on  clinical  guidelines  they  were  able  to  save  30%  respectively).126  However,  if  joint 
assessments are not taken up, they can actually add  an additional layer of duplication, as an 
HTA body may end up working both on a joint assessment and a national assessment for the 
same technology.  

To  conclude,  the  current  duplication  and  low  uptake  imply  that  investments  into  the 
cooperation both in terms of resources from the EU budget and the human resources from 
the Member States are not used optimally. 

The example of EU pharmaceutical legislation  

The  issue  of  duplication  of  national  efforts  was  an  important  aspect  triggering  the 
development  of  the  pharmaceutical  legislation  and  the  set-up  of  the  European  Medicines 
Agency (EMA) as illustrated in the box below. While it should be noted that the scope and 
technical content of HTA differs from marketing authorisation of pharmaceuticals, the set-up 
of  EMA  provides  a  useful  example  of  how  scientific  and  technical  cooperation  can  be 
organised at European level and of the type of benefits it can lead to.   

The  development  of  the  pharmaceutical  legislation  and  the  setup  of  the  European  Medicines 
Agency (EMA) 

Despite  considerable  efforts  at  harmonisation,  at  the  beginning  of  the  1990s  the  European 
pharmaceutical market remained more fragmented, along  Member State boundaries, than any other 
market for consumer products, as the granting of marketing authorisations were entirely in the hands 
of Member States.  

In  November  1990,  the  Commission  proposed  to  Council  and  Parliament  a  major  overhaul  of  the 
European  authorisation  system  for  medicinal  products,  including    the  introduction  of  a  'centralised' 
EU  authorisation  procedure  for  technologically  advanced  medicinal  products  and  a  'decentralised 
procedure', operated by the Member States with EU arbitration if needed,  for all other products.  To 
support the operation of the new system, the Commission proposed setting up a 'European Medicines 
Evaluation Agency' (EMEA) – later renamed EMA.   

EMA was set up in 1995 to harmonise the work of existing national medicine regulatory bodies. 

The centralised procedure was a success, as it effectively allowed access to the entire  EU market in 
little over a year, closely matching the review time of the Food and Drug Administration of the USA. 
Previously,  it  had  taken  on  average  six  years  for  a  new  medicine  to  be  authorised  in  a  significant 
number  of  Member  States.  In  turn,  despite  a  slow  start,  the  decentralised  procedure  proved  to  be  a 
real alternative to the centralised procedure. 

EMA has a 20+ year track record of ensuring efficacy and safety of human and veterinary medicines 
across Europe, and promoting research and innovation in the development of medicines. 

126 GÖG-LSE Study. Annex: HTA Bodies Focus group minutes 

38 

 
 
                                                            
EMA’s success is based on cooperation within the European medicines regulatory network – a unique 
partnership between the European Commission, the medicines regulatory authorities in the European 
Economic  Area  countries,  and  EMA.  Working  together  has  encouraged  the  exchange  of  knowledge, 
ideas and best practices, in order to ensure the highest standards in medicines regulation. This works 
because EU legislation requires that each Member State operates to the same rules and requirements 
regarding the authorisation and monitoring of medicines. By working closely together, Member States 
reduce duplication, share the workload and ensure the efficient and effective regulation of medicines 
across  the  EU.  Today,  seven  EMA  scientific  committees  and  more  than  30  working  parties  provide 
scientific expertise for the regulation of medicines by drawing on a pool of several thousand European 
scientific experts from the network. 

Problem 3. Unsustainability of HTA cooperation 

The  current  EU  cooperation  on  HTA  is  project-based.  This  means  its  funding  needs  to  be 
secured  and  re-negotiated  in  every  financial  cycle  and  there  is  no  guarantee  for  continuing 
activities in the long term. During the initiation and closing of such a large project (the Joint 
Action  3  involves  81  participants  and  benefits  from  an  EU  contribution  of  approximately 
EUR  16  000  000)  substantial  time  and  resources  are  spent  on  organisational  issues.  For 
instance, more than one year after the launch of Joint Action 3, there are still positions to be 
filled in key work packages and in the coordinating institution. This has resulted in drops of 
joint outputs linked to project cycles (Figure 7) as well as in inefficiencies (e.g. development 
of  a  new  IT  system  for  every  new  project).127  The  evaluation  of  EUnetHTA  Joint  Action  2 
also highlights that finalising a project and starting a new one at the same time stretched the 
resources of the participating organisations and caused delays.128  

127 http://www.eunethta.eu/joint-assessments  
128  EUnetHTA  JA2.  Report  on  evaluation  of  project  completion  including  assessment  of  impact  on  secondary 
users of HTA information. 2016 p. 39. 

39 

 
 
                                                            
Figure 7. EUnetHTA Joint Action outputs129 

The  need  for  sustainability  was  highlighted  by  many  of  the  contributors  to  the  public 
consultation. Among the limitations of the current model of cooperation most cited by public 
administrations were: the lack of flexibility of the  framework for EU-funded projects which 
require high efforts for the preparation of a proposal, difficulties to put in place a sustainable 
IT platform (including IT tools) for the use of all participants and access of joint work, delays 
in  performing  joint  work  which  affected  the  availability  of  joint  reports,  insufficient 
commitment from all partners to use the output, uncertainties about the quality of joint work, 
insufficient coordination and agreement on topic selection, lack of knowledge on the impact 
on decision-making and the limited participation of some categories of stakeholders such as 
health professionals and patients. 

Organisations  representing  stakeholders other than HTA bodies (e.g. academia, patients  and 
consumers representatives) expressed concerns related to the limited duration in time and the 
lack of a sustainable funding mechanism of the current EU cooperation on HTA.  

The following case study illustrates the problems described this section. 

A case study for metastatic cancer treatment 

The pharmaceutical received marketing authorisation in mid-2011. HTA was performed on the same 
product by 12 HTA bodies in 11 countries, resulting in considerable duplication of work for both HTA 
bodies and industry. The timeframe of the reports is between 2011 and 2016, indicating the differences 
of the time of market access. 

129 Own calculation http://www.eunethta.eu/joint-assessments and SEED project final report 2017. 

40 

 
 
 
                                                            
There  are  commonalities  but  also  important  methodological  differences  in  the  clinical  assessment 
(REA)  regarding  the  comparator  and  the  evidence  used.  In  all  HTA  reports,  the  primary  evidence 
stemmed  from  the  same  randomised,  double-blind  clinical  trial.  Three  agencies  used  indirect 
comparison to assess the pharmaceutical against another comparator; four others considered another 
phase III study, an observational study or referred directly to the marketing authorisation report; one 
agency  also  considered  another  comparator.  A  number  of  social  value  judgements  (e.g.  end  of  life 
criteria or advantages related to the method of administration) were identified in the report; in some 
countries  they  were  considered  in  a  systemic,  in  others  in  a  less  standardised  manner.  Half  of  the 
agencies  considered  the  treatment  important  because  of  unmet  medical  needs.  Three  pointed  out  its 
innovativeness. Nonetheless, the fact that the treatment improves the quality of life of patients was an 
important  consideration  in  all  HTA  reports.  Patients  were  involved  in  varying  degrees  in  the 
processes.  

Following the HTA process, most of the countries reimbursed the pharmaceutical with criteria, in the 
rest with no criteria. It should be noted that in one country the product was rejected due to its cost-
effectiveness;  the  decision  was  eventually  reversed  due  to  a  reduced  price.  During  this  period  the 
product was available on a case-by case basis through a special fund, which gives a good example of 
the  complexity  of  the  relationship  between  HTA  and  access.  In  the  majority  of  the  countries 
confidential pricing or a risk sharing pricing agreement is in place, which often requires the collection 
of real world evidence.  

3.  Why should the EU act?  

Article 114 of the Treaty on the Functioning  of the European Union (TFEU)  allows for the 
adoption of measures for the approximation of the provisions laid down by law, regulation or 
administrative action in the Member States, provided they are necessary for the establishment 
or functioning of the internal market, whilst at the same time ensuring a high level of public 
health protection. Article 168(4) provides for the adoption of measures setting high standards 
of quality and safety for medicinal products and devices for medical use. Article 168(1) TFEU 
states  that  a  high  level  of  human  health  protection  shall  be  ensured  in  the  definition  and 
implementation of all Union policies and activities.  

Most health technologies are products which benefit from the free movement of goods within 
the  internal  market.  Despite  this,  a  number  of  obstacles  to  their  free  movement  have  been 
outlined in section 2 of this report. The procedural and methodological differences, along with 
the  considerable  duplication  of  HTA  across  the  EU  Member  States,  have  a  significant 
negative  impact  on  when  and  where  health  technologies  reach  the  market,  thus  reducing 
business  predictability  for  companies,  particularly  SMEs.  This,  in  turn,  contributes  to 
differences  in  patient  access  to  innovative  health  technologies.  These  divergences  and 
duplication also result in considerable additional costs for HTA bodies and industry alike. 

The aims of this initiative cannot be achieved sufficiently without strengthened cooperation at 
EU level. As described in section 2, the diversity and multitude of approaches to HTA across 
the  Member  States  means  that,  due  to  their  scale  and  effect,  only  action  at  Union  level  can 
eliminate the obstacles described. Without action at EU level it is unlikely that national rules 
on how HTAs are carried out would be harmonised and thus the current fragmentation of the 
single market would persist. 

41 

 
 
While  the  on-going  cooperation,  namely  the  Joint  Actions  and  the  HTA  Network,  has 
illustrated benefits of EU cooperation, in terms of establishing the professional network, the 
tools  and  methodologies  for  cooperation  and  piloting  joint  reports,  the  current  cooperation 
model has not contributed to the removal of the fragmentation of the national systems and the 
duplication of efforts. Without an EU initiative, it is unlikely long-term cooperation on HTA 
between  Member  States  would  be  significantly  strengthened  through  bilateral  or  regional 
cross-border  initiatives.  There  are  clear  additional  costs  for  HTA  bodies  and  industry  from 
carrying out HTA on the same health technology in multiple Member States. By carrying out 
a HTA only  once at EU-level,  economies of scale, greater business predictability, increased 
quality and consistency of HTA and improved transparency for patients would be achieved in 
the long run. 

This  impact  assessment  report  has  identified  a  division  between  the  HTA  domains  (the 
clinical domains) which lend themselves to a common assessment at EU-level and those (the 
non-clinical domains) which have more country-specific elements. By making this distinction, 
this initiative will maximise the EU added value while at the same time ensuring an approach 
to HTA assessment that is proportionate and in keeping with the principle of subsidiarity by 
leaving Member States to continue carrying out the parts of HTA better achieved at national 
level.  

The  report  has  also  underlined  the  differences  between  the  pharmaceutical  and  the  medical 
technologies  sectors,  not  only  in  relation  to  the  different  market  access  path,  but  also  in 
relation to  the role  HTA plays in  the two sectors and the lower level of duplication/parallel 
processes  compared  with  pharmaceuticals.  In  order  to  ensure  a  proportionate  approach  is 
taken,  such  differences  are  reflected  in  the  design  and  comparison  of  the  identified  policy 
options along with the different categories of products within sectors (centrally authorised v. 
national authorisation etc) which are reflected in the product scopes considered in the various 
policy  options.  The  proportionality  of  the  initiative  is  further  considered  in  the  design  and 
comparison of policy options, and the measures envisaged do not go beyond what is necessary 
to remove obstacles to the free movement of goods within the internal market.  

Creating  a  system  of  HTA  at  EU-level  would  necessitate  some  financial  and  administrative 
costs for the Union and for EU Member States. Such costs also need to be considered in light 
of the current EU-level HTA cooperation and its lack of sustainability as outlined in section 2.  

The principle of subsidiarity is furthered ensured in the initiative by fully respecting Article 
168(7)  TFEU  which  stipulates  that  the  Union  shall  respect  the  responsibilities  of  Member 
States for the definition of their health policies and for the organisation and delivery of health 
services  and  medical  care.  In  particular,  Member  States  are  responsible  for  decisions  on 
pricing and reimbursement, which are not within the scope of this initiative. 

4.  Policy objectives  

The  general,  specific  and  operational  objectives  of  the  initiative  are  listed  below.  Figure  8 
provides an overview linking the objectives to the problems discussed in detail in section 2. 

General objectives  

The general objectives of the initiative are: 

  Ensure a better functioning of the internal market 
  Contribute to a high level of human health protection 

42 

 
 
Specific objectives  

The specific objectives of the initiative are: 

Improve the availability of innovative health technologies for EU patients 

 
  Ensure efficient use of resources and strengthen the quality of HTA across the EU 
 

Improve business predictability 

Operational objectives 

The operational objectives of the initiative are: 

  Promote convergence in HTA tools, procedures and methodologies 
  Reduce duplication of efforts for HTA bodies and industry 
  Ensure the uptake of joint outputs in Member States 
  Ensure the long-term sustainability of EU HTA cooperation 

Figure 8. Intervention logic 

5.  Policy options  

Based  on  identified  shortcomings,  experience  with  the  current  cooperation  and  comments 
from  stakeholders,  the  following  key  principles  were  identified  for  constructing  the  policy 
options: 

  The need to build on existing structures, activities and achievements and maintain a 

Member States driven approach;  

  The need to address the specificities of the different sectors: pharmaceuticals, medical 

and other technologies; 

  Ensure  a  high  level  of  quality,  transparency  and  independence  (scientific  and 

financial); 

  Ensure 

the  engagement  of  stakeholders, 

in  particular  patients,  health  care 

professionals and payers; 

  Support the development of HTA capacities at national level. 

43 

 
 
 
   
A  number  of  policy  options  (PO  1-5)  were  identified  already  in  the  Inception  Impact 
Assessment130.  Of  these,  policy  option  5  was  discarded  upfront  (as  discussed  in  section  5.1 
below), while policy options 1-4 were further developed and are discussed in more detail in 
subsequent sections. 

5.1.  Discarded policy option 

The  inception  impact  assessment  put  forward  a  legislative  option  which  includes  joint  full 
HTA reports ("option 5"), i.e. joint production of HTA reports which cover clinical and non-
clinical (e.g. economic, organisational, ethical) HTA domains. This option was also included 
in the analysis of options conducted in the GÖG-LSE Study and in the public consultation. 

However, it has become clear from the input received in the public consultation and other fora 
that such an option is not realistic. While there is broad agreement that voluntary cooperation 
on  methodologies  to  develop  full  HTA  reports  would  be  useful  to  increase  consistency  and 
predictability  of  assessments,  the  development  of  EU  legislation  mandating  joint  full  HTA 
reports at EU level would bring more challenges than benefits. This is mainly due to the fact 
that  full  HTA  reports  rely  to  a  large  extent  on  context-specific  information  (e.g.  economic, 
organisational,  ethical)  in  order  to  serve  national  decision-making.  These  issues  have  been 
highlighted by public authorities, experts as well as industry representatives. This implies that 
a joint full HTA report could not in practice at this point in time support improved governance 
or sustainability.  

Option  5  as  analysed  in  the  GÖG-LSE  study  therefore  raises  concerns  as  regards  its 
proportionality, Member States' responsibilities under Article 168(7) TFEU and its feasibility. 
This option is therefore discarded upfront and is not discussed further in subsequent sections 
of this Impact Assessment. 

5.2.  Key characteristics of the policy options  

The different policy options for EU cooperation on HTA after 2020 are defined along several 
key characteristics focusing on:  

(1)  Joint  outputs,  (areas  in  which  EU  cooperation  seems  possible/useful)  which  could  be 
included in the initiative: 

Technology Specific Reports 

  Early dialogues with health technology developers; 
Joint Relative Effectiveness Assessments (REA)131. 
 

Common tools and procedures 

  Methodologies to formulate the contents and design of assessments (e.g. EUnetHTA 

Core Model and Standard Operating Procedure/SOP); 

  Horizon Scanning; 

130  Inception  Impact  Assessment  on  Strengthening  of  the  EU  cooperation  on  Health  Technology  Assessment 
(HTA), 
http://ec.europa.eu/smart-regulation/roadmaps/docs/2016_sante_144_health_technology_ 
assessments_en.pdf 
131 REA can take place at the time of market launch, or later. 

see 

44 

 
 
 
                                                            
  Procedural framework (clarifying inter alia the rights, obligations and involvement of 
transparency; 

stakeholders,  such  as  patients  and  health  care  professionals; 
independence etc.); 

  Submission  templates  and  templates  for  other  key  documents,  including  assessment 

reports and summaries; 

  Database  for  horizon  scanning,  planned,  ongoing  and  finalised  early  dialogues; 

 

planned, ongoing assessments and finalised assessments; 
IT  tools  for  exchanging  (confidential)  information,  for  supporting  the  collection  of 
Real World Data and for training and other capacity building activities. 

The common tools and procedures should build on the work already undertaken in Union-
funded actions on HTA, including EUnetHTA, Horizon 2020-funded actions. Preliminary 
results of regional initiatives such as the BeNeLuxA, and Valletta Declaration initiatives 
could  also  be  taken  into  account.  The  distinctive  characteristics  of  the  different  sectors 
(i.e.  pharmaceuticals,  medical  devices  and  others)  should  be  taken  into  account  in  the 
development of common tools and procedures.  

 (2) Technologies which could be covered by the initiative:  

  Pharmaceuticals; 
  Medical technologies (medical devices and in vitro diagnostics)132; 
  Other technologies (e.g. Screening or vaccination programmes, surgical procedures). 

(3) The choice of instrument used to establish and maintain the cooperation, including: 

  Voluntary cooperation of national bodies outside any EU agreement or framework (i.e. 
an  intergovernmental  approach  relying  exclusively  on  the  resources  and  political 
commitments  of  Member  States,  as  is  the  case  in  the  BeNeLuxA  or  other  similar 
initiatives); 

  Contractual obligations between (some/all) Member States with possible co-financing 
by  EU  programme/funding  but  no  dedicated  legal  framework  (i.e.  project  based 
approach); 

  Common legal framework (Regulation or Directive) 

Also combinations of these instruments could be envisaged. For example, some outputs could 
be governed by a legal framework, while others could be produced on a voluntary basis. 

(4) The choice of governance structure for the cooperation, including:  

  Project-based  secretariat:  a  coordinating  secretariat  is  set  up  and  run  for  the 
duration  of  a  project  by  a  Member  State  HTA  body  in  agreement  with  the  other 
participants of the consortium; 

  Member  State  secretariat:  a  secretariat  is  established  and  hosted  in  a    national/ 

regional HTA body;  

132 Medical devices and in vitro diagnostics as defined by Regulation (EU) 2017/745 of the European Parliament 
and  of  the  Council  of  5  April  2017  on  medical  devices  and  Regulation  (EU)  2017/746  of  the  European 
Parliament and of the Council of 5 April 2017 on in vitro diagnostic medical devices 

45 

 
 
 
 
                                                            
  Central  secretariat:  a  secretariat  is  established  and  hosted  in  the  European 

Commission, an existing EU Agency or a new EU agency133. 

(5) The financing (i.e. possible sources of funding): 

  EU funding, either through the Public Health Programme or another EU financial 

instrument134; 

  Funding by Member States participating in the cooperation, in cash and/or in kind;  

  Funding of joint assessments through industry fees have also been considered, but 
has been discarded in the first phase of this initiative. This consideration is based 
on a proportionality assessment weighing the relatively limited size of the structure 
foreseen for cooperation against the burden of setting up a fee structure. It was also 
considered  appropriate  to  evaluate  the  cooperation  after  a  certain  period  of  time 
based  on  experience  gathered  and  assess,  at  that  point  in  time,  whether  the 
introduction of a fee structure for joint assessments would be appropriate.  

From these elements, four policy options have been constructed, based on input from Member 
States, stakeholders and experts through the various forms of consultation activities (for more 
details on consultation activities, see Annex II). The study supporting the Impact Assessment 
process has also further discussed and validated the proposed Policy Option (See GÖG-LSE 
study, Chapter 4). 

The  policy  options  were  constructed  according  to  their  feasibility  (e.g.  mandatory  uptake 
considered  only  for  legislative  options)  as  well  as  logical  and  coherent  combinations  of 
elements (see section 5.3. below). The governance structure is assessed separately in section 
6.5. 

5.3.  Description of the policy options 

5.3.1.  Policy option 1 (Baseline scenario). No Joint Actions after 2020  

Rationale 

The  baseline  scenario  supposes  that  after  the  current  EUnetHTA  Joint  Action  3  will  end  in 
2020  there  would  be  no  further  Joint  Action  on  this  topic.  The  EU  funding  to  support 
scientific and technical cooperation among Member States would be discontinued.135   

The choice of the baseline scenario is justified by the fact that although the Joint Actions have 
been  successful  in  demonstrating  a  proof  of  concept,  a  continuation  in  the  form  of  a  fourth 

133  Two  further  possibilities  were  considered  and  disregarded:  1)  a  rotating  MS  Secretariat  and  2)  a  fully 
centralised  model  where  both  the  support  function  and  scientific  expertise  are  integrated.  Both  models  were 
considered  not  feasible  due  to  the  limited  support  received  in  the  public  consultation  and  the  limitations  they 
would  pose  in  terms  of  implementation  (e.g.  losing  know-how  for  the  rotational  model,  and  the  challenge  in 
building and maintaining the necessary know-how in a fully centralised model) and acceptance (e.g. the link to 
national HTA processes is lost in case of a fully centralised model).  
134  For  all  policy  options  the  source  and  the  amount  of  funding  are  dependent  on  the  negotiations  of  the 
Multiannual Financial Framework (within the European Commission and the other relevant EU institutions) and 
subject to renewal every budgeting period. 

46 

 
 
                                                            
 
Joint Action is deemed to be both ineffective and unrealistic (see section 1.4.2 and section 2, 
problem  2).    This  was  considered  the  most  likely  and  most-evidence  based  baseline  also  in 
light  of  the  indications  from  the  Court  of  Auditors,  which  considers  that  this  type  of 
actions/projects is not supposed to be renewed too many times. 

The  Joint  Actions  have  created  the  necessary  trust  to  enable  HTA  bodies  to  work  together, 
have developed common methodologies and tools and have demonstrated by pilots that joint 
outputs, including joint assessments can be done. This demonstration of "proof of concept" is 
in line with the purpose of Joint Actions (see box below for further information). 

The role of Joint Actions as an EU funding instrument 

Joint Actions are collaborative projects specific to the Health Programme aiming to develop / share / 
refine  /  test  tools,  methods  and  approaches  to  specific  issues  or  activities,  and  engage  in  capacity 
building in key areas of interest for the Member States and countries participating to the Programme. 
They are co-financed by the European Commission and authorities of the Member States. This type of 
project  was  introduced  during  the  2nd  Health  Programme  (2008-2013)  and  continued  under  the 
current  one  (2014-2020)  to  cover  specific  health-policy  needs  and  aimed  at  supporting  EU 
cooperation in the field of health with as many partners as possible from all countries participating in 
the Programme136.  

Joint actions are often started, after several years of cooperation between relevant stakeholders and 
participants are designated by Member States authorities, in a bid to secure political endorsement and 
optimise policy coordination. Joint Actions are grants, assigned through a non-competitive negotiated 
procedure  (i.e.  for  each  possible  topic  only  one  proposal  for  a  Joint  Action  will  be  submitted),  and 
they are an exception to the Financial Regulations. The Commission, in consultation with the relevant 
Member  State  Programme  Committee  defines  the  policy  area  and  provides  a  general  outline  of  the 
aim,  the  final  objective  and  the  budget  of  the  Action.  All  Member  States  are  invited  to  appoint  the 
organisations  they  consider  relevant  for  the  Action  and  propose  a  detailed  work  plan,  which  the 
Commission  is  expected  to  accept.    As  emphasised  also  in  the  Commission's  Ex-post  Evaluation 
Report  of  the  2nd  Health  Programme  2008-2013137,  "joint  actions  build  on  previous  achievements 
made  possible  through  project  grants  started  sometimes  10  or  more  years  ago".  EUnetHTA  was 
singled  out  as  an  example  where  a  third  joint  action  even  though  possible,  was  not  seen  as  an 
undisputable fact. 

However, while constituting a good tool for testing and validating new ways of cooperation, 
the  EUnetHTA  Joint  Actions  have  showed  important  shortcomings  in  terms  of  disruptions 
(regular renegotiations/reallocations of work packages, renewal of staff etc.), and difficulties 

136 http://ec.europa.eu/chafea/documents/health/ja-2017-guide-applicants_en.pdf 
137 https://ec.europa.eu/health/sites/health/files/programme/docs/ex-post_2nd-hp-2008-13_comm-swd.pdf  

47 

 
 
 
                                                            
in  ensuring  consistent  delivery  of  high  quality,  timely  output  and  uptake  (see  sections  1.4.2 
and 2, problem 2). 

In  addition,  as  described  above  the  objective  of  a  financing  through  Joint  Actions  is  not  to 
support permanent/long-term/recurrent actions. This was confirmed by the Court of Auditors 
in its Report on cross-border threats to health, stating that “…given their significant size, they 
(NB. Joint Actions) take more time to prepare and also require political backing and national 
co-funding.  This  means  that,  despite  their  potential  for  increasing  the  EU-wide  take  up  of 
outputs produced with health programme funding, there cannot be too many subsequent joint 
actions in one policy area ."138 

In  conclusion,  Joint  Actions  are  only  intended  to  kick-start  policy  coordination  between 
Member States and are not intended to run indefinitely and have proven to be not effective in 
feeding in to national decision making processes (see section 1.4.2)139.  

In view of the above, a fourth Joint Action is not considered a realistic and credible option to 
address the problems and achieve the objectives of the initiative.  

Description 

Under the baseline scenario (policy option 1), the European cooperation would be limited to 
the high-level strategic policy discussions within the HTA Network, which mainly consists of 
meetings  between  Ministries  of  Health  and/or  national  HTA  agencies  to  discuss  policy 
developments  which  are  relevant  to  HTA  both  a  national  and/or  European  level.  However, 
without  a  continuous  and  sustainable  support  to  coordinate  and  develop  technical/scientific 
activities, including the developments of joint outputs, it is not expected that Member States 
will devote resources to continue the cooperation at EU level in a broader and more organised 
way.  This  will  be  particularly  true  for  Member  States  with  limited  resources  and  less 
developed HTA systems, which will need to focus their resources on performing assessments 
or identify reports from other agencies and/or possible regional networks that can be adapted 
and used at national level.  

No  dedicated  governance  structure  is  foreseen  under  this  policy  option.  Member  States  are 
relying on national resources as illustrated above (section 1.4.1). The trend of more Member 
States developing their own specific HTA systems (mostly in Central and Eastern Europe) is 
also  expected  to  continue,  with  different  procedural  frameworks  and  methodologies,  and 
variability  in  practices,  procedures  and  methodologies.140  The  differences  as  regards  good 
governance  principles,  e.g.  transparency  of  the  HTA  processes,  stakeholders'  participation/ 
involvement and quality control mechanisms would also continue to exist. HTA bodies would 
continue  the  trend  of  duplicating  assessments  of  the  same  technology  carried  out  in  other 
Member  States,  in  particular  for  pharmaceuticals.  The  capacity  of  HTA  bodies  to  cover  all 
relevant innovative technologies would remain limited: higher for the already well-established 

138 European Court of Auditors, Special Report Dealing with serious cross-border threats to health in the EU: 
important steps taken but more needs to be done, 2016 
https://www.eca.europa.eu/Lists/ECADocuments/SR16_28/SR_HEALTH_EN.pdf  

140 EFPIA/ Charles River Associates. 2017. Assessing the wider benefits of the EU’s proposal on strengthening 
cooperation on health technology assessment from the industry perspective.  

48 

 
 
                                                            
 
 
HTA systems, while the others would have to continue to limit themselves to assessing only a 
lower number of technologies. Member States with limited resources would therefore not be 
able to  fully benefit from HTA in  their efforts to  address challenges  related to  rising health 
care  expenditures,  evolving  health  technologies,  ageing  populations  and  increasing  burden 
from chronic diseases141,142. Pooling of resources and re-use of jointly developed HTA reports 
are  not  expected  to  a  significant  extent,  as  there  would  be  no  EU-wide  mechanism  and  no 
specific  resources  dedicated  to  EU-wide  activities  by  Member  States.  Adaptation  of  reports 
from  other  HTA  bodies  may  continue,  especially  in  Member  States  with  limited  HTA 
capacity, as this would be the main source to build capacity and know-how. 

Without  EU  stable  cooperation,  joint  early  dialogues  would  be  limited  to  HTA  bodies 
participating  to  the  parallel  scientific  advice  procedure  offered  to  developers  by  EMA.  The 
achievements  of  the  current  Joint  Action,  such  as  the  single  platform  put  in  place  by 
EUnetHTA and EMA which ensures not only a coordinated advice from regulators, but also a 
coordinated opinion from several HTA bodies, would likely be jeopardised if the process is 
not sustained overtime. 

Regional cooperation is expected to continue on a voluntary basis, in particular in relation to 
the production of some joint assessments to be used in possible joint price negotiations and 
procurements  efforts.  On  the  other  hand,  as  different  regional  cooperation  networks  are 
developing across the EU, duplications between the regional networks are likely to occur, and 
divergences as regards processes and methodologies can be expected between these networks 
in  addition  to  the  continued  national  divergences.  Moreover,  as  recent  discussions  around  a 
potential  joint  horizon  scanning  activity  in  the  context  of  the  BeNeLuxA  initiative  have 
shown143,  there  are  significant  challenges  in  identifying  a  suitable  organisational,  legal  and 
financial framework to perform joint activities in a regional cooperation setting. 

The HTA Network  established  under Directive 2011/24/EU is  expected to  continue to  meet 
twice per year to share some high level national experiences.  

5.3.2.  Policy option 2.  Project-based cooperation on HTA activities  

This option foresees voluntary cooperation supported by EU funding organised in the form of 
project(s)144  other  than  Joint  Actions.  The  project(s)  would  be  funded  under  the  Health 
Programme or any other EU financing instruments (e.g. Horizon 2020).  

The  instrument  to  implement  this  option  would  be  project(s)  through  competitive  calls  for 
proposals  in  line  with  the  priorities/EU  added  value  criteria  identified  by  the  Commission 
following lessons learnt from EUnetHTA and other projects/initiatives.  

The  calls  would  support  the  development  of  a  defined  number  of  joint  outputs  (e.g.  joint 
assessments and/or early dialogues) in a given timeline. The selected project(s) is expected to 
last 36-48 months, during which it would need to deliver the planned output. To address the 
shortcoming  of  EUnetHTA,  the  eligibility  criteria  would  be  more  specific  and  prescriptive 
than the  ones  which can  be  used  for  Joint  Actions.  For example,  the  call  would  specify  the 

141 OECD. 2015. Pharmaceutical expenditure and policies: past trends and future challenges. 
142 European Commission, DG ECFIN - EPC. (2016) Joint Report on Health Care and Long-Term Care Systems 
& Fiscal Sustainability. Brussels: Publication Office of the European Union, p. 1-13. 
143 For more information on BeNeLuxA initiative, see http://www.beneluxa.org/. 
144 This could be done as one project, subsequent projects or multiple parallel projects. The assessment of PO2 in 
this report is based on the assumption of one project in line with the GÖG-LSE study.  

49 

 
 
                                                            
minimum  number  of  participants  from  different  Member  States  to  ensure  a  sufficient  EU 
coverage,  the  maximum  number  from  each  Member  States  (e.g.  two  agencies  per  Member 
States)  to  ensure  an  efficient  and  manageable  consortium.  Their  profile  and  role  in  the 
national  decision-making  process  will  also  need  to  be  well  defined  to  ensure  that  the  most 
relevant HTA Agencies are included and the necessary expertise participates. Conditionality 
clauses  would  also  be  included  to  encourage  uptake  of  results  (i.e.  final  payment  would  be 
subject to the level of uptake of the joint output). 

To  facilitate  commitment  from  technology  developers,  their  European  trade  associations 
could  also  be  included  in  the  project.  Involvement  of  patients,  clinical  societies  and/or 
healthcare professional organisations would also be foreseen to increase participation of these 
stakeholders in the development of joint outputs.  

Such  a  model  differs  from  a  Joint  Action  because  it  would  be  based  on  competitive  calls 
which may result in more than one group (i.e. Consortia) of Member States competing with 
each  other.  The  number  of  beneficiaries  in  the  selected  project  will  be  lower  and  with  a 
homogenous  profile  and  role  in  the  national  decision-making  process.  Engaging  in  a 
competitive  process  with  relatively  high  level  of  co-financing  from  beneficiaries  (normally 
50-40%)  is  expected  to  ensure  a  higher  commitment  to  the  objectives  and  outputs  of  the 
project than what is the case in a Joint Action, including the uptake of results.   

The  evaluation  process  and  the  subsequent  negotiation  with  the  winning  Consortium  is 
expected to result in a focused and defined work plan, which will be limited to the output for 
which cooperation has demonstrated a clear EU added value (e.g. focus on clinical aspects of 
the  HTA  process  for  joint  REA).  This  is  different  from  negotiations  within  Joint  Actions 
typically focusing on solving administrative issues due to the complexity of the different legal 
status of the appointed beneficiaries and the Commission's limited possibilities to modify the 
proposed work plan. 

A  similar  project-based  model  was  tested  for  Early  Dialogues  through  the  project  SEED 
(2012-2015).  It  could  address  some  of  the  shortcomings  identified  in  the  Joint  Action 
EUnetHTA  (delays,  high  number  and  heterogeneous  profile  and  number  of  participants, 
inconsistency of the quality and timely outputs). 

The  scope  of  the  cooperation  could  cover  all  categories  of  health  technologies: 
pharmaceuticals,  medical  technologies  and  other  health  technologies  (see  5.1.2).  For  joint 
technology-specific  reports  (joint  assessments  and  early  dialogues)  the  activity  would  be 
limited to  clinical domains. For other  types of activities (cooperation on  methodologies  and 
procedures), 
(e.g.  economic, 
organisational,  ethical  assessment).  This  approach  would  contribute  to  ensure  best  use  of 
resources and focus on outputs which have demonstrated major EU added value.  

the  projects  could  cover  also  non-clinical  domains 

No EU legal framework is foreseen under this policy option and the governance model would 
the  winning 
be  a  project  secretariat  managed  by  one  of 
consortium/consortia.  It  is  expected  that  similarly  to  a  Joint  Action,  a  national  HTA  body 
would  take  up  the  coordination  role  and  distribute  and  monitor  tasks  and  responsibilities 
between partners to ensure the delivery of the agreed joint outputs.  IT tools will be managed 
by  the  project  throughout  its  duration.  IT  tools  developed  by  EUnetHTA  could  be  possibly 
reused and built upon, provided the winning consortium would have the right to exploit them.  

the  beneficiaries  of 

The  cooperation  foreseen  under  this  option  could  also  continue  to  benefit  from  the  HTA 
Network, which would be a platform to share practices of the different activities and would 

50 

 
 
become an important mechanism to ensure inclusiveness of the cooperation to Member States 
which may not be directly involved in the cooperation in the winning project(s).   

This  option  foresees  a  top-down  approach  with  the  Commission  in  the  lead,  identifying 
priorities,  launching  the  call  for  proposal,  monitoring  the  projects  and  disseminating  the 
results. 

The financing of this option would rely on the EU budget and Member States' co-financing 
(normally by providing in kind contributions).  

5.3.3.  Policy option 3. Permanent cooperation on common tools, 

procedures and early dialogues 

Table 3. Overview of policy option 3. 

In  option  3,  the  joint  outputs  would  cover  early  dialogues  (the  only  technology  specific 
reports  under  this  option)  and  various  common  tools  and  procedures  (listed  in  Table  3).  As 
described  in  section  5.2,  the  common  tools  would  be  based  on  work  already  carried  out  by 
existing  cooperation  mechanisms  and  the  specific  characteristics  of  different  health 
technologies  (e.g.  pharmaceuticals  and  medical  technologies)  would  be  taken  into  account 
when  developing  the  common  tools  and  procedures.  The  option  foresees  upwards 
harmonisation of a basic set of tools and procedures (see Table 3 and further details in section 
5.2), to ensure a high level of quality throughout the EU. Common procedures will be aimed, 
in  particular,  at  ensuring  the  involvement  of  patients  and  external  expert  (e.g.  healthcare 
professionals)  in  the  HTA  process,  avoiding  conflicts  of  interest  and  ensuring  transparency 
(e.g. via publication of joint outputs). 

Option 3 would in principle cover all types of health technologies, subject to selection and 
prioritisation  criteria  in  accordance  with  the  needs  of  Member  States.145  It  should  also  be 

145 In principle Member States will select and prioritise health technologies which shall undergo early dialogue 
based on given criteria.  

51 

 
 
 
 
                                                            
noted that, as early dialogues would be initiated by the industry, it cannot be guaranteed that 
all technologies will benefit from them.  

templates,  IT 

The  instrument  used to  implement  this  option  would  be  a  (new)  EU  legislative  framework 
which  would  ensure  the  mandatory  uptake  by  HTA  bodies  of  the  common  tools  and 
procedures  and  of  joint  early  dialogues.  These  tools,  procedures  and  early  dialogues  would 
relate to the clinical aspects of HTA, thereby supporting the assessment at Member State level 
of  the  clinical  domains  of  HTA  (REA).  Mandatory  uptake  of  the  common  tools  (e.g. 
tools)  and  common  procedures  (e.g.  for  stakeholder 
methodologies, 
involvement)  implies  that  Member  States  shall  use  these  basic  tools/procedures  when 
conducting  joint  early  dialogues  and  clinical  HTA  work  at  national  level  to  ensure  high 
quality and to facilitate cooperation and use of each other's assessments. The development of 
these common tools and procedures is expected to rely mainly on results of the Joint Actions 
EUnetHTA  and  to  be  further  developed  by  experts  nominated  by  national  HTA  bodies. 
Mandatory  uptake  of  early  dialogues  means  that  Member  States  shall  use  the  joint  early 
dialogues in the same way that they would use a national early dialogue, i.e. they should not 
repeat at national level an early dialogue which has already been conducted jointly. 

The governance of the cooperation under this option would be ensured by a central structure 
which  could  provide  administrative,  scientific  and  IT  support  to  deliver  joint  outputs  of  a 
consistent high quality, in a transparent, independent and timely manner and with appropriate 
involvement/consultation  of  stakeholders.  To  deliver  the  joint  output,  the  cooperation  shall 
rely  on  HTA  experts  from  Member  States  HTA  bodies  organised 
in  dedicated 
committees/groups covering the outputs.  

The  financing  of  this  option  is  expected  to  rely  mainly  on  the  EU  budget  and  in  kind 
contributions from Member States, which would be asked to provide expertise through their 
experts.  For  early  dialogues,  depending  on  the  governance  structure  chosen,  a  fee  from 
industry  would  cover  the  costs  of  the  experts  and  the  overheads  needed  to  support  the 
production of this specific joint output.146  

Article  15  of  Directive  2011/24/EU  would  be  deleted  under  this  option  as  it  would  not  be 
compatible  with  the  legislative  approach  suggested  above:  the  HTA  Network  foresees  fully 
voluntary cooperation, the output of the cooperation have no legal status. While Art 15 would 
be  deleted  from  Directive  2011/24/EC,  the  foreseen  new  Legal  framework  would  maintain 
and  further develop the objectives  defined  by  the  article,  and  add  provisions  to  ensure  their 
achievements, which is currently limited (see section 2). It will also re-introduce key elements 
already foreseen by the article such as the involvement of stakeholders in the cooperation and 
it  will  use  similar  working  methods  already  applied  such  as  the  setting  up  of  dedicated 
Member  States  experts'  groups/subgroups  to  develop  the  specific  outputs,  it  will  further 
develop  its  good  governance  principles  in  a  dedicate  governance  structure.    In  addition  the 
new Legal framework would provide a more stable framework for granting aid to support the 
cooperation.  

146 Industry fees would only be possible if the tasks are carried out by an EU Agency.  

52 

 
 
 
                                                            
5.3.4.  Policy option 4. Permanent cooperation on common tools, 

procedures, early dialogues and joint REA  

Table 4. Overview of policy option 4 

Option  4  comprises  the  joint  outputs  included  in  option  3  (common  tools/procedures  and 
joint  early  dialogues,  as  described  more  detail  in  section  5.3.3)  and  in  addition  joint  REAs 
(i.e. assessments of the clinical HTA domains).  

For  pharmaceuticals,  the  joint  REAs  would  comprise  centrally  authorised  pharmaceuticals 
and other pharmaceuticals prioritised by Member States due to their high value, high budget 
impact, or their impact on public health / addressing unmet medical needs.147 

For  medical  technologies,  the  scope  for  joint  REAs  comprises  those  that  are  prioritised  by 
Member  States  based  on  their  potential  high  risk  (i.e.  devices  undergoing  the  EU  scrutiny 
mechanism)  or  potential  impact  on  public  health  and  health  systems  (e.g.  addressing  unmet 
medical need, potential to transform the organisation of care, high budget impact).148 

The option also foresees a phase-in approach for joint REAs, i.e. a gradual introduction of the 
full product scope while the system is built up. 

The  instrument  used to  implement  this  option  would  be  a  (new)  EU  legislative  framework 
which would not only ensure the mandatory uptake of the common tools and procedures, and 
joint early dialogues as in option 3, but also of joint REAs. Mandatory uptake of joint REAs 
implies  that  Member  States  would  use  the  joint  assessment  reports  in  the  same  way  as  a 
national assessment report is used today and that the REA should not be repeated at national 
level. Member States would however continue to be free to assess other (non-clinical HTA) 

147 See GÖG-LSE study (Table 7, section 4.3 and annexes 3 and 4). 
148 See GÖG-LSE study (Table 7, section 4.3 and annexes 3 and 4). 

53 

 
 
 
 
                                                            
domains at national or regional level and would continue to draw the overall conclusions on 
the basis of the (joint) clinical and (national) non-clinical assessment parts149.  

The  governance  structure  would  be  similar  to  option  3  but  taking  into  consideration  the 
extended scope in terms of joint outputs (joint REA). In this respect, Member States experts 
will be supported by a central secretariat providing administrative support (e.g. organisation 
of  meetings,  travel  arrangements  etc.),  scientific/technical  and  IT  support.  A  management 
board  including  representatives  of  Member  States'  HTA  bodies  would  manage  the  overall 
governance  and  would  meet  regularly  to  discuss  topic  prioritisation,  progress  with  outputs 
(e.g. quality, timeliness), provide guidance and steer the cooperation. The scientific-technical 
work of producing the joint outputs would be carried out by experts nominated by Member 
States' authorities.150 For joint REAs, Member States' experts acting as author/rapporteur and 
co-author/co-rapporteur  would  carry  out  the  clinical  assessment  of  the  application/dossier 
submitted by industry (complying with common tools and procedures as described in section 
5.2)  and  prepare  a  joint  assessment  report.  A  committee/group  including  experts  nominated 
by  Member  States  would  thereafter  examine  the  draft  and  approve  the  joint  report  which 
would  then  be  incorporated  in  national  HTA  processes  (see  more  detailed  explanations  on 
mandatory uptake above). 

The financing solution is the same as the one foreseen in option 3. 

Article  15  of  Directive  2011/24/EU  would  be  deleted  under  this  option  as  it  would  not  be 
compatible with the legislative approach suggested. As under option 3, some key components 
would be maintained and further developed in the new legal framework.  

Option 4 could be divided in two sub options:  

  Option  4.1-  an  'opt-in'  system:  For  joint  REA,  such  a  system  would  allow  Member 
States,  without  prejudice  to  the  need  to  achieve  the  stated  specific  objectives  and 
ensure legal feasibility, some flexibility to decide if / when to start participating in the 
EU-level  system  of  joint  REA  depending  on  their  individual  situation  in  terms  of 
needs of adjusting national law and practice etc. This decision to participate would be 
system-based  (i.e.  Member  States  would  decide  whether  or  not  to  participate  in  the 
system for all joint  REA conducted at EU level) and not  on a product-specific basis 
(i.e.  Member  States  would  not  decide  for  each  product  submitted  for  joint  REA 
whether  to  participate  or  not).  Member  States  not  participating  in  the  joint  REA 
system would still be obliged to use the common tools and procedures (option 3) when 
carrying out their own REA. 

  Option  4.2:  is  essentially  the  same  as  option  4.1  with  the  difference  that  this  option 
would be applicable to all Member States with no possibilities to opt in later or stay 
out.  

149  As  discussed  in  sections  1.2,  1.4.1  and  the  Mapping  study  on  HTA  processes  across  the  EU,    all  current 
national  HTA  processes  include  an  assessment  of  clinical  evidence  (REA),  but  in  addition  may  also  assess 
various non-clinical domains (e.g. economic, organisation, ethical) on the same health technology. 
150 This would be similar to the model implemented by EMA for the central marketing authorisation procedure 
for medicinal products. 

54 

 
 
 
 
                                                            
 Table 5. Overview of policy options  

55 

 
 
 
 
6.  Impacts of the policy options  

This chapter will identify and describe the expected impacts of the policy options 2, 3 and 4, 
compared to the baseline scenario (option 1) described in section 5.3.1. The following impacts 
have been identified as most relevant for the key stakeholders.  

Member States/Public 

Patients/consumers 

Industry (pharmaceutical and 

medical technologies) 

- Functioning of the internal 

market 

- Costs  

- Business predictability 

- Innovation, research and 

competitiveness  

- Administrative burden 

Economic Impacts 

- Costs  

Administrations 

- Efficient allocation of 

resources 

- Administrative burden 

- EU budget 

Social/health 

- Governance, participation 

- Participation/ 

impacts   

and good administration 

involvement 

- Sustainability of health 

- Availability of 

systems  

innovative health 

- Public health 

technologies 

Table 6. Summary of key impacts for stakeholders  

The key challenge in assessing and quantifying the impacts has been the fact that HTA is an 
(often advisory) input for decision making; the access to health technologies and their prices 
are  set  by  the  national  pricing  and  reimbursement  decisions.151  Therefore,  many  impacts  of 
the  HTA  cooperation,  in  particular  on  sustainability  and  public  health  are  indirect. 
Quantitative assessments have been completed with qualitative assessments when necessary. 

None of the options are likely to have considerable impact on the overall demand for health 
technologies.  Therefore,  no  substantial  changes  in  the  production  and  distribution  in  the 
pharmaceutical  and  medical  technologies  sector  are  expected.  As  regards  HTA-related 
employment, no major effects in HTA staffing in Member States HTA bodies are expected. 
Some efficiency gains for REA could be envisaged, but resources are likely to be shifted to 
increasing demands in assessing additional technologies, re-assessments etc. No impact on the 
overall employment in the sector is therefore expected136. No impacts have been identified on 
trade152, on environment and on fundamental rights.  

The  common  horizontal  aspects  of  the  governance  system  and  financing  are  analysed  in  a 
dedicated section (6.5). 

151 It should also be noted that there is a difference between the actual prices of pharmaceuticals paid by social 
insurances  and  the  publicly  available,  official  list  prices.  Actual  prices  tend  to  be  lower  due  to  arrangements 
between  industry  and  payers.  However,  such  discounts  and  rebates  are  typically  strictly  confidential  and 
therefore  the  actual  prices  are  not  known.  WHO.  Background  Paper  8.3  Pricing  and  Reimbursement  Policies: 
Impacts on Innovation. 2013. 
152 GÖG-LSE Study 

56 

 
 
 
 
 
 
 
 
                                                            
The  estimations  of  costs  included  in  this  Impact  Assessment  report  were  provided  by  the 
GÖG-LSE Study and took into account the following type of costs:  

-  costs  for  the  implementation  mechanism/governance  (e.g.  project-based,  permanent 
structure;  include  costs  of  human  resources,  IT  tools,  travel,  premises  etc.).  The  personnel 
costs and costs resulting from Member States’ expert committees were estimated based on the 
average  salaries  of  EUnetHTA  JA  3  partners  (for  project-based  cooperation),  and  the  Staff 
Regulation  of  Officials  of  the  European  Communities  (for  a  permanent  structure)  and 
Commission expert fees; 

-  costs  of  the  output  production  are  based  on  the  assumption  that  annual  joint  output  will 
increase  gradually from option 2 to 4, from 13  joint  early  dialogues (EDs) and 12 REAs in 
option  2  to  40  joint  EDs  and  65  joint  REAs  in  option  4.  The  number  of  joint  EDs  was 
estimated taking into account the number of requests for scientific advice received by EMA 
per year, the number of requests received by EUnetHTA JA2 and 3 and the SEED project, as 
well as the average value of early dialogues performed at national level by the Member States 
offering this service to technology developers. The number of joint REAs was approximated 
based on the average number of centrally authorised medicinal products per year (approx. 40 
new molecules) and the average number of assessments for medical technologies carried out 
at  national  level  (taking  into  account  that  in  some  Member  States  assessments  of  certain 
medical technologies are also mandatory).  

 Policy option 2. Project-based cooperation on HTA activities 

6.1.1.  Economic impacts  

Member States/Public administrations 

Compared to the baseline scenario (described in section 5.3.1), a large part of the duplication 
of work is expected to persist under policy option 2. The problem of duplication of work for 
national HTA bodies (discussed in detail under section 2, problem 2) would largely remain, 
entailing continued significant costs for HTA bodies e.g. related to the production of REAs at 
national level (see section 2, problem 2, text box).  

In particular, the legal uncertainty around the status/relevance of the joint outputs stemming 
from the project foreseen under this option is expected to remain. It cannot be expected that 
to  ensure 
their  national  HTA 
Members  States  will  adapt 
consistency/compatibility with  uptake of joint work (e.g. changes  to  language  requirements, 
formats, procedures) produced in the context of time-limited, voluntary EU-funded projects. 

frameworks 

legal 

Even  if  contractual  arrangements  are  foreseen  under  this  option  aimed  to  promote/require 
national  uptake,  it  can  be  expected  that  HTA  frameworks  defined  in  national  law  or 
administrative  provisions  (e.g.  the  social  code)  would  prevail  over  any  contractual 
arrangements  in  the  context  of  a  project.  As  the  GÖG-LSE  study  points  out,  while 
theoretically  such  contractual  arrangements  would  be  possible  if  parties  agree,  there  is  no 
effective  possibility  for  the  European  Commission  to  enforce  these  obligations  and  as  such 
address the issue153. 

153 GÖG-LSE Study Section 4.3 

57 

 
 
                                                            
In  addition,  a  contractual  obligation  to  promote/require  national  uptake  may  discourage 
participation  in  project(s),  especially  in  those  Member  States  where  HTA  process  and 
procedures are well developed and defined in national law. These are normally the Member 
States which are less in need of EU cooperation to satisfy their national HTA obligations, and 
as  such  they  may  find  the  contractual  constraints  of  the  project  not  proportionate  for  the 
benefit they may gain. 

Since  there  is  no  guarantee  of  uptake  of  joint  outputs  in  national  HTA  systems,  buy-in, 
commitment and resource investment by Member States into an EU project for production of 
joint outputs are also expected to remain limited. If uptake of joint outputs is not ensured, the 
joint work cannot be fully effective in terms of reducing duplication of efforts for HTA bodies 
and creating efficiency gains through joint work.  

The  duplication  of  work  under  option  2  will  consist  both  in  national  HTA  activities 
continuing  in  parallel  and  in  addition  to  joint  work  stemming  from  the  project.  Such 
duplication  is  expected  to  result  in  additional  costs  and  further  negative  impacts  on  the 
efficient use of resources.  

In  addition,  the  same  considerations  made  for  policy  option  1  in  relation  to  the  potential 
impact of regional cooperations would apply also under this policy option. But contrary to the 
option 1, in option 2 regional cooperations may have a negative impact on ensuring efficient 
use of resources as they may add further to the duplication described above as Member States 
could be active in both regional and/or European cooperation efforts, as it is currently the case 
with EUnetHTA. 

Nonetheless, compared  with  option 1, due to some requirements to  improve  the quality and 
consistency of the joint work that the option would put in place (e.g. strict conditions in the 
Terms  of  Reference  of  the  project  on  requirements  for  number  of  participants,  their  role  in 
national  decision  making  and  profile/expertise,  see  section  5.3.2),  this  option  is  expected  to 
improve the situation compared to the baseline and facilitate to some extent the possibility to 
make use of each other's work. 

Some efficiency gains in the allocation of resources could be foreseen and quantified. More 
specifically,  option  2  is  expected  to  provide  modest  cost  savings  compared  to  option  1  for 
HTA bodies resulting from the voluntary production and uptake of joint REAs and full HTAs, 
estimated at a total of EUR 383 000 (EUR 256 000 for pharmaceuticals and EUR 127 000 for 
medical technologies) per year.154 

This estimation is confirmed by the results of the survey on the expectations of HTA bodies, 
which indicate that the overall costs are expected to slightly decrease/remain stable for option 
2.  The  additional  workload  caused by  the  joint  work  in  addition to  the  national  activities  is 
expected to be partly offset by the gains of the cooperation.  

154  GÖG-LSE  Study Section 7.2.3.2 While  there  are  uncertainties  in  the cost calculation,  the overall  estimates 
confirm  that  the  impact  on  costs  is  neutral/mildly  positive  for  all  options  of  continued  HTA  cooperation. 
Particular challenges include the high variation of HTA-related costs both from the HTA bodies’ and industry's 
side;  or  the  assumptions  related  to  the  uptake  of  joint  reports.  Moreover,  a  number  of  impacts  could  not  be 
quantified, e.g. the reduction of national assessment due to  better cooperation, alignment of methodology, and 
data  availability;  reduction  of  costs of  evidence generation; reduction of costs due  to  a lower number of early 
dialogues. Sensitivity analysis was conducted. 

58 

 
 
                                                            
It should be noted that in its estimate the GÖG-LSE study assumed that any new EU project 
would  be  of  similar  size  as  the  EUnetHTA  Joint  Action  (2016-2020).  The  requirements  to 
limit the number and define the profile of participants foreseen under this option may further 
optimise  cost  savings  due  to  a  more  homogenous,  smaller  and  possibly  more  efficient 
Consortium. 

The  differences  caused  by  HTA  bodies  developing  their  own  practices,  processes  and 
methodologies independently as described under option 1 are expected to remain, but slightly 
decrease under option 2 due to the project-based cooperation at EU level.  

This  option  is  expected  to  have  some  negative  impact  on  administrative  burden  resulting 
from  the  requirements  related  to  the  reporting  obligations  associated  to  EU  co-funded 
projects.  

Concerning  the  governance  system  and  the  financing,  this  option  is  expected  to  have  an 
impact on the EU budget for running the project which is estimated at EUR 5 300 100155. 

Industry 

Duplication of efforts for industry to comply with different Member States' requirements, i.e. 
for parallel assessments, early dialogues and additional evidence generation (as discussed in 
detail  in  section  2,  problem  1),  are  expected  to  continue  under  policy  option  2.  However,  a 
focused  and  efficient  project-based  cooperation,  which  has  addressed  some  of  the 
shortcoming  of  the  current  cooperation  model,  may  reduce  differences  in  approaches  and 
facilitate some  joint  reports which may then be used in  national decision-making processes. 
This persisting but slightly improved fragmentation of the internal market is in line with the 
pharmaceutical industry's own expectations as revealed by their replies to the survey carried 
out by the GÖG-LSE Study and the public consultation. 

Regarding  costs  for  the  pharmaceutical  industry  under  policy  option  2  compared  to  the 
baseline scenario, modest savings were identified for the entire pharmaceutical sector across 
the EU compared  with  the estimates under the baseline scenario.  The cost  savings resulting 
from the production of joint REAs and full HTAs are estimated at EUR 3 700 000 per year 
compared with the baseline156. This corresponds with the expectations of the pharmaceutical 
industry  which  indicated  that  option  2  could  lead  to  a  slight  reduction  of  costs  and 
administrative  burden.  On  the  other  hand,  in  the  public  consultation  and  the  EFPIA/CRA 
study it was highlighted that without mandatory uptake, the benefits of the joint work would 
not materialise for industry and therefore it would be more and more challenging to identify 
companies  which  would  be  willing  to  engage  in  voluntary  submissions  for  Joint 
Assessments157. 

Business predictability is expected to remain low under policy option 2 and the benefits in 
terms of innovation and competitiveness would not materialise. This is well in line with the 
responses provided by the pharmaceutical industry to the public consultation stating that the 
current model of cooperation based on voluntary joint work, which has limited effect on the 

155 GÖG-LSE Study Section 7.2.3.2 
156 GÖG-LSE Study Section 7.2.3.2 
157 It should be noted that under a voluntary framework of cooperation, experience has demonstrated that for 
pharmaceuticals Joint assessments are most efficiently carried out with the active engagement of the Industry, 
which however also remain voluntary.   

59 

 
 
                                                            
convergence  of  HTA  processes  and  methodologies  across  EU,  leads  to  low  business 
predictability (93%) and discourages innovation (74%). 

For the medical technologies sector, the economic impacts of option 2 would differ given that 
the  current  market  access  path  is  more  diverse  and  the  role  of  HTA  in  the  process  is 
substantially less developed than for pharmaceuticals. 

Modest  cost saving for the sector are expected compared to  the baseline scenario related to 
the preparation and submission of joint REAs and full HTAs, estimated at EUR 92 000 per 
year. According to the results of the survey conducted by the GÖG-LSE Study, the medical 
technologies sector indicated that stable/slightly increased costs are expected under option 2 
compared with the baseline scenario, but with efficiency gains in terms of administration.  

The medical technologies sector,  both the large companies and SMEs, perceives this option 
very  positively.  Industry  representatives  from  this  sector  have  expressed  support  for  a 
voluntary, non-legislative system 158 and argued in favour of a process that is demand-driven 
by  decision-makers  responsible  for  the  coverage  and  funding  of  health  technologies.  The 
medical  technology  industry  also  expects  that  option  2  would  reduce  fragmentation  and 
increase predictability, competitiveness and innovation. However, such expectations have not 
been substantiated by any specific evidence and from our analysis of the past and current EU 
cooperation on HTA, such benefits are not expected, especially taking into account the issues 
identified in section 2.  

Overall, commitment and resource investment by industry to an EU system for production of 
joint outputs are expected to remain limited, as there is no mandatory national uptake of joint 
outputs foreseen under this option, nor any obligation to industry to submit technologies for 
joint  assessment.  Continued  low  uptake  by  national  HTA  systems  is  expected  to  limit  the 
impacts  of  this  option  on  reduced  duplication  of  efforts,  increased  efficiency  gains  and 
improved business predictability for industry. This option is therefore only expected to have 
limited  positive  impact  on  improving  the  functioning  of  the  single  market  compared  to  the 
baseline. 

6.1.2.  Social impacts  

Member States/Public administrations 

As described above, joint reports such as early dialogues and joint REA would be done in the 
framework  of  a  project.  Considering  that  participation  in  the  project  is  voluntary  in 
combination  with  high  requirements  as  regards  profile  role  and  number  of  participants  and 
conditionality for uptake, it is likely that not all Member States will be able or willing to meet 
the conditions identified in the Terms of Reference/Call. This together with other hurdles to 
national uptake described above is expected to preserve the significant differences throughout 
the EU as regards the number of HTAs performed, e.g. only in some well-developed systems, 
HTA  bodies  would  be  able  to  assess  all  newly  authorised  medicines.  Therefore,  this  policy 
option  is  expected  to  bring  no  or  very  limited  benefits  on  sustainability  of  the  health 
systems and public health in general.  

In  addition,  the  limitations  in  participants  and  their  profile,  which  would  be  necessary  for 
improving the likelihood of timely output of consistent quality in a project based cooperation, 

158 Public consultation, GÖG-LSE Study 

60 

 
 
                                                            
is likely to negatively affect the inclusiveness of the option and as such not provide EU wide 
capacity building opportunities, which are particularly necessary in Member States with less 
developed  HTA  systems  which  are  often  the  ones  more  in  need  of  enhanced  sustainability. 
The role of the HTA Network in disseminating the knowledge across the EU could mitigate 
this, but only to a limited extent.  

The impact of the joint work is expected to be limited for decision-making by Member States 
due to the difficulties to enforce any contractual obligations. Also, the intermittent/disruptive 
nature of the cooperation based on a renewable project, is expected to have negative impact 
on governance as it would prevent planning and overall sustainability.   

Since, as discussed above, uptake of joint outputs in national HTA systems cannot be ensured, 
this  option  is  expected  to  have  only  limited  impacts  on  strengthening  evidence-based 
decision-making for the benefit of Member States health systems. 

Patients 

As  regards  participation  and  good  administration,  the  stakeholders'  involvement  and 
transparency would slightly improve compared with option 1. The requirement which could 
be introduced in the call for proposals/terms of reference to include patient representatives in 
the project(s) to increase their involvement in the preparation of Joint outputs, while may be 
implemented  correctly  in  Joint  output,  is  unlikely  to  have  significant  impact  on  national 
practices.  Stakeholder  involvement,  while  valued  by  many  HTA  bodies,  is  seen  as  a  major 
challenge to implement due to the resources needed. Current experience of patients and other 
stakeholders  is  that  the  Joint  Actions  did  not  sufficiently  address  the  issue  of  patients’ 
involvement  in  the  joint  HTA  evaluations.159  This  is  confirmed  by  the  results  of  the  survey 
carried  out  by  the  GÖG-LSE  Study,  showing  that  HTA  bodies  do  not  expect  stakeholder 
involvement to increase. 

The currently observed delays and divergences in the availability of innovative new health 
technologies  to  patients  across  Europe  would  likely  remain  unchanged  under  this  policy 
option.  As  emphasised  by  patients  in  the  public  consultation,  current  differences  in  HTA 
methodologies/procedures contribute to diverging outcomes of HTA reports (83% of patient 
replies) and disincentives to innovation (58% of patient replies), with negative consequences 
for the availability to patients. 

However,  while  the  Option  foresees  a  number  of  requirements  to  ensure  more  timely  and 
consistent quality output (see description in policy option 2), it should be noted that there are 
important  risks  and  issues  of  feasibility  related  to  a  project-based  cooperation  model  as 
suggested  under  this  policy  option.  The  success  of  joint  REA  would  continue  to  largely 
depend  on  the  use  of  the  report  in  national  decision-making  and  the  voluntary  industry 
submission. Therefore, there would be no guarantees for fully realising these benefits.160 As 

159 E.g. contribution to the public consultation by European Cancer Patient Coalition, European Organisation for 
Research and Treatment of Cancer (EORTC) Association Internationale de la Mutualité, European Social 
Insurance Platform 
160 While there are uncertainties in the cost calculation, the overall estimates confirm that the impact on costs is 
neutral/mildly  positive  for  all  options  of  continued  HTA  cooperation.  Particular  challenges  include  the  high 
variation of HTA-related costs both from the HTA bodies’ and industry's side; or the assumptions related to the 
uptake  of  joint  reports.  Moreover,  a  number  of  impacts  could  not  be  quantified,  e.g.  the  reduction  of  national 
assessment  due  to  better  cooperation,  alignment  of  methodology,  and  data  availability;  reduction  of  costs  of 

61 

 
 
                                                            
 
uptake of joint outputs in national HTA systems is not made mandatory under this option, the 
impacts  of  this  option  on  strengthening  evidence-based  decision-making  for  the  benefit  of 
patients will remain limited. 

As stated by EURORDIS in their contribution to the public consultation as long as  "work is 
voluntary  for  both  HTA  and  industry,  there  is  no  virtuous  cycle:  industry  may  hesitate  to 
participate  in  a  joint  assessment,  HTA  bodies  may  hesitate  to  participate  as  authors,  other 
hesitate to use the HTA reports in parts or in totality, so convincing evidence that such joint 
work is useful to all is difficult to generate." 

6.2.  Policy option 3. Permanent cooperation on common tools, 

procedures and early dialogues  

6.2.1.  Economic impacts  

Member States/Public administrations 

This  option  is  expected  to  have  a  moderate  positive  impact  for  Member  States/public 
administrations.  

For  HTA  bodies,  the  overall  costs  are  expected  to  slightly  decrease  due  to  the  joint  early 
dialogues which would largely replace national dialogues as well as the stronger convergence 
of processes and methodologies (including horizon scanning, IT tools etc.), which is expected 
to  facilitate  reuse  of  national  HTA  reports.  Such  reuse  of  national  HTA  reports  can  be 
particularly  beneficial  to  Member  States  that  have  limited  HTA  resources.  It  may  not 
primarily result in resource savings, but in an increase of quality.  

This  option  may  also  enable  further  voluntary  cooperation  on  joint  outputs  among  Member 
States outside the framework (e.g. regional cooperation with participation of a limited number 
of Member States and on specific activities), which may also contribute to some cost savings 
and more efficient use of resources for those involved.  

On the other hand, Member States would be faced with some administrative costs/burden to 
implement  and  adapt  their  systems  to  the  common  tools  and  procedures.  This  is  a  one-off 
cost/burden appearing at the start of the cooperation which is expected to affect all Member 
States but to different extent depending on the HTA rules/procedures in place at national level 
(as described in annexes VIII and IX). Member States with more detailed national rules will 
have to adapt more, but at the same time would benefit from already developed rules/policy at 
national  level  as  regards  quality,  transparency,  stakeholder  involvement  etc.  Member  States 
with a less developed system and legal framework will have to adopt new rules according to 
the proposed common rules and procedures. Considering that they have little in place this is 
not expected to be over demanding. In any case, these administrative costs are expected to be 
more  than  compensated  by  work-sharing  arrangements,  especially  through  the  facilitated 
reuse of national HTA reports due to the common tools and procedures.  

Impacts would differ across the EU depending on the current activities of the national HTA 
body.  For  instance,  for  smaller  agencies  which  currently  conduct  only  a  limited  number  of 
HTA activities, cooperation could increase the scope of their activities.  

evidence  generation;  reduction  of  costs  due  to  a  lower  number  of  early  dialogues.  Sensitivity  analysis  was 
conducted. 

62 

 
 
                                                                                                                                                                                          
It  has  to  be  noted  that  the  main  drivers  of  cost  changes  in  policy  option  3  are  difficult  to 
quantify  due  to  the  lack  of  data  (e.g.  on  the  number  of  national  early  dialogues  conducted 
across Europe), as further explained in the GÖG-LSE study. In the survey and the focus group 
discussion, the conclusion was that stronger EU cooperation would lead to a cost decrease per 
product.  Current  experience  suggests  that  sharing  the  work  decreases  the  costs  for  national 
agencies  significantly  (in  one  case  where  only  two  agencies  agreed  to  cooperate  on  clinical 
guidelines they were able to save 30% respectively).161  

Public  administrations'  response  to  the  public  consultation  confirmed  the  usefulness  of  the 
joint  tools  (responded  very  much  (75%)  or  to  some  extent  (25%)  to  their  needs)  and 
guidelines  (responded  very  much  (55%)  or  to  some  extent  (37%)  not  at  all  (8%)  to  their 
needs). 

Industry 

Under  option  3  the  expected  economic  impacts  and  their  magnitude  differ  for  the 
pharmaceutical and medical technologies sectors.  

Industry  would  face  fewer  differences  when  dealing  with  multiple  national  systems,  as  the 
tools and procedures (e.g. submission templates, data requirements, early dialogues) would be 
streamlined. This is expected to remove part of the current distortion on the internal market 
and facilitate more equal market access for health technologies throughout the EU.  

For  the  pharmaceutical  industry,  the  most  important  economic  impact  is  related  to  the 
expected  benefits  in  terms  of  predictability,  leading  to  better  innovation  and  increased 
competitiveness.  The  joint  early  dialogues  would  have  an  important  positive  impact  on 
predictability in so far as they could optimise the selection and design of the clinical trials and 
reduce the risk of investing in costly trials that do not produce relevant acceptable data for all 
HTA bodies, i.e. improve value for money.  
According  to  industry162,163,  early  dialogues  are  very  useful,  improving  transparency  on 
evidence  requirements  and  thereby  also  business  predictability.  It  represents  an  opportunity 
for  companies  to  receive  a  clear  ‘red  light’  message  on  certain  aspects  of  medicine 
development and reduce risks associated with the development of the product. Therefore, this 
exercise  can  guide  developers  to  invest  their  resources  in  viable  developments  from  both 
regulatory  and  HTA  perspectives.  Carrying  out  joint  early  dialogues  in  a  systematic  way 
would remove the possibility of parallel national dialogues with conflicting messages for the 
developers, thus contributing to a more effective design and more efficient funding of clinical 
trials which would better meet the needs of HTA bodies as well as improved capacity to bring 
innovations to market and increased competitiveness. 

The  relevance  of  early  dialogues  and  increased  predictability  are  particularly  relevant  for 
SMEs164. According to SME interviews conducted by the Deerfield Institute, SMEs confirm 
the potential of early dialogues and parallel scientific advice in reducing risks in the overall 
development  of  a  health  technology.  The  report  also  points  out  the  particular  relevance  for 
SMEs  creating  the  right  clinical  trial  design  from  the  beginning,  due  to  the  typically  less 

161 GÖG-LSE study, Annex 8 
162 EFPIA/CRA Study 
163 GÖG-LSE Study, survey and interviews Section 7.1.13 
164 Deerfield Institute. 2015. EuropaBio survey on Regulatory and HTA advice for SMEs.  

63 

 
 
                                                            
available  funding.    This  is  underlined  by  the  fact  that  that  more  and  more  SMEs  request 
parallel scientific advice with HTA. For example, in 2014 no such procedure was requested, 
whilst in 2016, 26 % of such advice was finalised for SMEs.165 

In addition, the total number of national early dialogues and the associated costs due to fees, 
are  expected  to  decrease  under  this  option.  Nevertheless,  the  actual  cost  saving  of  this 
decrease is estimated to be low due to the limited number of early dialogues currently carried 
out and their costs compared to the overall investment on the product development.166 

Also, the alignment of procedures and tools foreseen under this policy option is expected to 
improve  efficiencies  for  industry  and  reduce  the  costs  of  complying  with  multiple  systems 
under the baseline scenario.  These cost savings are  relatively  limited looking  at the broader 
perspective and would materialise once the necessary adaptations to the common format have 
been made. 

Regarding  administrative  burden,  this  policy  option  would  not  impose  any  mandatory 
obligation for industry. Early dialogues would be initiated by manufacturers and they would 
be voluntary. Streamlining tools and methodologies means that there is better alignment in the 
information that manufacturers need to provide in the national processes (i.e. reduced need to 
adapt to multiple national requirements) with some limited initial costs in order to adapt to the 
common  format.  The  alignment  is  expected  to  be  particularly  beneficial  for  SMEs  with 
limited resources and typically no national affiliates to engage with national authorities. 

In  the  public  consultation,  over  90%  of  the  respondents  from  the  pharmaceutical  industry 
(non-SMEs)  considered  joint  tools,  guidelines  and  early  dialogues  useful  to  very  much 
respond to their needs. SMEs were also positive, around 75% responding that such joint work 
would respond to their needs.  

The  positive  impacts  described  above  may  be  more  limited  for  the  medical  technologies 
industry  due  to  the  fact  that  HTA  processes  are  less  prevalent  and  in  particular  that  early 
dialogues  are  much  less  frequent  in  this  sector.  Only  28%  of  the  companies  reported 
participating  in  early  dialogues  vs  70%  of  pharmaceutical  companies,  and  even  when 
participants responded yes, they clarified that their experience was limited to one procedure.  

The  medical  technologies  industry  also  indicated  strong  concerns  that  a  legislative 
framework imposing mandatory uptake at EU level is expected to substantially increase their 
costs and administrative burden, which may have a strong negative impact on predictability, 
innovation  and  competitiveness.  Medical  technologies  representatives  interpreted  policy 
options 3-5 as (leading to) legally mandating REA (or full HTA) at the time of market launch, 
and as such they felt that it would substantially increase HTA activities in Member States for 
medical  technologies  and  fundamentally  change  the  current  business  model,  which  is  based 
largely  on  public  procurement  at  local  level.  As  clarified  in  the  description  of  the  policy 
options, option 3 is limited to cooperation on common tools, procedures and early dialogues 
and  does  not  foresee  Joint  REA  at  market  launch  for  any  technology  in  the  scope  of  the 
option. The statement that this option would fundamentally change the current business model 
of  the  sector  is  thus  unfounded.  In  addition,  it  should  be  noted  that,  in  any  case,  HTA  is 
currently being developed and broadened at national level, including on medical technologies, 

165 European Medicines Agency. SME Office annual report 2016.  

64 

 
 
                                                            
 
(see section 1.4.1) although in a divergent manner and that the joint early dialogues foreseen 
under  policy  option  3  would  only  take  place  at  the  initiative  of  industry.  Therefore  the 
concerns expressed by industry in this sector do not appear to be fully justified. This analysis 
is  also  supported  by  the  fact  that  in  the  public  consultation,  most  respondents  from  the 
medical  technologies  sector,  both  large  companies  and  SMEs,  considered  that  HTA  tools 
somewhat respond to their needs; they considered guidelines most relevant, followed by joint 
tools and early dialogues. The responses from SMEs showed more variations.   

However, it is understandable that, considering the recent changes to the Union legislation on 
medical  technologies,  which  is  still  in  the  process  of  being  implemented,  the  medical 
technologies industry is particularly sensitive to any further changes in processes which may 
have  any  impact  on  the  predictability  of  the  market  access  pathway,  their  well-established 
practices and business models. In this context it should be kept in mind that this initiative is 
envisaged post 2020. 

6.2.2.  Social impacts  

Member States/Public administrations 

For the HTA bodies, the joint  horizon scanning, joint  early dialogues and the reuse of their 
national assessments developed based on a common methodology mean that better evidence 
would  be  available  for  national  decision-making.  This  is  particularly  relevant  for  smaller 
agencies or agencies that are still developing their capacities.  

An increase in the consistency of the methods used to assess a technology through reliance on 
common tools and an expected increase in the relevance of the evidence generated via joint 
early  dialogues  would  also  have  a  positive  impact  on  the  sustainability  of  health  systems 
and ultimately public health. This is particularly important in lower income Member States 
as the opportunity costs of making a 'bad' decision are higher.167 This is well in line with the 
feedback  received  from  research  organisations  (such  as  EORTC),  and  industry  who  have 
pointed out that harmonised clinical data requirements across European HTA agencies would 
lead to a stronger expression of the European data needs and thereby ensure that requirements 
of HTA bodies and decision-makers would be adequately reflected by drug developers in the 
design of the clinical trials. 

On  governance  and  as  far  as  good  administration  is  concerned,  setting  up  a  permanent 
structure to enable HTA bodies to cooperate on a continuous basis on agreed joint outputs, is 
also expected to contribute to building capacities in Member States, ensure and efficient use 
of  the  resources  devoted  to  the  cooperation  and  ultimately  have  a  positive  impact  on  the 
quality of the output.  

Regarding  the  risks  and  issues  of  feasibility,  option  3  has  stronger  guarantees  for  the 
implementation  than  option  2  due  to  the  legal  framework  foreseeing  the  common  tools  and 
procedures. The political buy-in, particularly from Member States with well-developed HTA 
systems  is  a  key  success  factor  as  they  are  the  ones  that  might  need  to  adapt  their  systems 
more, which entails upfront investment. 

Patients 

167 Kaló, Z., Gheorghe, A., Huic, M., Csanádi, M., and Kristensen, F. B. (2016) HTA Implementation Roadmap 
in Central and Eastern European Countries. Health Econ., 25: 179–192. 

65 

 
 
                                                            
The partial removal of the current market distortion envisaged above (see economic impact on 
industry) and the resulting improved market access for health technology products throughout 
the EU would mean an improved availability of innovative health technologies for patients. 
This  option  is  also  expected  to  improve  patient  participation  to  the  HTA  process  and 
transparency. 

As  highlighted  by  patient  organisations  such  as  Eurordis  and  the  European  Patient  Forum, 
early  dialogues,  if  conducted  with  the  involvement  of  the  patients,  not  only  improve 
transparency  but  also  provide  valuable  input  to  the  discussion  with  technology  developers 
(e.g. on patient needs and preferences, relevance of particular health outcomes and quality of 
life). Patients should be aware of the plans to develop a new technology for their disease and 
have an opportunity to be part of the dialogue with the developers. Moreover, early dialogues 
can  reduce  futile  clinical  research,  and  maximise  the  chances  that  the  end  results  of  the 
development are relevant for HTA, which can then result in more timely decisions on patient 
access.168  Joint  early  dialogues  together  with  common  templates  and  common  processes 
would  bring  benefits  in  the  21  Member  States  currently  without  such  a  process,  but  also 
improve  transparency  and  patient  involvement  in  the  Member  States  where  early  dialogues 
are currently conducted at national level. Patient organisations noted in the public consultation 
that  joint  early  dialogues  would  be  more  efficient  for  them  than  participation  in  several 
national  early  dialogues,  considering  the  time,  financial  resources  and  training  needed  for 
patient participation. Joint early dialogues at European level would also give Member States 
and patients more influence in steering R&D investment decisions by industry towards health 
technologies with added value for patients. 

The use of common templates implies that elements of value for patients are also adopted in 
Member States where they are not currently captured; common processes can increase patient 
involvement and transparency. In particular, patient involvement would substantially improve 
in  over  half  of  the  Member  States  as  currently  only  12  Member  States  indicated  involving 
patients in the assessment process of pharmaceutical (11 for medical technologies).169 

6.3.  Policy option 4. Permanent cooperation on common tools and 

procedures, early dialogues and joint REA 

As explained in section 5.3.4., policy option 4 includes two sub-options:  

  Option 4.1 allowing Member States to decide if and when to start applying the joint 

REA;   

  Option 4.2 requiring all Member States to apply the joint REA. 

Option 4.1 would have the advantage of allowing Member States to select the time at which 
they  would  wish  to  join  the  system  which  would  ensure  a  strong  political  commitment  and 
willingness to participate. HTA bodies and stakeholders would have time to adapt to the new 
system,  thus  minimising  disruptions  which  are  inevitable  when  moving  from  established 
national processes to a Union approach. At the same time, this option implies a risk that some 

168  EURORDIS  public  consultation  contribution,  ,  EPF  position  paper  "Core  Principles  from  the  Patients' 
Perspective on the Value and Pricing of Innovative Medicines." 
169 Patient Involvement in Health Technology Assessment in Europe. Results of the European Patients' Forum 
Survey. 2013.  

66 

 
 
                                                            
Member States would choose to stay out for a very long time, or permanently, which could 
have  a  significant  adverse  impact  on  the  internal  market,  an  outcome  which  would  be  in 
contradiction with the underlying objective of the measure as defined. Such a risk therefore 
raises concerns as regards its legal feasibility. 

Considering,  however,  the  high  interest  in  cooperation  in  joint  REA  indicated  by  Member 
States in the public consultation and bilateral discussions as well as the wide interest from all 
Member States in the EUnetHTA Joint Action (2016-2020), it is expected that over time most 
Member States would join the system, but impacts would be spread over a longer period of 
time than under option 4.2. The opt-in approach in option 4.1 thus makes it difficult to predict 
which and how many Member States would benefit from the new system and in which time 
frame and raises the question as to whether the specific objectives of reducing duplication and 
increasing  uptake  of  joint  work  could  be  fully  met.  On  the  other  hand,  option  4.2  would 
ensure full coverage within a shorter period of time. 

As the impacts of these two sub-options are expected to be similar, they are assessed together. 
Differences  are  expected  to  be  limited  to  the  timing/moment  when  they  occur  and  the 
coverage throughout the EU. 

6.3.1.  Economic impacts 

Member States/Public administrations 

In  addition  to  the  economic  impacts  described  under  option 3,  option  4  is  expected to  have 
further positive impacts for Member States/public administrations, which are due to the joint 
production  and  mandatory  uptake  of  joint  REA  foreseen  under  this  option.  The  mandatory 
uptake foreseen under this option will be ensured by requiring Member States to use the joint 
REA in the national system in the same way as they are currently using a national REA and to 
report/notify their uptake to the Commission and to other Member States.  

The cost savings related to the joint REA have been estimated at EUR 1 560 000 per year for 
HTA bodies for option 4.1 and EUR 2 670 000 for option 4.2. This is in line with the results 
of  the  focus  group  meeting  with  public  administrations,  who  agreed  that  a  stronger  EU 
cooperation would lead to a cost decrease per joint output (once the system is well established 
and running).170 Moreover, a high quality joint REA is also expected to contribute to broader 
economic benefits for Member States which result from more efficient healthcare investments 
decisions. However, such broader economic benefits are more difficult to quantify. 

In  addition  to  the  adaptation  to  common  tools  and  procedures  foreseen  under  option  3, 
Member  States  would  be  faced  with  some  administrative  costs/burden  related  to  the 
national  participation  in  the  joint  REA.  In  the  first  phase,  this  is  expected  to  mainly  affect 
those Member States with a more developed system as experts from those Member States are 
most likely to be selected as assessors/co-assessors for the joint REA (see section 7.3.2.1. of 
GÖG-LSE study). In these cases, the joint work will replace the national assessment and be 
used  in  the  same  way  to  inform  national  pricing  and  reimbursement  decisions.  As  an 
additional benefit, national HTA bodies providing experts will be compensated for their work. 
Member  States  with  less  developed  resources  will  mainly  benefit  from  work  carried  out  by 

170  Section  7.2.3.2  in  contrast  to  this  finding,  the  survey  performed  by  GÖG-LSE  Study,  indicated  a  slight 
increase  of  costs.  Such  contradictory  results  were  discussed  in  the  focus  group  and  could  be  explained  by 
overheads and coordination costs at the beginning of the cooperation.   

67 

 
 
                                                            
others in the initial phase until they have built up own expertise. In a longer term, the work 
sharing will be more equal and expertise developed across the EU. In addition, Member States 
will need to ensure the use of the joint REA, e.g. to align processes and notify uptake. In any 
case,  these  costs/burden  are  expected  to  be  more  than  compensated  by  the  long-term  work-
sharing  arrangements  foreseen  under  this  option,  i.e.  on  joint  REA,  early  dialogues  and 
common  tools  and  procedures.  The  piloting  of  joint  REAs  under  EUnetHTA  has  already 
indicated the potential for efficiency gains from work-sharing arrangements: e.g. in one case 
reported, a national HTA assessor was able to prepare a national REA in 5 days by adapting a 
EUnetHTA  pilot  joint  REA,  whereas  preparation  of  a  national  REA  from  scratch  usually 
takes 25 days in the respective HTA body171. It is expected that these efficiency gains would 
increase further in the more streamlined joint REA preparation foreseen under option 4. The 
efficiency gain estimated here is dependent on the selection of health technologies where joint 
assessment  is  of  EU  wide/common  interest.  This  option  may  also  foresee  some  additional 
administrative  burden  for  Member  States  which  have  HTA  provisions  set  out  in  national 
legislation; these are normally the ones with more developed HTA systems. However, such a 
burden, which is inevitable when moving from established national processes to a European 
approach, is likely to be compensated for by the benefits from work sharing agreements (cost 
savings, efficiency gains) as described above, in particularly for the Member States with more 
developed HTA systems, as they are the ones most likely to perform Joint Work, at least in an 
initial phase of the cooperation. 

While there are efficiencies to be attained for HTA bodies, this option is not likely to lead to 
an overall reduction in employment, as existing staff would be able to engage in further HTA 
related activities (including economic assessments) that are relevant at national/regional level. 
As indicated in the survey carried out by the GÖG-LSE Study, participating HTA bodies even 
expect  that  the  increase  in  output  production  and  the  mandatory  uptake  of  REA  would  be 
accompanied by an increase in staff. 

The  extent  and  timing  of  these  impacts  would  vary  between  sub  option  4.1  and  4.2  in  the 
sense that they would occur at a slower pace in option 4.1 with the risk of never reaching the 
full impact described, contrary to option 4.2. Option 4.1 would allow Member States to decide 
when, and even if, to join the system. However, a strict implementation of option 4.2 may not 
fully accommodate Member State requests for time to adapt their national HTA processes to 
the new EU system. 

Industry 

Both sub option 4.1 and 4.2 are expected to bring further positive economic impacts for the 
industry compared with option 3, in particular for the pharmaceutical sector. 

For the pharmaceutical industry, the introduction of joint REA introduced under options 4.1 
and 4.2 would greatly reduce parallel clinical assessments for pharmaceuticals, as joint REAs 
are  produced  jointly  and  taken  up  at  national  level,  thereby  contributing  to  improved 
timeliness  and  convergence  of  outcomes  of  national  HTA  assessments.  This  is  expected  to 
further  reduce  the  current  internal  market  distortion  and  improve  market  access  for 
innovative health technologies. Whilst improved conditions for the functioning of the single 
market are expected under both policy options 4.1 and 4.2, it should be noted that considering 
the opt-in possibility under option 4.1 there is no guarantee under this option– in contrast to 

171 Example from presentation by Wim Goettsch (CAPR Meeting, Malta, 2017) 

68 

 
 
                                                            
option  4.2  –  that  the  internal  market  distortion  would  be  significantly  reduced  across  all 
Member States. 

The  joint  REA  is  also  expected  to  result  in  cost  savings  estimated  for  policy  option  4.1  at 
EUR  35  000  000  and  for  policy  option  4.2  at  EUR  64  000  000  annually  for  the  sector.  172 
Scope for savings related to a joint REA was confirmed in the focus group meetings, where it 
has been estimated that a joint report could recover 20-25% of the local HTA costs if there is 
no requirement for translation/adaption. Also, no fees are foreseen for the joint REAs. Costs 
of national submissions are  also  expected to  slightly decrease  due  to  aligned methodologies 
and tools (see option 3). 

However, companies would  still have to address national requirements not  pertaining to the 
EU cooperation (i.e. provide information for the assessment of non-clinical HTA domains), as 
well  as  national  reimbursement  procedures;  and  there  are  costs  related  to  the  production  of 
joint  REA.173  These  potential  savings  for  industry  are  minor  if  considered  in  the  broader 
context of R&D for pharmaceuticals. The overall drug development per drug is estimated at 
EUR 1 926 000 000. 174 Overall, the pharmaceutical industry does not expect any significant 
changes  in  their  current  costs,  as  confirmed  by  both  the  HTA  GÖG-LSE  Study  (baseline, 
interviews,  study,  focus  group)  and  the  EFPIA/CRA  study.  In  their  view,  the  benefits  from 
this  option  would  manifest  themselves  in  other  indicators  (such  as  business  predictability) 
rather than costs related to the production of HTA submissions (see below). 

Industry  

Joint REA  

Joint Early Dialogues 

Additional evidence 
generation following joint 
early dialogues 

Estimated impacts 

  Savings due to single Joint REA per product estimated at   

EUR 35 117 000 (option 4.1) and EUR 63 833 000 (option 4.2)175  
  Costs of national submissions can slightly decrease due to aligned 

 

 

methodologies and tools 
Improved timeframe for market access for certain countries  

Improved predictability, potentially very limited saving, one 
central process could replace more national processes (~ option 3) 

 

Improved predictability, no cost change (~ option 3) 

Table 7. Summary of economic impacts for pharmaceutical industry option 4 (source: GÖG-
LSE Study) 

A more important positive impact for the pharmaceutical industry relates to the timelines for 
market  access.  The  CRA/EFPIA  study  found  that  within  a  sample  of  16  countries,  the 
availability of joint REA at the time of marketing authorisation can speed up market access in 
12 of them from 2 to 6 weeks. The impacts of this 2-6 weeks of earlier access on a group of 
originator  pharmaceuticals  launched  in  Q2  2008  (see  Annex  VIII)  was  estimated.  Results 
show that this (one-month) earlier access for innovative products launched in one quarter can 
mean EUR 130 315 010 additional revenue over 12 years; which is a 1 % increase. A similar 

172 Estimations for 30 EDs, 40 REAs, without industry fees.  
173 Costs of joint REA for industry is estimated at EUR 140 000.  
174 DiMasi et al, Journal of Health Economics, January 2016  
175 GÖG-LSE Study. Cost estimations for 40 joint REAs for pharmaceuticals/year 

69 

 
 
 
                                                            
gain can be expected for other innovative products launched. In the long run, this may reduce 
differences  in  access  to  market,  improve  the  functioning  of  the  internal  market  and  have  a 
positive  impact  on  business  predictability,  competitiveness  and  innovation.176  Accessing 
first  markets  quickly  is  particularly  relevant  for  SMEs  that  depend  on  fast  access  to  first 
revenues. Again, the uncertainty of full coverage and timeframe under sub option 4.1 needs to 
be taken into account compared with sub option 4.2. 

It should be noted that, less heterogeneity among EU markets would not necessarily translate 
immediately into higher revenues since the negotiations on the pricing and reimbursement of 
pharmaceuticals  will  still 
to 
pharmaceuticals is not expected to increase.  

the  overall  public  budget  allocated 

take  place  and 

Despite the efficiencies it is unlikely that in-country or central HTA-related human resources 
would  be  reduced,  although  it  may  be  necessary  to  relocate  staff  to  a  central  level,  so  no 
overall impact on employment is expected. 177  

In  the  public  consultation,  the  vast  majority  of  pharmaceutical  industry  respondents 
considered  joint  REA  very  useful  and  indicated  a  preference  for  mandatory  uptake.  SMEs 
were slightly less positive, still over 75% considered joint REA very useful or useful. 

By  contrast,  representatives  of  the  medical  technologies  industry  anticipate  significant 
negative  economic  impacts  if  there  is  a  legally  mandated  joint  REA  at  the  time  for  market 
launch. The representatives of this sector argue that a joint REA at time of launch would not 
support  decision-making  but  would  delay  market  access.178  If  HTA  is  mandatory  at  market 
launch, this would negatively affect innovators, the 'first movers', because they are obliged to 
generate  comprehensive  evidence.  As  innovations  in  medical  technologies  are  incremental 
and do not receive the same patent protection as pharmaceuticals, the early followers can use 
the generated evidence and enjoy the benefits of quicker market access.  Thus, a situation is 
created  where  the  first  mover  has  a  considerable  disadvantage,  with  a  potential  negative 
impact on innovation.179  

Moreover,  due  to  the  currently  limited  role  of  HTA  before  market  access  of  medical 
technologies, the extent of current duplications (therefore the scope for efficiencies) is more 
limited  than  for  pharmaceuticals.  However  as  described  in  section  2  (problem  2),  current 
trends  for  increased  use  of  HTA  for  medical  technologies  may  increase  the  scope  for 
efficiency gains also on this sector. The cost calculation estimated little potential for savings 
for the medical technologies industry due to reduction of duplication: EUR 3 000 000 (option 
4.1)  and  EUR  7  000  000  (option  4.2)  annually  for  the  sector.  It  is  unlikely  that  in  the  first 
years  of  collaboration  the  number  of  joint  assessments  would  reach  25  so  even  this  modest 
saving  would  be  reduced.  Similarly  to  the  pharmaceutical  sector,  these  costs  are  not 
significant compared with the size of the sector. 

It should be noted that the GÖG-LSE study team concluded in the context of its overall study 
findings  that  some  of  the  anticipated  negative  impacts  expressed  by  medical  technologies 
industry representatives seem to be overestimated, which may reflect the lower level of actual 

176 GÖG-LSE Study. Section 7.2.3.2. 
177 GÖG-LSE Study Survey, focus group interviews Annex VI-VII.  
178 Online Public consultation report. Strengthening of the EU cooperation on Health Technology Assessment. 
2017. 
179 GÖG-LSE Study Focus group interviews Annex VI-VII. 

70 

 
 
                                                            
experience with  HTA  among  respondents  from  the  medical  technologies  industry  compared 
with  respondents  from  the  pharmaceutical  sector  where  HTA  is  already  more  established.  
Also,  the  medical  technologies  industry  seems  to  challenge  conducting  HTA  as  such,  not 
specifically  joint  EU  assessments  and  as  explained  above  (see  also  section  7.3.4.7  of  the 
GÖG-LSE study), the mapping shows an increase of national HTA on medical technologies 
regardless  of  an  EU  initiative  or  not.  Furthermore,  the  medical  technologies  industry 
statement mainly relates to concerns with mandatory HTA at market launch.  

In  addition,  there  are  reasons  to  believe  that,  contrary  to  what  is  suggested  by  industry  as 
referred  to  above,  a  joint  REA  on  medical  technologies  can  have  benefits  in  terms  of 
innovation as it may rationalise investment decisions for developers, clarify the EU-wide data 
needs,  reduce  uncertainties  as  regards  procedures  and  ensure  performance  of  health 
technologies. 

6.3.2.  Social impacts  

Member States/Public administrations 

Sub  options  4.1  and  4.2  are  expected  to  further  strengthen  the  positive  social  impacts  of 
option 3 for Member States/public administrations although the impacts are more certain and 
predictable under option 4.2 as under option 4.1 some Member States could choose to stay out 
in long term or permanently.  

The availability of timely and good quality joint REAs means better evidence available for the 
national  decision-making,  sustainability  of  health  systems  and  ultimately  public  health. 
Member  States  with  less  developed  HTA  systems  and/or  less  capacities  and  stronger 
pressures  on  their  health  budget  can  particularly  benefit  from  such  evidence.  Focusing  the 
joint  assessment  and  mandatory  uptake  on  the  clinical  aspects  will  avoid  duplication  and 
ensure  that  the  work  is  relevant  for  decision-makers,  while  at  the  same  time  not  interfering 
with Member States' subsequent decisions on making available certain health technologies to 
patients  or  on  pricing  and  reimbursement.  The  earlier  market  access  referred  to  above 
(economic impact on industry) would also increase the positive impact on health. 

It  would  also  allow  pooling  of  expertise,  with  potential  specialisation  of  HTA  bodies  in 
certain  therapeutic  areas  or  types  of  health  technologies,  with  a  subsequent  increase  in  the 
quality of joint outputs. 

It  is  expected  that  joint REAs  would  provide  a stronger  evidence  base for  price  negotiation 
with industry especially in Member States with less developed HTA systems. 

In terms of risks and issues of feasibility, key factors for the success of this option are (1) the 
timely and good quality joint REA and (2) the uptake of the joint work - especially as some 
well-developed HTA systems would need to make necessary adaptations. These risks would 
need  to  be  addressed  through  a  well-developed  implementation  mechanism,  the  legal 
guarantees and the monitoring and evaluation. 

Patients 

The  joint  REA  will  further  improve  the  participation  of  patients,  transparency  and 
availability of innovative health technologies compared with option 3. 

Patient involvement in joint REA can enhance the quality and relevance of the HTA report by 
improving the understanding of the impact of technologies in a real-life context (e.g. barriers 

71 

 
 
to complying with current therapy, side-effects etc.), providing input related to quality of life 
aspects and can lead to a higher accuracy in assessing the needs and preferences of patients.180 

Compared  with  the  multiple  parallel  procedures  for  patient  involvement  in  national 
assessments under option 3, joint REAs would improve streamlining of patient involvement 
by  creating  one  procedure  with  the  necessary  dedicated  resources.  This  is  also  expected  to 
lead to efficiency gains for patient representatives (e.g. in terms of time and training needs, as 
already discussed in the context of joint early dialogues under option 3).  

Regarding availability of innovative health technologies to EU patients, as explained in the 
economic impact section above, a high quality and timely joint REA has the potential to speed 
up assessment timelines and thereby reduce delays in the availability of innovative medicines. 
For  patients,  an  accelerated  access  by  2-6  weeks  can  be  important181,  because  in  many 
diseases,  earlier  therapy  is  associated  with  better  health  outcomes.  High  quality  and  timely 
evidence  on  the  added  therapeutic  value  of  a  product  provided  by  the  joint  REA  can  also 
contribute to promoting high quality and improved coherence of national full HTA reports. 

Joint REAs would also facilitate the involvement in the HTA process and awareness of HTA 
results of other relevant stakeholders such as health professionals. From the perspective of the 
health professionals, a joint REA would facilitate their access to reliable, timely and objective 
information  on  medical  technologies  and  support  them  in  taking  better  informed  decisions 
with  their  patients  on  the  best  treatment.182  Timely  uptake  of  positive  HTA  results  in 
evidence-based clinical treatment guidelines (which are often developed by scientific/learned 
societies  at  European  level)  can  further  contribute  to  facilitating  patient  access  in  clinical 
practice183. 

The  public  consultation  showed  strong  support  from  patient  organisations  to  not  only  use 
common tools and methodologies and perform joint early dialogues (see option 3), but to also 
carry out the joint REA as foreseen by option 4. 87% of respondents in the category "patient 
and  consumers"  noted  that  joint  REAs  would  very  much  meet  their  needs,  while  the 
remaining respondents in this category replied that it would to some extent respond to their 
needs.  Of  note,  some  patient  representatives  even  expressed  support  for  a  joint  full  HTA 
report, although they also recognised the inherent complexities in implementing such a joint 
full HTA. 

6.4.  Analysis of the governance structure and financing system 

6.4.1.  Description of the governance arrangements  

As indicated in section 5, the following governance arrangements have been considered:  

  Project secretariat 

  Member State secretariat  

  Central secretariat  

180 Patient Involvement in Health Technology Assessment in Europe. Results of the European Patients' Forum 
Survey. 2013. 
181 EFPIA/CRA Study 2017 
182 Standing Committee of European Doctors (CPME) public consultation  
183 Input from the European Society of Cardiology to the European Commission online public consultation 

72 

 
 
                                                            
o  established and hosted in a new EU Agency; 

o  established and hosted in an existing EU Agency;  

o  established and hosted in the European Commission.  

No  specific  governance  structure  is  foreseen  for  the  baseline  scenario  and  the  project 
secretariat is suitable only for policy option 2. Policy options 3 and 4 could, theoretically, be 
supported  by  any  of  the  remaining  structures  (a  Member  State  secretariat  or  a  central 
secretariat  with  different  locations)  although  some  of  the  governance  structures  are  much 
more adequate than others as illustrated below.  

Project secretariat 

This type of secretariat refers to a governance/coordination structure responsible for managing 
the  day-to-day  operations  of  a  project,  making  sure  the  participants  respect  their  tasks  and 
achieve their objective by agreed deadlines. A project consortium is chosen by the European 
Commission or an EU Executive Agency (depending on the funding instrument) following a 
call for tender /call for proposals and evaluation procedure. 

In  EU-funded  projects  a  project  management  and  coordination  work  package  is  considered 
pivotal for achieving the project' objectives. Its aim is to establish the management structure. 
It includes  the day-to-day  management and the quality supervision of the project as  well as 
reporting  to  the  European  Commission/Executive  Agency.  The  main  expected  result  is  to 
ensure a smooth coordination of the different steps of the project so they are realised on time 
within the budget limits and according to the predefined objectives. Another expected result is 
the  coordination  with  partners  so  they  are  properly  involved  and  regularly  updated  on  the 
implementation of the project. 

Figure 9. Coordination of an EU-funded project 

Member State secretariat 

The  secretariat  is  set  up,  hosted  and  run  by  a  national/regional  HTA  body  in  one  Member 
State for an agreed duration. 

73 

 
 
 
Figure 10 Diagram for the organisation of the Member States secretariat (based on GÖG-
LSE study) 

The Member State secretariat would be organised and have the following tasks: 

1) Administrative support 
•  Organisation of  meetings, travel arrangements  and other administrative issues relevant  to 
the  overall  coordination  and  to  the  operation  of  Member  States  representatives  and  Experts 
organised in working parties (WPs); 
•  Managing financial issues, especially important with regards to handling reimbursement of 
national experts and any other financial issues; 
•  Communication   
•  Providing  support  to  the  Member  States  representatives  overseeing  the  overall  EU 
cooperation on HTA.  

2) Scientific/technical support  

•  Support  the  production  of  outputs  (Standard  Operating  Procedures  for  identifying  and 
organising the work of experts from national authorities in WPs; provide scientific/technical 
support to authors and co-authors of the joint outputs); 
•  Quality management (both from a scientific and editorial perspective); 
•  Liaison with stakeholders (patients, industry, health professionals, academia, payers etc.).  

3) Provide IT support 

• 

intranet, communication tools, database etc. 

Central secretariat 

74 

 
 
  
 
 
 
Figure 11. Diagram for the central secretariat (based on GÖG-LSE study) 

Irrespective of  its  location  in  an  EU  agency  or  in  the  Commission,  the  tasks  of  this  central 
secretariat  would  be  similar  to  those  of  the  Member  State  secretariat.  Depending  on  the 
ambition of the policy option and foreseen outputs, it would include: 

1) Administrative support 
•  Organisation of  meetings, travel arrangements  and other administrative issues relevant  to 
the  overall  coordination  and  to  the  operation  of  the  Management  Board  and  Expert 
committees; 
•  Managing financial issues, especially important with regards to handling reimbursement of 
national experts, any industry fees (only possible if an agency), and legal aspects; 
•  Communication and 
•  Providing support to Management Board.  

2) Scientific/technical support (scientific secretariat to output-producing HTA bodies and MS 
expert Committees) 

•  Support  the  production  of  output  (Standard  Operating  Procedures  for  identifying  and 
organising  the  work  of  experts  from  national  authorities  in  Member  States  Expert 
Committees;  provide  scientific/technical  support  to  authors  and  co-authors  of  the  joint 
outputs); 
•  Quality management (both from a scientific and editorial perspective); 
•  Liaison with stakeholders (patients, industry, health professionals, academia, payers etc.);  
•  Provide support for national implementation (e.g. training). 

3) Provide IT support 

•  Submission system, intranet, communication tools, database etc. 

It  has  to  be  emphasised  that  the  secretariat  has  no  tasks  related  to  the  production  of  the 
different joint outputs, which is ensured by experts nominated by Member States HTA bodies 
organised in Committees. 

As  regards  human  resources,  the  staffing  depends  on  the  type  and  number  of  planned  joint 
outputs, as shown below and thus also depends on the policy option chosen. 

Central 
Secretariat 
(PO3)  

  Central coordination management - Total 14 FTE 

-  Head (1 FTE) 
-  Administrative support (total 4 FTE) 

75 

 
 
 
 
 
Central 
Secretariat 
(PO4.2) 

o  Head of administration (1 FTE)  
o  Project Manager (1 FTE) 
o  Administrative staff (2 FTE) 

-  Scientific/technical support (total 6 FTE) 

o  Head (1 FTE)  
o  Scientific officers (2 FTE) 
o  Methodology, guidelines, templates (2 FTE) 
o  Administrative staff (1 FTE) 
IT (total 3 FTE) 
o  Internal support l (1 FTE) 
o  Maintenance of tools and databases (2 FTE) 

- 

  Output production contracted to HTA bodies  
  Central coordination management - Total 34,5 FTE  

  Head (1 FTE) 
  Administrative support (total 11 FTE) 
o  Head of administration (1 FTE)  
o  Project Manager (4 FTE) 
o  Administrative (6 FTE) 

-  Scientific/technical support (total 18,5 FTE) 

o  Head (1 FTE)  
o  Scientific officers (9,5 FTE) 
o  Methodology, guidelines, templates (2,5 FTE) 
o  Administrative (5,5 FTE) 
IT support internal (4 FTE) 
o 
Internal support (1,5 FTE) 
o  Maintenance of tools and databases (2,5 FTE) 

- 

  Output production contracted to HTA bodies  

Table 9. Characteristics and staff of the central secretariat as estimated for policy options 3 
and 4.2. (based on GÖG-LSE study, for 65 joint REA/year) 

6.4.2.  Feasibility and efficiency of the governance arrangements  

In  order to choose the most appropriate  solution, the pros and cons  of the above mentioned 
governance  arrangements  are  described  below,  including  their  feasibility  to  support  the 
various policy options assessed within this Impact Assessment. 

Project secretariat 

Pros: 
-  It  is  an  adequate  mechanism  for  running  projects  with  defined  objectives  for  a  limited 
duration in time.    
- Allows for flexibility, does not oblige Member States to commit for a long period of time. 

Cons: 
- Contractual obligations cannot guarantee uptake of the results/outputs because they do not 
supersede national legal provisions. 
- Does not provide guarantees for full EU geographic coverage.  
- Member States have limited influence in prioritisation of projects and determining scope.  

Conclusions:  

 

It is suitable only for option 2. 

Member State secretariat 

76 

 
 
 
 
 
 
Pros: 
- It is close to national expertise and processes and can ensure a strong Member State driven 
approach.  
-  Staff  from  HTA  bodies  has  experience  with  national  HTA  processes  and  the  needs  for 
national decision-making. 

Cons: 
- Uncertainties/challenges related to hosting (location, decision mechanism for the nomination 
of the hosting Member State).  
- Uncertainties/challenges related to funding (recurrent contribution from EU budget).  
- Uncertainties/challenges related to the possibility of collecting and redistributing fees from 
industry to other HTA bodies and to enforce and ensure uptake will persist. 
-  Risk  to  steer  the  cooperation  towards  the  model  of  one  Member  State,  which  may  not  be 
suitable to all. 
- It received low support from stakeholders in the public consultation (see Fig. 12). 

Conclusions:  

  Particular  challenges  are  related  to  the  selection  of  the  Member  State  hosting  the 
secretariat, possible rotation, political acceptability and sustainability of financing. 
  Would be difficult to reconcile with a legal framework as suggested in option 3 and 
especially  option  4,  therefore  this  governance  structure  was  not  considered  in  the 
following section assessing the costs.184 

Central secretariat 

a) Central secretariat established and hosted in a new EU Agency 

Pros 
- An EU agency for HTA would preserve independence of HTA bodies from other influences 
(e.g. regulatory, industry) 
- Staff would be recruited in order to ensure expertise in all areas of HTA (i.e. EDs, REA, full 
HTA) and for all type of health technologies 
-  It  would  be  a  permanent  structure,  with  no  additional  administrative  burden  related  to 
renewal  of  the  coordination  structure,  and  allowing  for  a  continuous  production  of  joint 
outputs.   

Cons 
- Currently there are important political constraints not to create new EU agencies 
-  It  requires  a  longer  start-up  phase  and  higher  costs  due  to  start-up  costs  and  over  heads 
(human resources, financial etc.) 
- It requires a selection mechanism and a decision on location  
- It would be less relevant if human resources and estimated output the agency would be very 
limited in size. 

Conclusion:  

184 However, the costs were estimated in the GÖG-LSE study (section 7.2.) 

77 

 
 
 
 
 
 
 
 
                                                            
 
  Suitable for EU cooperation encompassing a broader number of activities for which 

no existing EU agency has appropriate expertise and a considerable size.  
  Allows for collection of fees in case of services provided to industry (i.e. ED) 
  Currently  not  a  feasible  governance  arrangement  due  to  important  political 
constraints, therefore this governance structure was not considered in the following 
section assessing the costs.185 

b) Central secretariat established and hosted in an existing EU Agency 
The  pros  and  cons  of  the  two  EU  Agencies  in  the  field  of  health  (i.e.  European  Medicines 
Agency/EMA and European Centre for Disease Control/ECDC) are presented below.  

EMA 

Pros 
- It is an established agency, so it would require less start-up costs 
- It has experience running Member States' expert committees with a rapporteur-co-rapporteur 
system  (similar  to  the  current  mechanism  for  carrying  out  joint  assessment  by  EUnetHTA 
with author and co-author) 
-  It  has  already  developed  well  established  cooperation  with  HTA  bodies  and  has  already 
some capacity and expertise in the area of HTA for pharmaceuticals.  
- It can collect industry fees and has a fee structure in place 
- It received some support from public consultation, especially from patient organisations 
- It could ensure synergies in the area of pharmaceuticals between regulatory and HTA issues  
- Would ensure continuous production of joint outputs 

Cons 
-  Some  Member  States  expressed  concerns  or  a  clear  opposition  (because  of  perceived 
conflict of interest between authorisation and HTA processes) 
-  The  medtech  sector  expressed  opposition  due  to  the  lack  of  mandate,  expertise  and 
experience in the field of medical devices and IVDs 
- Uncertainty over future location and future capacity 
- Requires a change in mandate for carrying out tasks in the area of HTA  

ECDC  

Pros 
- It is an established agency, so it would require less start-up costs 
- In principle ECDC could collect industry fees, but has currently no fee structure set up  

Cons 
- Requires a change in mandate for carrying out tasks in the area of HTA 
-  Need  to  expand  the  agency's  mandate  with  associated  risks  of  lengthy  discussions  on  its 
mandate or its further expansion. 
- It would require to set up a structure for collecting fees from industry (e.g. for ED) 

Conclusion:  

185 However, the costs were estimated in the GÖG-LSE study (section 7.2.) 

78 

 
 
 
 
 
 
 
 
                                                            
 
  Suitable  for  EU  cooperation  encompassing  most  types  of  joint  outputs  (i.e.  common 

tools and methodologies, horizon scanning, ED, REA).  

  Central functions are already in place. 
 
 
  Concerns expressed by some key stakeholders  

It allows for collection of fees in case of services provided to industry (i.e. ED).  
It would require changes in the mandate and staffing of both agencies. 

c) Central secretariat established and hosted in the European Commission. 
Pros 
-    It  would  avoid  the  debate  on  which  agency  should  be  more  appropriate  to  take  over  the 
HTA tasks and the discussions related to the changes of its mandate  
- It is an honest broker 
- It has experience with running Member States expert Committees and scientific Committees 
(e.g.    Scientific  Committee  on  Health,  Environmental  and  Emerging  Risks/SCHEER, 
Scientific Committee on Consumer Safety/SCCS) 
-  Several  DGs  already  employ  staff  with  scientific  profile186  (e.g.  DG  JRC,  SANTE,  RTD, 
CNECT)  and  experts  from  Member  States  HTA  bodies  could  be  seconded  for  an  agreed 
period of time. 
- It has broad support from the public consultation (see Figure 12) 
-  It  would  have  the  necessary  infrastructure  to  facilitate  the  cooperation  in  relatively  short 
time.  

Cons 
- It cannot collect and redistribute fees from industry. 

Conclusion:  

  Suitable  for  EU  cooperation  encompassing  most  types  of  joint  outputs  (i.e.  common 

 

tools and methodologies, horizon scanning, ED, REA). 
It  is  a  reasonable  solution  as  long  as  human  resources  and  estimated  outputs  are  
limited  

In  addition  to  the  pros  and  cons,  the  contribution  of  the  most  feasible  governance 
arrangements  to  the  achievement  of  the  operational  objectives  is  outlined  below  (table 
10). 

Operational objectives 

Project Secretariat  Central Secretariat  

  Promote  convergence 

in 
HTA  tools,  procedures  and 
methodologies 

Possible, but with no 
guarantees 

  Reduce 

of 
efforts  for  HTA  bodies  and 

duplication 

No 

EC 
Yes 

Yes 

EU Agency 
Yes 

Yes 

186  Scientific  profiles  for  both  European  Commission  staff  and  national  experts  include:  HTA  assessors, 
pharmacists,  pharmacologists,  biologists,  doctors,  experts  in  biotechnology,  engineers  with  expertise  in  the 
development  of  medical  devices  and  in  vitro  diagnostics,  statisticians,  researchers  in  the  field  of  health 
technologies. 

79 

 
 
 
 
 
 
 
                                                            
industry 

  Ensure  the  uptake  of  joint 
outputs in Member States 
the 

long-term 
sustainability  of  EU  HTA 
cooperation 

  Ensure 

No 

No 

Yes 

Yes 

Yes. But 
collection of fees 
from industry is 
not possible  

Yes. Can contribute 
to the financial 
sustainability by 
collecting fees from 
industry. 

Table 10. The contribution of the most feasible governance arrangements to the achievement 
of the operational objectives  

6.4.3.  Input from studies and stakeholders on the feasibility and 

efficiency of the governance arrangements  

The study supporting the Impact Assessment carried out by GÖG and LSE concluded that a 
central permanent governance structure supported by legislative cooperation could overcome 
the  current  fragmentation  of  European  HTA  systems  regarding  both  HTA  processes  and 
related  outcomes.  Support  functions  can  be  more  readily  centralised  in  a  permanent 
cooperation model as compared to a temporary/short-term one. Such a secretariat is expected 
to increase the efficiency of processes and ensure greater consistency in outcomes. It would 
also enable national agencies and their experts to keep a primary focus on the scientific work 
and not on the administrative and coordination functions, which supports production of high 
quality  joint  outputs  (e.g.  organisation  of  meetings,  interaction  with  experts  from  other 
countries and/or stakeholders etc.). 

The majority of the stakeholders who contributed to the online public consultation expressed 
support for a stable, central secretariat. 

Figure 12. Analysis of the overall replies provided to the online public consultation regarding 
the governance mechanism of the future EU cooperation on HTA 

In relation to the governance model, representatives of public administrations responding to 
the  public  consultation  emphasised  the  importance  of  separating  the  regulatory  and  HTA 
functions and ensuring the independence of HTA agencies. Many respondents indicated that a 
structure/unit to support HTA at EU level could be seen as a practical solution, especially if 
EUnetHTA structures and tools (such as POP database, intranet) could be easily incorporated. 
While some respondents were against the creation of a new EU agency, others expressed their 

80 

 
 
 
preference for this governance mechanism which would better reflect the specific needs of the 
HTA sector, with competencies clearly and transparently defined.  

Academia (e.g. EORTC) and patients' representatives (e.g. EURORDIS, EPF) advocated for 
a  centralised  HTA  system,  similar  to  the  central  marketing  authorisation  model  involving 
EMA,  in  order  to  ensure  harmonised  assessment  of  new  technologies,  especially  if  it 
addresses  the  clinical  assessment.  This  system  should  entail  permanent  administrative  and 
technical staff interacting with  standing  committee(s) of Member States. The system should 
benefit  from  strong  governance  and  appropriate  resources,  ensuring  its  independence  and 
guaranteeing high scientific standards developed and agreed by Member States experts. The 
high-quality  and  transparency  of  the  assessments  should  be  maximal,  and  stakeholders 
including clinicians, patients and industry, should be involved through appropriate permanent 
mechanisms  regulated  by  solid  and  well  defined  conflict  of  interest  provisions,  which  are 
considered key for the successful implementation of any of the policy options. A centralised 
system  was  seen  as  the  appropriate  mechanism  for  ensuring  adequate  funding  for  patients' 
involvement  (e.g.  training  activities,  developing  methods  to  obtain  relevant  patients'  views, 
coordination activities, and contribution to guidelines development. 

Representatives  of  pharma  industry  (e.g.  EFPIA,  EuropaBio,  Leem  -  Les  Entreprises  du 
Medicament) emphasised that any secretarial/organisational support function should be based 
on  high  scientific  standards,  should  receive  appropriate  resources,  and  joint  scientific 
assessments should be carried out by committee(s) of Member States experts.  

Representatives  of  the  medical  technologies'  industry  observed  that  setting  up  a  new  EU 
agency does not seem feasible and, while EMA is a good model for a successful agency in the 
field  of  pharmaceuticals,  due  to  its  limited/lack of  expertise  would  be  an  inappropriate  host 
for the EU cooperation on HTA on medical technologies. In this context, an existing structure 
within the European Commission was seen as a potential solution for providing support from 
a secretarial and organisational point of view.  

6.4.4.  Costs related to the governance arrangements 

Irrespective of the governance arrangements, two types of costs were estimated and analysed. 
This section describes first the running costs followed by the description of the costs directly 
related to the joint outputs. The following section refers to costs when the system is fully 
operational.  

Running costs 

The key drivers of the running costs are the following:  

-  The scope and number/volume of joint outputs foreseen in the policy options. These 
costs would mainly be related to the services provided directly to experts drafting the 
joint reports (e.g. committees for early dialogues and/or Joint REAs); 

-  The  geographical  location  of  the  secretariat.  Depending  on  the  price  level  of  the 
location, the costs of the central secretariat can change by 30%, driven mainly by the 
cost of premises and the indexation of the salaries; 

-  The  size  of  the  secretariat;  a  smaller  secretariat  would  typically  have  higher 

overheads; 

81 

 
 
Table  11  summarises 
mechanism(s). 187 

the  running  costs  per  policy  option(s)  and 

implementation 

Table 11. Summary of running costs188. All costs are compared to the baseline scenario which 
entails only minor expenses from EU budget (i.e. financing on average of two meetings of the 
HTA Network per year – approximately EUR 120 000). 

Cost of the joint outputs 

The costs of the joint outputs range are presented in Table 12. 

187 Option 2 was not included in the tables summarising the costs, because it does not have an overall governance 
arrangement. A new EU Agency as potential governance arrangement was also discarded based on the political 
constraints mentioned in section 6.5.2.3.  
188 Adapted from tables 20, 52, 53 in the GÖG-LSE Study 

82 

 
 
 
 
 
                                                            
Table  12.  Summary  of  joint  outputs'  costs  per  POs  and  implementation  mechanism189.  All 
costs are compared to the baseline scenario which does not include EU budget earmarked for 
production of joint outputs (i.e. cost for joint outputs = 0). 

These costs are presented separately from the running costs of the secretariat for two reasons:  

- These  costs depend  on the number of joint  outputs  estimated to  be carried out  under each 
policy option and are therefore directly linked to the policy option; 

- These costs correspond to a fully operational system, with a broad scope and a high number 
of joint  outputs  (i.e. ED, REA).  In  practice it is expected that these costs will not reach the 
values presented in the table from the beginning, but will gradually increase to the maximum 
foreseen in the calculation as the cooperation produces the foreseen number of joint products.   

The costs of the joint outputs include also the remuneration of HTA bodies carrying out the 
joint  work (i.e.  joint  REA,  joint  early  dialogues)  as  authors, co-authors  and  reviewers.  Fees 
from  industry  could  be  foreseen  to  cover  part  of  these  expenses  (i.e.  for  early  dialogues) 
depending on the governance structure chosen. However, the proportion and the mechanism 
of  the  industry  fees  should  be  carefully  considered  to  prevent  any  conflicts  of  interest  and 
guarantee the scientific independence of the work.190 

189 Adapted from tables 20, 53 and 56 in the GÖG-LSE Study 
190 Fees for joint assessments are not foreseen in the first stage as it has been considered disproportionate to the 
relatively limited size of the structure envisaged. However, it is suggested to evaluate the situation after a certain 
period of time to consider if fees would be appropriate. A dedicated impact assessment and appropriate proposal 
would be foreseen to examine industry fees.   

83 

 
 
 
 
                                                            
Overall costs 

The  overall  costs  (adding  the  running  to  the  joint  outputs'  costs)  are  presented  in  Table  13 
below. 

Table 13. Summary of the overall costs per POs and implementation mechanism. All costs are 
compared  to  the  baseline  scenario  which  entails  only  minor  expenses  from  EU  budget  (i.e. 
financing on average of two meetings of the HTA Network per year – approximately EUR 120 
000), with no EU budget allocated for production of joint outputs (i.e. cost for joint outputs = 
0). 

7.  Comparing policy options 

The  policy  options  presented  above  are  compared  against  the  criteria  of  effectiveness  (the 
extent to which the option would achieve the objective), efficiency (balance between costs vs 
benefits) and coherence (with the overarching objectives of EU policies).  

84 

 
 
 
 
 
General 
Objectives 
Specific  
Objectives 

Operational 
Objectives 

Policy  
Option 1 
(baseline) 

No Joint 
Actions after 
2020 

Effectiveness 

Ensure better functioning of the internal market 
Contribute to a high level of human health protection 
Improve the availability of innovative health technologies for EU patients 
Improve business predictability 
Ensure efficient use of resources and strengthen the quality of HTA across the 
EU 
Promote 
convergence in 
HTA 
Procedures 
methodologies 
- - - 
Persisting or even 
increasing 
divergences. 

Ensure long term 
sustainability of 
EU HTA 
cooperation 

Reduce 
duplication of 
efforts for HTA 
bodies Industry 

Increase the 
uptake of joint 
output in MS 

N/A 
No joint outputs  

- - - 
Duplication of 
work, parallel 
national/regional 
processes 

- - - 
EU cooperation is 
limited to the high-
level strategic 
policy discussions 
within HTA 
Network. No 
comprehensive 
scientific and 
technical EU 
cooperation is 
foreseen. If 
cooperation takes 
place, is expected 
between cluster of 
MS. 

Efficiency 
(benefit to cost) 

Subsidiarity and 
Proportionality 

Coherence 
- A deeper and fairer 
internal market 
- Support health systems 
- Foster research and 
innovation 

- - - 
High costs (parallel 
systems) with no or 
limited benefit for both 
HTA bodies and 
industry. 

- - - 
Fragmented internal 
market. 
Low business 
predictability. No positive 
stimulus for innovation or 
EU competitiveness. 
No support to health care 
systems to address 
challenges. 

- - 
MS cannot address 
current fragmentation 
at national level. 
The output of the 
national effort would 
not be proportionate to 
the investments. 

85 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
General 
Objectives 
Specific  
Objectives 

Operational 
Objectives 

Policy 
Option 2 

Project-based 
cooperation 
on HTA 
activities 

Effectiveness 

Reduce 
duplication of 
efforts for HTA 
bodies Industry 

Ensure better functioning of the internal market 
Contribute to a high level of human health protection 
Improve the availability of innovative health technologies for EU patients 
Improve business predictability 
Ensure efficient use of resources and strengthen the quality of HTA across the 
EU 
Promote 
convergence in 
HTA 
Procedures 
methodologies 
0 
Some convergence 
on methodologies 
may appear but 
differences in 
assessment practices 
and their outcomes 
are expected to 
remain. 

- - 
Limited uptake of 
joint assessments 
expected to 
persist as no 
enforcement is 
foreseen. 

- -  
No long term 
sustainability is 
ensured as the 
approach is project-
based. 

Ensure long term 
sustainability of 
EU HTA 
cooperation 

Increase the 
uptake of joint 
output in MS 

- -  
Some pooling of 
resources may 
appear but 
duplication of 
work due to 
parallel 
national/regional 
process is 
expected to 
continue. 

Efficiency 
(benefit to cost) 

Subsidiarity and 
Proportionality 

Coherence 
- A deeper and fairer 
internal market 
- Support health systems 
- Foster research and 
innovation 

- - 
Limited benefits for 
patients, industry and 
health care systems due 
to the scattered approach 
to the cooperation and 
limited use of joint 
assessments at national 
level. 

- - 
Fragmented internal 
market for health 
technologies persists. 
Business predictability is 
expected to remain low as 
well as the stimulus to 
innovation and 
competitiveness.  
Support to health care 
systems remains 
inefficient. 

- - 
MS cannot fully 
address current 
fragmentation through 
a project-based 
approach. 
The necessary 
resources invested are 
not expected to be 
proportionate to the 
output. 

86 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Efficiency 
(benefit to cost) 

Subsidiarity and 
Proportionality 

Coherence 
- A deeper and fairer 
internal market 
- Support health systems 
- Foster research and 
innovation 

+ 
Patients are expected to 
benefit from some 
convergence in HTA 
methodology and also 
improved participation 
in the HTA process. 
For HTA bodies' better 
evidence is available and 
more efficient use of 
resources. For industry 
improved predictability. 
Slight costs savings 
expected. 

+ 
Moderate positive 
performance concerning 
the contribution of this 
option to a fairer internal 
market of health 
technologies.  and patients 
are expected to benefit 
from it. Business 
predictability is expected 
to improve Health care 
systems benefit from better 
quality evidence. 

+ 
This option provides 
for a pooling of 
expertise and resources 
in the area of common 
tools, procedures and 
EU early dialogues, 
providing an EU added 
value to MS activities. 

General 
Objectives 
Specific  
Objectives 

Operational 
Objectives 

Policy 
Option 3 

Permanent 
cooperation 
on:  
common 
tools  
procedures 
early 
dialogues 

Effectiveness 

  Ensure better functioning of the internal market 
  Contribute to a high level of human health protection 
 

Improve the availability of innovative health technologies for EU 
patients 
Improve business predictability 

 
  Ensure efficient use of resources and strengthen the quality of HTA 

across the EU 

Promote 
convergence in 
HTA 
Procedures 
methodologies 
+ 
Ensured 
convergence in 
terms of tools and 
procedures used by 
EU MS. 

Reduce 
duplication of 
efforts for HTA 
bodies Industry 

+ 
EU MS can 
benefit/rely on 
each other's 
assessments in an 
easier way due to 
the use of 
common tools and 
procedures. 
However, 
duplication of 
work due to 
parallel 
national/regional 
HTA is expected 
to remain 

Increase the 
uptake of joint 
output in MS 

++ 
Mandatory 
uptake by HTA 
bodies of 
common tools, 
procedures and 
conclusions of 
joint early 
dialogues is 
ensured. 

Ensure long term 
sustainability of 
EU HTA 
cooperation 

+++ 
Long term 
sustainability is 
ensured by the 
permanent structure 
and the stable 
funding from EU 
budget + MS kind 
contribution.  

87 

 
 
 
 
 
 
 
 
 
 
 
 
  
 
 
 
 
 
 
General 
Objectives 
Specific  
Objectives 

Operational 
Objectives 

Policy 
Option 4.1 

Permanent 
cooperation on:  
common tools  
methodologies 
early dialogues 
joint REA (MS 
opt-in) 

Effectiveness 

  Ensure better functioning of the internal market 
  Contribute to a high level of human health protection 
 

Improve the availability of innovative health technologies for EU 
patients 
Improve business predictability 

 
  Ensure efficient use of resources and strengthen the quality of HTA 

across the EU 

Reduce 
duplication of 
efforts for HTA 
bodies Industry 

++ 
No duplication of 
work expected 
between the 
participating MS. 
However absence 
of duplication 
cannot be ensured 
if some EU MS 
do not join.  

Increase the 
uptake of joint 
output in MS 

++ 
Mandatory 
uptake by HTA is 
ensured for 
participating MS 
but does not 
cover the MS not 
joining. 

Ensure long term 
sustainability of 
EU HTA 
cooperation 

+++ 
Long term 
sustainability is 
ensured by the 
permanent structure 
and the stable 
funding from EU 
budget + MS in 
kind contribution.  

Promote 
convergence in 
HTA 
Procedures 
methodologies 
++ 
Ensured 
convergence 
between the MS that 
opt-in. 
Lower risk of 
divergent outcomes, 
therefore business 
predictability 
increases and 
ultimately patients 
will benefit from the 
availability of HTA. 

88 

Efficiency 
(benefit to cost) 

Subsidiarity and 
Proportionality 

Coherence 
- A deeper and fairer 
internal market 
- Support health systems 
- Foster research and 
innovation 

++ 
This option provides 
for a pooling of 
expertise and resources 
providing an EU added 
value to MS activities 
in the area of HTA. 
However the full 
potential of EU 
cooperation is 
exploited in a limited 
manner. 

++ 
Patients from 
participating EU MS are 
expected to benefit from 
an improved availability 
of innovative health 
technologies and also 
improved participation in 
the HTA process. 
For HTA bodies' better 
evidence is available and 
more efficient use of 
resources. For industry 
improved business 
predictability. 
Costs savings expected 
for the participating MS. 

++ 
Moderate positive 
performance concerning 
the contribution of this 
option to a fairer internal 
market of health 
technologies depending on 
the number of EU MS 
participating and patients 
are expected to benefit 
from it. Business 
predictability is expected 
to improve according to 
the number of EU MS 
participating. Health care 
systems of EU MS 
participating benefit from 
better quality evidence and 
efficiency gains. 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
General 
Objectives 
Specific  
Objectives 

Operational 
Objectives 

Policy 
Option 4.2 

Permanent 
cooperation on:  
common tools  
methodologies 
early dialogues 
joint REA  
(all MS from the 
start) 

Effectiveness 

  Ensure better functioning of the internal market 
  Contribute to a high level of human health protection 
 

Improve the availability of innovative health technologies for EU 
patients 
Improve business predictability 

 
  Ensure efficient use of resources and strengthen the quality of HTA 

across the EU 

Increase the 
uptake of joint 
output in MS 

+++ 
Mandatory 
uptake by HTA 
bodies is ensured.  

Reduce 
duplication of 
efforts for HTA 
bodies Industry 

+++ 
No duplication of 
work. Efficient 
pooling of 
resources and 
expertise. 
Expected increase 
of quality of 
HTAs. 

Ensure long term 
sustainability of 
EU HTA 
cooperation 

+++ 
Long term 
sustainability is 
ensured by the 
permanent structure 
and the stable 
funding from EU 
budget + MS in 
kind contribution + 
industry fees for 
early dialogues.  

Promote 
convergence in 
HTA 
Procedures 
methodologies 
+++ 
Ensured 
convergence in 
HTA procedures 
and methodologies 
in all EU MS. 
No risk of divergent 
outcomes for the 
clinical assessment, 
therefore business 
predictability 
considerably 
improves and 
ultimately patients 
will benefit from the 
availability of HTA. 

Efficiency 
(benefit to cost) 

Subsidiarity and 
Proportionality 

Coherence 
- A deeper and fairer 
internal market 
- Support health systems 
- Foster research and 
innovation 

+++ 
This option provides 
for a pooling of 
expertise and resources 
providing an EU added 
value to MS activities 
in the area of HTA. 

+++ 
EU Patients: improved 
availability of innovative 
health technologies and 
also improved 
participation in the HTA 
process. 
For HTA bodies: better 
evidence is available, 
efficient allocation and 
use of resources 
/expertise. For industry 
business predictability 
considerably improves. 
Costs savings are 
expected. Benefit to cost 
ratio is expected to be 
the most advantageous 
compared to the other 
options. 

+++ 
Positive performance 
concerning the 
contribution of this option 
to a fairer and deeper 
internal market of health 
technologies and EU 
patients are expected to 
benefit from it. 
The identified obstacles 
impeding a well-
functioning internal 
market are addressed. 
Business predictability is 
expected to improve. 
Health care systems of EU 
MS will benefit from 
better quality evidence and 
efficiency gains. 

Table 14. Comparison of policy options 

89 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
Legend: 
Significant positive performance 
Positive performance 
Moderate positive performance 
Neutral 
Moderate negative performance 
Negative performance 
Significant negative performance 

+ + +  
+ +  
+ 
0              
 - 
 - -   
- - -  

+ 

++ 

+++ 

PO 
1 
2 
3 
4.1 
4.2 
5 
Table 15. Overall scoring (Stars indicate the number of positive and negative scores the option has received) 

* 
* 
******* 

- - 
* 
****** 

* 
****** 

***** 

* 

0 

- 

- - - 
***** 

n/a 
* 

******* 

Total 
-17 
-12 
+10 
+15 
+21 

90 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
8.  Preferred policy option 

8.1.  Description of the preferred option 

As illustrated in  section 7, option 4.2 receives the highest  scores  when comparing  the other 
options.  However,  this  option  implies  a  certain  risk  considering  the  view  of  some  Member 
States  that  they  need  adequate  time  to  adapt  to  the  system.  This  has  been  addressed  by 
integrating  elements  from  other  policy  options  (in  particular  policy  option  2  and  4.1)  and 
allowing for some adjustments based on the assessment carried out in section 6 and comments 
received from stakeholders. Such adjustments are discussed in more detail below. 

8.1.1.  Joint outputs 

The preferred option comprises all types of joint outputs related to clinical aspects of HTA, 
i.e.  common  tools/procedures,  joint  early  dialogues  and  joint  REAs  (see  option  4  and  more 
detailed descriptions of the different types of outputs in sections 5.3.3 and 5.3.4). 

8.1.2.  Technology scope 

Pharmaceuticals 

The preferred option defines the scope of joint REAs as follows: 

Pharmaceuticals undergoing the central marketing authorisation procedure which in addition 
meet one of the following criteria: 
  new products containing new active substances 
  existing products for which the marketing authorisation is extended to a new therapeutic 

indication 

The  timing  of  the  procedure  for  joint  REAs  will  be  linked  to  that  of  the  central  marketing 
authorisation  procedure  (i.e.  the  joint  REA  will  be  available  at  the  time  of  or  shortly  after 
marketing authorisation), ensuring timeliness for supporting Member States decision-making 
at  the  time  of  market  launch.  This  approach  is  consistent  with  current  HTA  timelines  in 
Member  States,  i.e.  around  or  shortly  after  the  time  of  marketing  authorisation  of 
pharmaceuticals (see section 1.4, Figure 3). 

The  scope  takes  into  account  the  level  of  current  duplication  among  Member  States  HTA 
bodies  (which  is  most  prominent  around  or  shortly  after  marketing  authorisation),  the  EU 
added  value  of  a  joint  approach  and  stakeholder  views  (see  sections  8.2  and  8.3).  It  also 
respects the different remits of marketing authorisation and HTA (see section 1.3, text box), 
while supporting synergies where possible (see section 8.2, coherence). 

The preferred option will also allow for updates of joint REAs, i.e. re-assessments at a later 
point  in  time,  if  significant  additional  evidence  becomes  available  (e.g.  from  post-
authorisation studies).191 

Medical technologies 

The preferred option limits the scope of joint REAs as follows:  

191  Note  that  such  updates  of  joint  REAs  could  also  draw  on  increased  availability  of  real-world  data,  as 
explained in more detail in section 1.3, text box. 

91 

 
 
                                                            
Medical technologies in the highest risk classes192 and which in addition have been selected 
by Member States for a joint REA based on the following criteria: 
  unmet medical need 
  potential impact on patients, public health, or healthcare systems (e.g. burden of disease, 

budget impact, transformative technology) 

  significant  cross-border  dimension/  Union  wide  added  value  (e.g.  relevance  to  a  large 

number of Member States) 

Taking into account the more decentralised market access pathway for medical technologies, 
the timing of the joint REA will not be linked to the timing of the conformity assessment, i.e. 
it will not necessarily be at the time of market launch. Instead, Member States will define the 
most appropriate time point for a joint REA as part of the selection process mentioned above. 
Relevant considerations for the selection of an appropriate time point are expected to include 
market access of the technology in a significant number of Member States and availability of 
evidence for HTA purposes. 

This limited scope for medical technologies takes into account the level of current duplication 
among Member States HTA bodies, the EU added value of a joint approach and stakeholder 
views and concerns (see sections 8.2 and 8.3). 

Other technologies 

For other technologies (i.e. pharmaceutical and medical technology products not covered by 
the  above-described  scope,  or  other  health  technologies),  the  legislative  framework  will 
include provisions for voluntary cooperation. Production and uptake of joint outputs related to 
these  technologies  would  be  fully  voluntary,  but  Member  States  could  benefit  from  the 
organisational  framework  (e.g.  committee  and  secretariat  structures)  established  by  the 
legislation.193 

By  leaving  joint  work  on  other  technologies  at  a  voluntary  level,  the  preferred  option 
incorporates elements of policy option 2 (voluntary cooperation between Member States and 
cooperation on a broader scope of technologies). 

8.1.3.  Instrument 

Type of legal instrument 

The preferred option envisages the adoption of a new Union legislative act that could take the 
form of a directive or regulation. In considering the most appropriate form of instrument, it is 
necessary to assess the nature of the harmonisation intended to be achieved by the proposed 
measure,  the  nature  and  extent  of  implementing  measures  that  such  harmonisation  entails, 
including, in particular, the extent of discretion afforded to Member States in the choice and 
application of harmonised norms. 

192  Mechanism  for  scrutiny  of  conformity  assessments  of  certain  class  III  and  class  IIb  devices  (as  defined  in 
Regulation  (EU)  2017/745,  Article  55)  and  Mechanism  for  scrutiny  of  conformity  assessments  of  class  D 
devices (as defined in Regulation (EU) 2017/746, Article 50)  
193 Note the provisions for voluntary cooperation in the new legislative framework would replace the existing 
voluntary cooperation on HTA as defined in Article 15 of the Cross-border Healthcare Directive 2011/24/EU, 
i.e. the respective Article 15 would be repealed. 

92 

 
 
                                                            
In this context, it is notable that a key element of the preferred option is the establishment of 
procedures and structures for cooperation on joint REAs at Union level. While inevitably such 
a  transition  to  a  Union  wide  approach  will  require  some  adjustments  to  national  rules,  for 
example, as regards allowing for the use of joint REAs at national level as part of the overall 
HTA,  that  transition  does  not  result  in  a  need  for  significant  implementing  measures 
establishing those procedures and structures at national level. 

The study mapping of HTA processes across the EU shows that 26 Member States currently 
have  legal/procedural  frameworks  in  place  for  HTA.  Typically,  key  aspects  related  to  the 
HTA  bodies  roles  and  responsibilities  and  the  HTA  assessments  are  outlined  in  national 
legislation, while  much  of  the  details  e.g.  on  procedures  are  contained in  the  administrative 
provisions  of  Member  States’  HTA  bodies  (see  sections  1.4.1  and  Mapping  study  on  HTA 
processes across the EU). 

This suggests that a suitable adaptation period before the date of application of a regulation 
would  be  a  more  adequate  and  proportionate  approach  than  the  transposition  needed  for  a 
directive, in ensuring uptake of joint REA and use of common tools. 

Thus, in the light of the aims of the initiative, and the nature and level at which harmonisation 
intended to be achieved, it is considered that a regulation would be the most appropriate form 
of instrument for the proposed measure. 

The new regulation foreseen under the preferred option will amend Directive 2011/24/EU, i.e. 
Article  15  will  be  deleted  from  the  Directive.  The  HTA  Network,  which  currently  provides 
strategic  guidance  and  policy  orientation  for  the  scientific-technical  cooperation  under 
EUnetHTA  (see  section  1.4.2),  will  be  replaced  by  an  HTA  high-level  group  composed  by 
experts  from  the  Member  States  as  part  of  the  governance  system  defined  in  the  new 
regulation  (see  section  8.1.4  for  further  details  on  governance).  The  new  regulation  will 
ensure  that  the  good  governance  principle  (including  transparency  and  independence  of 
expertise), which is referred to in Article 15 of Directive 2011/24/EU, is re-introduced in the 
new  legislative  framework.  Current  provisions  for  granting  Union  aid  under  Article  15  of 
Directive  2011/24/EU  will  also  be  replaced  by provisions  for  Union  funding  under  the  new 
regulation foreseen by the preferred option (see section 8.1.4 for further details).  

Ensuring mandatory uptake 

The legislative framework foreseen by the preferred option will ensure the mandatory uptake 
of  the  common  tools/procedures,  joint  early  dialogues  and  joint  REAs  for  pharmaceuticals 
and  medical  technologies  that  are  within  the  scope  as  defined  in  section  8.1.2.  Mandatory 
uptake of joint REAs implies that Member States shall not repeat the REA at national level. 
Member States shall use the joint REA in the same way as they would use a national clinical 
assessment,  i.e.  they  shall  incorporate  it  in  their  overall  national  HTA  process.  This  means 
that Member States continue to be free to assess non-clinical HTA domains (e.g. economic, 
organisational, ethical) at national level and to draw conclusions on the overall added value of 
the technology. 

Enforcement  of  mandatory  uptake will  be  supported  by  a  requirement on  Member  States  to 
share with the Commission / other Member States the national HTA report which incorporates 
the joint REA carried out at EU level. This will be facilitated by IT tools developed as part of 
the  common  tools  of  the  EU  cooperation.  Moreover,  as  for  any  EU  legislation,  the 
Commission  may  take  the  necessary  action  foreseen  by  the  Treaty  (e.g.  infringement 

93 

 
 
proceedings),  if  informed  of  failure  to  comply  with  the  mandatory  uptake  or  any  other 
requirement of the legislation foreseen by the preferred option. 

Allowing time for adaptation and adequate safeguard provisions 

The  EU  legislative  framework  foreseen  by  the  preferred  option  will  include  provisions  to 
ensure that Member States have adequate time to adapt their national HTA frameworks to the 
new EU system. This takes into account the diversity of HTA frameworks across the EU (see 
section 1.4.1 and Mapping study on HTA processes across the EU) and the views of public 
administrations  in  the  public  consultation  (discussed  further  in  section  8.3.).  In  particular,  a 
deferred  application  from  the  date  of  entry  into  force  will  allow  for  the  alignment  of 
procedures  and  processes.  After  the  date  of  application,  a  transitional  period  is  foreseen 
during  which  Member  States  can  delay  their  participation  in  joint  REAs  and  joint  early 
dialogues. 

The legislative framework  foreseen under the preferred option will also include a safeguard 
clause allowing a Member State to carry out a national REA in addition to a joint REA if this 
can be justified by a need to protect public health which is specific to that Member State.  

Progressive implementation of joint work 

The preferred option will include provisions for stepwise build-up of the new EU system for 
joint work during its first years until it becomes fully operational.  

In  particular,  the  product  scope  for  pharmaceuticals  foreseen  by  the  preferred  option  (see 
section 8.1.2 and Annex XI) will be implemented in a progressive manner. This implies that 
all pharmaceuticals identified in the scope are expected to go through a joint REA once the 
system is fully operational. The numbers of e.g. joint REAs are expected to increase gradually 
during  the  first  years  of  joint  work  under  the  new  EU  system,  taking  into  account  the 
capacities and priorities of Member States. Selection criteria (same as those used permanently 
for  medical  technologies,  see  section  8.1.2)  will  be  listed  in  the  proposed  legislation  and 
Member States will use these to agree which health technologies will be subject to joint work 
during the building-up phase.194  

For  medical  technologies,  the  system  will  remain  based  on  a  prioritisation  mechanism  to 
ensure that joint REA are only performed on medical technologies selected by Member States 
according to agreed criteria within a limited well identified scope (see section 8.1.2). 

It  should  be  noted  that  by  allowing  adequate  time  for  adaptation  and  progressive 
implementation as discussed above, the preferred policy option combines elements of policy 
option  4.1  and  4.2  in  an  optimal  way.  It  allows  sufficient  flexibility  for  Member  States  to 
adapt over a period of several years, while at the same time ensuring that all Member States 
join the new EU system. The need for both sufficient time to adapt and join the system was an 
important point which has been raised by several Member States in bilateral meetings when 
considering a mandatory uptake of the output.   

194 More details on the progressive implementation, including number of expected outputs (joint early dialogues, 
joint REA ets.) over the first years before the system becomes fully operational are set out in Annex XI.  

94 

 
 
                                                            
8.1.4.  Governance and financing 

Governance 

As  shown  in  section  6.4.  the  analysis  of  the  pros  and  cons  of  each  possible  governance 
arrangement  together  with  their  potential  contribution  to  the  achievement  of  the  operational 
objectives of this initiative shows that at this stage the most feasible governance option is a 
central  secretariat  hosted  by  the  European  Commission.  Compared  to  the  other  governance 
options, it offers a reasonable solution for an initial phase which requires a relatively limited 
number of human resources while providing a stable structure for the EU cooperation on HTA 
encompassing  most  types  of  joint  outputs  (i.e.  common  tools  and  methodologies,  horizon 
scanning,  early  dialogue,  REA).  The  main  disadvantage  indicated  in  section  6.4,  i.e.  the 
impossibility to collect and redistribute fees from industry, may be addressed in the future by 
a possible transfer of the secretariat to an EU Agency. In addition, the governance option of a 
European Commission secretariat received high support in the public consultation (see section 
6.4.3). 

On  the  basis  of  the  analysis  carried  out  (see  section  6.5.),  the  most  suitable  governance 
arrangement for the preferred option is considered a central secretariat hosted by the European 
Commission, at least in an initial phase building up the cooperation.  

As presented in section 5.3.4 and 6.5, the central secretariat will provide: 

- administrative support (e.g. organisation of meetings, travel arrangements etc.),  

-  scientific/technical  support  (e.g.  support  for  the  technical  meetings  of  the  experts, 
preparation  of  the  documentation,  manage  procedures  for  involving  stakeholders,  ensure 
quality management, support implementation of joint tools and procedures, etc.) and 

-  IT  support  (e.g.  host  and  maintain  the  electronic  submission  system,  the  intranet  and 
internet, the databases/repositories of joint and national HTA reports, etc.).  

A  high  level  group/management  board  including  representatives  of  Member  States'  HTA 
bodies would manage the overall governance and would meet regularly to discuss the annual 
work  programme,  topic  prioritisation,  progress  with  outputs  (e.g.  quality,  timeliness)  and 
provide guidance and steer the cooperation.  

95 

 
 
Figure 13. Diagram of the governance arrangement (i.e. EC Central secretariat) for the 
preferred option 

The scientific-technical work of producing the joint outputs would be carried out by experts 
nominated  by  Member  States'  authorities  organised  in  Committees/groups  dedicated  to  the 
various types of joint work (e.g. for Joint REA, for joint early dialogues, for horizon scanning, 
for common tools and methodology etc.).  For example, for the joint REAs, Member States' 
experts  acting  as  author/rapporteur  and  co-author/co-rapporteur  would  carry  out  the  clinical 
assessment  of  the  application/dossier  submitted  by  industry  (complying  with  common  tools 
and procedures as described in section 5.2) and prepare a draft opinion. The HTA High Level 
Group would thereafter examine the draft and approve the joint report which would then be 
used in national HTA processes (see more detailed explanations on mandatory uptake above). 

Financing 

As  regards  financing,  the  costs  of  the  secretariat  should  be  supported  from  the  EU  budget 
Approx. EUR 17 000 000 overall costs, of which EUR are 8 000 000 running costs/year, and 
EUR  9  000  000  are  costs  of  joint  outputs/year.  The  remuneration  of  Member  States  HTA 
bodies  carrying  out  the  joint  work  as  authors  and  co-authors  is  included  in  the  costs  of  the 
joint outputs. Travel  expenses for Member State experts  contributing to the activities  of the 
dedicated committees/working groups (e.g. for joint REA, for joint early dialogues) are also 
covered by the EU budget and are included in the running costs.  An in-kind Member States 
contribution  would  be  possible  in  the  form  of  a)  seconded  national  experts195  from 
experienced  HTA  bodies  who  would  also  ensure  the  smooth  transfer  of  the  know-how 

195  Seconded  national  experts  are  national  civil  servants  or  persons  employed  in  the  public  sector  who  are 
working temporarily for an EU Institution. They remain in the service of that employer throughout the period of 
secondment and receive a daily allowance from the European Commission in line with the provisions in the Staff 
Regulation. 

96 

 
 
 
                                                            
 
developed by EUnetHTA to the central secretariat and the interoperability between the central 
secretariat  and  Member  States  HTA  bodies  and  b)  staff  in  national  HTA  bodies,  who  will 
contribute to the activities of the relevant committees/working groups (e.g. on joint REA, on 
joint early dialogues). 

More  detailed  figures,  including  costs  per  year  during  the  building-up  phase  are  set  out  in 
Annex XI.196   

Review clause 

The  EU  legislative  framework  foreseen  by  the  preferred  option  will  also  include  a  review 
clause. This will enable a review of the new system once it has been fully operational for a 
sufficient  period  of  time,  e.g.  in  terms  of  the  need  for  any  changes  to  the  financing  and 
governance  provisions,  with  a  view  to  a  possible  transfer  of  the  central  secretariat  to  an 
appropriate  EU  body.  This  could  include  an  evaluation  of  the  need  to  introduce  a  fee-for-
service system (e.g. industry fees to contribute to the cost of conducting joint REAs). 

8.2.  Justification of the preferred option 

The  preferred  option  is  expected  to  provide  for  the  best  combination  of  effectiveness  and 
efficiency,  while  ensuring  proportionality,  subsidiarity  and  coherence  with  related  EU 
policies. It also considers the need to address potential risks and implementation challenges. 

Effectiveness and efficiency 

The EU legislative framework foreseen by the preferred option will ensure mandatory uptake 
of joint work (see section 8.1.3 for further details). It will provide legal clarity and certainty 
with regard to the legal status of joint outputs and enable and justify the necessary adaptations 
in  national  legal/procedural  HTA  frameworks.  As  discussed  in  more  detail  in  section  2 
(problem  2),  uncertainty  around  the  legal  status  of  joint  outputs  in  the  context  of  existing 
national legal/procedural frameworks is a major reason for the low uptake under the current 
voluntary,  project-based  Joint  Action  EUnetHTA.  As  explained  in  sections  5.3.2  and  6.2, 
these  hurdles  could  also  not  be  sufficiently  addressed  by  other  forms  of  project-based 
cooperation  (even  if  contractual  arrangements  to  promote  uptake  were  envisaged).  An  EU 
legislative framework which ensures mandatory uptake of joint work is therefore necessary to 
effectively address the current problem of low uptake. 

In addition, the EU legislative framework will also address other factors that currently hinder 
uptake,  in  particular  concerns  around  quality  and  timelines  (discussed  in  more  detail  in 
section 2, problem 2). The EU legal framework will define and ensure, inter alia, governance 
and  work-sharing  mechanisms,  high  scientific  standards  of  methodologies  and  assessments 
(e.g.  via  pooling  of  expertise,  input  by  external  experts  and  stakeholders,  quality  assurance 
mechanisms),  and  standardisation  and  timeliness  of  procedures  for  the  production  of  joint 
outputs. This  will enable  Member States to  perform clinical HTA  work at  consistently  high 
quality and in a timely and efficient manner. 

196 The figures included in the Annex are estimates. The contribution from the EU budget post 2020 will be 
discussed within the framework of the preparation of the Commission's proposals for the next Multiannual 
Financial Framework (MFF) and will reflect the outcome of the negotiations on the MFF post 2020. 

97 

 
 
                                                            
The  preferred  option  will  allow  for  the  best  possible  achievement  of  the  objectives  of  the 
initiative. In particular, the preferred option will ensure that all Member States participate in 
the new EU system, maximising the positive impacts of the initiative on the functioning of the 
internal  market  and  public  health  across  the  EU.  Mandatory  uptake  of  common  tools, 
procedures  and  high  quality  joint  work  on  clinical  aspects  of  HTA  will  promote  evidence-
based  decision-making,  contributing 
innovative  health 
technologies  to  patients.  The  preferred  option  will  also  provide  Member  States  with  a 
sustainable  framework  for  cooperation  and  enable  them  to  use  their  HTA  resources  more 
efficiently.  Industry  will  benefit  from  efficiency  gains  and  from  improved  business 
predictability with regard to evidence requirements and HTA outcomes. 

improved  availability  of 

to 

The preferred option is cost efficient in the sense that the costs are significantly outweighed 
by savings for Member States and industry, as a result of pooling of resources and avoiding 
duplications (see sections 6.4.1, 6.5 and 8.3). 

Proportionality and subsidiarity 

Ensuring  mandatory  uptake  by  an  EU  legislative  framework  as  foreseen  by  the  preferred 
option  is  proportionate  in  that  it  is  an  effective  and  necessary  response  to  addressing  the 
current problem of low uptake of joint work (see previous section on effectiveness).  

The  inclusion  of  a  provision  for  mandatory  uptake  is  deemed  necessary,  as  without  this 
obligation,  there  would  be  a  high  risk  that  the  EU  legislative  framework  foreseen  by  the 
preferred  option  could  not  fully  deliver  on  the  objectives  of  the  initiative  (see  section  4). 
While improvements in quality assurance mechanisms and timeliness of procedures compared 
to the current project-based cooperation may encourage and facilitate spontaneous (voluntary) 
uptake  by  Member  States,  there  would  be  no  guarantee  that  all  Member  States  would 
consistently take up the joint outputs. In fact, there would be a risk that some Member States 
may choose to decide on uptake of joint outputs (e.g. joint REAs) on a case by case basis, and 
possibly only once the joint output has been produced. Such an approach would run counter to 
the work-sharing, scientific consensus-building and decision-making mechanisms foreseen by 
the  preferred  option  (see  section  8.1.4  for  further  details  on  governance).  If  there  is  no 
obligation  to  take  up  the  joint  outputs,  some  Member  States  may  not  fully  invest  their 
capacities and resources into the scientific consensus-building on joint outputs at EU level. If 
some  Member  States  continue  to  repeat  jointly  conducted  work  again  at  national  level,  the 
objectives of the initiative to ensure efficient use of resources (both for Member States and the 
EU) and reducing current duplication of efforts by HTA bodies would not be fully achieved. 
Moreover,  the  current  problems  of  duplication  of  efforts  for  industry  and  lack  of  business 
predictability  could  not  be  fully  addressed.  Without  mandatory  uptake  for  Member  States, 
manufacturers may be confronted with situations where after submission of a dossier for joint 
REA,  they  are  requested  by  individual  Member  States  to  submit  additional  dossiers  for 
national  REAs  (with  possibly  different  requirements  and  outcomes).  In  fact,  mandatory 
submission by industry for joint REAs (as foreseen by the preferred option) would be difficult 
to  justify  and  could  even  be  considered  disproportionate,  if  uptake  by  Member  States 
continued  to  be  voluntary.  Finally,  without  an  obligation  for  mandatory  uptake  by  Member 
States,  the  expected  benefits  of  the  initiative  in  terms  of  quality  and  efficiency  gains  and 
associated benefits for patients and public health may be unevenly distributed across the EU 
(depending  on  the  extent  to  which  individual  Member  States  take  up  joint  work).  In 
conclusion, there would be a considerable risk that the initiative does fully deliver on its core 
objectives of improving both the functioning of the single market and public health across the 

98 

 
 
EU. This is substantiated by the current legal framework for the cooperation provide by article 
15  of  Directive  24/2011/EU,  which  defines  ambitious  objectives  but  has  not  been  able  to 
achieve them. 

The mandatory uptake foreseen by the preferred option is also proportionate in that it does not 
go  beyond  what  is  necessary  to  ensure  that  joint  outputs  (e.g.  joint  REAs)  are  incorporated 
into  national  HTA  processes  (see  sections  8.1.3,  6.4.1,  5.3.3  and  5.3.4  for  more  detailed 
explanations on mandatory uptake).  

Proportionality  and  subsidiarity  are  further  ensured  by  focusing  the  joint  work  to  clinical 
aspects  of  HTA,  where  EU  cooperation  can  bring  both  quality  and  efficiency  gains.  The 
assessment  of  more  context-specific  HTA  domains  (e.g.  economic,  organisational,  ethical) 
and  decision-making  on  pricing  and  reimbursement  remain  at  Member  States  level. 
Mandatory uptake of e.g. a joint REA will not interfere with the assessment at national level 
of non-clinical HTA domains. Moreover, mandatory uptake of a joint REA does not pre-empt 
the  national  appraisal  process  which  will  continue  to  conclude  on  the  presence/absence  or 
extent of added value (based on the scientific assessment of clinical evidence presented in the 
joint REA and any additional non-clinical assessments conducted at national level). 

The preferred option is also proportionate in that it limits the scope of mandatory production 
and uptake of joint work to specific types of pharmaceuticals and medical technologies, and 
allows flexibility concerning the timing of joint REAs for medical technologies (see section 
8.1.2).  This  takes  into  account  the  differences  between  technology  sectors  and  their  market 
access pathways (see section 1.3 and Annex V) and the EU added value of a joint approach 
also  in  terms  of  focusing  on  the type  of  products  where  current  duplication  of  work  among 
HTA  bodies  is  most  prominent  (see  section  2,  problem  2).  For  other  technologies,  the 
preferred option facilitates further voluntary cooperation (see section 8.1.3). 

Finally,  the  preferred  option  respects  the  principle  of  proportionality  by  allowing  sufficient 
time for both Member States and industry to adapt to the new EU system (see section 8.1.3) 
and by providing adequate safeguard provisions allowing Member States to carry out national 
REAs in addition to joint REAs in duly justified circumstances related to the specific situation 
in those Member States. 

Coherence 

The identified option constitutes a coherent approach, well in line with the EU's overarching 
objectives, including a smooth functioning of the internal market, sustainable health systems 
and  an  ambitious  research  and  innovation  agenda.  In  addition  to  coherence  with  these  EU 
policy  objectives,  the  option  is  also  coherent  with  and  complementary  to  existing  EU 
legislation  related  to  pharmaceuticals  and  medical  technologies197.  For  example,  there  are 
opportunities for mutual information-sharing and better alignment of the timing of procedures 
between the joint  REA  and the centralised marketing  authorisation for pharmaceuticals (see 
Figure  3)198.  Synergies  are  also  expected  between  joint  REAs  for  medical  technologies  and 

197  Relevant  legislation  includes  Regulation  (EC)  No  726/2004,  Regulation  (EU)  536/2014,  Regulation  (EU) 
2017/745 and Regulation (EU) 2017/746 (see section 1.3 and Annex V for further details) 
198 Note that the need for improved synergies, while respecting the different remits of marketing authorisation 
and  HTA,  has  been  recognised  by  Member  States  in  the  HTA  Network  Reflection  Paper  "Synergies  between 
regulatory and HTA issues on pharmaceuticals" as well as by EUnetHTA and EMA in their joint "Report on the 
implementation of the EMA-EUnetHTA three-year work plan 2012-2015". 

99 

 
 
                                                            
some of the provisions foreseen by the new EU Regulations for medical devices and in vitro 
diagnostics (e.g. strengthened rules on clinical evaluation and clinical investigation; EU-level 
expert  panels  for  certain  high-risk  medical  devices)199.  Moreover,  the  joint  early  dialogues 
foreseen under the preferred option will contribute to the objectives of related EU legislation 
on clinical trials to ensure that the evidence generated in clinical studies is robust and benefits 
patients and public health. The option could also provide useful input to and synergies with 
the Digital Single Market agenda by encouraging innovation and research of high-tech/digital 
medical  products  and  by  supporting  the  development  of  a  European  IT  infrastructure 
supporting  EU  cooperation  on  HTA.  The  initiative  is  expected  to  play  an  important  role  in 
supporting innovation for the benefit of patients by influencing longer-term R&D investment 
decisions by industry (see section 1.3). 

Addressing potential risks and unintended consequences 

A potential risk to the implementation of the preferred option could be posed by challenges in 
achieving  scientific  consensus  on  joint  outputs  on  clinical  HTA  aspects.  As  discussed  in 
section 1.4.1, there are still divergences in the methodologies currently used by HTA bodies 
in  their  national  clinical  HTA  work.  However,  EUnetHTA  and  other  European  projects 
(SEED project, EU-funded research projects) have already delivered the proof of concept that 
convergence  in  methodologies  and  production  of  joint  outputs  at  European  level  is  possible 
(e.g.  common  methodological  guidelines,  joint  early  dialogues  and joint  REAs  produced  by 
EUnetHTA, see section 1.4.2). The improved governance structure foreseen by the preferred 
option  is  expected  to  further  facilitate  consensus-building.  In  particular,  it  will  ensure  the 
involvement  of  all  Member  States,  both  in  the  selection/prioritisation  of  the  technology 
undergoing  a  joint  REA  and  in  the  preparation  of  outputs  by  technical  staff  and  the  final 
approval of joint outputs by high-level representatives (see section 8.1.4). Such a governance 
structure is expected to ensure that outputs are relevant and acceptable to all Member States. 
For example, there may be situations where a minority group of Member States would prefer 
a  different  comparator  in  a  joint  REA.  Such  needs  can  be  accommodated  by  including 
analyses  against  several  comparators  in  a  joint  REA  (as  has  already  been  discussed  in  the 
context of EUnetHTA). Finally, consensus-building will be facilitated by limiting the content 
of  the  joint  REA  to  a  scientific  analysis  and  discussion  of  the  clinical  effects  observed  for 
different  health  outcomes,  in  light  of  the  available  clinical  evidence.  HTA  appraisals,  i.e. 
conclusions  on  the  presence/absence  or  extent  of  added  value  (e.g.  therapeutic,  economic, 
societal), will continue to remain at Member States level (see section 8.2, proportionality and 
subsidiarity).   

Another potential risk to the production of joint work under the preferred option relates to the 
sharing of the work load among Member States. Particularly in the initial build-up phase of 
the  new  EU  system,  some  Member  States  may  hesitate  to  take  a  leading  role  (e.g.  as  lead 
authors of a joint REA) because of lack of experience with the new EU system. However, as 
the joint work under the preferred option will build on best practices among Member States 
HTA  bodies  and  experiences  with  joint  work  under  EUnetHTA,  it  is  expected  that  HTA 
bodies will be  able to  quickly  familiarise themselves  with  the new system.  Within the Joint 
Actions  agencies  with  experiences  and  capacities  in  HTA  have  been  leading  main  work 

199  Wild  C,  Sauerland  S,  Schnell-Inderst  P.  Closing  the  gap  between  regulatory  and  HTA  requirements  for 
approval  and  reimbursement  of  high-risk  medical  devices  in  Europe,  Journal  of  Medical  Device  Regulation, 
Volume 14 (4), 2017 

100 

 
 
                                                            
packages  and  there  is  no  reason  to  believe  that  they  may  change  approach  once  the 
cooperation  becomes  more  structured  and  permanent,  rather  the  contrary.  Moreover,  as 
discussed  in  more  detail  in  section  8.3,  it  is  anticipated  that  Member  States  with  advanced 
HTA systems will take on the leading role particularly in the first years of the new system. 
Member  States  with  currently  limited  resources  and  capacities  for  HTA  may  initially 
primarily  participate  by  providing  input  and  comments  during  the  drafting  process of  e.g.  a 
joint REA. However, as confirmed by public authorities in the public consultation, joint work 
at  EU  level  can  be  expected  to  result  in  increased  capacity  building  on  HTA  over  time, 
particularly  for  countries  with  more  limited  resources.  It  also  provides  opportunities  for 
building capacities and developing a leading role in specific areas (e.g. particular therapeutic 
areas or types of technologies). The benefit of such a specialisation of expertise among HTA 
bodies  has  been  recognised  by  Member  States  representatives  in  the  HTA  Network200.  It 
should  also  be  noted  that  such  trends  for  specialisation  have  been  observed  among  national 
regulatory  authorities  in  the  context  of  the  EU  marketing  authorisation  of  pharmaceuticals. 
Finally,  the  preferred  option  will  foresee  dedicated  administrative,  scientific  and  financial 
support  by  the  central  secretariat  (including  special  allowances  for  lead  authors),  to  further 
encourage and incentivise active participation by all Member States in the production of joint 
work (see sections 8.1.4 and 8.3).          

Provisions  of  the  preferred  option  to  address  the  above-mentioned  challenges  are  further 
discussed in section 8.3. 

8.3. Implications for Member States and other stakeholders 

Member States 

The production of joint outputs and their mandatory uptake foreseen by the preferred option 
will require some adjustments to the legal/procedural HTA frameworks currently in place in 
Member States. Individual Member States will be affected to different extents, depending on 
whether they already have established HTA systems and how detailed the provisions in their 
current  legal/procedural  frameworks  are  (see  section  1.4.1  for  further  information  on  HTA 
systems  and  processes  across  the  EU).  However,  allowing  adequate  time  for  adaptation  of 
national frameworks and the progressive implementation of joint work during the first years 
of the new EU system (see section 8.1.3) will ensure sufficient time for all Member States to 
make the necessary adjustments. 

With regard to  resource implications for Member States, it is  expected that  any initial  costs 
related  to  the  above-mentioned  adjustments  to  national  HTA  framework  will  be  more  than 
off-set by the efficiency gains from joint work (see sections 6.3.1 and 6.4.1). As explained in 
section  8.1.4,  the  preferred  option  foresees  that  most  of  the  costs  of  producing  joint  work 
(support functions of the EU secretariat, travel of Member States experts to meetings, special 
allowances to remunerate Member States bodies carrying out the work as lead authors of joint 
outputs) will  be covered from the EU budget. Member States  may contribute in  kind by: a) 
sending seconded national experts to support the EU secretariat function, and b) through staff 
of  national/regional  HTA  bodies,  who  will  contribute  to  the  activities  of  the  relevant 
committees/working groups (e.g. on joint REA or joint early dialogues, see section 8.1.4). As 
discussed in section 8.1.4., for each joint output, some Member States will assume a leading 

200 HTA Network. Reflection paper on "Reuse of joint work in national HTA activities". 2015 

101 

 
 
                                                            
role  (e.g.  author  and  co-author  drafting  a  joint  REA)  while  the  other  Member  States  have 
opportunities  to  review  and  comment,  and  the  final  output  is  then  approved  by  all  Member 
States.  

It  is  expected  that  Member  States  with  advanced  HTA  systems  (significant  institutional 
capacities, resources and expertise) will assume a larger part of the workload (e.g. by serving 
as lead authors in joint assessments), particularly in the first years. This would be similar to 
existing  experiences  with  sharing  of  work  load  among  national  regulatory  authorities  in  the 
context  of  the  EU  marketing  authorisation  of  pharmaceuticals,  where  some  Member  States 
take a more prominent role in assuming the roles of main authors/rapporteurs201. It would also 
be consistent with experience from the project-based EU cooperation on HTA (Joint Actions 
EUnetHTA, SEED project, research projects on HTA, see section 1.4.2), where participants 
from Member States with advanced HTA systems have been particularly active (e.g. serving 
as  work  package  leaders).  Under  the  preferred  option,  Member  States  with  advanced  HTA 
systems which assume a leading role in the joint work will also benefit from efficiency gains, 
as part of the REAs which they currently have to produce on their own at national level will 
be replaced by joint REAs with other Member States as lead authors. It should be noted that 
Member States with advanced HTA systems already routinely conduct national REAs for the 
technologies covered by the mandatory scope of the preferred option (as described in section 
8.1.2)  and  some  of  them  conduct  national  REAs  for  an  even  broader  scope  of  technologies 
(see  section  1.4.1).  Member  States  with  advanced  HTA  systems  are  therefore  expected  to 
have both the capacity and willingness to take a leading role in the production of joint work, 
in  particular  in  the  first  years  of  the  new  EU  system.  In  addition,  the  EU  secretariat  will 
provide compensation to Member States’ bodies carrying out the joint outputs as lead authors 
in the form of special allowances (and will also cover the travel expenses of the other Member 
States  experts  attending  the  meetings  of  the  relevant  committees/working  groups  providing 
input to and approving the joint work, see section 8.1.4).  

Member States with less advanced HTA systems and limited capacities, the efficiency gains 
from joint work will be even greater: While they are currently only able to perform HTA for a 
limited  number  of  technologies  due  to  resource  constraints,  joint  REAs  will  enable  them  to 
take better informed decisions on larger number of technologies. Member States with limited 
resources could also benefit from efficiency gains through increased specialisation in an EU 
system  for  joint  work.  For  example,  they  could  build  up  capacity  in  specific  areas  (e.g. 
therapeutic indications, types of technologies) and over time assume an increasing role as lead 
author for e.g. joint REAs in these areas.202 A well-defined product scope focusing on health 
technologies  with  EU  added  value  and  cross  border  relevance  with  appropriate 
selection/prioritization criteria will allow Member States to focus on joint work relevant for 
all.   

In  addition  to  the  above-mentioned  efficiency  gains,  Member  States  will  also  benefit  from 
quality  gains  through  joint  work.  As  already  discussed  in  section  8.2.,  the  EU  legislative 
framework foreseen by the preferred option will include common procedures such as quality 
assurance mechanisms, procedures for consulting external experts (e.g. in the development of 
methodologies  or  the  production  of  joint  REAs),  and  conflict  of  interest  rules  to  ensure 

201 See EMA Annual Report 2016, Section on European medicines regulatory network 
202  Note  that  similar  specialisation  trends  have  been  observed  among  national  regulatory  authorities  in  the 
context of the EU marketing authorisation of pharmaceuticals. 

102 

 
 
                                                            
independence  of  expertise.  These  common  procedures  will  build  on  current  best  practices 
among  HTA  bodies  across  the  EU.  Member  States  with  less  advanced  HTA  systems  and 
limited resources will particularly benefit from the ensured high quality of joint outputs. But 
even Member States with advanced HTA systems are expected to benefit from further quality 
gains, by pooling their expertise and resources, and by drawing on the best available external 
expertise  across  Europe  (e.g.  specialists  for  particular  therapeutic  areas,  including  rare 
diseases, or experts on new and complex technologies). 

The preferred option takes into account the views expressed by Member States representatives 
in the HTA Network and by public administrations in the public consultation. Member States 
have  expressed  their  willingness,  in  principle,  to  take  up  joint  work  on  clinical  assessments 
(REA), provided that current hurdles such as the legal uncertainty around the status of joint 
outputs and concerns around quality assurance and timeliness are addressed. For example, the 
HTA  Network  (which  includes  representatives  at  policy  level  from  all  Member  States,  see 
section  1.4.2)  has  called  for  the  strengthening  of  joint  work  (e.g.  via  improved  quality 
assurance  and  process  management),  for  addressing  administrative/legal  hurdles  and  for 
increasing  the  uptake  of  joint  work  in  national  activities.  203,204  In  the  public  consultation, 
public  administrations  also  expressed  support  for  the  principle  of  uptake  of  joint  work.  In 
particular,  the  majority  of  public  administrations  expressed  support  for  the  notion  that  once 
institutions  participate  in  joint  work,  uptake  should  be  mandatory  for  them.  With  regard  to 
participation in joint work, a number of public administrations stressed the need to allow time 
to  build  up  the  new  EU  system  and  to  adapt  national  processes  accordingly.  The  preferred 
option takes into account this need for time to adapt by foreseeing both a deferred application 
and  a  transitional  period  during  which  Member  States  may  delay  their  participation  in  joint 
work (see section 8.1.3).  

Moreover,  the  provisions  foreseen  by  the  preferred  option  for  improved  transparency  (see 
sections  5.2,  5.3.3  and  5.3.4)  are  consistent  with  the  general  acknowledgement  among 
Member  States  of  the  importance  of  the  good  governance  principle.  For  example,  the  HTA 
Network  has  recognised  that  "cooperation  is  based  on  the  principle  of  good  governance, 
including:  transparency,  objectivity,  independence  of  expertise,  fairness  of  procedure  and 
appropriate  stakeholder  involvement"203.  Moreover,  the  HTA  Network  has  supported 
specifically  that  "authorities  responsible  for  HTA  should  aim  at  ensuring  that  HTAs  are 
electronically  accessible  and  understandable 
importance  of 
transparency  has  also  been  highlighted  by  stakeholders  in  the  public  consultation  (also  see 
following section on patients and healthcare professionals). 

to  stakeholders"203.  The 

The  preferred  option  does  not  prevent  Member  States  from  continuing  or  initiating  work  at 
national or  regional level  as  far as  non-clinical domains are concerned, thus  mutual  support 
between EU cooperation, regional cooperation and national work can be envisaged. 

Patients and healthcare professionals 

The  EU  legislative  framework  foreseen  by  the  preferred  option  will  ensure  adherence  to 
common procedures for stakeholder involvement and transparency (see sections 5.2, 5.3.3 and 
5.3.4), building on current best practices of HTA bodies across the EU. Considering that some 
HTA  bodies  currently  do  not  involve  patients  and  external  experts  such  as  healthcare 

203 HTA Network. Strategy for EU Cooperation on Health Technology Assessment. 2014 
204 HTA Network. Reflection paper on "Reuse of joint work in national HTA activities". 2015 

103 

 
 
                                                            
professionals or involve them only to a limited extent (see section 1.4.1), the preferred option 
is expected to lead to significant improvements. For example, common procedures for patient 
involvement will ensure that patients can express their views and provide their disease-related 
experience when issues such as quality of life or importance of particular symptoms and side 
effects are discussed in the context of a REA.  

Patients  and  healthcare professionals  will  also benefit  from  increased  access  to  high-quality 
HTA  reports.  It  should  be  noted  that,  as  highlighted  by  the  GÖG-LSE  study,  some  HTA 
bodies  currently  do  not  publish  their  HTA  reports.  The  preferred  option  will  contribute  to 
improving transparency in HTA across the EU, e.g. by ensuring publication of joint REAs and 
other joint outputs. 

In addition to pooling the expertise of HTA bodies across the EU, the preferred option also 
foresees common procedures for ensuring input by external experts which can contribute up-
to-date specialist knowledge (e.g. on complex therapeutic areas, new technologies, methods in 
clinical  study  design/analysis  and  evidence-based  medicine)  to  the  development  of  joint 
outputs such as joint REAs or common methodologies. This will lead to improved quality and 
more consistency in work on clinical aspects by HTA bodies across the EU. It is also in line 
with  the  responses  received  in  the  public  consultation  from  scientific/medical  societies  and 
healthcare  professional  associations,  who  have  expressed  their  willingness  to  become  more 
involved and contribute their expertise to the EU cooperation. Finally, improved quality and 
consistency  of  work  on  clinical  aspects  of  HTA  across  the  EU  will  respond  to  the  views 
expressed  by  patient  organisations  in  the  public  consultation:  While  patients  acknowledged 
that HTA bodies may reach different conclusions on non-clinical aspects of HTA (e.g. due to 
economic differences between countries), they also noted that is does not make sense to them 
that HTA bodies currently differ in their scientific assessments of the same clinical evidence 
in the context of the clinical part of HTA (see section 2, problem 1). Patient representatives 
have therefore called for this issue to be addressed by the EU cooperation.    

Industry 

As  already  discussed  in  detail  in  section  6.4.1.,  the  pharmaceutical  industry  is  expected  to 
overall  benefit  from  the  preferred  option,  which  is  in  line  with  views  expressed  by  the 
industry representatives. 

In the public consultation and the survey conducted by the GÖG-LSE study, representatives 
of  the  medical  technology  industry  expressed  a  number  of  concerns  (see  section  6.4.1).  In 
particular, they considered that their industry would be negatively affected if joint REAs were 
applied to the full range  of medical technologies (rather than focusing on technologies with 
particular relevance to health systems across the EU) and if joint REAs were conducted at the 
time  of  market  launch  (causing  potential  delays  to  market  access).  By  limiting  the  product 
scope of mandatory production and uptake of joint REAs and allowing flexibility with regard 
to  time  point  of  the  assessment  (see  section  8.1.2.),  the  preferred  option  takes  into  account 
these concerns and at the same time ensures proportionality and EU added value (see section 
8.2.). 

In addition to the benefits already discussed, SMEs from both sectors are expected to benefit 
from the fact that no industry fees are foreseen under the preferred option. This is expected to 
increase  participations  of  SMEs  in  early  dialogues,  which  are  particularly  beneficial  for 
smaller companies with limited HTA expertise in the medical technology sector.  

104 

 
 
Finally,  industry  representatives  of  both  the  pharmaceutical  and  the  medical  technologies 
sectors have expressed a need for sufficient time to adapt to the new EU system, which will 
be ensured by the preferred option (see section 8.1.3). 

For further details on impacts of the preferred option on different stakeholders, see Annex III. 

9.  Monitoring and evaluation 

Monitoring  and  evaluation  of  the  specific  objectives  will  have  to  use  several  means  of  data 
collection, as not all objectives are equally quantifiable and some monitoring may depend on 
a qualitative evaluation based for example on feedback from stakeholders obtained through a 
survey. 

This  section will mainly suggest possible indicators on how to  measure the effectiveness of 
the preferred option in relation to the specific objectives stated in the impact assessment. As 
much as possible quantitative indicators will be considered but qualitative ones may also be 
used.  As  regards  the  implementation  of  the  proposed  legislation,  data  collection  can  be 
qualitative  by  gathering  information  on  the  legal  and  administrative  measures  taken  by 
Member States to implement the legislation. Table 16 below illustrates different alternatives 
for monitoring the outputs and to assess further impacts of the preferred policy option using 
indicators on effectiveness. 

Operational 
Objectives 
1.  Promote 

convergence 
in HTA tools, 
procedures 
and 
methodologies 

Core indicators 

Source of data 

Target 

  Member State National 
Contact Points (NCP).  
  Member State authorities 

All Member States within 
the foreseen application 
(and possible transitional) 
period. 

  Adjustment made by 

Member States to their 
legal/procedural HTA 
frameworks, to enable 
use of common tools, 
procedures, and 
methodologies and 
joint REA 
(qualitative) 

  Use of methodologies 
and tools in MS HTA 
activities. (qualitative) 

105 

 
 
 
 
 
 
 
 
 
2.  Reduce 

  Number of joint REA 

duplication of 
efforts for 
HTA bodies 
and industry  

3.  Ensure the 

uptake of joint 
output in 
Member 
States 

for innovative 
pharmaceutical 
products (quantitative) 

  Number of national 
REA for innovative 
pharmaceutical 
products (quantitative) 

  Number of joint ED 
(quantitative) 
  Number of national 
ED (quantitative) 
  HTA reports used by 
other Member States 
in national/regional 
HTA activities 
(quantitative) 
  Number of FTE 

needed to produce a 
national full HTA 
report (quantitative) 
  Timeliness or joint 

REAs 

  Number of joint REA 
used in MS HTA 
activities.  
(quantitative) 

  Use of methodologies 
and tools in MS HTA 
activities. (qualitative/ 
quantitative) 

  Permanent secretariat of 
the EU cooperation. 
  Member State authorities 
  National Contact Points 
  Qualitative evaluation 

(e.g. comparison Member 
State report with EU 
assessment) 

Initial target 65 REA  

 
  Long term target: 
joint REA on all 
pharmaceuticals 
receiving a positive 
recommendation by 
EMA  

  No duplication of 
joint REA. 
  No additional 

 

Member State REA 
(unless duly justified 
dependent on 
legislation). 
Joint REAs for 
pharmaceuticals 
available at or shortly 
after the time of 
marketing 
authorisation 

  Member State HTA 

  Full uptake of joint 

authorities.  

  Secretariat of the EU 

cooperation 

  National Contact Points.  

REA if MS decides to 
start assessment 
procedure on the 
same technology.  

  Comprehensive use 

of methodologies and 
tools provided by the 
EU cooperation in 
national HTA 
activities.  

4.  Ensure the 
long-term 
sustainability 
of EU HTA 
cooperation. 

  Budget devoted to 
support EU 
cooperation 

  Production rate of the 
cooperation (number 
of joint outputs/year)  

  Legislation 
  Member State HTA 

authorities. 

Timeline to be indefinite 
(subject to review) to 
ensure sustainability.   

Table 16. Suggested core indicators for monitoring 

Effectiveness indicators of actions and outputs in relation to the specific objectives (illustrated 
in Table 16) will be part of the development of a broader monitoring programme, which will 
also  include  specific  indicators  related  to  efficiency  and  coherence  with  other  policies  (e.g. 
EU legislation on medicinal products and medical devices). 

In addition to the monitoring programme, it is suggested that an evaluation will be carried out 
at  later  point  in  time  (when  the  system  has  been  fully  operational  for  a  sufficient  period  of 
time to enable a meaningful evaluation), to assess the wider impacts of the implementation of 
the preferred policy option. In particular, the evaluation will look at addressing the problems 
identified in the impact assessment. In addition, cost benefit analyses on the performance of 
the implementation mechanism should be performed as part of future evaluations. According 

106 

 
 
 
 
 
 
 
 
 
to  the  financial  regulation,  an  evaluation  will  also  be  required,  as  the  cost  of  the 
implementation mechanism is expected to be above the EUR 5 000 000 threshold.205 

In summary, there are a number of quantitative indicators to assess the future cooperation, but 
assessment  will  also  depend  on  qualitative  data  sources.  Monitoring  and  evaluation  of  the 
wider impacts will require a number of qualitative tools such as desk research, surveys, focus 
groups  and  Delphi  surveys  to  assess  many  of  the  potential  impacts.  However,  as  regards 
monetary  costs  of  the  cooperation,  quantifiable  data  is  expected  to  be  available  at  a  greater 
extent. 

205 Financial Regulation Article 30 and Rules of Application article 18 

107 

 
 
 
 
                                                            
Annexes 

Annex I. Procedural Information  

1. Identification.  

The Directorate for Health and Food Safety (DG SANTE) is the lead DG on the initiative on 
Strengthening of EU cooperation on Health Technology Assessment.  

The initiative is in the European Commission's Work Programme for 2017, in Annex I New 
Initiatives,  under  the  heading  A  Deeper  and  Fairer  Internal  Market  with  a  Strengthened 
Industrial Base. The initiative has received the validation in the Agenda Planning on the 15th 
September  2016  (reference  2016/SANTE/144)  when  the  Inception  Impact  Assessment  was 
published.  

2. Organisation and timing 

An Inter-Service Steering Group was set up and met on the 15th September 2016, 24th March, 6th 
June, 1st and 11th of September 2017. Next to the SG (Secretariat-General) and LS (Legal Service) 
the  following  Commission  services  took  part  in  the  ISSG:  BUDG  (Budget),  GROW  (Internal 
Market,  Industry,  Entrepreneurship  and  SMEs),  RTD  (Research  and  Innovation),  CNECT 
(Communications Networks, Content and Technology), ECFIN (Economic and Financial Affairs), 
EMPL  (Employment),  TRADE  (Trade),  COMP  (Competition)  and  the  JRC  (Joint  Research 
Centre).  The  members  of  the  Inter-Service  Steering  Group  were  regularly  informed  on  the 
progress of the initiative and invited to relevant meetings.  

In addition, there were close contacts with the European Medicines Agency and the Consumers, 
Health, Agriculture and Food Executive Agency (CHAFEA) on this file and related projects and 
studies. 

3. Consultation of the RSB 

The current initiative of DG SANTE was the first one to benefit from the new opportunity for 
an upstream meeting with the Regulatory Scrutiny Board. The meeting took place on the 7th 
December 2016.  
A  first  version  of  this  Impact  Assessment  Report  was  submitted  to  the  RSB  on  the  27th  of 
September  2017,  the  meeting  took  place  on  the  25th  of  October  2017  and  the  RSB  written 
opinion was received on the 27th of October 2017. 

The Board concluded that the draft report required further work and asked for a resubmission. 

The Opinion of the Board acknowledged the efforts to conduct an analysis of Member States' 
structures,  resources  and  processes  in  place  for  the  development  and  use  of  HTAs.  It  also 
acknowledges the quality of the stakeholder consultation.  

However it identified shortcomings that need to be addressed, concerning the following key 
aspects: 

Justification  of  why  considering  the  continuation  of  the  current  joint  actions  is 

(1) 
unsustainable. 

108 

 
 
(2) 

Justification of the choice of the baseline, and definition of the options.  

Demonstration that the initiative is a proportionate and effective response to low HTA 

(3) 
uptake.  

Explanation  of  what  the  proposed  measures  would  imply  for  Member  States  with 
(4) 
regard to  resources  or  adjustments  to  national regulatory  frameworks  and  practices.  Specify 
the measures to improve patients' and consumers' participation in HTAs.  

Further  analyse  the  preferred  option  and  the  delivery  mechanisms  of  the  initiative, 

(5) 
including related resource implications. 

DG  SANTE  carefully  addressed  all  the  comments  received,  including  the  technical  ones 
submitted directly to the DG in order to improve the quality of the IA Report. 
A second version of the Impact Assessment Report was submitted to the RSB on the 21th of 
November  2017  and  the  second  RSB  written  opinion  was  received  on  the  4th  of  December 
2017. The Opinion of the Board acknowledged the improvements to the previous version and 
issued a positive opinion, while noting a number of remaining adjustment requirements to be 
made  by  DG  SANTE.  DG  SANTE  carefully  addressed  the  comments  received  in  the  final 
version  of  the  Impact  Assessment  Report.  An  overview  of  the  main  adjustments  made  in 
response to the main considerations of the RSB is shown in the Table below. 

RSB main considerations 

1) The baseline is treated as an option and not 
as a comparator for the options. 

Adjustments  made  in  final  version  of  IA 
report 

The final version of the IA report ensures that 
policy  options  are  consistently  compared  to 
the  baseline  scenario.  This  has  also  been 
clarified for figures related to governance and 
budget.  Adjustments  were  made  accordingly 
in sections 5.3.1 and 6.5. 

2)  The  report  provides  indications  that  the 
mandatory  uptake  of  joint  work  would  be 
sufficient  to  address  many  of  the  current 
shortcomings.  However, 
not 
convincingly demonstrate that it is necessary. 
It is not clear what the resulting amendments 
to the existing Directive are. 

does 

it 

3) The report provides insufficient indications 
of Member States' support for key aspects of 
the options. 

the 

and 

clarifying 

Further  clarifications  have  been  provided  on 
the  proportionality  of  the  preferred  option, 
elaborating  why  mandatory  uptake  of  joint 
work  is  considered  necessary  (see  section 
of 
8.2) 
legal/procedural hurdles to uptake (sections 2 
and  8.1).  Moreover,  the  final  version  of  the 
IA report clarifies that some of the principles 
referred  to  in  the  current  Article  15  of 
Directive 2011/24/EU (e.g. good governance, 
transparency) will also be present in the new 
legislative  framework  proposed  under  the 
preferred option (sections 3 and 8.1). 

issue 

Further  details  have  been  provided  on 
expected  Member  States  support  for  key 
aspects 
including 
acceptability  of  mandatory  uptake  of  joint 
work,  willingness  and  capability  to  take  a 
leading role in an EU framework and support 

initiative, 

the 

of 

109 

 
 
for  transparency  measures  (section  8.3).  The 
choice  of  a  Commission-hosted  secretariat  is 
also further elaborated (section 8.1.4). 

Risks  and  possible  unintended  consequences 
of  the  initiative  have  been  further  discussed, 
to  better  contextualise/qualify  the  expected 
benefits  of  the  initiative  (sections  8.2  and 
8.3). 

4) The revised report insufficiently discusses 
the  uncertainties, 
trade-offs  and 
implementation  challenges  associated  with 
the preferred option. 

risks, 

4. Evidence 

The  Impact  Assessment  has  strongly  built  on  the  consultation  and  experience  of  Member 
States,  through  the  two  arms  of  the  current  HTA  cooperation:  the  HTA  Network  and  the 
EUnetHTA  Joint  Action  3  as  well  as  dedicated  bilateral  meetings.  Input  from  stakeholders 
was gathered through the Open Public Consultation, which was open between 21st October 
and  13th  January.  SMEs  were  targeted  through  the  European  Medicines  Agency  and  DG 
GROW's  Enterprise  Europe  Network.  A  number  of  bilateral  meetings  took  place  with 
stakeholders. For more information, see Annex 2 Synopsis Report.  

5. External expertise 

The Impact Assessment was supported by three studies:  
-  Study  on  impact  analysis  of  policy  options  for  Strengthened  EU  cooperation  on  HTA206, 
which  was  undertaken  by  SOGETI  Luxembourg  S.A.,  the  Austrian  Public  Health  Institute 
(GÖG)  and  the  London  School  of  Economics  (LSE),  referred  to  as  GÖG-LSE  study.  The 
study  provided  an  in  depth  analysis  of  (1)  the  role  of  HTA  in  the  market  access  (baseline 
scenario),  (2)  the  potential  impacts  of  identified  options;  (3) systematic  literature  review  on 
HTA, with a specific focus on Europe. The final report of the study is available (Annex X)  

- Mapping of HTA national organisations, programmes and processes in EU Member States 
and Norway207 by Julia Chamova (Annex VIII)  

-  Mapping  of  HTA  methodologies  in  EU  Member  States  and  Norway  by  Finn  Borlum 
Kristensen208 (Annex IX) 

In addition the draft deliverable from EUnetHTA Joint Action 3, Descriptive write up of HTA 
processes  (Work  Package  7  National  Implementation  deliverable  -  unpublished)  was  also 
used.  

The main sources from the literature used are listed in the final report of the GÖG-LSE study 
(please see Annex X). 

The  following  table  summarises  the  surveys  and  questionnaires  supporting  the  Impact 
cooperation. 
of 
Assessment 

future 

HTA 

EU 

the 

the 

on 

206 CHAFEA Framework Contract 2016/Health/16 
207 DG SANTE Contract 17010402/2016/734820 
208 DG SANTE Contract 17010402/2016/736040 

110 

 
 
 
                                                            
Surveys and questionnaires supporting the Impact 
Assessment on the future of the EU HTA cooperation 

Timing of 
survey 
21/10/2016
-13/1/2017 

20/12/2016
-20/1/2017 

18/12/2016
-
22/12/2017 

Target group 
All citizens and 
organisations. Patient 
organisations,  public 
authorities, HTA 
bodies, payers, 
companies developing 
pharmaceuticals, 
medical devices and 
other technologies as 
well as associations 
representing 
stakeholders. SME 
outreach through EMA 
SME office.  
SMEs, outreach 
through the Enterprise 
Europe Network 
national contact points 

Two targeted surveys  
1.  Patient 
organisations, public 
authorities, HTA 
bodies, payers 
2. Technology 
developers (industry)  

3/1/2017-
31/1/2017 

Selected technology 
developers (of the 40 
technologies) 

Name 
Public consultation 

By 
DG SANTE 

SME panel 

DG 
GROW/SAN
TE 

Study  on 
the 
Impacts 
of Policy 
Options 

Survey 
on the 
Impacts 
of Policy 
Options 

Austrian 
Public 
Health 
Institute 
(GÖG) 

Case 
studies 

London 
School of 
Economics 
(LSE) 

HTA process 
mapping study 

Julia 
Chamova  

Aim 
To gather views and 
opinions related to 
the future of the EU 
cooperation on HTA, 
as proposed in the 
IIA. The results of this 
public consultation 
will feed into the 
impact assessment 
process 

A simplified and 
targeted version of 
the open public 
consultation for 
SMEs 
Gather  data and 
evidence on the 
impacts of the policy 
options per 
stakeholder to  
support the Impact 
Assessment process 
of the EC 
Gather evidence to 
map the role of HTA 
in market access 
process through a 
selection of 40 
technologies (20 
pharma, 15 med 
tech, 5 other) 
Map the HTA 
processes in EU MS 
and EEA 

HTA methodology 
mapping study 

Finn Børlum 
Kristensen 

Map the HTA 
methodologies in EU 
MS and EEA 

7/2016-
2/2017 

6/2016-
2/2017 

HTA bodies of MS and 
EEA countries - survey 
distributed via 
EUnetHTA JA3 WP7 
HTA bodies of MS and 
EEA countries - survey 
distributed via 
EUnetHTA JA3 WP7 

111 

 
 
 
 
Annex II. Stakeholder Consultation: Synopsis Report 

1. 

Introduction 

The  synopsis  report  documents  all  the  consultation  activities  accompanying  the  Impact 
Assessment on the initiative for strengthening EU cooperation on HTA.  

The  aim  of  the  stakeholder  consultation  was  to  collect  all  stakeholders'  views  on  the  EU 
cooperation  on  HTA,  encompassing  their  experience  with  the  on-going  cooperation 
mechanisms,  their  specific  needs  and  their  opinion  on  the  proposed  approaches  described  in 
the Inception Impact Assessment. 

For  reaching  all  interested  stakeholders  and  in  order  to  ensure  a  high  quality  and  balanced 
input, a combination of consultation methods was used:  

- The main channel to collect the opinions of stakeholders was the open public consultation. 
Besides  the  feedback  received  in  response  the  publication  of  the  Inception  Impact 
Assessment, DG SANTE launched a broad online public consultation to which all categories 
of  stakeholders  provided  input.  In  addition,  position  statements  from  different  organisations 
were received by email.  

- Bilateral meetings with interested stakeholder representatives were organised to allow in-
depth discussion on specific topics and the expression of non-organized interests. 

-  Experts  consultation  was  carried  out  through  the  existing  cooperation  mechanisms, 
EUnetHTA  Joint  Action  and  the  HTA  Network.  Since  these  are  the  stakeholders  who  are 
already participating in the cooperation and they will be the ones who continue cooperating 
after  2020,  these  meetings  allowed  to  openly  discuss  the  options  proposed  in  the  Inception 
Impact Assessment for the future EU cooperation in HTA.  

- Presentations to external events were used to reach out to stakeholders, to explain the main 
elements of the initiative, to invite them to participate in the public consultation, and listen to 
their views and opinions.  

A summary of all the above mentioned consultations is presented in the subsequent sections. 

2. 

Open public stakeholder consultation 

2.1.  Feedback on the inception Impact Assessment 

Following the  publication  of  the  Inception  Impact  Assessment  on  "Strengthening  of  the  EU 
cooperation on  Health  Technology  Assessment  (HTA)"  on  14  September  2016209  and  up  to 
now, the Commission received a number of 9 positions and statements. Three were submitted 
by national authorities, 4 by trade organisations, and 2 by industry210.  

Most  of  the  contributions  were  supportive,  highlighting  the  need  for  continuing  efforts  to 
facilitate  EU  cooperation  on  HTA,  but  also  pointing  out  the  challenges  of  the  current 

209 http://ec.europa.eu/smart-
regulation/roadmaps/docs/2016_sante_144_health_technology_assessments_en.pdf  
210 https://ec.europa.eu/health/technology_assessment/consultations/cooperation_hta_en  

112 

 
 
                                                            
collaboration  for  the  national  uptake  of  the  joint  work.  It  was  acknowledged  that  neither 
option 1 nor 2 provide for appropriate means not only to preserve but also to further develop 
achievements  to  date,  whilst  an  EU  legislative  framework  could  enable  a  permanent, 
systematic  collaboration.  In  contrast,  contributors  emphasised  that  they  are  not  supporting 
joint work on full HTA reports (proposed as option 5) which was seen as having direct impact 
on  national  pricing  and  reimbursement  processes  which  are  under  Member  States 
competence.  One  national  authority  expressed  reservations  about  the  legal  base  of  the 
initiative,  and  reminded  about  the  legal  provisions  in  the  Cross  border  Healthcare Directive 
which limit EU cooperation on HTA to voluntary cooperation. 

On  the  other  hand,  contributors  representing  medical  technologies'  industry  were  more 
negative, calling for an in depth analysis of the medtech sector and expressing their concerns 
that the particularities of their sector would not be  taken into account. They also questioned 
the potential added value of EU cooperation on HTA for medical technologies, arguing that 
decision making is often local and that currently HTA is rarely used on these types of health 
technologies. One of the contributors (i.e. manufacturer of medical devices) pointed out that 
depending  on  the  existing  regulations  in  different  Member  States,  HTA  might  become  a 
barrier to innovation and limit patient access to quality care. They were in favour of solutions 
based on improved voluntary cooperation among HTA bodies.  

2.2.  Online public consultation 

2.2.1. Description of the questionnaires and of the respondents 

The  Commission  launched  an  online  public  consultation  which  ran  from  21  October  2016 
until 13 January 2017211.  

Due  to  the  technical  nature  of  health  technology  assessment,  and  in  order  to  cover  all 
interested stakeholders, the online public consultation was carried out via two questionnaires. 
One  questionnaire  was  dedicated  to  citizens  and  was  made  available  in  all  EU  official 
languages.  A  second  one,  available  only  in  English  was  directed  to  administrations  (both 
public  and  private  administrations  with  a  public  service  obligation),  economic  stakeholders 
(in  particular  pharmaceutical  and  medical  technologies212'  industry),  as  well  as  associations 
and  organisations  representing  stakeholders  (e.g.  patients  and  consumers,  healthcare 
providers,  payers213,  industry  and  service  providers,  academia  and  scientific  societies).  A 
simplified  version  of  the  questionnaire  dedicated  to  administrations,  associations  and 
organisations,  tailored  for  SMEs  was  circulated  via  the  SME  Network  of  DG  GROW.  This 
questionnaire was also made available in all EU official languages. 

Citizens' consultation focused on their general awareness of HTA and national HTA systems, 
EU  cooperation  on  HTA,  as  well  as  usefulness  and  need  to  access  HTA  information  by 
patients,  consumers  and  healthcare  professionals.  The  consultation  of  administrations, 

211 https://ec.europa.eu/health/technology_assessment/consultations/cooperation_hta_en  
212 Medical technology, or medtech, encompasses a wide range of healthcare products and is used to diagnose, 
monitor or treat diseases or medical conditions affecting humans. In this report medical technologies refer to 
medical devices and in vitro diagnostics (IVD). 
213 For the purpose of this report, payers should be understood as insurance organisations or organisations 
acting on behalf of a public authority responsible for the payment of healthcare services. 

113 

 
 
                                                            
economic stakeholders, associations and organisations was directed to get their opinions on 
the current state of play and EU cooperation on HTA, and on EU cooperation on HTA beyond 
2020.  

The online public consultation and the SME consultation gathered a total of 249 replies. Of 
these  responses,  25%  (63  submissions)  were  from  individuals/citizens  and  the  rest  from 
administrations,  economic  stakeholders,  associations  or  organisations  ("non-individual 
respondents")  (75%,  186  submissions).  Of  the  186  non-individual  contributions,  36  replies 
were  received  in  response  to  the  questionnaire  dedicated  to  SMEs  distributed  to  the  SME 
Network  in  DG  GROW.  The  distribution  of  responses  per  Member  State  and  type  of 
organisation is shown below. 

  Profile of individual respondents 

As  regards  the  geographical  distribution  of  individual  responses,  contributions  from 
citizens/individuals came from 21 EU Member States (62) and Switzerland (1). The highest 
number  of  replies  came  from  citizens  in  Germany  and  Netherlands  (8/MS),  followed  by 
Spain, France and Italy (6/MS), Portugal and United Kingdom (4/MS), Belgium and Sweden 
(3/MS) and Greece, Ireland and Poland (2/MS). Only one reply came from citizens in Austria, 
Bulgaria, Cyprus, Estonia, Finland, Malta, Romania and Slovakia. 

Analysis  of  the  information  provided  in  relation  to  the  level  of  education,  work  experience 
and  sector  of  employment  showed  that  the  large  majority  of  the  individual  respondents  are 
well-educated, with expertise and work experience in either or both public and private sectors, 
in  areas  relevant  for  this  consultation  (e.g.  healthcare  sector,  HTA  sector,  public 
administration, and health technologies 'industry). A large majority (78%) of the respondents 
also  indicate  knowing  how  their  national  HTA  system  is  organised  and  being  aware  of  the 
current EU cooperation on HTA (63%), confirming the contributors' interest and expertise in 
this field.  

  Profile  of  respondents  to  the  questionnaire  dedicated  to  administrations,  economic 

stakeholders, associations and organisations  

Concerning  the  contributions  to  the  questionnaire  dedicated  to  administrations,  economic 
stakeholders, associations and organisations, industry was the major contributor with 52 % of 
all  replies,  followed  by  public  administration  (14%),  patients  and  consumers  associations 
(13%)  healthcare  providers'  organisations  and  scientific  societies  (13%)  and  payers  (3%). 
More details concerning the profile of each category of respondents are presented below: 

- Concerning input from industry, most of the contributions were submitted by SMEs (46%) 
followed  by  big  commercial  operators  (27%),  trade  associations  (26%)  and  consultancies 
(1%).  Additionally,  most  of  the  companies  contributing  to  the  public  consultation  are 
European  or  international  companies,  active  in  more  than  one  Member  State  or  beyond  the 
EU  (64%).  A  similar  number  of  contributions  were  received  from  both  pharmaceutical  and 
medical technologies' industry (32 and 35 contributions respectively).  

- As regards public administrations, most of the contributions were provided by HTA bodies 
(10  replies),  as  well  as  organisations  with  multiple  responsibilities  (8),  Ministries  of  Health 
(4), payers (1) and other national or regional organisations (3). Concerning the geographical 
distribution  of  responses  from  public  administrations,  contributions  came  from  15  EU 
Member  States  (Italy  with  5  contributions,  Germany,  Finland  and  Spain  with  3 
contributions/Member  State,  Slovenia  with  2  contributions,  and  Austria,  Belgium,  Czech 

114 

 
 
Republic,  Croatia,  France,  Hungary,  Ireland,  Poland,  Portugal  and  United  Kingdom  with  1 
contribution per country) and Norway (1 contribution).  

- Patients and consumers were represented by an equal number of national (12 organisations) 
and  European  patients'  associations  (12).  Most  of  these  associations  (63%)  acknowledged 
their  interest  for  both  pharmaceuticals  and  medical  technologies.  Additionally  4%  of  these 
organisations specified being interested in all health technologies.  

-  Healthcare  providers  were  represented  in  the  consultation  by  national  associations  (50%), 
followed  by  European  (31%)  and  regional  organisations  (19%).  Fifty  per  cent  of  the 
respondents  in  this  category  indicated  representing  hospitals  and  the  rest  provided  input  on 
behalf of doctors, community pharmacists, optometrists and public health trusts. 

-  Respondents  from  academia  were  mostly  European  organisations  (63%),  but  also  national 
and international ones (i.e. ISPOR).  

- Payers were mostly represented by national associations (60%).  

-  The  category  "other"  was  selected  by  non-profit  organisations  promoting  public  health, 
information on pharmaceuticals and therapeutic and diagnostic strategies, improved access to 
medicines  and  their  rational  use,  or  the  development  of  therapies  in  specific  areas  such  as 
cancer or regenerative medicine. 
The results of the online public consultation were published in May 2017214 and a summary is 
included below. 

2.2.2. Citizens opinions  

Almost  all  individual  respondents  (98%)  consider  that  it  is  useful  to  compare  new  health 
technologies with existing ones and assess whether they work better, equally well or worse, in 
order to provide guidance to decision makers. Citizens emphasised that patients should have 
access  to  the  best  possible  treatment,  with  the  least  possible  cost,  with  HTA  supporting 
"rational  decision  making  and  control  the  health  care  budget".  The  usefulness  of  HTA  as  a 
tool supporting disinvestment of obsolete technologies, allowing for and better allocation of 
resources for truly innovative health technologies was also highlighted.  

Almost all individual respondents confirmed the need to ensure key elements when carrying 
out assessments: 1) transparency of HTA processes, 2) independence from industry or other 
influences 3) appropriate expertise of the assessors, and last but not least 4) timely delivery of 
assessments for informed decision making.  

As regards the possibility of performing joint EU clinical and economic assessments, 57% of 
the citizens indicated that national/regional HTA bodies should not perform clinical/medical 
assessments  of  the  same  health  technologies  in  parallel,  independently  from  each  other. 
However, a similar percentage of the respondents (56%) were against carrying out EU joint 
economic assessments.  

The survey also showed that most individuals believe that HTA information should be easily 
accessible to doctors to enable an informed decision when prescribing the treatment of their 
patients  (95%)  and  also  to  patients  (84%).  The  involvement  of  relevant  stakeholders  and 
representatives of the public (i.e. patients who can provide good understanding of the patients' 

214 https://ec.europa.eu/health/sites/health/files/technology_assessment/docs/20161020_frep_en.pdf  

115 

 
 
                                                            
point  of  view,  especially  on  topics  such  as  unmet  needs,  quality  of  life  data  and  patients' 
preferences)  by  HTA  bodies  when  preparing  and/or  reviewing  HTA  reports  was  strongly 
advocated.  

2.2.3. Opinions from administrations, economic stakeholders, associations and organisations 

Firstly the questionnaire aimed to verify whether the issues identified in the Inception Impact 
Assessment  are  shared  by  stakeholders.  In  this  respect,  most  of  the  respondents  strongly 
agreed or agreed with the existence of differences in HTA processes and methodologies (80% 
confirmed  the  existence  of  different  HTA  methodologies  for  clinical  assessments,  85% 
acknowledged  the  differences  in  HTA  methodologies  for  economic  assessments,  and  91% 
agreed with the existence of differences in national HTA procedures).  

Fig. 1. Overview of the opinions expressed by administrations, organisations and associations on the 
existence of differences in HTA processes and methodologies across EU 

Furthermore,  the  contributors  to  the  public  consultation  confirmed  that  differences  in  HTA 
processes and methodologies across the EU translate into diverging outcomes of HTA reports 
which may affect patients' access to new technologies (e.g. delays, restricted access) (81% of 
contributions),  duplication  of  work  for  both  HTA  bodies  and  industry  (54%),  decrease  in 
business predictability (53%), higher costs for the actors (38%) and even affect innovation in 
a negative way (37%). 

116 

 
 
 
 
Fig. 2. Consequences of differences in HTA process and methodologies across EU as identified by 
public administrations, organisations and associations  

As  regards  the  current  EU  cooperation  on  HTA,  the  consultation  showed  that  32%  of  the 
respondents participated in EU-funded projects and joint actions and 47% of the contributors 
state that even though they did not directly participate, they were aware of EU cooperation on 
HTA. Whilst participation and awareness were relatively high among public administrations, 
payers,  industry,  healthcare  providers  and  academia,  it  was  very  low  for  SMEs.  From  the 
respondents who confirmed their participation in or awareness of EU funded activities, 69% 
considered EU cooperation on HTA useful or to some extent useful, with most benefit seen by 
public administrations, payers and academia (100%) (Fig. 3). Among the most cited benefits 
of the EU cooperation on HTA are sharing knowledge and best practices among participating 
organisations, capacity building as well as increased trust between participants and increased 
awareness on HTA issues (Fig. 4). 

Fig.3. Usefulness of EU cooperation on HTA (i.e. EU-funded projects and joint actions) per category 
of respondents 

117 

 
 
 
 
 
Fig.4. Benefits of the current EU cooperation on HTA as reported by public administrations, 
organisations and associations (Total number of replies = 157) 

A negative opinion about the EU cooperation on HTA was reported by medical technologies 
industry, SMEs, and a minority of respondents from the pharmaceutical industry (Fig. 3).  

The  survey  showed  that  despite  participation  and/or  awareness,  the  uptake  of  joint  work 
remained low, with significant variations in the estimations provided by different category of 
respondents. Overall, less than 11% of the respondents estimated that joint tools, EUnetHTA 
guidelines and joint early dialogues and joint reports (either REA or full HTA) were used to a 
great extent and up to 51% estimated they were used to a limited extent (Fig. 5).  

Fig. 5. Uptake of joint work from EU-funded projects and joint actions per type of joint activities – all 
categories of respondents (Total number of replies = 150)  

With regard to the future EU cooperation on HTA, a large majority of the respondents (87%) 
consider that EU cooperation on HTA should continue beyond 2020 when EUnetHTA Joint 
Action 3 will end. 

Concerning  the  scope  of  the  future  cooperation,  a  large  majority  of  the  respondents  found 
useful  and  to  some  extent  useful  to  continue  EU  cooperation  on  HTA  in  the  field  of 
pharmaceuticals  (80%),  but  also  in  the  areas  of  medical  technologies  (72%)  and  other 
technologies (54%) (Fig. 6).  

118 

 
 
 
 
As regards the policy options, the questionnaire outlined three options with focus on the type 
of  participation  (i.e.  voluntary  or  mandatory)  and  uptake  by  participating  Member  States' 
HTA  bodies  (i.e.  voluntary  or  mandatory).  In  this  regard,  the  "voluntary  participation  with 
mandatory  uptake  option"  was  generally  favoured,  being  at  the  same  time  the  option  with 
overall  lowest  opposition  and  the  highest  percentage  of  neutral  opinions.  In  contrast  the 
options  voluntary  participation  with  voluntary  uptake  and  mandatory  participation  with 
mandatory uptake registered a significantly higher opposition (50% or more) and less support. 
(Fig. 7). 

In relation to the potential funding mechanisms of the future EU cooperation on HTA, more 
than half of the respondents (53%) pointed towards a mix of contributions from EU budget, 
industry fees and Member States contributions (Fig. 8). 

Fig. 6. Overview of the opinions regarding the 
scope of EU cooperation on HTA beyond 2020Fig.  

Fig.7.  Overview  of  the  opinions  on  the 
policy 
continuing  EU 
for 
cooperation on HTA 

options 

Fig. 8. Overview of the opinions on the funding 
mechanism of the future EU cooperation on HTA 

Fig.9.  Overview  of  the  opinions  on  the 
governance  mechanism  of  the  future  EU 
cooperation on HTA 

With  respect  to  the  governance  mechanism,  the  consultation  shows  an  overall  preference 
towards  an  existing  EU  agency,  followed  by  the  European  Commission.  Respondents 
expressed similar preferences for a new EU agency, Member States HTA bodies on rotational 
basis  and  other  mechanisms,  most  of  them  consisting  of  elements  of  the  current  voluntary 
cooperation and/or EMA-like models (Fig. 9). 

Overall,  the  contributors  to  the  online  public  consultation  were  positive  in  regards  to  the 
future  of  EU  cooperation  on  HTA  beyond  2020,  pointing  out  to  the  outputs  responding  to 

119 

 
 
 
 
 
 
 
their needs and the benefits of harmonising the use of common tools, and of outputs such as 
early  dialogues  and  clinical  assessments/REA.  It  was  highlighted  that  in  the  process  of 
shaping  the  future  EU  cooperation on  HTA,  consideration  should  be  given  to  the  following 
issues:  distinguish  between  assessment  and  appraisal  (the  latter  being  the  responsibility  of 
national health care services and local insurance bodies), clear separation between regulatory 
assessment  (which    informs  decision  on  marketing  authorisation)  and  HTA  (that  informs 
decisions  on  added  value,  use  of  technologies  and  reimbursement  and  pricing),  step-wise 
approach  as  potential  key  success  factor,  appropriate  selection  and  prioritisation,  focus  on 
selected 
technologies,  clear  and  strong  coordination/governance/secretarial  support,  
extension  of  the  scope  of  early  dialogues  to  guidelines  on  technology  development,  and 
appropriate  stakeholder  involvement.  Reduced  duplication  of  efforts  and  costs,  better 
allocation of resources and last, but not least, the contribution to a faster and more equitable 
access  to  new  health  technologies  for  EU  patients  were  among  the  most  quoted  benefits.  
However  some  categories  of  stakeholders,  including  medtech  industry,  SMEs  and  some 
national authorities were mostly in favour of a voluntary cooperation.  

3. Bilateral meetings with stakeholders 

Commission  services  organised  senior  level  meetings  with  10  Member  States'  Ministries  of 
Health and HTA bodies interested in receiving clarifications and providing an early input to 
the  initiative.  Bilateral  meetings  took  place  at  the  request  of  all  interested  stakeholders:  
payers  (5  meetings),  patients'  and  consumers'  representatives  (8  meetings),  with  healthcare 
providers and academia representatives (8 meetings), as well as with industry representatives 
and their trade associations (20 meetings)215.  

4. Experts Consultation 

Following the publication of the Inception Impact Assessment, several meetings with Member 
States HTA experts were organised. Two closed sessions with Member States representatives 
to  HTA  Network  took  place  on  10  November  2016216  and  23  March  2017217.  In  addition, 
brainstorming sessions with members of the EUnetHTA Executive Board were also organised 
in December 2016, March and May 2017. These discussions were open and constructive, and 
showed engagement by HTA Network members and EUnetHTA experts to contribute to the 
development of the policy initiative. 

5. External events 

The initiative was presented during several meetings such as the EUnetHTA Forum (Brussels, 
October 2016),  the annual meeting of the International Society  for Pharmacoeconomics and 
Outcomes Research/ISPOR (Vienna, October 2016), and the European Health Forum Gastein 
(Bad  Gastein,  September  2016),  which  allowed  constructive  discussions  with  stakeholders 
(i.e. HTA experts, healthcare professionals, public health specialists, patients representatives, 
industry representatives) and represented good opportunities for raising awareness about the 
Commission's online public consultation. 

215 Minutes available at https://ec.europa.eu/health/technology_assessment/events_en#anchor3  
216 https://ec.europa.eu/health/sites/health/files/technology_assessment/docs/ev_20161110_mi_en.pdf  
217 https://ec.europa.eu/health/sites/health/files/technology_assessment/docs/ev_20170329_mi_en.pdf  

120 

 
 
                                                            
6. Conclusions  

The  consultation  activities  showed  that  there  is  broad  support  for  the  initiative  and  a  high 
level of stakeholder interest in concrete implementation of a pragmatic solution, from which 
will  benefit  not  only  public  authorities  or  industry  but  also  patients  and  healthcare 
professionals.  

A  large  majority  of  stakeholders  emphasise  that  EU  cooperation  beyond  2020  is  needed  to 
ensure  a  constant  exchange  of  information  and  knowledge  between  HTA  institutions  in 
Europe, to increase synergies between Member States, to streamline HTA methodologies, to 
increase transparency and evidence-based decision making, as well as business predictability. 
The possibility to access a larger number of HTA reports with less duplication of work and 
better  allocation  of  resources  by  HTA  bodies  was  highlighted.  Patients'  organisations, 
healthcare professionals and academia stress that EU cooperation can enhance access to added 
value  and  affordable  technologies  in  a  timely  manner  and  in  the  long  run  can  also  lead  to 
savings, improving resilience and contributing to the sustainability of health systems. Several 
stakeholders note that significant public resources have been invested in EU cooperation on 
HTA and all the results achieved so far should be capitalised to support sustainable healthcare 
systems  and  guarantee  equitable  access  to  technologies  with  added  value  to  all  patients  in 
Europe. 

While all representatives of public administrations are in favour of continuing EU cooperation 
on  HTA  beyond  2020,  some  indicate  a  preference  for  voluntary  cooperation  while  others 
support a system with mandatory elements (i.e. legal framework for EU cooperation on HTA 
to  streamline  interoperability  of  HTA  national  systems).  Most  contributors  highlight  that  in 
case of a mandatory system, uptake of joint work should be limited to clinical and technical 
matters, whereas assessment of non-clinical domains (e.g. economic, legal, ethical) should be 
carried  out  individually  or  jointly  by  interested  Member  States/HTA  bodies  on  a  voluntary 
basis. The idea of a phase-in approach was also raised.  

Citizens,  patients  and  consumers  representatives,  as  well  as  healthcare  providers  and 
academia  were  extremely  positive,  with  most  of  them  in  favour  of  a  collaboration  covering 
both  clinical and economic assessments. They underline the need  for involving patients and 
healthcare professionals in the HTA process, the need of transparency (e.g. summary of HTA 
reports  publicly  available,  including  criteria  and  rationale  for  evaluation),  and  last  but  not 
least the need to ensure independence of HTA bodies from industry and other interests. 

As  regards  industry,  pharmaceutical  industry  and  their  trade  associations  support  the 
harmonisation of European relative efficacy  assessments  at time of launch,  accompanied by 
an alignment at EU level of the evidence requirements between regulators, HTA bodies and 
payers.  Many  representatives  of  the  pharmaceutical  industry  advocate  for  voluntary 
participation  for  both  Member  States  and  manufacturers  until  the  process  of  joint  work  has 
proven  itself,  however  with  mandatory  uptake  of  joint  work.  It  was  stressed  that  economic 
assessments  should  remain  the  responsibility  of  Member  States.  Medical  technologies' 
operators  and  their  trade  associations  reiterate  the  importance  of  taking  into  account  the 
particularities  of  their  sector  and  the  need  for a Member  States-driven  approach (i.e.  timing 
and  selection  of  technologies  to  be  assessed  should  be  decided  by  HTA  bodies  and  not 
centrally  at  EU  level).  It  was  underlined  that  HTA  should  focus  on  products  that  are 

121 

 
 
 
 
 
 
 
innovative and address high  unmet  patient  needs in  disease areas where appropriate clinical 
and economic evidence has been or can be generated (e.g. transformative in-vitro diagnostics 
and medical devices). 

These  results  are  fully  taken  into  account  in  the  proposed  preferred  option  presented  in  the 
Impact Assessment, particularly with regard to: 
- subsidiarity (i.e. focus on joint early dialogues and joint clinical assessments, with economic 
assessments to be performed at national level), and 
-  proportionality  (i.e.  national  uptake  understood  as  implementation  of  joint  work  within 
national  HTA  processes,  addressing  the  differences  between  the  pharmaceuticals  and  the 
medical technologies sector in relation to the different market access path and the role HTA 
plays in the two sectors, progressive implementation of the product scope). 

122 

 
 
 
 
 
 
Annex III. Who is Affected by the Initiative 

The aim of this annex is to set out the practical implications of the preferred policy option 
for the main stakeholders affected by it.  

Who is 
affected? 

Public 
administrations 

Patients 

How are they affected? 

Main positive outcomes include: 

  Better evidence for national decision-makers (i.e.  due to high quality 
and  timely  joint  reports)  to  support  sustainability  of  national  health 
systems  and  foster  public  health.    Member  States  with  less  HTA 
capabilities  and  higher  pressure  on  their  healthcare  budgets  will 
particularly  benefit  from  such  evidence.  Furthermore,  focusing  joint 
reports  on  clinical  data  makes  them  relevant  to  all  decision-makers, 
without  affecting  national  competences  on  pricing  and  reimbursement 
decisions.  

  Cost savings and optimisation of resources. The foreseen work sharing 
is expected to result in cost savings for public administrations in the long 
run, also allowing for a better allocation of resources.  

  Pooling  expertise  and  enhanced  capacity  to  address  more  health 
technologies. HTA bodies in the EU will be able to specialise in different 
topics, rather than to keep a general profile of both their tasks and staff. 
Therefore,  the  existing  staff  is  expected  to  specialise  (e.g.  orphan 
medicines, medical devices), developing complementary expertise which 
would ensure the desired high quality of joint REA reports.  

The introduction of an EU system may be accompanied by an initial increase in 
costs  (mainly  human  resources)  related  to  the  initial  implementation  of  new 
procedures and methodologies at national level. However, this is expected to be 
compensated  by  the  work-sharing  arrangements  and  avoidance  of  duplication 
foreseen under the preferred policy option. Member States' HTA bodies carrying 
out  the  joint  assessments  as  authors  and  co-authors  would  be  remunerated  for 
their work.  The HTA bodies which will not be actively participating as authors 
and co-authors will benefit from the work produced by the EU system and could 
therefore save the relevant resources that they would have had to invest.  

This  is  expected  to  counterbalance  the  initial  increased  spending  due  to  the 
adaptation to the new EU system. 

  An EU HTA system would provide for a framework for the involvement 
of  patients  in  the  HTA  processes  at  national  and  EU  level  (i.e. 
common  procedures  for  involving  patients,  financial  support  to  cover 
participation of patients to the meeting of the expert Committees carrying 
out the joint work).  

  The  publication  of  the  joint  assessment/REA  reports  will  also  increase 
the transparency in of decision-making in relation to the availability of 
health technologies and the consistency of the clinical HTA assessments 
across  the  EU  as  they  will  be  based  on  common  procedures  and 
methodologies.  

123 

 
 
 
 
Healthcare 
professional 
and providers 

  An EU HTA system would provide for a framework for their 

involvement in the HTA process (i.e. common procedures for involving 
healthcare professional and providers).  

  Additionally, the publication of the joint assessment/REA reports would 
facilitate access to reliable, timely and objective information on medical 
technologies  allowing  for  better  informed  decisions  on  the  best 
treatment for their patients. 

Pharmaceutical 
industry 

The introduction of a joint REA with mandatory uptake will be accompanied by 
positive economic impacts for the pharmaceutical industry:  

 

Improved business predictability. A high quality and timely joint 
assessment report should reduce divergent decisions related to access on 
the market of new innovative technologies. 

  Potential positive impact on time to market. A high quality and timely 
joint assessment report has the potential to accelerate the next steps to be 
carried  out  at  national  level  (i.e.  appraisal,  pricing  and  reimbursement). 
In  this  regard,  quick  first  access  to  markets  is  particularly  relevant  for 
SMEs which depend on first access to first revenues.  
It will  reduce duplication of work through harmonisation of tools and 
methodologies and one submission for joint early dialogues/joint REA.  

 

  A  more  predictable  HTA  system  has  the  potential  to 

increase 

investments in R&D activities in Europe.   

Regarding costs, in an initial phase the introduction of joint REA at the time of 
market  authorisation  is  expected  to  lead  to  certain  costs  associated  with  the 
adaptation  to  the  new  EU  system.  However,  the  implementation  of  the  product 
scope  in  a  progressive  manner  during  the  first  years  of  the  new  EU  system  is 
expected to allow a smoother transition to the new system. 

Additionally, while the duplication of certain tasks/requirements for the staff of 
pharmaceutical  companies  in  EU  Member  States  will  be  reduced,  the  costs 
associated  with  national  pricing  and  reimbursement  procedures  will  persist. 
Overall, according to the GÖG-LSE study the, the pharmaceutical industry does 
not  expect  any  significant  savings,  but  the  gains  in  terms  of  business 
predictability are expected to overcome the costs related to the implementation of 
an EU system.  

124 

 
 
 
 
Medical 
technologies 
industry 

SMEs 

Cost  calculations  estimate  that  reduction  of  duplications  could  lead  to  tangible 
savings for the medical devices industry. 

When  representatives  of  this  sector  expressed  the  opinion  that  no  positive 
economic  impact  if  there  is  any  obligatory  REA  at  the  time  of  CE  marking, 
many  assumed  that  HTA  would  become  mandatory  for  all  type  of  medical 
technologies at the time of market launch.  

It is important to highlight that the preferred option foresees joint REAs only for 
medical  technologies  which  have  undergone  the  scrutiny  mechanism  in  the 
context  of  their  conformity  assessment  and  which  in  addition  have  been 
selected  by  Member  States  for  a  joint  REA  based  on  specific  criteria  (e.g.  EU 
wide  added  value,  potential  impact  of  the  technology  on  patients,  public  health 
and healthcare systems across the EU).  

Additionally, taking into account the more decentralised market access pathway 
for medical technologies, the timing of the joint REA will not be linked to the 
timing of the conformity assessment, i.e. it will not necessarily be at the time of 
market launch. This will avoid putting an additional burden on the manufacturers 
at market launch. 

Contrary to what is foreseen for pharmaceuticals, for which the  product scope is 
expected  to  be  fully  covered  over  time  (all  pharmaceuticals  identified  in  the 
scope  are  expected  to  go  through  a  joint  REA  at  the  end  of  transitional 
arrangements),  for  medical  technologies  the  system  will  remain  based  on  a 
prioritisation mechanism to ensure that joint REA are only performed on medical 
technologies  in  which  Member  States  identify  that  EU  cooperation  brings  an 
added value.  

Overall,  a  predictable  HTA  system  is  expected  to  re-direct  medtech  industry 
resources  towards  development  of  and  investment  in  products  which  would  for 
instance  address  unmet  medical  needs  and  lead  to  the  improvement  of  health 
outcomes for patients.  

In the area of pharmaceuticals SMEs are mostly engaged in the discovery phase 
of  new  molecules,  and  a  very  low  number  apply  for  central  marketing 
authorisation  and  undergo  HTA  process.  The  number  of  applications  for  joint 
REA from SMEs is expected to be very low, and since no fees are foreseen for 
this type of joint outputs, the compliance costs are expected to be low. As regards 
joint  early  dialogues,  no  fees  will  be  foreseen;  therefore  this  should  become  a 
very advantageous service provided to SMEs developing products in the area of 
pharmaceuticals.  

A  similar  treatment  would  be  applied  for  SMEs  in  the  field  of  medical 
technologies (no fee in case of voluntary application for a joint early dialogues, 
no  compliance  fee  in  case  of  a  joint  REA).  SMEs  manufacturing  medical 
technologies which will have to undergo the scrutiny mechanism in the context 
of  their  conformity  assessment  are  expected  to  benefit  from  the  improved 
convergence in procedures and methodologies and reduced duplications/parallel 
REA. 

125 

 
 
 
 
 
Annex IV. Analytical Models 

To support the analysis of the impact assessment three studies  were contracted. Firstly, two 
smaller studies were launched: 1) Mapping of HTA national organisations, programmes  and 
processes  in  EU  and  Norway218,  and  2)  Mapping  of  HTA  methodologies  in  EU  and 
Norway219.  Thereafter,  a  study  with  a  larger  scope  was  launched  with  the  following 
objectives: 

  To collect data and evidence and provide an in-depth analysis of what would happen 
in the absence of further action at EU level including its impacts (baseline scenario); 
  To  collect  data  and  evidence  and  provide  analysis  on  the  potential  impacts  of 

identified policy options for cooperation of the European Commission; 

  To collect relevant literature on HTA, with a specific focus on the European Union, to 

understand the way how it is used across EU Member States (MS). 

The  study  has  been  performed  by  a  consortia  consisting  of  SOGETI  Luxembourg  S.A.,  the 
Austrian  Public  Health  Institute  (GÖG)  and  the  London  School  of  Economics  (LSE) 
(henceforth the GÖG-LSE study).  

In the GÖG-LSE study, to establish the baseline scenario, a case study comprising a product 
sample of health technologies was analysed which included 20 pharmaceuticals, 15 medical 
devices and 5 “other technologies” (including complex health interventions). The study team 
collected  detailed  information  on  the  HTA-process  each  technology  underwent  in  Member 
States. The case study also analysed the influence of the regulatory framework on technology 
developers.  In  addition,  the  costs  of  performing  a  HTA  were  identified  for  both,  the 
technology  developer  and  the  HTA  body  through  desk  research  and  an  online  survey 
disseminated  to  all  stakeholder  groups,  where  mainly  public  administrations  and  health 
technology developers responded.  

To  analyse  possible  impacts  of  identified  policy  options  of  the  European  Commission,  a 
survey  was  performed  on  the  economic  and  social  impacts  of  the  identified  policy  options, 
complemented  by  focus  groups,  interviews  and  findings  from  literature  review.  The  study 
investigated the following impacts, for which one or more indicators were defined: 

The impacts investigated included economic (EC) and social health (SH) criteria: 

218 Mapping of HTA national organisations, programmes and processes in EU and Norway, 2017, DG SANTE 
Contract 17010402/2016/734820 (Annex VIII) 
219  Mapping  of  HTA  methodologies  in  EU  and  Norway,  2017,  DG  SANTE  Contract  17010402/2016/736040 
(Annex IX) 

126 

 
 
                                                            
 
EC1: Costs 
EC2: Administrative Burden 
EC3:  Competitiveness  of  EU  health  technology 
sector 
EC4: Innovation and Research 
EC5: 
Research 
EC6:  Functioning  of  the  internal  market  and 
competition 
EC7: Consumers and households 
EC8: Macroeconomic environment 

International  Trade 

Innovation  and 

SH1: Employment 
SH2:  Governance,  participation  and 
good administration 
SH3:  Access  to  social  protection  and 
health systems 
SH4: Sustainability of Health Systems 
SH5: Public Health  

*Environmental impacts were considered not to be relevant to the analysis at an early stage.  

The study also provides a description of the implementation mechanisms and an estimation of 
the costs based on data gathered from desk research (including data from existing agencies), 
and responses in relation to the online survey.  

127 

 
 
 
 
Annex V: Health Technology Sectors 

In  this  Impact  Assessment,  the  term  health  technology  is  understood  in  a  broad  sense, 
comprising  pharmaceuticals,  medical 
in  vitro 
diagnostics)220 and other technology-based tools for disease prevention, diagnosis or treatment 
used in healthcare.  

(medical  devices  and 

technologies 

This annex provides a description of the pharmaceutical and the medical technologies sectors 
as the two key health technology sectors, by giving an overview of the size of each sector, the 
actors,  products  and  regulatory  framework.  Both  sectors  are  highly  innovative  and  play  an 
important  role  in  the  European  growth  and  competitiveness.  The  pharmaceutical  sector  is 
characterised  by  stronger  concentration  of  actors  compared  to  the  medical  technologies, 
where  95%  of  the  companies  are  small  and  medium  enterprises  (SMEs).  The  regulatory 
approval process and market access path (including pricing and reimbursement decision) for 
pharmaceuticals is more centralised. The medical technologies sector is more heterogeneous 
in  terms  of  the  products  and  the  market  access  path.  When  a  pharmaceutical  arrives  on  the 
market after a lengthy, costly and risky research and development process it is protected by a 
patent.  This  is  different  for  medical  technologies,  where  a  quick  innovation  cycle  can  be 
observed due to the rather short lifecycle of products (18-24 months). Last but not least, the 
Health  Technology  Assessment  methodologies  have  been  primarily  developed  for  assessing 
pharmaceuticals; addressing the particular methodological challenges relevant to the medical 
technologies sector is still on-going.  

A  simplified  diagram  showing  the  HTA  step  within  the  lifecycle  of  pharmaceuticals  and 
medical devices in the context of the EU legal framework is presented below. As illustrated 
below,  HTA  typically  takes  place  following  pharmaceutical  market  authorisation  or  CE 
marking under the medical devices legislation. Enhanced cooperation on HTA as suggested in 
this impact assessment is fully coherent with the legislation on clinical trials (Regulation (EU) 
536/2014),  pharmaceuticals  (Regulation  (EC)  No  726/2004),  medical  devices  and  in  vitro 
diagnostics  (Regulations  (EU)  2017/745  and  2017/746)  and  the  Transparency  Directive 
Council Directive 89/105/EEC). 

220 Medical devices and in vitro diagnostics as defined by Regulation (EU) 2017/745 and Regulation (EU) 
2017/746. 

128 

 
 
 
 
 
 
 
 
                                                            
Pharmaceuticals 

Medical devices and in vitro diagnostics 

Figure 1. HTA step within the lifecycle of pharmaceuticals and medical devices 

I. Pharmaceutical Industry  

1. Sector overview 

The pharmaceutical sector in the EU employs approximately 800,000 people. It is one of the 
industries with the highest labour productivity. The  research and development investment is 
high in the sector, thus it plays an important role in the European competitiveness. 221 

The  pharmaceutical  sector  is  characterized  by  a  strong  R&D  activity  and  a  high  level  of 
regulation.  On  the  supply  side,  the  market  is  divided  between  originator  companies222  and 
generic manufacturers. The life cycle of a drug can be divided into 3 phases: 
1) From R&D to market launch 
2) Time on market under patent protection 
3) Expiration of patent and access to the market of the generic drug. 

1.1. Actors 

The following table shows the top 10 pharma companies in million EUR 2016. 

COMPANY 
Pfizer 
Novartis 
Roche 
Merk&Co 
Sanofi 
Gilead  Sciences 
Johnson & Johnson 
GlaxoSmithKline 
AstraZeneca 
Abbvie 

REVENUE 
$ 43 112 
$ 42 467 
$ 38 733 
$ 35 244 
$ 34 896 
$ 32 151 
$ 29 864 
$27 051 
$23 264 
$ 22 724 

221 Pharmaceutical Industry: A Strategic Sector for the European Economy. SWD(2014) 216 final/2 
222 Companies active in research, development, production and marketing of products protected by patents. 

129 

 
 
 
  
 
 
 
 
                                                            
1.2. Products223 

All  new  medicines  introduced  into  the  market  are  the  result  of  lengthy,  costly  and  risky 
research and development (R&D) conducted by pharmaceutical companies: 

o  By the time a medicinal product reaches the market, an average of 12–13 years would 

have passed since the first synthesis of the new active substance; 

o  The  cost  of  researching  and  developing  a  new  chemical  or  biological  entity  is 
estimated  at  €  1  926  million  ($  2  558  million  in  year  2013)  in  2016  (DiMasi  et  al, 
Journal of Health Economics, January 2016); 

o  On  average,  only  one  to  two  of  every  10  000  substances  synthesised  in  laboratories 
will  successfully  pass  all  stages  of  development  required  to  become  a  marketable 
medicine. 

2. The Regulatory Framework 

Medicinal products are (largely) regulated at EU-level throughout their lifecycle with the dual 
objectives  of  facilitating  their  free  movement  within  the  single  market  and  ensuring  a  high 
level  of  public  health  protection.  Directive  2001/83/EC  and  Regulation  (EC)  No  726/2004    
are considered the core pieces of legislation covering the placing on the market, production, 
labelling,  classification,  distribution  and  advertising  of  medicinal  products  for  human  use. 
They  are  supplemented  by  a  number  of  other  Acts  which  focus  on  a  particular  step  in  the 
product lifecycle or on specific types of medicinal products. The regulatory framework for the 
main  steps  in  this  lifecycle  is  outlined  below  with  a  particular  focus  on  marketing 
authorisation procedures due to their links with HTA. 

2.1 Clinical Trials 

Once  the  initial  research  phase  on  a  prospective  new  medicine  has  been  completed,  such 
products  need  to  undergo  clinical  trials,  which  are  governed  by  Regulation  (EU)  536/2014 
(which  replaces  the  former  Directive  2001/20/EC).  The  clinical  trials  legislation  lays  down 
rules on the protection of patients, informed consent, the manufacturing and clinical practice 
of  those  medicines  to  be  used  in  clinical  trials,  and  the  authorisation  of  trials  and  the 
information available on them. The regulation is designed to ensure a streamlined application 
procedure with defined deadlines. An EU clinical portal will provide for a single entry point 
for  sponsors  and  a  single  contact  point  by  Member  States.  When  making  an  application  a 
single set of documents will be needed and the applications will be assessed based on the new 
harmonised assessment procedure consisting of two parts – a joint assessment by all Member 
States  concerned  and  a second  part of  separate assessments  by  those  Member  States.  These 
new  rules  will  become  applicable  once  the  new  portal  and  database  run  by  the  European 
Medicines Agency are fully functional, which is expected in 2018.  

223 EFPIA Key data 2016. Link: the-pharmaceutical-industry-in-figures-2016.pdf 

130 

 
 
 
 
 
 
 
 
                                                            
2.2 Marketing Authorisation Procedures 

According to the rules governing medicinal products in  the EU, such  products may only  be 
placed  on  the  market  once  a  marketing  authorisation  has  been  issued,  either  by  a  Member 
State authority or at Union level. These authorisations are granted based on an assessment of 
the quality, safety and efficacy of the product in question and, in addition to a purely national 
authorisation  limited  to  one  Member  State,  can  be  issued  through  one  of  three  distinct 
procedures: 

1.  Centralised procedure; 
2.  Mutual recognition procedure; 
3.  Decentralised procedure. 

Whichever procedure is used the application is made using the Common Technical Document 
consisting  of  the  information  on  quality,  non-clinical  data,  and  clinical  data.  In  addition  to 
these standard procedures it should be pointed out that special rules exist for the authorisation 
of  medicinal  products  for  paediatric  use,  orphan  drugs,  traditional  and  herbal  medicinal 
products,  and  homeopathic  medicinal  products  which  provide  for  simplified  rules  for  the 
authorisation of such products.  

Centralised procedure 

The  majority  of  new  innovative  medicines  in  the  EU  are  authorised  via  the  centralised 
procedure.  This  procedure  is  governed  by  Regulation  (EC)  No  726/2004  and  allows 
applicants  to  obtain  a  marketing  authorisation  that  is  valid  throughout  the  EU.  It  is 
compulsory  for  medicinal  products  manufactured  using  biotechnological  processes,  for 
orphan medicinal products and for human products containing a new active substance which 
was not authorised in the EU before 20 May 2004 and which are intended for the treatment of 
AIDS, cancer, neurodegenerative disorder or  diabetes. The centralised procedure is optional 
for any other products containing new active substances not authorised before 20 May 2004 
or for products which constitute a significant therapeutic, scientific or technical innovation or 
for which a central authorisation is in the interests of patients or animal health at Union level. 

When  a  company  wishes  to  place  on  the  market  a  medicinal  product  that  is  eligible  for  the 
centralised procedure, it sends an application directly to the European Medicines Agency, to 
be  assessed  by  the  Committee  for  Medicinal  Products  for  Human  Use  (CHMP).  The 
procedure  results  in  a  Commission  Decision,  which  is  valid  in  all  EU  Member  States. 
Centrally  authorised  products  may  be  marketed  in  all  Member  States.  Full  copies  of  the 
marketing  authorisation  application  file  are  sent  to  a  rapporteur  and  a  co-rapporteur 
designated  by  the  competent  EMA  scientific  committee.  They  co-ordinate  the  EMA's 
assessment  of  the  medicinal  product  and  prepare  draft  reports.  Once  the  draft  reports  are 
prepared, they are sent to the CHMP, whose comments or objections are communicated to the 
applicant.  The  rapporteur  is  therefore  the  privileged  interlocutor  of  the  applicant  and 
continues to play this role, even after the marketing authorisation has been granted. 

The  rapporteur  and  co-rapporteur  then  assess  the  applicant's  replies,  submit  them  for 
discussion  to  the  CHMP  and,  taking  into  account  the  conclusions  of  this  debate,  prepare  a 
final  assessment  report.  Once  the  evaluation is  completed,  the  CHMP  gives  a  favourable  or 
unfavourable opinion as to whether to grant the authorisation. When the opinion is favourable, 

131 

 
 
 
 
 
 
 
 
it shall include the draft summary of the product's characteristics, the package leaflet and the 
texts proposed for the various packaging materials. 

The  time  limit  for  the  evaluation  procedure  is  210  days.  The  EMA  then  has  fifteen  days  to 
forward its opinion to the Commission. This is the start of the second phase of the procedure: 
the  decision-making  process.  The  Agency  sends  to  the  Commission  its  opinion  and 
assessment report, together with annexes containing: 

 
 

 

the summary of product characteristics (Annex 1); 
the particulars of the manufacturing authorisation holder responsible for batch release, 
the  particulars  of  and  the  manufacturer  of  the  biological  active  substance  and  the 
conditions of the marketing authorisation (Annex 2); and 
the labelling and the package leaflet (Annex 3). 

During  the  decision-making  process,  the  Commission  services  verify  that  the  marketing 
authorisation complies with Union law. The Commission has fifteen days to prepare a draft 
decision. The medicinal product is assigned a Community registration number, which will be 
placed on its packaging if the marketing authorisation is granted. 

The draft decision is then sent to the Standing Committee on Medicinal Products for Human 
Use.  Member  States  have  fifteen  days  to  return  their  linguistic  comments  and  22  days  for 
scientific  and  technical  ones.  This  procedure  is  conducted  in  writing  but  if  a  duly  justified 
objection is raised by one or more Member States, the committee holds a plenary meeting to 
discuss it. When the opinion is favourable, the draft decision is adopted via the empowerment 
procedure.  The  Commission  then  notifies  the  Commission  Decision  to  the  marketing 
authorisation  holder.  The  decision  is  then  published  in  the  Community  Register.  Marketing 
authorisations are valid for five years. Applications for renewal must be made to the EMA at 
least six months before this five-year period expires. 

132 

 
 
 
 
 
Fig. 2. Centralised procedure 

Mutual recognition procedure 

As laid down in Directive 2001/83/EC, the mutual recognition procedure is compulsory for all 
medicinal products to be marketed in a Member State other than that in which they were first 
authorised. Any national marketing authorisation granted by an EU Member State's national 
authority  can  be  used  to  support  an  application  for  its  mutual  recognition  by  other  Member 
States. 

The mutual recognition procedure is based on the principle of the mutual recognition by EU 
Member  States  of  their  respective  national  marketing  authorisations.  An  application  for 
mutual  recognition  may  be  addressed  to  one  or  more  Member  States.  The  applications 
submitted must be identical and all Member States must be notified of them. As soon as one 
Member  State  decides  to  evaluate  the  medicinal  product  (at  which  point  it  becomes  the 
"Reference  Member  State"),  it  notifies  this  decision  to  other  Member  States  (which  then 
become  the  "Concerned  Member  States"),  to  whom  applications  have  also  been  submitted. 
Concerned Member States will then suspend their own evaluations, and await the Reference 
Member State's decision on the product. 

This  evaluation  procedure  undertaken  by  the  Reference  Member  State  may  take  up  to  210 
days, and ends with the granting of a marketing authorisation in that Member State. It can also 
occur  that  a  marketing  authorisation  had  already  been  granted  by  the  Reference  Member 

133 

 
 
 
 
 
 
 
State. In such a case, it shall update the existing assessment report in 90 days. As soon as the 
assessment is completed, copies of this report are sent to all Member States, together with the 
approved  summary  of  product  characteristics  (SPC),  labelling  and  package  leaflet.  The 
Concerned  Member  States  then  have  90  days  to  recognise  the  decision  of  the  Reference 
Member  State  and  the  SPC,  labelling  and  package  leaflet  as  approved  by  it.  National 
marketing  authorisations  shall  be  granted  within  30  days  after  acknowledgement  of  the 
agreement. 

Should  any  Member  State  refuse  to  recognise  the  original  national  authorisation,  on  the 
grounds of potential serious risk to public health, the issue will be referred to the coordination 
group.  Within  a  timeframe  of  60  days,  Member  States  shall,  within  the  coordination  group, 
make  all  efforts  to  reach  a  consensus.  In  case  this  fails,  the  procedure  is  submitted  to  the 
appropriate EMA scientific committee (CHMP or CVMP, as appropriate), for arbitration. The 
opinion  of  the  EMA  Committee  is  then  forwarded  to  the  Commission,  for  the  start  of  the 
decision  making  process  (described  below).  As  in  the  centralised  procedure,  this  process 
entails consulting  various  Commission  departments  and  the  Standing  Committee  on  Human 
Medicinal Products. 

Decentralised procedure 

Introduced  by  Regulation  (EC)  No  726/2004,  the  decentralised  procedure  is  similar  to  the 
mutual recognition procedure in terms of procedural steps. The key difference is that there is 
no existing marketing authorisation in any Member State before an application is made. An 
identical application for marketing authorisation is submitted simultaneously to the competent 
authorities of the Reference Member State and of the Concerned Member States. At the end 
of the procedure, the draft assessment report, SPC, labelling and package leaflet, as proposed 
by  the  Reference  Member  State,  are  approved.  The  subsequent  steps  are  identical  to  the 
mutual recognition procedure. 

2.3 Manufacturing and distribution 

Once  a  marketing  authorisation  has  been  granted,  companies  then  require  a  manufacturing 
authorisation before medicinal products can be manufactured in the Union based on the rules 
laid  down  in  Directive  2001/83/EC  as  well  as  the  detailed  rules  on  Good  Manufacturing 
Practice  (GMP)  set  out  in  Directive  2003/94/EC.  Once  a  manufacturer  has  made  an 
application,  an  inspection  is  carried  out  prior  to  the  national  competent  authority  issuing  a 
GMP  certificate.  Applicants  are  assessed  on  the  suitability  of  their  premises,  their  technical 
equipment and control facilities, staff, and compliance with GMP including the use of active 
substances  complying  with  GMP.  A  manufacturing  authorisation  is  also  necessary  for 
companies importing medicinal products into the EU from third countries. 

Distribution  of  medicinal  products  is  governed  by  the  rules  on  Good  Distribution  Practice 
(GDP).  A  distribution  authorisation  is  necessary  in  order  to  distribute  medicinal  products. 
This allows either manufacturers to distribute products themselves or to sell products to those 
in possession of a distribution authorisation. In turn distribution authorisation holders can sell 
medicinal products to entities entitled by the MS to sell medicinal products to the public. The 
retail of medicinal products is regulated at Member State level.  

134 

 
 
 
 
 
 
 
 
 
2.4 Pharmacovigilance 

Once  medicinal  products  have  been  placed  on  the  EU  market  pharmacovigilance  is  used  to 
ensure  that  the  safety  of  medicinal  products  is  fully  monitored  and  that  action  is  taken  to 
reduce  the  risks  and  increase  the  benefits  of  such  products.  The  system  requires  data  from 
healthcare  professionals  working  with  medicinal  products  to  be  collected,  managed,  and 
evaluated  and  decisions  taken  where  necessary  to  protect  public  health.  When  granting  a 
marketing  authorisation,  certain  conditions  may  be  made  requiring  the  collection  of  further 
data post-authorisation, particularly on the efficacy of a product. Where safety issues do arise 
this can result in further action being taken including the suspension, withdrawal, revocation 
or  non-renewal  of  the  marketing  authorisation.  The  basic  rules  and  requirements  for 
pharmacovigilance are laid down in directive 2001/83/EC and Regulation (EC) No 726.2004.  

2.5 Market access 

In most Member States, pricing and reimbursement decisions for pharmaceuticals are taken at 
national  level  at  the  time  of  market  launch  (or  shortly  thereafter).  While  pricing  and 
reimbursement  are  a  Member  States  competence,  the  Transparency  Directive  (Council 
Directive  89/105/EEC)  defines  procedural  requirements  to  ensure  the  transparency  of  these 
processes for pharmaceuticals, including the setting of timeframes for decision-making. 

It  is  important  to  note  that  HTA  does  not  comprise  pricing  and  reimbursement  decisions. 
Rather,  the  role  of  HTA  is  to  provide  a  scientific  assessment  to  inform  evidence-based 
decision-making on pricing and reimbursement. In line with this distinction, the principle of 
subsidiarity and Article 168(7) TFEU, decisions on pricing and reimbursement are not within 
the scope of the initiative analysed in this impact assessment. Nevertheless, strengthened EU 
cooperation  on  HTA  can  be  considered  coherent  with  the  objectives  of  the  Transparency 
Directive in terms of supporting timely and transparent decision-making by Member States224. 

II. Medical Technology Industry 

1. Sector Overview 

There  are  about  25  000  medical  technology  companies  in  Europe.  About  95%  of  them  are 
Small and Medium-sized Enterprises (SMEs). The market is estimated to employ about 575 
000 people. Total sales amount to EUR 100 billion per year, of which 8 to 10% are invested 
in R&D activities225. On average, the European medical technology market has been growing 
by 4% per annum over the past seven years. Demand fell in 2009 due to the global economic 
crisis, resulting in a growth rate of only 1% in that year. The market recovered in 2010, but 
growth  rates  fell  back  in  2011226.  The  sector  is  expected  to  grow,  given  the  technological 

224 Note that this relates in particular to supporting decision-making on clinical aspects of HTA (compare policy 
option 3 and 4). 
225 Medical devices, DG GROW https://ec.europa.eu/growth/sectors/medical-devices_en 
226  WHO;  G.  Donahoe  and  G.  King  –  Estimates  of  Medical  Device  Spending  in  the  United  States,  F.S.A., 
M.A.A.A., AdvaMed, 2012 

135 

 
 
 
 
 
 
 
                                                            
advancements  and  improvements  in  healthcare,  combined  with  the  steady  increase  in  life 
expectancy rates over the last decades.   

Two main underlying characteristics of the industry is - a high variety of products and a quick 
innovation cycle227. There are more than 500, 000 medical technologies currently available on 
the market. Additionally, on average, products have a lifecycle of between 18 and 24 months 
before  an  improved  product  enters  the  market,  which  is  a  strong  driver  for  research  and 
innovation.228  

1.1. Actors 

Most of the 25 000 medical technology companies in Europe are based in Germany, the UK, 
Italy, Switzerland, Spain and France229.  

The following table shows the top 10 medical technology companies in 2015 in the world. 

COMPANY230 
1 Medtronic 

2 Johnson & Johnson (medical device 
segment) 
3 Philips Healthcare (Royal Philips 
Electronics) 
4 GE Healthcare(General Electric) 
5 Fresenius (medical care segment) 
6 Siemens Healthineers (Siemens) 
7 Cardinal Health  (medical segment) 
8 Becton, Dickinson (medical segment) 
9 Baxter (medical products segment) 
10 Stryker 

1.2. Products 

2015 REVENUE 
$28,833,000,000 

$25,137,000,000 

$19,817,952,415 

$17,639,000,000 
$16,739,425,600 
$11,652,847,873 
$11,395,000,000 
$10,282,000,000 
$9,968,000,000 
$9,946,000,000 

A  distinct  characteristic  of  the  medical  technology  industry  is  the  wide  variety  of  products 
covered  by  it.  There  are  more  than  500,000  registered  medical  devices  –  from  syringes, 
surgical  kits  and  hip  replacements,  to  pacemakers,  in  vitro  diagnostic  devices  and 
radiotherapy units.  

The rather high level of R&D activities in the field results in a constant flow of innovations in 
the  sector.  Accordingly,  the  highest  number  of  patents  filed  to  the  European  Patent  Office 
(EPO)  in  2015  came  from  the  medical  technology  industry  (7.8%)  –  more  than  the  ones  in 

227 MedTech Europe. The European Medical Technology industry in figures. 2016 
228 The European Medical Technology Industry – in figures 2016 
229 Ibid. 
230 Medical Design & Outsourcing annual look at the world’s 100 largest medical technology companies 2016 - 
2016 Top 100 Medical devices companies.pdf 

136 

 
 
 
 
                                                            
digital and computer communication.231The number of medical devices receiving CE marking 
in 2015 is estimated to be around 4,500. 232  

1.3 Classification of Products  

The  European  classification  depends  on  rules  that  involve  the  medical  device's  duration  of 
body contact, invasive character, use of an energy source, effect on the central circulation or 
nervous system, diagnostic impact, or incorporation of a medicinal product.  

The  initial  classification  of  medical  devices  in  the  EU  is  set  out  in  Article  IX  of  Council 
Directive  93/42/EEC.  Essentially,  they  fall  under  four  categories:  non-invasive  devices, 
invasive  medical  devices,  actives  devices  and,  last,  but  least,  special  rules,  which  include 
contraceptive,  disinfectant,  and  radiological  diagnostic  medical  devices).  Additionally, 
medical  devices  are  further  segmented  into  classes.  While  there  is  a  separate  classification 
scheme  for  IVDs  and  implantable  devices  do  not  follow  the  same  classification  system  as 
provided by the Medical Devices Directive, they are subject to similar requirements as Class 
III. There are three groups covering from low to high risk devices: 

  Class I – Provided non-sterile or do not have a measuring function (low risk) 
  Class I – Provided sterile and/or have a measuring function (low/medium risk) 
  Class IIa (medium risk) 
  Class IIb (medium/high risk) 
  Class III (high risk) 

2. Regulatory Framework 

The existing regulatory framework for the medical devices dates back to the 1990s.  The three 
Directives  (Council  Directive  90/385/EEC,  Council  Directive  93/42/EEC  and  Council 
Directive 98/79/EC) have been recently revised, as an array with issues with divergences in 
the interpretations and applications of the regulations, the technological progress, along with 
some  incidents  which  involved  malfunctions  of  medical  devices  highlighted  the  need  for  a 
review.  

In April 2017, two legislative proposals on medical and in-vitro diagnostic Regulations were 
adopted, replacing the existing Directives. Firstly, it is Regulation (EU) 2017/745 on medical 
devices, amending Directive 2001/83/EC, Regulation (EC) No 178/2002 and Regulation (EC) 
No 1223/2009 and repealing Council Directives 90/385/EEC and 93/42/EEC. Secondly, it is 
Regulation  (EU)  2017/746  on  in  vitro  diagnostic  medical  devices  repealing  Directive 
98/79/EC and Commission Decision 2010/227/EU. 

The new regulation was designed to ensure a high  level  of consistency of health  and safety 
protection  for  EU  citizens  using  medical  devices.  Moreover,  the  legislation  safeguards  the 
free  trade  of  products  throughout  the  Union,  and  responds  to  the  significant  scientific  and 
technological developments, which have occurred in the sector over the last two decades.  

231 MedTech Europe. The European Medical Technology industry in figures. 2016 
232 MedTech Europe. The European Medical Technology industry in figures. 2016 

137 

 
 
                                                            
The new regulations include a number of improvements to modernise the existing regulatory 
system. These improvements include:  

  Provision for a stricter ex-ante control for high-risk devices through a new pre-market 

scrutiny mechanism;  

  A reinforcement of the criteria for designation and processes for oversight of Notified 

 

 
 

Bodies;  
inclusion of some aesthetic devices which, under the scope of these Regulations, 
present the same characteristics and risk profile as analogous medical devices; 
introduction of a new risk classification system for in vitro diagnostic medical devices; 
improved transparency is assured through the establishment of a comprehensive EU 
database on medical devices; along with this a device traceability system is also 
introduced based on Unique Device Identification; 

  an "implant card" containing information about implanted medical devices for a 

 

patient; 
reinforcement of the rules on clinical evidence, including an EU-wide coordinated 
procedure for authorisation of multi-centre clinical investigations; 

  strengthening of post-market surveillance requirements for manufacturers; 
 

last, but not least, an improved coordination mechanisms between EU countries in the 
fields of vigilance and market surveillance.  

These new rules will only enter into force after three years for the Regulation on medical 
devices (spring 2020) and after five years for the Regulation on in vitro diagnostic medical 
devices (spring 2022). 

2.1 Post-market surveillance 

The enforcement of the harmonised legislation on medical devices is the responsibility of the 
relevant  authorities  in  the  Member  States.  They  are  expected  to  gather  record  and  analyse 
relevant  data  on  the  quality,  performance  and  safety  of  devices.  The  necessary  conclusions 
need to be drawn, and any preventive and corrective actions have to be determined.  

2.2 Market surveillance 

The new regulations provide clearer rights and obligation of market surveillance authorities, 
along  with  clearer  procedures  for  national  provisional  measures.  Member  States  are 
anticipated to exchange mutual information and control. 

2.3 Vigilance  

The  new  regulations  provide  the  establishment  of  an  EU  vigilance  portal.  Additionally, 
serious  incidents  are  expected  to  be  centrally  reported.  Following  such  cases,  field  safety 
corrective  actions  are  expected  to  be  taken.  Moreover,  trend  reporting  and  enhanced 
coordination between authorities are expected to take place under the new regulations. 

138 

 
 
 
2.4 Market Access  

The  market  access  process  presents  a  more  heterogeneous  picture  for  medical  technologies, 
compared  to  the  pharmaceutical  products.  Health  Technology  Assessments  for  medical 
technologies are currently carried out less frequently and on a smaller, less centralised scale 
(See Fig. 3).  The pricing and reimbursement judgement is typically taken in a decentralised 
manner, through local (hospital level) decisions or procurement, with limited input from HTA 
reports developed on a national, or centralised, level. Public procurement (instead of a central 
decision on reimbursement) is often the final step of the market access path.  

3. Other considerations 

For medical technologies, a number of issues should be taken into account when considering 
HTA:  

  Heterogeneity of the products, including multiple uses for the same device. 
 

Incremental  innovation  and  a  short  lifecycle.  The  quick,  incremental  innovations, 
which can take place within 18-24 month on the medical technologies, may affect the 
efficacy and cost of the device. 

  Learning  curve  of  the  device  user  (patient  or  health  professional)  means  that  the 

outcomes are less favourable during the period of training.  

  Wider  economic  and  organisational  implications,  such  as  required  trainings, 

operational costs or savings due to shorter hospitalisation. 

  Availability  of  evidence;  randomised  controlled  trials  can  be  more  challenging  to 

design, due to the learning curve, difficulties in blinding or randomisation.  

  For  IVDs,  there  are  two  further  challenges.  Firstly,  the  value  of  improved  diagnosis 
cannot  be  separated  from  the  value  of  the  treatment  determined  by  the  diagnosis. 
Secondly, technologies may have multiple uses. 233 234 235  

Such  methodological  challenges  were  pointed  out  in  a  number  of  submissions  in  the  public 
consultation (e.g. COCIR, Siemens, etc.)  

233 Rummel, P, Hawlik, K, Wild, C. Health Technology Assessments on Medical Devices in Europe. Final 
Report. LBI-HTA Rapid Assessment Nr. 12; 2016. Vienna: Ludwig Boltzmann Institute for Health Technology 
Assessment. 
234 Drummond, M., Griffin, A. and Tarricone, R. (2009), Economic Evaluation for Devices and Drugs—Same or 
Different?, Value in Health, 12: 402–404. 
235 MedTech and COCIR contributions to the open public consultation 

139 

 
 
                                                            
Figure 3. Schematic market access process of health technologies 

140 

 
 
 
 
 
Annex VI. European Cooperation on HTA 

The EU Cooperation on Health Technology Assessment (HTA) has had a long history, from 
the  first  main  steps  in  the  beginning  of  the  1990's  through  different  networking  and 
coordination projects to the largest ever Joint Action funded by the Public Health Programme, 
the EUnetHTA Joint Action 3. To date, over EUR 50 million will have been invested in the 
EU cooperation jointly by Member States and the EU by 2020. This Annex summarises the 
main elements of the projects and cooperation conducted so far.  

PUBLIC HEALTH PROGRAMME 
  EUR-ASSESS (1994-1997) 
  ECHTA-ECHAI (1999-2001) 
  EUnetHTA project (2006-2008) 
  EUnetHTA Joint Action 1 (2010-2012) 
  EUnetHTA Joint Action 2 (2012-2015) 
  EUnetHTA Joint Action 3 (2016-2020) 

FP 7 RESEARCH PROGRAMME 

  AdhopHTA  (7th Framework Programme) 
  MedtecHTA (7th Framework Programme) 
 
Integrate-HTA (7th Framework Programme) 
  Advance-HTA (7th Framework Programme) 

INNOVATIVE MEDICINES INITIATIVE  

  ADAPT SMART (Innovative Medicines Initiative)  
  GET REAL (Innovative Medicines Initiative) 

PUBLIC HEALTH PROGRAMME  

EUR-ASSESS (1994-1997) 

Between  1994  and  1997  the  EUR-ASSESS  project  took  place  with  the  following  aims:  to 
improve methods of priority setting, to develop and formulate HTA methodologies, to ensure 
that effective dissemination strategies were being used throughout European agencies, and to 
improve decision making by stimulating wider use of technology assessments.  

All the then 15 Member States and Switzerland took part in the project and it was concluded 
that  there  was  a  need  to  promote  the  field  of  HTA,  improve  its  use  and  impact  in  decision 
making and that further EU cooperation was needed. One identified example of the need of 
further  cooperation  was  duplication  of  effort;  where  it  was  exemplified  that  certain 
technologies  could  have  up  to  10  different  assessments  with  limited  coordination  between 
different HTA agencies. 

The European Collaboration for Assessment of Health Interventions-Health Technology 
Assessment (ECHTA/ ECAHI) (1999-2001) 

Following  the  EUR-ASSESS,  with  the  ECHTA/ECAHI  (1999-2001)  project,  the  ambition 
level increased further. The project assembled 6 different working groups on different aspects 
of HTA and possible ways of cooperation. The project had the following aims: 

141 

 
 
 
o  To  assess  health  promotion  and  disease  prevention  activities  in  terms  of 
benefits, risks and economic, social and ethical implications as a complement 
to community health indicators. 

o  To develop systems for routine exchange of information between programmes 

on: 

  Emerging technology issues 
  Priorities for future evaluation 
  Conduct  and  timing  of  ongoing  evaluations,  including  findings  from 

evaluations. 

o  To identify possible joint assessments and to co-ordinate findings and existing 

resources within the community to support joint assessments. 

o  To  develop  and  disseminate  best  practice  in  undertaking  and  reporting 

assessments. To identify needs for methodological development. 

o  To  develop  and  co-ordinate  education  and  support  networks  for  individuals 
and organisations undertaking or using assessment of health interventions. To 
identify needs in the field and assist in the establishment of new provisions. 
o  To  identify  and  share  successful  approaches  to  link  findings  of  assessments, 

their contribution to health indicators and health care decision-making. 

It was concluded that a more sustainable network was needed to support the EU cooperation 
on HTA and that project based solutions would not be adequate to proceed further. Therefore, 
there was a call for a permanent coordination secretariat funded by the European Commission 
with a caveat that it should respect the subsidiarity principle.  

EUnetHTA project (2006-2008) 

The  project was  led  by the  National  Board  of  Health  of  Denmark,  Danish  Centre  for  HTA. 
The  EUnetHTA  network  collaboration  was  initiated  in  2006  with  the  EUnetHTA  project  to 
connect  public  national  health  technology  assessment  (HTA)  agencies,  research  institutions 
and  health  ministries,  enabling  an  affective  exchange  of  information  and  support  to  policy 
decisions  by  Member  States.    The  concept  of  the  (paper  based)  HTA  Core  Model  and  the 
framework for producing and sharing structured HTA information were developed. 

EUnetHTA Joint Action 1 (2010-2012) 

The  EUnetHTA  Joint  Action  1  (JA1)  was  led  by  Danish  Health  and  Medicines  Authority 
(DHMA)  and  involved  33  collaborating  partners  from  26  EU  Member  States,  Norway  and 
Croatia.  The  budget  of  JA1  was  6  million  EUR.  EUnetHTA  JA1  built  on  the  methods  and 
tools developed by the earlier EUnetHTA project (2006-2008) and EUnetHTA Collaboration 
2009. JA1 was a voluntary, time-limited initiative with a defined work plan. 

The  JA1  developed  a  background  review  and  a  HTA  Core  Model  for  rapid  Relative 
Effectiveness  Assessment  of  pharmaceuticals,.  It  also  developed  the  POP  (Planned  and  On-
going Projects) database which in 2011 turned into an online tool. The JA also collaborated 
with EMA on the improvement of European Public Assessment Reports (EPARs), which are 
the full scientific assessment report published by EMA for every medicine granted a central 
marketing  authorisation.  The  JA  also  signed  a  Memorandum  of  Understanding  with 
INAHTA, the International Network of Agencies for Health Technology Assessment, which 
links 52 Member agencies working with HTA, in 29 countries. 

EUnetHTA Joint Action 2 (2012-2015) 

142 

 
 
EUnetHTA  Joint  Action  2  (JA2)  was  led  by  the  National  Board  of  Health  of  Denmark, 
Danish Centre for HTA and involved 69 organisations in 31 countries across Europe. A total 
number of 49 government-appointed organisations from 28 EU Member States and Norway  
participated in the work. The budget of the JA2 was 9,5 million EUR.  

The JA2 tested the tools and methodologies developed in the first EUnetHTA JAs through the 
cross-border  HTA  pilots.  The  aim  was  to  generate  evidence  on  the  costs,  quality  and 
feasibility of European cooperation as applied to concrete assessment projects. 

Through  joint  work  the  partners  produced  approximately  20  joint  reports,  including  REA 
(focusing  on  the  clinical/therapeutic  added  value)  and  Full  HTA  (including  assessment  of 
economic and organisational aspects). The cooperation also facilitated over 20 early dialogues 
between technology developers and HTA bodies, which help industry to design the studies in 
terms of regulatory and HTA requirements. 

EUnetHTA Joint Action 3 (2016-2020)236 
The EUnetHTA Joint Action  3 (JA3)  with  a budget  of 20  million EUR is  led by the Dutch 
National Healthcare Institute (ZIN). JA3 comprises around 80 HTA bodies from all Member 
States,  Norway  and  Switzerland.  The  challenges  have  been  the setup of  a  new  coordinating 
office  and  the  involvement  of  many  new  organisations  (80  versus  69  in  previous  Joint 
Actions). 

Under  JA3  further  progress  is  expected  from  the  previous  Joint  Actions  since  the 
collaboration foresees 80 joint reports and 35 early dialogues by 2020. Increased uptake of the 
joint work at national level is also an important aim of the Joint Action. In addition, JA3 will 
also perform a revision of current guidelines, models, methodologies and other tools, as well 
as the development of new ones, with the aim of facilitating HTA collaboration at EU level 
beyond the end of the project in 2020. 

FP 7 FRAMEWORK PROGRAMME (2007-2013) 

Through the FP7 work programme (2007-2013) managed by DG RTD, several projects were 
launched  focusing  on  developing  methodologies,  tools  and  guidelines  on  different  topics 
related to HTA which started 2013 and were finalised in 2015. These were:  

AdHopHTA – Adopting hospital-based Health Technology Assessment in Europe237 

The  project  AdHopHTA  aimed  to  bolster  the  use  and  impact  of  high-quality  health 
technology  assessment  (HTA)  in  hospital  settings.  The  ultimate  goal  was  to  facilitate  the 
adoption  of  health  technologies  with  proven  value  in  hospitals  and  to  keep  costly  pseudo-
innovations without proven benefit at bay.  

AdHopHTA findings and results have been collected in a “handbook for hospital-based HTA 
(HB-HTA)” that includes a set of guiding principles for good practices in HB-HTA units. The 
handbook  has  an  accompanying  toolkit  to  put  it  into  practice.  The  project  has  developed  as 
well a database of hospital-based HTA reports produced by the project partners. 

236 http://www.eunethta.eu/  
237 http://www.adhophta.eu/  

143 

 
 
 
                                                            
INTEGRATE-HTA238  
The  INTEGRATE-HTA  project  developed  conceptual  and  methodological  guidance  for  a 
comprehensive,  patient–centered,  and  integrated  assessment  of  complex  technologies.  The 
assessment  comprises  effectiveness  as  well  as  economic,  socio-cultural,  ethical,  and  legal 
factors and takes into account patient-specific factors, context, and implementation issues. Its 
starting point is the perspective of the stakeholders involved, palliative care has been chosen 
as a case study. 

MedtecHTA239 

The  general  objective  of  MedtecHTA  was  to  identify  improvements  of  HTA  methods  by 
making  evaluation  of  medical  devices  more  comprehensive  and  by  acknowledging 
complexities rising from their integration into clinical practice. The project aims at filling the 
gap  on  the  current  research  debate  on  the  challenges  to  the  available  methodological 
framework for HTA when applied to medical devices.  

Advance HTA240   

The objective of ADVANCE-HTA project was to contribute to the advancement of HTA by 
addressing  areas  of  intense  methodological  debate  (value  for  money,  value  assessment, 
quality  of  life  measurement,  rare  and  orphan  diseases,  HTA  for  selected  medical  devices, 
HTA in emerging settings). Ultimately, it aims to bring about improvement in HTA methods 
which  can  be  taken  further  by  competent  authorities  at  national  and  supra-national  levels 
towards a common understanding of choices in healthcare decision-making.  

INNOVATIVE MEDICINES INITIATIVE (IMI) 

The Innovative Medicines Initiative (IMI) is Europe's largest public-private partnership with a 
€2 billion budget funded equally between the European Commission and industry, aiming to 
improve  the  drug  development  process  by  supporting  a  more  efficient  discovery  and 
development  of  better  and  safer  medicines  for  patients.  IMI  is  since  2015  undertaking  two 
projects relevant to the EU cooperation on HTA.  
GetReal241 
GetReal  aims  to  show  how  robust  new  methods  of  Real  World  Evidence  (RWE)  collection 
and synthesis could be developed and considered for adoption earlier in pharmaceutical R&D 
and the healthcare decision making process. This will require companies, healthcare decision 
makers and other stakeholders to work together to generate a consensus on best practice in the 
use of RWE in regulatory and reimbursement decision-making. 

It  is  expected  that  alternative  evidence  generating  strategies  will  deliver  more  focused 
research in pharmaceutical research and development , and allow healthcare decision makers 
to be more certain when providing patients with access to new treatments. GetReal is carrying 
out  work  to  develop  intelligence,  evidence,  tools,  techniques  and  training  to  realise  the  full 
potential of RWE. 

238 http://www.integrate-hta.eu/  
239 http://www.medtechta.eu/wps/wcm/connect/site/medtechta/home  
240 http://www.advance-hta.eu/  
241 http://www.imi-getreal.eu/  

144 

 
 
 
                                                            
Accelerated Development of Appropriate Patient Therapies (AdaptSMART)242 

AdaptSMART  aims  to  establish  a  platform  for  coordinating  activities  related  to  'Medicines 
Adaptive  Pathways  to  Patients'  (MAPPs)  and  enable  stakeholder  dialogue  in  this  field.  The 
main aims of the project are the following:  

 

Identify  the  scientific  challenges  and  opportunities  related  to  the  implementation  of 
MAPPs  and  foster  the  aligned  understanding  of  consortium  members  and  their 
constituents. 

  Support  new  IMI2  research  and  innovation  actions  by  facilitating  the  inclusion  of 
MAPPs enablers, tools and methodologies to address its challenges and opportunities. 
  Conduct horizon scanning and research on key topics to produce actionable advice and 
recommendations for IMI and other stakeholders to further the broad implementation 
and adoption of MAPPs. 

  Distribute  findings,  key  discoveries  and  case  studies  from  ongoing  or  completed 
MAPPs pilot projects, creating a MAPPs repository of knowledge and opportunities. 

242 http://adaptsmart.eu/  

145 

 
 
 
 
                                                            
Annex VII: International Outlook  

While  Europe  is  generally  considered  a  leading  example  in  applying  HTA  in  decision-
making, it is interesting to note developments in other parts of the world.  

Most developed and many developing countries are implementing HTA systems. Most OECD 
countries have national agencies responsible for HTA, although they have varying capacities, 
institutional  settings,  scope  and  mandates.  In  the  majority  of  cases,  HTA  is  used  to  inform 
coverage  and  pricing  decisions.  In  this  regard,  when  it  comes  to  pharmaceuticals,  HTA 
agencies  only  provide  scientific  assessment,  while  the  particular  government,  third-party 
payers  or  joint  associations  of  medical  bodies  make  the  final  coverage  and  pricing 
decisions243. 

HTA is  often, yet not  always  used  for medical technologies in  OECD states. Depending  on 

243 Structural Policy Country Notes: Medium-term Policy Challenges; Southeast Asian Economic Outlook 2013, 
OECD  https://www.oecd.org/dev/49954247.pdf  

146 

 
 
                                                            
 
the type of medical devices, HTA is used to inform coverage decisions relative to either the 
medical  device  itself  (e.g.  implantable  devices)  or  to  diagnostic  and  therapeutic  procedures 
using  the  device  (e.g.  imaging  or  surgery).  According  to  a  recent  study,  carried  out  by  the 
OECD244,  two-thirds  of  its  member  countries  use  HTA  to  make  decisions  on  devices  or 
interventions, whether systematically or in "some circumstances". 

Looking at particular countries, Canada is one to offer a positive international example in the 
realm of HTA. The country,  which has  federal structure, consists of three territories and 10 
provinces.  Before  the  2000s  the  HTA  processes  in  Canada  were  decentralised  –  each 
jurisdiction was  performing  its  own  HTA.  This  led  to  significant  discrepancies  between  the 
sheer numbers and types of drugs assessed in the different parts of the country.  In an attempt 
to reduce the differences between the healthcare services Canadians were receiving based on 
where they lived, Canadian Agency for Drugs and Technologies in Health (CADTH) was set 
up245246.  It  is  a  national  organisation,  where  federal,  provincial  and  territorial  healthcare 
assessments are made. Quebec is the only territory which is does not participate in CADTH 
due  to  constitutional  provisions.  Consequently,  Canada  managed  to  reduce  the  divergences 
between the different provinces. In brief, the move towards harmonisation in Canada has met 
its objective to a large degree thanks to the establishment of the Canadian Agency for Drugs 
and Technologies in Health and by establishing common HTAs.  

Australia has also a well-established HTA system comparable to the ones in place in several 
Member States, i.e. based on independent body providing recommendations to the pricing and 
reimbursement  authority.  The  assessment  of  pharmaceutical  and  other  technologies  is 
conducted  by  a  specialised  agency,  the  Medical  Services  Advisory  Committee,  and, 
increasingly by insurers.247  It is interesting to note that also Australia regularly undertakes a 
review  of  its  HTA  system  to  reduce  duplication  and  fragmentation  of  the  processes  which 
have been identified as having cost and time implications for economic stakeholders, patients 
and administrations.248 

In USA, due to the very different nature of healthcare system, based mainly on many private 
payers  providing  care  to  its  members,  HTA  is  equally  fragmented.  Providers  of  care  may 
perform  directly  of  via  academic  institutions,  evidence  reviews  or  comparative  analysis, 

244 Structural Policy Country Notes: Medium-term Policy Challenges; Southeast Asian Economic Outlook 2013, 
OECD  https://www.oecd.org/dev/49954247.pdf   
245 CADTH website accessed 27 July 2017: https://www.cadth.ca/about-cadth/who-we-are/history  
246 Science-Matrix, Evalution of CADTH, December 2016 : 
https://www.cadth.ca/sites/default/files/pdf/2016%20CADTH%20Evaluation.pdf  
247 Healy J, Sharman E, Lokuge B. Australia: Health system review. Health Systems in Transition 2006; 8(5): 1–
158. 
248 Australian Government-Productivity Commission (2015) Efficiency in Health. Available at: 
http://www.pc.gov.au/research/completed/efficiency-health/efficiency-health.pdf    

147 

 
 
                                                            
however  the  systems  is  not  really  comparable  with  the  ones  in  place  in  most  of  European 
countries249.   

In  South  Korea,  HTAs  were  first  introduced  in  the  1990s  within  the  National  Health 
Insurance (NHI). Swiftly increasing expenditures for healthcare have been a challenge of the 
NHI,  which  considered  health  technology  management  as  a  cost  controlling  measure. 
Currently, the HTA process is governed by a governmental committee within the Ministry of 
Health,  Welfare  and  Family  Affairs  (MIHWFA).  It  comprises  of  twenty  members  who 
technically  supported  by  the  HTA  centre  created  within  the  National  Health  Insurance 
structure.  The  institutionalisation  of  HTA  in  South  Korea  has  been  driven  mainly  by  the 
requirements  of  the  NHI.  It  has  manifested  both  strengths  as  well  as  weaknesses250.  Most 
recently  the  South  Korean  government  is  establishing  a  new  organization  for  HTA, 
independent from the NHI. 

Another  proof  for  the  worldwide  interest  in  HTA  has  been  established  by  international 
scientific societies, such as  ISPOR (International Society  for Pharmaceutical and Outcomes 
Research) or HTAi (Health Technology Assessment international). They work to improve the 
quality of HTAs and their role in decision-making. 

249 European Obervatory on Health Systems and Policy (2013) United States of America. Health system review. 
Vol. 15 No. 3 2013. Available at: http://www.euro.who.int/__data/assets/pdf_file/0019/215155/HiT-United-
States-of-America.pdf  
250 Health Technology Assessment in South Korea, Chang-yup Kim (2009) International Journal of Technology 
Assessment in Health Care, 25:Supplement 1, 219-223 https://www.cambridge.org/core/services/aop-cambridge-
core/content/view/S0266462309090667   

148 

 
 
 
 
                                                            
Annex VIII. Earlier Market Access Calculations 

One of the proposed policy options (PO 4) in the Inception Impact Assessment on Strengthening the 
EU  cooperation  on  Health  Technology  Assessment  foresees  a  EU  'joint  REA'  for  all  innovative 
pharmaceuticals,  which  is  available  at  the  time  of  the  marketing  authorisation  by  EMA.  This  joint 
REA  has  the  potential  to  speed  up  the  market  access  process  for  innovative  pharmaceuticals.  The 
extent of this time gains have been estimated at 2-6 weeks by the CRA/EFPIA study251. The following 
calculation aims to estimate the monetary gains from the industry's perspective of this earlier access.  

Methods: 

The calculation was done for a cohort of pharmaceuticals that were launched in quarter 2 2008  

The  total  turnover  at  manufacturer  price  newly 
launched  non-generic  products  (13 
pharmaceuticals) was extracted from IMS aggregated across all available countries (covering most of 
the  EEA)  and  retail/hospital  sectors  for  Q1  2008-  Q3  2016  (column  'current  revenues').  The  cohort 
was  defined  as  non-generic  medicinal  products  for  which  no  sales  data  were  reported  in  the  first 
quarter  of  2008  and  sales  data  were  reported  for  all  subsequent  quarters.  This  cohort  concerns  13 
medicinal  products  (as  distinguished  in  the  data-set  by  their  international  product  name).  All  13 
products are originator products (no biological products were present in this cohort). 

The  hypothetical  revenues  of  a  1  month  quicker  market  access  was  calculated  (column  `1  month 
quicker  market  access  revenues')  for  the  first  12  years  following  market  launch.  It  can  be  assumed 
after 12 years the concerned product  markets would be genericised.  Revenues for the first  8.5 years 
(34 quarters) were directly taken from the IMS database. For the remaining 3.5 years (14 quarters) it 
was assumed revenues had plateaued at the observed level of the 34th quarter following market launch 
(available data confirm that revenues had stabilized after 7 years at an aggregate turnover of around 
EUR 400 million). Revenue flows were discounted to their 2008 quarter 1 value assuming an assumed 
(annual) 2% inflation rate. A sensitivity analysis with 1% discount rate gave similar results (0.97 % 
instead of 1.06%).  

It  was  assumed  that  such  an  earlier  HTA  report  does  not  affect  the  length  of  the  subsequent  steps 
(appraisal, pricing negotiations, etc.).  

Results:  

For  the  selected  products  launched  in  one  quarter,  the  one  month  earlier  access  means  EUR 
130.315.010  additional  revenue  over  the  12  years;  which  is  a  1  %  increase.  A  similar  gain  can  be 
expected for all innovative products launched.  

Calculations:  

251 EFPIA/ Charles River Associates, 2017, Assessing the wider benefits of the EU’s proposal on strengthening 
cooperation on health technology assessment from the industry perspective 

149 

 
 
                                                            
150 

YearQuarterCurrent revenues1 month quicker market access revenues0002.508.758117.489.10713.688.9771225.959.88133.207.7041347.467.77151.566.1091459.467.65164.219.1132573.357.96678.720.8972689.005.03294.878.12027106.097.741111.107.49228120.528.816125.103.80939133.590.794149.952.111310181.772.621193.075.711311214.616.761226.704.152312249.639.987251.761.445413254.740.105255.008.146414254.282.245258.785.786415266.470.396247.667.150416209.023.290215.210.439517226.460.826234.655.215518249.804.230274.252.199519321.552.295318.990.281520312.316.250314.258.464621316.571.768317.637.486622318.189.769325.051.358623337.101.525338.531.931624339.706.802334.925.638725323.756.529329.688.632726339.866.107335.953.804727326.508.758327.923.923728329.120.849333.386.936829340.230.571340.792.139830340.226.754344.143.985831350.240.229343.587.787832328.651.826336.365.295933350.054.936350.874.201934350.771.875350.771.875935349.039.615349.039.615936347.315.910347.315.9101037345.600.717345.600.7171038343.893.995343.893.9951039342.195.701342.195.7011040340.505.794340.505.7941141338.824.233338.824.2331142337.150.976337.150.9761143335.485.982335.485.9821144333.829.210333.829.2101245332.180.620332.180.6201246330.540.172330.540.1721247328.907.825328.907.8251248327.283.539327.283.539total 12.827.396.35212.957.711.362difference between current and earlier revenues130.315.010percentage101,016% 
 
 
 
Annex IX. Implementation Mechanisms and Policy Options 

For the five policy options, the following five implementation mechanisms were considered.  

  Project-based coordination 
  Member States (MS) secretariat 
  European Commission (EC)  secretariat 
  Existing EU agency 
  New EU Agency 

In one instance, for Policy Option 2 Project-based cooperation on HTA activities, only one 
implementation mechanism was suitable, the project-based cooperation.  

For  the  policy  options  foreseeing  a  permanent  mechanism  (PO  3-5),  more  than  one 
implementation  mechanism  is  conceivable.  Still  even  for  these  options,  the  implementation 
mechanisms are not freely interchangeable. For instance, more centralised production of the 
joint output requires more centralised structure. Also, policy options requiring the work 15-35 
full  time  staff  cannot  be  hosted  by  an  independent  agency.  The  following  table  shows  the 
conceivable pairing of the implementation mechanism per policy option, based on input from 
Member  States  experts,  stakeholder  consultation,  LSE-GOG  study  and  experience  with 
EUnetHTA Joint Actions.  

Scoring table – Selection of governance mechanisms per policy option (based on input 
from MS experts, stakeholder consultation, LSE-GÖG study and experience with EUnetHTA 
Joint Actions) 

Project-
based 
coordination 

MS  
secretariat 

EC  
secretariat 

Existing  
EU agency 

New EU 
Agency 

PO2 
PO3 
PO4 
PO5 
Observations 

Suitable 
implementation 
mechanism for 
voluntary 
cooperation 

Suitable for EU 
cooperation 
limited to some 
joint outputs, 
however with 
challenges 
related to the 
selection of the 
MS hosting the 
secretariat and 
sustainability of 
financing  

Suitable for EU 
cooperation 
encompassing 
most types of 
joint outputs (i.e. 
common tools 
and 
methodologies, 
horizon 
scanning, ED, 
REA) especially 
in case of a 
phase-in 
approach 

Suitable for EU 
cooperation 
encompassing 
most types of 
joint outputs (i.e. 
common tools 
and 
methodologies, 
horizon 
scanning, ED, 
REA) 

Suitable for EU 
cooperation 
encompassing 
all type of joint 
outputs, 
including Full 
HTA, for which 
no existing EU 
agency has 
appropriate 
expertise 

151 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Legend 
Most adequate  
Not adequate 
For the sake of calculations and presentation of the findings, the study paired each policy 
option with one implementation mechanism.  

152 

 
 
 
 
 
Annex X. Costs of Joint Outputs and Overall Costs per Policy Option and 
Implementation Mechanisms 

Table 1. Costs of joint outputs per POs and implementation mechanism (Amount in 1000 EUR) 
(Adapted from tables 20, 53 and 56 in the GÖG-LSE Study) 

Type of Costs 

Costs of joint outputs per POs and implementation mechanism (Amount x1000 
EUR) 

PO2 

PO3 

Implementation 
mechanisms 

Staff 

Estimated Joint outputs 

Project-
based 
coordinati
on 

15 FTEs 

13 ED 
11-15 REA 

MS 
secretariat 
(cat 1 MS) 

EC 
secretaria
t 

EC 
secretaria
t 

14 FTEs 

40 ED 

PO4 

Existing 
EU 
agency 

35.5 FTEs 

40 ED 
65 REA 

New  
EU Agency 

Uptake of joint outputs 

voluntary 

mandatory 

mandatory 

Costs for Common tools, 
templates and 
methodologies 
(Maintenance) 
Costs for Common tools, 
templates and 
methodologies 
(Development) 
Costs for Joint Early 
Dialogues 
Costs for Joint REA 

210.0 

Included in implementation mechanism 

300.0 

300.0 

300.0 

596.3 
1,598.3  

1,834.8 
N/R 

Costs for Joint full HTA 

N/R 

N/R 

Total costs of joint 
outputs 

2,704.6 

2,134.8 

1,834.8 
6,821.6 

N/R 

8,956.4 

153 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Table 2. Overall costs per POs and implementation mechanism (Amount x1000 EUR) (Adapted from 
tables 20, 52, 53 and 56 in the GÖG-LSE Study) 

Overall costs per POs and implementation mechanism (Amount x1000 EUR) 

PO2 

PO3 

PO4 

Implementation 
mechanisms 

Staff 

Estimated Joint outputs 

Project-
based 
coordinati
on 
15 FTEs 

13 ED 
11-15 REA 

MS 
secretariat 
(cat 1 MS) 

EC 
secretaria
t 

14 FTEs 

40 ED   

EC 
secretariat 

Existing 
EU agency 

New  
EU Agency 

35.5 FTEs 

40 ED 
65 REA 

Uptake of joint outputs 

voluntary 

mandatory 

mandatory 

Type of costs 

Start-up costs 

Costs running secretariat 

Costs joint outputs 

Total costs 
(Running + joint outputs) 
Fees foreseen (100% of 
costs of EDs, to be paid by 
industry ) 

Total costs by EU  

N/R 

N/R 

N/R 

N/R 

N/R 

2,614.5 

2,704.6 

3,683 

3,101 

2,134.8 

2,134.8 

6,926.9 

8,956.4 

+ 5-10% of 
running costs 
in first years  
8,210 

8,210 

8,956.4 

8,956.4 

5,319.10 

5817.8 

5235.8 

15,883.3 

17,166.4 

17,166.4 

-596.3 

0 

0 

0 

-1,834.80 

-1,834.80 

4,722.80 

5817.8 

5235.80 

15,883.3 

15,331.60 

15,331.60 

154 

 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Annex XI. Implementation Tables for Preferred Policy Option 

Table  1.  ESTIMATED  HR  IMPACTS  AND  COSTS  PER  YEAR  OF  THE 
PREFERRED  POLICY  OPTION  (EUR  x1000)  –  Comparison  of  the  preferred 
governance arrangement (EC Secretariat) with a secretariat run by an EU Agency 

Preferred option – FULLY OPERATIONAL 

Staff 

35 FTEs 

Estimated Joint outputs 

40 Joint early dialogues (ED) per year 

65 Joint clinical assessments/REA per year 

EC 

secretariat 

Existing 

EU agency 

NA 

8,200 

Start-up costs 

NA 

Costs running secretariat 

7,000 

(staff,  premises, 
IT, 
meetings + overheads) 

expert 

Costs joint outputs* 

9,000 

9,000 

(joint  early  dialogues, 
clinical 
horizon scanning) 

assessments, 

joint 
joint 

Total costs 

(Running + joint outputs) 

16,000 

Fees foreseen (100% of costs of 
EDs, to be paid by industry ) 

0 

Total costs by EU  

16,000 

17,200 

0 

17,200 

Most feasible in an initial phase 

*The  costs  of  joint  outputs  refers  mainly  to  the  remuneration  of  an  author  and  a  co-author 
HTA body from the Committees dedicated to carry out joint work (e.g. Committee for joint 
REA, Committee for joint early dialogues). In special situations more than 2 authors could be 
envisaged.  

155 

 
 
 
 
 
 
 
 
 
  
Table 2. Estimated staffing, workload and costs/year for the progressive 
implementation of the preferred policy option  
Type of costs 

EC Secretariat - YEARS 1-5  

% of costs from a  fully operational system 

YEARS 

YEAR 1 

YEAR 2 

YEAR 3 

YEAR 4 

YEAR 5 

FTEs (details in Table 
3) 

running 
Costs 
secretariat 
  (%  from 
total running costs of a 
operational 
fully 
million 
secretariat, 
EUR) 

  Staff + premises 
  Expert 

Committee 

meetings 
    - CG meetings  
   (nr meetings, million 
EUR) 
   - Joint Committee 
meetings 
   (nr meetings, million 
EUR) 
  - WG meetings 
   (nr meetings, million 
EUR) 
Costs joint outputs* # 

(% from total costs of 
joint outputs of a fully 
operational secretariat, 
million EUR) 

 

 

early 

in 

clinical 

Joint 
dialogues/ED 
(number,  costs 
million EUR) 
Joint 
assessments/REA 
(number,  costs 
million EUR) 

12 

15 

22 

30 

35 

4 
ADMIN/AST 
(EC) 

8 
SCIENTISTS/ 
SPECIALIST
S (Seconded 
national 
experts/EC 
staff with 
scientific 
profile) 

4 ADMIN/AST 
(EC) 

5 ADMIN/AST 
(EC) 

8 ADMIN/AST 
(EC)  

11 
SCIENTISTS/ 
SPECIALISTS 
(Seconded 
national 
experts/EC 
staff with 
scientific 
profile) 

17 
SCIENTISTS/ 
SPECIALISTS 
(Seconded 
national 
experts/EC 
staff with 
scientific 
profile) 

22 
SCIENTISTS/ 
SPECIALISTS 
(Seconded 
national 
experts/EC 
staff with 
scientific 
profile) 

10 
ADMIN/AST 
(EC) 

25 
SCIENTISTS/ 
SPECIALIST
S (Seconded 
national 
experts/EC 
staff with 
scientific 
profile) 

30%  

2.10 

1.60  

0.50 

2 
0.12  

10 
0.30  

8 
0.08  

20% 

1.80  

40% 

2.80 

2.00  

0.80 

3 
0.18   

15 
0.45  

17 
0.17  

30% 

2.70  

60% 

4.20 

3.00  

1.20 

4 
0.24  

25 
0.75  

21 
0.21  

50% 

4.50  

80% 

5.60 

100% 

7.00 

4.10  

1.50 

4 
0.24   

35 
1.05  

21 
0.21  

70% 

6.30 

4.80  

2.20 

4 
0.24  

52 
1.56  

40 
0.40  

100% 

9.00  

10 ED 
0.46  

14 ED 
0.64  

20 ED 
0.92  

30 ED 
1.38  

40 ED 
1.84  

10 REA 
1.10  

16 REA 
1.82  

30 REA 
3.30  

43 REA 
4.73  

65 REA 
7.00 

in 

156 

 
 
 
 
 
 
 

horizon 
Joint 
scanning, 
SOPs, 
guidelines  (number, 
costs in million EUR) 

Total costs by EU 
(million EUR) 

0.24  

0.24  

0.24  

0.19  

0.16  

3.90  

5.50  

8.70  

11.90  

16.00  

*Costs of joint outputs include the remuneration for the Member States bodies carrying out the joint work as authors and co-
authors. 
1 Coordination Group meeting = EUR 60,000   
1 Joint Committee meeting = EUR 30,000  
1 Working Group meeting = EUR 10,000  

#  As  regards  scope,  health  technologies  to  be  assessed  will  be  prioritised  from  the  already  limited  scope  (i.e.  centrally 
authorised pharmaceuticals – new active substances and extension of indication; medical technologies - technologies which 
have  undergone  the  scrutiny  mechanism  in  the  context  of  their  conformity  assessment)  according  to  agreed  criteria  (e.g. 
unmet medical needs; potential impact on patients, public health, or healthcare systems; significant cross-border dimension; 
major Union-wide added value). 

Table 3. EC secretariat - Staff numbers and profiles for the progressive implementation 
of the preferred policy option 

YEAR
S 

FTEs 

Profil
es 

YEAR 1 

YEAR 2 

YEAR 3 

YEAR 4 

YEAR 5 

12 

15 

22 

30 

35 

4 ADMIN/AST 
(EC*) 

4 ADMIN/AST 
(EC*) 

5 ADMIN/AST 
(EC*) 

8 ADMIN/AST 
(EC*)  

10 ADMIN/AST 
(EC) 

8 SCIENTISTS/ 
SPECIALISTS 
(Seconded national 
experts**/EC staff 
with scientific 
profile*) 

Head (1 FTE) 
Administrative 
support (3 FTE) 
o  Head 

of 
administration 
(1 FTE)  

11 SCIENTISTS/ 
SPECIALISTS 
(Seconded national 
experts**/EC staff 
with scientific 
profile*) 

Head (1 FTE) 
Administrative 
support (4 FTE) 
o  Head 

of 
administration 
(1 FTE)  

17 SCIENTISTS/ 
SPECIALISTS 
(Seconded national 
experts**/EC staff 
with scientific 
profile*) 

Head (1 FTE) 
Administrative 
support (6 FTE) 
o  Head 

of 

administration 
(1 FTE)  

o  Project 

o  Project 

o  Project 

Manager 
FTE) 

(1 

Manager 
FTE) 

(1 

o  Administrativ

o  Administrativ

e staff (1 FTE) 
Scientific/technic
al support (5 
FTE) 
o  Head (1 FTE)  
o  Scientific 
officers 
FTE) 

(2 

o  Methodology, 
guidelines, 
templates 
FTE) 

(1 

o  Administrativ

e staff (2 FTE) 
Scientific/technic
al support (7 
FTE) 
o  Head (1 FTE)  
o  Scientific 
officers 
FTE) 

(3 

o  Methodology, 
guidelines, 
templates 
(2FTE) 
o  Administrativ

e staff (1 FTE) 

e staff (1 FTE) 

IT support (3 
FTE) 
o 

Internal 
support 

l 

(1 

IT support (3 
FTE) 
o 

Internal 
support 

l  (1 

Manager 
FTE) 

(2 

o  Administrative 
staff (3 FTE) 
Scientific/technical 
support (12 FTE) 
o  Head (1 FTE)  
o  Scientific 

officers (5 FTE) 

o  Methodology, 
guidelines, 
templates 
FTE) 

(3 

o  Administrative 
staff (3 FTE) 

IT support (3 
FTE) 
o 

Internal  support 
l (1 FTE) 

o  Maintenance  of 
and 

tools 

22 SCIENTISTS/ 
SPECIALISTS 
(Seconded national 
experts**/EC staff 
with scientific 
profile*) 

Head (1 FTE) 
Administrative 
support (8 FTE) 
o  Head 

of 
administration 
(1 FTE)  

o  Project 

Manager 
FTE) 

(3 

o  Administrativ

e staff (4 FTE) 
Scientific/technic
al support ( 17 
FTE) 
o  Head (1 FTE)  
o  Scientific 
officers 
FTE) 

(8 

o  Methodology, 
guidelines, 
templates 
FTE) 

(3 

o  Administrativ

e staff (5 FTE) 

25 SCIENTISTS/ 
SPECIALISTS 
(Seconded national 
experts**/EC staff 
with scientific 
profile*) 

Head (1 FTE) 
Administrative 
support (11 FTE) 
o  Head 

of 

administration 
(1 FTE)  

o  Project 

Manager 
FTE) 

(5 

o  Administrative 
staff (5 FTE) 
Scientific/technica
l support (19 
FTE) 
o  Head (1 FTE)  
o  Scientific 
officers 
FTE) 

(10 

o  Methodology, 
guidelines, 
templates 
FTE) 

(3 

o  Administrative 
staff (5 FTE) 

IT support (4 
FTE) 
o 

Internal 
support 

l 

(2 

IT support (4 
FTE) 
o 

Internal 
support 

l 

(2 

157 

 
 
 
FTE) 

FTE) 

o  Maintenance 

o  Maintenance 

databases 
FTE) 

(2 

FTE) 

o  Maintenance 

tools  and 
(2 

of 
databases 
FTE) 

tools  and 
(2 

of 
databases 
FTE) 

tools  and 
(2 

of 
databases 
FTE) 

FTE) 

o  Maintenance of 
and 
(2 

tools 
databases 
FTE) 

1FTE  EUR 138,000 (EUR 115,000 staff related costs + EUR 23,000 costs related to premises) 

*Scientific  profiles  for  both  European  Commission  staff  and  national  experts  include: 
pharmacists, pharmacologists, biologists, medical doctors, experts in biotechnology, engineers 
with  expertise  in  the  development  of  medical  devices  and  in  vitro  diagnostics,  statisticians, 
legal experts, researchers in the field of health technologies. 

As  regards  the  European  Commission,  DG  JRC,  DG  SANTE,  DG  RTD,  DG  CNECT  have 
staff with appropriate scientific profiles. 

**Seconded  national  experts  are  national  civil  servants  or  persons  employed  in  the  public 
sector who are working temporarily for an EU Institution. They remain in the service of that 
employer  throughout  the  period  of  secondment  and  receive  a  daily  allowance  from  the 
European Commission in line with the provisions in the Staff Regulation. 

ESTIMATIONS 

Data for the estimation of the running costs: Study on Impact Analysis of Policy Options for 
Strengthened  EU  Cooperation  on  HTA.  2017.  Sogeti,  Austrian  Public  Health  Institute, 
London School of Economics. (CHAFEA/2016/Health/16) 

The running costs of an EU Agency are based on data from the European Medicines Agency 
(the  only  EU  Agency  in  the  field  of  health  with  experience  in  working  with  MS  expert 
Committees and collecting industry fees). The running costs of a project based secretariat and 
Member States secretariat are based on the data from EUnetHTA Joint Action 2 and 3. 

Data for the estimation of the outputs costs: Study on Impact Analysis of Policy Options for 
Strengthened  EU  Cooperation  on  HTA.  2017.  Sogeti,  Austrian  Public  Health  Institute, 
London School of Economics. (CHAFEA/2016/Health/16).  

Estimated  number  of  joint  outputs:  Study  on  Impact  Analysis  of  Policy  Options  for 
Strengthened  EU  Cooperation  on  HTA.  2017.  Sogeti,  Austrian  Public  Health  Institute, 
London  School  of  Economics.  (CHAFEA/2016/Health/16).  Based  on  needs  identified  by 
HTA bodies and the volume of ongoing activities carried out of EUnetHTA Joint Action 3. 

Estimated  progress  of  work  in  the  first  5  years  –  estimation  by  DG  SANTE  based  on  the 
ongoing  work  of  EUnetHTA  and  opinions  expressed  during  bilateral  meeting  and  public 
consultation  by  HTA  bodies  that  a  phased-in  approach  would  allow  a  smooth  transition  for 
incorporating EU joint activities into the national processes. 

158