REPORT FROM THE COMMISSION TO THE EUROPEAN PARLIAMENT AND THE COUNCIL on the experience acquired with the procedures for authorising and supervising medicinal products for human use, in accordance with the requirements set out in the EU legislation on medicinal products for human use
Date: 2021-08-31
Institutions: Directorate-General for Health and Food Safety, European Commission
Subjects: coronavirus disease, drug surveillance, epidemic, market approval, medicinal product, public health

EN 

  EN 

   EUROPEAN COMMISSION  Brussels, 31.8.2021  COM(2021) 497 final  REPORT FROM THE COMMISSION TO THE EUROPEAN PARLIAMENT AND THE COUNCIL on the experience acquired with the procedures for authorising and supervising medicinal products for human use, in accordance with the requirements set out in the EU legislation on medicinal products for human use  
 
 
1. 

INTRODUCTION 

At  least  every  10  years1,  the  Commission  must  publish  a  general  report  on  the 
experience  acquired  from  operating  the  procedures  for  medicinal  products  for  human 
use  laid  down  in  Regulation  (EC)  No  726/20042  and  in  Chapter  4  of  Title  III  of 
Directive  2001/83/EC3.  There  are  different  procedures  in  the  EU  for  granting  a 
marketing  authorisation  for  medicinal  products.  This  report  covers  the  centralised, 
decentralised and mutual  recognition  procedures. It  does not  cover the purely national 
procedure for authorising medicines for human use in a single Member State4.  

This  report  links  to  the  pharmaceutical  strategy  for  Europe5  and  will  inform  its 
implementation, with regard to possible legislative and non-legislative measures. It also 
complements  the  ongoing  revisions  of:  (i)  the  EU regulations  on  medicines  for  rare 
diseases  and  on  medicines  for  children6;  and  (ii)  the  Regulation  on  the  European 
Medicines Agency’s fee system7. 

2.  REGULATORY FRAMEWORK AND CONTEXT 

Objectives of the framework 

The EU regulatory framework for medicinal products has three main objectives, set out 
in the bullet points below. 

  Its  first objective is  to  guarantee a high  level  of  health protection for the people of 
Europe. It seeks to do this in two main ways. Firstly, the framework aims to provide 
patients  as  swiftly  as  possible,  with  high  quality,  safe  and  effective  medicinal 
products – both innovative and off-patent. Secondly, it aims to increase monitoring 
of  medicinal  products  after  they  have  been  authorised  thanks  to  strengthened 
procedures for monitoring and pharmacovigilance. 

  Its  second  objective  is  to  both:  (i)  complete  the  single  market  in  pharmaceutical 
products,  taking  account  of  the  implications  of  globalisation;  and  (ii)  support  the 
competitiveness of the European pharmaceuticals sector. 

  Its  third  objective  is  to  rationalise  and  simplify  the  regulatory  system  as  much  as 
possible,  thus  improving  its  overall  consistency,  efficiency  and  transparency  in 
running procedures and decision-making. 

1 Article 86 of Regulation (EC) No 726/2004 and of Article 38(2) of Directive 2001/83/EC. 
2 Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down 
Community procedures for the authorisation and supervision of medicinal products for human and veterinary use 
and establishing a European Medicines Agency, OJ L 136, 30.4.2004, p.1.  
3 Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community 
code relating to medicinal products for human use, OJ L 311, 28.11.2001, p.67.  
4 Section 2 gives further details on the different procedures for marketing authorisation of medicinal products for 
human use in the EU. 
5 COM(2020) 761. 
6  Joint  evaluation  of  Regulation  (EC)  No  1901/2006  on  medicinal  products  for  paediatric  use  and  Regulation 
(EC) No 141/2001 on orphan medicinal products (SWD(2020) 163 final). 
7 Evaluation of the European Medicines Agency’s fee system (SWD(2019) 335 final). 

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Marketing-authorisation procedures 

Since the first piece of EU pharmaceutical legislation in 1965, the EU has aimed at two 
overarching objectives: ensuring a high level of public health protection and removing 
obstacles  to  the  free  movement  of  pharmaceuticals.  Council  Directive  65/65/EEC8 
established  the  fundamental  principle  of  EU  pharmaceutical  legislation  that  no 
medicinal product may be placed on the EU market unless a marketing authorisation has 
been  granted  for  that  product.  The  authorisation  of  medicines  builds  on  three  key 
criteria: quality, safety and efficacy. This helps to ensure that products administered to 
patients are of suitable quality and provide a positive benefit-risk balance.  

Since  1965,  a  large  body  of  legislation  has  been  developed.  There  has  been  a 
progressive  harmonisation  across  the  entire  European  Economic  Area  (EEA)  of  the 
requirements  for:  (i)  granting  marketing  authorisations;  and  (ii)  post-marketing 
monitoring.  The  two  main  legal  acts  currently  regulating  marketing  authorisation 
procedures  for  medicinal  products  in  the  EEA  are  Regulation  (EC)  No 726/2004  and 
Directive  2001/83/EC,  as  amended.  There  are  four  different  procedures  to  obtain  a 
marketing  authorisation:  a  centralised  procedure;  a  decentralised  procedure;  a 
mutual recognition procedure; and a purely national procedure.  

The centralised procedure allows the applicant to gain a single authorisation with EEA-
wide  effect9.  The  application  is  assessed  by  the  European  Medicines  Agency  (EMA) 
through  its  scientific  Committee  for  Medicinal  Products  for  Human  Use  (CHMP)  and 
the  authorisation  granted  by  the  European  Commission.  The  centralised  procedure  for 
medicines  for  human  use  has  two  different  ‘scopes’.  The  first  scope  is  a  mandatory 
scope  that  covers:  (i)  medicines  developed  by  certain  biotechnological  processes;  (ii) 
advanced therapy medicines; (iii) medicines for rare diseases;  and  (iv) new medicines 
for specific diseases, e.g. cancer, AIDS  and diabetes. The second scope is an optional 
scope  that  covers,  for  example:  (i)  medicines  in  other  disease  areas;  (ii)  medicines 
containing  a  new  active  substance;  or  (iii)  existing  medicines  presenting  a  significant 
innovation. 

The  other  procedures  allow  the  applicant  to  gain  a  national  authorisation  in  only  one 
Member  State  market  (purely  national  procedure)  or  several  Member  States’  markets 
(decentralised procedure and mutual recognition procedure).  

The mutual  recognition  procedure  applies when  the applicant already holds a national 
authorisation  for  a  medicinal  product  in  one  Member  State  and  wishes  to  obtain  a 
national authorisation for the same medicinal product in other Member States.  

8  Council  Directive  65/65/EEC  of  26  January  1965  on  the  approximation  of  provisions  laid  down  by  Law, 
Regulation or Administrative Action relating to proprietary medicinal products, OJ 22, 9.2.1965, p. 369.  
9  In  accordance  with  the  EEA  agreement,  a  national  procedure/step  to  give  the  EU  marketing  authorisations 
effect is necessary for Norway, Iceland and Liechtenstein.  

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The decentralised procedure is for products not yet authorised in any Member State and 
where  the  applicant  wishes  to  obtain  a  national  marketing  authorisation  in  more  than 
one Member State.  

The initial marketing authorisation procedures are framed by two types of activities, set 
out in the bullet points below. 

  The  first  type  of  activities  are  pre-authorisation  activities.  These  are  activities 
that  take  place  before  an  application  for  marketing  authorisation  has  been  made. 
They include: (i) seeking and receiving scientific advice;  (ii) assessing applications 
for  orphan  designations;  (iii)  agreeing  on  paediatric  investigation  plans;  and  (iv) 
other  formal  and  informal  activities  to  support  the  development  of  medicines  and 
prepare applications for marketing authorisations. 

  The  second  type  of  activities  are  post-marketing  activities.  These  include 
pharmacovigilance activities (continuous safety monitoring), referrals and variations 
to marketing authorisations. 

European medicines regulatory network  

To  fulfil  their  obligations  in  authorising  and  supervising  medicines,  a  partnership  has 
been  formed  by  the  Commission,  the  EMA,  and  the  national  competent  authorities  in 
the  Member  States.  This  partnership  is  commonly  referred  to  as  the  ‘European 
medicines  regulatory  network’.  In  this  network,  the  parties  have  different  roles  as  set 
out below. 

  The  EMA  is  responsible  for  the  scientific  evaluation,  supervision,  and  safety 
monitoring  of  medicines  in  the  EU.  The  EMA  also  coordinates  scientific  expertise 
from across the EU. 

  The  national  competent  authorities  are  responsible  for  granting  national  marketing 
authorisations and supervising medicines in the respective Member States. They also 
provide expertise to the network.  

  The European Commission grants EU marketing authorisations following assessment 
by the EMA. It also monitors and oversees the network’s activities. In addition, it is 
responsible for ensuring that EU law is applied correctly. 

The  parties  work  in  close  collaboration  with  all  the  stakeholders  involved  in  
developing, manufacturing, distributing and administering medicines. This collaboration 
aims to ensure that the objectives of the legislation are achieved.  

3.  BACKGROUND ANALYSIS STUDY  

The Commission engaged an external contractor to provide a supporting study for this 
report.  

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Study  on  the  experience  acquired  from  operating  the  procedures  for  authorising 
and monitoring medicinal products for human use10 

The study assessed the extent to which the current  marketing-authorisation system  for 
medicines met its objectives in the period 2010-2017. More specifically, the study: 

  collected available data and evidence on the operation of the centralised procedure, 

the decentralised procedure, and the mutual recognition procedure; 

  assessed  the  effectiveness  (achievement  of  objectives  set  by  the  regulatory 
framework) and efficiency (relationship between the resources used and the changes 
made, which included an examination of the administrative and regulatory burden) of 
the procedures and the system in place, including an assessment of the functioning of 
the European medicines regulatory network;  

  summarised the results of its analysis and drew useful conclusions based on lessons 
learnt from the experience acquired from the marketing authorisation procedures; 
  compared the current situation with the findings of the 2010 study and followed up 

on the implementation of the recommendations made in 2010; 

  identified  options  for  possible  actions  that  may  need  to  be  taken  to  remove  any 
existing  barriers  and  obstacles  to  optimal  performance,  and  analysed  the  pros  and 
cons of each option. 

As part of the study, relevant stakeholders were consulted on their experience with the 
system and on its strengths and weaknesses. Consultation activities included: 

  interviews  with  representatives  of  the  EMA,  the  Commission,  the  European 
Parliament, the pharmaceutical industry, umbrella organisations, patient groups, and 
healthcare professional organisations; 

  attending  meetings  of  the  CHMP  and  the  Coordination  Group  for  the  Mutual 

Recognition and Decentralised Procedures – human (CMDh);  

  a written questionnaire sent to national competent authorities, followed by follow-up 

telephone interviews; 

  an online survey sent to committees’ experts; 
  eight  Member-State  case  studies  (Czechia,  Denmark,  Estonia,  Germany,  Italy, 

Portugal, Spain and Sweden). 

Review of the study report 

The Commission circulated this contractor’s study among the Member States, the EMA 
and the CMDh. The comments it received clarified inconsistencies and inaccuracies in 
the study. However, the comments also suggested to investigate further certain potential 
efficiency  gains  and  to  adapt  the  current  procedures  and  guidelines  to  innovation  and 
progress  in  science.  For  instance,  the  comments  called  for:  (i)  greater  coordination 

10 Insert link when published. 

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between  national  competent  authorities,  the  EMA,  and  health  technology  assessment 
(HTA) bodies; and (ii) actions to streamline procedures to reduce administrative burden. 
The  comments  also  pointed  to  issues  not  appropriately  investigated  in  the  study,  but 
which  will  be  further  addressed  in  the  evaluation  of  the  pharmaceutical  legislation  as 
part of the pharmaceutical strategy for Europe. 

4.  RESULTS  FROM  THE  STUDY  ON  THE  ASSESSMENT  OF  AUTHORISATION  AND 

MONITORING PROCEDURES 

Effectiveness 

Overall, data provided by the contractor’s study indicate that the current system of EU 
procedures for marketing authorisation and monitoring of medicinal products for human 
use, meets the objectives laid down in the legislation. In particular, the current system 
guarantees a high level of health protection for the people of Europe. The system also 
broadly  meets  its  objectives  of  completing  the  internal  market  in  pharmaceutical 
products  and  of  creating  a  regulatory  framework  that  supports  the  competitiveness  of 
the European pharmaceutical sector.  

Rapid  scientific  developments  have  resulted  in  new  challenges  for  the  system.  The 
system  has  therefore  become  more  complex  through  the  setting-up  of:  (i)  new 
committees  via  new  EU legislation;  (ii)  supportive  expert  working  parties;  and  (iii) 
other  additional  EU  legal  requirements.  The  rapid  scientific  developments  continue  to 
challenge  the  system,  and  solutions  to  these  challenges  will  be  proposed  through  the 
implementation of the pharmaceutical strategy for Europe. 

In the period 2010-2017, activities in the network have generally increased. As a result, 
most  national  competent  authorities  have  allocated  more  resources  to  EU-level 
activities.  The  increased  fee  income  of  the  EMA  (+81%)  is  also  an  indicator  of 
increased activities, given that the fees are paid by applicants to apply for and maintain 
marketing  authorisations.  The  number  of  initial  marketing  authorisation  applications 
under the centralised procedure remained stable at around 90-100 applications per year. 
Nevertheless,  these  new  applications  add  to  the  ever-growing  portfolio  of  authorised 
products that the EMA, the network and the Commission must manage. This has led to 
constant  growth  in  the  number  of  post-marketing  procedures  and  related  supervisory 
and monitoring activities. The number of variations to marketing authorisations granted 
under  the  centralised  procedure  increased  from  about  4 100  in  2010  to  approximately 
6 200 in 2017 (+ 51%)11. 

In  addition,  the  number  of  procedures  for  orphan,  paediatric  and  advanced  therapy 
medicines have increased substantially in 2010-2017. The advanced therapy medicinal 
products  classification  requests  increased  from  20  requests  submitted  in  2013  to  46 

11  Based  on  publicly  available  EMA  annual  reports:  https://www.ema.europa.eu/en/about-us/annual-reports-
work-programmes     

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submitted in 2017 (i.e. an increase of 130%), while orphan designations submitted every 
year  grew from  174 to  260 (up 49%), and  all paediatric investigation plan procedures 
increased from 318 to 421 (up 32%).  

The number of finalised mutual recognition and decentralised procedures has fluctuated 
between 1 640 procedures in 2010 and 1 515 procedures in 2017. In 2014, there was a 
dip to 1 046 procedures12. The CMDh plays an important role in the mutual recognition 
and decentralised procedures, both pre- and post-authorisation. It fosters harmonisation, 
for example by examining disagreements between Member States and providing general 
guidance on the procedures. In the study period, the number of referrals for the mutual 
recognition  and  decentralised  procedures  has  decreased  over  time  through  dialogue, 
cooperation and harmonisation in the CMDh. The number of referrals to the CMDh fell 
from  17  in  2010  to  11  in  2017,  with  a  peak  of  27  referrals  in  2012.  The  number  of 
referrals from the CMDh to the CHMP fell from 7 in 2010 to only 1 in 201712.  

Requests to  the EMA from  developers of medicines for scientific advice  and protocol 
assistance increased by more than 40% between 2010 and 2017. The contractor’s study 
recognised that the EMA has ensured a well-functioning process overall.  

Support to small and medium-sized enterprises (SMEs) has increased from 366 requests 
for  scientific  advice  to  the  EMA  in  2013  to  436  in  2017.  In  that  period,  SMEs 
consistently accounted for around 30% of all requests at EMA level. 

Even though the level of activity increased, the system has remained effective overall. 
The system relies on resources and expertise from the Member States, and benefits from 
mechanisms  that  ensure  internal  coordination  within  the  system.  The  EMA  plays  an 
effective coordination and scientific support role, adapting organisational structure and 
working  methods  to  ensure  efficiency  gains  in  response  to  the  increased  level  of 
activity. 

There  was  greater  harmonisation  and  coordination  among  Member  States  after  the 
enlargements of the EU. More and more Member States began taking an active role in 
procedures  as:  (i)  (co-)rapporteur;  (ii)  part  of  a  multi-national  assessment  team  in  the 
centralised  procedure;  or  (iii)  reference  Member  State  in  the  mutual  recognition  and 
decentralised procedures.  

Market  surveillance  and  safety  monitoring  have  been  strengthened.  One  of  the  main 
reasons for this was the implementation of the revised pharmacovigilance legislation in 
2012,  which  led  to:  (i)  the  establishment  of  the  Pharmacovigilance  Risk  Assessment 
Committee;  and  (ii)  coordinated  signal  management.  There  has  been  continuous 
development of the mechanisms to report adverse drug reactions. Patients have played a 
particularly  significant  role  in  this,  with  patient-submitted  adverse  drug  reactions 
reaching the number of about 90 000 in 201713. The review procedure for adverse drug 

12 CMDh statistics 2017. 
13 EMA, Annual Report 2017. 

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reactions  identifies  potential  risks  and  gives  the  EMA,  the  Commission  and  national 
competent  authorities 
take  necessary  regulatory  steps.  The 
to 
identification  of  these  potential  risks  could  be  further  improved  by  integrating  real-
world  data  into  the  procedures.  This  would  require  building  expertise  on  big  data, 
pharmacoepidemiology and statistics. 

the  possibility 

Efficiency 

The  study  found  that  the  efficiency  of  the  current  system  can  be  improved.  The  main 
elements  for  improvement  are  outlined  below  and  will  be  further  explored  and 
addressed in the implementation of the pharmaceutical strategy for Europe.  

Even before the COVID-19 pandemic, some of the experts who participated in the study 
survey  indicated  that  there  was  insufficient  capacity  to  address  emergency  needs  and 
shortages through post-marketing procedures. Experts said that the system is unable to 
react  quickly  and  flexibly,  due  to:  a)  the  lack  of  EU legislation  to  address  emergency 
needs  (e.g.  for  medicine  shortages);  and  b) the  cumbersome  coordination  approaches 
between  Member  States  and  between  committees,  which  should  be  streamlined  and 
formalised. 

The  recently  adopted  legal  proposals  under  the  European  health-union  package14  will 
help to address emergency needs. In addition, the pharmaceutical strategy for Europe5 
will explore how to minimise the risk of medicine shortages through specific measures 
such  as:  (i)  stronger  obligations  for  supply;  (ii)  earlier  notification  of  shortages  and 
withdrawals;  (iii)  greater  transparency  of  stocks;  and  (iv)  stronger  EU  coordination 
mechanisms to monitor, manage and avoid shortages. Furthermore, the pharmaceutical 
strategy  for  Europe  will  consider  measures  that  improve  the  availability  of  medicines 
throughout the EU. This will also have a (preventive) impact on shortages. 

The contractor’s study suggests that, beyond the authorisation process regulated by the 
EU  pharmaceutical  legislation,  coordination  between  the  EMA,  national  competent 
authorities  and  HTA  bodies  could  be  improved.  This  would  help  to  ensure  that 
medicines  become  accessible  to  patients  more  quickly.  Since  2010,  there  has  been  an 
increase  in  procedures  for  the  joint  issuing  of  scientific  advice  by  the  EMA/national 
competent  authorities  on 
the  one  hand  and  HTA  bodies/pricing  authorities/ 
reimbursement authorities on the other. Joint scientific-advice procedures would allow 
the  design  of  clinical  trials  and  real-world  data  studies  to  generate  evidence  with 
multiple uses. Not only could this evidence be used to support marketing authorisations, 
it could also be used to support pricing and reimbursement decisions at national level. 
This  would  ensure  faster  access  for  patients  to  medicines.  As  a  result,  greater 
cooperation between these parties will make the system more efficient to the benefit of 
patients, improving their access to medicines. 

14 European health-union package: COM(2020) 724, COM(2020) 725, COM(2020) 726, COM (2020) 727. 

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To reduce administrative burden and free valuable time and capacity for both regulators 
and industry, the regulatory framework should be simplified. This could be achieved by 
streamlining  certain  procedures  and  processes.  To  streamline  authorisation  procedures 
and optimise the authorisation framework, collaboration with  – and inspiration from – 
other mature systems in other parts of the world could be considered.  

Variations  to  the  terms  of  a  marketing  authorisation  include  very  different  types  of 
amendments.  For  example,  variations  can:  (i)  add  a  new  therapeutic  indication  to  a 
product;  (ii)  modify  information  on  contraindications;  or  (iii)  change  the  address  of  a 
marketing authorisation holder. National  competent authorities, the EMA and industry 
stakeholders share the view that variations create a high workload for all three parties 
and  that  simplification  is  necessary.  Efficient  business  processes  are  therefore  very 
important. 

The contractor’s study also raises the possibility of continuing to improve coordination 
between  the  EMA  committees.  In  particular,  the  study  suggested  coordination  that 
would  improve  the  consistency  of  outcomes,  by  better  aligning  procedures  amongst 
committees for the delivery of the final scientific opinions and recommendations by the 
EMA.  The  ongoing  revision  of  the  regulations  on  medicines  for  rare  diseases  and  on 
medicines  for  children  is  already  assessing  possible  solutions  to:  (i)  better  coordinate, 
when necessary, the work of the concerned committees; and (ii) simplify and streamline 
certain procedures.  

Applications  for  centralised  authorisation  submitted  by  SMEs  have  increased  over  the 
the  proportion  of  products  not  receiving 
study  period  2010-2017.  However, 
authorisation is higher for applications by SMEs than applications submitted by larger 
companies. The contractor’s study indicates that this may be due to the complexity, cost 
and accessibility of the  procedure. SMEs  also  lack staff  and money, and are therefore 
challenged  by  the  quality  and  clinical  requirements  for  obtaining  a  marketing 
authorisation. In addition, SMEs may not necessarily bring to the market themselves the 
medicines they have developed. This is because promising medicines are often acquired 
by larger pharmaceutical companies at a late stage of development. 

Significant  progress  in  the  support  provided  to  SMEs  has  been  observed  in  the  study 
period 2010-2017 thanks to:  

  a dedicated support function and incentives for SMEs (e.g. regulatory guidance 

 

and fee incentives) put in place by the EMA;  
support from national competent authorities to industry – including SMEs – by 
providing them with early advice. 

Further ways  to  support SMEs will be explored as part of the pharmaceutical  strategy 
for Europe.  

Digital  transformation  is  changing  the  health  sector.  However,  digital  expertise  and 
infrastructure  are  not  yet  sufficiently  available  across  the  Member  States  and  the 

8 

 
 
 
 
network. Development of expertise and infrastructure is particularly needed in big data 
and the use of artificial intelligence.  

All  stakeholders  agreed  that  EU  telematics  systems  also  play  an  important  role  in 
contributing to  the efficiency of the system. However, the stakeholders  also  identified 
room  for  improvement.  National  competent  authorities  pointed  to  a  very  complex 
governance  system  for  EU  telematics.  Some  stakeholders  said  that  EMA  telematics 
systems and national systems were not fully compatible, leading to duplication of work. 
Various stakeholders and national competent authorities remarked that the network was 
falling  behind  in  the  development  of  EU-wide  IT  systems.  This  is  due  to  competing 
priorities  between  the  need  to  set  up  new  IT  systems  required  by  legislation  and  the 
need  to  maintain  existing  systems  with  the  resources  currently  available.  This  may 
result  in  decreased  efficiency  in  the  near  future  if  not  addressed  in  a  timely  and  agile 
manner. 

National  competent  authorities,  the  EMA  and  experts  reported  that  the  system  has 
difficulties handling products that lie on the borderline between medicines and medical 
devices that are not easily classified in one or the other category (i.e. products where the 
primary mode of action  cannot  be easily determined).  Improved coordination between 
the sectors is necessary, so as not to hamper innovation in the EU. Difficulties are also 
caused by borderlines between medicines and other product categories (i.e. substances 
of human origin, biocides and food supplements). 

The EMA committees and working parties need to build further access to new areas of 
expertise in order to intergrade the latest scientific and technological knowledge into the 
development and evaluation of novel medicines. This is particularly true in areas such 
as  advanced  therapy  medicinal  products  where  the  development  of  expertise  in  the 
national  competent  authorities  is  of  increasing  importance,  considering  the  substantial 
pipeline of products.  

5. 

INITIAL  LESSONS  LEARNT  FROM  COVID-19  AS  REGARDS  AUTHORISATION  AND 
MONITORING PROCEDURES  

The  COVID-19  pandemic  highlighted  both  limitations  and  strengths  of  the  European 
medicines regulatory system. It also showed opportunities to improve the system.  

The  EU  delivered  fast  assessment  and  authorisation  of  COVID-19  vaccines  and 
therapeutics  thanks  to  rapid  scientific  advice  and  rolling  review  by  EMA  of  scientific 
evidence  as  it  becomes  available  and  expedited  decision-making  procedures  by  the 
European  Commission.  Remote  inspections  and  remote  clinical  trials  enabled  the 
system to keep delivering under the constraints of the pandemic, but also made possible 
to test alternative ways of working. Conditional marketing authorisations were granted 
for  COVID-19  vaccines  and  other  medicines  in  view  of  the  emergency  situation. 

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Member States provided access at national level to COVID-19 medicines in advance of 
their (conditional) authorisation, through compassionate-use15 mechanisms on the basis 
of harmonised advice provided by EMA. This has been done for remdesivir before the 
conditional  marketing  authorisation  was  granted,  dexamethasone  and  medicines 
consisting of monoclonal antibodies against SARS-CoV216. 

To  efficiently  respond  to  the  emergency,  regulatory  flexibilities  were  introduced,  for 
instance for requirements for labelling and on timelines and languages for consultation 
during  the  decision  making  process.  Those  flexibilities  were  explained  in  several 
guidance  documents  published  by  the  Commission17,  EMA18  and  the  Heads  of 
Medicines Agencies19. 

The  pandemic  also  reinforced  the  importance  of  early  dialogue  with  medicine 
developers and academia to keep the EMA and the experts of the EU network abreast of 
emerging  new  technologies  and  to  prepare  for  their  review,  which  facilitated  the  fast 
approval  of  COVID-19  vaccines  and  therapies.  The  COVID-19  EMA  pandemic  Task 
Force20  brought  together  the  best  expertise  from  the  network  and  ensured  a  fast  and 
coordinated response to the pandemic, from the provision of early advice to developers 
to steering scientific discussions at key milestones. This lesson learnt has already been 
taken up in the proposal of the Health Union package to strengthen EMA’s mandate21, 
where it is proposed to formally establish such a task force at EMA to lead the scientific 
response  to  any  future  public  health  emergency.  The  same  proposal  also  includes 
mechanisms for crisis management and for monitoring and mitigation of shortages. 

The pandemic added an extremely high pressure on the European medicines regulatory 
network,  stretching  its  resources.  Mitigation  measures  were  taken  to  ensure  business 
continuity22. Member States reported unprecedented workload, both for the assessment 
and  for  the  pharmacovigilance  of  COVID-19  vaccines  and  therapeutics.  The  heavy 
workload  could  be  sustained  for  a  short  period  without  serious  impact  on  normal 
business  and  with  remarkable  results  as  regards  the  efficient  support  provided  to 
COVID-19  medicine  and  vaccine  developers  and  the  timely  authorisation  of  four 

15 https://www.ema.europa.eu/en/human-regulatory/research-development/compassionate-use 
16 https://www.ema.europa.eu/en/human-regulatory/post-authorisation/referral-procedures/article-53-opinions 
17 E.g. vaccinesstrategy_labellingpackaging_en.pdf (europa.eu) and guidance_regulatory_covid19_en.pdf 
(europa.eu). 
18 EMA guidance can be found here: Guidance for medicine developers and other stakeholders on COVID-19 | 
European Medicines Agency (europa.eu). 
19 Heads of Medicines Agencies: COVID-19 (hma.eu). 
20 COVID-19 EMA pandemic Task Force: https://www.ema.europa.eu/en/human-regulatory/overview/public-
health-threats/coronavirus-disease-covid-19/emas-governance-during-covid-19-pandemic#covid-19-ema-
pandemic-task-force-section. 
21COM (2020) 725. 
22 EMRN COVID19 BCP (europa.eu). 

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COVID-19 vaccines. However, this requires further monitoring to ensure that the right 
steps are taken to sustain and strengthen the capacity of the network. 

The possibility to integrate, with adjustments, the tools and flexibilities, applied during 
the COVID-19 pandemic, into the normal business is already being considered and will 
be further  assessed, including impact  on  resources, in  the context  of the  evaluation  of 
the general pharmaceutical legislation under the pharmaceutical strategy for Europe.  

6.  CONCLUSIONS 

The current report and the supporting study demonstrate that overall the EU has a well-
functioning authorisation system for medicines. However, it also highlights issues that 
could  be  improved.  Moreover,  the  study  pre-dates  recent  disruptive  events  that  had  a 
direct impact on the marketing authorisation procedures and business continuity, such as 
Brexit  and  the  COVID-19  pandemics.  Lessons  from  these  experiences  should  inform 
any  follow-up  action.  Some  initial  lessons  learnt  from  the  COVID-19  pandemic  are  
discussed in section 5 above. 

The  implementation  of  the  pharmaceutical  strategy  for  Europe5,  which  covers 
challenges to the medicines framework and a wide range of issues along the life-cycle 
of  medicines,  provides  an  opportunity  for  a  holistic  response  to  the  issues  outlined 
above.  

The implementation will explore several issues, such as: (i) how to address shortages of 
medicines;  (ii)  how  to  streamline  procedures  and  life-cycle  management,  including 
those  for  variations;  (iii)  how  to  increase  cooperation  between  sectors  and  relevant 
parties along the life-cycle of medicines; and (iv) how to ensure relevant expertise in the 
network. This will also benefit SMEs by reducing the administrative burden for industry 
and  increasing  cooperation  between  medicines  regulators,  HTA  bodies,  pricing  and 
reimbursement authorities. 

Some actions would require changes in the legislation, whereas others can be achieved 
by non-legislative means (e.g. through guidelines and enhanced coordination). Evidence 
for the need for specific legislative actions will be gathered through an evaluation of the 
pharmaceutical legislation under the pharmaceutical strategy for Europe.  

The  work  of  the  implementation  of  the  pharmaceutical  strategy  for  Europe5  will  be 
undertaken  in  close  collaboration  with:  (i)  the  EMA;  (ii)  Member  States  and  national 
competent  authorities;  (iii)  representatives  of  patients’  and  healthcare  professionals’ 
organisations; (iv) academia; (v) industry; and (vi) other relevant stakeholders. 

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