COMMUNICATION FROM THE COMMISSION TO THE EUROPEAN PARLIAMENT, THE COUNCIL, THE EUROPEAN ECONOMIC AND SOCIAL COMMITTEE AND THE COMMITTEE OF THE REGIONS Pharmaceutical Strategy for Europe
Date: 2020-11-25
Institutions: Directorate-General for Health and Food Safety, European Commission
Subjects: EU strategy, antimicrobial resistance, competitiveness, health care, innovation, pharmaceutical industry, pharmaceutical legislation, pricing of medicines, research and development, security of supply

EN 

  EN 

   EUROPEAN COMMISSION  Brussels, 25.11.2020  COM(2020) 761 final  COMMUNICATION FROM THE COMMISSION TO THE EUROPEAN PARLIAMENT, THE COUNCIL, THE EUROPEAN ECONOMIC AND SOCIAL COMMITTEE AND THE COMMITTEE OF THE REGIONS Pharmaceutical Strategy for Europe  {SWD(2020) 286 final}  
 
 
1.  Medicines - a strong ecosystem at an important crossroads 

Good health is central to wellbeing and depends on a multitude of factors including healthy 
lifestyles and fair and equitable access to healthcare, a central pillar of the European way of 
life. Healthcare in turn requires safe, effective and affordable medicines.  

Great progress has been made on human health in the European Union in recent years, with 
average  life  expectancy  at  birth  in  the  EU  having  increased  by  3.3  years  since  20021.  New 
medicines,  vaccines  and  treatments  have  helped  to  tackle  some  of  the  leading  causes  of 
disease and life threatening illnesses. 

Milestones of major progress in treatments in the EU in the past 20 years:  

Biotech products offer treatment for many chronic conditions such as diabetes, or anaemia in patients 
with renal failure. A new generation of antiviral medicines for the treatment of chronic hepatitis C has 
become available since 2014. 

Several widely used vaccines offer protection against hepatitis B, papilloma virus or cholera. In 2020 
the Commission authorised the first Ebola vaccine. 

Personalised therapies dramatically improved the prognosis of patients with some cancers, an example 
being  trastuzumab  that  improved  cure  rate  of  HER22  positive  breast  cancer  and  advanced  disease 
overall survival. 

Advanced  therapy  medicinal  products  such  as  cell-based  and  gene  therapy  products  are  paving  the 
way for new promising therapies. CAR-T cell3 therapies for the treatment of certain blood cancers and 
a medicine to treat transfusion-dependent beta thalassaemia, a blood disorder, were authorised more 
recently. 

At the same time, though we are experiencing a period of rapid change and innovation, many 
patients  do  not  benefit  from  that  innovation,  because  medicines  are  either  unaffordable  or 
unavailable.  And  there  is  greater  awareness  of  the  need  to  ensure  that  our  use  of 
pharmaceuticals is sustainable.  

The  COVID-19  pandemic  has,  and  continues  to  have,  a  very  serious  impact  on  Europe. 
Though  Europe’s  response  has  demonstrated  strengths,  existing  vulnerabilities  have  been 
thrown into sharp focus, including those related to data availability, the supply of medicines 
or  the  availability  of  manufacturing  capacities  to  adapt  and  support  the  production  of 
medicines.  The  conclusion  of  advance  purchase  agreements  for  vaccines  is  nevertheless  an 
example  of  effective  co-operation  between  public  and  regulatory  authorities,  industry  and 
civil society organisations. The anticipated widespread and equitable availability of safe and 
effective  vaccines  in  record  time  raises  hope  for  an  exit  from  the  crisis  and  provides 
inspiration  for  a  renewed,  innovative,  patient-centred  and  world-leading  pharmaceutical 
sector. 

A new EU approach is needed to ensure we have a strong, fair competitive and green industry 
that  delivers  for  patients,  and  which  draws  on  the  potential  of  the  digital  transformation  of 

1   Eurostat: mortality and life expectancy statistics. 
2    Human epidermal growth factor receptor 2. 
3   Chimeric antigen receptor T cells. 

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health and care, driven by technological advances in fields such as artificial intelligence and 
computational  modelling.  We  need  well-functioning  international  supply  chains  and  a  well 
performing  single  market  for  pharmaceuticals,  through  an  approach  that  covers  the  entire 
lifecycle  of  pharmaceutical  products,  from  production  to  distribution,  consumption  and 
disposal. 

In this context, the Commission proposes a new pharmaceutical strategy for Europe. It is a 
patient-centred  strategy  that  aims  to  ensure  the  quality  and  safety  of  medicines,  while 
boosting the sector’s global competitiveness. It is a key pillar of the Commission’s vision to 
build a stronger European Health Union4, which President von der Leyen set out in her 2020 
State of the Union speech. 

The  new  pharmaceutical  strategy  acknowledges  that  the  EU  is  starting  from  strong 
foundations. Europe has a comprehensive pharmaceuticals system, from the development and 
authorisation  of  medicines  to  their  post-authorisation  monitoring.  The  Commission,  the 
European  Medicines  Agency  (EMA),  the  medicines  regulatory  authorities  in  the  Member 
States and the European Economic Area work together in the European medicines regulatory 
network to ensure that patients have access to high-quality, effective and safe medicines. 

EU Member State health systems making use of these medicines are a crucial part of Europe's 
high  levels  of  social  protection  and  cohesion  and  build  on  the  common values  of  universal 
access to good quality care, equity and solidarity.  

There  is  a  strong  and  competitive  pharmaceutical  industry  in  the  EU.  Together  with  other 
public and private actors, it serves public health and acts as a driver of job creation, trade and 
science.  Medicine  producers  made  the  biggest  contribution  to  research  investment  in  2019, 
with  over  €37 billion.  The  sector  provides  800 000  direct  jobs  and  a  €109.4 billion  trade 
surplus5. The EU is  the second largest  market  in  the world  for pharmaceuticals,  with  many 
stakeholders  involved,  from  start-ups  to  large  companies,  from  producers  of  patented 
medicines to  generics and biosimilars, from  wholesalers and distributors to parallel  traders, 
from medical device to software developers. Emerging biopharmaceutical companies account 
for over 70% of the research pipeline6, contributing to a vibrant sector. 

The  Pharmaceutical  Strategy  for  Europe  builds  on  these  foundations.  It  will  foster  patient 
access  to  innovative  and  affordable  medicines.  It  will  support  the  competitiveness  and 
innovative  capacity  of  the  EU’s  pharmaceutical  industry.  It  will  develop  the  EU  open 
strategic autonomy and ensure robust supply chains so that Europe can provide for its needs, 
including  in  times  of  crisis.  And  it  will  ensure  a  strong  EU  voice  on  the  global  stage.  The 
strategy  has  four  work  strands  which  flow  from  these  objectives.  Each  strand  contains 
flagship  initiatives  and  flanking  measures  to  ensure  the  objectives  deliver  tangible  results. 
Taken  together,  they  will  ensure  Europe's  pharmaceutical  policy  evolves  in  line  with  the 
green and digital transitions, demographic change and remains relevant given the realities of 
today and the ambitions of tomorrow, as part of a stronger Health Union. 

4   European Health Union package: COM(2020) 724, COM(2020) 725, COM(2020) 726, COM (2020) 727.  
5   Eurostat, international trade in goods by type of good. 
6  

IQVIA Institute for Human Data Science (2019), ‘The global use of medicine in 2019 and outlook to 2023’. 

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The  strategy  will  also  help  to  deliver  other  Union  objectives.  By  boosting  innovation  to 
address unmet needs, including vaccination against treatable infections that cause cancer, as 
well as medicines for paediatric and rare cancers, it directly contributes to ‘Europe’s Beating 
Cancer  Plan’.  Together,  the  Pharmaceutical  Strategy  and  the  Cancer  Plan  will  ensure  that 
patients across Europe can access high-quality treatment and new therapies when they need 
them and ensure the availability and  affordability  of essential  medicines for cancer patients 
across the EU. The strategy’s actions to address access to medicines will also help to meeting 
EU-level commitments under the UN’s sustainable development goals. 

The  strategy7  is  also  complementary  to  the  European  Green  Deal8  and  more  particular  the 
Zero  Pollution  ambition  for  a  toxic-free  environment,  notably  through  the  impact  of 
pharmaceutical substances on the environment. The pharmaceutical strategy paves a way for 
the  industry  to  contribute  to  EU’s  climate  neutrality,  with  a  focus  on  reducing  greenhouse 
emissions  along  the  value  chain.  It  also  contributes  to  the  action  plan  to  implement  the 
European  Pillar  of  Social  Rights9,  the  strategic  frameworks  on  achieving  a  Union  of 
Equality10,  the  upcoming  Green  Paper  on  Ageing,  the  strategy  on  Shaping  Europe’s  digital 
future11, the European strategy for data12, the work on the creation of a European health data 
space, the European One Health Action Plan against antimicrobial  resistance13 and the new 
industrial strategy for Europe14. 

Finally,  the  strategy  is  of  key  relevance  for  non-EU  countries  as  well,  in  particular  in  the 
Western  Balkans  and  the  EU’s  neighbourhood,  as  candidate  countries,  potential  candidates 
and DCFTA15 countries  have an obligation to  align to  the EU acquis  of the pharmaceutical 
legislation. 

2.  Delivering for patients: fulfilling unmet medical needs and ensuring accessibility 

and affordability of medicines 

2.1.  Prioritising unmet medical needs 

Investment  in  research and development  (R&D)  for innovative medicines and treatments  is 
essential for making progress in preventing and treating diseases. Access to safe, high quality 
and  effective  medicines  is  a  key  element  of  social  well-being,  including  for  persons  from 
disadvantaged,  vulnerable  groups,  such  as  people  with  disabilities,  people  with  a  minority 

7   The  implementation  of  the  Strategy  will  be  compatible  with  the  resources  available  in  the  2021-2027 

Multiannual Financial Framework and will be aligned with the relevant programmes and policies. 

8   COM(2019) 640. 
9   https://ec.europa.eu/social/main.jsp?catId=1226&langId=en  
10   See the Gender Equality Strategy (COM(2020) 152), the Anti-racism action plan COM(2020) 565), the EU 
Roma  strategic  framework  for  equality,  inclusion  and  participation  (COM(2020)  620)  and  the  LGBTIQ+ 
equality strategy and the forthcoming Strategy for the Rights of Persons with Disabilities the Action Plan on 
Integration and Inclusion 2020-2027. 

11   European Commission (2020), Shaping Europe’s digital future (ISBN 978-92-76-16363-3). 
12   COM(2020) 66.  
13   https://ec.europa.eu/health/sites/health/files/antimicrobial_resistance/docs/amr_2017_action-plan.pdf  
14   COM(2020) 102. 
15   Deep  and  Comprehensive  Free  Trade  Areas  (DCFTA)  are  established  between  the  European  Union,  and 

Georgia, Moldova and Ukraine, respectively. 

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ethnic or racial background and older people. There is a growing consensus that policies need 
to  be  rethought  so  as  to  stimulate  innovation  in  particular  in  areas  of  unmet  needs,  and  for 
pharmaceutical  innovation  to  be  more  patient-centred,  health  system  oriented  and  take 
account of multi-disciplinary requirements, such as in long-term care settings. 

Currently,  investment  does  not  necessarily  focus  on  the  greatest  unmet  needs,  due  to  the 
absence  of  commercial  interest  or  limitations  of  the  science.  Treatments  for  important 
diseases, for example, neurodegenerative diseases and paediatric cancers are still lacking. In 
addition, there are over 7 000 known rare diseases, including rare cancers, of which 95% still 
have  no  treatment  option16.  Other  shortcomings  concern  the  lack  of  development  of  new 
antimicrobials, treatments or vaccines for emerging health threats (including those similar to 
the present  pandemic, such as the severe acute respiratory syndrome coronavirus 2 (SARS-
CoV-2) or Middle East respiratory syndrome (MERS)) and the lack of treatments for specific 
population groups such as pregnant and breastfeeding women and older people.  

Development  of  novel  antimicrobials  or  alternatives  is  a  prime  example  of  unmet  medical 
need,  given  the  lack  of  therapeutic  options  to  address  antimicrobial  resistance  (AMR). 
AMR decreases our capability to treat infectious diseases, and threatens our ability to perform 
routine  surgery.  As  underlined  in  the  EU  One  Health  Action  Plan  on  AMR17,  it  is  a 
multifactorial problem of global concern, with serious health and economic ramifications. An 
important  challenge  is  the  excessive  and  inappropriate  use  of  antimicrobials  in  animal  and 
human  healthcare,  leading  to  the  development  of  resistance,  causing  an  estimated  33 000 
human  deaths  in  the  EU/EEA  every  year18.  While  measures  to  reduce  excessive  and 
inappropriate use, described elsewhere, must be pursued, they can have the unintended effect 
of  reducing  investment  in  new  antibiotics.  Current  incentive  models  do  not  provide  a 
sustainable  solution;  new  business  approaches  are  required,  including  new  incentives  to 
develop antimicrobials as well as new pricing systems. 

16   Joint  evaluation  of  Regulation  (EC)  No  1901/2006  of  the  European  Parliament  and  of  the  Council  of  12 
December 2006 on medicinal products for paediatric use and Regulation (EC) No 141/2000 of the European 
Parliament and of the Council of 16 December 1999 on orphan medicinal products  SWD(2020) 163. 
17   https://ec.europa.eu/health/sites/health/files/antimicrobial_resistance/docs/amr_2017_action-plan.pdf 
18   Cassini  et  al.,  (2019)  ‘Attributable  deaths  and  disability-adjusted  life-years  caused  by  infections  with 
antibiotic-resistant  bacteria  in  the  EU  and  the  European  Economic  Area  in  2015:  a  population-level 
modelling analysis’, in Lancet Infect Dis. Vol.19, issue 1, pp. 55-56. 

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Flagship initiatives related to antimicrobial resistance 

  Pilot  innovative  approaches  to  EU  R&D  and  public  procurement  for  antimicrobials 
and  their  alternatives  aiming  to  provide  pull  incentives  for  novel  antimicrobials  – 
target date 2021. 

  Promote 

investment  and  coordinate 

research,  development,  manufacturing, 
deployment  and  use  for  novel  antibiotics  as  part  of  the  new  EU  Health  Emergency 
Response Authority, prior to the start of the authority’s operations preparatory action 
on AMR – 2021. 

  Consider  in  the  review  of  the  pharmaceutical  legislation19  to  introduce  measures  to 
restrict  and  optimise  the  use  of  antimicrobial  medicines.  Explore  new  types  of 
incentives for innovative antimicrobials – 2022. 

Other action 

  Propose non-legislative measures and optimise the use of existing regulatory tools to 
combat  antimicrobial  resistance,  including  harmonisation  of  product  information, 
draft evidence-based guidance on existing and new diagnostics; promote the prudent 
use of antibiotics and communication to healthcare professionals and patients – 2021. 

Our  reply  to  the  challenges,  raised  by  persisting  unmet  medical  needs,  should  be  multi-
facetted.  Research  priorities  should  be  aligned  to  the  needs  of  patients  and  health 
systems.  Enabling  collaboration  between  scientific  disciplines  by  involving  regulators, 
academia,  healthcare  professionals,  patients’  organisations  and  healthcare  deliverers  and 
payers  at  early  stages  of  R&D,  as  pioneered  by  innovative  partnerships  for  health  research 
and innovation, can support this ambition.  

We need to break silos so that various public authorities responsible for authorisation, health 
technology assessment, healthcare provision, health insurance and financing, work together. 
Increased cooperation in scientific advice and convergence on key concepts, such as ‘unmet 
medical  need’,  will  facilitate  the  design  of  clinical  trials,  generation  of  evidence  and 
assessment, ensuring that innovation matches the needs of patients and of the national health 
systems. The outcomes of these discussions could also guide funding into specific areas, such 
as basic research in new therapeutic areas. 

To complement existing cross-country collaborative approaches in public procurement, joint 
pricing  and  reimbursement  negotiations,  new  ways  of  information  sharing,  such  as  horizon 
scanning, should be  considered. The proposed Health Technology  Assessment Regulation20 
will,  when  adopted,  promote  evidence-based  investment  decisions  in  innovative  health 
technologies with added clinical value for patients. 

19   References to the ‘pharmaceutical legislation’ are to 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)  and  Regulation  (EC)  No  726/2004  of  the  European  Parliament  and  of  the 
Council of 31 March 2004 laying down Union procedures for the authorisation and supervision of medicinal 
products for human use and establishing a European Medicines Agency (OJ L 136, 30.4.2004, p. 1). 

20   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). 

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A process of reflection has begun on how to tailor the system of incentives provided by the 
EU  pharmaceuticals  framework  better  in  order  to  stimulate  innovation  in  areas  of  unmet 
medical  needs  (e.g.  neurodegenerative  and  rare  diseases  and  paediatric  cancers).  Wide 
stakeholder engagement and multidisciplinary input will be sought. The findings of the study 
on pharmaceutical incentives21 and the evaluation of the legislation on medicines for children 
and  rare  diseases22  will  inform  any  future  review,  in  line  with  the  principles  of  better 
regulation.  

Flagship initiatives on unmet needs 

  Propose  to  revise  the  legislation  on  medicines  for  children  and  rare  diseases  to 
improve  the  therapeutic  landscape  and  address  unmet  needs  (e.g.  in  paediatric 
cancer) through more tailored incentives – 2022. 

  Facilitate collaboration on unmet needs and evidence generation in joint meetings of 
existing  committees/networks  of  regulators,  health  technology  assessment  (HTA) 
bodies and payers, involving key actors in the development, authorisation and access 
to  medicines  for  a  lifecycle  approach  and  improved  availability  and  affordability. 
Work  with  the  European  Parliament  and  the  Council  towards  the  adoption  of  the 
Regulation on health technology assessment – 2021.  

Other actions 

  Incorporate  the  European  Medicines  Agency  (EMA)  priority  medicines  scheme 
(PRIME)  in  the  regulatory  framework  to  provide  enhanced  support  so  as  to 
accelerate product development and authorisation in areas of unmet needs – 2022. 

  Enable  parallel  scientific  advice  on  clinical  study  design  for  medicines  by  HTA 

bodies and the EMA, as provided for by the proposed HTA Regulation – 2021. 

2.2.  Ensuring patients’ access to medicines 

Innovative and promising therapies do not always reach the patient, so patients in the EU still 
have different levels of access to medicines. Companies are not obliged to market a medicine 
in  all  EU  countries;  they  may  decide  not  to  market  their  medicines  in,  or  withdraw  them 
from, one or more countries. This can be due to various factors, such as national pricing and 
reimbursement  policies,  size  of  the  population,  the  organisation  of  health  systems  and 
national administrative procedures resulting in smaller and less wealthy markets in particular 
facing  these  problems.  Experience  in  the  area  of  medicines  for  children  and  rare  diseases 
illustrates the problem. The availability of such medicines has increased since the adoption of 
the specific regulations, but access varies considerably across Member States. 

Lack of transparency of  research  costs or return  on investment  can influence decisions that 
impact affordability and ultimately access for patients. Drawing on this and wider experience, 
the  Commission  will  review  the  system  of  incentives.  This  may  include  greater 
‘conditionality’  of  incentives  to  support  broader  access  for  patients  and  ways  to  increase 

21   Study  on  the  economic  impact  of  supplementary  protection  certificates,  pharmaceutical  incentives  and 

rewards in Europe: final report (2018). 

22   SWD(2020) 163. 

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competition.  The  Commission  will  also  launch  a  pilot  project  to  understand  better  the  root 
causes of deferred market launches, including as regards cancer, to inform the evaluation of 
the pharmaceutical legislation. 

Generic  and  biosimilar  medicines  provide  a  large  number  of  patients  with  accessible  and 
affordable treatments. They also allow health systems potential savings in costs through their 
positive  effect  on  pricing  competition.  The  Commission  will  consider  targeted  policies  that 
support  greater  generic  and  biosimilar  competition,  based  on  the  sound  functioning  of  the 
single market, appropriate market protection mechanisms, the removal of barriers that delay 
their timely entry to market and increased uptake by health systems. This may include further 
clarifying the provisions for the conduct of trials on patented products to support generic and 
biosimilar marketing authorisation applications (the so-called ‘Bolar’ provision).  

The  aforementioned  policies  will  be  accompanied  by  enforcement  of  the  EU  competition 
rules. The Commission’s Report on competition enforcement in the pharmaceutical sector23 
has shown that originator companies sometimes implement  strategies to  hinder the entry or 
expansion of the more affordable medicines of their generic and biosimilar competitors and 
that such strategies may require competition law scrutiny. The Commission will also continue 
to  carefully  review  mergers  between  pharmaceutical  companies  to  avoid  distortion  of 
competition. 

New health technologies should demonstrate their clinical added-value and cost-effectiveness 
compared  to  what  is  already  available.  Health  technology  assessment  is  a  tool  to  support 
this  analysis  and  inform  national  pricing  and  reimbursement  decisions.  Currently,  such 
assessment  is  highly  fragmented  across  the  EU.  The  proposed  Health  Technology 
Assessment Regulation will enable cooperation on clinical evidence requirements and clinical 
trial  design.  It  can  therefore  support  Member  States’  timely  and  evidence-based  decision-
making on patient access to new medicines.  

Actions  in  the  area  of  public  procurement  can  foster  competition  and  improve  access. 
Public buyers should design smart and innovative procurement procedures, e.g. by assessing 
the  role  of  ‘winner-takes  it  all’  procedures  and  improving  related  aspects  (such  as  price 
conditionality,  timely  delivery,  ‘green  production’  and  security  and  continuity  of  supply) 
including via the Big Buyers initiative launched under the SME Strategy.  

This would allow to address through the use of public purchasing instruments some important 
key policy objectives. National authorities will be able to share their experience and develop 
common approaches based on best practices.  

In  addition,  health  systems  and  private  companies  can  cooperate  by  using  the  new 
‘innovation partnership’ tender procedure that allow public buyers to establish a partnership 
for  the  development,  manufacturing  and  subsequent  purchase  of  medicines  with  limited 
demand.  

23   COM(2019) 17.  

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Finally,  the  Commission  will  support  regional  initiatives  of  joint  negotiation  or  joint 
tendering, as these can also contribute to improving access to medicines24. 

Flagship initiatives on access to medicines 

  Propose  to  revise  the  system  of  incentives  and  obligations  in  the  pharmaceutical 
legislation  taking  into  account  the  relationship  with  intellectual  property  rights,  to 
support innovation, access and the affordability of medicines across the EU – 2022. 

  Review the pharmaceutical legislation to address market competition considerations 
including 

to  generic  and  biosimilar  medicines, 

and 
interchangeability and the ‘Bolar’ exemption – 2022. 

improve  access 

thus 

Other actions 

  Initiate a pilot together with the EMA and Member States, with the engagement of 
future  marketing  authorisation  holders,  to  understand  the  root  causes  of  deferred 
market launches – 2021. 

  Encourage  buyers  from  the  health  sector  to  cooperate  in  view  of  implementing 
innovative  procurement  approaches  for  the  purchases  of  medicine  or  medical 
devices, in the framework of the Big Buyers initiative  – 2021. 

2.3.  Ensuring affordability of medicines for patients and health systems’ financial and fiscal 

sustainability 

The  affordability  of  medicines  has  implications  for  both  public  and  household  finances.  It 
poses a growing challenge for the majority of Member States. The business model has moved 
from selling blockbusters to marketing ‘niche-busters’. Often, new products are priced even 
higher,  with  growing uncertainty as to  their real-life effectiveness  and related overall costs. 
This puts the budgetary sustainability of health systems at risk, and reduces the possibilities 
for patients to have access to these medicines.  

There  is  a  lack  of  transparency  (in  particular  in  R&D  costs)  and  consensus  on  costing 
principles.  Better  understanding  and  greater  clarity  are  fundamental  as  a  basis  for  policy 
debates  on  the  pricing  of  niche  medicines  and  ‘fair  return’  on  research  contributions. 
Changing business models (e.g. high value acquisitions of promising pipeline products) and 
novel payment approaches, such as risk-sharing arrangements and deferred payment schemes, 
may  have  long-term  implications,  and  thus  affect  affordability  of  new  medicines.  The 
Commission will foster transparency of price information to help Member States take better 
pricing  and  reimbursement  decisions,  also  considering  possible  knock-on  effects  for 
innovation.  

Expenditure on medicines in hospital settings is incompletely reported at EU level and it is 
growing  rapidly.  Pharmaceutical  budgets  account  for  20-30%  of  hospital  expenditures  and 
are  growing  faster  than  retail  spending25.  This  is  to  be  expected  given  budget  increases  for 
the 
specialised  medicines  administered 

in  hospitals.  The  Commission  will  assess 

24   An example of such an initiative is the Beneluxa initiative, see https://beneluxa.org/collaboration. 
25   European Commission, State of health in the EU: companion report 2019 (ISBN 978-92-76-10194-9). 

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effectiveness  of  current  financial  protection  mechanisms,  working  to  optimise  these  to 
safeguard the affordability of medicines for individual patients and health systems. Improved 
knowledge on the efficiency and accessibility of medicinal care in Member States will inform 
country-specific knowledge on health systems (e.g. in the European Semester and the ‘state 
of  health  in  the  EU’  cycle)  and  possible  reforms  in  the  Member  States.  Minimising  waste 
and optimising the value of spending on medicines are also  critical  to  achieving efficient 
and  sustainable  health  systems.  A  mix  of  policy  levers  can  support  this  goal,  including: 
ensuring value for money through health technology assessment; exploiting potential savings 
from  generics  and  biosimilars;  encouraging  responsible  prescribing;  and  improving  patient 
adherence. 

Decisions  on  the  pricing  and  reimbursement  of  medicines  are  the  purview  of  Member 
States.  The  Commission  will  step  up  co-operation  with  and  among  Member  States  on  the 
affordability and cost-effectiveness of medicines and will launch a group to steer cooperation 
between national pricing and reimbursement authorities and healthcare payers. It will support 
mutual  learning  through  information  and  best-practice  exchange,  including  on  public 
procurement  and  the  coverage  of  pharmaceutical  costs  by  social  protection  systems,  price-
increase criteria and rational prescribing.  

Certain conditions such as newly launched niche products for a small number of patients or 
the  absence  of  automatic  substitution  rules  for  biologicals,  can  create  market  barriers.  This 
means that competing generics, biosimilars and ‘older’ products may find it hard to enter or 
stay  in  the  market.  This  lack  of  competition  thus  inhibits  price  savings  once  innovative 
products  lose  their  market  exclusivities.  Rules  that  do  not  directly  regulate  prices  or 
reimbursement  levels  may  nevertheless  have  a  bearing  on  the  affordability  and  cost-
effectiveness  of  medicines  through  indirect  effects  on  the  contestability  of  markets  or  the 
economic viability of products in more mature markets. The Commission will take this into 
account  in  the  review  of  the  pharmaceutical  legislation,  to  see  how  sound  competition  can 
best be fostered, leading to downward effect on prices of medicines. It will also continue to 
work, including through the exchange of best practices, on the uptake of biosimilars, in order 
to stimulate competition.  

Flagship initiatives on affordability 

  Propose  to  revise  the  pharmaceutical  legislation  addressing  aspects  that  impede  the 
competitive  functioning  of  the  markets  and  to  take  account  of  market  effects 
impacting on affordability – 2022. 

  Develop  cooperation in  a group of  competent  authorities, based on mutual  learning 
and  best-practice  exchange  on  pricing,  payment  and  procurement  policies,  to 
improve  the  affordability  and  cost-effectiveness  of  medicines  and  health  system’s 
sustainability, including on cancer treatment – 2021-2024. 

Other actions 

  Engage  with  Members  States  in  implementing  non-legislative  measures  to  improve 
transparency,  such  as  guidelines  on  principles  and  costing  methods  for  establishing 
the R&D costs of medicines – 2021-2024.  

  Continue  the  assessment  through  the  European  semester  of  the  adequacy  and 
sustainability of national health systems and issue country specific recommendations 
as relevant to ensure they are accessible and efficient. 

9 

 
 
3. 

Supporting a competitive and innovative European pharmaceutical industry  

3.1.  Providing a fertile environment for Europe's industry 

A competitive and resource-efficient EU pharmaceutical industry is of strategic interest 
for public health, economic growth, jobs, trade and science. The EU aims to support industry 
to be competitive and resilient so that, in turn, it can better respond to patients’ needs. The 
sector is changing rapidly. Established businesses are increasingly outsourcing functions and 
are  focusing  investment  on  a  limited  number  of  therapeutic  areas,  while  disinvesting  from 
others. New players, especially technology companies, have entered the market. The coming 
together  of  these  separate  industry  segments  will  transform  current  business  models  and 
markets.  

The new industrial strategy for Europe26 provides key actions supporting industry in the EU. 
Drawing  on  that  framework,  the  pharmaceutical  strategy  will  create  a  stable  and  flexible 
regulatory  environment  that  offers  legal  certainty  for  investment  and  accommodates 
technological  trends.  This  includes  providing  balanced  and  fair  incentives  to  reward  and 
protect innovation and create the right conditions for companies of all sizes in the EU to be 
competitive. 

Intellectual  property  rights  offer  protection  for  innovative  products  and  processes,  but  in 
particular for patents and supplementary protection certificates, there are differences in their 
application in Member States.  

This leads to duplications and inefficiencies thus hampering the competitiveness of industry. 
The  Commission’s  intellectual  property  action  plan27,  includes  measures  to  simplify  and 
streamline  the  EU  pharmaceutical  intellectual  property  system,  notably  as  regards 
supplementary protection certificates.  

Secure and efficient access to health data is key to fully exploiting the huge potential of new 
technologies  and  digitalisation.  Industry  and  regulators  require  access  to  data  through  a 
robust EU-wide data infrastructure to support innovation. An interlinked system that gives 
access  to  comparable  and  interoperable  health  data  from  across  the  EU  would  be  a  real 
multiplier  in  terms  of  research,  regulation  and  evidence  generation.  The  Commission  will 
propose  a  European  health  data  space  and  establish  interoperable  data  access 
infrastructure, which will improve exchange, federated access and cross-border analysis of 
health  data  in  the  EU.  This  will  support  better  healthcare  delivery  and  health  research, 
policymaking and regulation, while protecting individuals’ fundamental rights, notably their 
rights to privacy and data protection28.  

It is essential to continue creating quality employment opportunities in the EU throughout the 
pharmaceutical  value-chain.  To  that  end,  a  competitive  pharmaceuticals  industry  requires 

26   Cf. fn. 10. 
27    COM(2020) 760. 
28   In full compliance with the General Data Protection Regulation - Regulation (EU) 2016/679 of the European 
Parliament  and  of  the  Council  of  27  April  2016  on  the  protection  of  natural  persons  with  regard  to  the 
processing  of  personal  data  and  on  the  free  movement  of  such  data,  and  repealing  Directive  95/46/EC 
(General Data Protection Regulation), OJ L 119, 4.5.2016, p. 1. 

10 

 
 
                                                           
access to a skilled  and specialised  workforce. NextGenerationEU provides unprecedented 
funding  opportunities  to  support  the  availability  of  a  skilled  workforce  as  well  as  its 
adaptability and the Skills Agenda for Europe29 sets the way to make it happen. Specifically, 
it should help ensure that all key players in the pharmaceutical sector pool their resources and 
invest  in  upskilling  and  reskilling  of  all  employees  throughout  the  value  chain,  including 
through  commitments  that  need  to  materialise  under  the  Pact  for  Skills30,  launched  on  10 
November 2020. Contributing to the increase in STEM31 specialists the skills agenda will aim 
to  boost  the  number  of  (male  and  female)  STEM  graduates  and  teachers  by  making  these 
studies and careers more attractive. Researchers are at the forefront of science and innovation 
and require a specific set of skills too. More will be done to upskill scientists, in line with the 
skills agenda, and to foster their mobility across Europe. 

Diverse  sources  of  funding  are  an  essential  tool  to  support  innovation.  A  key  element  of 
support  to  the  Strategy  will  be  the  new  and  ambitious  standalone  EU4Health  programme. 
In addition,  Horizon  Europe,  Cohesion  Policy,  the  European  Defence  Fund,  public-private 
and  public-public  investment  partnerships  such  as  the  Innovative  Health  Initiative32,  and 
national  schemes  are  important  enablers  for  R&D,  including  for  small  and  medium-sized 
enterprises  (SMEs)  and  academia.  Some  of  these  partnerships  may  help  early  uptake  of 
innovation  in  health  systems.  Commission  initiatives  such  as  the  SME  strategy  for  a 
sustainable and digital Europe33, Startup Europe34, the European Innovation Council and the 
European Institute of Innovation and technology will help provide the right environment for 
SMEs and start-ups active in the health sector to grow and attract venture capital. Likewise, 
there  are  opportunities  to  invest  in  international  partnerships  in  the  area  of  health  through 
international cooperation instruments such as the EU external investment plan. At the same 
time  increased  transparency  in  the  cost  of  research  and  development  of  pharmaceuticals  is 
needed. 

Flagship initiatives on competitiveness 

  Optimise  the  supplementary  protection  certificates  system,  to  make  it  more 
transparent and efficient as foreseen in the Intellectual Property Action Plan – 2022. 

  Legislative  proposal  on  a  European  Health  Data  Space,  enabling  better  healthcare, 

health research, innovation and evidence-based decisions – 2021. 

  Establish  by  2025  interoperable  data  access  infrastructure  for  the  European  Health 
Data Space in order to facilitate secure cross-border analysis of health data; tested in 
2021 with a pilot project involving EMA and national authorities – 2021 – 2025. 

  Support public-private and public-public partnerships, financially and technically for 
example through the Innovative Health Initiative, with particular attention to SMEs, 
academia,  not-for  profit  organisations,  and  through  the  health  care  systems 
transformation partnerships – 2021. 

29   COM(2020) 274. 
30   The Pact for Skills: mobilising all partners to invest in skills. 
31   Science, technology, engineering and mathematics. 
32   European Partnership for Innovative Health (Initiative). 
33   COM(2020) 103. 
34   https://ec.europa.eu/digital-single-market/en/startup-europe.  

11 

 
 
                                                           
Other action 

  Prioritise skills investment  to  support the availability of  a skilled workforce and its 
adaptability  through  the  NextGenerationEU,  and  within  the  new  Recovery  and 
Resilience Facility and through commitments under the pact for skills  – 2022. 

3.2.  Enabling innovation and digital transformation 

Patients in the EU expect that they can benefit from state-of-the art healthcare. Scientific and 
technological advances are crucial to improve patients’ health, and support a more efficient, 
and cost effective, way of discovering and using medicines. These advances can translate not 
only into entirely new medicines, but also into alternative uses for existing ones.  

Advanced therapy medicinal products and some medicines for rare diseases are challenging 
concepts, both in terms of science and manufacturing. An increasing number of gene and cell 
therapies  under  development  may  offer  curative  treatments  and  would  require  a  new 
business  model  to  address  the  shift  in  cost  from  chronic  to  one-time  treatment.  ‘Bedside’ 
manufacture35 of more individualised medicines could be a future trend. 

Vaccines, early detection and the improvement of well-being can affect the management of 
diseases  and  the  use  of  treatments.  The  COVID-19  pandemic  has  shown  that  innovative 
approaches to the development, approval and post-authorisation monitoring of vaccines and 
repurposing  of  medicines  are  needed.  Platforms  to  monitor  the  safety  and  effectiveness  of 
vaccines  post-authorisation  will  be  developed  in  addition  to  regular  pharmacovigilance. 
COVID19 has also underlined the importance of collaboration between different stakeholders 
and secure, open access to different kind of health data, such as databases of molecules held 
by  companies,  using  data  sharing  agreements.  This  requires  open  platforms  and  greater 
collaboration to identify datasets that can be made available for reuse36. 

Digital  transformation  is  affecting  the  discovery,  development,  manufacture,  evidence 
generation,  assessment,  supply  and  use  of  medicines.  Medicines,  medical  technologies  and 
digital  health  are  becoming  increasingly  integral  to  overarching  therapeutic  options.  These 
include  systems  based  on  artificial  intelligence  for  prevention,  diagnosis,  better  treatment, 
therapeutic monitoring and data for personalised medicines and other healthcare applications.  

Personalised medicine is an integrated package of healthcare solutions comprising elements 
of medicines and medical devices that are structured to meet an individual patient’s needs. In 
the  future,  patients  may  still  be  prescribed  a  tablet  but  that  tablet  may  be  combined  with  a 
new  technology  to  determine  the  right  usage,  the  right  schedule  and  the  right  dosage 
according  to  their  personal  situation.  This  can  also  support  treatments  in  multi-disciplinary 
settings like long-term care. Digital therapeutics can use app-based platforms to help patients 
manage  chronic  diseases  such  as  diabetes,  depression  and  heart  conditions,  and  reduce 
medication.  

35   This  refers  to  a  shift  from  the  production  of  personalised  medicines  from  being  exclusively  in  the  factory 

towards a fine-tuning at the patient’s bedside. 

36   In line with the EU data strategy, notably for data reuse and business to government data sharing. 

12 

 
 
                                                           
Initiatives such as the ‘1+ million genome’37 are exploring ways to access genetic data with 
the  potential  to  improve  disease  prevention,  also  via  an  enhanced  understanding  of  the 
impacts of environmental determinants such as climate change and pollution, allow for more 
personalised  treatments  and  provide  sufficient  scale  for  new  clinically  impactful  research 
including on different cancer types.  

High  performance  computing  and  artificial  intelligence  can  help  accelerate  the 
identification of potential active substances for repurposing and reduce the high failure rates. 
Supercomputing  is  used  in  the  COVID-19  pandemic  for  example  through  the  Commission 
project Excalate4COV. Due care should be taken to avoid any gender, race or other bias in 
the  data  produced  by  artificial  intelligence.  Technological  advances  can  also  support  the 
‘3 Rs’ principles (replace, reduce, refine) for the ethical use of animals in medicine testing. 

The  main  source  of  evidence  for  the  authorisation  of  innovative  medicines  should  remain 
robust clinical trials with suitable comparators reflecting the standard of care in the EU. The 
full  implementation  of  the  Clinical  Trials  Regulation38  will  put  in  place  a  harmonised, 
highly coordinated, robust and agile system for the assessment and oversight of clinical trials 
in the EU. It will improve transparency of information, independently of the outcome of the 
trials,  to  allow  public  scrutiny  and  will  address  new  developments  such  as  adaptive  and 
complex  trials,  and  the  use  of  in-silico  techniques  and  virtual  approaches.  Experience  with 
EU funded R&I projects with adaptive trials shows that research can initiate changes that can 
reduce costs and decrease development times.  

The  Commission  will  work  to  ensure  that  the  new  framework  supports  innovative  trial 
designs.  Moreover,  in  coordination  with  the  European  regulators,  patient  groups  and 
stakeholders,  it  will  support  more  patient-oriented  design,  planning  and  conduct  of  clinical 
trials  through  harmonised  international  guidance  documents  and  taking  into  account  the 
experience acquired from clinical trials for COVID-19 vaccines and treatments. This includes 
representative participation of population groups, for example gender and age groups, that are 
likely  to  use  the  medicinal  product  investigated  in  the  clinical  trials  to  ensure  appropriate 
safety  and  efficacy.  Pragmatic  trials,  where  the  treatment  is  prescribed  and  used  as  in 
normal daily practice, can improve patient compliance and treatment tolerance by identifying 
optimal  posology  and  use  with  other  treatments.  Commercial  interest  is  often  weaker  for 
these trials, so they are mainly organised by academia, where the price of trial medication and 
insufficient regulatory knowledge can be bottle-necks.  

The  Commission  supports  initiatives  to  improve  academic  researchers  and  not-for-profit 
stakeholders’ regulatory knowledge via scientific and regulatory advice so that the evidence 
they generate can be seamlessly used to repurpose off-patent medicines for new therapeutic 
uses. Industry engagement and partnership in this process will be promoted. 

The  new  models  of  product  development  and  care  delivery  mean  that  regulators  are  seeing 
the  limits  of  the  legislation  and  the  potential  needs  for  regulatory  adaptability.  The  rapid 

37   Towards access to at least 1 million sequenced genomes in the EU by 2022;  

https://ec.europa.eu/digital-single-market/en/european-1-million-genomes-initiative. 

38   Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical 

trials on medicinal products for human use (OJ L 158, 27.5.2014, p. 1). 

13 

 
 
                                                           
developments  in  products  combining  medicines  and  medical  devices  are  reflected  in  new 
legislation39, but some challenges remain. These include clarifying roles and responsibilities, 
streamlining requirements and procedures, and building up the necessary regulatory expertise 
and  collaboration  between  sectors.  Access  to  testing  facilities  to  test  devices  involving 
artificial intelligence is important to ensure the quality of these devices. 

The Commission will propose to revise the pharmaceutical legislation to consider how to 
make  best  use  of  this  transformation.  This  includes  new  methods  of  evidence  generation 
and  assessment,  such  as  analysis  of  big  and  real  world  data  to  support  the  development, 
authorisation and use of medicines. Regulators may require access to the raw data at the time 
of  authorisation  to  fully  appreciate  these  innovative  elements  of  the  treatment.  Also 
incentivising  the  development  and  validation  of  relevant  biomarkers  would  support  the 
effectiveness  in  the  intake  of  some  new  and  expensive  medicines,  but  also  generics, 
contributing to the sustainability of healthcare systems. 

Flagship initiatives on innovation 

  Propose  to  revise  the  pharmaceutical  legislation,  to  adapt  to  cutting-edge  products, 
scientific developments  (e.g.  genomics or personalised medicine) and technological 
transformation  (e.g. data analytics  and digital  tools) and provide tailored incentives 
for innovation – 2022.  

  Enhance  dialogue  among  regulatory  and  other  relevant  authorities  in  the  area  of 
medicines and medical devices to increase cooperation on evidence generation within 
their respective fields – 2021. 

  Support collaborative projects bringing together stakeholders to take forward the use 
of  high  performance  computing  and  artificial  intelligence  in  combination  with  EU 
health data for pharmaceutical innovation – 2021-2022. 

  Establish  the  secure  federated  access  to  10  million  genomes  across  borders  for 
research,  innovation  and  clinical  applications,  including  personalised  medicine  – 
2025. 

Other actions 

  Full  implementation  of  the  regulatory  framework  for  clinical  trials,  which  supports 
innovative trial designs and a more patient-oriented medicine development  – 2021. 

  Launch a pilot project with engagement of industry and academia to test a framework 
for repurposing of off-patent medicines and inform possible regulatory action – 2021. 

  Launch  a  vaccine  platform  to  monitor  the  effectiveness  and  safety  of  vaccines, 

supported by an EU-wide clinical trials network – 2021.  

39   Regulation  (EU)  2017/745  of  the  European  Parliament  and  of  the  Council  of  5  April  2017  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, OJ L 117, 5.5.2017, p. 1 and Regulation (EU) 
2017/746  of  the  European  Parliament  and  of  the  Council  of  5  April  2017  on  in  vitro  diagnostic  medical 
devices  and  repealing  Directive  98/79/EC  and  Commission  Decision  2010/227/EU,OJ L 117,  5.5.2017, 
p. 176. 

14 

 
 
                                                           
  Strengthen  support  and  training  of  academia  and  not-for-profit  organisations  in 
regulatory science for better translation of research into product development – 2022.  

  Initiative for regulatory pilots in a ‘sandbox’ environment provided by the EMA and 
the  Commission  to  test  the  adaptability  of  the  pharmaceuticals  framework  for  new 
cutting-edge product developments – 2022.  

3.3.  A sound and flexible regulatory system 

Regulatory  efficiency  is  a  prerequisite  for  a  modern  pharmaceutical  system.  The  EU  has 
continuously updated its framework to ensure that a comprehensive system covers the entire 
lifecycle  of  medicines.  It  builds  on  a  dual  system  where  the  Commission  authorises 
innovative medicines for the entire EU on the basis of a positive opinion from the EMA and 
national regulators authorise a great number of generic and other essential medicines.  

The Commission will explore the need to recognise more formally the role of the network of 
national  medicines agencies (Heads of Medicines Agencies)  and its  operational  structure in 
the regulatory system.  

The Commission will evaluate procedures to explore new approaches to assessing scientific 
evidence for the safety and efficacy of medicines as part of the review of the legislation and 
seek to bring EU regulatory approval times onto par with those in other parts of the world. 
It  will  consider  how  infrastructure  and  adapted  regulatory  processes  could  leverage  digital 
technology  and  artificial  intelligence  to  support  regulatory  decision-making  and  increase 
efficiency. The experience of the EMA during the COVID-19 pandemic (e.g. in  the rolling 
review of incoming scientific evidence to speed up assessment) will inform future action. The 
Commission  aims  to  revisit  existing  regulatory  tools,  such  as  priority  review  and  scientific 
advice to support companies, especially SMEs, in the development of innovative products for 
unmet medical needs.  

A  study40  on  the  authorisation  and  monitoring  of  medicines  for  human  use  will  inform  the 
evaluation of the regulatory framework to simplify and streamline procedures and reduce 
costs.  The  management  of  variations  of  marketing  authorisations  and  the  assessment  of 
quality files relating to active substances are two examples of areas in which simplification is 
required.  A  process  of  reflection  will  be  initiated  on  the  functioning  of,  and  synergies 
between the scientific committees and on the role of patients and healthcare professionals.  

Moreover, better use of product information in electronic format (ePI) could facilitate the 
delivery of information on the medicine to healthcare professionals and patients in the EU’s 
multilingual environment and support wider availability of medicines across Member States. 
Any measures should take into account the needs of all patients and healthcare professionals. 
Measures should also be considered with the aim to ensure that medicines are safely handled 
by workers, including when administrating treatments. 

The  Commission  will  assess  classification  and  interplay  challenges  relating  to  other 
regulatory procedures (such as medical devices and substances of human origin) and consider 

40   Study  on  the  experience  acquired  as  a  result  of  the  procedures  for  authorisation  and  monitoring  of 

medicinal products for human use – to be published in 2021. 

15 

 
 
                                                           
action  to  increase  cooperation  between  regulatory  sectors  and,  where  necessary,  clarity  for 
stakeholders on innovative products, while maintaining high standards of quality, safety and 
efficacy.  

The regulatory requirements for the authorisation of medicines for human use that contain or 
consist of genetically modified organisms (GMOs) should be fit for purpose when it comes 
to  addressing  the  specificities  of  medicines  and  the  conduct  of  clinical  trials  with  those 
products  in  the  EU  (which  is  currently  hindered  by  the  fragmentation  of  national 
requirements).  Solutions  will  be  explored  during  the  evaluation  of  the  pharmaceutical 
legislation. In general, consideration should be given to mechanisms for the continuous and 
timely adaptation of its technical requirements in light of emerging science and technologies 
with  a  view  to  enhance  effectiveness  to  protect  human  health  whilst  minimising  harmful 
impacts on the environment. 

Regulators also need to adapt to new scientific and technological developments by upscaling 
necessary  expertise  and  achieving  operational  excellence  to  deal  with  novel  and  more 
complex therapies. One important factor in this respect is the availability of sufficient funding 
at  all  levels.  The  EMA  fees  system  is  key  in  funding  regulatory  activities  at  EU  level  and 
ensuring coverage of the relevant costs. The Commission will consider this in the upcoming 
revision of the EMA fee legislation.  

Flagship initiatives on regulatory efficiency 

  Propose  to  revise  the  pharmaceutical  legislation  to  provide  for  simplification,  the 
streamlining  of  approval  procedures  and  flexibility  for  the  timely  adaptation  of 
technical  requirements  to  scientific  and  technological  developments,  in  order  to 
address  the  challenges  relating  to  the  interplay  of  medicines  and  devices,  and  to 
strengthen pro-competitive elements – 2022. 

  Propose  to  revise  the  variation  framework  for  medicines,  through  changes  in 
legislation  and  guidelines,  to  make  the  lifecycle  management  of  medicines  more 
efficient and adapted to digitalisation – 2021-2023. 

Other actions 

  Proposal for revised EMA fee legislation – 2021. 

  Provide for a single assessment process across Member States for active substances 
used for different generic medicines (active substance master files) to facilitate their 
authorisation and life-cycle management – 2022.  

  Consider  adapting  regulatory  requirements  in  the  pharmaceutical  legislation, 
applicable to medicines for human use that contain or consist of genetically modified 
organisms (GMOs) – 2022. 

  Upgrade  the  Commission’s  Union  Register  of  centrally  authorised  products  to 
include a statistical dashboard and make data fully available for secondary use as part 
of the EU open data initiative – 2021.  

  Develop  and  implement  electronic  product  information  (ePI)  for  all  EU  medicines 
with  involvement  of  Member  States  and  industry,  evaluate  and  revise  relevant 
provisions in the legislation – 2022. 

16 

 
 
  Propose  to  revise  legislation  to  give  regulatory  authorities  more  power  to  adapt  on 
their  own  initiative  the  terms  of  marketing  authorisations  on  the  basis  of  scientific 
evidence – 2022.  

  Simplify  and  streamline  the  system  of  penalties  to  address  non-compliance  in  a 

proportionate and efficient way – 2024.  

4.  Enhancing resilience: Diversified and secure supply chains; environmentally 
sustainable pharmaceuticals; crisis preparedness and response mechanisms 

4.1.  Secure the supply of medicines across the EU and avoid shortages 

The European Council41 has recognised that “achieving strategic autonomy while preserving 
an  open  economy  as  a  key  objective  of  the  Union”.  Shortages  of  medicines  have  been  a 
serious  concern  in  the  EU  for  several  years  and  have  increased  during  the  COVID-19 
pandemic. Shortages compromise patient health and severely burden healthcare systems and 
healthcare  professionals.  They  can  lead  to  under-treatment  and  increased  hospital  stays. 
Shortages are increasingly frequent for products that have been on the market for many years 
and are widely  used42. The reasons  are  complex;  they include marketing strategies, parallel 
trade,  scarce  active  pharmaceutical  ingredients  and  raw  materials,  weak  public  service 
obligations, supply quotas or issues linked to pricing and reimbursement.  

Building  up  EU’s  open  strategic  autonomy  in  the  area  of  medicines  requires  actions  to 
identify strategic dependencies in health, and to  propose measures to  reduce them, possibly 
including  by  diversifying  production  and  supply  chains,  ensuring  strategic  stockpiling,  as 
well as fostering production and investment in Europe. Minimising the impact of medicines 
shortages  on  patient  care  will  require  both  preventative  and  mitigating  measures  to 
significantly  reinforce  the  obligation  of  continuous  supply.  This  year,  the  Commission 
launched  a  study  to  map  the  root  causes  of  shortages  and  assess  the  legal  framework.  The 
study will inform the evaluation and revision of the current legislation. Legislative measures 
could  include  stronger  obligations  on  industry  to  ensure  the  supply  of  medicines,  earlier 
notification of shortages and withdrawals, enhanced transparency of stocks across the supply 
chain, and a stronger coordinating role for the EMA in monitoring and managing shortages. 
Such measures will be complemented by enhanced cooperation between Member States, for 
example  improved  procurement  approaches  and  strategies,  joint  procurement  for  critical 
medicines and EU-level cooperation on tools and instruments for national policy making on 
prices  and  reimbursement.  For  products  with  small  volumes  or  limited  use,  new  business 
contracting and/or payment models will be crucial.  

Pharmaceutical  manufacturing  and  supply  chains  are  complex,  increasingly  globalised 
and  sometimes  not  sufficiently  diversified.  Multiple  actors,  in  different  parts  of  the  world 
with varying environmental performance of the production processes, may be involved in the 
various  production  steps  for  a  single  ingredient.  Certain  technologies  necessary  for  the 
production  of  raw  materials  are  no  longer  available  in  the  EU.  Even  before  the  COVID-19 
pandemic there were concerns about the resilience of pharmaceutical manufacturing chains, 

41   European Council Conclusions of 2 October 2020 (EUCO 13/20). 
42   Pharmaceutical Group of European Union (PGEU), Medicine shortages survey: 2019 results.  

17 

 
 
                                                           
both the European Parliament and Member States have called on the Commission to address 
this issue43. Notably as regards the supply of raw pharmaceutical materials, intermediates and 
active  pharmaceutical  substances,  which  may  contribute  to  risks  of  shortages  of  critical 
medicines. The pandemic showed that public authorities do not often have access to complete 
information about the structure of the manufacturing and supply chains. An appropriate crisis 
response  requires  resilient  and  sufficiently  diversified  supply  chains  that  operate  in  a 
predictable and resource-efficient trading environment.  

The Commission will therefore initiate and steer a structured dialogue with the actors in the 
pharmaceuticals  manufacturing  value  chain,  public  authorities,  patient  and  health  non-
governmental  organisations  and  the  research  community.  In  its  first  phase,  the  structured 
dialogue will aim to gain a better understanding of the functioning of  global supply chains 
and identify the precise causes and drivers of different potential vulnerabilities, including 
potential  dependencies  threatening  the  supply  of  critical  medicines,  active  pharmaceutical 
ingredients and raw materials based on data collection and analysis.  

In  a  second  phase,  the  structured  dialogue  will  serve  to  put  forward  a  set  of  possible 
measures  to  address  the  identified  vulnerabilities  and  formulate  policy  options  to  be 
considered  by  the  Commission  and  other  authorities  in  the  EU  to  ensure  the  security  of 
supply  and  the  availability  of  critical  medicines,  active  pharmaceutical  ingredients  and  raw 
materials. While it is important to assess whether manufacturing capacity for certain critical 
medicines  may  be  required  in  the  EU  from  the  perspective  of  public  health  and  crisis 
preparedness,  any  potential  measures  would  have  to  be  in  full  compliance  with  EU 
competition and World Trade Organization (WTO) rules.  

In order to improve the predictability of the trading environment in health products, including 
medicines, the EU will work together with WTO members on an initiative that would aim 
to  facilitate  trade  in  healthcare  products  and  contribute  to  an  effective  response  to  a  health 
emergency.  Such  an  initiative  would  help  to  strengthen  the  resilience  and  robustness  of 
supply  chains  in  the  EU  and  in  all  other  WTO  partners.  It  would  rely  on  increased 
cooperation  of  trade  partners  to  avoid  unnecessary  disruptions  in  the  production  and 
distribution of essential goods, which is crucially important in times of need. 

Flagship initiatives on open strategic autonomy 

  Propose  to  revise  the  pharmaceutical  legislation  to  enhance  security  of  supply  and 
address  shortages  through  specific  measures  including  stronger  obligations  for 
supply and transparency, earlier notification of shortages and withdrawals, enhanced 
transparency  of  stocks  and  stronger  EU  coordination  and  mechanisms  to  monitor, 
manage and avoid shortages – 2022. 

  Follow up on the European Council request for open strategic autonomy and launch a 
the  pharmaceuticals 
structured  dialogue  with  and  between 
manufacturing  value  chain  and  public  authorities  to  identify  vulnerabilities  in  the 
global  supply  chain  of  critical  medicines, 
raw  pharmaceutical  materials, 
intermediates  and  active  pharmaceutical  substances  in  order  to  formulate  policy 

the  actors 

in 

43   European Parliament  resolution of 17 September  2020 on the shortage of  medicines —  how to address an 
emerging problem (2020/2071(INI)) and European Council Conclusions of 2 October 2020 (EUCO 13/20). 

18 

 
 
                                                           
options and propose actions to strengthen the continuity and security of supply in the 
EU – 2021. 

  Consider actions to ensure that the industry increases the transparency on the supply 

chains through voluntary process – 2021. 

Other actions 

  Encourage  Member  States  and  provide  support  to  engage  in  close  cooperation 
through  funding  provided  by  EU4Health  to  develop  guidelines,  measures  and  tools   
that  could  be  used  both  at  EU  level  and  in  national  policy  making  to  address 
structural shortages – 2021-2022.  

  Promote  WTO-based  actions  to  increase  the  resilience  in  global  supply  chains  in 

essential goods – 2021. 

4.2.  High quality, safe and environmentally sustainable medicines  

The  recent  experience  with  the  unexpected  presence  of  nitrosamines  impurities  in  some 
medicines44 has highlighted the importance of a sound system for detecting quality problems 
and of compliance management.  Strengthening oversight  of the  global manufacturing  chain 
and  ensuring  more  transparency  across  the  supply  chain  are  vital.  The  accountability  of  all 
actors  for  the  quality  of  the  medicines  is  essential,  but  especially  that  of  the  marketing 
authorisation holders. Compliance with good manufacturing and distribution practices should 
be enhanced.  

Internationally,  the  EU  plays  an  active  role  in  the  promotion  of  good  manufacturing 
practices, which ensure the highest quality of pharmaceutical products. This can be achieved 
through fora such as the International Council for Harmonisation of Technical Requirements 
for  Pharmaceuticals  for  Human  Use  (ICH)  and  bilateral  and  multilateral  cooperation  in  the 
area  of  inspections.  Bilateral  cooperation  mechanisms  are  beneficial,  in  particular  mutual 
reliance  on  inspections  avoiding  duplicative  efforts  and  allowing  a  more  efficient  use  of 
inspectors.  Within  the  EU,  the  Commission  will  support  Member  State  cooperation  on 
inspections and help improve capacity.  

The  Commission  will  also  analyse  the  regulatory  impact  of  emerging  new  manufacturing 
methods  such  as  decentralised  or  continuous  manufacturing.  These  methods  create  new 
manufacturing  models,  with  a  shift  from  industrial  to  ‘bedside’  manufacturing.  While 
speeding  up  production  times,  they  create  new  challenges  in  terms  of  appropriate  quality, 
inspection and oversight.  

The production, use and disposal of medicines have environmental implications, as residues 
and waste products may enter the environment. Not only does this have a negative impact on 
the environment itself, some waste and residues may have endocrine-disrupting potential and 
others  may  increase  the  risk  of  antimicrobial  resistance.  The  presence  of  antimicrobial 
pharmaceuticals in water and soil may play a role in accelerating the development of resistant 
bacteria.  The  European  Green  Deal  zero-pollution  ambition  aims  to  protect  both  public 

44   https://www.ema.europa.eu/en/human-regulatory/post-authorisation/referral-procedures/nitrosamine-

impurities  

19 

 
 
                                                           
health  and  ecosystems.  Action  is  required  throughout  the  lifecycle  of  medicines  to  reduce 
resource use, emissions and levels of pharmaceutical residues in the environment. The overall 
exposure to such residues should be minimised and reduced as much as possible. There is still 
a lot of waste from unused medicines. The Commission recently adopted  guidelines on the 
separate  collection  of  household  hazardous  waste,  which  include  pharmaceuticals45.  Further 
measures limiting such waste should be considered including the reduction of pack sizes and 
their alignment to real use. The circular economy action plan46 and the chemicals strategy for 
sustainability47  establish  a  framework  for  generating  an  overall  shift  to  a  production  and 
consumption  of  resources  and  pharmaceutical  raw  materials  which  are  safe,  and  create  the 
lowest  possible  impact  on  the  environment  and  on  the  climate.  Moreover,  the  EU  strategic 
approach  to  pharmaceuticals  in  the  environment48  and  the  EU  one  health  action  plan  on 
antimicrobial  resistance49  set  out  targeted  actions  that  are  currently  being  implemented 
(among others, actions that improve environmental risk assessment and waste management).  

The  Pharmaceutical  Strategy  for  Europe  builds  on  those  measures  and  complements  them, 
especially  in  the  review  of  the  pharmaceutical  legislation,  including  by  reviewing  the 
provisions on environmental risk assessment. Innovation for environmentally sustainable and 
climate-neutral  pharmaceuticals  and  manufacturing  should  become  a  driver  for  the  EU 
pharmaceuticals industry which should apply the best available techniques at manufacturing 
level to reduce emissions and contribute to EU’s climate ambition along their supply chains.  

The Commission will further encourage, through international cooperation, actions to address 
the  environmental  risks  in  other  countries  where  pharmaceutical  emissions  from 
manufacturing and other sources may contribute to the spread of AMR. The extent to which 
AMR  can  be  addressed  through  the  good  manufacturing  practices  should  be  assessed.  The 
Commission  will  cooperate  with  the  World  Health  Organization  (WHO),  other  key 
international  organisations  and  bilaterally  to  raise  awareness  of  environmental  risks, 
including by sharing best practices and developing international guidelines. The Commission 
will  foster  the  environmentally  sustainable  production  and  disposal  of  medicines  globally 
including through political dialogue and voluntary commitments from the sector. 

Flagship initiatives on quality and environmental sustainability 

  Propose  to  revise  the  manufacturing  and  supply  provisions  in  the  pharmaceutical 
legislation  to  improve  the  transparency  and  reinforce  oversight  of  the  supply  chain 
and clarify  responsibilities to  ensure overall environmental sustainability, safeguard 
the  quality  of  medicines  and  ensure  preparedness  for  new  manufacturing 
technologies – 2022. 

  Propose to revise the pharmaceutical legislation to strengthen the environmental risk 

45   Commission Notice - Separate Collection of Household Hazardous Waste (2020/C 375/01), 06.11.2020. 
46   COM(2020) 98.  
47   COM(2020) 667. 
48   COM(2019) 128. Additional information on the progress of the implementation of the strategic approach to 
pharmaceuticals  in  the  environment  can  be  found  at  https://ec.europa.eu/environment/water/water-
dangersub/pharmaceuticals.htm. 

49   COM(2017) 339. 

20 

 
 
                                                           
assessment requirements and conditions of use for medicines, and take stock of the 
results of research under the innovative medicines initiative – 2022.  

Other actions 

  Review the framework on good manufacturing practice and encourage inspections of 

good manufacturing and distribution practice to improve compliance – 2022. 

  Work with the Member States to enhance their capacity to participate in international 

inspection and audit programme – ongoing. 

  Engage  with  international  partners  through  cooperation  to  ensure  the  quality  and 
environmental sustainability of the active pharmaceutical ingredients imported from 
non-EU countries – ongoing. 

  Assess  with  Member  States  and  EMA  the  feasibility  of  improving  information  in 
existing databases or linked repositories with regard to manufacturing sites, their use 
for products authorised in the EU and inspection status – 2022. 

  Continue  the  implementation  of  the  actions  under  the  strategic  approach  to 
pharmaceuticals  in  the  environment,  including  the  environmentally  safe  disposal  of 
medicines and reducing pack size and packaging – ongoing. 

  Engage  with  Member  States  and  stakeholders  in  developing  best  practices  for 

decarbonising value chains – 2021.  

4.3.  Enhancing Europe’s health crisis response mechanisms  

Effective co-operation between the public and private sector has been central to the Union’s 
response  to  the  COVID-19  pandemic.  The  ability  to  conclude  multiple  advance  purchase 
agreements  for  vaccine  are  evidence  of  a  diverse  ecosystem  of  small  and  medium  sized 
pharmaceutical  enterprises  and  established  industry  multinationals,  enabled  by  a  robust 
regulatory  and  financing  environment.  The  nature  and  speed  of  the  response  to  COVID-19 
nevertheless  illustrates  the  need  for  a  more  structural  approach  to  preparedness,  as  well  as 
weaknesses  in  the  sector’s  ability  rapidly  to  respond  to  and  prepare  for  emergency  health 
events, with potential consequences for European strategic autonomy. 

The  European  Health  Union  package  is  a  first  step  to  develop  structural  and  future-proof 
solutions  improvements  to  increase  the  EU's  preparedness  and  resilience  to  cross-border 
health threats. It extends the role of the EMA to serve as central hub for scientific excellence. 
It  gives EMA the  ability to  fast  track scientific advice and  evaluation  procedures, to  assess 
supply capacity and to monitor, quantify and mitigate shortages of crucial medicines during a 
crisis. It strengthens the mandate of the European Centre for Disease Prevention and Control 
to  provide  hands-on  support  to  Member  States  and  the  European  Commission  through 
epidemiological surveillance, scientific recommendations for appropriate health measures to 
deal  with  health  crisis.  Finally,  it  includes  a  Regulation  on  serious  cross-border  threats  to 
improve  preparedness  and  response,  including  the  announcement  of  a  European  Health 
Emergency Response Authority (HERA).  

HERA fills a major structural gap in the EU’s crisis preparedness and response infrastructure, 
and will strengthen the coordination of operations across the whole value chain and develop 
strategic investments for research, development, manufacturing, deployment, distribution and 

21 

 
 
use of medical countermeasures. This will require the assembly of ecosystems of public and 
private capabilities that jointly enable a rapid response when the need arises. 

HERA will anticipate specific threats and enable technologies through horizon scanning and 
foresight. It will identify and address investment gaps in key countermeasures including the 
development of innovative antimicrobials. It will monitor and pool production capacity, raw 
material  requirements  and  availability,  thus  addressing  supply  chain  vulnerabilities.  It  will 
support  the  development  of  crosscutting  technological  solutions  (e.g.  vaccine  platform 
technologies) that sustain preparedness and response planning for future public health threats 
and development of specific countermeasures, including through research, clinical trials and 
data infrastructure. 

During  an  EU  health  emergency,  additional  resources,  such  as  large  scale  and  timely 
procurement mechanisms or stockpiles, will be required to adequately react in the interest of 
all  Member  States.  Building  on  the  experience  with  COVID-19  vaccines  development  and 
their  common  procurement,  the  Commission  will  evaluate  and  launch  a  preparatory  action 
focusing on emerging threats to human health, such as infectious diseases and anti-microbial 
resistance.  In  parallel,  it  will  launch  an  impact  assessment  and  consultation  on  the 
establishment of an EU authority, with a view to proposing in 2021 a properly mandated and 
resourced dedicated structure to start operations swiftly. Synergies and complementarity with 
existing EU bodies and relevant spending programmes will be ensured. 

A  variety  of  additional  measures  to  support  resilience  are  planned.  The  EU4Health 
programme  and  public  private  partnerships  will  complement  national  policies  aiming  to 
protect people from serious cross-border health threats and contribute to crisis preparedness 
and  response.  Part  of  the  review  of  the  legislation  will  also  examine  how  to  create  a  more 
crisis-resilient  system.  In  parallel,  R&I,  global  value  and  supply  chains,  international 
cooperation  and  convergence  and  enhanced  and  diversified  production  facilities  will 
complete the picture. In the context of its IP action plan, the Commission will analyse tools to 
better make intellectual property related to critical technologies available in times of crisis. 

Flagship initiative on Europe’s health crisis response mechanisms 

  Proposal for an EU Health Emergency Response Authority – 2021. 

5.  Ensuring a strong EU voice globally 

The EU regulatory system on pharmaceuticals is recognised as a well-developed reliable and 
mature system. In addition, the pharmaceuticals sector is economically strategic for the EU in 
terms of global trade.  

In its work at global level, the Commission will work closely with the EMA and the national 
competent authorities in the regulatory network.  

The Commission will continue its open dialogue with other regions and countries, including 
with low and middle income countries. It will explore how to make the procedure for issuing 
opinions on medicines intended exclusively for markets outside the EU more appealing as a 
means  of  cooperating  with  other  countries  and  facilitating  access  to  medicines  outside  the 
EU.  Furthermore,  the  EU  will  continue  its  work  in  multilateral  fora  towards  enhanced 
regulatory  cooperation  and  where  possible  convergence,  namely  in  the  International 

22 

 
 
Pharmaceutical  Regulators  Programme50  and  the  International  Coalition  of  Medicines 
Regulatory Authorities51.  

The  EU  has  an  interest  in  working  with  its  international  partners  towards  more  ambitious 
level  of  quality,  efficacy  and  safety  standards  in  international  cooperation  fora  and 
organisations.  Common  international  standards  are  a  key  tool.  They  facilitate  the  global 
development of pharmaceuticals. The EU will continue to play a leading role in international 
organisations  that  promote  such  standardisation,  such  as  the  ICH,  whose  guidelines  are 
increasingly referred to  as global  standards. The Commission  will  actively work  with  other 
ICH  partners  to  set  the  agenda  for  preparing  and  updating  guidelines  for  further 
harmonisation.  

Global markets are an essential source of growth, including for SMEs. This includes ensuring 
a  level  playing  field  and  a  regulatory  environment  that  are  conducive  to  innovation  and 
competitiveness.  In bilateral relations with other countries, the Commission will defend EU 
interests,  including  reciprocal  access  to  procurement  markets  in  third  countries,  but  also 
identify common areas of strategic interest. In particular, Africa is an important partner with 
whom to explore cooperation on innovation, production and technology transfer. It will focus 
on  international  cooperation,  strengthening  global  governance  and  alliances  with  partner 
countries, including through a WTO-based initiative or action to facilitate trade in healthcare 
products. 

The EU will support the work of the World Health Organization (WHO) in strengthening 
regulatory capacity through encouraging reliance mechanisms and establishing a framework 
for designating regulators as WHO Listed Authorities.  

Flagship initiative on international cooperation 

  Work  at  global  level,  with  the  EMA  and  the  network  of  national  regulators,  in 
international  fora  and 
to  promote  regulatory 
through  bilateral  cooperation 
convergence to ensure access to safe, effective high-quality and affordable medicinal 
products globally – ongoing. 

Other actions 

  Advance international harmonisation by proactively proposing topics in line with the 
latest  scientific  developments;  promoting  the  uptake  and  implementation  of 
international  standards,  and  ensuring  a  level  playing  field  for  operators  on  the 
international  market  by  enhancing  the  EU’s  bilateral  and  multilateral  relations   
ongoing. 

6.  Working together for success: a cooperative and layered approach to 

implementing the strategy 

The  pharmaceutical  strategy  will  ensure  that  we  continue  to  deliver  high  quality,  safe 
medicines and that the benefits of innovation reach patients in the EU. It will guarantee that 

50   http://www.iprp.global/home  
51   http://www.icmra.info/drupal/en/home  

23 

 
 
                                                           
the EU remains an attractive place to invest, research and develop medicines. It will improve 
the resilience and crisis preparedness of the EU system. And it will strengthen the EU’s voice 
on the global stage. 

To ensure that this strategy succeeds we need a comprehensive, integrated approach that 
addresses  the  challenges  and  breaks  down  silos,  working  together  across  disciplines  and 
regulatory  competences  throughout  the  lifecycle  of  medicines  and  medical  technologies  to 
find the right policy approaches.  

A successful transition will depend on collaborative dialogue, as exemplified by the various 
consultation  activities  in  the  preparation  of  this  strategy.  The  Commission  is  ready  to 
continue this dialogue. Accordingly, it intends to reach out to all relevant national authorities 
and stakeholders so that they may not only provide their input, but also become partners in 
this  process.  An  inclusive  civil  society  dialogue  building  on  existing  structures  will  be 
used  to  facilitate  the  interaction  with  stakeholders:  public  authorities,  industry,  healthcare 
professionals, patient, consumer and civil society organisations and the research community. 

The  Commission  will  regularly  report  on  the  progress  made  and  will  keep  the  European 
Parliament  and  the  Council  fully  informed  and  engaged  in  all  relevant  actions,  taking 
account of their role in policy shaping and law making. 

The Commission will pursue the objectives of the strategy and implement specific actions in 
partnership  with  the  Member  States,  through  enhanced  dialogue,  close  interaction  and  a 
proactive  exchange  of  information  between  the  Member  States  and  the  Commission.  The 
main forum for discussion with the Member States will be the Pharmaceutical Committee52 
while  other  existing  collaboration  mechanisms  in  the  EU  will  be  strengthened  and 
streamlined for this purpose. 

This strategy sets out a multi-year vision. It is the beginning of a process that will ensure that 
the  EU's  pharmaceutical  policy  delivers  and  serves  public  health  in  an  economically, 
environmentally and socially sustainable manner in an ever-changing environment that brings 
transformation  in  both  science  and  markets.  Its  implementation  requires  long-term 
engagement  and  mobilisation  of  resources.  Its  success  will  depend  on  the  commitment  and 
contribution of all actors in the pharmaceutical value chain to build common ownership.  

52   Council Decision 75/320/EEC of 20 May 1975 setting up a pharmaceutical committee (OJ L 147, 9.6.1975, 

p. 23). 

24