Pharmaceutical Strategy for Europe - European Commission
←
→
Page content transcription
If your browser does not render page correctly, please read the page content below
PHARMACEUTICAL STRATEGY FOR EUROPE
Pharmaceutical
Strategy for Europe
2020
2020
1
Contents
04 15
1. Medicines - a strong 3. Supporting a
ecosystem at an competitive and
important crossroads innovative European
pharmaceutical
09 industry
3.1. Providing a fertile environment
2. Delivering for patients: for Europe’s industry
fulfilling unmet 3.2. Enabling innovation and digital
medical needs and transformation
ensuring accessibility 3.3. A sound and flexible regulatory
and affordability of system
medicine
2.1 Prioritising unmet medical
needs
2.2 Ensuring patients’ access to
medicines
2.3 Ensuring affordability of
medicines for patients and
health systems’ financial and
2 fiscal Sustainability
22 30
4. Enhancing resilience: 5. Ensuring a strong EU
Diversified and voice globally
secure supply chains;
environmentally
sustainable 32
pharmaceuticals; crisis
preparedness and 6. Working together for
response mechanisms success: a cooperative
and layered approach
4.1. Secure the supply of medicines to implementing the
across the EU and avoid strategy
shortages
4.2. High quality, safe and
environmentally sustainable
medicines
4.3. Enhancing Europe’s health crisis
response mechanisms
3
202O
1. Medicines - a strong
PHARMACEUTICAL STRATEGY FOR EUROPE
ecosystem
at an important
crossroads
4
PHARMACEUTICAL STRATEGY FOR EUROPE
Good health is central to wellbeing and At the same time, though we are
depends on a multitude of factors including experiencing a period of rapid change and
healthy lifestyles and fair and equitable access innovation, many patients do not benefit
to healthcare, a central pillar of the European from that innovation, because medicines
way of life. Healthcare in turn requires safe, are either unaffordable or unavailable. And
effective and affordable medicines. there is greater awareness of the need to
ensure that our use of pharmaceuticals is
Great progress has been made on human sustainable.
health in the European Union in recent years,
with average life expectancy at birth in the The COVID-19 pandemic has, and continues
EU having increased by 3.3 years since 20021. to have, a very serious impact on Europe.
New medicines, vaccines and treatments have Though Europe’s response has demonstrated
helped to tackle some of the leading causes of strengths, existing vulnerabilities have
disease and life threatening illnesses. been thrown into sharp focus, including
those related to data availability, the
supply of medicines or the availability of
M I L E S TON ES OF MAJ OR manufacturing capacities to adapt and
P RO G RE SS I N TR EATME N TS support the production of medicines. The
I N T H E E U I N THE PA ST 20 conclusion of advance purchase agreements
Y E A RS : for vaccines is nevertheless an example
of effective co-operation between public
Biotech products offer treatment for many and regulatory authorities, industry and
chronic conditions such as diabetes, or anaemia civil society organisations. The anticipated
in patients with renal failure. A new generation widespread and equitable availability of safe
of antiviral medicines for the treatment of and effective vaccines in record time raises
chronic hepatitis C has become available since hope for an exit from the crisis and provides
2014. inspiration for a renewed, innovative, patient-
centred and world-leading pharmaceutical
Several widely used vaccines offer protection
sector.
against hepatitis B, papilloma virus or cholera.
A new EU approach is needed to ensure
In 2020 the Commission authorised the we have a strong, fair competitive and
first Ebola vaccine. Personalised therapies green industry that delivers for patients,
dramatically improved the prognosis of and which draws on the potential of
2020
patients with some cancers, an example being the digital transformation of health and
trastuzumab that improved cure rate of HER21 care, driven by technological advances in
positive breast cancer and advanced disease fields such as artificial intelligence and
overall survival. computational modelling. We need well-
functioning international supply chains
Advanced therapy medicinal products such and a well performing single market for
as cell-based and gene therapy products are pharmaceuticals, through an approach that
covers the entire lifecycle of pharmaceutical
paving the way for new promising therapies.
products, from production to distribution,
CAR-T cell2 therapies for the treatment of
consumption and disposal.
certain blood cancers and a medicine to treat
transfusion-dependent beta thalassaemia, a In this context, the Commission proposes
blood disorder, were authorised more recently. a new pharmaceutical strategy for
Europe. It is a patient-centred strategy
that aims to ensure the quality and safety
2. Human epidermal growth factor receptor 2 . of medicines, while boosting the sector’s
3. Chimeric antigen receptor T cells. global competitiveness. It is a key pillar of
the Commission’s vision to build a stronger
1. Eurostat: mortality and life expectancy statistics.
5
PHARMACEUTICAL STRATEGY FOR EUROPE
European Health Union4 2, which President medicines. It will support the competitiveness
von der Leyen set out in her 2020 State of and innovative capacity of the EU’s
the Union speech. pharmaceutical industry. It will develop the
EU open strategic autonomy and ensure
The new pharmaceutical strategy robust supply chains so that Europe can
acknowledges that the EU is starting provide for its needs, including in times of
from strong foundations. Europe has a crisis. And it will ensure a strong EU voice on
comprehensive pharmaceuticals system, the global stage. The strategy has four work
from the development and authorisation strands which flow from these objectives.
of medicines to their post-authorisation Each strand contains flagship initiatives and
monitoring. The Commission, the European flanking measures to ensure the objectives
Medicines Agency (EMA), the medicines deliver tangible results. Taken together, they
regulatory authorities in the Member will ensure Europe’s pharmaceutical policy
States and the European Economic Area evolves in line with the green and digital
work together in the European medicines transitions, demographic change and remains
regulatory network to ensure that patients relevant given the realities of today and the
have access to high-quality, effective and ambitions of tomorrow, as part of a stronger
safe medicines. Health Union.
EU Member State health systems making The strategy will also help to deliver other
use of these medicines are a crucial part of Union objectives. By boosting innovation to
Europe’s high levels of social protection and address unmet needs, including vaccination
cohesion and build on the common values of against treatable infections that cause
universal access to good quality care, equity cancer, as well as medicines for paediatric
and solidarity. and rare cancers, it directly contributes to
‘Europe’s Beating Cancer Plan’. Together,
There is a strong and competitive the Pharmaceutical Strategy and the Cancer
pharmaceutical industry in the EU. Together Plan will ensure that patients across Europe
with other public and private actors, it can access high-quality treatment and new
serves public health and acts as a driver of therapies when they need them and ensure
job creation, trade and science. Medicine the availability and affordability of essential
producers made the biggest contribution to medicines for cancer patients across the EU.
research investment in 2019, with over €37 The strategy’s actions to address access to
billion. The sector provides 800 000 direct medicines will also help to meeting EU-level
2020
jobs and a €109.4 billion trade surplus 3. The commitments under the UN’s sustainable
EU is the second largest market in the world development goals.
for pharmaceuticals, with many stakeholders
involved, from start-ups to large companies, The strategy 5 is also complementary to the
from producers of patented medicines to European Green Deal 6 and more particular
generics and biosimilars, from wholesalers the Zero Pollution ambition for a toxic-free
and distributors to parallel traders, from environment, notably through the impact
medical device to software developers. of pharmaceutical substances on the
Emerging biopharmaceutical companies environment. The pharmaceutical strategy
account for over 70% of the research paves a way for the industry to contribute
pipeline 4, contributing to a vibrant sector. to EU’s climate neutrality, with a focus on
reducing greenhouse emissions along the
The Pharmaceutical Strategy for Europe value chain. It also contributes to the action
builds on these foundations. It will foster plan to implement the European Pillar of
patient access to innovative and affordable
2. European Health Union package: COM(2020) 724, COM(2020) 725, COM(2020) 726, COM (2020) 727.
3. Eurostat, international trade in goods by type of good.
4. IQVIA Institute for Human Data Science (2019), ‘The global use of medicine in 2019 and outlook to 2023’.
5. 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.
6. COM(2019) 640.
6
PHARMACEUTICAL STRATEGY FOR EUROPE
Social Rights 7, the strategic frameworks on
achieving a Union of Equality 8, the upcoming
Green Paper on Ageing, the strategy on
Shaping Europe’s digital future 9, the
European strategy for data 10, the work on the
creation of a European health data space,
the European One Health Action Plan against
antimicrobial resistance 11 and the new
industrial strategy for Europe 12.
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 DCFTA 13 countries have an obligation to
align to the EU acquis of the pharmaceutical
legislation.
2020
7. https://ec.europa.eu/social/main.jsp?catId=1226&langId=en
8. 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.
9. European Commission (2020), Shaping Europe’s digital future (ISBN 978-92-76-16363-3).
10. COM(2020) 66.
11. https://ec.europa.eu/health/sites/health/files/antimicrobial_resistance/docs/amr_2017_action-plan.pdf
12. COM(2020) 102.
13. Deep and Comprehensive Free Trade Areas (DCFTA) are established between the European Union, and Georgia, Moldova and Ukraine, respectively.
7
2020
2. Delivering for patients:
PHARMACEUTICAL STRATEGY FOR EUROPE
fulfilling unmet
medical needs and
ensuring accessibility
and affordability of
medicines
PHARMACEUTICAL STRATEGY FOR EUROPE
2.1. Prioritising unmet medical threatens our ability to perform routine
needs surgery. As underlined in the EU One Health
Action Plan on AMR 15, it is a multifactorial
Investment in research and development problem of global concern, with serious
(R&D) for innovative medicines and health and economic ramifications. An
treatments is essential for making progress important challenge is the excessive and
in preventing and treating diseases. Access inappropriate use of antimicrobials in
to safe, high quality and effective medicines animal and human healthcare, leading to
is a key element of social well-being, the development of resistance, causing an
including for persons from disadvantaged, estimated 33 000 human deaths in the EU/
vulnerable groups, such as people with EEA every year 16. While measures to reduce
disabilities, people with a minority ethnic or excessive and inappropriate use, described
racial background and older people. There is elsewhere, must be pursued, they can have
a growing consensus that policies need to the unintended effect of reducing investment
be rethought so as to stimulate innovation in new antibiotics. Current incentive models
in particular in areas of unmet needs, and do not provide a sustainable solution; new
for pharmaceutical innovation to be more business approaches are required, including
patient-centred, health system oriented new incentives to develop antimicrobials as
and take account of multi-disciplinary well as new pricing systems.
requirements, such as in long-term care
settings.
Flagship initiatives related to
Currently, investment does not necessarily antimicrobial resistance
focus on the greatest unmet needs, due
to the absence of commercial interest ▶ Pilot innovative approaches to EU
or limitations of the science. Treatments R&D and public procurement for
for important diseases, for example, antimicrobials and their alternatives
neurodegenerative diseases and paediatric aiming to provide pull incentives for
cancers are still lacking. In addition, there are novel antimicrobials – target date
over 7 000 known rare diseases, including 2021.
rare cancers, of which 95% still have no
treatment option 14. Other shortcomings ▶ Promote investment and coordinate
concern the lack of development of new research, development, manufacturing,
antimicrobials, treatments or vaccines for deployment and use for novel
2020
emerging health threats (including those antibiotics as part of the new EU
similar to the present pandemic, such as Health Emergency Response Authority,
the severe acute respiratory syndrome prior to the start of the authority’s
coronavirus 2 (SARS-CoV-2) or Middle East operations preparatory action on AMR
respiratory syndrome (MERS)) and the lack – 2021.
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
14. 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.
15. https://ec.europa.eu/health/sites/health/files/antimicrobial_resistance/docs/amr_2017_action-plan.pdf
16. 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.
9
PHARMACEUTICAL STRATEGY FOR EUROPE
cooperation in scientific advice and
▶ Consider in the review of the convergence on key concepts, such as ‘unmet
pharmaceutical legislation 1 to medical need’, will facilitate the design
introduce measures to restrict and of clinical trials, generation of evidence
optimise the use of antimicrobial and assessment, ensuring that innovation
medicines. Explore new types matches the needs of patients and of the
of incentives for innovative national health systems. The outcomes of
antimicrobials – 2022. these discussions could also guide funding
into specific areas, such as basic research in
Other action new therapeutic areas.
▶ Propose non-legislative measures To complement existing cross-country
and optimise the use of existing collaborative approaches in public
regulatory tools to combat procurement, joint pricing and reimbursement
antimicrobial resistance, including negotiations, new ways of information
harmonisation of product information, sharing, such as horizon scanning, should be
draft evidence-based guidance on considered. The proposed Health Technology
existing and new diagnostics; promote Assessment Regulation 17 will, when adopted,
the prudent use of antibiotics promote evidence-based investment
and communication to healthcare decisions in innovative health technologies
professionals and patients – 2021. with added clinical value for patients.
1. 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).
Our reply to the challenges, raised by
2020
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 A process of reflection has begun on how to
healthcare deliverers and payers at early tailor the system of incentives provided
stages of R&D, as pioneered by innovative by the EU pharmaceuticals framework
partnerships for health research and better in order to stimulate innovation
innovation, can support this ambition. in areas of unmet medical needs (e.g.
neurodegenerative and rare diseases and
We need to break silos so that various paediatric cancers). Wide stakeholder
public authorities responsible for engagement and multidisciplinary input
authorisation, health technology assessment, will be sought. The findings of the study
healthcare provision, health insurance on pharmaceutical incentives 18 and the
and financing, work together. Increased evaluation of the legislation on medicines for
17. 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).
18. Study on the economic impact of supplementary protection certificates, pharmaceutical incentives and rewards in Europe: final report (2018).
10PHARMACEUTICAL STRATEGY FOR EUROPE
children and rare diseases 19 will inform any market a medicine in all EU countries; they
future review, in line with the principles of may decide not to market their medicines
better regulation. in, or withdraw them from, one or more
countries. This can be due to various factors,
such as national pricing and reimbursement
Flagship initiatives on unmet needs policies, size of the population, the
organisation of health systems and national
▶ Propose to revise the legislation administrative procedures resulting in smaller
on medicines for children and rare and less wealthy markets in particular facing
diseases to improve the therapeutic these problems. Experience in the area of
landscape and address unmet needs medicines for children and rare diseases
(e.g. in paediatric cancer) through illustrates the problem. The availability of
more tailored incentives – 2022. such medicines has increased since the
adoption of the specific regulations, but
▶ Facilitate collaboration on unmet access varies considerably across Member
needs and evidence generation in States.
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
2020
support so as to accelerate product
development and authorisation in
areas of unmet needs – 2022.
Lack of transparency of research costs or
▶ Enable parallel scientific advice on
return on investment can influence decisions
clinical study design for medicines by
that impact affordability and ultimately
HTA bodies and the EMA, as provided
access for patients. Drawing on this and
for by the proposed HTA Regulation –
wider experience, the Commission will review
2021.
the system of incentives. This may include
greater ‘conditionality’ of incentives to
support broader access for patients and ways
to increase competition. The Commission
2.2. Ensuring patients’ access to will also launch a pilot project to understand
medicines better the root causes of deferred market
launches, including as regards cancer, to
Innovative and promising therapies do not inform the evaluation of the pharmaceutical
always reach the patient, so patients in the legislation.
EU still have different levels of access to
medicines. Companies are not obliged to
19. SWD(2020) 163.
11PHARMACEUTICAL STRATEGY FOR EUROPE
Generic and biosimilar medicines provide assessing the role of ‘winner-takes it all’
a large number of patients with accessible procedures and improving related aspects
and affordable treatments. They also allow (such as price conditionality, timely delivery,
health systems potential savings in costs ‘green production’ and security and continuity
through their positive effect on pricing of supply) including via the Big Buyers
competition. The Commission will consider initiative launched under the SME Strategy.
targeted policies that support greater generic
and biosimilar competition, based on the This would allow to address through the
sound functioning of the single market, use of public purchasing instruments some
appropriate market protection mechanisms, important key policy objectives. National
the removal of barriers that delay their authorities will be able to share their
timely entry to market and increased uptake experience and develop common approaches
by health systems. This may include further based on best practices.
clarifying the provisions for the conduct of
trials on patented products to support generic In addition, health systems and private
and biosimilar marketing authorisation companies can cooperate by using the new
applications (the so-called ‘Bolar’ provision). ‘innovation partnership’ tender procedure that
allow public buyers to establish a partnership
The aforementioned policies will be for the development, manufacturing and
accompanied by enforcement of the EU subsequent purchase of medicines with
competition rules. The Commission’s limited demand.
Report on competition enforcement in the
pharmaceutical sector 20 has shown that Finally, the Commission will support regional
originator companies sometimes implement initiatives of joint negotiation or joint
strategies to hinder the entry or expansion tendering, as these can also contribute to
of the more affordable medicines of their improving access to medicines 21.
generic and biosimilar competitors and that
such strategies may require competition Flagship initiatives on access to
law scrutiny. The Commission will also medicines
continue to carefully review mergers between
pharmaceutical companies to avoid distortion ▶ Propose to revise the system of
of competition. incentives and obligations in the
pharmaceutical legislation taking
New health technologies should demonstrate into account the relationship with
2020
their clinical added-value and cost- intellectual property rights, to
effectiveness compared to what is already support innovation, access and the
available. Health technology assessment affordability of medicines across the
is a tool to support this analysis and inform EU – 2022.
national pricing and reimbursement decisions.
Currently, such assessment is highly ▶ Review the pharmaceutical legislation
fragmented across the EU. The proposed to address market competition
Health Technology Assessment Regulation considerations and thus improve
will enable cooperation on clinical evidence access to generic and biosimilar
requirements and clinical trial design. It can medicines, including interchangeability
therefore support Member States’ timely and and the ‘Bolar’ exemption – 2022.
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
20. COM(2019) 17.
21. An example of such an initiative is the Beneluxa initiative, see https://beneluxa.org/collaboration.
12PHARMACEUTICAL STRATEGY FOR EUROPE
reimbursement decisions, also considering
Other actions possible knock-on effects for innovation.
▶ Initiate a pilot together with the Expenditure on medicines in hospital
EMA and Member States, with the settings is incompletely reported at EU level
engagement of future marketing and it is growing rapidly. Pharmaceutical
authorisation holders, to understand budgets account for 20-30% of hospital
the root causes of deferred market expenditures and are growing faster than
launches – 2021. retail spending 22. This is to be expected given
budget increases for specialised medicines
▶ Encourage buyers from the health administered in hospitals. The Commission
sector to cooperate in view of will assess the effectiveness of current
implementing innovative procurement financial protection mechanisms, working to
approaches for the purchases of optimise these to safeguard the affordability
medicine or medical devices, in the of medicines for individual patients and
framework of the Big Buyers initiative health systems. Improved knowledge on the
– 2021. efficiency and accessibility of medicinal
care in Member States will inform country-
specific knowledge on health systems (e.g.
2.3. Ensuring affordability of in the European Semester and the ‘state of
medicines for patients and health in the EU’ cycle) and possible reforms
health systems’ financial and in the Member States. Minimising waste
and optimising the value of spending
fiscal sustainability on medicines are also critical to achieving
The affordability of medicines has efficient and sustainable health systems.
implications for both public and household A mix of policy levers can support this
finances. It poses a growing challenge for goal, including: ensuring value for money
the majority of Member States. The business through health technology assessment;
model has moved from selling blockbusters exploiting potential savings from generics
to marketing ‘niche-busters’. Often, new and biosimilars; encouraging responsible
products are priced even higher, with growing prescribing; and improving patient adherence.
uncertainty as to their real-life effectiveness
and related overall costs. This puts the
budgetary sustainability of health systems at
2020
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
22. European Commission, State of health in the EU: companion report 2019 (ISBN 978-92-76-10194-9).
13PHARMACEUTICAL STRATEGY FOR EUROPE
Decisions on the pricing and
reimbursement of medicines are the Flagship initiatives on affordability
purview of Member States. The Commission
will step up co-operation with and among ▶ Propose to revise the pharmaceutical
Member States on the affordability and legislation addressing aspects that
cost-effectiveness of medicines and impede the competitive functioning
will launch a group to steer cooperation of the markets and to take account
between national pricing and reimbursement of market effects impacting on
authorities and healthcare payers. It will affordability – 2022.
support mutual learning through information
and best-practice exchange, including on ▶ Develop cooperation in a group of
public procurement and the coverage of competent authorities, based on
pharmaceutical costs by social protection mutual learning and best-practice
systems, price-increase criteria and rational exchange on pricing, payment and
prescribing. procurement policies, to improve the
affordability and cost-effectiveness
Certain conditions such as newly launched of medicines and health system’s
niche products for a small number of patients sustainability, including on cancer
or the absence of automatic substitution treatment – 2021-2024.
rules for biologicals, can create market
barriers. This means that competing generics, Other actions
biosimilars and ‘older’ products may find
it hard to enter or stay in the market. This ▶ Engage with Members States
lack of competition thus inhibits price in implementing non-legislative
savings once innovative products lose their measures to improve transparency,
market exclusivities. Rules that do not such as guidelines on principles and
directly regulate prices or reimbursement costing methods for establishing the
levels may nevertheless have a bearing on R&D costs of medicines – 2021-2024.
the affordability and cost-effectiveness of
medicines through indirect effects on the ▶ Continue the assessment through the
contestability of markets or the economic European semester of the adequacy
viability of products in more mature markets. and sustainability of national health
The Commission will take this into account in systems and issue country specific
the review of the pharmaceutical legislation, recommendations as relevant to
2020
to see how sound competition can best be ensure they are accessible and
fostered, leading to downward effect on efficient.
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.
14202O
PHARMACEUTICAL STRATEGY FOR EUROPE
3. Supporting a
competitive and
innovative European
pharmaceutical
industryPHARMACEUTICAL STRATEGY FOR EUROPE
3.1. Providing a fertile incentives to reward and protect innovation
environment for Europe’s and create the right conditions for companies
industry of all sizes in the EU to be competitive.
A competitive and resource-efficient EU Intellectual property rights offer
pharmaceutical industry is of strategic protection for innovative products and
interest for public health, economic growth, processes, but in particular for patents and
jobs, trade and science. The EU aims to supplementary protection certificates, there
support industry to be competitive and are differences in their application in Member
resilient so that, in turn, it can better States.
respond to patients’ needs. The sector is
changing rapidly. Established businesses are This leads to duplications and inefficiencies
increasingly outsourcing functions and are thus hampering the competitiveness of
focusing investment on a limited number of industry. The Commission’s intellectual
therapeutic areas, while disinvesting from property action plan 24, includes measures
others. New players, especially technology to simplify and streamline the EU
companies, have entered the market. The pharmaceutical intellectual property system,
coming together of these separate industry notably as regards supplementary protection
segments will transform current business certificates.
models and markets.
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
2020
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
protection 25.
The new industrial strategy for Europe 23 It is essential to continue creating quality
provides key actions supporting industry employment opportunities in the EU
in the EU. Drawing on that framework, the throughout the pharmaceutical value-chain.
pharmaceutical strategy will create a stable To that end, a competitive pharmaceuticals
and flexible regulatory environment industry requires access to a skilled and
that offers legal certainty for investment specialised workforce. NextGenerationEU
and accommodates technological trends. provides unprecedented funding opportunities
This includes providing balanced and fair to support the availability of a skilled
workforce as well as its adaptability and
23. 26 Cf. fn. 10.
24. COM(2020) 760.
25. 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.
16PHARMACEUTICAL STRATEGY FOR EUROPE
the Skills Agenda for Europe 26 sets the way
to make it happen. Specifically, it should Flagship initiatives on
help ensure that all key players in the competitiveness
pharmaceutical sector pool their resources
and invest in upskilling and reskilling of ▶ Optimise the supplementary
all employees throughout the value chain, protection certificates system,
including through commitments that need to make it more transparent
to materialise under the Pact for Skills 27, and efficient as foreseen in the
launched on 10 November 2020. Contributing Intellectual Property Action Plan –
to the increase in STEM 28 specialists the skills 2022.
agenda will aim to boost the number of (male
and female) STEM graduates and teachers ▶ Legislative proposal on a European
by making these studies and careers more Health Data Space, enabling
attractive. Researchers are at the forefront of better healthcare, health research,
science and innovation and require a specific innovation and evidence-based
set of skills too. More will be done to upskill decisions – 2021.
scientists, in line with the skills agenda, and
to foster their mobility across Europe. ▶ Establish by 2025 interoperable data
access infrastructure for the European
Diverse sources of funding are an essential Health Data Space in order to
tool to support innovation. A key element of facilitate secure cross-border analysis
support to the Strategy will be the new and of health data; tested in 2021 with
ambitious standalone EU4Health programme. a pilot project involving EMA and
In addition, Horizon Europe, Cohesion Policy, national authorities – 2021 – 2025.
the European Defence Fund, public-private
and public-public investment partnerships ▶ Support public-private and public-
such as the Innovative Health Initiative 29, and public partnerships, financially and
national schemes are important enablers for technically for example through
R&D, including for small and medium-sized the Innovative Health Initiative,
enterprises (SMEs) and academia. Some of with particular attention to SMEs,
these partnerships may help early uptake of academia, not-for profit organisations,
innovation in health systems. Commission and through the health care systems
initiatives such as the SME strategy for a transformation partnerships – 2021.
sustainable and digital Europe 30, Startup
2020
Europe 31, the European Innovation Council Other action
and the European Institute of Innovation
and technology will help provide the ▶ Prioritise skills investment to support
right environment for SMEs and start-ups the availability of a skilled workforce
active in the health sector to grow and and its adaptability through the
attract venture capital. Likewise, there NextGenerationEU, and within the new
are opportunities to invest in international Recovery and Resilience Facility and
partnerships in the area of health through through commitments under the pact
international cooperation instruments such for skills – 2022.
as the EU external investment plan. At
the same time increased transparency in
the cost of research and development of
pharmaceuticals is needed.
26. COM(2020) 274.
27. The Pact for Skills: mobilising all partners to invest in skills.
28. Science, technology, engineering and mathematics.
29. European Partnership for Innovative Health (Initiative).
30. COM(2020) 103.
31. https://ec.europa.eu/digital-single-market/en/startup-europe.
17PHARMACEUTICAL STRATEGY FOR EUROPE
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 reuse 33.
Digital transformation is affecting the
discovery, development, manufacture,
evidence generation, assessment, supply
and use of medicines. Medicines, medical
technologies and digital health are becoming
3.2. Enabling innovation and increasingly integral to overarching
digital transformation therapeutic options. These include systems
based on artificial intelligence for prevention,
Patients in the EU expect that they can diagnosis, better treatment, therapeutic
benefit from state-of-the art healthcare. monitoring and data for personalised
Scientific and technological advances are medicines and other healthcare applications.
crucial to improve patients’ health, and
support a more efficient, and cost effective, Personalised medicine is an integrated
way of discovering and using medicines. package of healthcare solutions comprising
These advances can translate not only elements of medicines and medical devices
2020
into entirely new medicines, but also into that are structured to meet an individual
alternative uses for existing ones. patient’s needs. In the future, patients may
still be prescribed a tablet but that tablet
Advanced therapy medicinal products and may be combined with a new technology to
some medicines for rare diseases are determine the right usage, the right schedule
challenging concepts, both in terms of and the right dosage according to their
science and manufacturing. An increasing personal situation. This can also support
number of gene and cell therapies under treatments in multi-disciplinary settings like
development may offer curative treatments long-term care. Digital therapeutics can use
and would require a new business model to app-based platforms to help patients manage
address the shift in cost from chronic to one- chronic diseases such as diabetes, depression
time treatment. ‘Bedside’ manufacture 32of and heart conditions, and reduce medication.
more individualised medicines could be a
future trend. Initiatives such as the ‘1+ million
genome’ 34 are exploring ways to access
Vaccines, early detection and the genetic data with the potential to
improvement of well-being can affect improve disease prevention, also via an
32. 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.
33. In line with the EU data strategy, notably for data reuse and business to government data sharing.
34. 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.
18PHARMACEUTICAL STRATEGY FOR EUROPE
enhanced understanding of the impacts into account the experience acquired from
of environmental determinants such as clinical trials for COVID-19 vaccines and
climate change and pollution, allow for treatments. This includes representative
more personalised treatments and provide participation of population groups, for
sufficient scale for new clinically impactful example gender and age groups, that
research including on different cancer types. are likely to use the medicinal product
investigated in the clinical trials to ensure
High performance computing and appropriate safety and efficacy. Pragmatic
artificial intelligence can help accelerate trials, where the treatment is prescribed and
the identification of potential active used as in normal daily practice, can improve
substances for repurposing and reduce the patient compliance and treatment tolerance
high failure rates. Supercomputing is used in by identifying optimal posology and use
the COVID-19 pandemic for example through with other treatments. Commercial interest
the Commission project Excalate4COV. Due is often weaker for these trials, so they are
care should be taken to avoid any gender, mainly organised by academia, where the
race or other bias in the data produced by price of trial medication and insufficient
artificial intelligence. Technological advances regulatory knowledge can be bottle-necks.
can also support the ‘3 Rs’ principles (replace,
reduce, refine) for the ethical use of animals The Commission supports initiatives to
in medicine testing. improve academic researchers and not-for-
profit stakeholders’ regulatory knowledge
The main source of evidence for the via scientific and regulatory advice so
authorisation of innovative medicines should that the evidence they generate can be
remain robust clinical trials with suitable seamlessly used to repurpose off-patent
comparators reflecting the standard of medicines for new therapeutic uses. Industry
care in the EU. The full implementation of engagement and partnership in this process
the Clinical Trials Regulation 35 will put will be promoted.
in place a harmonised, highly coordinated,
robust and agile system for the assessment The new models of product development
and oversight of clinical trials in the EU. It and care delivery mean that regulators are
will improve transparency of information, seeing the limits of the legislation and the
independently of the outcome of the trials, potential needs for regulatory adaptability.
to allow public scrutiny and will address new The rapid developments in products
developments such as adaptive and complex combining medicines and medical devices
2020
trials, and the use of in-silico techniques are reflected in new legislation 36, but some
and virtual approaches. Experience with EU challenges remain. These include clarifying
funded R&I projects with adaptive trials roles and responsibilities, streamlining
shows that research can initiate changes that requirements and procedures, and building
can reduce costs and decrease development up the necessary regulatory expertise and
times. collaboration between sectors. Access to
testing facilities to test devices involving
The Commission will work to ensure that artificial intelligence is important to ensure
the new framework supports innovative the quality of these devices.
trial designs. Moreover, in coordination
with the European regulators, patient The Commission will propose to revise the
groups and stakeholders, it will support pharmaceutical legislation to consider
more patient-oriented design, planning and how to make best use of this transformation.
conduct of clinical trials through harmonised This includes new methods of evidence
international guidance documents and taking generation and assessment, such as
35. 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).
36. 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.
19PHARMACEUTICAL STRATEGY FOR EUROPE
analysis of big and real world data to support
the development, authorisation and use of ▶ Launch a pilot project with
medicines. Regulators may require access to engagement of industry and academia
the raw data at the time of authorisation to to test a framework for repurposing
fully appreciate these innovative elements of off-patent medicines and inform
of the treatment. Also incentivising the possible regulatory action – 2021.
development and validation of relevant
biomarkers would support the effectiveness ▶ Launch a vaccine platform to monitor
in the intake of some new and expensive the effectiveness and safety of
medicines, but also generics, contributing to vaccines, supported by an EU-wide
the sustainability of healthcare systems. clinical trials network – 2021.
▶ Strengthen support and training
of academia and not-for-profit
Flagship initiatives on innovation organisations in regulatory science
for better translation of research into
▶ Propose to revise the pharmaceutical product development – 2022.
legislation, to adapt to cutting-edge
products, scientific developments (e.g. ▶ Initiative for regulatory pilots in a
genomics or personalised medicine) ‘sandbox’ environment provided by the
and technological transformation EMA and the Commission to test the
(e.g. data analytics and digital tools) adaptability of the pharmaceuticals
and provide tailored incentives for framework for new cutting-edge
innovation – 2022. product developments – 2022.
▶ Enhance dialogue among regulatory
and other relevant authorities in 3.3. A sound and flexible
the area of medicines and medical regulatory system
devices to increase cooperation on
evidence generation within their Regulatory efficiency is a prerequisite for
respective fields – 2021. a modern pharmaceutical system. The EU
has continuously updated its framework to
▶ Support collaborative projects ensure that a comprehensive system covers
bringing together stakeholders to take the entire lifecycle of medicines. It builds
on a dual system where the Commission
2020
forward the use of high performance
computing and artificial intelligence in authorises innovative medicines for the entire
combination with EU health data for EU on the basis of a positive opinion from
pharmaceutical innovation – 2021- the EMA and national regulators authorise a
2022. great number of generic and other essential
medicines.
▶ Establish the secure federated access
to 10 million genomes across borders The Commission will explore the need to
for research, innovation and clinical recognise more formally the role of the
applications, including personalised network of national medicines agencies
medicine – 2025. (Heads of Medicines Agencies) and its
operational structure in the regulatory
Other actions system.
▶ Full implementation of the regulatory The Commission will evaluate procedures
framework for clinical trials, which to explore new approaches to assessing
supports innovative trial designs and scientific evidence for the safety and
a more patient-oriented medicine efficacy of medicines as part of the review
development – 2021. 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
20PHARMACEUTICAL STRATEGY FOR EUROPE
technology and artificial intelligence to The regulatory requirements for the
support regulatory decision-making and authorisation of medicines for human use
increase efficiency. The experience of the that contain or consist of genetically
EMA during the COVID-19 pandemic (e.g. modified organisms (GMOs) should be fit
in the rolling review of incoming scientific for purpose when it comes to addressing the
evidence to speed up assessment) will specificities of medicines and the conduct
inform future action. The Commission aims of clinical trials with those products in
to revisit existing regulatory tools, such the EU (which is currently hindered by the
as priority review and scientific advice to fragmentation of national requirements).
support companies, especially SMEs, in the Solutions will be explored during the
development of innovative products for evaluation of the pharmaceutical legislation.
unmet medical needs. In general, consideration should be given to
mechanisms for the continuous and timely
A study 37 on the authorisation and monitoring adaptation of its technical requirements in
of medicines for human use will inform the light of emerging science and technologies
evaluation of the regulatory framework to with a view to enhance effectiveness to
simplify and streamline procedures and protect human health whilst minimising
reduce costs. The management of variations harmful impacts on the environment.
of marketing authorisations and the
assessment of quality files relating to active Regulators also need to adapt to new
substances are two examples of areas in scientific and technological developments by
which simplification is required. A process of upscaling necessary expertise and achieving
reflection will be initiated on the functioning operational excellence to deal with novel
of, and synergies between the scientific and more complex therapies. One important
committees and on the role of patients and factor in this respect is the availability of
healthcare professionals. sufficient funding at all levels. The EMA fees
system is key in funding regulatory activities
Moreover, better use of product at EU level and ensuring coverage of the
information in electronic format (ePI) relevant costs. The Commission will consider
could facilitate the delivery of information on this in the upcoming revision of the EMA fee
the medicine to healthcare professionals and legislation.
patients in the EU’s multilingual environment
and support wider availability of medicines
across Member States. Any measures should
2020
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 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.
37. 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.
21PHARMACEUTICAL STRATEGY FOR EUROPE
Flagship initiatives on regulatory ▶ Consider adapting regulatory
efficiency requirements in the pharmaceutical
legislation, applicable to medicines for
▶ Propose to revise the pharmaceutical human use that contain or consist of
legislation to provide for simplification, genetically modified organisms (GMOs)
the streamlining of approval procedures – 2022.
and flexibility for the timely adaptation
of technical requirements to scientific ▶ Upgrade the Commission’s Union
and technological developments, in order Register of centrally authorised products
to address the challenges relating to the to include a statistical dashboard and
interplay of medicines and devices, and make data fully available for secondary
to strengthen pro-competitive elements use as part of the EU open data initiative
– 2022. – 2021.
▶ Propose to revise the variation ▶ Develop and implement electronic
framework for medicines, through product information (ePI) for all EU
changes in legislation and guidelines, medicines with involvement of Member
to make the lifecycle management of States and industry, evaluate and revise
medicines more efficient and adapted to relevant provisions in the legislation –
digitalisation – 2021-2023. 2022.
Other actions ▶ Propose to revise legislation to give
regulatory authorities more power to
▶ Proposal for revised EMA fee legislation adapt on their own initiative the terms of
– 2021. marketing authorisations on the basis of
scientific evidence – 2022.
▶ Provide for a single assessment process
across Member States for active ▶ Simplify and streamline the system of
substances used for different generic penalties to address non-compliance in a
medicines (active substance master proportionate and efficient way – 2024.
files) to facilitate their authorisation and
life-cycle management – 2022.
2020
22202O
PHARMACEUTICAL STRATEGY FOR EUROPE
4. Enhancing resilience:
Diversified and
secure supply chains;
environmentally
sustainable
pharmaceuticals; crisis
preparedness and
response mechanismsPHARMACEUTICAL STRATEGY FOR EUROPE
4.1. Secure the supply of
medicines across the EU and
avoid shortages
The European Council 38 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 used 39.
The reasons are complex; they include
marketing strategies, parallel trade, scarce
active pharmaceutical ingredients and raw cooperation between Member States, for
materials, weak public service obligations, example improved procurement approaches
supply quotas or issues linked to pricing and and strategies, joint procurement for critical
reimbursement. medicines and EU-level cooperation on tools
and instruments for national policy making
Building up EU’s open strategic autonomy
on prices and reimbursement. For products
in the area of medicines requires actions to
with small volumes or limited use, new
identify strategic dependencies in health,
business contracting and/or payment models
and to propose measures to reduce them,
will be crucial.
possibly including by diversifying production
and supply chains, ensuring strategic Pharmaceutical manufacturing and
stockpiling, as well as fostering production supply chains are complex, increasingly
and investment in Europe. Minimising globalised and sometimes not sufficiently
the impact of medicines shortages on
2020
diversified. Multiple actors, in different parts
patient care will require both preventative of the world with varying environmental
and mitigating measures to significantly performance of the production processes,
reinforce the obligation of continuous supply. may be involved in the various production
This year, the Commission launched a study steps for a single ingredient. Certain
to map the root causes of shortages and technologies necessary for the production
assess the legal framework. The study will of raw materials are no longer available in
inform the evaluation and revision of the the EU. Even before the COVID-19 pandemic
current legislation. Legislative measures there were concerns about the resilience of
could include stronger obligations on pharmaceutical manufacturing chains, both
industry to ensure the supply of medicines, the European Parliament and Member States
earlier notification of shortages and have called on the Commission to address
withdrawals, enhanced transparency of this issue 40. Notably as regards the supply of
stocks across the supply chain, and a raw pharmaceutical materials, intermediates
stronger coordinating role for the EMA in and active pharmaceutical substances, which
monitoring and managing shortages. Such may contribute to risks of shortages of
measures will be complemented by enhanced critical medicines. The pandemic showed that
38. 41 European Council Conclusions of 2 October 2020 (EUCO 13/20).
39. Pharmaceutical Group of European Union (PGEU), Medicine shortages survey: 2019 results.
40. 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).
24You can also read
