Foreword
European Healthcare: So much we can build on, so much left to do
NATHALIE MOLL
EFPIA Director General
No one sector has all the answers and by working together we can continue to transform the lives of patients and their families, make Europe a world leader in medical R&D and drive the growth of the European economy.
During 2019, EFPIA will look to continue to promote collaboration as a partner in the fight against disease
We will continue to work with governments and health system stakeholders to find solutions that ensure patients get rapid access to new treatments
We can continue to drive Europe’s health success story
FIND OUT MORE
The challenges faced by healthcare in Europe is often the subject of debate in Brussels. And despite the many challenges, it really is worth remembering that the rising demand for health services driven by an ageing population is the result of a European success story.
Over the last 50 years, life expectancy across the EU has risen by over a decade. We have seen dramatic advances in prevention and in treatment for diseases such as cancer, heart disease, HIV, inflammatory disease and hepatitis C. With over 7000 medicines in development and rapid advances in diagnostics, devices, imaging and data analytics there are many reasons to be optimistic about the future health of Europe.
To realise this potential will require new levels of partnership and collaboration. No one sector has all the answers and by working together we can continue to transform the lives of patients and their families, make Europe a world leader in medical R&D and drive the growth of the European economy.
Much of the focus in 2018 has been on partnership and collaboration to tackle some of these grand challenges together. For example, the EU Health Summit, in which EFPIA was a contributing partner, brought together 29 organisations from across healthcare and the life sciences sector, developing concrete recommendations for the new EU Commission and Parliament to action on health.
Discussions around Horizon Europe and Europe’s new Public Private Partnership for Health have focused on going beyond medicines to include partners from diagnostics, devices and data analytics. EFPIA entered in to a strategic partnership with the International Consortium for Health Outcomes Measurement to standardise definitions of outcomes to facilitate more patient-centred, outcomes-focused health systems. There are many more examples included in this Annual Report.
During 2019, EFPIA will look to continue to promote collaboration as a partner in the fight against disease. Our role in that fight is to develop new treatments and cures that can transform the lives of patients. Access to those new treatments is a goal we share with everyone in healthcare. We need to create an environment that fosters and supports the development of new treatments for diseases like dementia, diabetes, AMR and cancer knowing that innovation is only meaningful when patients get access to it. In 2019 we will continue to work with governments and health system stakeholders to find solutions that ensure patients get access to the new wave of treatments coming through and we can continue to drive Europe’s health success story.
Foreword
European Healthcare: So much we can build on, so much left to do
STEFAN OSCHMANN
EFPIA President
Chairman of the Executive Board and CEO of Merck
Patients must play a central role in shaping how medicines are developed, how services are designed and how chronic conditions are managed. As an industry, we are committed to engaging patients in all of these processes.
When I took the reins as EFPIA president, we focused on three factors: patient engagement, data and collaboration.
FIND OUT MORE
Europe has made great progress in healthcare. An EU citizen born today can expect to live 30 years longer than his or her ancestors a century ago. And there is much to suggest that this trend will persist. From immuno-oncology to gene and cell therapy, we are currently seeing the emergence of new medicines that give hope to patients who until recently had no treatment options.
I am personally convinced that there has never been a more promising time for pharmaceutical innovation. However, Europe also faces major healthcare challenges. Demographic change will see ageing societies exert significant cost pressure on healthcare systems. At the same time, high unmet medical need remains in diseases like cancer or Alzheimer’s. This requires continued large-scale investments in research and development. The pharmaceutical industry has a critical role to play in tackling these challenges.
When I took the reins as EFPIA president, we focused on three factors: patient engagement, data and collaboration. Patients must play a central role in shaping how medicines are developed, how services are designed, and how chronic conditions are managed. As an industry, we are committed to engaging patients in all of these processes.
Working through collaborative vehicles we have moved from talking about patients to talking with patients to help shape policy and practice. This approach was typified by last year’s EU Health Summit, where organizations from across healthcare came together to make recommendations to support the creation of patient-centred health systems.
Furthermore, the broad availability of health data and digital technologies open new possibilities. Just one example: by using data more effectively, we can accelerate the transition to outcomes-focused healthcare systems.
To that end we have been working with the International Consortium for Health Outcomes Measurements to bring together experts, patients and their families to build a holistic view of the outcomes that matter to patients. These enable meaningful measurement of what healthcare systems are actually delivering. Finally, collaboration is and remains key. During my presidency, we celebrated the 10th anniversary of the Innovative Medicines Initiative (IMI). Under this initiative, which was jointly launched by the European Commission and EFPIA, partners have initiated projects across a huge range of therapeutic areas. It has led to big advances in identifying biomarkers, developing a vaccine for the Ebola crisis and contributed to our understanding of – and how we manage – disease. Two years ago, we launched the #WeWontRest story that underlines our commitment, dedication and passion to discover, develop and deliver new medicines for patients. That commitment remains as strong as ever. Today, we can look forward to continuing to work with partners across the research and health communities to meet the needs of patients across Europe.
About EFPIA
EFPIA Activity 2018
Click on each dates to view activity details
- JAN 18
LEGAL AFFAIRS
- EFPIA commissioned research on GDPR implementation which highlighted the need for harmonised approaches and for a more proactive response from responsible authorities
- EFPIA response to the Commission’s consultation on supplementary protection certificates (SPCs) and patent research exemptions
- Publication of the Review of CRA’s Report “Assessing the economic impacts of changing exemption provisions during patent and SPC protection in Europe” written by OHE
VACCINES EUROPE
An article was published in the journal Vaccine on “Innovation Partnership for a Roadmap on Vaccines in Europe (IPROVE): A vision for the vaccines of tomorrow”
EUROPEAN BIOPHARMACEUTICAL ENTERPRISES
EBE published a reflection paper on “Medicinal product incorporating a drug delivery device component: An industry perspective on the EU marketing application technical requirements, regulatory review process and post-approval device related change assessment”
- FEB 18
SCIENCE POLICY AND REGULATORY AFFAIRS
- EFPIA joined industry and RTO associations in calling for an appropriate design and budget for the Horizon Europe programme
- Joint industry (EFPIAAESGP-MfE) submission on EU Pharmaceutical Environment Strategy
- Presentation on ICH to the Turkish regulator, TITCK, Ankara
- MAR 18
SCIENCE POLICY AND REGULATORY AFFAIRS
- Kick-off of the IMI PARADIGM project on Patient Engagement
- IMI Call 14 launched: 4 topics on immunology, remote clinical trials and machine learning, €167 million budget
- EFPIA response to the EU consultation on funding for research and innovation
- IMI ADAPT-SMART project which EFPIA was a member of held its closing event in Budapest
VACCINES EUROPE
VE issued a paper on “Immunisation Information Systems at the cornerstone of improved vaccination policies in a digital Europe”
- APR 18
COMMUNICATIONS AND PARTNERSHIPS
- “Working Together with Patient Organisations” workshop in Berlin, Germany
- Launch of Brighter Tomorrow
INTERNATIONAL AFFAIRS
- EFPIA workshop with China Pharmaceutical Industry Association (CPIA) on common regulatory matters for the international and Chinese manufacturers
- EFPIA co-organised the launch workshop for the Eurasian Common Medicines Market with AIPM and the Eurasian Commission
LEGAL AFFAIRS
Presentation at the workshop on SPC manufacturing waiver held by the Permanent Representation of Poland to the European Union
SCIENCE POLICY AND REGULATORY AFFAIRS
- ICON, Medidata and Q-linea joined as Partners in Research
- EFPIA-IMI workshop on Disease Interception
- IMI2 SGG Digital Health and Patient Centric Evidence Generation kick-off
VACCINES EUROPE
VE issued a position on the IPROVE Industry R&D priorities
EUROPEAN BIOPHARMACEUTICAL ENTERPRISES
EBE organised 3 sessions at the DIA Europe 2018 conference focusing on advanced therapies’ development, implementation of the in vitro diagnostics regulation, and health economics linked to personalised medicine
- MAY 18
ECONOMIC AND SOCIAL AFFAIRS
EFPIA created Alzheimer’s Working Group
INTERNATIONAL AFFAIRS
EFPIA participated in the St. Petersburg International Economic Forum (SPIEF)
LEGAL AFFAIRS
The GDPR came into effect
PUBLIC AFFAIRS
EFPIA exhibition “Unlocking Tomorrow’s Cures” in the European Parliament
- JUN 18
COMMUNICATIONS AND PARTNERSHIPS
- EFPIA 40th Anniversary
- Health Collaboration Awards Call for Applications
INTERNATIONAL AFFAIRS
- Turkey’s observer membership of ICH was approved at the ICH general assembly
- Russian authorities approved amendments to Federal Law 61, making it possible for international firms to carry out the GMP certification process in parallel with marketing authorization procedures
LEGAL AFFAIRS
Third disclosure of transfers of value to HCPs, HCOs and POs around Europe by EFPIA members as part of the industry commitment to transparency was carried out.
SCIENCE POLICY AND REGULATORY AFFAIRS
- IMI 10th Anniversary - Conference on Medical Innovations
- IMI Think Big workshop on Advanced Therapies
- Science 37 joined as Partner in Research
- EFPIA statement on Horizon Europe calling for an ambitious health research programme and supporting European Partnerships
- HSA, Singapore, MFDS, Korea and NMPA, China become members of ICH Management Committee and TFDA, Chinese Taipei become member of ICH
- EFPIA experts participated and engaged in Commission Expert Working Group on developing guidance on Genetically altered animals
VACCINES EUROPE
- An article was published in the journal Nature on “Impact of the Innovative Medicines Initiative on vaccine development”, coauthored by the VE Executive Director Magdalena de Azero and EFPIA Executive Director for Science Policy and Regulatory Affairs Magda Chlebus
- VE issued a paper on “Improving Access and Convenience to Vaccination”
- JUL 18
INTERNATIONAL AFFAIRS
The expanded EU-Japan Mutual Recognition Agreement (MRA) on Good Manufacturing Practices (GMP) inspections entered into force
SCIENCE POLICY AND REGULATORY AFFAIRS
IMI Calls 15 & 16 launched: 15 topics, including the AMR Accelerator programme, integrated research platforms, blockchain enabled healthcare, liquid biopsies, safety tools for human immuno-biology, synaptic plasticity, digital endpoints in neurodegeneration and immune diseases, €433 million budget
EUROPEAN BIOPHARMACEUTICAL ENTERPRISES
- EBE published together with EFPIA an evidence-based analysis demonstrating the benefits of personalised medicines to patients, society and healthcare systems
- EBE published together with EFPIA a reflection paper “Industry perspective on Article 117 of the EU Medical Devices Regulation and the impact on how Medicines are assessed”
- AUG 18
SCIENCE POLICY AND REGULATORY AFFAIRS
A multistakeholder working group formed under STAMP to further discuss the proposal on repurposing
- SEP 18
ECONOMIC AND SOCIAL AFFAIRS
HMA/EMA workshop on shortages and availability of medicines (involving EU-28 Heads of Medicines Agencies, Commission and Stakeholders)
STRATEGY AND HEALTHCARE SYSTEMS
EFPIA together with ICHOM and Pharma Industry Finland organised a multi-stakeholder event on outcomes-based healthcare in Helsinki
LEGAL AFFAIRS
- EFPIA Position Paper: EU Directive introducing Europe-wide representative actions for the protection of collective interests of consumers
- EFPIA participated in a technical seminar on the SPC manufacturing waiver organised by the Permanent Representation of Denmark to the European Union
SCIENCE POLICY AND REGULATORY AFFAIRS
- EFPIA poster at the 21st European Congress on Alternatives to Animal Testing and 18th Annual Congress of EUSAAT (European Society for Alternatives to Animal Testing)
- EFPIA and CEFIC informed by the Commission of a successful submission to ensure pharmaceutical companies will be placed on the carbon leakage list
- EFPIA and other life science associations presented at European Chemicals Agency (ECHA) committees on diversities between the chemical and pharmaceutical sectors
VACCINES EUROPE
VE participated in the launch of the EU Joint Action on Vaccination as a collaborative stakeholder for the next 3 years of the project
EUROPEAN BIOPHARMACEUTICAL ENTERPRISES
EBE developed a joint position with EFPIA on the need to consider a risk-based approach for biomarker assay development in clinical trials as an alternative to CE marking
- OCT 18
STRATEGY AND HEALTHCARE SYSTEMS
- EFPIA together with ICHOM and pharma. be organised an event on outcomes-based healthcare in Brussels
- EFPIA together with ICHOM and Farmaindustria organised a multistakeholder event on outcomes-based healthcare in Madrid
- EFPIA Diabetes Platform supported the launch of the European Diabetes Forum (EUDF)
LEGAL AFFAIRS
- EFPIA organised a workshop to raise concerns regarding the impact on clinical research
- EFPIA published a position paper on the Copenhagen Economic (CE) study on “the economic impact of supplementary protection certificates, pharmaceutical incentives and rewards in Europe”
- EFPIA published a position paper on the Max Planck Institute (MPI) legal study on “the legal aspects of Supplementary Protection Certificates in the EU”
SCIENCE POLICY AND REGULATORY AFFAIRS
- EFPIA participated in a Commission workshop to review and develop templates for Non-technical Project Summaries and the results of Retrospective Assessment in animal experiment design
- IMI 10th anniversary Scientific Symposium
- IMI Stakeholder Forum on the value of cross-sectorial health research and innovation
- Big Data for Better Outcomes Programme’s Stakeholder symposium
EUROPEAN BIOPHARMACEUTICAL ENTERPRISES
EBE published jointly with EFPIA a position paper on Next Generation Sequencing (NGS) to explain the complexity of the technology involved and the regulatory challenges faced by pharmaceutical companies in Europe
- NOV 18
COMMUNICATIONS AND PARTNERSHIPS
Launch of ForTheChance, the patient story perspective of the #WeWontRest campaign
STRATEGY AND HEALTHCARE SYSTEMS
- EFPIA together with ICHOM and ABPI organised a multi-stakeholder event on outcomes-based healthcare in London
- EFPIA and 27 other European health policy organisations organised the EU Health Summit in Brussels, to present and discuss 20 recommendations on EU health policy for the next European Commission and Parliament that they had developed jointly over 9 months
INTERNATIONAL AFFAIRS
EFPIA Vice-President Jean-Christophe Tellier represented EFPIA during the annual EFPIA Japan Day, co-organised with EFPIA Japan
LEGAL AFFAIRS
EFPIA presented comments to the MPI study on “the legal aspects of Supplementary Protection Certificates in the EU”
SCIENCE POLICY AND REGULATORY AFFAIRS
- BenevolentAI joined as Partner in Research
- Kick-off of the IMI EHDEN project (Electronic health data in a European network)
- EFPIA with 5 inter-sector health industries joined forces calling for a future health Public Private Partnership
- DEC 18
COMMUNICATIONS AND PARTNERSHIPS
EFPIA & ITN Productions launched the joint project #WeWontRest film
ECONOMIC AND SOCIAL AFFAIRS
EFPIA and EUnetHTA meeting to discuss the Joint Action 3
INTERNATIONAL AFFAIRS
EFPIA became a member of the European Chamber of Commerce (EUCCC) in China
SCIENCE POLICY AND REGULATORY AFFAIRS
- EFPIA Submission to the Chemicals agency, ECHA, on microplastics in the pharma sector which lead to a derogation from restrictions for pharma sector
- EFPIA response to the EC Roadmap to establish a Format for a European Electronic Health Record (EHR) Exchange
- Common cross-industry response on ICH Q12 was submitted to EMA
- Inter-association response on the “Guidance on detection and notification of shortages of medicinal products for Marketing Authorisation Holders (MAHs) in the Union (EEA)” and EMA/548142/2018 Draft Version “Proposed metrics of shortage”
EUROPEAN BIOPHARMACEUTICAL ENTERPRISES
- EBE organised its 7th annual regulatory conference on Advanced Therapies: “Advancing the delivery of ATMPs to patients”
- EBE published a concept paper on “Management and control of raw materials used in the manufacture of biological medicinal products and ATMPs”
#WeWontRest
Illness never sleeps, so neither do we. The research-based pharmaceutical industry is working tirelessly to develop new treatments and cures for patients and #WeWontRest until we win the fight against disease.
With nearly 7,000 medicines in development, 74% of which are potentially first-in-class, the exciting new wave of medical innovation will play a key role in addressing the challenges faced by patients and healthcare systems.
CANCERS
CARDIOVASCULAR
DISORDERS
DIABETES
HIV / AIDS
IMMUNOLOGICAL
DISORDERS
INFECTIOUS
DISEASES
MENTAL HEALTH
DISORDERS
NEUROLOGICAL
DISORDERS
Source: PhRMA
84
medicines
recommended
for approval
by EMA
42
new active
substances
EFPIA shares a common goal with all partners in healthcare: to ensure that patients across Europe get rapid access to the latest, effective and life-saving medicines.
CANCER
Every year, more than 3.4 million people are diagnosed with cancer in Europe. (2) 558,000 breast cancer patients treated between 2007-2017, resulted in a gain of 1,180,000 Healthy Life Years (HLYs)* across the patient population. (3)
The EFPIA Oncology Platform aims to transform the lives of patients diagnosed with cancer. #WeWontRest until cancer is nothing to fear.
ALZHEIMER’S
Today, over 11 million people in Europe are living with Alzheimer’s Disease and other forms of dementia, with the number expected to increase to 14 million by 2030. (4,5) There are currently 135 investigational trials ongoing in treatments for Alzheimer’s disease. (6)
The EFPIA Alzheimer’s Platform aims to ensure a brighter today and tomorrow for people with Alzheimer’s disease. #WeWontRest until Alzheimer’s patients have a brighter future.
DIABETES
Nearly 1 out of 10 people in Europe have diabetes, amounting to around 60 million people. (7) By 2045 this number is expected to increase by 22% to 81 million people. (8)
The EFPIA Diabetes Platform aims to improve the lives of everyone
affected by diabetes. #WeWontRest in the fight against diabetes.
Collaboration
Europe is facing significant challenges across its healthcare systems. No one sector has all the answers. Collaboration is key. By working together, we can win the fight against disease. #WeWontRest in working with partners across the health and research sectors to better the lives of patients. Here are just some of the examples of EFPIA working with partners to deliver more for patients.
119
IMI projects
(signed grant agreements) as of end 2018
20
grants
for a total value of EUR 546 million
13 projects kicked off in 2018
- PARADIGM
- EHDEN
- c4c
- Hypo-Resolve
- iConsensus
- PIONEER
- EBOVAC3
- AIMS-2-TRIALS
- GetReal Initiative
- ReSOLUTE
- EFOEUPATI
- WEB-RADR2
- ESCulab
26
Partners in Research
that committed EUR 36.7 million to IMI2 projects as of 31 December 2018
3
calls
were launched, including 19 topics, with a total budget of EUR 600 million for which 17 proposals were selected for funding
20+ active industry members
20+ active patient organisations
11 overarching topics covered through 2018
4 “Working Together” national workshops to date (CEE Region, Germany, Greece, Turkey)
80+ projects applied for the Patient Think Tank initiated Health Collaboration Awards
28+ partner organisations
400 participants
20 recommendations
3 prioritised recommendations by the audience on outcomes, prevention and collaboration
Continuation of the initiative beyond the Summit – EU Health Coalition with growing number of partner organisations
& 
4
workshops
organised in 2018 in Helsinki, Madrid, Brussels and London. Always in collaboration with our national trade association members
2 workshops to be organised in 2019 in Lisbon and Warsaw
450 participants with a diversified composition of patients, industry, payers, healthcare professionals, hospitals, national medicines agencies, national and EU policy-makers
The economic impact of the industry
The research-based pharmaceutical industry can play a critical role in restoring Europe to growth and ensuring future competitiveness in an advancing global economy.
Europe is a global leader in highly-skilled, science-based workforce
EU-28 trade balance – High technology sectors (€ million) – 2018
The research-based pharmaceutical industry is a key asset of the European economy – one of Europe’s top performing high-technology sectors.a
The impact of vaccines
#WeWontRest until we live in a world in which no one suffers or dies from a vaccine-preventable disease
Global measles deaths have declined by 84% from an estimated 550,000 deaths in 2000 to 89,780 in 2016. Accelerated immunisation activities have had a major impact on reducing measles deaths.(1)
Immunization currently prevents between
2,000,000 – 3,000,000
deaths every year. Immunization is one of the most successful and cost-effective public health interventions.
An additional of
1,500,000
could be avoid, however, if global vaccination coverage improves.
More children are being immunized worldwide than ever before. During 2016, an estimated 116.5 million (about 86%) children under the age of one year worldwide received three doses of diphtheria-tetanus-pertussis (DTP3) vaccine. These children are protected against infectious diseases that can cause serious illness or disability, and be fatal.
Influenza vaccination helps reduce complications from chronic diseases
-28%
deaths in diabetic patients
-79%
hospital
admission
-50%
heart attack
occurrence
Risk of experiencing a Heart Attack after systemic respiratory infections
-24%
the risk of
stroke
occurrence
Risk of experiencing a Stroke after systemic respiratory infections
significant
reduction of
exacerbation
N°1 of Risk group for influenza mortality
Vaccines are critical in helping to combat the growing antimicrobial resistance (AMR) crisis
PNEUMOCOCCAL
Universal coverage by a pneumococcal conjugate vaccine could avert 11.4 million days of antibiotic use per year in children younger than five(3).
Childhood immunisation against S. pneumoniae is the most effective public health measure for preventing IPD both among vaccine recipients (direct effect), and among unimmunised populations(4).
ANTIBIOTIQUE USE
MOST EFFECTIVE PUBLIC HEALTH MEASURE
EFPIA represents the pharmaceutical industry in Europe. Through its direct membership of 36 national associations and 39 leading pharmaceutical companies, EFPIA’s mission is to create a collaborative environment that enables our members to innovate, discover, develop and deliver new therapies and vaccines for people across Europe, as well as contribute to the European economy. Our vision is for a healthier future for Europe – a future based on prevention, innovation, access to new treatments and better outcomes for patients.
European Medicines Agency (EMA, 2018). Human medicines highlights 2018. Retrieved from https://www.ema.europa.eu/en/documents/report/human-medicines-highlights-2018_en.pdf
(2) Bengt Jönsson et al. Comparator Report on Patient Access to Cancer Medicines in Europe Revisited, 2016 - page 4: Cancer incidence increased by 31% from 1995 to 2012 (external reference: Ferlay J, et al. Cancer incidence and mortality patterns in Europe: estimates for 40 countries in 2012. Eur J Cancer. 2013;49(6):1374-403)
(3) EFPIA (2019). The economic and Societal Footprint of the Pharmaceutical Industry in Europe
(4) Alzheimer Europe. Cost of illness and burden of dementia in Europe – Prognosis to 2030. Retrieved from www.alzheimer-europe.org/Research/European-Collaboration-on-Dementia/Cost-of-dementia/Prognosis-to-2030
(5) EurActiv (2016). Rising dementia numbers in EU causes alarm. Retrieved from www.euractiv.com/section/health-consumers/news/rising-dementia-in-the-eu-causes-alarm/
(6) European Medicines Agency (EMA, 2018). Revised guideline on clinical studies for Alzheimer’s disease medicines. Retrieved from www.ema.europa.eu/en/news/revised-guideline-clinical-studies-alzheimers-disease-medicines
(7,8) IDF Diabetes Atlas Eight Edition, International Diabetes Federation 2017
(9) EUROSTAT (2015). Retrieved from https://ec.europa.eu/eurostat/statistics-explained/index.php?title=Glossary:Healthy_life_years_(HLY)
* Healthy Life Years (HLYs), also called disability-free life expectancy (DFLE), is defined as the number of years that a person is expected to continue to live in a healthy condition.(9)
(1) Recommendations in full: https://www.euhealthsummit.eu/wp-content/uploads/Future-of-Health-recommendations-in-full-new.pdf; key recommendations: https://www.euhealthsummit.eu/wp-content/uploads/Future-of-Health-key-recommendations.pdf
(1) World Health Organisation (WHO, 2018). 10 facts on immunisation. Retrieved April 08, 2019, from https://www.who.int/features/factfiles/immunization/en/
(2) Euractiv (2018). Vaccination: raising awareness against fake news. Retrieved from https://en.euractiv.eu/wp-content/uploads/sites/2/special-report/EURACTIV-Special-Report-Vaccination-Raising-awareness-against-fake-news.pdf
(3) Laxminarayan R. et al (2016). Access to effective antimicrobials: a worldwide challenge. http://dx.doi.org/10.1016/S0140-6736(15)00474-2 (4) European Centre for Disease Prevention and Control. Prevention and control measures for pneumococcal disease. Retrieved April 08, 2019, from https://ecdc.europa.eu/en/pneumococcal-disease/prevention-and-control
*CORRECT AT DATE OF PUBLICATION