26 June 2019 - 12:30 - 14:00
Martin Seychell, Yann Le Cam, Tamsin Rose

The EU has already made great progress on the treatment of rare diseases. However, persistent gaps remain. Of the 5000 to 8000 rare diseases that exist in the EU, 95% do not have an effective therapy. On top of that, about a quarter of orphan drugs fail to reach marketing authorisation. Our 2017 debate on the subject revealed that budget pressures, bureaucratic obstacles and problems of scale are preventing rare disease patients from getting the treatment they need. During this lunch debate, we want to explore how the EU can move from evidence and incentives to equal access to new medicines for patients across the different health systems in the EU.

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Health is an economic and social driver for Europe. Demographic shifts, greater demand and ongoing financial restrictions are putting European health systems under extreme pressure. 


Networking lunch and registration of participants


Equal access to care for rare diseases

Rare diseases are both a European success story and an area of persistent gaps. The good news: the 2000 EU Orphan Medicines Regulation has improved the drug development pipeline, resulting in over 150 new medicines authorised on the market. Similarly, the 2007 Paediatric Regulation has led to 267 new medicines and 43 new pharmaceutical forms appropriate for children. With an estimate of 5000 to 8000 distinct rare diseases existing in the EU, 95 per cent still do not have an available therapy, and a large percentage of orphan drugs fail to reach marketing authorisation. Gaps still persist in knowledge and treatments, frequently with an overlap because rare diseases often occur in children.

The European Commission published end of 2017 an evaluation and fitness check roadmap for its joint evaluation of the legislation on medicines for children and rare diseases, which will serve as a basis for the 2018 review and analysis of impact of pharmaceutical incentives on innovation as well as the availability and accessibility of medicines. 

•    Has the EU Orphan Medicines Regulation achieved its aims? What impact did it have?
•    How can the EU move from incentives and evidence to action and equal access to new medicines for patients across the different health systems in the EU?
•    Launched around two years ago, what impact are the European Reference Networks (ERNs) having? 
•    What impact does the advancement on genomics and the growth in ‘personalised medicines’ have on development for rare diseases?
•    How to encourage and foster the repurposing of drugs?

Martin Seychell
, Deputy Director-General for Health and Food Safety
Yann Le Cam, Chief Executive Officer at EURORDIS

Moderated by:
Tamsin Rose
, Senior Fellow at Friends of Europe


End of debate

Martin Seychell
Deputy Director-General for Health and Food Safety
An expert in chemistry and pharmaceutical technology, Martin Seychell has held important positions in several government boards and commissions in Malta, including the post of Director of Environment as well as Advisor to the Prime Minister. He was in charge of implementing a number of EU directives in the areas of risk assessment, food safety and chemicals legislation. Within his current role as European Commission Deputy Director-General at DG Santé, Seychell spearheads the directorates dealing with Consumer affairs, Public health and Health systems and products, including the performance of national health systems, country knowledge and EU health programmes.
Yann Le Cam
Chief Executive Officer at EURORDIS
Yann Le Cam has dedicated 25 years to raising awareness of rare diseases as a public health priority in Europe and beyond. In addition to co-founding and leading Eurordis, which gathers today 751 rare disease patient organisations in 66 countries, he has held a number of high-ranking positions in leading medical research NGOs and patient organisations. He is also recognised for his particular contribution to the adoption of many European regulations that impact the lives of rare diseases patients. In 2016, Le Cam was elected to the Management Board of the European Medicines Agency.
Tamsin Rose
Senior Fellow
Tamsin Rose is Senior Fellow at Friends of Europe. Having studied international relations, she has 25 years of experience working across the European continent from Ireland to Mongolia. A natural communicator, Tamsin has been a radio reporter, worked on press for the EU Delegation in Moscow and is currently a member of the external speaker team for the European Commission Directorate General for Communication, describing how the EU works and key policies to visitor groups from around the world. Since 2002 she has specialised in public health and public participation issues, serving as Secretary General of the European Public Health Alliance (EPHA), and providing strategic advice for health groups on how to engage successfully with the EU.



This event is exclusively for Friends of Europe’s members, EU institution representatives and media.

Clara Casert, Programme Assistant
Tel.: +32 2 893 98 25
Email: clara.casert@friendsofeurope.org

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