EQUAL ACCESS TO CARE FOR RARE DISEASES

26 June 2019 - 12:30 - 14:00
Rute Fernandes, Yann Le Cam, Martin Seychell, Till Voigtländer, Tamsin Rose
Introduction

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.

Related content:

Topics

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. 

Programme
12.30-13.00

Networking lunch and registration of participants

13.00-14.00

Rare diseases are both a European success story and an area of persistent gaps. The good news: the 2000 EU Orphan Medicines Regulation has significantly improved the drug development pipeline, resulting in over 150 new medicines authorised on the market. With an estimate of 5000 to 8000 distinct rare diseases existing in the EU, 95% still do not have the available therapy, and a large percentage of orphan drugs fails 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 will soon adopt a joint evaluation of existing legislation on medicine for children and rare diseases, taking a two-pronged approach. First and foremost, the Commission intends to assess the efficiency of EU legislation by analysing the impact that pharmaceutical incentives have on innovation. Secondly, it plans to gauge the availability and accessibility of medicines in the EU. Building on the foundations set out by the last four EU mandates on health, the priority now is to determine an effective strategy for translating EU incentives, knowledge and networks into better access to treatment for rare disease patients.

  • Has the EU Orphan Medicines Regulation achieved its aims? If so, what impact has it had?
  • 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?

Speakers:
Rute Fernandes
, Group Vice President and Head of Rare Diseases for Europe and Canada at Takeda
Yann Le Cam, Chief Executive Officer of the European Organisation for Rare Diseases (EURORDIS)
Martin Seychell, European Commission Deputy Director General for Health and Food Safety
Till Voigtländer, Austrian Representative to the Board of Member States of the European Reference Networks (ERNs)

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

14.00

End of debate

Speakers
Rute Fernandes
Group Vice President and Head of Rare Diseases for Europe and Canada at Takeda
Rute Fernandes is a member of the Europe and Canada Executive Team of Takeda, the patient-focused, values-based and Research and Development-driven global biopharmaceutical company. In her current role, Fernandes leads the Rare Disease Franchise covering 3 disease areas in Rare Metabolic Disorders, Rare Hematology and Rare Immunology, across 38 countries. Previously, Fernandes held senior executive roles at Shire as General Manager Switzerland, Head of Global Commercial Operations, Head of Patient Services and Commercial Excellence for International. Over the years, she has also worked for Novartis, Procter and Gamble and Vodafone, building on her extensive experience in strategy, finance, commercial development and industry.
Yann Le Cam
Chief Executive Officer of the European Organisation for Rare Diseases (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.
Martin Seychell
European Commission 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.
Till Voigtländer
Austrian Representative to the Board of Member States of the European Reference Networks (ERNs)
Till Voigtländer is the Austrian Representative to the Board of Member States of the European Reference Networks – virtual networks of European healthcare providers that aim to facilitate discussion on rare diseases. In his home country of Austria, he heads the National Coordination Centre for Rare Diseases and was in charge of drawing up the National Action Plan for Rare Diseases. Voigtländer has also been the Austrian Coordinator for Orphanet, a reference database for rare diseases, since 2004. In addition to that, he is Associate Professor at the Medical University of Vienna.
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.
Partners
Takeda_logo EURORDIS logo
   
   

 

Registration

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

Augusta Ramaccioni, Programme Executive
Tel: +32 2 893 98 23  
Email: augusta.ramaccioni@friendsofeurope.org

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