IMPROVING ACCESS, CARE AND DIAGNOSIS FOR RARE DISEASES

21 June 2017 - 12:30 - 14:00
Yann Le Cam, Nathalie Moll, Xavier Prats Monné, Tamsin Rose, Maurizio Scarpa, Kim Stratton
Introduction

People with a rare disease face significant challenges and most rare diseases still take a long time to be diagnosed. Many patients go through years of testing and misdiagnosis before finally getting to see a specialist who knows what they are seeing in front of them. Only 5% of rare diseases have therapies to date – few curative, some transformative, most improving and prolonging life. Rare diseases are an area where the added value of the EU is obvious, as it can coordinate the provision of appropriate diagnosis and care to patient populations that would otherwise be too small and disparate. The most effective strategies are collaborative across borders, and the EU has a track record of great achievements through cross-country collaboration. Rare diseases are also a good example of the benefits of a universal access health system – allowing the sharing of risk across the whole population and ensuring the supply of expensive diagnosis and medicines that would be unaffordable for the families concerned on an individual basis. To respond to the challenges, the EU has introduced legislation to create a favourable environment to invest in new medicines to treat rare diseases, which have increased the range of therapies available through appropriate incentives. But improving the pipeline of new medicines is only part of the picture for rare diseases: key hurdles include decisions by competent authorities on whether such therapies would be reimbursed and therefore accessible to patients. What more could the EU do to support member state efforts to improve the current system through analysis, recommendations and pilot projects? The EU has also launched new European Reference Networks (ERNs), which aim to provide highly specialised care for patients with complex conditions. They bring together clinicians, researchers and patients to improve the knowledge base as well as care through cross-border collaboration.

  • What role can ERNs play to increase the chances for rare disease patients to receive an accurate diagnosis and treatment?
  • Which mechanisms in healthcare systems need to be changed in order to get different actors in health to work together more effectively?
  • As personalised medicine becomes more of a reality, how can patients with rare diseases benefit?
  • How can the EU help member states improve access to medicines for rare diseases and provide appropriate care at reasonable cost?

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Programme
12.30 – 13.00

Welcome lunch and registration of participants

13.00 – 14.00

Café Crossfire debate

14.00

End of debate

Moderators
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.
Speakers
Yann Le Cam
Chief Executive Officer of 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.
Nathalie Moll
Director-General of the European Federation of Pharmaceutical Industries and Associations (EFPIA)
Nathalie Moll leads the federation that represents the pharmaceutical industry operating in Europe. She has spent 20 years working for the biotech industry at the EU and national level in associations and corporate positions. Moll previously worked as Secretary-General of EuropaBio, which was ranked as the most effective European trade association in Brussels in 2013. In this post, she promoted a dynamic and innovative environment for the biotech industry in Europe. Moll was named one of the 15 leading women in biotech in Europe in 2017.
Xavier Prats Monné
European Commission Director-General for Health and Food Security
Xavier is responsible for EU policies and programmes in the field of health and food safety. With a goal of providing all EU citizens with equal access to quality healthcare while responding to their specific needs, Xavier has supported the adoption of efficiency-boosting measures such as establishing 24 European Reference Networks (ERNs) for rare diseases. Before his current assignment, Xavier spent more than a decade in employment and education policy circles and served as European Commission Director-General for Education and Culture.
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.
Maurizio Scarpa
Director of the Horst Schmidt Klinik Institute for Rare Diseases and Coordinator of the European Reference Network for Hereditary Metabolic Diseases (MetabERN)
Maurizio Scarpa has extensive expertise in the treatment of rare paediatric disorders. As coordinator of MetabERN, he oversees a comprehensive network that connects existing highly specialised healthcare providers across the European Union to improve the care and treatment of all patients affected by rare metabolic disorders. Scarpa is also the president of the Brains for Brains (B4B) Foundation, which seeks to promote an international network of health professionals in rare disorders, specifically in the field of rare neurological, paediatric diseases.
Kim Stratton
Head of International Commercial at Shire
Kim Stratton is a member of the Executive Committee of Shire, a leading global biotechnology company focused on serving people with rare diseases. In her position as Head of International Commercial, she optimises the company’s international performance and leads all new market entries. Stratton joined Shire from Novartis where she served as Global Head of Group Country Management and External Affairs. Prior to that, she held many roles of increasing responsibility in leading pharmaceutical companies. Stratton qualified as a State Registered Nurse at the Royal North Shore Hospital in her native Australia.
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