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.
Networking lunch and registration of participants
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?
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)
Tamsin Rose, Senior Fellow at Friends of Europe
End of debate
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
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