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
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
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.
Clara Casert, Programme Assistant
Tel.: +32 2 893 98 25