21 March 2019 - 13:00 - 14:00
Tamsin Rose

Patients with rare diseases are not getting the drugs they need because of budget pressures, bureaucratic obstacles and problems of scale, panelists told a Friends of Europe ‘Café Crossfire’ lunch debate on Wednesday 21 June 2017. Speakers at the event called for more collaboration between doctors, industry, insurers, patients and EU governments to solve this issue.

This follow-up debate will explore the progress made since our first discussion and establish what role the EU can play to ensure equal access to care for all citizens across Europe.

Related content:


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. 


Registration & networking

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 improved the drug development pipeline, resulting in over 100 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 the available therapy, and about a quarter 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 18 months 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 drug development for rare diseases?
  • How to encourage and foster the repurposing of drugs?  

Moderated by
Tamsin Rose, Senior Fellow at Friends of Europe


End of debate

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.

Sarah Bentz, Senior Programme Manager
Tel.: +32 2 893 98 23
Email: sarah.bentz@friendsofeurope.org

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