Equal access to care for rare diseases

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.