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.
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?
Friends of Europe