EU rare disease action plan: Main findings from the European Added Value Assessment
New European Parliament study maps options for an EU rare disease action plan.
A new European Parliament Research Service study, "EU Rare Disease Action Plan - European Added Value Assessment", highlights the potential of stronger EU-level action to improve the lives of people living with rare diseases. Affecting an estimated 36 million people in the European Union, rare diseases still face challenges such as delayed diagnosis, unequal access to care, and fragmented research. The study outlines 31 measures that could form the basis of a future EU action plan, covering areas such as diagnosis, access to therapies, research, data sharing, and patient support.
It highlights the central role of European Reference Networks (ERNs) as unique cross-border platforms that connect expertise, support clinical decision-making, and improve patient outcomes. Initiatives such as the European Rare Disease Research Alliance (ERDERA) are also recognised for their contribution to integrating research, data, and patient involvement at EU level.
Building on these existing strengths, the study calls for a more integrated approach that links research, clinical practice, and data ecosystems, ensuring that innovation can more effectively translate into real-world patient care.
Read the full study and executive summary HERE!