Eurordis - Rare Disease Europe

Today’s plenary vote by the European Parliament on pharmaceutical reforms marks a significant stride forward for the 30 million Europeans living with rare diseases and their families. Against the backdrop of 94% of rare diseases still lacking a dedicated treatment, we welcome the genuine political will that has been demonstrated to improve treatment development and access.

We support the MEPs’ approval of crucial proposals, such as introducing progressive market exclusivity for orphan drugs, refining the ‘significant benefit’ criteria, and clearer ‘High Unmet Medical Needs’ classification. These changes, along with enhancing the PRIME scheme and ensuring patient participation in EMA consultations, represent not just progress but the result of our community’s advocacy to close the gap between fast-paced scientific discoveries and patient care.

The commitment to an EU policy framework for rare diseases, endorsed by the European Parliament position, aligns with our longstanding call for a European Action Plan for Rare Diseases. Importantly, this endorsement builds on an already strong and broad political consensus, spanning EU institutions and Member States, on the urgent need for such an Action Plan.

As we progress to the next stages of the proposed reform, Member States should acknowledge and build upon the Parliament’s recommendations. Additionally, the Council should seize the opportunity that remains to further refine the legislation by incorporating an ‘Orphan Drug Development Plan’ in the legislation, to help navigating the journey of more and better rare disease treatments to patients.