Eurordis - Rare Disease Europe

We welcome the ENVI Committee’s approval of compromises reached across political lines, which enhance the original proposals by the European Commission. From the start of these legislative reforms, our community has tirelessly called for the proposals to evolve the current framework, given that 94% of rare diseases still lack specific treatments. We believe that the proposals passed today mark an important moment in European policymaking on rare diseases, with a meaningful step taken toward bridging the gap between rapid scientific progress and the slower advancement in patient care.

We welcome the compromise amendments that ensure patient organisations’ voices are heard in regulatory processes and create a robust toolkit to enhance research and improve access to medicinal products across the EU. We are particularly encouraged by amendments requiring mandatory patient consultation, establishing progressive market exclusivity for research-intensive entities, facilitating central procurement for swifter access to orphan medicinal products, and extending the PRIME scheme.

Finally, our community will celebrate the amendment calling for the delivery of an EU policy framework for rare diseases. Our community, many Members of the European Parliament, over 20 Member State governments, and the European Economic and Social Committee have all endorsed the call for such a policy framework, in the form of a European Action Plan for Rare Diseases. Recently, the European Commissioner herself affirmed that the Commission is well-equipped to implement a detailed and resourced strategy on rare diseases, mirroring the comprehensive approach adopted in the cancer strategy launched in 2021.

As we approach the Strasbourg plenary session votes on the pharmaceutical reforms next month, it is imperative that all political groups uphold the compromises already reached. At the same time, we call upon Member States to take note of the progresses made in the European Parliament and build upon them as they continue their reading of the proposed reform.