DRAFT REPORT with recommendations to the Commission on a European Union rare disease action plan (2025/2130(INL)) Committee on Public Health Rapporteur: Nicolás González Casares

https://www.europarl.europa.eu/doceo/document/SANT-PR-785192_EN.html On 2 March, MEP Nicolás González Casares presented a draft own-initiative legislative report to the European Parliament’s Public Health (SANT) Committee, calling on the European Commission to propose legislation establishing a European framework for rare diseases. The report highlights that, among the 6,000 to 8,000 identified rare diseases affecting an estimated 27 to 36 million people in the EU, around 95% still lack an authorised treatment. The report calls for a comprehensive approach addressing the full rare disease pathway, including improved coordination of research, faster and more equitable diagnosis, better access to therapies, strengthened support for patients and families, and enhanced data sharing, notably through the European Health Data Space. It also emphasises the need to reduce inequalities across Member States and reinforce the role of the European Reference Networks. EURORDIS welcomed the report as an important step forward in the long-standing effort to establish an EU Action Plan for Rare Diseases. “We are very happy with this draft report, which reflects a growing recognition in Europe that rare diseases cannot be addressed through fragmented national policies alone. The scale of the challenge demands a coordinated European approach,” said Valentina Bottarelli, Head of Policy & Public Affairs at EURORDIS. MEPs on the SANT Committee had until 26 March to submit amendments to the draft report. A vote in Committee is expected in June, followed by a plenary vote in the European Parliament, likely in September.