From coordination to regulation? Analysis of three scenarios on the future of EU rare disease policy

Rare diseases affect an estimated 30 million people in Europe, with persistent inequalities in access to diagnosis, treatment and care. While EU action has expanded in the last decade, it remains largely based on coordination mechanisms, with uneven impact across the EU. This presentation builds on a European Added Value Assessment for the European Parliament, combining literature review, legal analysis, stakeholder interviews, case studies and assessment of 31 potential EU measures. Drawing on this research, the analysis develops three forward-looking scenarios reflecting distinct legal and policy approaches. The first scenario assumes no major new initiative, relying on Directive 2011/24/EU and existing coordination mechanisms. While this may consolidate ongoing activities, it is unlikely to reduce fragmentation or inequalities in access. The second one builds on strengthened coordination through an EU action plan implemented via a non-legislative instrument, similar to Europe’s Beating Cancer Plan. This could provide a more structured framework, but its impact would depend on voluntary uptake and national implementation. The third scenario presents a more interventionist approach, as proposed in the European Parliament’s own-initiative legislative report (2025/2130(INL)). It points towards the adoption of legislation to underpin the development of an EU action plan, representing an ambitious expansion of action in health with implications for feasibility, Member State competence, and balance between coordination and regulation. Rare disease policy illustrates the limits of coordination-based instruments. Growing pressure for more structured EU action, combined with evidence of its added value and political support, suggests an action plan is likely to emerge. However, it remains uncertain whether it will take a more ambitious regulatory form, which could set a precedent for broader EU intervention in health and raise questions about the scope of EU competences.

Key messages

• Coordination alone is unlikely to reduce inequalities in rare disease care.

• stronger EU rare disease action could improve access, but its legal form will shape feasibility, impact and precedent.