Momentum grows for an EU Action Plan on Rare Diseases
On 15 October 2026, EURORDIS launched the European Blueprint for Rare Diseases, a landmark document outlining a set of proposals for the structure, priorities and implementation of a future EU Action Plan on Rare Diseases.
Co-created by patients, clinicians, researchers, public authorities and industry representatives from across Europe over the course of ten months, the Blueprint calls on the European Commission to introduce a European Action Plan on Rare Diseases. Such a plan would build on existing EU and national-level initiatives to create a political framework specific to rare diseases, improving coordination and addressing persistent gaps in access to diagnosis, treatment and specialised care.
The Blueprint is intended to serve as the rare disease community’s contribution to designing the EU Action Plan on Rare Diseases. It proposes 18 priority actions across six areas where EU coordination stands to make the greatest difference:
In addition to these actions, the Blueprint calls for an Action Plan which includes:
- A shared vision, common goals and SMART targets linking EU and national action;
- Dedicated resources under the EU’s Multiannual Financial Framework (MFF) 2028-2034;
- Patient partnership embedded in governance through a multi-stakeholder Advisory Board;
- A common monitoring framework with public reporting and independent evaluation.
The Blueprint’s publication comes at a time of growing political momentum for a more coordinated approach to rare diseases for the EU. On 28 September, the European Parliament Committee on Public Health (SANT) adopted a legislative initiative resolution calling for a dedicated EU law addressing rare diseases. This resolution, initially presented by MEP Nicolás González Casares last March, calls on the European Commission to develop a comprehensive European rare disease action framework. Following its adoption by SANT, the resolution will be tabled for a vote at an upcoming plenary session of the European Parliament. Once adopted by a majority of all MEPs, the European Commission will have three months to respond to the proposal.
For decades, the rare disease community and initiatives such as the European Reference Networks and the orphan medicines framework have demonstrated just how much can be accomplished when Europe works together. Now, there is a consensus in the community that it is time to move beyond the existing initiatives and national plans that were promoted by the 2009 Council recommendation by introducing a common European framework. The approximately 30 million people living with a rare disease in Europe are facing delayed access to diagnosis and inequal access to treatment, based largely on where they live. Both the Blueprint and the legislative resolution currently being examined in Parliament aim to address these challenges through a comprehensive, ambitious European Action Plan on Rare Diseases which will lead to tangible improvements in people’s lives and greater social justice for everyone living with a rare disease.
Moving forward, the Blueprint will be presented at the upcoming meeting, "Research as care: Why rare diseases matter for Europe's future" on 13 November 2026 in Dublin, Ireland. This event, organised by Rare Diseases Ireland and EURORDIS in association with the Irish Presidency of the Council of the EU, will explore how research, innovation, patient partnership and EU policy can improve rare disease care and strengthen Europe's life sciences ecosystem.
