Towards an EU Action Plan on Rare Diseases

In early 2026, two important documents were published that could shape the future of rare disease care across Europe. The first is the Casares report, adopted by the European Parliament’s Health Committee, which sets out a political vision for a European Action Plan on Rare Diseases. The second is a study by the European Parliamentary Research Service (EPRS), which examines the evidence for where European cooperation can make the biggest difference. Together, they represent a significant step forward for the rare disease community — including people living with cystic fibrosis and their families.

Two documents, one direction

The Casares report and the EPRS study approach the same challenge from different angles, but point in the same direction. The Casares report focuses on fairness, access to care, and reducing differences between countries. It calls for clear goals, timelines, and dedicated funding for a European Action Plan. The EPRS study looks at the evidence: where does working together at EU level actually lead to better results than countries acting alone?

In short, one document explains what needs to be done; the other shows why and how it can work. Used together, they make a compelling case that a European Rare Disease Action Plan is both necessary and realistic.

The core problem: where you live should not decide the care you receive

Both documents highlight the same fundamental injustice: people living with rare diseases in Europe do not have equal opportunities depending on where they live. This can mean waiting years for a diagnosis, having little or no access to treatments available in neighbouring countries, being unable to participate in research, receiving little support as a family, and facing serious financial hardship as a result.

These are not abstract policy concerns — they are the daily reality for hundreds of thousands of patients and families across Europe.

What needs to change

Both documents identify the same priorities for action: earlier and more accurate diagnosis, including newborn screening; fair access to treatments across all Member States; sustained investment in research and innovation; better data sharing and use of patient registries; stronger support for patients and families; and improved coordination between countries.

What this means for CF organisations

This is a moment for national CF organisations to act. These documents provide strong European backing for advocacy at the national level. Organisations can use them to engage policymakers on shared European priorities, make the case for fair and timely access to treatments, promote patient participation in research and European Reference Networks, and push for better social and financial support for patients and families.

EURORDIS, the European rare disease patient organisation, broadly supports the Casares report as a strong foundation for an EU Action Plan. It welcomes its ambition while emphasising the importance of a practical, patient-centred approach — with flexible implementation, stronger use of data, sustainable funding, and meaningful patient involvement at every stage.

Conclusion

The publication of these two documents creates a real and timely opportunity. Together, they combine political direction with evidence-based analysis to build the case for more coordinated, consistent action across Europe. For patients and families living with rare diseases, that means a genuine chance of more equitable care, faster diagnosis, better access to treatments, and stronger support — wherever they live in Europe.

For the full text of the Casares report and the EPRS study, see the links in the references below.

[1] https://www.europarl.europa.eu/doceo/document/SANT-PR-785192_EN.html

[2] https://www.europarl.europa.eu/RegData/etudes/STUD/2026/774708/EPRS_STU(2026)774708_EN.pdf