Rare Diseases Forum 2026

- There is a clear consensus from across the rare disease community as well as the Council and European Parliament, for the urgency of an EU Action Plan on Rare Diseases. As the 10-year Global Action Plan is developed in 2026, reviewed in 2027 and implemented from 2028, when and how will the European Commission propose and implement a corresponding EU Action Plan aligned with it?

- How can an EU Action Plan effectively address the diagnostic and treatment odyssey faced by people living with rare diseases, while providing sustained support to patients, their families, and carers?

- How will the EU Health Technology Assessment Regulation, Review of the EU Pharmaceutical Package, and Critical Medicines Act affect patient access to orphan medicines? 

- What strategies can Member States adopt to reconcile affordability with innovation, including pricing and reimbursement models that ensure system sustainability? 

- Should the European Commission consider establishing a Priority Review Voucher system similar to the US, and what other regulatory incentives could encourage orphan drug development? 

- How can the EU Life Sciences Strategy and Biotech Act stimulate biotech and pharmaceutical companies to invest in rare disease therapies while maintaining timely patient access? 

- How can the European Commission and Member States leverage ERDERA, JARDIN, and the European Health Data Space to accelerate research and improve diagnosis? 

- What breakthroughs would AI bring forward for patients?  

- What frameworks should patient organisations, regulators, and healthcare providers follow to deploy AI, genomics, and digital health tools ethically and equitably? 

- What public-private partnership models, including initiatives like “Rare Diseases Moonshot” or “Together for Rare,” can the EU and Member States support to finance R&D sustainably? 

- How can the European Commission and EMA promote international collaboration, including EMA–FDA regulatory convergence and global trial networks? 

- What actions can Member States take to train and retain a healthcare workforce specialised in rare diseases? 

- How can the European Commission, Member States, and patient organisations create sustainable frameworks for cross-border care? 

- What policies can EU institutions and national governments implement to integrate social protection, education, and workplace rights into rare disease strategies?