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? 

- What can Europe learn from other established incentives like the US Priority Review Voucher and the Kingdom of Saudi Arabia Breakthrough Designation to encourage orphan drug development?

- How can current EU pharmaceutical files like the EU Life Sciences Strategy, the Biotech Act and the Critical Medicines Act stimulate biotech and pharmaceutical companies to invest in rare disease therapies and achieve timely patient access

- How can the EU support early-diagnosis frameworks into its rare disease agenda to reduce long-term clinical and economic burden in Member States?

- What role can JCA play in addressing the challenges to equitable rare disease access to innovation in EU member states?

- What are the learnings from JCA so far, and what's needed to make it fit for rare diseases?

- Are countries ready to implement JCAs, and is the rare disease ecosystem ready to adapt to the new requirements?

- 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? 

- Which collaborative models and public–private partnerships have proven most effective in advancing rare disease R&D, and how can they be scaled globally? In particular, how can initiatives such as “Rare Diseases Moonshot” or “Together for Rare” be supported by the EU and Member States to ensure sustainable R&D financing?

- How do global initiatives such as IRDiRC, ERDERA, and IHI illustrate effective models of international collaboration, and what lessons can be drawn to strengthen and expand these approaches?