Session 11

Advancing national policy solutions for equitable rare disease access and care

Organised by Alexion, AstraZeneca Rare Disease, with the participation of Brains for Brain Foundation & HLM4Rare, in collaboration with Biogen and PTC Therapeutics & EUCOPE

The 30-36 million people living with rare diseases in Europe still face stark disparities in access to diagnosis, orphan therapies and optimal care. More than a decade after the last review by the European Commission of national policies, fragmentation persists – and we lack up-to- date evidence on how the national policy landscape has evolved. This interactive session brings together renowned experts, national policymakers, healthcare leaders, and patient advocates to explore concrete national policy solutions grounded in their direct, front-line experience across European healthcare systems. The discussion will focus on adapting access pathways to the realities of rare disease clinical drug development and strengthening rare disease infrastructure – including national registries, screening, European Reference Networks, and meaningful patient involvement.

Through an interactive discussion and open debate, panellists and participants will draw on their collective experience to discuss what the national policy landscape looks like for rare disease access and care across Europe and how it can elvove to better serve patient needs. The goal: “Health for a stronger European Union” means moving from analysis to scalable policy solutions that deliver faster, fairer access – strengthening solidarity and health equity, reinforcing Europe’s competitiveness in biotechnology and ultimately improving health and societal outcomes.