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, PTC Therapeutics & EUCOPE

People living with rare diseases across Europe continue to face major inequalities in diagnosis and care. More than a decade after the European Commission’s last review of national rare disease policies, important gaps remain and evidence on what works best is still fragmented. This session will present a new multi-stakeholder Joint Impact Analysis of rare disease policy frameworks across 10 European countries, developed with the insights of patient leaders, policymakers, academics, and industry experts. The discussion will explore which national policy measures most effectively support timely and equitable access to care, from registries, diagnosis and coordinated care pathways to tailored assessment and funding approaches. Participants, through innovative interactive modalities, will help identify practical, “no-regrets” actions that can be adapted across Member States. The session aims to move from analysis to scalable policy solutions that strengthen health equity, solidarity, and Europe’s competitiveness in rare disease innovation.

Speakers to be announced