Rosa Castro
Public Affairs Director, EURORDIS-Rare Diseases Europe
Rare diseases offer Europe a strategic edge — fuelling biotech, genomics and AI innovation. Learn how a new WHO resolution could shape future healthcare systems and competitiveness.
Across Europe, rare diseases are being recognised as more than a quest for social justice. They are emerging as a strategic opportunity — a lever for scientific excellence, smart regulation and patient-centred innovation. That shift is real, and it is encouraging — but here’s what should excite us even more: Europe has only begun to tap this potential.
Rare diseases as a strategic priority
Rare diseases sit at the crossroads of the challenges that national and regional competitiveness strategies aim to solve — and the strengths they seek to build. From the EU’s Life Sciences Strategy and Biotech Act to the UK’s Rare Diseases Action Plan, recent initiatives have increasingly recognised rare diseases as a sector where meaningful progress meets scientific opportunity.
An overwhelming 95%
of rare diseases still lack
an approved treatment.
System gaps in rare disease
The case is clear. An overwhelming 95% of rare diseases still lack an approved treatment, yet more than 70% have a genetic origin.1 This places rare diseases at the forefront of breakthroughs in genomics, newborn screening and advanced therapies. Rare diseases are also a testing ground for AI-enabled tools and real-world data to accelerate diagnosis, deepen research and inform smarter regulation.
However, leadership in rare diseases demands more than technological promise. It requires systems that connect innovation to access alongside policies that reward complexity without leaving patients behind. Rare diseases reveal where those systems falter — and how to make them stronger.
Europe poised for action
The recent adoption of the first-ever WHO Resolution on Rare Diseases opens a new window for pan-European action. At the 13th European Conference on Rare Diseases and Orphan Products (ECRD), from 2–4 June 2026 in Prague and online, EURORDIS will launch a multi-stakeholder dialogue on a roadmap for implementing the Resolution across the WHO European Region. Europe has the tools. It has the ambition. Now is the time to deliver.
[1] The landscape for rare diseases in 2024. The Lancet Global Health, Volume 12, Issue 3, e341.
