Across Europe, more than 30 million people, or around one in 16, live with a rare disease1. Many rare diseases are chronic, lifelong and disabling, and roughly 90% lack approved therapies – even amid scientific breakthroughs2. While rare diseases affect more Europeans than diabetes and cancer combined, patients wait an average of five years for a diagnosis, and complex cases may take a decade or more of referrals before answers arrive3. For patients and families on this journey, time is not just precious; it is everything. With the new Multiannual Financial Framework pending, and an EU Action Plan on Rare Disease being discussed in the European Parliament, Europe has a window of opportunity to change lives and act now.
The Rare Diseases Forum 2026 comes at a crucial time for EU health policy: register and join the conversation
In order to deliver for rare diseases, coordination across three priorities is required. First, we must accelerate diagnosis through harmonised newborn screening and better medical education. Every child should have equal access to early detection regardless of where they are born. Second, sustain innovation by maintaining predictable incentive frameworks and deepening collaboration between academia, industry, and patient communities.
With only about a 6.2 per cent probability of success from laboratory to patient4, rare disease R&D needs long-term commitment and coherent policies. Third, ensure equitable access by sharing best practices among member states and enabling innovative payment models tailored to the unique nature of rare diseases. National action matters; European coordination, however, can multiply impact and reduce inequality.
With the World Health Organization expected to present a Global Rare Disease Action Plan by 2028, now is the time for Europe to shape the future
A European Rare Diseases Action Plan would provide an end-to-end framework, from discovery to faster diagnosis to equitable access. The action plan should strengthen funding for European Reference Networks (ERNs), promote public-private partnerships that accelerate discovery, and facilitate cross-border healthcare for specialised treatments.
Europe has a legacy to build on. The Orphan Medicinal Products Regulation has enabled over 260 treatments to reach patients5; ERNs already connect expertise across borders, so no patient faces their condition alone. With the World Health Organization expected to present a Global Rare Disease Action Plan by 20286, now is the time for Europe to shape the future, aligning regulations, funding and research to deliver results that matter to people’s lives.
The question is not whether Europe can afford to act, but whether we can afford the status quo that impacts more than 30 million people
The question is not whether Europe can afford to act, but whether we can afford the status quo that impacts more than 30 million people waiting for years for answers and treatments. Echoing the European Economic and Social Committee7, we look to the European Commission to table a funded Rare Disease Action Plan: one that strengthens ERNs, supports newborn screening, sustains incentives for innovation, and enables equitable access across member states. We also call for policymakers to work with a multi-stakeholder forum bringing together patient communities, industry, healthcare professionals, payers and member states to shape and implement this initiative together.
Europe can set the global standard for rare diseases. If we match political will with scientific progress, we can shorten the diagnostic journey, unlock much-needed therapies, and ensure access for all who need it – turning rare diseases into a policy priority, and not a postcode lottery. Together, we can turn momentum into measurable impact with purpose.
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