There is a quiet contradiction at the heart of Europe’s approach to rare diseases.
Over the past two decades, the EU has built something substantial. It has funded world-leading research, established cross-border networks that bring together specialised expertise, and created regulatory incentives that turn scientific possibility into real treatments. In doing so, Europe has recognised rare diseases as a shared public responsibility.
The Rare Diseases Forum 2026 comes at a crucial time for EU health policy: register and join the conversation
But what has emerged is an ecosystem rather than a strategy.
The European Reference Networks connect Centres of Expertise, yet their integration into national systems remains uneven. Research funding delivers breakthroughs, but often without long-term continuity. Data initiatives are advancing, but remain fragmented. For the 30 million people in Europe living with a rare disease, access to diagnosis, treatment and care still depends too heavily on where they live.
This is not a failure of ambition, but of alignment.
The EU-supported Rare 2030 foresight study already called for a coordinated framework – a long-standing call widely supported by the rare disease community, researchers, clinicians and policymakers, now echoed in work within the European Parliament led by rapporteur Nicolás González Casares.
An EU Action Plan on Rare Diseases would set priorities across currently siloed areas and require them to move in the same direction
An EU Action Plan on Rare Diseases would set priorities across currently siloed areas and require them to move in the same direction. Research would align more closely with unmet needs. Healthcare systems would connect more effectively with European expertise. Data would be optimised, not just collected. Patient involvement would become standard.
The value of EU action is clear. Even if rare diseases are collectively very common, their individual rarity often makes them too difficult for any one country to address alone. Expertise is scarce. Data is dispersed. Investment carries higher risk. An Action Plan is therefore essential.
What an Action Plan offers is leverage. It allows Europe to make more of what it has already built. Recent findings by the European Parliamentary Research Service point to “the benefits of a more strategically aligned EU approach to consolidating existing initiatives, reducing fragmentation, strengthening cross-border coordination and enhancing equity, efficiency and sustainability in rare disease policy”.
Today, progress depends on navigating a landscape of initiatives and policies that were not designed together. This creates friction at every step. A shared roadmap would make this landscape navigable, with clear timelines and measurable indicators to track progress.
The question is no longer whether Europe can lead on rare diseases, but whether it can organise its existing leadership into something coherent
And it would send a signal. That rare diseases are a priority. That equity across member states matters. That Europe is willing to organise itself around complex, long-term challenges.
The question is no longer whether Europe can lead on rare diseases, but whether it can organise its existing leadership into something coherent.
The European Conference on Rare Diseases and Orphan Products (ECRD) 2026, taking place on 3-4 June in Prague, will be a moment to answer that question. It will bring together the people and institutions already doing the work, alongside the presentation of EURORDIS’ European Blueprint for Rare Diseases – a collective effort by voices from across the community to define a shared roadmap and set out recommendations for a future EU Action Plan. What is needed now is the political will to connect it.
We have the pieces. We just need the plan.
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