Europe stands at a crossroads for its health and innovation agenda. As global competition for life sciences investment intensifies, the EU needs to both deliver scientific progress and ensure that innovation translates into tangible benefits for patients and society. Rare diseases bring this challenge into sharp focus, offering an opportunity for Europe to lead.
The Rare Diseases Forum 2026 comes at a crucial time for EU health policy: register and join the conversation
The forefront of innovation
Rare diseases are not a niche issue. Rather, rare disease research sits at the forefront of scientific and medical innovation. It often generates insights that extend far beyond rare diseases, informing our understanding of more common conditions and driving significant scientific advances in fields such as biotechnologies and precision medicines.
However, in a field where patient populations are small, diseases are complex and unmet need is high, policy frameworks are crucial to ensuring this innovation lives up to its potential.
Rare disease innovation as a strategic priority
Given its broad-reaching potential impact, rare disease innovation should be understood as a strategic priority that delivers measurable returns in patient outcomes, economic resilience and Europe’s competitiveness.
Rare diseases are not a niche issue. Rather, rare disease research sits at the forefront of scientific and medical innovation
Developing therapies for small and often poorly understood patient populations requires sustained investment as it involves significant uncertainty and risk for companies. This can only be achieved with relevant supportive policies. The EU has taken important steps to strengthen its policy ecosystem, including through the European Life Sciences Strategy and the proposed Biotech Act 1. Focused initiatives such as the European Reference Networks (ERNs) have also driven progress in recognising and rewarding innovation in the rare disease space.
However, more must be done.
The need to act now
The EU already faces growing global competition for pharmaceutical R&D and biotech investment. Without a strong, predictable and competitive framework for rare diseases, Europe risks further relocation of research, fewer clinical trials, slower development of new therapies and delayed access for patients – and thus falling behind in the global innovation race.
An EU Rare Disease Action Plan would send a clear signal that Europe is serious about supporting innovation, driving competitiveness, and reaffirming its position as a leader in life sciences
At the same time, global momentum in this field is accelerating. The 2025 World Health Assembly resolution on rare diseases, which provides for the development of a ten-year Global Action Plan, sets a clear direction for action. Europe now has an opportunity – and a responsibility – to translate this global ambition into concrete, and coordinated, regional action.
An EU Rare Disease Action Plan
An EU Rare Disease Action Plan would provide a coherent framework to coordinate efforts and accelerate progress. This includes supporting earlier and more accurate diagnosis, improving awareness and expertise, enabling more coordinated care, and facilitating better access to innovative treatments.
The EU does address rare diseases within wider legislation that governs research, cross-border healthcare, and the regulation of medicines. However, this has brought about a patchwork of policies creating uncertainty and inconsistencies. An EU Rare Disease Action Plan would show that the EU acknowledges the needs of rare disease patients.
It would offer a clear and coordinated approach that could guide policy development and implementation, ensuring consistency across healthcare, research, and industrial policy. Backed by a broad coalition of stakeholders, an Action Plan would send a clear signal that Europe is serious about supporting innovation, driving competitiveness, and reaffirming its position as a leader in life sciences.
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