This year must be a turning point for rare disease policy in Europe. The ambition is here. The frameworks are emerging. What we need now is political will and commitment – starting with stronger national delivery and clear, binding direction at EU level.
People living with a rare disease still face avoidable delays in diagnosis, unequal access to care, and fragmented systems of support. These challenges persist despite major EU-level advances. Now is the time to close the gap between policy adoption and real-world implementation.
Held during the Danish Presidency of the EU Council, the Nordic Rare Disease Summit 2025 offers a unique opportunity to turn dialogue into action. It can align national strategies, guide the implementation of new EU frameworks, and demonstrate how the Nordic region, grounded in collaboration, digital leadership, and strong patient communities, can shape a more inclusive and resilient health system across Europe.
A European Action Plan
The conversation on rare diseases is gaining momentum. In 2024, the European Economic and Social Committee, supported by the Hungarian Presidency and patient organisations, called for a comprehensive EU Action Plan. A new intergroup in the European Parliament is also keeping rare diseases on the agenda.
Yet, Europe still lacks a binding, coherent policy that brings together Member States and EU institutions. Fragmented approaches hold back progress. National strategies matter – but they are not enough. We need a cross-border framework with shared objectives, timelines, and accountability. That means:
- Setting measurable goals for early diagnosis, data sharing, and equitable treatment access;
- Ensuring consistent implementation across countries;
- Centring the patient experience in design, delivery, and evaluation.
Making EU Frameworks Work
New EU frameworks, such as the HTA Regulation and the European Health Data Space, are now in force.
To ensure it delivers for our community, the HTA Regulation must accommodate the complexity of rare diseases – by using adapted methodologies, integrating real-world data, and valuing patient-reported outcomes. The EHDS must guarantee secure and meaningful access to health data for people living with rare diseases, while enabling responsible use of data to drive research, diagnosis, and innovation.
This article is part of The Parliament's Guide to the Danish Presidency of the Council of the EU.
The Nordics, drawing on their advanced registries, interoperable systems, and spirit of cross-border collaboration, are uniquely positioned to contribute valuable examples and practical models that could help inspire progress across Europe.
Making Systems Work with People
Empowerment must be embedded as a structural principle – not just a theme.
People living with rare diseases must be recognised as equal partners in shaping healthcare and policy, provided with the appropriate training, tools, and support to engage meaningfully, and represented in all decision-making spaces – local, national, and EU-wide. That requires legal guarantees, stable funding, and established mechanisms for patient participation in research, regulatory processes, and care design.
The Nordic Rare Diseases Roadmap reflects this approach. It was shaped through broad collaboration and calls for the integration of lived experience at every level of the system. Denmark’s renewed national plan, developed with patient input, shows how these principles can be embedded in national policy – and should serve as a model across Europe.
What We Need Next
The Nordic Rare Disease Summit is not just a platform – it’s a lever. It can:
- Align Nordic and EU efforts;
- Showcase best practices in data use, patient engagement, and access to innovation; and
- Fastrack the implementation of the Nordic Rare Diseases Roadmap
Crucially, the Summit will build on the momentum of the 2024 Roadmap’s EU launch – a strategic moment supported by the Danish Permanent Representation. Together, these initiatives can connect the dots between regional leadership and European ambition.
Now we need clear political ownership at EU and national levels, sustained investment in patient-driven solutions, innovation, and cross-border care, and robust monitoring to ensure accountability and long-term impact.
The Danish EU Presidency – and the Trio’s focus on health cooperation – offers a timely opportunity to mainstream rare diseases in the European health agenda.
Let’s seize this moment. Let’s push boundaries, align systems, and put the voices of people living with rare diseases at the centre of European policy.
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