The Declaration on the European Innovation and Care Ecosystem for Rare and Complex Diseases has been released following the High-Level Meeting on a European research and innovation ecosystem for rare diseases. This meeting took place on 9-11 December 2025 in Brussels bringing together EU policymakers, industry representatives, patient organisations, researchers, and healthcare professionals to foster multi-stakeholder dialogue at a critical time for rare disease policy.

The document provides a strategic Roadmap for Priority Action aimed to address the critical challenges that the European Union faces in the area of rare disease care and research, including declining competitiveness, unequal patient access to innovative treatments, significant barriers related to orphan medicines, structural and financial constraints of European Reference Networks (ERNs), and persistent diagnostic delays.

There are eight priority actions identified that require a more coherent political framework supported by dedicated, earmarked resources:

• Establish a high-level Consultative Group to assess the coherence and impact of the rare disease regulatory framework and monitor outcomes through a dedicated dashboard;
• Strengthen ERN collaboration and research capacity by formalising the ERN Academy, thereby expanding EU-level training opportunities and knowledge exchange in the field of rare diseases.
• Accelerate equitable access to diagnosis by expanding capacity, including newborn screening and the use of data and AI to shorten diagnostic timelines.
• Foster EU leadership in rare diseases clinical trials through structured collaboration among the Academy, ERNs, patient groups and industry.
• Establish at least one Comprehensive Rare Disease Infrastructure Cluster (CoRDIC) per Member State for research, innovation and care.
• Boost the generation of real-world evidence by ensuring FAIR-compliant data collection and governance within the European Health Data Space.
• Develop new financially sustainable early access for innovative orphan therapies and diagnostics, supported by a European Guarantee Fund.
• Secure dedicated funding for ERNs under the 2028–2034 Multiannual Financial Framework.

What emerges is a call for an immediate and decisive review of the EU’s approach to revitalise   innovation and care ecosystem for rare diseases.

Fondazione Benzi fully embraces this vision, as it shares the challenges of research in rare diseases through its experience and active involvement in major European initiatives, such as ERDERA, INVENTS and ERAMET.

For this reason, a clear and coordinated call for action, as mandated by the High-Level Meeting Rare 2025, is not only timely, but necessary.