Rare Disease Moonshot welcomes the EU Competitiveness Compass

The European Commission’s Competitiveness Compass recognizes biotechnology, life sciences, and innovation as key drivers of economic growth and competitiveness. This communication builds on previous EU policy initiatives, including the European Green Deal and the Digital Strategy, which emphasized the need for sustainable and technology-driven economic resilience. The Competitiveness Compass follows the 2024 recommendations of the Draghi Report, which identified Europe's lag in productivity and innovation as critical challenges and present legislation and communication initiatives to boost European competitiveness over the next five years vis-à-vis other parts of the world. Additionally, the 2024 European Industrial Strategy outlined the urgency of investing in key technologies, including biomanufacturing and advanced medical research. These milestones have shaped the Competitiveness Compass, reinforcing the EU's commitment to reducing dependencies on external markets and fostering research excellence. By integrating these lessons, the Competitiveness Compass now provides a structured roadmap for strengthening Europe's leadership in life sciences and rare disease research. It highlights the need to strengthen public-private partnerships, reduce regulatory fragmentation, and create a supportive research environment.

These priorities align with the Rare Disease Moonshot’s mission to accelerate innovation and address gaps in rare disease research.

Since its launch in December 2022, the Rare Disease Moonshot has made significant strides. In 2024, it led to the publication of three Research Needs Recommendations on: clinical trials, diagnosis, and translational research. These inspired the RealiseD project under the Innovative Health Initiative was launched, pioneering new clinical trial models for small populations. Additionally, the ERDERA Accelerator will expand public-private collaboration capacities, strengthening translational research pathways. Through open online courses and webinar series with the European Joint Programme on Rare Diseases, we have enhanced knowledge-sharing, while a collaboration with the International Rare Diseases Research Consortium helped define industry contributions to rare disease research.

The Competitiveness Compass sets a strong foundation for fostering Europe’s leadership in life sciences. Several key initiatives further support rare disease research:

Within health biotech, the European Biotech Act will aim to streamline regulatory processes and boost investment in health technologies, accelerating the development of new therapies. Furthermore, it should support the integration of cutting-edge advancements, such as gene and cell therapies and personalized medicine, into mainstream healthcare by providing incentives for research. Overall, by fostering a more agile regulatory framework, this act should enable researchers and companies to navigate approval processes with greater efficiency, reducing the time required to bring innovative treatments to clinical use.
The Life Sciences Strategy will seek to enhance collaboration among academia, industry, and regulatory bodies by establishing standardized frameworks for data sharing, streamlining approval pathways for innovative treatments, and fostering joint research initiatives. This approach ensures that diagnostics and treatments are developed more efficiently, reducing redundancies and accelerating the transition from discovery to clinical application. By creating a cohesive network of stakeholders, the strategy promotes transparency, shared resources, and the alignment of research objectives with regulatory expectations.
The European Innovation Act will improve access to venture capital, strengthen intellectual property protections, and create an innovation-friendly regulatory framework to expedite rare disease breakthroughs.
The Multiannual Financial Framework and Framework Programme 10 present crucial funding opportunities for expanding rare disease research under a structured European funding framework that ensures stability and long-term investment.

To maximize impact on EU’s competitiveness and Life Sciences Strategy, it would be crucial to:

Integrate rare disease research into these funding and policy frameworks.
Ensure the Biotech Act and Life Sciences Strategy explicitly prioritize rare diseases.
Expand Framework Programme 10 funding to foster public-private partnerships in rare disease research and development.

By embedding rare disease research within the broader competitiveness agenda, Europe ensures that scientific advancements contribute to economic growth while addressing urgent patient needs.

Europe’s leadership in Rare Disease Research is reachable

Europe is uniquely positioned to lead the world in rare disease research and innovation—not because of future potential, but because of the existing structures, funding instruments, and partnerships that are already in motion. The challenge ahead is ensuring these tools are used to their full potential. Of course, Competitiveness is not a responsibility for the EU level alone. All stakeholders and players need to step up to meet the challenge, working together in a joint endeavour and taking commitment and cooperation to a new level. The Rare Disease Moonshot is ready to push forward. The time to act is now. Let’s fully leverage Europe’s opportunities to transform the future of rare disease research.