In European rare disease research, the main challenge often lies not in discovery itself, but in the progression of promising scientific outputs into assets that are sufficiently robust, usable and mature to support further development, regulatory engagement and, ultimately, impacting people’ lives.

This challenge is especially acute in rare disease white spots, where scientific knowledge, infrastructure, data resources and commercial incentives are weak from the outset. The problem is therefore not only the level of funding, but how funding is structured, deployed and connected to downstream use.

How can Europe improve the impact of rare diseases research?

Read the white paper here. 

Europe stands at a crossroads. In a world shaped by geopolitical tensions, economic fragmentation and accelerating global competition, the choices made today will determine Europe’s ability to compete, innovate and lead in the decades ahead.

Join us on 23 June in Brussels to engage in a forward-looking discussion on how Europe can turn today’s challenges into opportunities – and make the right choices for its future.

Register here.