Rare Disease Moonshot

Initiative leader: The Rare Disease Moonshot coalition

Duration: Ongoing

Of the 6,000-7,000 identified rare diseases, 95% don’t have an approved therapy, and for most of these diseases research is effectively non-existent. The search for new treatments is thwarted by enormous scientific challenges, and at the current pace, it would take over 100 years to develop treatments for all rare conditions.

The coalition will work together to pool expertise, reduce fragmentation in research and foster greater collaboration between organisations. It will bring together an ecosystem of rare disease experts and research to explore opportunities for collaboration and support a range of public-private partnerships to:

• Enhance translational research ecosystem to fill the research pipelines with new therapeutic options
• Optimise clinical trials and regulatory pathways for very small patient populations to de-risk and optimise development
• Develop infrastructure to accelerate the journey to diagnosis and treatment

The Rare Disease Moonshot is also a response to the European Commission's call to address unmet medical needs — and the belief that the science and translational capability gap in rare diseases cannot be addressed by one party or even one sector alone, nor can it be optimally served by a “patchwork” of unrelated initiatives. In order to improve the rare disease ecosystem in Europe, policy solutions need to be devised all along the lifecycle of medicines, from screening to patient access. This complex challenge calls for a vision which captures the sum of public and private knowledge and capabilities to accelerate innovation - not only in joint undertakings, but also through vital funding initiatives at European and National levels, and in coordination with international initiatives.

ECRIN's Role in The Rare Disease Moonshot

ECRIN is one of the 7 partners in the Rare Disease Moonshot initiative.