The ‘Rare Disease Moonshot’, a new initiative to boost research and development into rare and paediatric diseases, has launched at the European Health Summit.
The initiative is a commitment and collaboration between seven organisations to break down the barriers to finding new treatments and cures for the world’s rarest and severe conditions. It aims to speed up research into more than 7000 identified rare diseases, of which a staggering 95% have no approved treatment or cure.
The coalition will work together to pool expertise, reduce fragmentation in research and foster greater collaboration between organisations.
The seven organisations are the Critical Path Institute (C-Path), the European Infrastructure for Translational Medicine (EATRIS), the European Clinical Research Infrastructure Network (ECRIN), the European Federation of Pharmaceutical Industries and Associations (EFPIA), the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), EuropaBio, and EURORDIS-Rare Diseases Europe.
For decades, innovation has significantly advanced, with the emergence of new technologies like cell-editing and gene therapies. Between 2000 and 2021, more than 200 new orphan medicines were approved by the EMA, addressing the needs of up to 6.3 million rare disease patients. But significant unmet needs remain.
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.