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A new moonshot for rare diseases

Words by GOLD newsdesk

Rare diseases are gaining some well-deserved attention as a new initiative to boost R&D was launched at the European Health Summit on 8 December 2022.

The ‘Rare Disease Moonshot’ is a collaboration between seven pharmaceutical bodies and rare disease groups aiming to find treatments and cures for the rarest conditions in the world. The scheme has been set up in response to the European Commission’s call for unmet medical needs to be addressed on the continent. Currently, 95% of rare diseases have no approved treatment or cure, and many of these conditions affect children.

The initiative is a collaboration between the European Federation of Pharmaceutical Industries and Associations, the Critical Path Institute – known as C-Path – the European Infrastructure for Translational Medicine, the European Clinical Research Infrastructure Network, EuropaBio, EURORDIS-Rare Diseases Europe and the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE).

Commenting on the launch, Alexander Natz, Secretary-General, EUCOPE, said: “Rare diseases represent a pioneering domain for therapeutic innovation. With the moonshot, our goal is to unlock science pathways for as many underserved patients and diseases as possible.”

Current hurdles to rare disease drug discovery include poor translational research ecosystems leading to a shortage of drugs in pipelines, regulatory pathways discriminating against smaller patient populations and lacking infrastructure for diagnosing and treating these conditions.

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