Booster toward new scientific breaktroughs in rare and pediatric diseases

ABOUT THE MOONSHOT

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

Public-private partnerships could help pool resources to solve problems more quickly, reduce fragmentation and scale up existing initiatives to make a real difference for patients. That’s why a coalition of partners is joining forces to accelerate research in rare and paediatric diseases and bring new therapies to patients.

Latest news

28 Feb 2023

How the Rare Disease Moonshot can unlock science for patients

A guest blog by Magda Chlebus, Executive Director Science Policy and Regulatory at EFPIA Sometimes the most exciting areas to work in are those facing complex challenges, where the scope for making...
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12 Dec 2022

TOWARDS A RARE DISEASE MOONSHOT: SCALING UP PUBLIC PRIVATE PARTNERSHIPS TO ACCELERATE RESEARCH

If you missed the launch of the Rare Disease Moonshot, you can watch it here...
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08 Dec 2022

‘Rare Disease Moonshot’ – Scaling-up public-private partnerships to accelerate research into world’s rarest diseases

A new initiative to boost research and development into rare and paediatric diseases will launch today at the European Health Summit. The ‘Rare Disease Moonshot’ is a commitment and collabora...
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