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

12 Feb 2024

Recording Now Available: EJP RD-EFPIA Joint Advanced Webinar on Real-World Data, Machine Learning, and Deep Analytics

We are delighted to announce the successful outcome of the EJP RD-EFPIA Joint Advanced Webinar on Real-World Data, Machine Learning, and Deep Analytics in rare diseases, which accumulated enthusiasti...
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17 Jan 2024

Training Webinar: Real-World data, Machine learning and Deep analytics in rare diseases: Regulatory grade data collection for marketing authorization submissions – what is buzz, what is realistic?

Marc Van Dijk (UCB) and Luis Pinheiro (EMA) will present views of industry and regulators on various facets of collecting, processing, and using data for research, regulatory and healthcare decisio...
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10 Jan 2024

Rare Disease Moonshot: Europe’s Public-Private Coalition to Erase the Rare Disease “White Spots”

The knowledge and infrastructure required to unlock rare disease “white spots,” diseases where there is currently no science or translational capability, call for a different strategic paradig...
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