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.

Initiatives

Discover initiatives bringing together public and private stakeholders to advance research, accelerate innovation and improve outcomes for people living with rare diseases. Use the search and filters below to explore initiatives by category and their relevance to rare diseases.

Latest news

26 Oct 2026

The Moonshot approach is advancing rare disease R&D

The Moonshot approach is advancing rare disease R&D The Rare Disease Moonshot, launched in 2022, set an ambitious objective to accelerate progress for people living with rare diseases by short...
Read more
27 May 2026

Rewiring research funding to deliver impact in Rare Disease White Spots

The Rare Disease Moonshot contribution to the EURORDIS’ Blueprint for an EU Action Plan on Rare Diseases In European rare disease research, the main challenge often lies not in discovery itself,...
Read more
27 Feb 2026

Impact of Innovative Medicines/Health Initatives projects on rare diseases

Rare diseases face fundamental research and implementation challenges driven by small, scattered patient populations, incomplete natural history data, limited trial methodologies adapted to ultra-rare...
Read more