Booster toward new scientific breaktroughs in rare and pediatric diseases


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

11 Jul 2023

Clinical trials research needs recommendations

The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on.    The RD Moonshot partn...
Read more
14 Jun 2023

Rare Diseases Moonshot partners welcome the pre-publication of the draft future Innovative Health Initiative (IHI) call topic: ‘Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases’

The Rare Disease (RD) Moonshot partners welcome the inclusion of the rare and ultra-rare diseases in a recent IHI call topic which aims to establish novel approaches to improve clinical trials to ulti...
Read more
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...
Read more