The French Institute for Research on Rare Diseases is a consortium administratively bound to Inserm, which brings together public bodies, research institutions and charities involved in rare diseases research or committed to the welfare of patients suffering from rare diseases. Its major roles are to help define research policy on rare diseases, stimulate the involvement of scientists in this research area, facilitate multidisciplinary approaches and optimize work resources, in particular, by federating assets on tools of collective interest for research.

The strategic activities carried out by the Institute, which are in keeping with the French National Roadmap for Rare Diseases, include the definition and funding of transversal actions and the management of annual calls for proposals. These activities are financially supported mostly by the French Ministry of Research through the French National Research Agency (ANR) and the French Muscular Dystrophy Association (AFM). On average, the French Institute for research on Rare Diseases has been committing € 5 million per year to research on rare diseases.