Speakers

Conference title: Health data applied to rare diseases: review of the national plan for rare diseases 3 and prospects in 2024 for the national plan for rare diseases 4
Since 2004, with the launch of three successive national plans, France has put in place a unique system to support sufferers and their families. This has been made possible by the high level of medical and research expertise in France, but also by the strong mobilization of families, associations and professionals. This co-construction and co-working approach has enabled us to pursue and amplify the contribution of patient associations and their families to the definition and implementation of policy in favor of rare diseases. The 7th European Rett Syndrome Congress is a step in this direction.

Objectives
Because data is scarce, clinical observation of rare diseases must be used for research.
The patient's own experience and experience helps to improve our knowledge of the natural history of the disease.
Certain questions or criteria that are important for research can thus be enriched by the data provided by the clinic.
In this way, the clinic becomes an important source of knowledge, helping to build more appropriate care pathways.
We regularly work closely with the Banque nationale de données maladies rares (BNDMR) to share data between care and research, in conjunction with the national health data system.