The network's stakeholders have chosen 3 priority areas
The operationalization of clinical research
Research in rare and ultra-rare diseases faces a major methodological and operational issue related to small numbers, dissemination and heterogeneity of patients. Clinical trials are therefore international in scope. The challenge is to facilitate the framework for evaluating and carrying out therapeutic trials to increase the inclusion of French patients in trials and allow them to benefit from the latest innovations.
The development of AI
The development of artificial intelligence, the collection and use of health data, opens up promising horizons for rare diseases in terms of major innovations around diagnosis and the identification of new treatments.
Accessibility of treatments
The aim is to develop real and quick accessibility to treatments – orphan drugs – in order to allow patients suffering from rare diseases in France to benefit from the same innovations as their European neighbours.
