- scientific and medico-economic evaluation;
- laboratory control and advertising control;
- inspection of industrial sites.
The Agency also coordinates vigilance activities relating to all products for which it is relevant. It also takes policing decisions in the name of the State with respect to two principles: contradiction and transparency. Decisions are discussed and motivated through the evaluation by a network of internal and external experts. The ANSM operates as the unique portal for applications. In order to conduct a clinical gene therapy trial in France several bodies have to be consulted. Final authorization is granted by the head of the ANSM. A specific streamlined procedure has been set up.
Gene therapy trial sponsors have to submit a single report to the ANSM according to a specific application format including all information required by the review body, the High Council for Biotechnology (HCB). The evaluation of the risk/benefit ratio of the proposed trial, the review of manufacturing processes, quality of the end product including viral validation and inspections are under the control of the ANSM. Finally, the initiation of a clinical trial requires approval by a local bioethics committee, called Comité de Protection des Personnes (CPP).
When taking a final decision, ANSM takes into account its own evaluation, the advice of the HCB and the opinion of the CPP. An approval can only be provided with the agreement of the Minister of Research (MESR), covering the contained use aspect (production/administration to the patients).
See also: Gene therapy in Germany and in Europe: regulatory issues. Qual. Assur. J. 1997; 2: 141-152. giving an outline on the regulatory issues of gene therapy in France.
(Last reviewed by the Competent Authority: 29 January 2013)