title: Overview of the Regulatory Oversight Implemented by the French Regulatory Authorities for the Clinical Investigation of Gene Therapy and Cell Therapy Products.
abstract: Advanced therapy medicinal products, a new class of products with promising therapeutic effects, have been classified as medicinal products and as such should be developed according to a well-structured development plan, to establish their quality, safety and efficacy profile and conclude, at the time of the marketing authorisation evaluation, on a positive risk/benefit balance for patients. An important part of this development plan is achieved through clinical trials, which have also to be approved according to a well-established regulatory process, prior any initiation. This chapter is dedicated to describe the regulatory pathway to be followed in France, before initiating any clinical trial with those investigational advanced therapy medicinal products. In France, to get the final authorisation to initiate a clinical trial, the legislation imposes to run in parallel two independent but complementary authorisation procedures. The first procedure is aimed at assessing the ethical aspect of the biomedical research, while the second has to review the safety and regulatory aspects. A third procedure has to be envisaged where in case the investigational product consists or contains a genetically modified organism. The French system herein described is in line with the EU regulation on clinical trial and follows the respective deadlines for granting the final approval. The complexity of the procedure is in fact more due to the complexity of the products and protocols to be assessed than to the procedure itself which is now very close to the well-known procedure applied routinely for more conventional chemical or biological candidate medicinal products.
authors.affiliation: BIOVAC Directorate, National Safety Agency for Drug and Health Products, Management of Organic Products, Saint Denis, France. sophie.lucas-samuel@ansm.sante.fr.
authors.forename: Sophie
authors.initials: S
authors.lastname: Lucas-Samuel
authors.affiliation: BIOVAC Directorate, National Safety Agency for Drug and Health Products, Management of Organic Products, Saint Denis, France. Nicolas.ferry@ansm.sante.fr.|Committee for Advanced Therapies (CAT), European Medicines Agency, London, UK. Nicolas.ferry@ansm.sante.fr.
authors.forename: Nicolas
authors.initials: N
authors.lastname: Ferry
authors.affiliation: Public Health and Health Products, School of Pharmacy, Paris-Descartes University, Paris, France. jean-hugues.trouvin@parisdescartes.fr.
authors.forename: Jean-Hugues
authors.initials: JH
authors.lastname: Trouvin
mesh_terms: D000818:Animals; D001688:Biological Products / Q000627:therapeutic use; D064987:Cell- and Tissue-Based Therapy / Q000941:ethics* / Q000379:methods; D002986:Clinical Trials as Topic; D004274:DNA, Recombinant / Q000627:therapeutic use; D017277:Drug Approval / Q000331:legislation & jurisprudence*; D004335:Drug and Narcotic Control / Q000331:legislation & jurisprudence*; D005602:France; D015316:Genetic Therapy / Q000941:ethics / Q000331:legislation & jurisprudence*; D005822:Genetic Vectors / Q000627:therapeutic use; D006801:Humans; D017387:Investigational New Drug Application / Q000331:legislation & jurisprudence; D061214:Patient Safety / Q000331:legislation & jurisprudence; D017410:Practice Guidelines as Topic; D012107:Research Design; D057170:Translational Research, Biomedical / Q000941:ethics / Q000331:legislation & jurisprudence*
chemical_list: D001688:Biological Products; D004274:DNA, Recombinant
keywords: Advance therapy medicinal product; Application dossier; Clinical trial; French regulation; Investigational medicinal product