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 classifi ed as medicinal products and as such should be developed according to a well-structured development plan, to establish their quality, safety and effi cacy profi le and conclude, at the time of the marketing authorisation evaluation, on a positive risk/benefi t 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 fi nal authorisation to initiate a clinical trial, the legislation imposes to run in parallel two independent but complementary authorisation procedures. The fi rst 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 modifi ed organism.The French system herein described is in line with the EU regulation on clinical trial and follows the respective deadlines for granting the fi nal 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.