FDA Commissioner Scott Gottlieb unveils regulatory framework to promote gene therapy development

FDA Commissioner Scott Gottlieb on Wednesday outlined a regulatory framework for the development of gene therapies. Gottlieb said the agency will issue a total of six scientific guidance documents that will form the basis of a modern, comprehensive framework for advancing gene therapy. "These policies are part of our efforts to communicate the steps we’re taking to provide clear recommendations to sponsors and researchers, so that we can better support innovation," the Commissioner stated.

As part of the framework, Gottlieb noted that the agency is releasing the first three disease-specific draft guidance documents on the development of gene therapy products. Gottlieb explained that the first document, which covers gene therapies designed for the treatment of haemophilia, will provide recommendations concerning the agency's current thinking about clinical trial design and preclinical considerations to support the development of these products.

Meanwhile, the Commissioner said that the second draft guidance, which is about gene therapies for retinal disorders provides recommendations regarding product development, preclinical testing and clinical trial design for such products. In addition, the third draft guidance, which is focusing on gene therapy products for rare diseases aims to assist companies in the design of clinical development programmes and issues related to interpretation of efficacy.

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Gottlieb also said that the FDA is providing updates to three existing guidance documents that address manufacturing issues related to gene therapy. The first document, which covers chemistry, manufacturing and control information for new drug applications for gene therapies, provides recommendations for submitting such information to ensure the safety and potency of the products.

According to Gottlieb's statement, the second document on testing for retroviral vector-based gene therapies provides additional recommendations concerning the proper testing of replication-competent retroviruses during production of retroviral vector-based gene therapy products as well as patient monitoring after treatment. The final document provides recommendations on the design of long-term follow-up observational studies to assess delayed adverse events after treatment with a gene therapy product. The FDA noted that once finalised, the three documents will replace guidance issued in November 2006 and April 2008.

Gottlieb has unveiled a number of initiatives since being confirmed as agency commissioner last year. In October 2017, Gottlieb announced several proposals to speed the development of complex generics. Later, the Commissioner disclosed plans to speed the development of new drugs, especially cancer therapies, using early data.

For related analysis, read ViewPoints: FDA looking to take flexible stance on regulating gene therapies.

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