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40.2 Regulation of gene therapy

Two federal agencies, the Food and Drug Administration (FDA) and the National Institute of Health, Office of Biotechnology Activities (NIH, OBA) belonging to the Department of Health and Human Services (DHHS) regulate the gene therapy clinical trials. FDA is involved in the determination of the quality, safety, purity potency and identity of the gene therapy product so that it can be safely administered to patients whereas the NIH is responsible to oversee the quality of science or research used in the development of the gene therapy. NIH provides funds to institutes and scientists involved in gene therapy research work. It also precisely evaluates and reviews the composition of Institutional Bio safety Committees (IBC). Public reviews are conducted and full awareness program of the various novel clinical trials is communicated and discussed through the quarterly meetings of the Recombinant DNA Advisory Committee (RAC). Confidentiality of the review of Investigational New Drugs (INDs) is maintained by the FDA reviewers. The pros and cons of the preclinical and clinical investigations are carefully determined by the FDA and the NIH whereas the ethical, legal and social issues are carefully considered by the RAC. FDA and the NIH have to be informed with proper document submission prior to initiation of human gene therapy clinical trials. IND application has to be submitted by the sponsors of a gene therapy clinical trial in a prescribed format to the Centre for Biologics Evaluation and Research (CBER). The approval of the Institutional review Board (IRB) and the IBC is essential for getting a clearance to conduct the clinical trials. The complete information and the protocol have to be submitted by the investigators to the NIH, OBA in accordance with the prescribed NIH guidelines. The protocol is also submitted to the RAC which after careful review and public discussion gives comments to the investigator and sends recommendations to the IRB, IBC and the FDA. By conducting public reviews the ethical, societal issues are addressed and the acceptance and progress of gene therapy is determined.