Regulatory Concerns in Human Gene Therapy

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Abstract
Gene therapy in humans is now being undertaken in an investigational setting. Such therapy involves the administration of biological products to human patients. A document entitled, “Points to Consider in Human Somatic Cell Therapy and Gene Therapy” has been prepared by the Center for Biologics Evaluation and Research (CBER) of the Food and Drug Administration (FDA) and is published elsewhere in this issue. This paper provides explanatory material about the CBER regulatory process and the scientific and regulatory basis for the requests for data in that document. The two institutions that have major regulatory responsibility over human gene therapy are the NIH and the FDA. The NIH has previously published its “Points to Consider” document. The FDA publishes a draft of its proposed “Points to Consider” on pages 251–256 of this issue. In this accompanying article, Epstein provides the background information on which the FDA “Points” are based.