title: United States Food and Drug Administration Regulation of Gene and Cell Therapies.
abstract: The United States (US) Food and Drug Administration (FDA) is a regulatory agency that has oversight for a wide range of products entering the US market, including gene and cell therapies. The regulatory approach for these products is similar to other medical products within the United States and consists of a multitiered framework of statutes, regulations, and guidance documents. Within this framework, there is considerable flexibility which is necessary due to the biological and technical complexity of these products in general. This chapter provides an overview of the US FDA regulatory oversight of gene and cell therapy products.
authors.affiliation: Office of Cellular, Tissue, and Gene Therapies (OCTGT), Center for Biologics Evaluation and Research (CBER), United States Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA. alexander.bailey@fda.hhs.gov.
authors.forename: Alexander M
authors.initials: AM
authors.lastname: Bailey
authors.affiliation: Office of Cellular, Tissue, and Gene Therapies (OCTGT), Center for Biologics Evaluation and Research (CBER), United States Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
authors.forename: Judith
authors.initials: J
authors.lastname: Arcidiacono
authors.affiliation: Office of Cellular, Tissue, and Gene Therapies (OCTGT), Center for Biologics Evaluation and Research (CBER), United States Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
authors.forename: Kimberly A
authors.initials: KA
authors.lastname: Benton
authors.affiliation: Office of Cellular, Tissue, and Gene Therapies (OCTGT), Center for Biologics Evaluation and Research (CBER), United States Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
authors.forename: Zenobia
authors.initials: Z
authors.lastname: Taraporewala
authors.affiliation: Office of Cellular, Tissue, and Gene Therapies (OCTGT), Center for Biologics Evaluation and Research (CBER), United States Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
authors.forename: Steve
authors.initials: S
authors.lastname: Winitsky
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000941:ethics / Q000379:methods*; D002986:Clinical Trials as Topic; D004335:Drug and Narcotic Control / Q000331:legislation & jurisprudence*; D005069:Evaluation Studies as Topic; D015316:Genetic Therapy / Q000941:ethics / Q000331:legislation & jurisprudence*; D006801:Humans; D061214:Patient Safety / Q000331:legislation & jurisprudence; D017410:Practice Guidelines as Topic; D011786:Quality Control; D014481:United States; D014486:United States Food and Drug Administration / Q000331:legislation & jurisprudence*
keywords: Cell therapy; Clinical development; Clinical trial; Gene therapy; Marketing application; Product licensure; Public health; US Food and Drug Administration (US FDA)