United States Food and Drug Administration Regulation of Gene and Cell Therapies.

Bailey AM1, Arcidiacono J2, Benton KA2, Taraporewala Z2, Winitsky S2.

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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.

KEYWORDS:

Cell therapy; Clinical development; Clinical trial; Gene therapy; Marketing application; Product licensure; Public health; US Food and Drug Administration (US FDA)