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Regulation of Cellular Therapy in the United States

Book Chapter

Fisher, Nicole; Ibenana, Laarni; El Fiky, Ashraf; Anderson, Robert

Cell Therapy: cGMP Facilities and Manufacturing


Springer International Publishing

Gee, Adrian P.



HCT/P; FDA; Good tissue practices; Good manufacturing practices; Regenerative Medicine Advanced Therapy; HPC, cord blood; Biologics regulations; Biologics License Application; BLA

In the United States, the Food and Drug Administration (FDA) is the jurisdictional authority for the regulation of cellular therapy products. The approach to regulatory oversight is tiered, with human cell, tissue, and cellular and tissue-based products (HCT/Ps) separated into two categories based on the risks associated with the use of the product. Products that meet the requirements for a lower level of regulatory oversight are regulated solely under Section 361 of the Public Health Services (PHS) Act, which provides the statutory authority to create and enforce regulations deemed necessary to prevent the introduction, transmission, or spread of communicable diseases. Under Section 361 of the PHS Act, HCT/Ps must comply with Title 21 of the Code of Federal Regulations 1271 [21 CFR 1271], but do not require premarket approval. HCT/Ps that do not meet the specified criteria to be regulated solely under Section 361 of the PHS are regulated as drugs, biologics, and/or devices under Section 351 of the PHS Act and the Food, Drug, and Cosmetic (FD&C) Act. These products require premarket approval and must also comply with applicable regulations under Title 21 of the CFR, including good manufacturing practices [21 CFR 210 and 211] and 21 CFR 1271. Relevant provisions in Part 1271 applicable to all HCT/Ps include donor eligibility and good tissue practices. Biological drug products must conform to the biological product regulations [21 CFR 600-680] and require a license, which is granted upon demonstration that the product meets prescribed requirements of safety, purity, and potency. In order to collect the safety and efficacy data supporting licensure, the product is studied in clinical trials under an Investigational New Drug (IND) application, in accordance with 21 CFR 312. Some cellular therapies are combined with gene therapy, which is intended to modify a patient’s genome in order to cure or treat a disease. These cell/gene therapy products are subject to the same statutory requirements as HCT/Ps regulated as drugs, biologics, and/or devices; however, the FDA has issued several guidance documents to address unique characteristics of gene therapy products. Cell and gene therapy products that meet certain criteria may be eligible for expedited development programs at the FDA, including Regenerative Medicine Advanced Therapy (RMAT) designation.

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