Regulation of cellular therapies falls under different pathways depending on processing, intended use, country of collection and manufacture.
The Food and Drug Administration’s Center for Biologics Evaluation and Research is responsible for regulation of cellular and tissue-based therapies in the U.S. An overview of FDA ’s regulatory approach is described below.
Based on a tiered, risk-based approach for the regulation of cellular and tissue-based therapies, the FDA has established two primary regulatory pathways for these products. Under the first scheme, cellular therapy products that meet all the criteria in 21 CFR 1271.10(a) are regulated solely as HCT/Ps and are not required to be licensed, approved or cleared. These products are commonly referred to as "361 products" because they are regulated solely under Section 361 of the Public Health Service (PHS) Act. The regulatory focus on these products is prevention of communicable disease.
Under the second scheme, if a cellular therapy product does not meet all the criteria in 21 CFR 1271.10(a) it is regulated as a drug, device, and/or biological product under the Federal Food, Drug, and Cosmetic Act (FDCA) and Section 351 of the PHS Act, and as such is commonly referred to as a "351 product." The FDA requires premarket review of safety and efficacy data before these cellular therapy products are introduced into interstate commerce, and the products must comply with applicable provisions in Title 21 of the CFR 600 series (biologicals), 200 series (drugs), and 800 series (devices).
Autologous cellular and tissue-based products collected and transplanted in the same surgical procedure, without significant manipulation, are not subject to FDA oversight. Vascularized organs and minimally manipulated bone marrow cells both are regulated by the Health Resources and Services Administration.
Cellular therapies are an increasingly international activity. The FDCA mandates that the FDA collaborate with foreign regulatory authorities to reduce regulatory burdens, harmonize regulatory requirements, and establish appropriate reciprocal arrangements. The FDA's International Programs have grown in recent years — the agency has opened offices in Africa, Asia, Europe, India and Latin America. The Center for Biologics Evaluation and Research also has established an International Program focused on partnering with other organizations to enhance regulatory harmonization, regulatory capacity building, information sharing, international standards setting, and collaborative research across the globe for products under its jurisdiction.
The World Health Organization (WHO) is the directing and coordinating authority for health within the United Nations. The WHO Guiding Principles on Human Cell, Tissue and Organ Transplantation, which are intended to provide an orderly, ethical and acceptable framework for the acquisition and transplantation of human cells, tissues and organs for therapeutic purposes, have greatly influenced professional codes and practices as well as legislation around the world for almost two decades.
Regulations for cellular therapies outside the U.S. are determined by regional and local competent authorities. A listing of some regulatory authorities is available at
International Competent Authorities.
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September 15, 2023
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