The Center for Biologics Evaluation and Research (CBER)/Office of Cellular, Tissue, and Gene
Therapies (OCTGT) is issuing this guidance to assist sponsors and investigators in designing
early-phase clinical trials for cellular therapy (CT) and gene therapy (GT) products. CT and GT
products will be referred to collectively as CGT products. This guidance provides OCTGT’s
current recommendations regarding clinical trials in which the primary objectives are the initial
assessments of safety, tolerability, or feasibility of administration of investigational products.
Such trials include most Phase 1 trials, including the initial introduction of an investigational
new drug into humans, and some Phase 2 trials of CGT products.
The scope of this guidance is limited to products for which OCTGT has regulatory authority.
CGT products within the scope of this guidance meet the definition of “biological product” in
section 351(i) of the Public Health Service (PHS) Act (42 U.S.C. 262(i)) and include CT and GT
products that are used as therapeutic vaccines. This guidance does not apply to those human
cells, tissues, and cellular- and tissue-based products (HCT/Ps) regulated solely under section
361 of the PHS Act (42 U.S.C. 264), as described in Title 21 of the Code of Federal Regulations
(CFR) Part 1271 (21 CFR Part 1271), or to products regulated as medical devices under the
Federal Food, Drug, and Cosmetic Act, or to therapeutic biological products for which the
Center for Drug Evaluation and Research (CDER) has regulatory responsibility.
There is increasing interest and activity in the development of CGT products because of their
potential to address unmet medical needs. This guidance is intended to facilitate such
development by providing recommendations regarding selected aspects of the design of earlyphase
clinical trials of these products. This guidance does not provide detailed information about
the preclinical and chemistry, manufacturing, and controls (CMC) components of an investigational new drug application (IND), as we have previously provided recommendations in connection with these components (Refs. 1, 2, and 3). This guidance is intended to complement the information in those guidances.
This guidance finalizes the draft guidance of the same title dated July 2013