The Center for Biologics Evaluation and Research (CBER)/Office of Cellular, Tissue, and Gene Therapies (OCTGT) is issuing this guidance to assist sponsors of Investigational New Drug Applications (INDs) for cellular therapy (CT) and gene therapy (GT) products. CT and GT products will be referred to collectively as CGT products. This guidance provides recommendations to assist in designing early-phase clinical trials of CGT products. When this guidance is finalized, we believe it will clarify OCTGT’s current expectations 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)). 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 Code of Federal Regulations (CFR) Part 1271 (21 CFR Part 1271), 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.


Posted on the FDA website on 2 July 2013
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 early-phase clinical trials of these products. This guidance does not provide detailed information about the preclinical and chemistry, manufacturing, and controls (CMC) components of an IND, as we have previously provided recommendations in connection with these components