FDA Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products

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Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products[附网盘链接]由FDA于不久之前发布，适用于美国。

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Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products _______________________________________________________ Guidance for Industry This guidance represents the Food and Drug Administration’s (FDA’s or Agency’s) current thinking on this topic. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. You can use an alternative approach if the approach satisfies the requirements of the applicable statutes and regulations. If you want to discuss an alternative approach, contact the FDA staff responsible for implementing this guidance using the contact information on the title page of this guidance. I. INTRODUCTION 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 1

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 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 1

Many of the principles in this guidance may apply to combination products involving a biological product under OCTGT’s regulatory authority.

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