U.S. flag An official website of the United States government

On Oct. 1, 2024, the FDA began implementing a reorganization impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.

U.S. Food and Drug Administration
  1. Home
  2. Regulatory Information
  3. Search for FDA Guidance Documents
  4. Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial
  1. Search for FDA Guidance Documents

GUIDANCE DOCUMENT

Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial Guidance for Industry November 2022

Download the Final Guidance Document Read the Federal Register Notice
Final
Docket Number:
FDA-2021-D-0776
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

The purpose of this guidance is to provide recommendations to sponsors interested in studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial for a single disease.  Sponsors have expressed interest in gathering preliminary evidence of safety and activity using multiple versions of a cellular or gene therapy product in a single clinical trial.  Although multiple versions of a product can be studied together in a single clinical trial, each version of the product is distinct and is generally submitted to FDA in a separate investigational new drug application (IND).  The objective of these early-phase clinical studies is to guide which version(s) of the product to pursue for further development in later-phase studies.  Thus, these studies are not intended to provide primary evidence of effectiveness to support a marketing application and generally are not adequately powered to demonstrate a statistically significant difference in efficacy between the study arms.  In this guidance, we, FDA, provide recommendations for studies that evaluate multiple versions of a cellular or gene therapy product, including how to organize and structure the INDs, submit new information, and report adverse events.  This guidance finalizes the draft guidance of the same title dated September 2021.

Submit Comments

Submit Comments Online

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2021-D-0776.

Back to Top