Studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial: guidance for industry

Studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial: guidance for industry

Collection:
Health Policy and Services Research
Contributor(s):
Center for Biologics Evaluation and Research (U.S.), issuing body.
Publication:
Silver Spring, MD : Center for Biologics Evaluation and Research, November 2022
Language(s):
English
Format:
Text
Subject(s):
Cell- and Tissue-Based Therapy
Clinical Trials as Topic
Genetic Therapy
Government Regulation
United States
United States. Food and Drug Administration
Genre(s):
Guideline
Technical Report
Abstract:
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 This guidance finalizes the draft guidance of the same title dated September 2021. In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required.
Copyright:
The National Library of Medicine believes this item to be in the public domain. (More information)
Extent:
1 online resource (1 PDF file (i, 10 pages))
Illustrations:
Illustrations
NLM Unique ID:
9918590086206676 (See catalog record)
Permanent Link:
http://resource.nlm.nih.gov/9918590086206676