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

Description

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  This guidance finalizes the draft guidance of the same title dated September 2021.

Key Topics

Terms and concepts identified from this document

• 21CFR312.40§ 312.40 General requirements for use of an investigational new drug in a clinical investigation.

  (a) An investigational new drug may be used in a clinical investigation if the following conditions are met:Read full regulation →

• 21CFR56.103§ 56.103 Circumstances in which IRB review is required.

  (a) Except as provided in §§ 56.104 and 56.105 , any clinical investigation which must meet the requirements for prior submission (as required in parts 312, 812, and 813) to the Food and Drug Administration shall not be initiated unless that investigation has been reviewed and approved by, and remaiRead full regulation →

• 21CFR312.42§ 312.42 Clinical holds and requests for modification.

  (a) General. A clinical hold is an order issued by FDA to the sponsor to delay a proposed clinical investigation or to suspend an ongoing investigation. The clinical hold order may apply to one or more of the investigations covered by an IND. When a proposed study is placed on clinical hold, subjectRead full regulation →

• 21CFR312.32§ 312.32 IND safety reporting.

  (a) Definitions. The following definitions of terms apply to this section:Read full regulation →

• 21CFR312.33§ 312.33 Annual reports.

  A sponsor shall within 60 days of the anniversary date that the IND went into effect, submit a brief report of the progress of the investigation that includes:Read full regulation →

Deficiencies cited in Warning Letters referencing the same regulations

• Clinical Investigator (Sponsor)

  Angela D. Ritter, M.D.

  2024-06-18

Korean regulatory guidelines covering similar topics

• 세포·유전자치료제 초기 임상시험 디자인 가이드라인
  TitleMedium
• 임상시험용 세포치료제·유전자치료제 비교동등성 평가 가이드라인
  TitleMedium

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