Key Takeaways

  • FDA releases three draft guidance documents to support the development of CGTs.
  • The FDA is inviting comments on these drafts until November 24, and December 24.
  • Recommendations for designing clinical trials in small patient populations including trial design, endpoints, and data generation to support product licensure.
  • Guidance on accelerating development and review of regenerative medicine therapies, covering five FDA expedited programs.
  • Recommendations for generating safety and efficacy data after product approval, with attention to long-term effects and small patient cohorts.

 

The US Food and Drug Administration (FDA) has issued three draft guidance documents outlining advice for the development of novel cell and gene therapies. These documents include guidelines for innovative trial designs, expedited programmes for regenerative medicine therapies, and post-approval recommendations

The FDA has invited comments on the guidance. The deadline to respond to the first two documents is 24 November 2025, while comments on the third document are required by 24 December 2025.

Document 1: Innovative Trial Designs

The Agency’s first document is titled Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations. This guidance sets out recommendations to sponsors of CGT clinical trials for small patient populations, like those with a rare disease. Innovative Designs for Clinical Trials provides recommendations for trial design, endpoints, and data generation to support product licensure.

Document 2: Expedited Programmes for Regenerative Medicine

This document, dubbed Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, provides guidance to sponsors in developing regenerative medicine therapies. Therein, the FDA sets out recommendations for accelerating the development and review process of these drugs.

It looks at five expedited programmes offered by the FDA, and how they may apply to regenerative medicines. These programmes include Fast Track designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy (RMAT) designation, accelerated approval, and priority review designation.

Document 3: Post-Approval Study Guidance

The final document to be released by the FDA this September is called Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products. This draft guidance discusses the generation of safety and efficacy data after a CGT product has been approved. Herein, it outlines recommendations on the approaches and methods to do so.

Document three acknowledges the potential for long-lasting effects of CGT products and small patient cohorts in their trials. Therefore, it states that “post-approval monitoring is important for gathering data on product safety and effectiveness over time.”