Draft Guidance with Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products

Recommendation

7/8 May 2024

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Regulatory Requirements and Practical Implementation

Due to the  increasing interest and activity in the development of CGT products and because of their potential to address unmet medical needs the FDA published this draft guidance. It is intended to facilitate this development by providing recommendations regarding selected aspects of the design of early-phase clinical trials for these products.

The design of early-phase clinical trials of CGT products often differs from the design of clinical trials for other types of pharmaceutical products. These differences are necessitated by the distinctive features of these products, and may also reflect previous clinical experience. This Guidance is supposed to assist sponsors of Investigational New Drug Applications (INDs) for cellular therapy (CT) and gene therapy (GT) products. 

Amongst an introduction and the background information, the guidance includes the following contents:

1. Features of CGT products that influence clinical trial design

• Product Characteristics
• Manufacturing Considerations
• Preclinical Considerations

2. Clinical Trial Design

• Early-Phase Trial Objectives
• Choosing a Study Population
• Control Group and Blinding
• Dose Selection
• Treatment Plan
• Monitoring and Follow-up

Please see the draft "Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products" for more detailed information.