CBER - Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products

Recommendation

9/10 May 2023
Heidelberg, Germany

Annex 2 & Co. - GMP Compliance for Biopharmaceuticals
Regulatory Requirements and Practical Implementation

In June the FDA Center for Biologics Evaluation and Research (CBER)/ Office of Cellular, Tissue, and Gene Therapies (OCTGT) published a guidance for industry "Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products". It finalizes the draft guidance of the same title dated July 2013.

This guidance document should assist sponsors and investigators in designing early-phase clinical trials for cellular therapy (CT) and gene therapy (GT) products. The guideline shows OCTGT’s current recommendations regarding clinical trials in which the primary objectives are the initial assessments of safety, tolerability, or feasibility of administration of investigational products.

There is a need of such a specified guidance document, because the design of early-phase clinical trials of CGT products often differs from the design of clinical trials for other types of pharmaceutical products. Except of the surrounding sections like Introduction, Background and References, the document covers the following segments:

Features of CGT products that influence clinical trial phases

• Product Characteristics
• Manufacturing Considerations
• Preclinical Considerations

Clinical trial design

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

Meetings with OCTGT

Guidance on submitting an IND

More details can be found direvtly in the guideline "Considerations for the Design of Early-Phase Clinical Trials ofCellular and Gene Therapy Products".