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

Recommendation
20/21 May 2025
Vienna, Austria
With a workshop about the development of ADCs
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 It finalizes the draft guidance of the same title dated July 2013.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".
Related GMP News
12.05.2025EMA Proposes Revisions to GMP Guideline for ATMPs
07.05.2025FDA Initiates Transition to Non-Animal Testing Methods for Monoclonal Antibodies
16.04.2025FDA Warning Letter: Unauthorised Distribution of Fecal Microbiota Transplant Products
16.04.2025Development of a Risk-based Quality System for CMC R&D Laboratories in Drug Development
03.04.2025European Pharmacopoeia Commission Adopts First General Texts on mRNA Vaccines