The US Food and Drugs Administration (FDA) has issued the final version of its Guidance for Industry document on the “Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products” (link to document). The guidance document addresses the specific issues associated with clinical trials of cellular and gene therapy (CGT) products based on experience gained from such trials, and includes information on:
Features of CGT products that influence clinical trial design, including product characteristics, manufacturing considerations, and preclinical (nonclinical) considerations
Clinical trial design, including early-phase trial objectives, choosing a study population, control groups and blinding, dose and regimen, treatment plan, and monitoring and follow up
Meetings with the Office of Cellular, Tissue, and Gene Therapies (OCTGT)
Guidance on submitting an Investigational New Drug application (IND)
The document is aimed at developers of CGT products who wish to conduct FDA-approved clinical trials in the US. Similar guidance for EU clinical trials is published by EMA in the Guideline on Human Cell-Based Medicinal Products (EMEA/CHMP/410869/2006).
Author: Patrick Ginty