Officials from the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) asserted that the duration of clinical trials for gene therapies depends on the nature of the disease being treated, and that diseases that are more progressive and have a rapid onset may involve shorter trials.
This was one of the learnings imparted by officials during a 7 February virtual town hall meeting to answer stakeholder questions on the clinical development of gene therapies for rare diseases outside the hematology and oncology space. The town hall is part of a series to address questions from stakeholders on topics under OTAT’s remit.
The town hall addressed the factors sponsors should consider when determining the study population for early phase gene therapy trials; the office’s criteria for allowing single arm, externally controlled trial to provide the primary evidence of effectiveness for approval; and the agency’s guidelines for determining the duration of gene therapy clinical trials…
