Data indicates a smaller percentage of studies intended for product registration or indication expansion are single-arm trials, compared to the proportion for all gene therapy trials.
Sponsors might not be confident that single-arm studies can support gene therapy approvals in the US despite Peter Marks’ statements to the contrary.
Marks, the director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research, wants to grow gene...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
