Ideally, the studies would be underway at the time of approval, as the US FDA’s new authority allows, but Office of Therapeutic Products Director Nicole Verdun sys there will be exceptions.
Confirmatory study timing for cell and gene therapies gaining accelerated approval is not going to be mandated, new Office of Therapeutic Products Director Nicole Verdun said.
Verdun prefers that confirmatory trials be underway at the time of approval, but acknowledged there will be exceptions.
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
