Retiring Office of Tissues and Advanced Therapies’ Director Wilson Bryan expects the expedited pathway will be used more frequently moving forward, but worries that ambiguous confirmatory trial results many years down the line will still leave efficacy questions unanswered.
Accelerated approval is likely to be used more extensively in the future for gene therapies that treat very rare diseases, but the slowly progressive nature of some conditions means there may be many years of uncertainty about the clinical benefits provided by the one-time treatments, the head of the US FDA’s Office of Tissues and Advanced Therapies said.
OTAT Director Wilson Bryan opined on the future use of accelerated approval for gene therapies, worries about ambiguous confirmatory trial...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
