Forthcoming guidance is expected to describe areas of “low-hanging fruit” and those that are more challenging for use of the expedited pathway, Center for Biologics Evaluation and Research Director Peter Marks said.
A forthcoming US Food and Drug Administration guidance on accelerated approval for gene therapies will address what Center for Biologics Evaluation and Research Director Peter Marks described as three “buckets” of scenarios for the expedited pathway’s use.
Marks discussed CBER’s plans for a guidance that will try to more clearly define the pathway for gene therapies and...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
