Running multiple product-specific postmarket trials for 15 years or longer would not be cost-effective and one industry stakeholder suggests workstreams eventually could merge.
Standardization of methods and trials may be necessary for long-term post-approval data collection on cell and gene therapies to be feasible.
US Food and Drug Administration officials want 15 years or more of postmarketing follow-up for new cell and gene therapies,...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
