The long-term follow-up requirements expected for many gene therapies could raise consent and drop-out issues, which could hinder efforts to track safety and efficacy.
Pediatric patients will change a lot in the decade or more after receiving a gene therapy, which has raised concerns about postmarketing tactics to ensure safety and efficacy tracking can continue in the long-term.
US Food and Drug Administration officials are advising sponsors that postmarketing follow-up for gene therapies could last up to 15...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
