CBER director does not want to hold back potentially beneficial products, but also warned that patients will have difficult treatment decisions to make since an immune response might prevent a patient from receiving another gene therapy using the same vector ever again.
Some gene therapies that reach the market through accelerated approval may not ultimately show clinical benefit, but that’s acceptable, said Peter Marks, director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research.
However, he also said the consequences for patients could be substantial in those cases and that they must understand the...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
