Commercial scale-up during Phase III trials could create labeling issues and force additional clinical trials, US FDA officials said.
Gene therapy sponsors risk review problems and development delays if they attempt manufacturing changes during pivotal clinical studies.
Commercial production processes should be in place for the entire Phase III trial, said Graeme Price, Gene Therapy Branch 4...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
