In final guidance documents on development of CAR-T cell products and human genome therapies incorporating genome editing, US FDA says that sponsors should plan for continued follow-up, with funding, even if they cease operations or withdraw the IND.
Gene and cell therapy product sponsors should have a plan for ensuring that long-term safety follow-up is completed even if they go out of business, the US Food and Drug Administration said.
Two final guidance documents released on 29 January, “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products”...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
