Trial protocols, annual reports, and adverse event reports on gene therapies will only go to US FDA under now-finalized amendments, a sign that the product category is maturing and can be regulated like other drugs and biologics.
The US National Institutes of Health has finalized a proposal it made last summer to reduce its role in the oversight of gene therapy. Because the proposal has now been finalized without meaningful changes, we are reprinting our initial story.
In announcing the finalization, NIH noted that by far most of the comments on the proposal favored no longer requiring...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
