A process in which the FDA coordinates reviews with other regulators would allow for better leveraging of global patient populations with ultra-rare diseases and attract more commercial interest in a given disease area, CBER’s Peter Marks tells the Biopharma Congress. The biologics center also looks to apply the philosophy underlying the Real-Time Oncology Review program.
The US Food and Drug Administration is eyeing potential adoption of a Project Orbis-type approach for cell and gene therapies to treat rare diseases.
A process where the FDA can coordinate reviews with other regulators would allow for better leveraging of global patient populations...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
