Biologics center director was skeptical that RMAT would offer much beyond US FDA's existing breakthrough therapy pathway, but he has since come to appreciate the flexibility offered by less stringent designation criteria and leeway in the types of potential confirmatory evidence.
The US Food and Drug Administration’s Regenerative Medicine Advanced Therapy (RMAT) expedited pathway has evolved into a more useful “tool” than Center for Biologics Evaluation and Research Director Peter Marks originally expected when the program was established in December 2016 through the 21st Century Cures Act.
Speaking at the Food and Drug Law Institute annual meeting on 20 May, Marks said he initially was skeptical that...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
