Stakeholders propose centralized oversight approaches for managing quality of “like-for-like” fleets of self-contained manufacturing units that could roam the US – or the world. The US FDA shares the industry’s thinking on how best to adapt the site-focused regulatory scheme it enforces to the emerging variety of distributed manufacturing systems.
Industry stakeholders have proposed ways to adapt the facility-specific regulation of drug manufacturing quality for emerging distributed and point-of-care manufacturing technologies that would reduce compliance burden but also in some cases could reduce quality assurance.
The proposals were prompted by a discussion paper the US Food and Drug Administration issued in October 2022. (Also see...
Many platform designation requests have been from sponsors eager to cite other sponsors’ products, but CBER Director Peter Marks said in an interview with the Pink Sheet his office likely is years away from accepting those applications.
CBER Director Peter Marks outlines a streamlined process to approval for treating different mutations of the same gene. NCATS’ Philip Brooks tells the Pink Sheet the approach avoids the need to “start from scratch for every new mutation.”
New draft guidance defines the necessary components of a platform technology designation request, including that sponsors demonstrate how it will save the FDA review time.
CBER’s Nicole Verdun said the FDA is using the principles of the platform approach in situations that don’t qualify for the agency’s new incentive.
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.
