In an interview with the Pink Sheet, Marks said the guidances on regenerative medicines make clear what the agency will be enforcing.
Cracking the whip against bad actors in the regenerative medicines arena, such as noncompliant stem cell clinics, has always been a priority for the Center for Biologics Evaluation and Research (CBER). But the center plans on taking its enforcement actions a step further in 2018, CBER Director Peter Marks says.
"Enforcement actions on stem cell clinics that were putting out products that could put patients at risk that were unapproved...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
