US FDA declined stakeholder requests to remove recommendation for placebo controls from a final guidance but added qualifying language on their use ‘when feasible.’ While the agency excised language on biomarker validation from the final guidance, it reaffirmed the need for early establishment of critical quality attributes and critical process parameters.
The US Food and Drug Administration declined stakeholder requests to eliminate a recommendation for placebo controls in a final guidance document on rare disease gene therapies but provided more flexibility around their use "when feasible."
In the final guidance, Human Gene Therapy for Rare Diseases, the agency bowed to industry concerns about the 2018...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.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.
