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Accelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks says - Endpoints
The FDA will increasingly use accelerated approval for gene therapies aimed at rare conditions, according to Peter Marks, the FDA’s top official overseeing cell and gene therapies, the clearest signal yet of how widely the agency will deploy its expedited pathway.
How biomarkers can support regulatory rare disease pathways - BioNews
On February 21, the Biotechnology Innovation Organization’s (BIO) soon-to-be CEO, John F. Crowley, joined an event about biomarkers and rare disease regulatory pathways, hosted by the Reagan-Udall Foundation for the Food and Drug Administration in Washington, D.C.
Natural History Studies and Registries in the Development of Rare Disease Treatments
The Reagan-Udall Foundation for the FDA, in collaboration with FDA’s Rare Diseases Team within the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine; Office of New Drugs; Center for Drug Evaluation and Research and the National Institutes of Health’s Division of Rare Diseases Research Innovation within the National Center for Advancing Translational Sciences, is hosting a public workshop.