US FDA’s New ‘N of 1’ Guidance Essentially A Down Payment On Critical Advice
Draft guidance will help sponsor-investigators working on individualized gene therapies. The focus on basic administrative and procedural aspects of interaction with the FDA leaves many policy and commercialization questions about how these personalized investigational products should be studied unanswered.
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CBER’s heavy COVID-19 workload is pushing other things to the side, causing delays in sponsor meetings and issuance of new guidance documents, and a slowdown in efforts to streamline development of individualized therapeutics, biologics center leaders tell BIO Digital 2020.
CBER Director Peter Marks said the agency could streamline development by allowing sponsors to depend on an already-approved manufacturing platform and reviewing any modifications that are proposed.
Recent cases of individual designed treatments have FDA playing a bit of catch up. The concept has been on the horizon, but now “bespoke therapies” are here.