FDA proposes new system for approving customized drugs and therapies for rare diseases

Associated Press (AP)EN 3 min read 100% complete by By MATTHEW PERRONEFebruary 23, 2026 at 05:53 PM

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The FDA proposed new guidelines on Monday, September 22, 2025, to accelerate the development of customized treatments for rare diseases. The preliminary guidelines would create a new approval pathway for bespoke therapies, including gene editing, that have been tested on only a few patients due to the difficulty of conducting larger studies. FDA Commissioner Marty Makary stated the goal is to remove barriers and encourage scientific advances for rare diseases. This announcement follows a recent decision by the FDA to drop the requirement of two clinical trials for standard drug reviews. The FDA will accept public comments on the draft guidance for 60 days before finalizing it.

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Donald Trump 40% Washington 50% Children's Hospital of Philadelphia 50% University of Pennsylvania 50% CRISPR 60% Marty Makary ×280% Food and Drug Administration ×790%

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fda 100% rare diseases 100% customized treatments 90% drug approval 80% gene editing 70% bespoke therapies 60% pharmaceutical industry 60% clinical trials 50% regulatory flexibility 50%

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Associated Press (AP)

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