FDA moves to facilitate personalized therapies for rare diseases

Published February 23, 2026 12:15pm ET



The Food and Drug Administration is making it possible for pharmaceutical companies to produce bespoke medicines for individual patients, an effort to revolutionize the standard of care for rare diseases. 

Trump administration officials announced on Monday new draft guidance from the FDA that would update approval standards for personalized genetic medicines, creating a new approval pathway for therapies targeted to patient groups too small for traditional clinical trials.

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