WASHINGTON, Feb. 23, 2026 — The U.S. Food and Drug Administration proposed a new “plausible mechanism” framework to speed approvals of individualized therapies for rare, life‑threatening genetic diseases, allowing developers to rely on small, well‑controlled studies when traditional trials are not feasible, according to Reuters. The framework is described in the FDA’s draft guidance.
The proposal is intended to support genome‑editing and RNA‑based medicines tailored to tiny patient populations and to expand access where full‑scale randomized trials are impractical, as outlined by Reuters and reported by AP.
Why the framework now
Health officials say the U.S. has more than 10,000 rare diseases affecting over 30 million Americans, a scale highlighted by Spectrum News and NPR.
Because many conditions affect only a handful of people worldwide, traditional trials can take years and cost millions, limiting commercial incentives, according to AP and Spectrum News.
How approvals would work
Under the draft, sponsors must show a plausible biological rationale and evidence that a therapy targets the underlying genetic or molecular cause, criteria described in the FDA’s guidance and summarized by Spectrum News.
The FDA said it will still require safety monitoring and post‑approval evidence for individualized products, an approach noted by Reuters and echoed in Spectrum News coverage of long‑term monitoring.
Promise and safeguards
At Monday’s announcement, HHS Secretary Robert F. Kennedy Jr. said families had been told to wait for science, while FDA Commissioner Marty Makary said rare diseases had long been an afterthought, comments quoted by NPR and Spectrum News.
NPR and AP both pointed to a Pennsylvania infant treated at Children’s Hospital of Philadelphia with a customized CRISPR therapy as a case that showed individualized gene‑editing can work when conventional options fail.
Reuters and the FDA guidance both emphasize that evidence of safety and regulatory‑quality manufacturing remains central even as approvals move faster.
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Image: “DNA in a circle” by Ryan Kissinger, Courtesy of NIAID (NIH BioArt). Public domain. Source: https://commons.wikimedia.org/wiki/File:DNA_(NIH_BioArt_126_-_629405).png. Modifications: resized and cropped to 1920×1080 (16:9).
