HHS and FDA unveil draft “plausible mechanism” framework to speed individualized rare‑disease therapies HHS announced draft FDA guidance creating a plausible mechanism pathway to allow approval of individualized and ultra‑rare therapies based on strong mechanistic rationale plus confirmatory evidence when randomized trials are infeasible. Officials emphasized safeguards including long‑term monitoring and transparency.

HHS and FDA just unveiled a groundbreaking draft guidance that could revolutionize how we approve ultra-rare genetic therapies, speeding life-saving treatments to patients who need them most!

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