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FDA leaders propose a 'plausible mechanism' pathway for individualized therapies
A statement by a ministry, institution or international organisation, or a change in regulations. It is not evidence about how well a treatment works.
Writing in NEJM, FDA leaders described an approval pathway called the 'plausible mechanism' pathway for individualized therapies for which randomized trials cannot be conducted. This pathway is being considered primarily for rare genetic diseases that cause death or severe disability in children. Real-world data will need to be collected after approval.
What does it mean for patients and families?
Individualized gene therapies may get a more structured approval process in the future. This development points to that. The details are not yet clear.
Source
RAPS: FDA leaders propose new 'plausible mechanism' pathway for bespoke medicines
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.