FDA draft guidance sets framework for individualized rare disease therapies
The US Food and Drug Administration published draft guidance in February 2026 establishing a plausible mechanism framework for individualized therapies that target rare genetic diseases. The framework is a genuine advance, but its five criteria are not equally tractable, and meeting them will require substantial groundwork for most rare disease programs. The commentary appeared in Nature Genetics.
The framework could speed approval for patients with rare genetic conditions. Implementation challenges remain.
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