NCHR Written Comment to FDA on the Plausible Mechanism Framework for Developing Individualized Therapies for Genetic Conditions
April 27, 2026: NCHR raises concerns about the FDA’s proposed plausible mechanism framework for therapies targeting rare and genetic diseases, warning that reliance on such very limited evidence makes it difficult or impossible to make informed choices based on safety and effectiveness, and means even more very high costing unproven drugs, which patients will pay for directly or through health insurance.
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