Comment from ALS United
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Summary: ALS United, a nationwide partnership of nonprofit organizations, supports the FDA's draft guidance on the Plausible Mechanism Framework for individualized therapies. They argue that the framework should specifically include mutation-defined subsets of ALS while maintaining rigorous standards for biomarker validation, confirmatory studies, and safety oversight.
Please see the attached comments submitted on behalf of ALS United in response to FDA’s draft guidance, “Considerations for the Use of the Plausible Mechanism Framework to Develop Individualized Therapies That Target Specific Genetic Conditions With Known Biological Cause.
ALS United appreciates FDA’s work to develop a practical and scientifically grounded pathway for individualized therapies in ultra-rare settings and respectfully requests that FDA consider the attached recommendations as it finalizes the guidance.