Comment from N=1 Collaborative
AnonymousSupportAdvocacy
Summary: The N=1 Collaborative (N1C), a nonprofit consortium of clinicians, researchers, and patient advocates, supports the draft guidance as a necessary step for developing individualized therapies for rare genetic diseases. They recommend specific improvements, including making data sharing a requirement, calibrating CMC standards for small-batch manufacturing, expanding the platform concept to multiple genes, and establishing a formal process for developing clinical effectiveness standards.
N1C submits these comments in response to the Food and Drug Administration's draft guidance entitled "Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause" (hereafter "the Guidance"). We welcome the Guidance as a meaningful and urgently needed step toward a regulatory framework that reflects the scientific and clinical realities of an individualized approach to genetic medicine development. The Guidance opens an important door, and the N1C community is committed to coordinating and enabling new programs using this guidance while iteratively learning with the Agency. Our full comments and signatories are attached.