Comment from American College of Medical Genetics and Genomics

Summary: The American College of Medical Genetics and Genomics (ACMG) supports the FDA's draft guidance on the plausible mechanisms framework for developing individualized therapies for rare genetic conditions. They commend the FDA's approach but request specific clarifications and additional guidance regarding diagnostic rigor, basket trials, biomarker acceptance, natural history data for ultra-rare diseases, and decentralized manufacturing requirements.