Comment from National Organization for Rare Disorders

Summary: The National Organization for Rare Disorders (NORD) supports the FDA's interest in developing a Plausible Mechanism Framework for rare disease therapies but requests specific revisions to terminology, scope, and clarity on existing tools. They advocate for clarifying that the framework applies to non-genetic medicines, removing the term "individualized" to avoid misleading implications, and establishing a holistic framework for manufacturing and commercialization.