FDA Draft Guidance Refines Approval Process for Individualized Genetic Therapies
The U.S. Food and Drug Administration (FDA) has released draft guidance titled 'Considerations for the Use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause.' This guidance aims to adapt the regulatory approval process for individualized genetic interventions, particularly for rare genetic disorders affecting a small number of patients. Traditionally, each variant-specific modification would require its own investigational new drug application (IND) and extensive testing, which is economically and logistically infeasible for 'n-of-1' or 'n-of-few' patient populations. The new framework outlines five key elements for approval: a specific genetic abnormality with a clear link to disease, a therapy targeting the underlying pathology, a well-characterized natural history of the disease, confirmation of successful targeting, and demonstrated improvement in clinical outcomes. The draft guidance provides more detail on nonc... 
