FDA, CMS Unveil Coordinated Moves on Individualized Gene Therapies
HHS announced coordinated actions by FDA and CMS on October 27, 2026 to advance individualized gene therapies. FDA issued draft guidance allowing companies to seek approval for multiple patient-specific versions of a genome-editing therapy under one Biologics License Application, using an "Individualized Feature Space" to define which product components may vary without separate clinical trials for each variant, limited to single-gene disorders with known pathogenic variants. CMS separately announced plans to expand its Cell and Gene Therapy Access Model, which uses outcomes-based agreements with manufacturers to improve Medicaid access, beyond its initial focus on sickle cell disease. The guidance is in draft form and does not change FDA's approval standards or existing expedited-review designations.