Federal health agencies announced two steps on October 27 aimed at developing individualized gene therapies and broadening access through Medicaid.
The Food and Drug Administration issued draft guidance describing how developers could seek approval for genome-editing treatments tailored to different genetic mutations within one biologics application.
The proposed approach defines boundaries for how a product may vary among patients. Within those boundaries, laboratory evidence could support adding patient-specific variants without additional clinical trials, HHS said. The document also addresses manufacturing, safety testing and unintended genetic changes.
The guidance remains a draft open for public comment. It does not approve a particular treatment.
Separately, the Centers for Medicare & Medicaid Services plans to invite manufacturers treating conditions beyond sickle cell disease into its voluntary Cell and Gene Therapy Access Model. CMS negotiates outcomes-based agreements on participating states’ behalf.
HHS said a manufacturer application request would follow in coming months. The announcement does not establish that an expanded program is already operating or that a particular patient has coverage.
Sources: HHS — October 27 announcement: https://www.hhs.gov/press-room/fda-cms-advance-gene-therapies-rare-diseases-patient-access.html