BACKGROUND: Genetic
therapies are a promising treatment for children born with
spinal muscular atrophy (SMA); however, their high price tags can evoke coverage restrictions. OBJECTIVE: To assess variation in coverage guidelines across fee-for-service state Medicaid programs for 2 novel genetic therapies,
nusinersen and
onasemnogene abeparvovec, that treat SMA. We also assessed the association of these coverage guidelines with use of the 2 genetic therapies. METHODS: We evaluated fee-for-service Medicaid coverage policies for
nusinersen and
onasemnogene abeparvovec from publicly available websites for the period February 2020-March 2020. We then documented areas of agreement and disagreement across 4 key coverage domains. We used 2018 and 2019 state Medicaid drug utilization data to calculate the use of
nusinersen across Medicaid programs and assessed that use against the restrictiveness of the coverage guidelines. RESULTS: We identified 19 state Medicaid coverage guidelines for
nusinersen. Most states agreed on diagnostics requirements; however, there were disagreements based on
ventilator status. We identified 17 state Medicaid coverage guidelines for
onasemnogene abeparvovec. There was more discordance in these coverage guidelines compared with
nusinersen, notably in domains of SMN2 gene count and
ventilator status. When comparing utilization of
nusinersen with coverage restrictions, we found that the more restrictive states had considerably lower utilization of
nusinersen. CONCLUSIONS: There was significant variation across fee-for-service Medicaid coverage policies for
nusinersen and
onasemnogene abeparvovec. Although states can impose individual coverage guidelines for each
drug, we presented policy options that could reduce variation and potentially decrease the cost burden of these drugs. DISCLOSURES: This study was funded by Arnold Ventures. The authors have no conflicts of interest to disclose.