Abstract
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.