A global model for symptomatic obstructive hypertrophic cardiomyopathy to assess the cost-effectiveness of mavacamten: results from a Dutch societal perspective

OBJECTIVES: To explore the cost-effectiveness of mavacamten + beta-blocker/calcium channel blocker therapy (BB/CCB) versus BB/CCB monotherapy for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (HCM). MATERIALS AND METHODS: A 5-state Markov model (New York Heart Association classes I-IV, death) that included treatment sequencing was developed. It used a Dutch societal perspective and lifetime horizon stratified into short-term (mavacamten + BB/CCB: 30 weeks; BB/CCB: 46 weeks) and long-term (i.e. post short-term) periods. The model population reflected the EXPLORER-HCM trial intention-to-treat population. Model parameters were aligned with 2016 Zorginstituut Nederland guidelines, including annual discount rates of 4.00% and 1.50% for costs and health outcomes. Costs (2022/2023 Euros), life-years (LYs) and quality-adjusted LYs (QALYs) per patient, incremental costs and LYs/QALYs, and incremental cost-utility ratios were estimated. Sensitivity and scenario analyses were conducted to evaluate the robustness of the results. RESULTS: Treatment with mavacamten + BB/CCB resulted in an incremental discounted gain of 3.09 QALYs and 3.17 LYs versus the BB/CCB monotherapy strategy. Incremental discounted costs were €49,388 over a lifetime; the additional costs of mavacamten were driven by increased treatment acquisition costs but partly offset by savings in healthcare resource utilization and indirect costs, particularly informal care costs. Mavacamten + BB/CCB was cost-effective at a €50,000 per QALY threshold versus BB/CCB monotherapy at €15,961 per QALY gain. The deterministic and probabilistic sensitivity and scenario analyses supported the robustness of the model results. CONCLUSIONS: In the Netherlands, mavacamten + BB/CCB is a cost-effective treatment strategy for symptomatic obstructive HCM compared to BB/CCB monotherapy.