Cost-effectiveness of Mavacamten for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy using a French healthcare perspective.

backgroundThe objective of this study was to assess the cost-effectiveness of mavacamten in combination with the standard of care (SoC), composed of beta-blockers and calcium channel blocker monotherapy, compared to the SoC alone in the treatment of patients with obstructive hypertrophic cardiomyopathy with persistent symptoms, from a French collective perspective.

methodsA four state Markov model defined by NYHA classification levels I to III/IV and death was developed to simulate the evolution of disease severity over a lifetime horizon. Clinical inputs and utilities were derived from EXPLORER-HCM trial. According to HAS recommendations, costs, life-years (LYs) and quality-adjusted LYs (QALYs) per patient, incremental costs and LYs/QALYs, incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) were estimated. Model uncertainty was assessed through deterministic, probabilistic sensitivity analyses and scenario analyses.

resultsMavacamten in combination with SoC was associated with a QALY gain of 1.52, driven by an improved NYHA distribution of mavacamten patients. Incremental discounted costs were €122,651, owing to increased treatment acquisition costs, partly offset by savings in healthcare resource utilization, resulting in an ICUR of €80,799/QALY. The sensitivity analyses showed the results of the model were relatively robust. The probabilistic sensitivity analysis confirmed base-case ICUR, with 80% probability of mavacamten being cost effective at a willingness-to-pay of €93,750/QALY. The scenario analysis simulating a limited societal perspective highlighted the potential benefit of mavacamten treatment on productivity loss costs.

conclusionOn a public list price basis, mavacamten in combination with SoC is a cost-effective strategy for symptomatic obstructive HCM compared to SoC alone.