Key result
Mavacamten in combination with standard of care was associated with a gain of 1.52 QALYs and an incremental cost of €122,651 compared to standard of care alone, resulting in an ICUR of €80,799 per QALY gained.
Why the study?
The cost-effectiveness of adding mavacamten to standard of care for obstructive hypertrophic cardiomyopathy with persistent symptoms needed to be evaluated from a French collective perspective.
Does mavacamten in combination with standard of care improve cost-effectiveness in adult patients with symptomatic obstructive hypertrophic cardiomyopathy compared to standard of care alone?
Does mavacamten in combination with standard of care improve cost-effectiveness in adult patients with symptomatic obstructive hypertrophic cardiomyopathy compared to standard of care alone?
Effect estimate: ICUR €80,799/QALY
Absolute Event Rate: 14.9% vs 13.38%
Mavacamten added to standard of care is a cost-effective strategy for symptomatic obstructive hypertrophic cardiomyopathy in France, with an ICUR of €80,799/QALY.
May support mavacamten consideration in France if ICUR thresholds allow; leaves open real-world validation and generalizability.
BACKGROUND: The 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. METHODS: A 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. RESULTS: Mavacamten 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. CONCLUSION: On a public list price basis, mavacamten in combination with SoC is a cost-effective strategy for symptomatic obstructive HCM compared to SoC alone.
No takes yet. Share an insight, caveat, or question.
Cotté et al. (2025) studied Symptomatic obstructive hypertrophic cardiomyopathy. Mavacamten in combination with standard of care vs. Standard of care alone was evaluated on Incremental cost-utility ratio (ICUR) (ICUR €80,799/QALY). Mavacamten in combination with standard of care was associated with a gain of 1.52 QALYs and an incremental cost of €122,651 compared to standard of care alone, resulting in an ICUR of €80,799 per QALY gained.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: