A global model for symptomatic obstructive hypertrophic cardiomyopathy to assess the cost-effectiveness of mavacamten: results from a Dutch societal perspective.
Ektare V, Simons C, et al. • Journal of medical economics • 2026
Mavacamten + BB/CCB is cost-effective compared to BB/CCB monotherapy for symptomatic obstructive HCM in the Netherlands, at €15,961 per QALY gained, well below the €50,000 per QALY threshold.
Key Findings
Results
Mavacamten plus BB/CCB therapy resulted in an incremental discounted gain of 3.09 QALYs compared to BB/CCB monotherapy.
The model used a 5-state Markov model (NYHA classes I–IV, death) with a lifetime horizon.
The model population reflected the EXPLORER-HCM trial intention-to-treat population.
Annual discount rates of 4.00% for costs and 1.50% for health outcomes were applied per Dutch guidelines.
The short-term period was 30 weeks for mavacamten + BB/CCB and 46 weeks for BB/CCB monotherapy.
Results
Mavacamten plus BB/CCB therapy resulted in an incremental discounted gain of 3.17 life-years compared to BB/CCB monotherapy.
Life-years (LYs) were estimated per patient over a lifetime horizon.
The model was stratified into short-term and long-term (post short-term) periods.
Results are reported in 2022/2023 Euros.
Results
Incremental discounted costs of mavacamten plus BB/CCB versus BB/CCB monotherapy were €49,388 over a lifetime.
The additional costs were driven primarily by increased treatment acquisition costs for mavacamten.
Cost increases were partly offset by savings in healthcare resource utilization.
Indirect costs, particularly informal care costs, also contributed to cost offsets.
Costs are expressed in 2022/2023 Euros.
Results
Mavacamten plus BB/CCB was cost-effective at the Dutch willingness-to-pay threshold of €50,000 per QALY, with an incremental cost-utility ratio of €15,961 per QALY gained.
The €50,000 per QALY threshold was used as the Dutch reference for cost-effectiveness.
The incremental cost-utility ratio of €15,961 per QALY is well below the €50,000 threshold.
Results were evaluated from a Dutch societal perspective.
The model was aligned with 2016 Zorginstituut Nederland guidelines.
Results
Deterministic and probabilistic sensitivity and scenario analyses supported the robustness of the model results.
Both deterministic sensitivity analyses and probabilistic sensitivity analyses were conducted.
Scenario analyses were also performed to evaluate robustness.
All analyses supported the base-case finding that mavacamten + BB/CCB is cost-effective versus BB/CCB monotherapy.
Methods
The model incorporated treatment sequencing and was stratified into distinct short-term and long-term periods to capture the clinical trial data and extrapolated long-term outcomes.
Short-term period was 30 weeks for mavacamten + BB/CCB and 46 weeks for BB/CCB monotherapy, reflecting different clinical trial follow-up durations.
Long-term period was defined as the post short-term phase extending to a lifetime horizon.
The model population was based on the EXPLORER-HCM trial intention-to-treat population.
The 5-state Markov model used NYHA functional classes I–IV and death as health states.
What This Means
This research used a mathematical model to compare the costs and health benefits of adding mavacamten (a new type of heart medication called a cardiac myosin inhibitor) to standard drug therapy versus standard drug therapy alone for patients in the Netherlands with a heart condition called symptomatic obstructive hypertrophic cardiomyopathy (HCM). HCM causes the heart muscle to thicken and obstruct blood flow, leading to symptoms like shortness of breath and fatigue. The model followed patients over their entire lifetime and was built to meet Dutch healthcare evaluation standards.
The study found that patients treated with mavacamten in addition to standard medications lived approximately 3.17 years longer on average and gained 3.09 quality-adjusted life years (a measure that accounts for both length and quality of life) compared to those on standard medications alone. Although mavacamten treatment costs an additional €49,388 over a patient's lifetime, these costs were partially reduced by savings in hospital care use and informal caregiving costs. The resulting cost of €15,961 per quality-adjusted life year gained is well below the Dutch healthcare system's threshold of €50,000, meaning mavacamten is considered good value for money in the Netherlands.
This research suggests that mavacamten offers meaningful health benefits for patients with obstructive HCM at a cost that represents reasonable value for the Dutch healthcare system. The findings were consistent across multiple sensitivity and scenario analyses, indicating the conclusions are robust. These results may help inform healthcare decision-makers in the Netherlands when considering whether to reimburse mavacamten for eligible patients.
Ektare V, Simons C, Johannesen K, Krause T, Zema C, Buisman L, et al.. (2026). A global model for symptomatic obstructive hypertrophic cardiomyopathy to assess the cost-effectiveness of mavacamten: results from a Dutch societal perspective.. Journal of medical economics. https://doi.org/10.1080/13696998.2026.2675850