Kosteneffectiviteit van mavacamten bij obstructieve HCM: gezondheidswinst, maar een hoge prijs

<sec><st>Introduction</st> <p>The clinical management of Obstructive Hypertrophic Cardiomyopathy remains challenging, and the phase III EXPLORER-HCM trial has demonstrated that the novel cardiac myosin inhibitor Mavacamten can significantly improve patients&rsquo; symptomatic and functional status.</p> </sec> <sec><st>Purpose</st> <p>This study aimed to evaluate the cost-effectiveness of the MBC regimen (Mavacamten plus a &beta;-blocker or a calcium-channel blocker) versus the PBC regimen (placebo plus a &beta;-blocker or a calcium-channel blocker) for treating Obstructive Hypertrophic Cardiomyopathy from the perspective of the Chinese Healthcare System.</p> </sec> <sec><st>Methods</st> <p>A Markov model was constructed using data from the EXPLORER-HCM clinical trial. Cost and utility inputs were sourced from public databases and published literature. Total costs, life-years (LY), quality-adjusted life years (QALY) and the incremental cost-effectiveness ratio (ICER) were assessed. One-way deterministic sensitivity analysis and probabilistic sensitivity analysis were conducted to examine the robustness of the model.</p> </sec> <sec><st>Results</st> <p>Compared with the PBC regimen, Mavacamten treatment yielded an ICER of US$50 587.23/QALY. Its incremental cost was US$106 221.41, providing 2.1 additional QALY and 3.15 LY. Sensitivity analyses indicated that the drug price was the key driver influencing changes in the ICER value.</p> </sec> <sec><st>Conclusion</st> <p>This study confirms that Mavacamten provides substantial health benefits to patients, but its relatively high ICER exceeds the commonly accepted willingness to pay.</p> </sec>