Cost-effectiveness analysis of mavacamten for obstructive hypertrophic cardiomyopathy: a perspective from the Chinese healthcare system.

INTRODUCTION

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' symptomatic and functional status.

PURPOSE: This study aimed to evaluate the cost-effectiveness of the MBC regimen (Mavacamten plus a β-blocker or a calcium-channel blocker) versus the PBC regimen (placebo plus a β-blocker or a calcium-channel blocker) for treating Obstructive Hypertrophic Cardiomyopathy from the perspective of the Chinese Healthcare System.

METHODS

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.

RESULTS

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.

CONCLUSION

This study confirms that Mavacamten provides substantial health benefits to patients, but its relatively high ICER exceeds the commonly accepted willingness to pay.