ORIGINAL RESEARCH article
Front. Pharmacol.
Sec. Translational Pharmacology
Volume 16 - 2025 | doi: 10.3389/fphar.2025.1636732
Net Health Benefit of Mavacamten for the Treatment of Chinese Patients with Obstructive Hypertrophic Cardiomyopathy: A Model-based Economic Evaluation
Provisionally accepted
- 1Sun Yat-sen University, Guangzhou, China
- 2Bristol Myers Squibb, Shanghai, China
- 3Bristol Myers Squibb, Uxbridge, United Kingdom
- 4OPEN Health HEOR & Market Access, London, United Kingdom
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Objective: Obstructive hypertrophic cardiomyopathy (HCM) is a cardiovascular disorder characterized by significant symptom burden. This study aims to evaluate the net clinical benefit of mavacamten (a first-in-class cardiac myosin inhibitor) ± beta-blockers/calcium channel blockers (BB/CCBs) compared to BB/CCB monotherapy for Chinese patients with obstructive HCM. Methods: A five-state Markov model (NYHA classes I–IV and death) was developed from a Chinese healthcare system perspective. Patients were initialised in NYHA II or III states, mirroring the baseline distribution in the EXPLORER-CN trial (NCT05174416). State transitions were simulated using cycle-specific probabilities derived from trial data and validated extrapolation assumptions. All-cause mortality risks incorporated disease-specific and extra surgical mortality rates. Treatment sequencing rules reflected escalation pathways (e.g., non-response, adverse events, or eligibility for septural reduction therapy [SRT]), informed by Chinese clinical practice. Health utilities were mapped algorithmically from EXPLORER-CN patient-level EQ-5D responses. Results: Over a lifetime horizon, mavacamten ± BB/CCBs demonstrated superior health outcomes versus BB/CCB monotherapy, with incremental gains in life-years (15.76 vs. 14.40) and quality-adjusted life-years (QALYs: 13.69 vs. 11.38). Conclusions: Mavacamten provides substantial health benefits for Chinese obstructive HCM patients, including clinically meaningful improvements in survival and quality-of-life metrics relative to standard care.
Keywords: Net health benefits, Obstructive HCM, Hypertrophic Cardiomyopathy, economic evaluation, Mavacamten
Received: 28 May 2025; Accepted: 14 Oct 2025.
Copyright: © 2025 Bao, Jia, Wang, Hurst, Cheah, Ho and Xuan. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Han Bao, baoh9@mail.sysu.edu.cn
Jianwei Xuan, xuanjw3@sysu.edu.cn
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.