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Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma

Author

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  • Huimin Zou
  • Yan Xue
  • Xianwen Chen
  • Yunfeng Lai
  • Dongning Yao
  • Carolina Oi Lam Ung
  • Hao Hu

Abstract

Background: The objective of this study was to systematically analyse methodological and structural assumptions utilised in model-based health economic evaluations of systemic advanced hepatocellular carcinoma (HCC) therapies, discuss the existing challenges, and develop methodological recommendations for future models in advanced HCC. Methods: We performed literature searches using five databases (Embase, PubMed, Web of Science, Econlit, and CNKI) up to December 4, 2022. Technology appraisals from Canada, England, Australia, and the United States were also considered. Model-based full economic evaluations of systemic advanced HCC therapies in English or Chinese met the eligibility criteria. The reporting quality was assessed by using the Consolidated Health Economic Evaluation Reporting Standards 2022 checklist. Results: Of 12,863 records retrieved, 55 were eligible for inclusion. Markov model (n = 29, 53%) and partitioned survival model (n = 27, 49%) were the most commonly used modelling techniques. Most studies were based on health-state-driven structure (n = 51, 93%), followed by treatment-line-driven structure (n = 2, 4%) and combination structure (n = 1, 2%). Only three studies (5%) adopted external real-world data to extrapolate the overall survival or calibrate the extrapolation. Few studies reported the assumptions of transition probabilities. Utility modelling approaches were state-based (n = 51, 93%) and time-to-death (n = 1, 2%). Only 13 studies (24%) reported five types of model validation. Economic evaluation results of specific treatment strategies varied among studies. Conclusions: Disease modelling for health economic evaluations of systemic therapies in advanced HCC has adopted various modelling approaches and assumptions, leading to marked uncertainties in results. By proposing methodological recommendations, we suggest that future model-based studies for health economic evaluation of HCC therapies should follow good modelling practice guidelines and improve modelling methods to generate reliable health and economic evidence.

Suggested Citation

  • Huimin Zou & Yan Xue & Xianwen Chen & Yunfeng Lai & Dongning Yao & Carolina Oi Lam Ung & Hao Hu, 2023. "Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma," PLOS ONE, Public Library of Science, vol. 18(10), pages 1-35, October.
    • Handle: RePEc:plo:pone00:0292239
    DOI: 10.1371/journal.pone.0292239
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    References listed on IDEAS

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    1. Amir Shlomai & Moshe Leshno & Daniel A Goldstein, 2018. "Regorafenib treatment for patients with hepatocellular carcinoma who progressed on sorafenib—A cost-effectiveness analysis," PLOS ONE, Public Library of Science, vol. 13(11), pages 1-11, November.
    2. Kunichika Matsumoto & Yinghui Wu & Takefumi Kitazawa & Shigeru Fujita & Kanako Seto & Tomonori Hasegawa, 2018. "Cost of illness of hepatocellular carcinoma in Japan: A time trend and future projections," PLOS ONE, Public Library of Science, vol. 13(6), pages 1-12, June.
    3. P. Vemer & I. Corro Ramos & G. A. K. Voorn & M. J. Al & T. L. Feenstra, 2016. "AdViSHE: A Validation-Assessment Tool of Health-Economic Models for Decision Makers and Model Users," PharmacoEconomics, Springer, vol. 34(4), pages 349-361, April.
    4. Alessandro Liberati & Douglas G Altman & Jennifer Tetzlaff & Cynthia Mulrow & Peter C Gøtzsche & John P A Ioannidis & Mike Clarke & P J Devereaux & Jos Kleijnen & David Moher, 2009. "The PRISMA Statement for Reporting Systematic Reviews and Meta-Analyses of Studies That Evaluate Health Care Interventions: Explanation and Elaboration," PLOS Medicine, Public Library of Science, vol. 6(7), pages 1-28, July.
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