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

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

doi:10.1371/journal.pone.0292239

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

PLoS One. 2023 Oct 5;18(10):e0292239. doi: 10.1371/journal.pone.0292239. eCollection 2023.

Authors

Huimin Zou¹, Yan Xue¹, Xianwen Chen¹, Yunfeng Lai², Dongning Yao³, Carolina Oi Lam Ung^{1

4

5}, Hao Hu^{1

4

5}

Affiliations

¹ State Key Laboratory of Quality Research in Chinese Medicine, Institute of Chinese Medical Sciences, University of Macau, Macao SAR, China.
² School of Public Health and Management, Guangzhou University of Chinese Medicine, Guangzhou, China.
³ Department of Drug Regulatory Science and Pharmacoeconomics, School of Pharmacy, Nanjing Medical University, Nanjing, China.
⁴ Centre for Pharmaceutical Regulatory Sciences, University of Macau, Macao SAR, China.
⁵ Department of Public Health and Medicinal Administration, Faculty of Health Sciences, University of Macau, Macao SAR, China.

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.

Copyright: © 2023 Zou et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Australia
Canada
Carcinoma, Hepatocellular* / therapy
Cost-Benefit Analysis
Humans
Liver Neoplasms* / therapy

Grants and funding

This research is supported by the funding of the Science and Technology Development Fund, Macau SAR (SKL-QRCM(UM)-2023-2025) and the University of Macau (MYRG2022-00113-ICMS). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.