State Key Laboratory of Quality Research in Chinese Medicine, Institute of Chinese Medical Sciences, University of Macau, Macao SAR, China.
School of Public Health and Management, Guangzhou University of Chinese Medicine, Guangzhou, China.
PLoS One. 2023 Oct 5;18(10):e0292239. doi: 10.1371/journal.pone.0292239. eCollection 2023.
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.
背景：本研究旨在系统分析系统治疗晚期肝细胞癌（HCC）的基于模型的健康经济评估中使用的方法学和结构假设，讨论现有挑战，并为晚期 HCC 的未来模型制定方法学建议。
方法：我们使用五个数据库（Embase、PubMed、Web of Science、Econlit 和 CNKI）进行文献检索，检索时间截至 2022 年 12 月 4 日。还考虑了来自加拿大、英国、澳大利亚和美国的技术评估。符合纳入标准的是基于英语或中文的系统治疗晚期 HCC 的基于模型的全经济评估。使用 2022 年健康经济评估报告标准清单评估报告质量。
结果：从 12863 条记录中检索到 55 项符合纳入标准。最常用的建模技术是马尔可夫模型（n=29，53%）和分割生存模型（n=27，49%）。大多数研究基于健康状态驱动的结构（n=51，93%），其次是治疗线驱动的结构（n=2，4%）和组合结构（n=1，2%）。只有三项研究（5%）采用外部真实世界数据来外推总生存或校准外推。很少有研究报告转移概率的假设。效用建模方法是基于状态的（n=51，93%）和基于时间到死亡的（n=1，2%）。只有 13 项研究（24%）报告了五种类型的模型验证。特定治疗策略的经济评估结果在研究之间存在差异。
结论：晚期 HCC 系统治疗的健康经济评估中的疾病建模采用了各种建模方法和假设，导致结果存在显著不确定性。通过提出方法学建议，我们建议未来 HCC 治疗的基于模型的健康经济评估研究应遵循良好的建模实践指南，并改进建模方法，以生成可靠的健康和经济证据。
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