Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma.
<h4>Background</h4>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 methodolo...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
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Public Library of Science (PLoS)
2023-01-01
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| Series: | PLoS ONE |
| Online Access: | https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0292239&type=printable |
| _version_ | 1797637514895294464 |
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| author | Huimin Zou Yan Xue Xianwen Chen Yunfeng Lai Dongning Yao Carolina Oi Lam Ung Hao Hu |
| author_facet | Huimin Zou Yan Xue Xianwen Chen Yunfeng Lai Dongning Yao Carolina Oi Lam Ung Hao Hu |
| author_sort | Huimin Zou |
| collection | DOAJ |
| description | <h4>Background</h4>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.<h4>Methods</h4>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.<h4>Results</h4>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.<h4>Conclusions</h4>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. |
| first_indexed | 2024-03-11T12:49:40Z |
| format | Article |
| id | doaj.art-4c2b48d2c4ea4445bb5164295cb831e1 |
| institution | Directory Open Access Journal |
| issn | 1932-6203 |
| language | English |
| last_indexed | 2024-03-11T12:49:40Z |
| publishDate | 2023-01-01 |
| publisher | Public Library of Science (PLoS) |
| record_format | Article |
| series | PLoS ONE |
| spelling | doaj.art-4c2b48d2c4ea4445bb5164295cb831e12023-11-04T05:33:27ZengPublic Library of Science (PLoS)PLoS ONE1932-62032023-01-011810e029223910.1371/journal.pone.0292239Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma.Huimin ZouYan XueXianwen ChenYunfeng LaiDongning YaoCarolina Oi Lam UngHao Hu<h4>Background</h4>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.<h4>Methods</h4>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.<h4>Results</h4>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.<h4>Conclusions</h4>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.https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0292239&type=printable |
| spellingShingle | Huimin Zou Yan Xue Xianwen Chen Yunfeng Lai Dongning Yao Carolina Oi Lam Ung Hao Hu Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma. PLoS ONE |
| title | Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma. |
| title_full | Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma. |
| title_fullStr | Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma. |
| title_full_unstemmed | Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma. |
| title_short | Comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma. |
| title_sort | comparative analysis of disease modelling for health economic evaluations of systemic therapies in advanced hepatocellular carcinoma |
| url | https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0292239&type=printable |
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