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解决经济评价中患者异质性问题的进展:方法文献综述。

Advances in Addressing Patient Heterogeneity in Economic Evaluation: A Review of the Methods Literature.

机构信息

Manchester Centre for Health Economics, Division of Population Health, Health Services Research, and Primary Care, School of Health Sciences, Faculty of Biology, Medicine and Health, Manchester Centre for Health Economics, University of Manchester, Manchester, UK.

Social Care and Society, Division of Population Health, Health Services Research, and Primary Care, School of Health Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.

出版信息

Pharmacoeconomics. 2024 Jul;42(7):737-749. doi: 10.1007/s40273-024-01377-9. Epub 2024 Apr 27.


DOI:10.1007/s40273-024-01377-9
PMID:38676871
Abstract

Cost-effectiveness analyses commonly use population or sample averages, which can mask key differences across subgroups and may lead to suboptimal resource allocation. Despite there being several new methods developed over the last decade, there is no recent summary of what methods are available to researchers. This review sought to identify advances in methods for addressing patient heterogeneity in economic evaluations and to provide an overview of these methods. A literature search was conducted using the Econlit, Embase and MEDLINE databases to identify studies published after 2011 (date of a previous review on this topic). Eligible studies needed to have an explicit methodological focus, related to how patient heterogeneity can be accounted for within a full economic evaluation. Sixteen studies were included in the review. Methodologies were varied and included regression techniques, model design and value of information analysis. Recent publications have applied methodologies more commonly used in other fields, such as machine learning and causal forests. Commonly noted challenges associated with considering patient heterogeneity included data availability (e.g., sample size), statistical issues (e.g., risk of false positives) and practical factors (e.g., computation time). A range of methods are available to address patient heterogeneity in economic evaluation, with relevant methods differing according to research question, scope of the economic evaluation and data availability. Researchers need to be aware of the challenges associated with addressing patient heterogeneity (e.g., data availability) to ensure findings are meaningful and robust. Future research is needed to assess whether and how methods are being applied in practice.

摘要

成本效益分析通常使用人群或样本平均值,这可能掩盖了亚组之间的关键差异,并可能导致资源分配不理想。尽管在过去十年中开发了几种新方法,但对于研究人员来说,可用的方法并没有最近的总结。本综述旨在确定在经济评估中解决患者异质性的方法的进展,并概述这些方法。使用 Econlit、Embase 和 MEDLINE 数据库进行文献检索,以确定 2011 年后(之前关于该主题的综述日期)发表的研究。符合条件的研究需要有明确的方法学重点,涉及如何在全面经济评估中考虑患者异质性。本综述纳入了 16 项研究。方法多种多样,包括回归技术、模型设计和信息价值分析。最近的出版物应用了更常用于其他领域的方法,如机器学习和因果森林。与考虑患者异质性相关的常见挑战包括数据可用性(例如样本量)、统计问题(例如假阳性风险)和实际因素(例如计算时间)。有一系列方法可用于解决经济评估中的患者异质性,相关方法根据研究问题、经济评估的范围和数据可用性而有所不同。研究人员需要意识到解决患者异质性相关的挑战(例如数据可用性),以确保研究结果有意义且稳健。需要进一步研究评估方法在实践中的应用情况和适用性。

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[1]
A Review of Heterogeneity in Comparative Economic Analysis, with Specific Considerations for the Decentralized US Setting and Patient-Centered Care.

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本文引用的文献

[1]
Extensions of Health Economic Evaluations in R for Microsoft Excel Users: A Tutorial for Incorporating Heterogeneity and Conducting Value of Information Analyses.

Pharmacoeconomics. 2023-1

[2]
US cancer drug prices do not reflect benefits to patients.

Lancet Oncol. 2022-12

[3]
Real-World Evidence-Current Developments and Perspectives.

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[4]
An efficient approach for optimizing the cost-effective individualized treatment rule using conditional random forest.

Stat Methods Med Res. 2022-11

[5]
Tools for the Economic Evaluation of Precision Medicine: A Scoping Review of Frameworks for Valuing Heterogeneity-Informed Decisions.

Pharmacoeconomics. 2022-10

[6]
Developing Patient-Centered Real-World Evidence: Emerging Methods Recommendations From a Consensus Process.

Value Health. 2023-1

[7]
Machine Learning Methods in Health Economics and Outcomes Research-The PALISADE Checklist: A Good Practices Report of an ISPOR Task Force.

Value Health. 2022-7

[8]
Local Instrumental Variable Methods to Address Confounding and Heterogeneity when Using Electronic Health Records: An Application to Emergency Surgery.

Med Decis Making. 2022-11

[9]
Factors Limiting Subgroup Analysis in Cost-Effectiveness Analysis and a Call for Transparency.

Pharmacoeconomics. 2022-2

[10]
Cost-effectiveness of the fixed-dose combination tiotropium/olodaterol versus tiotropium monotherapy or a fixed-dose combination of long-acting β2-agonist/inhaled corticosteroid for COPD in Finland, Sweden and the Netherlands: a model-based study.

BMJ Open. 2021-8-4

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