Department of Health Sciences, Faculty of Science, Vrije Universiteit Amsterdam, Amsterdam Public Health Research Institute, Amsterdam, the Netherlands.
Department of Health Sciences, Faculty of Science, Vrije Universiteit Amsterdam, Amsterdam Movement Sciences Research Institute, Amsterdam, the Netherlands.
Health Econ. 2022 Dec;31(12):2680-2699. doi: 10.1002/hec.4603. Epub 2022 Sep 11.
The statistical quality of trial-based economic evaluations is often suboptimal, while a comprehensive overview of available statistical methods is lacking. Therefore, this review summarized and critically appraised available statistical methods for trial-based economic evaluations. A literature search was performed to identify studies on statistical methods for dealing with baseline imbalances, skewed costs and/or effects, correlated costs and effects, clustered data, longitudinal data, missing data and censoring in trial-based economic evaluations. Data was extracted on the statistical methods described, their advantages, disadvantages, relative performance and recommendations of the study. Sixty-eight studies were included. Of them, 27 (40%) assessed methods for baseline imbalances, 39 (57%) assessed methods for skewed costs and/or effects, 27 (40%) assessed methods for correlated costs and effects, 18 (26%) assessed methods for clustered data, 7 (10%) assessed methods for longitudinal data, 26 (38%) assessed methods for missing data and 10 (15%) assessed methods for censoring. All identified methods were narratively described. This review provides a comprehensive overview of available statistical methods for dealing with the most common statistical complexities in trial-based economic evaluations. Herewith, it can provide valuable input for researchers when deciding which statistical methods to use in a trial-based economic evaluation.
基于试验的经济学评价的统计质量往往不尽如人意,而缺乏对现有统计方法的全面概述。因此,本综述总结并批判性地评价了现有的用于基于试验的经济学评价的统计方法。通过文献检索,确定了处理基线不均衡、偏态成本和/或效果、相关成本和效果、聚类数据、纵向数据、缺失数据和删失的基于试验的经济学评价中统计方法的研究。提取了研究中描述的统计方法、它们的优点、缺点、相对性能和建议的数据。共纳入 68 项研究。其中,27 项(40%)评估了处理基线不均衡的方法,39 项(57%)评估了处理偏态成本和/或效果的方法,27 项(40%)评估了处理相关成本和效果的方法,18 项(26%)评估了处理聚类数据的方法,7 项(10%)评估了处理纵向数据的方法,26 项(38%)评估了处理缺失数据的方法,10 项(15%)评估了处理删失的方法。所有确定的方法都进行了叙述性描述。本综述全面概述了处理基于试验的经济学评价中最常见统计复杂性的现有统计方法。因此,它可以为研究人员在决定基于试验的经济学评价中使用哪种统计方法时提供有价值的参考。