The George Institute for Global Health, University of New South Wales, Sydney, NSW, 2042, Australia.
Faculty of Medicine and Health, School of Public Health, Edward Ford Building A27, University of Sydney, Sydney, NSW, 2006, Australia.
Pharmacoeconomics. 2021 Jan;39(1):63-80. doi: 10.1007/s40273-020-00963-x. Epub 2020 Oct 5.
Recently, there has been an increase in use of the stepped wedge trial (SWT) design in the context of health services research, due to its pragmatic and methodological advantages over the parallel group design.
Our objective was to summarise the statistical methods used when conducting economic evaluations alongside SWTs.
A systematic literature search extending to February 2020 was conducted in the PubMed, Scopus, Cochrane and National Health Service Economic Evaluation Database (NHS-EED) databases to find and evaluate studies where there was an intention to conduct an economic evaluation alongside an SWT. Studies were assessed for their eligibility, findings, reporting of statistical methods and quality of reporting.
Of the 586 studies retrieved from the literature search, 69 studies were identified and included in this systematic review. A total of 54 studies were published protocols, with eight economic evaluations and seven studies reporting full trial results. Included studies varied in terms of their reporting of statistical methods, in both detail and methodology. There were 34 studies that did not report any statistical methods for the economic evaluation, and only 16 studies reported appropriate methods, mainly using some form of mixed/multilevel model, and two used seemingly unrelated regression. Twelve studies reported the use of generic bootstrap methods and other modelling techniques, whilst the remaining studies failed to appropriately account for clustering, correlation or adjustment for time.
The use of appropriate statistical methods that account for time, clustering and correlation between costs and outcomes is an important part of SWT health economics analysis, one that will benefit from an effort to communicate the methods available and their performance.
由于实用和方法学上的优势,阶梯式随机临床试验(SWT)在卫生服务研究中的应用近来有所增加,已经超越了平行组设计。
我们的目的是总结在进行 SWT 时进行经济评估所使用的统计方法。
我们对 PubMed、Scopus、Cochrane 和英国国家卫生服务经济评价数据库(NHS-EED)进行了截至 2020 年 2 月的系统文献检索,以查找并评估有意图在进行 SWT 的同时进行经济评估的研究。我们评估了这些研究的合格性、结果、统计方法报告和报告质量。
从文献检索中检索到的 586 项研究中,有 69 项研究被确定并纳入本系统评价。其中,共有 54 项研究为发表的方案,8 项经济评估和 7 项研究报告了完整的试验结果。纳入的研究在统计方法的报告方面存在差异,无论是在细节还是方法学方面。有 34 项研究未报告任何经济评估的统计方法,只有 16 项研究报告了适当的方法,主要使用某种形式的混合/多级模型,有两项研究使用了看似不相关的回归。12 项研究报告了使用通用自举方法和其他建模技术,而其余研究未能适当考虑成本和结果之间的聚类、相关性或调整。
使用适当的统计方法来考虑时间、成本和结果之间的聚类和相关性是 SWT 健康经济学分析的重要组成部分,这将得益于努力交流可用方法及其性能。