Wu Changjin, Hao Jun, Xin Yu, Song Ruomeng, Li Wentan, Zuo Ling, Zhang Xiyan, Cai Yuanyi, Wu Huazhang, Hui Wen
School of Public Health, Chongqing Medical University, Chongqing, China.
Medical Research and Biometrics Centre, National Clinical Research Centre for Cardiovascular Diseases, Fuwai Hospital, National Centre for Cardiovascular Diseases, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China; Department of Clinical Sciences, Liverpool School of Tropical Medicine, Liverpool, UK; Institute for Global Health, University College London, London, UK.
J Clin Epidemiol. 2024 Dec;176:111535. doi: 10.1016/j.jclinepi.2024.111535. Epub 2024 Sep 21.
Economic evaluations based on well-designed and -conducted pragmatic randomized controlled trials (pRCTs) can provide valuable evidence on the cost-effectiveness of interventions, enhancing the relevance and applicability of findings to healthcare decision-making. However, economic evaluation outcomes are seldom taken into consideration during the process of sample size calculation in pragmatic trials. The reporting quality of sample size and information on its calculation in economic evaluations that are well-suited to pRCTs remain unknown. This study aims to assess the reporting quality of sample size and estimate the power values of economic evaluations in pRCTs.
We conducted a cross-sectional survey using data of pRCTs available from PubMed and OVID from 1 January 2010 to 24 April 2022. Two groups of independent reviewers identified articles; three groups of reviewers each extracted the data. Descriptive statistics presented the general characteristics of included studies. Statistical power analyses were performed on clinical and economic outcomes with sufficient data.
The electronic search identified 715 studies and 152 met the inclusion criteria. Of these, 26 were available for power analysis. Only 9 out of 152 trials (5.9%) considered economic outcomes when estimating sample size, and only one adjusted the sample size accordingly. Power values for trial-based economic evaluations and clinical trials ranged from 2.56% to 100% and 3.21%-100%, respectively. Regardless of the perspectives, in 14 out of the 26 studies (53.8%), the power values of economic evaluations for quality-adjusted life years (QALYs) were lower than those of clinical trials for primary endpoints (PEs). In 11 out of the 24 (45.8%) and in 8 out of the 13 (61.5%) studies, power values of economic evaluations for QALYs were lower than those of clinical trials for PEs from the healthcare and societal perspectives, respectively. Power values of economic evaluations for non-QALYs from the healthcare and societal perspectives were potentially higher than those of clinical trials in 3 out of the 4 studies (75%). The power values for economic outcomes in Q1 were not higher than those for other journal impact factor quartile categories.
Theoretically, pragmatic trials with concurrent economic evaluations can provide real-world evidence for healthcare decision makers. However, in pRCT-based economic evaluations, limited consideration, and inadequate reporting of sample-size calculations for economic outcomes could negatively affect the results' reliability and generalisability. We thus recommend that future pragmatic trials with economic evaluations should report how sample sizes are determined or adjusted based on the economic outcomes in their protocols to enhance their transparency and evidence quality.
基于精心设计和实施的实用随机对照试验（pRCT）进行的经济评估可为干预措施的成本效益提供有价值的证据，提高研究结果与医疗保健决策的相关性和适用性。然而，在实用试验的样本量计算过程中，经济评估结果很少被考虑在内。适合pRCT的经济评估中样本量及其计算信息的报告质量仍然未知。本研究旨在评估样本量的报告质量，并估计pRCT中经济评估的检验效能值。
我们使用2010年1月1日至2022年4月24日从PubMed和OVID获取的pRCT数据进行了一项横断面调查。两组独立评审员筛选文章；三组评审员分别提取数据。描述性统计呈现了纳入研究的一般特征。对有足够数据的临床和经济结果进行了统计检验效能分析。
电子检索识别出715项研究，152项符合纳入标准。其中，26项可用于检验效能分析。在152项试验中，只有9项（5.9%）在估计样本量时考虑了经济结果，且只有1项相应地调整了样本量。基于试验的经济评估和临床试验的检验效能值分别为2.56%至100%和3.21%至100%。无论从何种角度来看，在26项研究中的14项（53.8%）中，质量调整生命年（QALY）的经济评估检验效能值低于主要终点（PE）的临床试验检验效能值。在24项研究中的11项（45.8%）以及13项研究中的8项（61.5%）中，从医疗保健和社会角度来看，QALY的经济评估检验效能值分别低于PE的临床试验检验效能值。从医疗保健和社会角度来看，4项研究中的3项（75%）非QALY的经济评估检验效能值可能高于临床试验。Q1中经济结果的检验效能值不高于其他期刊影响因子四分位数类别。
从理论上讲，同时进行经济评估的实用试验可以为医疗保健决策者提供真实世界的证据。然而，在基于pRCT的经济评估中，对经济结果样本量计算的考虑有限且报告不足，可能会对结果的可靠性和可推广性产生负面影响。因此，我们建议未来进行经济评估的实用试验应在其方案中报告如何根据经济结果确定或调整样本量，以提高透明度和证据质量。
