Ye Fangshu, Wang Chong, O'Connor Annette M
Department of Statistics, Iowa State University, Ames, Iowa, United States of America.
Department of Veterinary Diagnostics and Production Animal Medicine, Iowa State University, Ames, Iowa, United States of America.
PLoS One. 2023 Dec 21;18(12):e0296020. doi: 10.1371/journal.pone.0296020. eCollection 2023.
Randomized clinical trials (RCTs) are designed for measuring the effectiveness of the treatments and testing a hypothesis regarding the relative effect between two or more treatments. Trial designers are often interested in maximizing power when the total sample size is fixed or minimizing the required total sample size to reach a pre-specified power. One approach to maximizing power proposed by previous researchers is to leverage prior evidence using meta-analysis (NMA) to inform the sample size determination of a new trial. For example, researchers may be interested in designing a two-arm trial comparing treatments A and B which are already in the existing trial network but do not have any direct comparison. The researchers' intention is to incorporate the result into an existing network for meta-analysis. Here we develop formulas to address these options and use simulations to validate our formula and evaluate the performance of different analysis methods in terms of power. We also implement our proposed method into the R package OssaNMA and publish an R Shiny app for the convenience of the application. The goal of the package is to enable researchers to readily adopt the proposed approach which can improve the power of an RCT and is therefore resource-saving. In the R Shiny app, We also provide the option to include the cost of each treatment which would enable researchers to compare the total treatment cost associated with each design and analysis approach. Further, we explore the effect of allocation to treatment group on study power when the a priori plan is to incorporate the new trial result into an existing network for meta-analysis.
随机临床试验(RCT)旨在衡量治疗方法的有效性,并检验关于两种或多种治疗方法相对效果的假设。当总样本量固定时,试验设计者通常希望最大化检验效能,或者在达到预先设定的检验效能时最小化所需的总样本量。先前研究人员提出的一种最大化检验效能的方法是利用荟萃分析(NMA)的先验证据来指导新试验的样本量确定。例如,研究人员可能有兴趣设计一项双臂试验,比较已经存在于现有试验网络中但没有任何直接比较的治疗方法A和B。研究人员的意图是将结果纳入现有的网络进行荟萃分析。在这里,我们开发了公式来处理这些选项,并使用模拟来验证我们的公式,并在检验效能方面评估不同分析方法的性能。我们还将我们提出的方法实现到R包OssaNMA中,并发布了一个R Shiny应用程序以方便应用。该包的目标是使研究人员能够轻松采用所提出的方法,该方法可以提高RCT的检验效能,因此节省资源。在R Shiny应用程序中,我们还提供了纳入每种治疗成本的选项,这将使研究人员能够比较与每种设计和分析方法相关的总治疗成本。此外,当预先计划将新试验结果纳入现有的荟萃分析网络时,我们探讨了治疗组分配对研究检验效能的影响。