Statistical lessons learned for designing cluster randomized pragmatic clinical trials from the NIH Health Care Systems Collaboratory Biostatistics and Design Core
Statistical lessons learned for designing cluster randomized pragmatic clinical trials from the NIH Health Care Systems Collaboratory Biostatistics and Design Core
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DOI：
10.1177/1740774516646578
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发表时间：
2016-10-01
期刊：
影响因子：
2.7
通讯作者：
Heagerty, Patrick J.
中科院分区：
文献类型：
--
作者：
Cook, Andrea J.;Delong, Elizabeth;Heagerty, Patrick J.
背景/目标：医疗保健系统中的实用临床试验为评估新的干预措施和治疗方法提供了重要机会。最近建立了网络，以支持切实有效的研究。务实的试验将导致改善我们如何提供医疗保健和承诺，更迅速地将研究成果转化为practice.Methods：美国国立卫生研究院（NIH）的医疗保健系统合作实验室成立进行务实的临床试验，并培养跨研究领域和学科的合作，为未来的研究开发最佳实践。通过两个阶段的资助过程，包括试点阶段（UH 2）和主要试验阶段（UH 3），合作实验室的研究人员有机会在启动这些试验之前共同努力改善这些试验的各个方面，并解决实施过程中出现的新问题。创建了七个核心以解决各种考虑因素，包括电子健康记录;表型，数据标准和数据质量;生物统计学和设计核心;患者报告的结局;医疗保健系统相互作用;监管/伦理;和利益相关者参与。本文的目的是总结生物统计学和设计核心在2012年至2014年期间进行的7项实用临床试验的初步试点阶段的经验教训。结果：5项整群随机试验（也称为组随机试验）产生了方法学问题，包括考虑交叉和阶梯楔形设计。我们概述了试验阶段的一般主题和挑战，并提出了解决方案，包括研究设计、随机化单位、样本量和统计分析等主题。我们的研究结果适用于医疗保健系统内进行的其他实用临床试验。结论：使用UH 2/UH 3资助机制的实用临床试验提供了一个机会，确保所有相关设计问题都得到充分考虑，以便可靠有效地评估新的干预措施和治疗方法。只有严格的设计和适当的分析选择成为研究方案的重要组成部分，才能确保试验结果的完整性和普遍性。
Background/aims: Pragmatic clinical trials embedded within health care systems provide an important opportunity to evaluate new interventions and treatments. Networks have recently been developed to support practical and efficient studies. Pragmatic trials will lead to improvements in how we deliver health care and promise to more rapidly translate research findings into practice.Methods: The National Institutes of Health (NIH) Health Care Systems Collaboratory was formed to conduct pragmatic clinical trials and to cultivate collaboration across research areas and disciplines to develop best practices for future studies. Through a two-stage grant process including a pilot phase (UH2) and a main trial phase (UH3), investigators across the Collaboratory had the opportunity to work together to improve all aspects of these trials before they were launched and to address new issues that arose during implementation. Seven Cores were created to address the various considerations, including Electronic Health Records; Phenotypes, Data Standards, and Data Quality; Biostatistics and Design Core; Patient-Reported Outcomes; Health Care Systems Interactions; Regulatory/Ethics; and Stakeholder Engagement. The goal of this article is to summarize the Biostatistics and Design Core's lessons learned during the initial pilot phase with seven pragmatic clinical trials conducted between 2012 and 2014.Results: Methodological issues arose from the five cluster-randomized trials, also called group-randomized trials, including consideration of crossover and stepped wedge designs. We outlined general themes and challenges and proposed solutions from the pilot phase including topics such as study design, unit of randomization, sample size, and statistical analysis. Our findings are applicable to other pragmatic clinical trials conducted within health care systems.Conclusion: Pragmatic clinical trials using the UH2/UH3 funding mechanism provide an opportunity to ensure that all relevant design issues have been fully considered in order to reliably and efficiently evaluate new interventions and treatments. The integrity and generalizability of trial results can only be ensured if rigorous designs and appropriate analysis choices are an essential part of their research protocols.