Epstein Leonard H, Bickel Warren K, Czajkowski Susan M, Paluch Rocco A, Moeyaert Mariola, Davidson Karina W
Department of Pediatrics.
Fralin Biomedical Research Institute.
Health Psychol. 2021 Dec;40(12):858-874. doi: 10.1037/hea0001055. Epub 2021 Aug 9.
The biomedical research community has long recognized that much of the basic research being conducted, whether in the biological, behavioral or social sciences, is not readily translated into clinical and public health applications. This translational gap is due in part to challenges inherent in moving research findings from basic or discovery research to applied research that addresses clinical or public health problems. In the behavioral and social sciences, research designs typically used in the early phases of translational research are small, underpowered "pilot" studies that may lack sufficient statistical power to test the research question of interest. While this approach is discouraged, these studies are often employed to estimate effect sizes before embarking on a larger trial with adequate statistical power to test the research hypothesis. The goal of this paper is to provide an alternative approach to early phase studies using single case designs (SCDs).
Review basic principles of SCDs; provide a series of hypothetical SCD replication experiments to illustrate (1) how data from SCDs can be analyzed to test the effects of an intervention on behavioral and biological outcomes and (2) how sample sizes can be derived for larger randomized controlled trials (RCTs) based on clinically meaningful effects from SCDs; and review feedback between SCDs and RCTs.
The paper illustrates the use of SCD reversal and multiple baseline designs for early phase translational research.
SCDs provide a flexible and efficient platform for the use of experimental methods in early phase translational research. (PsycInfo Database Record (c) 2022 APA, all rights reserved).
生物医学研究界早就认识到，正在进行的许多基础研究，无论是在生物科学、行为科学还是社会科学领域，都不容易转化为临床和公共卫生应用。这种转化差距部分归因于将研究结果从基础研究或探索性研究转化为解决临床或公共卫生问题的应用研究时所固有的挑战。在行为科学和社会科学中，转化研究早期阶段通常使用的研究设计是规模小、效力不足的“试点”研究，可能缺乏足够的统计效力来检验感兴趣的研究问题。虽然这种方法不被提倡，但这些研究通常用于在开展具有足够统计效力以检验研究假设的更大规模试验之前估计效应大小。本文的目的是提供一种使用单病例设计（SCD）进行早期阶段研究的替代方法。
回顾单病例设计的基本原理；提供一系列假设的单病例设计重复实验，以说明（1）如何分析单病例设计的数据来检验干预对行为和生物学结果的影响，以及（2）如何根据单病例设计具有临床意义的效应为更大规模的随机对照试验（RCT）确定样本量；并回顾单病例设计与随机对照试验之间的反馈。
本文阐述了单病例设计的反转和多基线设计在早期转化研究中的应用。
单病例设计为早期转化研究中使用实验方法提供了一个灵活且高效的平台。（PsycInfo数据库记录（c）2022美国心理学会，保留所有权利）
