The Clinical Trials Mosaic: Toward a Range of Clinical Trials Designs to Optimize Evidence-Based Treatment.
The Clinical Trials Mosaic: Toward a Range of Clinical Trials Designs to Optimize Evidence-Based Treatment.
复制标题
DOI：
10.17505/jpor.2017.03
复制
发表时间：
2017
影响因子：
--
通讯作者：
Cooper R
中科院分区：
文献类型：
--
作者：
Ridenour TA;Chen SK;Liu HY;Bobashev GV;Hill K;Cooper R
将临床试验设计分为概念性(例如，随机临床试验或RCT)和具体(例如，N-of-1或病例研究)，排除了在这些极端之间使用一系列混合设计和潜在的研究问题。这篇文章描述了独特的临床证据，可以使用具体的临床试验(ICTS)来补充随机对照试验数据。建议并在此说明的是，RCT和ICT的设计功能的创新组合可以为临床医生提供更全面的信息，用于测试治疗、进行务实试验和做出基于证据的临床决策。混合模型轨迹分析和统一结构方程建模与多基线设计相结合，在(A)旨在改善严重自闭症相关沟通障碍的真正N-of-1试点研究中，以及(B)两项旨在缓解轮椅不适的免费干预措施的小样本初步研究中。证据支持某些治疗结果的机制，并排除其他机制。效应大小包括平均阶段差异(即有效性)、轨迹斜率和研究阶段之间路径系数的差异。信通技术可以与随机对照试验同等严谨地分析，并产生与随机对照试验相当的效应大小，目的是开发混合设计以增强随机对照试验，用于试点测试创新治疗、对罕见疾病或其他小群体的疗效研究、量化人内过程，以及在许多随机对照试验不可行的情况下进行临床试验。
Dichotomizing clinical trials designs into nomothetic (e.g., randomized clinical trials or RCTs) versus idiographic (e.g., N-of-1 or case studies) precludes use of an array of hybrid designs and potential research questions between these extremes. This paper describes unique clinical evidence that can be garnered using idiographic clinical trials (ICTs) to complement RCT data. Proposed and illustrated herein is that innovative combinations of design features from RCTs and ICTs could provide clinicians with far more comprehensive information for testing treatments, conducting pragmatic trials, and making evidence-based clinical decisions Mixed model trajectory analysis and unified structural equations modeling were coupled with multiple baseline designs in (a) a true N-of-1 pilot study to improve severe autism-related communication deficits and (b) a small sample preliminary study of two complimentary interventions to relieve wheelchair discomfort. Evidence supported certain mechanisms of treatment outcomes and ruled out others. Effect sizes included mean phase differences (i.e., effectiveness), trajectory slopes, and differences in path coefficients between study phases. ICTs can be analyzed with equivalent rigor as, and generate effect sizes comparable to, RCTs for the purpose of developing hybrid designs to augment RCTs for pilot testing innovative treatment, efficacy research on rare diseases or other small populations, quantifying within-person processes, and conducting clinical trials in many situations when RCTs are not feasible.