He Zhe, Gonzalez-Izquierdo Arturo, Denaxas Spiros, Sura Andrei, Guo Yi, Hogan William R, Shenkman Elizabeth, Bian Jiang
Florida State University, Tallahassee, FL, USA.
University of College London, London, UK.
AMIA Annu Symp Proc. 2018 Apr 16;2017:849-858. eCollection 2017.
Clinical trials are indispensable tools for evidence-based medicine. However, they are often criticized for poor generalizability. Traditional trial generalizability assessment can only be done after the trial results are published, which compares the enrolled patients with a convenience sample of real-world patients. However, the proliferation of electronic data in clinical trial registries and clinical data warehouses offer a great opportunity to assess the generalizability during the design phase of a new trial. In this work, we compared and contrasted a priori (based on eligibility criteria) and a posteriori (based on enrolled patients) generalizability of Type 2 diabetes clinical trials. Further, we showed that comparing the study population selected by the clinical trial eligibility criteria to the real-world patient population is a good indicator of the generalizability of trials. Our findings demonstrate that the a priori generalizability of a trial is comparable to its a posteriori generalizability in identifying restrictive quantitative eligibility criteria.
临床试验是循证医学不可或缺的工具。然而，它们常常因普遍适用性差而受到批评。传统的试验普遍适用性评估只能在试验结果发表后进行，即把入组患者与一个方便抽样的真实世界患者样本进行比较。然而，临床试验注册库和临床数据仓库中电子数据的激增，为在新试验的设计阶段评估普遍适用性提供了一个绝佳机会。在这项工作中，我们比较并对比了2型糖尿病临床试验的先验（基于入选标准）和后验（基于入组患者）普遍适用性。此外，我们表明，将临床试验入选标准所选择的研究人群与真实世界患者人群进行比较，是试验普遍适用性的一个良好指标。我们的研究结果表明，在识别限制性定量入选标准方面，试验的先验普遍适用性与其后验普遍适用性相当。
