Pragmatic Clinical Trials: A Randomized Trial in Real-World Studies

doi:10.3969/j.issn.1673-5765.2022.12.003

Abstract

Abstract: Pragmatic clinical trials (PCT), as a important component of real-world research, attracted more and more attention in clinical study design. However, there exists some confusions in understanding and application of PCT. Therefore, this article provided technical specifications of PCT in terms of the concept, study planning and design, purpose, design types and core features, to make readers have a comprehensive and correct understanding of PCT; in addition, the application scope, basic statistical analysis principles, analysis requirements for various types of study design, and the report standards of PCT were also elaborated.

Key words: Pragmatic clinical trial; Real-world study; Clinical trial; Study design

摘要： 实效性临床试验（pragmatic clinical trials，PCT）作为真实世界研究的重要构成，在临床研究设计中越来越受到关注和重视。然而，目前对PCT的理解和应用仍存在的一些误区。因此，本文首先从PCT的概念、研究策划与设计、研究目的、常见的设计类型和核心特征等方面进行阐述，使读者全面和正确地认识PCT。其次，本文详述了PCT的使用范围、基本的统计分析原则与各类型试验设计的分析要求。最后，本文明确了此类研究的报告规范。

关键词: 实效性临床试验; 真实世界研究; 临床试验; 研究设计

ZHANG Yanli, JIN Aoming, PAN Yuesong. Pragmatic Clinical Trials: A Randomized Trial in Real-World Studies[J]. Chinese Journal of Stroke, 2022, 17(12): 1299-1303.

张艳丽, 金奥铭, 潘岳松. 实效性临床试验：真实世界研究的随机化试验[J]. 中国卒中杂志, 2022, 17(12): 1299-1303.

References

[1] SUN X，TAN J，TANG L. Real world evidence：experience and lessons from China[J/OL]. BMJ，2018，360：j5262[2022-10-10]. https://doi.org/10.1136/bmj.j5262.
[2] 吕志杰，吴丽，李秋爽，等. 中国实效性随机对照试验研究现状[J]. 中国医院统计，2021，28（6）：568-572，576.
[3] 青雪梅，房繄恭，刘保延，等. 实用性随机对照试验及其方法学特征思考[J]. 北京中医药大学学报，2008，31（1）：14-18.
[4] 孙鑫，谭婧，唐立，等. 重新认识真实世界研究[J]. 中国循证医学杂志，2017，17（2）：126-130.
[5] BALAS E A，BOREN S A. Managing clinical knowledge for health care improvement[J]. Yearb Med Inform，2000，9（1）：65-70.
[6] SCHWARTZ D，LELLOUCH J. Explanatory and pragmatic attitudes in therapeutical trials[J]. J Clin Epidemiol，2009，62（5）：499-505.
[7] MACRAE K D. Pragmatic versus explanatory trials[J]. Int J Technol Assess Health Care，1989，5（3）：333-339.
[8] THORPE K E，ZWARENSTEIN M，OXMAN A D，et al. A pragmatic-explanatory continuum indicator summary（PRECIS）：a tool to help trial designers[J]. J Clin Epidemiol，2009，62（5）：464-475.
[9] LOUDON K，TREWEEK S，SULLIVAN F，et al. The PRECIS-2 tool：designing trials that are fit for purpose[J/OL]. BMJ，2015，350：h2147[2022-10-10]. https://doi.org/10.1136/bmj.h2147.
[10] ZWARENSTEIN M，TREWEEK S，GAGNIER J J，et al. Improving the reporting of pragmatic trials：an extension of the CONSORT statement[J/OL]. BMJ，2008，337：a2390[2022-10-10]. https://doi.org/10.1136/bmj.a2390.
[11] 胡贵平，胡亚楠，詹思延. PRECIS研究进展[J]. 中华流行病学杂志，2016，37（3）：439-442.
[12] 唐立，康德英，喻佳洁，等. 实效性随机对照试验：真实世界研究的重要设计[J]. 中国循证医学杂志，2017，17（9）：999-1004.
[13] 温泽淮，李玲，刘艳梅，等. 实效性随机对照试验的技术规范[J]. 中国循证医学杂志，2019，19（7）：794-802.
[14] WANG Y L，LI Z X，ZHAO X Q，et al. Effect of a multifaceted quality improvement intervention on hospital personnel adherence to performance measures in patients with acute ischemic stroke in China：a randomized clinical trial[J]. JAMA，2018，320（3）：245-254.