方法对比

	Pragmatic Clinical Trial	自适应试验设计
领域	临床研究	临床研究
方法族	Process / pipeline	Process / pipeline
起源年份	2009-2015	1990s-2000s
提出者	Thorpe et al. (2009); PRECIS framework developed by international consortia	Stephen Pocock, Christopher Jennison, and statistical methodologists; FDA formalized guidance 2019
类型	Research Design	Research Design
开创性文献	Thorpe, K. E., Zwarenstein, M., Oxman, A. D., Treweek, S., Furberg, C. D., Altman, D. G., ... & Tugwell, P. (2009). A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. CMAJ, 180(10), E47–E57.	Pocock, S. J. (2005). Current issues in the design and interpretation of clinical trials. BMJ, 330(7500), 1118–1121.
别名	pragmatic trial, real-world trial, effectiveness trial, PRECIS-2	adaptive trial, adaptive design, response-adaptive randomization, RAR
相关	4	1
摘要	A pragmatic trial is designed to evaluate the real-world effectiveness of an intervention in typical clinical settings with diverse, representative patients, minimal exclusion criteria, and clinically relevant outcomes. Developed by Thorpe and colleagues (2009) and formalized via the PRECIS-2 framework (2015), pragmatic trials bridge the gap between explanatory efficacy trials (conducted in controlled research settings) and implementation science, answering the question 'Does this work in actual clinical practice?' rather than 'Can this work under ideal conditions?'	An adaptive trial design allows pre-specified modifications to the trial based on interim data—such as sample size re-estimation, stopping for futility or efficacy, dropping ineffective arms, or shifting randomization ratios toward better-performing treatments. Developed systematically in the 1990s–2000s by statisticians like Pocock and Jennison, and formalized by the FDA in 2019, adaptive designs accelerate drug development, reduce exposure to ineffective treatments, and improve efficiency without inflating false-positive rates when properly executed.