方法对比

	匹配的II期临床试验	匹配病例对照研究
领域	流行病学	流行病学
方法族	Process / pipeline	Process / pipeline
起源年份	1960s–1980s (formalized with Simon optimal designs, 1989)	1950s–1970s
提出者	Gehan (1961) for Phase II designs; matching frameworks adapted from case-control methodology	Brian MacMahon and others; systematised by Schlesselman (1982)
类型	Controlled clinical trial design	Observational analytic design
开创性文献	Gehan, E. A. (1961). The determination of the number of patients required in a preliminary and a follow-up trial of a new chemotherapeutic agent. Journal of Chronic Diseases, 13(4), 346–353.	Rothman, K. J., Greenland, S., & Lash, T. L. (2008). Modern Epidemiology (3rd ed.). Lippincott Williams & Wilkins. ISBN: 978-0781755474
别名	matched Phase II trial, historically matched Phase II study, propensity-matched Phase II trial, externally controlled Phase II trial	matched case-referent study, individually matched case-control, pair-matched case-control, matched case-control design
相关	5	5
摘要	A matched Phase II clinical trial is a single-arm or small-controlled early-efficacy study in which treated patients are paired with matched controls — drawn from historical databases, registries, or concurrent external cohorts — on key prognostic variables such as age, disease stage, and performance status. This design allows preliminary efficacy assessment without a concurrent randomized arm, trading randomization for feasibility while partially controlling for confounding through the matching process.	A matched case-control study is an observational epidemiological design in which each case (a person with the disease or outcome of interest) is paired with one or more controls (persons without the outcome) who share one or more characteristics — such as age, sex, or clinical setting — to control confounding. Exposure history is then compared between cases and their matched controls to estimate the odds ratio of the exposure-disease association.