方法对比

	I期临床试验	II期临床试验
领域	流行病学	流行病学
方法族	Process / pipeline	Process / pipeline
起源年份	1960s (formal regulatory framework established ~1963–1970s)	1960s–1970s (formalised in US federal drug regulation)
提出者	Regulatory and clinical pharmacology community; formalized in U.S. FDA IND regulations (1963) and ICH guidelines	U.S. Food and Drug Administration / ICH E8 guidelines (institutionalised framework)
类型	Interventional clinical study design	Interventional clinical study design
开创性文献	Storer, B. E. (1989). Design and analysis of phase I clinical trials. Biometrics, 45(3), 925–937.	Friedman, L. M., Furberg, C. D., DeMets, D. L., Reboussin, D. M., & Granger, C. B. (2015). Fundamentals of Clinical Trials (5th ed.). Springer. ISBN: 978-3319185392
别名	Phase 1 trial, first-in-human study, FIH study, dose-escalation study	Phase 2 trial, Phase II study, early efficacy trial, proof-of-concept trial
相关	6	6
摘要	A Phase I clinical trial is the first stage of human testing for a new drug, biologic, or intervention. Its primary objective is to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) rather than therapeutic efficacy. Small cohorts of participants — typically healthy volunteers or patients with advanced disease — receive sequentially increasing doses to identify the maximum tolerated dose (MTD) and the dose-limiting toxicities (DLTs) that define the boundary for subsequent trials.	A Phase II clinical trial is the second stage in the drug or intervention development pipeline, conducted after Phase I safety testing. Its primary goal is to assess whether the intervention shows preliminary efficacy signals in a relevant patient population at the dose established in Phase I, while continuing to characterise the safety and tolerability profile. Phase II trials are generally smaller than Phase III confirmatory trials and serve as critical go/no-go decision points before large-scale investment.