方法对比

	实用性II期临床试验	I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	Pragmatic framework: 1967; Phase II application: 1990s–2000s	1960s (formal regulatory framework established ~1963–1970s)
提出者	Conceptual basis: Daniel Schwartz & Joseph Lellouch (pragmatic vs. explanatory distinction, 1967); applied to Phase II context by drug developers and trialists from the 1990s onward	Regulatory and clinical pharmacology community; formalized in U.S. FDA IND regulations (1963) and ICH guidelines
类型	Interventional study design	Interventional clinical study design
开创性文献	Schwartz, D., & Lellouch, J. (1967). Explanatory and pragmatic attitudes in therapeutical trials. Journal of Chronic Diseases, 20(8), 637–648.	Storer, B. E. (1989). Design and analysis of phase I clinical trials. Biometrics, 45(3), 925–937.
别名	pragmatic Phase II trial, real-world Phase II trial, Phase II pragmatic RCT, Phase IIb pragmatic trial	Phase 1 trial, first-in-human study, FIH study, dose-escalation study
相关	6	6
摘要	A pragmatic Phase II clinical trial is an early-to-mid-stage interventional study that evaluates a new treatment's preliminary efficacy and safety under conditions that approximate real-world clinical practice rather than tightly controlled experimental settings. It sits between pure explanatory Phase II trials and large pragmatic Phase III confirmatory trials, prioritising practical feasibility and clinical relevance while still generating the signal needed to justify further development.	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.