手法を比較

	実用的な第II相臨床試験	第I相臨床試験
分野	疫学	疫学
系統	Process / pipeline	Process / pipeline
提唱年	Pragmatic framework: 1967; Phase II application: 1990s–2000s	1960s (formal regulatory framework established ~1963–1970s)
提唱者	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
種類	Interventional study design	Interventional clinical study design
原典	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.
別名	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
関連	6	6
概要	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.