罕见病小样本临床试验的统计设计及其应用  

Statistical design and application of clinical trials with small sample sizes for rare diseases

作　　者：高源 仲子航 陆梦依 陈峰 Gao Yuan;Zhong Zihang;Lu Mengyi;Chen Feng(Department of Biostatistics,School of Public Health,Nanjing Medical University,Nanjing 211166,China)

机构地区：[1]南京医科大学公共卫生学院生物统计学系,南京211166

出　　处：《中华流行病学杂志》2024年第12期1700-1704,共5页Chinese Journal of Epidemiology

摘　　要：罕见病由于病例少,难以开展大规模临床试验。本文介绍了几种潜在的可用于罕见病的小样本统计设计,包括交叉设计、自身多阶段交叉设计、随机安慰剂阶段设计、随机撤药设计、成组序贯设计、适应性设计等,讨论这些设计的优缺点及应用场景,进一步讨论了Bayes决策在罕见病临床试验中的优势。旨在为罕见病小样本临床试验的设计和实施提供参考。Due to the limited number of cases,conducting large-scale clinical trials for rare diseases is challenging.This review introduces several small sample statistical designs tailored for rare diseases,including crossover design,n-of-1 design,randomized placebo-phase design,randomized withdrawal design,group sequential design,and adaptive design.It discusses the advantages,disadvantages,and application scenarios of these designs.Additionally,it explores the benefits of Bayes decision-making in clinical trials for rare diseases.The aim is to provide a reference for designing and implementing small sample clinical trials for rare diseases.

关 键 词：罕见病 小样本 统计设计 临床试验 

分 类 号：R195.1[医药卫生—卫生统计学]