对体内诊断药物临床试验设计和统计分析的考虑  

The consideration for the clinical trial design and statistical analysis of in vivo diagnostic drugs

作　　者：李新旭 周军 唐智敏 王骏[1] LI Xin-xu;ZHOU Jun;TANG Zhi-min;WANG Jun(Center for Drug Evaluation,National Medical Products Administration,Beijing 100022,China)

机构地区：[1]国家药品监督管理局药品审评中心

出　　处：《中国临床药理学杂志》2019年第20期2639-2644,2648,共7页The Chinese Journal of Clinical Pharmacology

基　　金：国家“重大新药创制”科技重大专项基金资助项目(2017ZX09101001)

摘　　要：体内诊断药物的临床试验不同于治疗药物、医疗器械和体外诊断试剂,主要评价目标是诊断准确性和安全性。本文基于诊断准确性,探讨了体内诊断药物的临床试验设计和统计分析要点,如对选择对比品、金标准、试验类型和评价指标的考虑,对检验界值确定、试验假设建立、样本量估算、受试者选择、随机化和盲法等的考虑,以及对统计分析集、统计分析方法和统计推断等的考虑,并阐述了在对比品的使用、金标准的诊断准确性、诊断结果的复杂性、受试者的诊断时机、样本量的估算以及适应性设计的应用等方面所面临的技术挑战。鼓励申办方与监管机构沟通,共同化解体内诊断药物临床试验的技术难题。Clinical trials of in vivo diagnostic drugs are different from those of therapeutic drugs,medical devices and in vitro diagnostic reagents,and their primary endpoints are diagnostic accuracy and safety.Based on the diagnostic accuracy,this paper discussed the key points of clinical trial design and statistical analysis of in vivo diagnostic drugs,such as considering the selection of control drugs,gold standard,test types and endpoints,considering the determination of margin,establishment of test hypothesis,estimation of sample size,selection of subjects,randomization,blinding,etc.,and considering the statistical analysis set,statistical analysis method,statistical inference,etc.,and also discussed the technical challenges in the use of control drugs,diagnostic accuracy of gold standard,complexity of diagnostic results,diagnostic timing of subjects,estimation of sample size and application of adaptive design.The sponsors are encouraged to communicate with the regulatory authorities to solve the technical problems in clinical trials of in vivo diagnostic drugs.

关 键 词：体内诊断药物 临床试验设计 统计分析 考虑 

分 类 号：R97[医药卫生—药品]