新药临床试验量效关系研究的设计与分析方法评价_张春茂.pdf

Ch inese Jou rnal of N ew Drugs 2009, 18 ( 20) 1 1 2 1 1 , , , , ( 1 第四军医大学卫生统计学教研室, 西安 710032; 2 潍坊医学院卫生统计学教研室, 潍坊 26 1042) [] 剂量效应关系研究是新药研发的重 环节, 是判断新药有效性和安全性的基础通过不同临 床试验阶段的研究, 可以为给药剂量和给药方案提供依据但目前国内对量效关系研究重视程度不够, 临床 试验通常只设 一个固定剂量或 2 ~ 3 个剂量组, 剂量探索的过程流于形式文中从量效关系研究的目的入 手, 探讨了期临床试验中量效关系研究常见的试验设计和分析方法 [] 量效关系; 临床试验; 自适应性设计; 多重比较; 模型 [] R 969. 4 [] B [ ] 100 3- 3734( 2009) 20 - 1930 - 05 Statistcal design and analysis for doseresponse relationship in clinical trial 1 1 2 1 1 ZHANG Chunm ao , X IA Jiela i, WANG Suzhen , JIANG Zh iw ei , WANG L ing ( 1D ep artm ent of H ealth S ta tistics, F ourth M ilitary M edical Un iversity, X ian 7100 32, China; 2 D ep artm ent of H ealth S tatistics, Wefi ang M ed ical College, Wefi ang 261042, China [Abstract] D oserespon se relation sh ip stu dy is an mi portant factor fo r the new drug deve lopm ent program and it is the foundation to de term ine drug efficacy and safety. Through c lin ical trials, the study prov ide the b asis for dosage reg mi en. H ow ever, the dom est ic studies pay little attent ion to doserespon se re lation sh ip. U su ally, only one f ixeddose or 2 ~ 3 dose g roup s are designed in clin ica l tr ia,l doserespon se study is ignored. T his artic le focu ses on the mi portance o f the research, and review s the exp er mi enta l design and analysis m ethods in Phase c lin ica l trial. [K ey words] doserespon se re lation ship; clinical trial; adaptive design; m u ltiple com parison s; m odeling [ 2] , ( )