适应性设计在新药临床试验中的应用

Application of adaptive designs in clinical trials of developing new drugs

适应性设计因为具有设计灵活、节约成本、缩短研发时间、加快医药产品上市的优势,近年来越来越受到临床试验工作者和统计学家的关注。在临床试验探索期引入适应性设计很有意义,特别是复杂的新药临床试验。某降脂药临床试验采用双重适应性有偏硬币设计(doubly adaptive biased coin design,DBCD)适应性设计方案,在保证检验功效的前提下,将降脂药临床试验的Ⅱb期和Ⅱc期无缝链接起来。DBCD缩短了研发周期,减少了样本量,并且将更多的患者分配到较好的治疗组。

In recent years,adaptive design has been more and more popular among clinical trialists and statisticians. This is because the adaptive designs are often more flexibility,less cost,shorter in development time and faster in marketing pharmaceutical products. It is valuable and important to introduce adaptive designs in the exploration phase of clinical trials,especially complex drugs. In this study,we implement doubly adaptive biased coin design in one clinical trial of a new drug of reducing lipid-level. By combining phase IIb and phase IIc seamlessly,the proposed procedure maintains test power,greatly shortens the development time,and assigns more patients to better treatment groups.