标题: 临床试验之连续性满足点Phase II/III调适性设计
An Alternative Phase II/III Design in Clinical Trials for Continuous Endpoint
作者: 刘佳佩
Liu, Chia-Pei
萧金福
Hsiao, Chin-Fu
统计学研究所
关键字: 调适设计;临床试验;phase II/III设计;adaptive design;clinical trial;phase II/III design
公开日期: 2009
摘要: 医药研发是非常具有风险、复杂、高成本又费时的过程。其中大部分的时间和经费是用于临床试验。因此,我们需要更有效率且可靠的临床试验方法,来分析资料及评估药物的风险和效用,以减少病人样本数,缩短发展期间,进而降低药物开发成本。在本文中,我们发展一个连续性满足点的phase II / III试验设计,在phase II试验中评估不同剂量的试验药物与对照组两两之间比较。在phase II试验,随机分派病人接受一个试验药物剂量或对照组。如果一个或一些剂量有统计显着的疗效优于对照组,这些剂量将被选中进入phase III试验。此外,病人在被选中的剂量组与对照组将继续进入phase III试验。同时也将招募新的病人,随机分配至所选的药物剂量组或对照组。我们会计算每阶段的检定统计量之临界值,以确定各药物剂量在试验过程中是否应被淘汰或被选择,并计算合适的样本大小以方便招募病人。在我们的设计中,由于将传统的phase II试验及phase III试验合并成单一的试验,同时,在phase II试验收集的资料也会进入phase III试验做最后的分析,因此可以减少样本数量和节省试验时间。
The pharmaceutical development is very risky, complex, costly, and time-consuming. Much of time and costs were spent in clinical trials. Hence, we need the methods which can be more efficient and reliable to minimize sample size, shorten the period of development duration, and thus reduce the cost for drug development. In this paper, we demonstrate the phase II/III design for continuous endpoint based on pariwise comparisons at the phase II stage when evaluating the efficacy of drugs. For the phase II stage, patients are randomly assigned to receive either one of the several doses of the test drug or to the control group. If one or some doses are declared to have a statistically significantly superior efficacy over control, these doses will be selected for the phase III trial. In addition, the patients in the selected doses and control groups will be continued to the phase III stage. Also new patients will be recruited and randomized to receive either the selected doses of the test drug or to the control group. We will find the critical value at each stage to determine whether the treatment should be dropped out or selected in the process of the trials, and compute the required sample size for facilitating recruitment of patients. In our design, since we integrate the traditional phase II and III trials into a single trial, and the data collected from phase II stage will also be included into the final analysis, sample size reduction and trial time saving may be possible.
URI: http://140.113.39.130/cdrfb3/record/nctu/#GT079726510
http://hdl.handle.net/11536/45240
显示于类别:Thesis


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