Korn Edward L, Freidlin Boris
Biometric Research Branch, National Cancer Institute, Bethesda, MD 20892, USA.
Stat Med. 2006 Sep 15;25(17):2922-31. doi: 10.1002/sim.2516.
A trial of a new therapy is to be compared to results from a previous trial of patients treated with a standard therapy. For a given sample size for the trial of the new therapy, we desire the power, against a specific alternative hypothesis, for the hypothesis test of the null hypothesis that the therapies are equivalent. Alternatively, the sample size required for the trial of the new therapy is needed for a target power. We explain why a popular method for doing these calculations is wrong, and discuss alternative methods in the context of normal outcomes, binary outcomes, and time-to-event outcomes.
一种新疗法的试验结果将与先前接受标准疗法治疗的患者试验结果进行比较。对于新疗法试验的给定样本量，我们希望针对特定的备择假设，对两种疗法等效的原假设进行假设检验时具有检验功效。或者，对于目标检验功效，需要确定新疗法试验所需的样本量。我们解释了一种常用的计算方法为何错误，并在正态分布结局、二项分布结局和事件发生时间结局的背景下讨论了替代方法。
