Randomized clinical trial design for assessing noninferiority when superiority is expected
Randomized clinical trial design for assessing noninferiority when superiority is expected
复制标题
DOI：
10.1200/jco.2007.11.8711
复制
发表时间：
2007-11-01
影响因子：
45.3
通讯作者：
Gray, Robert
中科院分区：
文献类型：
--
作者：
Freidlin, Boris;Korn, Edward L.;Gray, Robert
随机临床试验（RCT）是新疗法明确评估的黄金标准。旨在证明新疗法的疗效并不逊色于标准疗法的随机对照试验称为非劣效性试验。传统上，非劣效性试验需要非常大的样本量。有时，具有良好毒性和/或耐受性的新治疗方案也有望在疗效上有一定程度的改善。在这种专门的环境中，我们描述了一种混合试验设计方法，该方法需要比标准非劣效性设计小得多的样本量。这种混合设计自然可以结合正式的优越性和非劣效性测试。
The randomized clinical trial (RCT) is the gold standard for definitive evaluation of new therapies. RCTs designed to show that the therapeutic efficacy of a new therapy is not unacceptably inferior to that of standard therapy are called noninferiority trials. Traditionally, noninferiority trials have required very large sample sizes. Sometimes, a new treatment regimen with a favorable toxicity and/or tolerability profile is also expected to have some modest improvement in efficacy. In such specialized settings we describe a hybrid trial-design approach that requires a dramatically smaller sample size than that of a standard noninferiority design. This hybrid design can naturally incorporate a formal test of superiority as well as noninferiority.