RECONSIDERING SOME ASPECTS OF THE TWO-TRIALS PARADIGM
RECONSIDERING SOME ASPECTS OF THE TWO-TRIALS PARADIGM
复制标题
重新考虑两次试验范式的某些方面
DOI：
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发表时间：
2002
影响因子：
1.1
通讯作者：
W. Maurer
中科院分区：
文献类型：
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作者：
J. Maca;P. Gallo;M. Branson;W. Maurer
在临床提交中证明疗效的一个共同标准是至少两项关键试验的统计学显著性结局（“两项试验惯例”）。当不同试验中的数据结构足够相似，可以合并试验间的数据进行综合分析时，我们认为这种分析是判断疗效的更合理和有效的基础。可以建立联合分析的标准，以确保与两项试验惯例相同的假阳性率保护。组合分析通常比具有相同假阳性率保护的两次试验方法的相应应用具有更大的功效。此外，我们描述了纯组合分析的修改版本的行为，通过限制单个试验p值的较大值，将试验结果的再现性纳入正式标准。这些修改显示，以保持理想的行为的纯组合分析，即，更高的权力相比，两个试验公约。
A common standard for the demonstration of efficacy in a clinical submission is a statistically significant outcome in at least two pivotal trials (“two-trials convention”). When the data structures in different trials are sufficiently similar to allow pooling of the data across trials for a combined analysis, we argue here that such an analysis is a more logical and efficient basis for a judgment regarding efficacy. Criteria for combined analyses may be established, which ensure the same false positive rate protection as the two-trials convention. A combined analysis will generally have much more power than the corresponding application of the two-trials approach that has the same false positive rate protection. In addition, we describe the behavior of modified versions of pure combined analysis, which incorporate a formal standard for reproducibility of trial results by limiting the larger of the individual trial p-values. These modifications are shown to maintain the desirable behavior of the pure combined analysis, namely, higher power compared to the two-trials convention.