二元终点的双臂优效和非优效试验的最佳样本量分配。

IF 1.3 4区 医学 Q4 PHARMACOLOGY & PHARMACY Pharmaceutical Statistics Pub Date : 2024-09-01 Epub Date: 2024-03-12 DOI:10.1002/pst.2375
Marietta Kirchner, Stefanie Schüpke, Meinhard Kieser
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引用次数: 0

摘要

临床试验的样本量必须足够大,以确保有足够的力量达到研究目的。另一方面,出于伦理和经济方面的考虑,样本量也不应超过必要的范围。样本量分配是影响所需总样本量的参数之一。对于二元终点的双臂优效和非劣效试验,我们在多种情况下进行了大量计算,以确定在所有其他参数固定的情况下,使总样本量最小的最佳分配比例。结果表明,对于优效和非劣效试验,最佳分配比例可能与两组样本量相等的情况有很大偏差。不过,与均衡分配相比,最佳分配总样本量所节省的样本量通常很小。
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Optimal sample size allocation for two-arm superiority and non-inferiority trials with binary endpoints.

The sample size of a clinical trial has to be large enough to ensure sufficient power for achieving the aim the study. On the other side, for ethical and economical reasons it should not be larger than necessary. The sample size allocation is one of the parameters that influences the required total sample size. For two-arm superiority and non-inferiority trials with binary endpoints, we performed extensive computations over a wide range of scenarios to determine the optimal allocation ratio that minimizes the total sample size if all other parameters are fixed. The results demonstrate, that for both superiority and non-inferiority trials the optimal allocation may deviate considerably from the case of equal sample size in both groups. However, the saving in sample size when allocating the total sample size optimally as compared to balanced allocation is typically small.

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来源期刊
Pharmaceutical Statistics
Pharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.70
自引率
6.70%
发文量
90
审稿时长
6-12 weeks
期刊介绍: Pharmaceutical Statistics is an industry-led initiative, tackling real problems in statistical applications. The Journal publishes papers that share experiences in the practical application of statistics within the pharmaceutical industry. It covers all aspects of pharmaceutical statistical applications from discovery, through pre-clinical development, clinical development, post-marketing surveillance, consumer health, production, epidemiology, and health economics. The Journal is both international and multidisciplinary. It includes high quality practical papers, case studies and review papers.
期刊最新文献
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