具有混合二元和连续反应的II期临床试验的先前有效样本量

IF 1.4 3区数学 Q2 STATISTICS & PROBABILITY

Statistica Neerlandica Pub Date : 2022-11-07 DOI:10.1111/stan.12283

Meghna Bose, J. Angers, A. Biswas

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引用次数: 0

摘要

在毒性和疗效是治疗反应载体的两个组成部分的II期临床试验中，寻找有效样本量(ESS)的问题被考虑。特别地，假设其中一个分量是二元的，另一个是连续的。研究了不同设置下不同先验分布的二元安全性和持续有效性情况。在各种情况下得到理论表达式。通过仿真研究对这些方法进行了评价和比较。然后，通过使用Covid - 19 II期疫苗试验的一些真实数据来说明所提出的方法。

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Prior effective sample size in phase II clinical trials with mixed binary and continuous responses

The problem of finding Effective Sample Size (ESS) in Phase II clinical trials where toxicity and efficacy are the two components of the treatment response vector is considered. In particular, one of the components is assumed to be binary and the other is assumed to be continuous. The case of binary safety and continuous efficacy is studied for different prior distributions under different set up. Theoretical expressions are obtained in various situations. The methods are evaluated and compared by simulation studies. The proposed method is then illustrated by using some real life data on a phase II vaccine trial for Covid‐19.

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来源期刊

Statistica Neerlandica 数学-统计学与概率论

CiteScore

2.60

自引率

6.70%

发文量

审稿时长

>12 weeks

期刊介绍： Statistica Neerlandica has been the journal of the Netherlands Society for Statistics and Operations Research since 1946. It covers all areas of statistics, from theoretical to applied, with a special emphasis on mathematical statistics, statistics for the behavioural sciences and biostatistics. This wide scope is reflected by the expertise of the journal’s editors representing these areas. The diverse editorial board is committed to a fast and fair reviewing process, and will judge submissions on quality, correctness, relevance and originality. Statistica Neerlandica encourages transparency and reproducibility, and offers online resources to make data, code, simulation results and other additional materials publicly available.