Determination of Sample Sizes of Bivariate Efficacy and Safety Outcomes

- Journal title : Korean Journal of Applied Statistics
- Volume 22, Issue 2, 2009, pp.341-353
- Publisher : The Korean Statistical Society
- DOI : 10.5351/KJAS.2009.22.2.341

Title & Authors

Determination of Sample Sizes of Bivariate Efficacy and Safety Outcomes

Lee, Hyun-Hak; Song, Hae-Hiang;

Lee, Hyun-Hak; Song, Hae-Hiang;

Abstract

We consider sample-size determination problem motivated by comparative clinical trials where patient outcomes are characterized by a bivariate outcome of efficacy and safety. Thall and Cheng (1999) presented a sample size methodology for the case of bivariate binary outcomes. We propose a bivariate Wilcoxon-Mann-Whitney(WMW) statistics for sample-size determination for binary outcomes, and this nonparametric method can be equally used to determine sample sizes of ordinal outcomes. The two methods of sample size determination rely on the same testing strategy for the target parameters but differs in the test statistics, an asymptotic bivariate normal statistic of the transformed proportions in Thall and Cheng (1999) and nonparametric bivariate WMW statistic in the other method. Sample sizes are calculated for the two experimental oncology trials, described in Thall and Cheng (1999), and for the first trial example the sample sizes of a bivariate WMW statistic are smaller than those of Thall and Cheng (1999), while for the second trial example the reverse is true.

Keywords

Sample sizes;bivariate WMW;efficacy and safety;

Language

Korean

Cited by

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