• The Korean Journal of Applied Statistics
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• v.22 no.2
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• pp.341-353
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• 2009

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.

• The Korean Journal of Applied Statistics
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• v.22 no.6
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• pp.1249-1263
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• 2009

The power function in sample size determination has to be characterized by an appropriate statistical test for the hypothesis of interest. Nonparametric tests are suitable in the analysis of ordinal data or frequency data with ordered categories which appear frequently in the biomedical research literature. In this paper, we study sample size calculation methods for the Wilcoxon-Mann-Whitney test for one- and two-dimensional ordinal outcomes. While the sample size formula for the univariate outcome which is based on the variances of the test statistic under both null and alternative hypothesis perform well, this formula requires additional information on probability estimates that appear in the variance of the test statistic under alternative hypothesis, and the values of these probabilities are generally unknown. We study the advantages and disadvantages of different sample size formulas with simulations. Sample sizes are calculated for the two-dimensional ordinal outcomes of efficacy and safety, for which bivariate Wilcoxon-Mann-Whitney test is appropriate than the multivariate parametric test.