A rank-based sample size method for multiple outcomes in clinical trials

Statistics in Medicine
Peng HuangPeter C O'Brien

Abstract

O'Brien (Biometrics 1984; 40:1079-1087) introduced a rank-sum-type global statistical test to summarize treatment's effect on multiple outcomes and to determine whether a treatment is better than others. This paper presents a sample size computation method for clinical trial design with multiple primary outcomes, and O'Brien's test or its modified test (Biometrics 2005; 61:532-539) is used for the primary analysis. A new measure, the global treatment effect (GTE), is introduced to summarize treatment's efficacy from multiple primary outcomes. Computation of the GTE under various settings is provided. Sample size methods are presented based on prespecified GTE both when pilot data are available and when no pilot data are available. The optimal randomization ratio is given for both cases. We compare our sample size method with the Bonferroni adjustment for multiple tests. Since ranks are used in our derivation, sample size formulas derived here are invariant to any monotone transformation of the data and are robust to outliers and skewed distributions. When all outcomes are binary, we show how sample size is affected by the success probabilities of outcomes. Simulation shows that these sample size formulas provide good control of...Continue Reading

References

Dec 30, 1993·Statistics in Medicine·J Whitehead
Jun 1, 1997·Controlled Clinical Trials·P C O'Brien, N L Geller
Mar 6, 2003·Statistical Methods in Medical Research·N RabbeeP Senchaudhuri
May 3, 2006·Archives of General Psychiatry·Helena Chmura KraemerJerome A Yesavage

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Citations

Mar 4, 2009·Clinical Trials : Journal of the Society for Clinical Trials·Cynthia R GrossWilliam Thomas
Jan 27, 2016·Biometrics·Ritesh RamchandaniDianne M Finkelstein
Jun 11, 2009·Movement Disorders : Official Journal of the Movement Disorder Society·Peng HuangUNKNOWN Parkinson Study Group
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Jan 4, 2020·Neurological Sciences : Official Journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology·Dafin F MuresanuJohannes Vester

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