A general approach for sample size calculation for the three-arm "gold standard" non-inferiority design

• Verfasst von: Stucke, Kathrin [VerfasserIn]
• Verfasst von: Kieser, Meinhard [VerfasserIn]
• Titel: A general approach for sample size calculation for the three-arm "gold standard" non-inferiority design
• Verf.angabe: Kathrin Stucke and Meinhard Kieser
• E-Jahr: 2012
• Jahr: 4 July 2012
• Umfang: 18 S.
• Fussnoten: Gesehen am 20.08.2020
• Titel Quelle: Enthalten in: Statistics in medicine
• Ort Quelle: Chichester [u.a.] : Wiley, 1982
• Jahr Quelle: 2012
• Band/Heft Quelle: 31(2012), 28, Seite 3579-3596
• ISSN Quelle: 1097-0258
• DOI: doi:10.1002/sim.5461
• Datenträger: Online-Ressource
• Sprache: eng
• Sach-SW: intersection-union test
• Sach-SW: multiple comparisons
• Sach-SW: non-inferiority
• Sach-SW: optimal allocation
• Sach-SW: sample size calculation
• Sach-SW: three-arm trial
• K10plus-PPN: 1576246280

Abstract

In the three-arm ‘gold standard’ non-inferiority design, an experimental treatment, an active reference, and a placebo are compared. This design is becoming increasingly popular, and it is, whenever feasible, recommended for use by regulatory guidelines. We provide a general method to calculate the required sample size for clinical trials performed in this design. As special cases, the situations of continuous, binary, and Poisson distributed outcomes are explored. Taking into account the correlation structure of the involved test statistics, the proposed approach leads to considerable savings in sample size as compared with application of ad hoc methods for all three scale levels. Furthermore, optimal sample size allocation ratios are determined that result in markedly smaller total sample sizes as compared with equal assignment. As optimal allocation makes the active treatment groups larger than the placebo group, implementation of the proposed approach is also desirable from an ethical viewpoint. Copyright © 2012 John Wiley & Sons, Ltd.