Assessment of statistical significance and clinical relevance

• Verfasst von: Kieser, Meinhard [VerfasserIn]
• Verfasst von: Friede, Tim [VerfasserIn]
• Verfasst von: Gondan, Matthias [VerfasserIn]
• Titel: Assessment of statistical significance and clinical relevance
• Verf.angabe: Meinhard Kieser, Tim Friede, Matthias Gondan
• Jahr: 2013
• Umfang: 13 S.
• Fussnoten: First published: 28 September 2012 ; Gesehen am 23.08.2018
• Titel Quelle: Enthalten in: Statistics in medicine
• Ort Quelle: Chichester [u.a.] : Wiley, 1982
• Jahr Quelle: 2013
• Band/Heft Quelle: 32(2013), 10, Seite 1707-1719
• ISSN Quelle: 1097-0258
• DOI: doi:10.1002/sim.5634
• Datenträger: Online-Ressource
• Sprache: eng
• Sach-SW: clinical relevance
• Sach-SW: effect size
• Sach-SW: probabilistic index
• Sach-SW: relative effect
• Sach-SW: responder analysis
• Sach-SW: sample size
• Sach-SW: statistical significance
• K10plus-PPN: 158032990X

Abstract

In drug development, it is well accepted that a successful study will demonstrate not only a statistically significant result but also a clinically relevant effect size. Whereas standard hypothesis tests are used to demonstrate the former, it is less clear how the latter should be established. In the first part of this paper, we consider the responder analysis approach and study the performance of locally optimal rank tests when the outcome distribution is a mixture of responder and non-responder distributions. We find that these tests are quite sensitive to their planning assumptions and have therefore not really any advantage over standard tests such as the t-test and the Wilcoxon-Mann-Whitney test, which perform overall well and can be recommended for applications. In the second part, we present a new approach to the assessment of clinical relevance based on the so-called relative effect (or probabilistic index) and derive appropriate sample size formulae for the design of studies aiming at demonstrating both a statistically significant and clinically relevant effect. Referring to recent studies in multiple sclerosis, we discuss potential issues in the application of this approach. Copyright © 2012 John Wiley & Sons, Ltd.