Validity and power considerations on hypothesis testing under minimization

Statistics in Medicine
Zhenzhen XuShiowjen Lee

Abstract

Minimization, a dynamic allocation method, is gaining popularity especially in cancer clinical trials. Aiming to achieve balance on all important prognostic factors simultaneously, this procedure can lead to a substantial reduction in covariate imbalance compared with conventional randomization in small clinical trials. While minimization has generated enthusiasm, some controversy exists over the proper analysis of such a trial. Critics argue that standard testing methods that do not account for the dynamic allocation algorithm can lead to invalid statistical inference. Acknowledging this limitation, the International Conference on Harmonization E9 guideline suggests that 'the complexity of the logistics and potential impact on analyses be carefully evaluated when considering dynamic allocation'. In this article, we investigate the proper analysis approaches to inference in a minimization design for both continuous and time-to-event endpoints and evaluate the validity and power of these approaches under a variety of scenarios both theoretically and empirically. Published 2016. This article is a U.S. Government work and is in the public domain in the USA.

References

Jan 1, 1978·Journal of Chronic Diseases·J P Matts, R B McHugh
Jun 1, 1987·Controlled Clinical Trials·L A Kalish, C B Begg
Apr 1, 1985·Statistics in Medicine·L A Kalish, C B Begg
Jun 1, 1985·Controlled Clinical Trials·N J Birkett
May 1, 1974·Clinical Pharmacology and Therapeutics·D R Taves
Sep 1, 1974·Journal of Chronic Diseases·M Zelen
May 30, 1996·Statistics in Medicine·M A Proschan
Feb 26, 2004·Statistics in Medicine·UNKNOWN Committee for Proprietary Medicinal Products (CPMP)
Feb 2, 2011·Biometrics·Michael ProschanLisa Kammerman
Jul 20, 2011·Statistics & Probability Letters·Richard Simon, Noah Robin Simon
Jul 6, 2012·Statistics in Medicine·Lee D Kaiser
Jun 14, 2013·Pharmaceutical Statistics·Baoguang HanDamian McEntegart

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Citations

Nov 22, 2016·Statistics in Medicine·Zhenzhen XuShiowjen Lee
Jan 29, 2021·Statistical Methods in Medical Research·Andrea CallegaroNaveen Karkada

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