Group sequential designs for negative binomial outcomes

Statistical Methods in Medical Research
Tobias MützeTim Friede

Abstract

Count data and recurrent events in clinical trials, such as the number of lesions in magnetic resonance imaging in multiple sclerosis, the number of relapses in multiple sclerosis, the number of hospitalizations in heart failure, and the number of exacerbations in asthma or in chronic obstructive pulmonary disease (COPD) are often modeled by negative binomial distributions. In this manuscript, we study planning and analyzing clinical trials with group sequential designs for negative binomial outcomes. We propose a group sequential testing procedure for negative binomial outcomes based on Wald statistics using maximum likelihood estimators. The asymptotic distribution of the proposed group sequential test statistics is derived. The finite sample size properties of the proposed group sequential test for negative binomial outcomes and the methods for planning the respective clinical trials are assessed in a simulation study. The simulation scenarios are motivated by clinical trials in chronic heart failure and relapsing multiple sclerosis, which cover a wide range of practically relevant settings. Our research assures that the asymptotic normal theory of group sequential designs can be applied to negative binomial outcomes when th...Continue Reading

References

Feb 17, 1998·Statistics in Medicine·D O Scharfstein, A A Tsiatis
Sep 3, 1999·Journal of Biopharmaceutical Statistics·W Jiang
Aug 15, 2001·Lancet·M K ParmarUNKNOWN CHART steering committee
Oct 31, 2006·Biostatistics·Nian-Sheng TangShun-Fang Wang
Sep 22, 2007·Journal of Biopharmaceutical Statistics·Qi Xia, Donald R Hoover
Jul 2, 2008·The European Respiratory Journal·O N KeeneJ A Anderson
Jan 30, 2009·Computational Statistics & Data Analysis·Inmaculada B AbanNsoki Mavinga
Feb 4, 2009·Circulation·Cyrus MehtaJames H Ware
Jun 1, 2007·Journal of Statistical Planning and Inference·Edsel A PeñaJuan R González
Nov 3, 2009·The European Respiratory Journal·O N KeeneP W Jones
Feb 11, 2010·Statistics in Medicine·Tim Friede, Heinz Schmidli
Sep 14, 2010·Contemporary Clinical Trials·Bart Spiessens, Muriel Debois
Nov 23, 2011·Circulation·James E Udelson
Sep 17, 2013·Pharmaceutical Statistics·Thomas GsponerHeinz Schmidli
Jun 17, 2014·Journal of Biopharmaceutical Statistics·Paul GalloVivian H Shih
Sep 22, 2015·Statistics in Medicine·Tobias MützeTim Friede
Nov 20, 2016·Statistics in Medicine·Tobias MützeTim Friede
Dec 14, 2016·The New England Journal of Medicine·Margaret M Redfield
Jan 1, 2015·Therapeutic Innovation & Regulatory Science·Roland FischHeinz Schmidli

❮ Previous
Next ❯

Citations

Nov 15, 2019·Clinical Pharmacology and Therapeutics·Heinz SchmidliFrank Bretz
Jan 8, 2021·Communications in Statistics: Theory and Methods·Michael J GraylingAdrian P Mander

❮ Previous
Next ❯

Software Mentioned

Pocock
R
R package
gscounts
ADDPLAN®
East®

Related Concepts

Related Feeds

Asthma

This feed focuses in Asthma in which your airways narrow and swell. This can make breathing difficult and trigger coughing, wheezing and shortness of breath.

Allergy and Asthma

Allergy and asthma are inflammatory disorders that are triggered by the activation of an allergen-specific regulatory t cell. These t cells become activated when allergens are recognized by allergen-presenting cells. Here is the latest research on allergy and asthma.

Related Papers

American Journal of Human Genetics
A M Lilienfeld
American Journal of Human Genetics
M E Bernstein
The Spanish Journal of Psychology
Miguel A García-Pérez, Vicente Núñez-Antón
© 2022 Meta ULC. All rights reserved