subtee: An R Package for Subgroup Treatment Effect Estimation in Clinical Trials

The investigation of subgroups is an integral part of randomized clinical trials. Exploration of treatment effect heterogeneity is typically performed by covariate-adjusted analyses including treatment-by-covariate interactions. Several statistical techniques, such as model averaging and bagging, we...

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Main Authors: Nicolas M. Ballarini, Marius Thomas, Gerd K. Rosenkranz, Björn Bornkamp
Format: Article
Language:English
Published: Foundation for Open Access Statistics 2021-10-01
Series:Journal of Statistical Software
Subjects:
Online Access:https://www.jstatsoft.org/index.php/jss/article/view/3652
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author Nicolas M. Ballarini
Marius Thomas
Gerd K. Rosenkranz
Björn Bornkamp
author_facet Nicolas M. Ballarini
Marius Thomas
Gerd K. Rosenkranz
Björn Bornkamp
author_sort Nicolas M. Ballarini
collection DOAJ
description The investigation of subgroups is an integral part of randomized clinical trials. Exploration of treatment effect heterogeneity is typically performed by covariate-adjusted analyses including treatment-by-covariate interactions. Several statistical techniques, such as model averaging and bagging, were proposed recently to address the problem of selection bias in treatment effect estimates for subgroups. In this paper, we describe the subtee R package for subgroup treatment effect estimation. The package can be used for all commonly encountered type of outcomes in clinical trials (continuous, binary, survival, count). We also provide additional functions to build the subgroup variables to be used and to plot the results using forest plots. The functions are demonstrated using data from a clinical trial investigating a treatment for prostate cancer with a survival endpoint.
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spelling doaj.art-b796e2c66b1b4325b78200df50ba0bf22023-06-01T18:48:04ZengFoundation for Open Access StatisticsJournal of Statistical Software1548-76602021-10-019911710.18637/jss.v099.i143493subtee: An R Package for Subgroup Treatment Effect Estimation in Clinical TrialsNicolas M. Ballarini0https://orcid.org/0000-0002-3432-8931Marius Thomas1https://orcid.org/0000-0003-2790-829XGerd K. Rosenkranz2https://orcid.org/0000-0002-8371-2059Björn Bornkamp3https://orcid.org/0000-0002-6294-8185Section for Medical Statistics, Center for Medical Statistics, Informatics, and Intelligent Systems, Medical University of Vienna, Vienna, AustriaNovartis Pharma AGSection for Medical Statistics, Center for Medical Statistics, Informatics, and Intelligent Systems, Medical University of Vienna, Vienna, AustriaNovartis Pharma AGThe investigation of subgroups is an integral part of randomized clinical trials. Exploration of treatment effect heterogeneity is typically performed by covariate-adjusted analyses including treatment-by-covariate interactions. Several statistical techniques, such as model averaging and bagging, were proposed recently to address the problem of selection bias in treatment effect estimates for subgroups. In this paper, we describe the subtee R package for subgroup treatment effect estimation. The package can be used for all commonly encountered type of outcomes in clinical trials (continuous, binary, survival, count). We also provide additional functions to build the subgroup variables to be used and to plot the results using forest plots. The functions are demonstrated using data from a clinical trial investigating a treatment for prostate cancer with a survival endpoint.https://www.jstatsoft.org/index.php/jss/article/view/3652model averagingbootstrapselection biastreatment effect heterogeneity
spellingShingle Nicolas M. Ballarini
Marius Thomas
Gerd K. Rosenkranz
Björn Bornkamp
subtee: An R Package for Subgroup Treatment Effect Estimation in Clinical Trials
Journal of Statistical Software
model averaging
bootstrap
selection bias
treatment effect heterogeneity
title subtee: An R Package for Subgroup Treatment Effect Estimation in Clinical Trials
title_full subtee: An R Package for Subgroup Treatment Effect Estimation in Clinical Trials
title_fullStr subtee: An R Package for Subgroup Treatment Effect Estimation in Clinical Trials
title_full_unstemmed subtee: An R Package for Subgroup Treatment Effect Estimation in Clinical Trials
title_short subtee: An R Package for Subgroup Treatment Effect Estimation in Clinical Trials
title_sort subtee an r package for subgroup treatment effect estimation in clinical trials
topic model averaging
bootstrap
selection bias
treatment effect heterogeneity
url https://www.jstatsoft.org/index.php/jss/article/view/3652
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AT bjornbornkamp subteeanrpackageforsubgrouptreatmenteffectestimationinclinicaltrials