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Subgroup identification in clinical trials via the predicted individual treatment effect

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  • N.M. Ballarini
  • G.K. Rosenkranz
  • Thomas Jaki
  • F. König
  • M. Posch
Article numbere0205971
<mark>Journal publication date</mark>18/10/2018
<mark>Journal</mark>PLoS ONE
Issue number10
Number of pages22
Publication StatusPublished
<mark>Original language</mark>English


Identifying subgroups of treatment responders through the different phases of clinical trials has the potential to increase success in drug development. Recent developments in subgroup analysis consider subgroups that are defined in terms of the predicted individual treatment effect, i.e. the difference between the predicted outcome under treatment and the predicted outcome under control for each individual, which in turn may depend on multiple biomarkers. In this work, we study the properties of different modelling strategies to estimate the predicted individual treatment effect. We explore linear models and compare different estimation methods, such as maximum likelihood and the Lasso with and without randomized response. For the latter, we implement confidence intervals based on the selective inference framework to account for the model selection stage. We illustrate the methods in a dataset of a treatment for Alzheimer disease (normal response) and in a dataset of a treatment for prostate cancer (survival outcome). We also evaluate via simulations the performance of using the predicted individual treatment effect to identify subgroups where a novel treatment leads to better outcomes compared to a control treatment. © 2018 Ballarini et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.