Identification of Predicted Individual Treatment Effects (PITE) in randomized clinical trials

Lamont, Andrea E. and Lyons, Mike and Jaki, Thomas Friedrich and Stuart, E. A. and Feaster, Daniel and Ishwaran, H. and Tharmaratnam, Kukatharmini and Van Horn, M. Lee (2018) Identification of Predicted Individual Treatment Effects (PITE) in randomized clinical trials. Statistical Methods in Medical Research, 27 (1). pp. 142-157. ISSN 0962-2802

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Abstract

In most medical research, treatment effectiveness is assessed using the average treatment effect or some version of subgroup analysis. The practice of individualized or precision medicine, however, requires new approaches that predict how an individual will respond to treatment, rather than relying on aggregate measures of effect. In this study, we present a conceptual framework for estimating individual treatment effects, referred to as predicted individual treatment effects. We first apply the predicted individual treatment effect approach to a randomized controlled trial designed to improve behavioral and physical symptoms. Despite trivial average effects of the intervention, we show substantial heterogeneity in predicted individual treatment response using the predicted individual treatment effect approach. The predicted individual treatment effects can be used to predict individuals for whom the intervention may be most effective (or harmful). Next, we conduct a Monte Carlo simulation study to evaluate the accuracy of predicted individual treatment effects. We compare the performance of two methods used to obtain predictions: multiple imputation and non-parametric random decision trees. Results showed that, on average, both predictive methods produced accurate estimates at the individual level; however, the random decision trees tended to underestimate the predicted individual treatment effect for people at the extreme and showed more variability in predictions across repetitions compared to the imputation approach. Limitations and future directions are discussed.

Item Type:
Journal Article
Journal or Publication Title:
Statistical Methods in Medical Research
Additional Information:
The final, definitive version of this article has been published in the Journal, Statistical Methods in Medical Research, 27 (1), 2018, © SAGE Publications Ltd, 2018 by SAGE Publications Ltd at the Statistical Methods in Medical Research page: http://journals.sagepub.com/home/smm on SAGE Journals Online: http://journals.sagepub.com/
Uncontrolled Keywords:
/dk/atira/pure/subjectarea/asjc/2600/2613
Subjects:
ID Code:
77529
Deposited By:
Deposited On:
07 Jan 2016 11:54
Refereed?:
Yes
Published?:
Published
Last Modified:
25 Oct 2020 03:42