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Kuhlemeier, Alena ; Jaki, Thomas ; Witkiewitz, Katie ; Stuart, Elizabeth A. ; Van Horn, M. Lee

Validation of predicted individual treatment effects in out of sample respondents

Kuhlemeier, Alena, Jaki, Thomas , Witkiewitz, Katie, Stuart, Elizabeth A. and Van Horn, M. Lee (2024) Validation of predicted individual treatment effects in out of sample respondents. Statistics in Medicine 43 (22), pp. 4349-4360.

Date of publication of this fulltext: 22 Sep 2025 06:58
Article
DOI to cite this document: 10.5283/epub.77771


Abstract

Personalized medicine promises the ability to improve patient outcomes by tailoring treatment recommendations to the likelihood that any given patient will respond well to a given treatment. It is important that predictions of treatment response be validated and replicated in independent data to support their use in clinical practice. In this paper, we propose and test an approach for validating ...

Personalized medicine promises the ability to improve patient outcomes by tailoring treatment recommendations to the likelihood that any given patient will respond well to a given treatment. It is important that predictions of treatment response be validated and replicated in independent data to support their use in clinical practice. In this paper, we propose and test an approach for validating predictions of individual treatment effects with continuous outcomes across samples that uses matching in a test (validation) sample to match individuals in the treatment and control arms based on their predicted treatment response and their predicted response under control. To examine the proposed validation approach, we conducted simulations where test data is generated from either an identical, similar, or unrelated process to the training data. We also examined the impact of nuisance variables. To demonstrate the use of this validation procedure in the context of predicting individual treatment effects in the treatment of alcohol use disorder, we apply our validation procedure using data from a clinical trial of combined behavioral and pharmacotherapy treatments. We find that the validation algorithm accurately confirms validation and lack of validation, and also provides insights into cases where test data were generated under similar, but not identical conditions. We also show that the presence of nuisance variables detrimentally impacts algorithm performance, which can be partially reduced though the use of variable selection methods. An advantage of the approach is that it can be widely applied to different predictive methods.



Involved Institutions


Details

Item typeArticle
Journal or Publication TitleStatistics in Medicine
Publisher:Wiley
Volume:43
Number of Issue or Book Chapter:22
Page Range:pp. 4349-4360
Date29 July 2024
InstitutionsInformatics and Data Science > Department Machine Learning & Data Science > Lehrstuhl für Computational Statistics (Prof. Dr. Thomas Jaki)
Identification Number
ValueType
10.1002/sim.10187DOI
Keywordsindividual treatment effects, personalized medicine, validation
Dewey Decimal Classification000 Computer science, information & general works > 004 Computer science
StatusPublished
RefereedYes, this version has been refereed
Created at the University of RegensburgPartially
URN of the UB Regensburgurn:nbn:de:bvb:355-epub-777712
Item ID77771

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