Machine learning approaches to evaluate heterogeneous treatment effects in randomized controlled trials: a scoping review.

Journal: Journal of clinical epidemiology
PMID: 39305940

Abstract

BACKGROUND AND OBJECTIVES: Estimating heterogeneous treatment effects (HTEs) in randomized controlled trials (RCTs) has received substantial attention recently. This has led to the development of several statistical and machine learning (ML) algorithms to assess HTEs through identifying individualized treatment effects. However, a comprehensive review of these algorithms is lacking. We thus aimed to catalog and outline currently available statistical and ML methods for identifying HTEs via effect modeling using clinical RCT data and summarize how they have been applied in practice.

Authors

  • Kosuke Inoue
    Department of Social Epidemiology, Graduate School of Medicine, Kyoto University, Sakyo, Kyoto, Japan.
  • Motohiko Adomi
    Department of Epidemiology, Harvard T.H. Chan School of Public Health, Boston, MA, USA.
  • Orestis Efthimiou
    Department of Psychiatry, Warneford Hospital, University of Oxford, Oxford, OX3 7JX, UK.
  • Toshiaki Komura
    Department of Epidemiology, School of Public Health, Boston University, Boston, MA, USA.
  • Kenji Omae
    Department of Innovative Research and Education for Clinicians and Trainees (DiRECT), Fukushima Medical University Hospital, Fukushima, Japan.
  • Akira Onishi
    Department of Advanced Medicine for Rheumatic Diseases, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Yusuke Tsutsumi
    Scientific Research Works Peer Support Group (SRWS-PSG), Osaka, Japan.
  • Tomoko Fujii
    Intensive Care Unit, Jikei University Hospital, Tokyo, Japan; Departments of Health Promotion and Human Behavior and of Clinical Epidemiology, Kyoto University Graduate School of Medicine/School of Public Health, Kyoto, Japan.
  • Naoki Kondo
    Department of Social Epidemiology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
  • Toshi A Furukawa
    Department of Health Promotion and Human Behavior, Kyoto University Graduate School of Medicine/School of Public Health, Kyoto University, Kyoto, Japan.

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