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Methods in dynamic treatment regimens using observational healthcare data: A systematic review

Title: Methods in dynamic treatment regimens using observational healthcare data: A systematic review
Authors: Liang, David; Paul, Animesh Kumar; Weir, Daniala L; Deneer, Vera H M; Greiner, Russell; Siebes, Arno; Gardarsdottir, Helga; Pharmacoepidemiology and Clinical Pharmacology; Afd Pharmacoepi & Clinical Pharmacology; PECP - Centre for Pharmacoepidemiology; PECP – Centre for Clinical Therapeutics; Sub Algorithmic Data Analysis
Publication Year: 2025
Subject Terms: Causal inference; Counterfactual methods; Dynamic treatment regimens; G-methods; Machine learning; Reinforcement learning; Software; Computer Science Applications; Health Informatics; SDG 3 - Good Health and Well-being
Description: We present a systematic review of methods used to estimate Dynamic Treatment Regimens (DTR) using observational healthcare data and provide a brief summary of their strengths and weaknesses, evaluation metrics, and suitable research problem settings. We considered all observational studies identified in PubMed or EMBASE between January 1950 until January 2022, including only studies that evaluated medical treatments or interventions as exposure and/or outcome in patients and where DTRs were estimated. 83 studies met our inclusion criteria; 44.6% estimating DTR utilizing reinforcement learning, 18.1% utilizing counterfactual-based models, 12.1% utilizing classification-based methods, and 9.6% utilized g-methods. Among the studies analyzed, 28.9% aimed to replicate human expert DTRs, while 71.1% aimed to refine and improve existing DTRs. Approximately two-thirds of studies (65.1%) reported the assumptions required for their applied methods, such as exchangeability, positivity, consistency, and Markov property. Most of the studies (83.1%) estimated DTRs with more than two treatment options; 50.6% mentioned time-varying confounders, only a few estimated conditional average treatment effects (7.2%). Most (85.5%) validated their methods, with 32.5% using expected outcomes (e.g., survival rates), 26.5% employing simulated data, and 25.3% conducting direct comparisons with observational data.
Document Type: article in journal/newspaper
File Description: application/pdf
Language: English
ISSN: 0169-2607
Relation: https://dspace.library.uu.nl/handle/1874/475722
Availability: https://dspace.library.uu.nl/handle/1874/475722
Rights: info:eu-repo/semantics/OpenAccess
Accession Number: edsbas.65C0556B
Database: BASE