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Tree-based methods for individualized treatment regimes

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  • E. B. Laber
  • Y. Q. Zhao

Abstract

Individualized treatment rules recommend treatments on the basis of individual patient characteristics. A high-quality treatment rule can produce better patient outcomes, lower costs and less treatment burden. If a treatment rule learned from data is to be used to inform clinical practice or provide scientific insight, it is crucial that it be interpretable; clinicians may be unwilling to implement models they do not understand, and black-box models may not be useful for guiding future research. The canonical example of an interpretable prediction model is a decision tree. We propose a method for estimating an optimal individualized treatment rule within the class of rules that are representable as decision trees. The class of rules we consider is interpretable but expressive. A novel feature of this problem is that the learning task is unsupervised, as the optimal treatment for each patient is unknown and must be estimated. The proposed method applies to both categorical and continuous treatments and produces favourable marginal mean outcomes in simulation experiments. We illustrate it using data from a study of major depressive disorder.

Suggested Citation

  • E. B. Laber & Y. Q. Zhao, 2015. "Tree-based methods for individualized treatment regimes," Biometrika, Biometrika Trust, vol. 102(3), pages 501-514.
  • Handle: RePEc:oup:biomet:v:102:y:2015:i:3:p:501-514.
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    File URL: http://hdl.handle.net/10.1093/biomet/asv028
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    References listed on IDEAS

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    1. Su Xiaogang & Zhou Tianni & Yan Xin & Fan Juanjuan & Yang Song, 2008. "Interaction Trees with Censored Survival Data," The International Journal of Biostatistics, De Gruyter, vol. 4(1), pages 1-26, January.
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    Cited by:

    1. Jingxiang Chen & Haoda Fu & Xuanyao He & Michael R. Kosorok & Yufeng Liu, 2018. "Estimating individualized treatment rules for ordinal treatments," Biometrics, The International Biometric Society, vol. 74(3), pages 924-933, September.
    2. Hyung G. Park & Danni Wu & Eva Petkova & Thaddeus Tarpey & R. Todd Ogden, 2023. "Bayesian Index Models for Heterogeneous Treatment Effects on a Binary Outcome," Statistics in Biosciences, Springer;International Chinese Statistical Association, vol. 15(2), pages 397-418, July.
    3. Hongming Pu & Bo Zhang, 2021. "Estimating optimal treatment rules with an instrumental variable: A partial identification learning approach," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 83(2), pages 318-345, April.
    4. Chunrong Ai & Yue Fang & Haitian Xie, 2024. "Data-driven Policy Learning for a Continuous Treatment," Papers 2402.02535, arXiv.org.
    5. Ying Huang & Juhee Cho & Youyi Fong, 2021. "Threshold‐based subgroup testing in logistic regression models in two‐phase sampling designs," Journal of the Royal Statistical Society Series C, Royal Statistical Society, vol. 70(2), pages 291-311, March.
    6. Yingchao Zhong & Chang Wang & Lu Wang, 2021. "Survival Augmented Patient Preference Incorporated Reinforcement Learning to Evaluate Tailoring Variables for Personalized Healthcare," Stats, MDPI, vol. 4(4), pages 1-17, September.
    7. Shuxiao Chen & Bo Zhang, 2021. "Estimating and Improving Dynamic Treatment Regimes With a Time-Varying Instrumental Variable," Papers 2104.07822, arXiv.org.
    8. Emily L. Butler & Eric B. Laber & Sonia M. Davis & Michael R. Kosorok, 2018. "Incorporating Patient Preferences into Estimation of Optimal Individualized Treatment Rules," Biometrics, The International Biometric Society, vol. 74(1), pages 18-26, March.
    9. Qian Guan & Eric B. Laber & Brian J. Reich, 2016. "Comment," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 111(515), pages 936-942, July.
    10. Yizhe Xu & Tom H. Greene & Adam P. Bress & Brandon K. Bellows & Yue Zhang & Zugui Zhang & Paul Kolm & William S. Weintraub & Andrew S. Moran & Jincheng Shen, 2022. "An Efficient Approach for Optimizing the Cost-effective Individualized Treatment Rule Using Conditional Random Forest," Papers 2204.10971, arXiv.org.
    11. Zhang, Haixiang & Huang, Jian & Sun, Liuquan, 2020. "A rank-based approach to estimating monotone individualized two treatment regimes," Computational Statistics & Data Analysis, Elsevier, vol. 151(C).

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