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Estimating Individualized Treatment Regimes to Optimize Incremental Cost-Effectiveness Ratio

Author

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  • Xinyuan Dong

    (Amazon.com, Inc)

  • Ying-Qi Zhao

    (Fred Hutchinson Cancer Center)

Abstract

Medical decision making can be challenging due to the trade-off between improving clinical efficacy and the associated medical costs. Evaluation of the incremental cost-effectiveness ratio (ICER) of different treatment programs is important for cost-effectiveness analysis. Individualized treatment regimes (ITRs) that consider patient heterogeneity can lead to varying health benefits and costs. To identify a promising ITR that balances efficacy and cost, the ICER criterion can be used to evaluate the quality of the ITR. We propose a method that considers both health benefits and costs to derive the the optimal ITR. We utilize Dinkelbach’s algorithm to transform a fractional program into a parametric program, which is easier to handle. We compare our method to ITRs that only optimize a single outcome (benefits or costs) through extensive simulation studies and show that our approach performs satisfactorily. To demonstrate the practical application of our method, we apply it to the Multicenter Automatic Defibrillator Implantation Trial with Cardiac Resynchronization Therapy (MADIT-CRT) study, a randomized trial. Our approach can help identify an optimal ITR that balances the trade-off between clinical efficacy and medical costs. Overall, our method provides a valuable tool for medical decision making that takes into account patient heterogeneity and cost-effectiveness analysis.

Suggested Citation

  • Xinyuan Dong & Ying-Qi Zhao, 2025. "Estimating Individualized Treatment Regimes to Optimize Incremental Cost-Effectiveness Ratio," Statistics in Biosciences, Springer;International Chinese Statistical Association, vol. 17(2), pages 366-385, July.
  • Handle: RePEc:spr:stabio:v:17:y:2025:i:2:d:10.1007_s12561-024-09440-x
    DOI: 10.1007/s12561-024-09440-x
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    References listed on IDEAS

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    1. Yingqi Zhao & Donglin Zeng & A. John Rush & Michael R. Kosorok, 2012. "Estimating Individualized Treatment Rules Using Outcome Weighted Learning," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 107(499), pages 1106-1118, September.
    2. Hongwei Zhao & Chen Zuo & Shuai Chen & Heejung Bang, 2012. "Nonparametric Inference for Median Costs with Censored Data," Biometrics, The International Biometric Society, vol. 68(3), pages 717-725, September.
    3. E. B. Laber & Y. Q. Zhao, 2015. "Tree-based methods for individualized treatment regimes," Biometrika, Biometrika Trust, vol. 102(3), pages 501-514.
    4. Yuanjia Wang & Haoda Fu & Donglin Zeng, 2018. "Learning Optimal Personalized Treatment Rules in Consideration of Benefit and Risk: With an Application to Treating Type 2 Diabetes Patients With Insulin Therapies," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 113(521), pages 1-13, January.
    5. Juhee Lee & Peter F. Thall & Yuan Ji & Peter Müller, 2015. "Bayesian Dose-Finding in Two Treatment Cycles Based on the Joint Utility of Efficacy and Toxicity," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 110(510), pages 711-722, June.
    6. S. A. Murphy, 2003. "Optimal dynamic treatment regimes," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 65(2), pages 331-355, May.
    7. Ying-Qi Zhao & Donglin Zeng & Eric B. Laber & Michael R. Kosorok, 2015. "New Statistical Learning Methods for Estimating Optimal Dynamic Treatment Regimes," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 110(510), pages 583-598, June.
    8. Eric B. Laber & Daniel J. Lizotte & Bradley Ferguson, 2014. "Set-valued dynamic treatment regimes for competing outcomes," Biometrics, The International Biometric Society, vol. 70(1), pages 53-61, March.
    9. Xinlei Mi & Fei Zou & Ruoqing Zhu, 2019. "Bagging and deep learning in optimal individualized treatment rules," Biometrics, The International Biometric Society, vol. 75(2), pages 674-684, June.
    10. Xinyang Huang & Jin Xu, 2020. "Estimating individualized treatment rules with risk constraint," Biometrics, The International Biometric Society, vol. 76(4), pages 1310-1318, December.
    11. Xin Zhou & Nicole Mayer-Hamblett & Umer Khan & Michael R. Kosorok, 2017. "Residual Weighted Learning for Estimating Individualized Treatment Rules," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 112(517), pages 169-187, January.
    12. Martí, Rafael & Resende, Mauricio G.C. & Ribeiro, Celso C., 2013. "Multi-start methods for combinatorial optimization," European Journal of Operational Research, Elsevier, vol. 226(1), pages 1-8.
    13. Werner Dinkelbach, 1967. "On Nonlinear Fractional Programming," Management Science, INFORMS, vol. 13(7), pages 492-498, March.
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