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Net benefit index: Assessing the influence of a biomarker for individualized treatment rules

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  • Yiwang Zhou
  • Peter X.K. Song
  • Haoda Fu

Abstract

One central task in precision medicine is to establish individualized treatment rules (ITRs) for patients with heterogeneous responses to different therapies. Motivated from a randomized clinical trial for Type 2 diabetic patients on a comparison of two drugs, that is, pioglitazone and gliclazide, we consider a problem: utilizing promising candidate biomarkers to improve an existing ITR. This calls for a biomarker evaluation procedure that enables to gauge added values of individual biomarkers. We propose an assessment analytic, termed as net benefit index (NBI), that quantifies a contrast between the resulting gain and loss of treatment benefits when a biomarker enters ITR to reallocate patients in treatments. We optimize reallocation schemes via outcome weighted learning (OWL), from which the optimal treatment group labels are generated by weighted support vector machine (SVM). To account for sampling uncertainty in assessing a biomarker, we propose an NBI‐based test for a significant improvement over the existing ITR, where the empirical null distribution is constructed via the method of stratified permutation by treatment arms. Applying NBI to the motivating diabetes trial, we found that baseline fasting insulin is an important biomarker that leads to an improvement over an existing ITR based only on patient's baseline fasting plasma glucose (FPG), age, and body mass index (BMI) to reduce FPG over a period of 52 weeks.

Suggested Citation

  • Yiwang Zhou & Peter X.K. Song & Haoda Fu, 2021. "Net benefit index: Assessing the influence of a biomarker for individualized treatment rules," Biometrics, The International Biometric Society, vol. 77(4), pages 1254-1264, December.
    • Handle: RePEc:bla:biomet:v:77:y:2021:i:4:p:1254-1264
    DOI: 10.1111/biom.13373
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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.
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    4. 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.
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