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Bayesian Phase I/II Biomarker-Based Dose Finding for Precision Medicine With Molecularly Targeted Agents

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  • Beibei Guo
  • Ying Yuan

Abstract

The optimal dose for treating patients with a molecularly targeted agent may differ according to the patient's individual characteristics, such as biomarker status. In this article, we propose a Bayesian phase I/II dose-finding design to find the optimal dose that is personalized for each patient according to his/her biomarker status. To overcome the curse of dimensionality caused by the relatively large number of biomarkers and their interactions with the dose, we employ canonical partial least squares (CPLS) to extract a small number of components from the covariate matrix containing the dose, biomarkers, and dose-by-biomarker interactions. Using these components as the covariates, we model the ordinal toxicity and efficacy using the latent-variable approach. Our model accounts for important features of molecularly targeted agents. We quantify the desirability of the dose using a utility function and propose a two-stage dose-finding algorithm to find the personalized optimal dose according to each patient's individual biomarker profile. Simulation studies show that our proposed design has good operating characteristics, with a high probability of identifying the personalized optimal dose. Supplementary materials for this article are available online.

Suggested Citation

  • Beibei Guo & Ying Yuan, 2017. "Bayesian Phase I/II Biomarker-Based Dose Finding for Precision Medicine With Molecularly Targeted Agents," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 112(518), pages 508-520, April.
    • Handle: RePEc:taf:jnlasa:v:112:y:2017:i:518:p:508-520
    DOI: 10.1080/01621459.2016.1228534
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    References listed on IDEAS

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    1. Ying Yuan & Guosheng Yin, 2009. "Bayesian dose finding by jointly modelling toxicity and efficacy as time‐to‐event outcomes," Journal of the Royal Statistical Society Series C, Royal Statistical Society, vol. 58(5), pages 719-736, December.
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    6. M.-K. Riviere & Y. Yuan & F. Dubois & S. Zohar, 2015. "A Bayesian dose finding design for clinical trials combining a cytotoxic agent with a molecularly targeted agent," Journal of the Royal Statistical Society Series C, Royal Statistical Society, vol. 64(1), pages 215-229, January.
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