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Designing precision medicine trials to yield a greater population impact

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  • Ying‐Qi Zhao
  • Michael L. LeBlanc

Abstract

Traditionally, a clinical trial is conducted comparing treatment to standard care for all patients. However, it could be inefficient given patients’ heterogeneous responses to treatments, and rapid advances in the molecular understanding of diseases have made biomarker‐based clinical trials increasingly popular. We propose a new targeted clinical trial design, termed as Max‐Impact design, which selects the appropriate subpopulation for a clinical trial and aims to optimize population impact once the trial is completed. The proposed design not only gains insights on the patients who would be included in the trial but also considers the benefit to the excluded patients. We develop novel algorithms to construct enrollment rules for optimizing population impact, which are fairly general and can be applied to various types of outcomes. Simulation studies and a data example from the SWOG Cancer Research Network demonstrate the competitive performance of our proposed method compared to traditional untargeted and targeted designs.

Suggested Citation

  • Ying‐Qi Zhao & Michael L. LeBlanc, 2020. "Designing precision medicine trials to yield a greater population impact," Biometrics, The International Biometric Society, vol. 76(2), pages 643-653, June.
    • Handle: RePEc:bla:biomet:v:76:y:2020:i:2:p:643-653
    DOI: 10.1111/biom.13161
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    References listed on IDEAS

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    1. 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.
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    Cited by:

    1. Rosamarie Frieri & William Fisher Rosenberger & Nancy Flournoy & Zhantao Lin, 2023. "Design considerations for two‐stage enrichment clinical trials," Biometrics, The International Biometric Society, vol. 79(3), pages 2565-2576, September.
    2. Nigel Stallard, 2023. "Adaptive enrichment designs with a continuous biomarker," Biometrics, The International Biometric Society, vol. 79(1), pages 9-19, March.

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