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Introduction to Basket Trials

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Bayesian Adaptive Design for Immunotherapy and Targeted Therapy

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Abstract

With the advent of targeted therapies and immunotherapies, precision medicine has entered clinical practice. Basket trials provide an important approach to evaluating treatment effects of targeted therapies and immunotherapies. Under the basket trial, patients with the same genetic or molecular aberrations, regardless of their cancer types, are enrolled in the trial for evaluating the effect of a targeted agent. The basket trial allows for the incorporation of precision medicine into clinical trials. This chapter introduces several frequentist and Bayesian designs for basket trials, with a special focus on borrowing information across cancer types using Bayesian hierarchical model approaches. Software for some designs are described.

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Notes

  1. 1.

    https://www.mskcc.org/departments/epidemiology-biostatistics/biostatistics/ basket-trials.

  2. 2.

    Precisely, the poisson-binomial distribution should be used to compute these p values.

  3. 3.

    https://cran.r-project.org/web/packages/bhmbasket/vignettes/reproduceExNex.html.

  4. 4.

    http://glimmer.rstudio.com/brbnci/BasketTrials/.

  5. 5.

    https://github.com/brbnci/BasketTrials/blob/91f42fa3398c137dd427eae19f42dcb1eec0e703/global.R.

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Authors and Affiliations

  1. Department of Biostatistics, St. Jude Children’s Research Hospital, Memphis, TN, USA

    Haitao Pan

  2. Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Ying Yuan

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  1. Haitao Pan
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  2. Ying Yuan
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Correspondence to Haitao Pan .

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Pan, H., Yuan, Y. (2023). Introduction to Basket Trials. In: Bayesian Adaptive Design for Immunotherapy and Targeted Therapy. Springer, Singapore. https://doi.org/10.1007/978-981-19-8176-0_8

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