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A Bayesian group sequential small n sequential multiple‐assignment randomized trial

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  • Yan‐Cheng Chao
  • Thomas M. Braun
  • Roy N. Tamura
  • Kelley M. Kidwell

Abstract

A small n, sequential, multiple‐assignment, randomized trial (called ‘snSMART’) is a small sample multistage design where participants may be rerandomized to treatment on the basis of intermediate end points. This design is motivated by the ‘A randomized multicenter study for isolated skin vasculitis’ trial (NCT02939573): an on‐going snSMART design focusing on the evaluation of three drugs for isolated skin vasculitis. By formulating an interim decision rule for removing one of the treatments, we use a Bayesian model and the resulting posterior distributions to provide sufficient evidence that one treatment is inferior to the other treatments before enrolling more participants. By doing so, we can remove the worst performing treatment at an interim analysis and prevent the subsequent participants from receiving the removed treatment. On the basis of simulation results, we have evidence that the treatment response rates can still be unbiasedly and efficiently estimated in our new design, especially for the treatments with higher response rates. In addition, by adjusting the decision rule criteria for the posterior probabilities, we can control the probability of incorrectly removing an effective treatment.

Suggested Citation

  • Yan‐Cheng Chao & Thomas M. Braun & Roy N. Tamura & Kelley M. Kidwell, 2020. "A Bayesian group sequential small n sequential multiple‐assignment randomized trial," Journal of the Royal Statistical Society Series C, Royal Statistical Society, vol. 69(3), pages 663-680, June.
    • Handle: RePEc:bla:jorssc:v:69:y:2020:i:3:p:663-680
    DOI: 10.1111/rssc.12406
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    References listed on IDEAS

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    Cited by:

    1. Sidi Wang & Kelley M. Kidwell & Satrajit Roychoudhury, 2023. "Dynamic enrichment of Bayesian small‐sample, sequential, multiple assignment randomized trial design using natural history data: a case study from Duchenne muscular dystrophy," Biometrics, The International Biometric Society, vol. 79(4), pages 3612-3623, December.
    2. Cole Manschot & Eric Laber & Marie Davidian, 2023. "Interim monitoring of sequential multiple assignment randomized trials using partial information," Biometrics, The International Biometric Society, vol. 79(4), pages 2881-2894, December.

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