Optimal allocation to treatments in a sequential multiple assignment randomized trial (2024)

Abstract

One of the main questions in the design of a trial is how many subjects should be assigned to each treatment condition. Previous research has shown that equal randomization is not necessarily the best choice. We study the optimal allocation for a novel trial design, the sequential multiple assignment randomized trial, where subjects receive a sequence of treatments across various stages. A subject's randomization probabilities to treatments in the next stage depend on whether he or she responded to treatment in the current stage. We consider a prototypical sequential multiple assignment randomized trial design with two stages. Within such a design, many pairwise comparisons of treatment sequences can be made, and a multiple-objective optimal design strategy is proposed to consider all such comparisons simultaneously. The optimal design is sought under either a fixed total sample size or a fixed budget. A Shiny App is made available to find the optimal allocations and to evaluate the efficiency of competing designs. As the optimal design depends on the response rates to first-stage treatments, maximin optimal design methodology is used to find robust optimal designs. The proposed methodology is illustrated using a sequential multiple assignment randomized trial example on weight loss management.

Original languageEnglish
Pages (from-to)2471-2484
Number of pages14
JournalStatistical Methods in Medical Research
Volume30
Issue number11
Early online date23 Sept 2021
DOIs
Publication statusPublished - Nov 2021

Keywords

  • cost constraint
  • efficiency
  • maximin designs
  • optimal allocation
  • response rates
  • sequential multiple assignment randomized trial trials

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  • 09622802211037066Final published version, 791 KBLicence: CC BY

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    Morciano, A. (2021). Optimal allocation to treatments in a sequential multiple assignment randomized trial. Statistical Methods in Medical Research, 30(11), 2471-2484. https://doi.org/10.1177/09622802211037066

    Morciano, Andrea ; Moerbeek, Mirjam. / Optimal allocation to treatments in a sequential multiple assignment randomized trial. In: Statistical Methods in Medical Research. 2021 ; Vol. 30, No. 11. pp. 2471-2484.

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    title = "Optimal allocation to treatments in a sequential multiple assignment randomized trial",

    abstract = "One of the main questions in the design of a trial is how many subjects should be assigned to each treatment condition. Previous research has shown that equal randomization is not necessarily the best choice. We study the optimal allocation for a novel trial design, the sequential multiple assignment randomized trial, where subjects receive a sequence of treatments across various stages. A subject's randomization probabilities to treatments in the next stage depend on whether he or she responded to treatment in the current stage. We consider a prototypical sequential multiple assignment randomized trial design with two stages. Within such a design, many pairwise comparisons of treatment sequences can be made, and a multiple-objective optimal design strategy is proposed to consider all such comparisons simultaneously. The optimal design is sought under either a fixed total sample size or a fixed budget. A Shiny App is made available to find the optimal allocations and to evaluate the efficiency of competing designs. As the optimal design depends on the response rates to first-stage treatments, maximin optimal design methodology is used to find robust optimal designs. The proposed methodology is illustrated using a sequential multiple assignment randomized trial example on weight loss management.",

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    Morciano, A 2021, 'Optimal allocation to treatments in a sequential multiple assignment randomized trial', Statistical Methods in Medical Research, vol. 30, no. 11, pp. 2471-2484. https://doi.org/10.1177/09622802211037066

    Optimal allocation to treatments in a sequential multiple assignment randomized trial. / Morciano, Andrea; Moerbeek, Mirjam.
    In: Statistical Methods in Medical Research, Vol. 30, No. 11, 11.2021, p. 2471-2484.

    Research output: Contribution to journalArticleAcademicpeer-review

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    T1 - Optimal allocation to treatments in a sequential multiple assignment randomized trial

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    AU - Moerbeek, Mirjam

    N1 - Funding Information:The authors received no financial support for the research, authorship and/or publication of this article.Publisher Copyright:© The Author(s) 2021.

    PY - 2021/11

    Y1 - 2021/11

    N2 - One of the main questions in the design of a trial is how many subjects should be assigned to each treatment condition. Previous research has shown that equal randomization is not necessarily the best choice. We study the optimal allocation for a novel trial design, the sequential multiple assignment randomized trial, where subjects receive a sequence of treatments across various stages. A subject's randomization probabilities to treatments in the next stage depend on whether he or she responded to treatment in the current stage. We consider a prototypical sequential multiple assignment randomized trial design with two stages. Within such a design, many pairwise comparisons of treatment sequences can be made, and a multiple-objective optimal design strategy is proposed to consider all such comparisons simultaneously. The optimal design is sought under either a fixed total sample size or a fixed budget. A Shiny App is made available to find the optimal allocations and to evaluate the efficiency of competing designs. As the optimal design depends on the response rates to first-stage treatments, maximin optimal design methodology is used to find robust optimal designs. The proposed methodology is illustrated using a sequential multiple assignment randomized trial example on weight loss management.

    AB - One of the main questions in the design of a trial is how many subjects should be assigned to each treatment condition. Previous research has shown that equal randomization is not necessarily the best choice. We study the optimal allocation for a novel trial design, the sequential multiple assignment randomized trial, where subjects receive a sequence of treatments across various stages. A subject's randomization probabilities to treatments in the next stage depend on whether he or she responded to treatment in the current stage. We consider a prototypical sequential multiple assignment randomized trial design with two stages. Within such a design, many pairwise comparisons of treatment sequences can be made, and a multiple-objective optimal design strategy is proposed to consider all such comparisons simultaneously. The optimal design is sought under either a fixed total sample size or a fixed budget. A Shiny App is made available to find the optimal allocations and to evaluate the efficiency of competing designs. As the optimal design depends on the response rates to first-stage treatments, maximin optimal design methodology is used to find robust optimal designs. The proposed methodology is illustrated using a sequential multiple assignment randomized trial example on weight loss management.

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    KW - response rates

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    Morciano A, Moerbeek M. Optimal allocation to treatments in a sequential multiple assignment randomized trial. Statistical Methods in Medical Research. 2021 Nov;30(11):2471-2484. Epub 2021 Sept 23. doi: 10.1177/09622802211037066

    Optimal allocation to treatments in a sequential multiple assignment randomized trial (2024)

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