Optimal allocation to treatments in a sequential multiple assignment randomized trial

Andrea Morciano, Mirjam Moerbeek

Research output: Contribution to journalArticleAcademicpeer-review

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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