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Full-Text Articles in Physical Sciences and Mathematics
Stochastic Optimization Of Adaptive Enrichment Designs For Two Subpopulations, Aaron Fisher, Michael Rosenblum
Stochastic Optimization Of Adaptive Enrichment Designs For Two Subpopulations, Aaron Fisher, Michael Rosenblum
Johns Hopkins University, Dept. of Biostatistics Working Papers
An adaptive enrichment design is a randomized trial that allows enrollment criteria to be modified at interim analyses, based on a preset decision rule. When there is prior uncertainty regarding treatment effect heterogeneity, these trial designs can provide improved power for detecting treatment effects in subpopulations. We present a simulated annealing approach to search over the space of decision rules and other parameters for an adaptive enrichment design. The goal is to minimize the expected number enrolled or expected duration, while preserving the appropriate power and Type I error rate. We also explore the benefits of parallel computation in the …
Sensitivity Of Trial Performance To Delay Outcomes, Accrual Rates, And Prognostic Variables Based On A Simulated Randomized Trial With Adaptive Enrichment, Tiachen Qian, Elizabeth Colantuoni, Aaron Fisher, Michael Rosenblum
Sensitivity Of Trial Performance To Delay Outcomes, Accrual Rates, And Prognostic Variables Based On A Simulated Randomized Trial With Adaptive Enrichment, Tiachen Qian, Elizabeth Colantuoni, Aaron Fisher, Michael Rosenblum
Johns Hopkins University, Dept. of Biostatistics Working Papers
Adaptive enrichment designs involve rules for restricting enrollment to a subset of the population during the course of an ongoing trial. This can be used to target those who benefit from the experimental treatment. To leverage prognostic information in baseline variables and short-term outcomes, we use a semiparametric, locally efficient estimator, and investigate its strengths and limitations compared to standard estimators. Through simulation studies, we assess how sensitive the trial performance (Type I error, power, expected sample size, trial duration) is to different design characteristics. Our simulation distributions mimic features of data from the Alzheimer’s Disease Neuroimaging Initiative, and involve …