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Stat Med. 2014 Oct 15;33(23):4017-28. doi: 10.1002/sim.6200. Epub 2014 May 12.

A Bayesian design for phase II clinical trials with delayed responses based on multiple imputation.

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Division of Clinical and Translational Sciences, Department of Internal Medicine, Medical School, The University of Texas Health Science Center at Houston, Houston, TX 77030, U.S.A.; Biostatistics/Epidemiology/Research Design Core, Center for Clinical and Translational Sciences, The University of Texas Health Science Center at Houston, Houston, TX 77030, U.S.A.


Interim monitoring is routinely conducted in phase II clinical trials to terminate the trial early if the experimental treatment is futile. Interim monitoring requires that patients' responses be ascertained shortly after the initiation of treatment so that the outcomes are known by the time the interim decision must be made. However, in some cases, response outcomes require a long time to be assessed, which causes difficulties for interim monitoring. To address this issue, we propose a Bayesian trial design to allow for continuously monitoring phase II clinical trials in the presence of delayed responses. We treat the delayed responses as missing data and handle them using a multiple imputation approach. Extensive simulations show that the proposed design yields desirable operating characteristics under various settings and dramatically reduces the trial duration.


continuous monitoring; delayed responses; multiple imputation

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Free PMC Article

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