Abstract
Screening trials are small trials used to decide whether an intervention is sufficiently promising to warrant a large confirmatory trial. Previous literature examined the situation where treatments are tested sequentially until one is considered sufficiently promising to take forward to a confirmatory trial. An important consideration for sponsors of clinical trials is how screening trials should be planned to maximize the efficiency of the drug development process. It has been found previously that small screening trials are generally the most efficient. In this paper we consider the design of screening trials in which multiple new treatments are tested simultaneously. We derive analytic formulae for the expected number of patients until a successful treatment is found, and propose methodology to search for the optimal number of treatments, and optimal sample size per treatment. We compare designs in which only the best treatment proceeds to a confirmatory trial and designs in which multiple treatments may proceed to a multi-arm confirmatory trial. We find that inclusion of a large number of treatments in the screening trial is optimal when only one treatment can proceed, and a smaller number of treatments is optimal when more than one can proceed. The designs we investigate are compared on a real-life set of screening designs. Copyright © 2013 John Wiley & Sons, Ltd.
Highlights
Bringing a drug to market is a long, expensive process [1] requiring multiple clinical trials of various sizes
We investigate two types of screening design for comparing multiple treatments: (1) the top-treatment design, in which if the test statistic of the most successful treatment at the screening stage is above a threshold, that treatment passes on to a confirmatory trial in which it is tested against a control treatment; and (2) the all-interesting-treatments design, in which all treatments with test statistics above a threshold go on to a multi-arm confirmatory trial
In this article we have explored optimal design of multi-arm screening trials
Summary
Bringing a drug to market is a long, expensive process [1] requiring multiple clinical trials of various sizes. A question of interest is whether changing the way screening trials are conducted could improve the efficiency of the drug development process as a whole. The problem of designing screening trials of a single new treatment to maximize the efficiency of a drug development program is discussed by Stallard [2]. The problem is considered from the view of a large funder of clinical trials, for whom many treatments are available for testing. Treatments are tested sequentially until one succeeds at the confirmatory trial. When the efficiency of the overall drug development process is considered, it has been found that conducting small screening trials on a larger number of treatments is efficient [3,4,5]
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