Competing designs for phase I clinical trials: a review |
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Authors: | Rosenberger William F Haines Linda M |
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Affiliation: | Department of Mathematics and Statistics, University of Maryland, Baltimore County, 1000 Hilltop Circle, Baltimore, MD 21250, USA. |
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Abstract: | Phase I clinical trials are typically small, uncontrolled studies designed to determine a maximum tolerated dose of a drug which will be used in further testing. Two divergent schools have developed in designing phase I clinical trials. The first defines the maximum tolerated dose as a statistic computed from data, and hence it is identified, rather than estimated. The second defines the maximum tolerated dose as a parameter of a monotonic dose-response curve, and hence is estimated. We review techniques from both philosophies. The goal is to present these methods in a single package, to compare them from philosophical and statistical grounds, to hopefully clear up some common misconceptions, and to make a few recommendations. This paper is not a review of simulation studies of these designs, nor does it present any new simulations comparing these designs. |
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Keywords: | continual reassessment method decision theory escalation with overdose control maximum tolerated dose quantile estimation random walk rules |
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