Continual reassessment and related designs in dose-finding studies

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چکیده

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Continual Reassessment and Related Dose-Finding Designs.

During the last twenty years there have been considerable methodological developments in the design and analysis of Phase 1, Phase 2 and Phase 1/2 dose-finding studies. Many of these developments are related to the continual reassessment method (CRM), first introduced by O'Quigley, Pepe and Fisher (1990). CRM models have proven themselves to be of practical use and, in this discussion, we inves...

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In the dose–finding setting, we discuss and compare adaptive designs, including those which allocate each new patient (or a cohort of patients) to the dose currently viewed as the best one, for example, the seemingly most efficacious. We call this type of allocation “best intention” designs and found that, similar to other areas of life, the best intention may pave the road to rather disastrous...

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A major practical impediment when implementing adaptive dose-finding designs is that the toxicity outcome used by the decision rules may not be observed shortly after the initiation of the treatment. To address this issue, we propose the data augmentation continual re-assessment method (DA-CRM) for dose finding. By naturally treating the unobserved toxicities as missing data, we show that such ...

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ژورنال

عنوان ژورنال: Statistics in Medicine

سال: 2011

ISSN: 0277-6715

DOI: 10.1002/sim.4215