Dose-Finding Experiments in Clinical Trials

Nancy Flournoy (Univeristy of Missouri)

Friday 7th October, 2011 15:00-16:00 Maths 203

Abstract

Consider two situations. In one, toxicity increases with dose. In the second, one considers efficacy in addition, and assumes efficacy increases with dose - so except at the extremes, the probability of efficacy without toxicity (success) will increase up to a point at which the toxicity is great enough to cause it to turn down. In the first case, typically, one seeks to identify a dose with a prescribed toxicity rate; in the second case, one seeks to identify the dose that maximizes the probability of success. These goals can be posed in terms of estimation or dose selection, as a finite number of doses are typically permitted. Two common classes of procedures that differ in many fundamental ways are described: up-and-down designs and best intention designs.

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