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J Biopharm Stat. 2007;17(6):1085-96.

Dose finding designs for continuous responses and binary utility.

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GlaxoSmithKline, Collegeville, Pennsylvania, USA.


Often in clinical trials the observed responses are continuous but a regulatory agency will approve the drug only if the probability is sufficiently large that the efficacy measure exceeds a predefined threshold and the toxicity does not exceed another given threshold. Thus the measure of interest (utility) is based on dichotomized responses. We consider normally distributed correlated responses and build a utility function using the probit transform. Locally optimal designs are used as benchmarks for more practical designs such as composite and adaptive designs. We focus on D-criterion (i.e., all parameters of the dose-response model are of interest) and consider only two-stage designs. It is shown that the practice of reporting dichotomized responses leads to a substantial loss in the precision of estimated parameters (or in the power loss).

[Indexed for MEDLINE]

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