Strategy for Designing In Vivo Dose–Response Comparison Studies.

ABSTRACT In preclinical drug discovery, at the step of lead optimization of a compound, in vivo experimentation can differentiate several compounds in terms of efficacy and potency in a biological system of whole living organisms. For the lead optimization study, it may be desirable to implement a dose–response design so that compound comparisons can be made from nonlinear curves fitted to the data. A dose–response design requires more thought relative to a simpler study design, needing parameters for the number of doses, the dose values, and the sample size per dose. This tutorial illustrates how to calculate statistical power, choose doses, and determine sample size per dose for a comparison of two or more dose–response curves for a future in vivo study. [ABSTRACT FROM AUTHOR]