Optimal designs for a multiresponse Emax model and efficient parameter estimation

Abstract

The aim of dose finding studies is sometimes to estimate parameters in a fitted model. The precision of the parameter estimates should be as high as possible. This can be obtained by increasing the number of subjects in the study, N, choosing a good and efficient estimation approach, and by designing the dose finding study in an optimal way. Increasing the number of subjects is not always feasible because of increasing cost, time limitations, etc. In this paper, we assume fixed N and consider estimation approaches and study designs for multiresponse dose finding studies. We work with diabetes dose-response data and compare a system estimation approach that fits a multiresponse Emax model to the data to equation-by-equation estimation that fits uniresponse Emax models to the data. We then derive some optimal designs for estimating the parameters in the multi- and uniresponse Emax model and study the efficiency of these designs.

Keywords: Dose-response studies; Multiresponse Emax models; Optimal designs; Precise parameter estimates; System estimation.