Design of clinical trials with multiple end points of different types
Seminar Room 1, Newton Institute
Several correlated endpoints are observed in almost any clinical trial. Typically one of them is claimed as a primary end point and the design (dose allocation and sample size) is driven by a single response model. I discuss the design problem with multiple end points which potentially may be of different nature. For instance, the efficacy end point may be continuous while the toxicity end point may be discrete. I emphasize the necessity to differentiate between the responses and utility functions. The response (end point) functions are what we observe while the utility functions are what should be reported or used in the decision making process. The discussed criteria of optimality are related to the latter and usually describe the precision of their estimators.