On the Comparison of Methods in Analyzing Bounded Outcome Score Data

Abstract

Clinical trial endpoints often take the form of bounded outcome scores (BOS) which report a discrete set of values on a finite range. Conceptually such endpoints are ordered categorical in nature, but in practice they are often analyzed as continuous variables, which may result in data range violations and difficulties to handle data skewness. Analysis methods dedicated for BOS data have been proposed; however, much confusion exists among pharmacometricians on how to compare the possible methods. This commentary reviews the main methods used in pharmacometrics applications and discusses their theoretical and practical comparisons. The expected performance of some conceptually appealing methods in different situations is discussed, and a guideline is provided on selecting analysis methods in practice.