Clinical Pharmacology and Pharmacometrics, Janssen Research & Development, LLC, 1400 McKean Road, PO Box 776, Spring House, Pennsylvania, 19477, USA.
AAPS J. 2019 Aug 26;21(6):102. doi: 10.1208/s12248-019-0370-6.
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.
临床试验终点通常采用有界结局评分（BOS）的形式，报告有限范围内的离散取值。从概念上讲，此类终点在性质上是有序分类的，但实际上它们通常被分析为连续变量，这可能导致数据范围违规和处理数据偏度的困难。已经提出了专门用于 BOS 数据的分析方法；然而，在如何比较可能的方法方面，药物计量学家之间仍然存在很多混淆。本评论回顾了药物计量学应用中使用的主要方法，并讨论了它们的理论和实际比较。讨论了一些在不同情况下从概念上吸引人的方法的预期性能，并提供了在实践中选择分析方法的指南。
