Other Weighted Effect Measures

Abstract

A number of other effect measures and analysis approaches for composite endpoints which take account of the different clinical relevance of the components were proposed in the literature; compare, for example, Lachin and Bebu (Clin Trials 12:627–633, 2015), Bakal et al. (Stat Methods Med Res 24:980–988, 2015), Bakal et al. (Eur Heart J 34:903–908, 2013), Pocock et al. (Eur Heart J 33:176–182, 2012), Buyse (Stat Med 29:3245–3257, 2010), and Hallstrom et al. (Control Clin Trials 13:148–155, 1992). As it goes beyond the scope of this book to present a complete and systematic literature review on weighted approaches, only a few exemplary methods will be presented and discussed within this chapter. Many of the weighting approaches presented in the literature should be used and interpreted with care as they are subject to general mathematical concerns. For example, unlike the weighting approaches presented in the previous chapters, many of the approaches proposed in the literature combine first and subsequent events irrespective of their underlying correlation structure and irrespective of the competing risk situation. Moreover, most of these approaches ignore the observational period making the approaches sensitive to changes in the follow-up distribution. Therefore, we generally recommend to apply weighted composite effect measures only when the properties of the method are really understood which usually requires a thorough mathematical look on the methodological aspects.