'All for one, one for all': Evaluating composite endpoints in clinical trials

摘要

The outcomes or endpoints that are chosen for a study are essentially the questions to be asked about the treatment: Will this treatment help patients live longer? Does this treatment cause more bleeding to occur? (The terms "outcomes" or "endpoints" can be used interchangeably; however, "endpoints" will be used in this article, as it implies something that can be measured.) Ideally, endpoints should be easy to choose depending on the questions the authors (and hopefully you, the readers) think need to be answered. However, there are important practical limitations as to which specific endpoints can be studied, as well as to how many different endpoints can be studied at the same time. One way to address either (or both) of these challenges is to use composite endpoints, but it is important to remember that doing so comes with its own limitations. These limitations need to be understood before the results of a study can be fully interpreted.