A key aspect of patient-focused drug development is identifying and measuring outcomes that are important to patients in clinical trials. Many medical conditions affect multiple symptom domains, and a consensus approach to determine the relative importance of the associated multiple outcomes ignores the heterogeneity in individual patient preferences. Patient-selected outcomes offer one way to incorporate individual patient preferences, as proposed in recent regulatory guidance for the treatment for migraine, where each patient selects their most bothersome migraine-associated symptom in addition to pain. Patient-ranked outcomes have also recently been proposed, which go further and consider the full ranking of the relative importance of all the outcomes. This can be assessed using a composite DOOR (Desirability of Outcome Ranking) endpoint. In this paper, we compare the advantages and disadvantages of using patient-selected versus patient-ranked outcomes in the context of a two-arm randomised controlled trial for multiple sclerosis. We compare the power and type I error rate by simulation, and discuss several other important considerations when using the two approaches.

翻译：以患者为中心的药物开发的一个关键方面是在临床试验中识别和测量对患者重要的结局。许多疾病会影响多个症状域，而采用共识方法来确定相关多重结局的相对重要性，忽略了个体患者偏好的异质性。患者选择结局提供了一种纳入个体患者偏好的方法，正如近期针对偏头痛治疗的监管指南所提议的那样，即每位患者除了疼痛外，还需选择其最困扰的偏头痛相关症状。患者排序结局最近也被提出，其更进一步考虑了所有结局相对重要性的完整排序。这可以通过一个复合的DOOR（结局合意性排序）终点来评估。在本文中，我们比较了在多发性硬化症双臂随机对照试验背景下，使用患者选择结局与患者排序结局的优缺点。我们通过模拟比较了检验效能和I类错误率，并讨论了使用这两种方法时其他几个重要的考量因素。