标题： 患者选择和患者排名结果在临床试验中的纳入方法比较 显示英文标题

标题： A comparison of approaches to incorporate patient-selected and patient-ranked outcomes in clinical trials

摘要： 患者为中心的药物开发的一个关键方面是确定和测量在临床试验中对患者重要的结果。 许多医学状况会影响多个症状领域，而一种共识方法来确定相关多个结果的相对重要性忽略了个体患者偏好的异质性。 患者选择的结果提供了一种纳入个体患者偏好的方式，如最近针对偏头痛治疗的监管指南所提出的，其中每位患者除了疼痛外，还会选择其最烦扰的偏头痛相关症状。 患者排名的结果也最近被提出，这进一步考虑了所有结果的相对重要性的完整排名。 可以使用复合DOOR（结果排名的可取性）终点来进行评估。 在本文中，我们比较了在多发性硬化症的两组随机对照试验背景下使用患者选择的结果与患者排名的结果的优势和劣势。 我们通过模拟比较了功效和第一类错误率，并讨论了使用这两种方法时的其他几个重要考虑因素。 显示英文摘要

摘要： 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.