Migraine Clinical Outcome Assessment System (MiCOAS)

项目总结摘要 拟议的偏头痛临床结果评估系统(MiCOAS)旨在改进和标准化 偏头痛临床试验中的终点和这些终点的评估。这种标准化的临床结果 评估(COA)，为纳入患者的声音而开发，并得到黄金标准的经验支持 仪器开发技术，将有助于解释偏头痛临床和跨临床结果 试验，进而推进偏头痛治疗方法的开发和批准。UG3阶段的 格兰特将首先召集一组利益相关者(实质性研究专家、患者权益倡导者、临床医生、 心理测量师、监管者和付款人)来指导项目(目标1)。全面系统地审查 偏头痛文献将被用于全面了解目前偏头痛的结果，包括急性和非急性偏头痛 偏头痛预防性试验(AIM 2)。使用以患者为中心的访谈(AIM)进行定性研究的结果 3)将确保在目标1和目标2期间确定为开发/改进目标的终端是 对病人有意义。 UH3拨款阶段将使用额外的以患者为中心的定性研究，以充分了解患者 关于UG3中确定的目标端点以及哪些方面对它们最重要的经验， 患者在讨论所述终点时的语言，以及以前未涉及的任何可能的研究领域 已识别(目标4)。根据患者访谈结果，将使用GOLD- 标准技术。在开发/提炼预防性质量的预期情况下- 生活/影响/残疾类型测量，将设计和执行偏头痛患者的新数据收集 并将采用黄金标准的心理测量技术对应聘者进行经验评估和提炼 项目，最终确定最终评估所选的项目，并评估初步有效性和适用性 结果评估。还将为草案设计、执行和分析单独的数据收集 用于急性偏头痛试验的结果评估(AIM 5)。这笔赠款的主要目的是 开发一套公开可用的偏头痛终点及其相关的偏头痛COA核心集合。鉴于此， 通过科学渠道以演示文稿和手稿的形式适当传播研究结果 必需(目标6)。成功完成拟议项目的总体目标将导致标准化 以及经验支持的偏头痛COA用于临床试验，这些试验对患者和统计数字都有意义 很健壮。

PROJECT SUMMARY ABSTRACT The proposed Migraine Clinical Outcome Assessment System (MiCOAS) aims to improve and standardize endpoints and the assessment of those endpoints in migraine clinical trials. Such standardized clinical outcome assessments (COAs), developed to incorporate the patient voice and empirically supported by gold-standard instrument development techniques, will aid in the interpretability of results from and across migraine clinical trials and, by extension, advance the development and approval of migraine treatments. The UG3 phase of the grant will first assemble a group of stakeholders (substantive research experts, patient advocates, clinicians, psychometricians, regulators, and payers) to guide the project (AIM 1). Comprehensive systematic review of the migraine literature will be undertaken to fully understand current migraine outcomes, in both acute and preventative migraine trials (AIM 2). Results from qualitative research using patient-centered interviews (AIM 3) will ensure that endpoints identified during AIM 1 and AIM 2 as targets for development/refinement are meaningful to patients. The UH3 grant stage will use additional patient-centered qualitative research to fully understand the patient experience with respect to the target endpoints identified in UG3 and what aspects are most important to them, patient language in discussing said endpoints, and any possible areas of study that were not previously identified (AIM 4). Using the patient-interview results, outcome measures will be developed using gold- standard techniques. In the expected case of developing/refining a preventative quality-of- life/impact/disability-type measure, a new data collection of migraine patients will be designed and executed and gold-standard psychometric techniques will be employed to empirically evaluate and refine candidate items, finalize the items selected to the final assessment, and evaluate the initial validity and fit-for-purpose of the outcome assessment. A separate data collection will also be designed, executed, and analyzed for the draft outcome assessments intended for acute migraine trials (AIM 5). The overarching purpose of this grant is to develop a publicly available core set of migraine endpoints and their related COAs for migraine. Given this, proper dissemination of findings through scientific outlets in the form of presentations and manuscripts is essential (AIM 6). Successful completion of the overall goal of the proposed project will result in standardized and empirically-supported migraine COAs for clinical trials that are meaningful to patients and statistically robust.