Preparing a clinical outcomes assessment set for nephrotic syndrome [Prepare-NS]

摘要 PREPARE-NS计划将开发和建立一套核心的临床心理测量特性， 肾病综合征的结局评估（COA），主要关注液体超负荷（FO）。期间 在UG 3阶段，将与FDA合作召集一个由专家和利益相关者组成的多元化团队， 确认条件，措施和锚点，并设计必要的研究，以确定一个严格和耐心的- 居中的FO端点定义和度量集。UH 3将专注于测试和实施核心集 在UG 3阶段确定的COA。在UH 3阶段进行的研究将使我们能够完善FO COA。最后的活动将侧重于向公众广泛提供最后一套核心行动方案， 用于试验。在这两个阶段，我们将与项目利益相关者密切协商， 临床医生专家小组，从患者、临床医生、监管机构、支付者和药物管理者的角度出发， 开发者利用这些资源，并依靠我们团队广泛的临床，COA开发， 心理测量专业知识，我们将产生一套核心的高度相关的COA，将推进以患者为中心的 肾病综合征的药物开发

Abstract The PREPARE-NS program will develop and establish the psychometric properties of a core set of clinical outcome assessments (COAs) for nephrotic syndrome, with primary focus on fluid overload (FO). During the UG3 phase, a diverse team of experts and stakeholders will be convened, in cooperation with the FDA, to confirm conditions, measures and anchors and design the studies necessary to identify a rigorous and patient- centered FO endpoint definition and measure set. The UH3 will focus on testing and implementing the core set of COAs identified in the UG3 phase. The research conducted in the UH3 phase will allow us to refine the FO COAs. The final activities will be focused on making the final core set of COAs widely available to the public for use in trials. For both phases, we will work in close consultation with the project stakeholder engagement and clinician expert panels, drawing from the perspectives of patients, clinicians, regulators, payers, and drug developers. Tapping these resources, and relying on our team's wide-ranging clinical, COA development, and psychometric expertise, we will generate a core set of highly-relevant COAs that will advance patient-focused drug development in nephrotic syndrome.