Data, Modeling,and Coordination Center for PrecISE Network

摘要 此U24应用程序的目标是建立数据、建模和协调中心(DMCC) NHLBI针对重度和/或易加重哮喘的精确干预(PRECISE)网络。这个 PRECISE的目标是精确地进行顺序的、适应性的、第二阶段/概念验证试验 对重症哮喘患者分层人群的干预。严重哮喘患者有 异质性，因此是一个很难治疗的群体。精准医疗干预，重点是 确定针对个别患者的最佳治疗方法，而不是针对大多数患者的最佳治疗方法，可能会 改善这一复杂患者群体的总体结局。精密网络即将解决的问题 这些问题。其成功的核心将是一个创新、前瞻性和富有成效的协调中心 这为参与临床中心的研究人员带来了凝聚力，他们分享了他们的愿景并提供了 精确试验的科学领导、组织支持和分析结果。关键要素将 加强协作和研究进展，包括经验丰富的领导、创新的设计和分析 方法论(例如，顺序自适应设计、机器学习、神经网络、Q学习)、 全面的研究运营支持、最先进的数据管理系统和卓越的 行政支持。我们的基础设施将同时提供公共和私人网站，其中包括 面向公众、参与者和调查人员的信息页面、常见问题、学习材料、 项目日历、通信枢纽等关键功能。我们将开发一种安全、用户友好的 高级数据管理系统，包括基于Web的调查、数据输入和研究访问 管理(包括全面报告、生物样本跟踪和基于业绩的报销 工具)、与外部应用程序的集成以及质量控制。我们的研究运作和管理 团队将通过促进沟通，包括会议安排，为PRECISE提供坚实的基础 会议纪要、协作文档准备、法规遵从性跟踪以及对摘要的支持 以及精准网络的出版准备工作。这一综合结构将共同搭建舞台 在精密网络中进行高影响、顺序、自适应的第二阶段/概念验证试验， 这可能导致为重症患者建立潜在的个性化治疗方法 和/或易加重的哮喘。

ABSTRACT The goal of this U24 application is to establish the Data, Modeling, and Coordination Center (DMCC) for the NHLBI's Precision Interventions for Severe and/or Exacerbation-Prone Asthma (PrecISE) Network. The objective of PrecISE is to conduct sequential, adaptive, phase II/proof of concept trials with precision interventions in stratified patient with severe asthma populations. Patients with severe asthma are heterogeneous, and thus a hard to treat population. Precision medicine interventions, which focus on identifying the best treatments for individual patients instead of the best treatments for the most patients, could improve overall outcomes among this complex patient population. The PrecISE Network is poised to address these issues. Central to its success will be an innovative, forward-looking, and productive Coordinating Center that brings cohesion to the participating Clinical Center investigators by sharing their visions and providing scientific leadership, organizational support and analytic results for PrecISE trials. Key elements that will enhance collaboration and research progress include experienced leadership, innovative design and analysis methodologies (e.g., sequential adaptive designs, machine learning, neural networks, Q-learning), comprehensive research operations support, a state-of-the-art data management system, and superior administrative support. Our infrastructure will provide both public and private websites which include PrecISE information pages for the public, participants, and investigators, frequently asked questions, study materials, project calendar, a communications hub, and other key features. We will develop a secure, user-friendly advanced data management system that includes web-based access for surveys, data entry, research management (including comprehensive reporting, biosample tracking and performance-based reimbursement tools), integration with external applications, and quality control. Our research operations and administration teams will provide a solid foundation to PrecISE by facilitating communication including meeting arrangements and minutes, collaborative document preparation, tracking of regulatory compliance, and support for abstract and publication preparation across the PrecISE Network. Together, this integrated structure will set the stage for the execution of high-impact, sequential, adaptive, phase II/proof of concept trials in the PrecISE Network, which can lead to the establishment of potential personalized treatment approaches for patients with severe and/or exacerbation-prone asthma.