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.
Severe asthma affects 10% of people worldwide, and despite currently available treatments, asthma remains poorly controlled for many patients. The National Institutes of Health is funding a new iteration of previous high- impact asthma clinical trials networks, which will take a personalized medicine approach to identify new therapies for severe asthma, tailored to an individual patient using data-based treatment algorithms that leverage information on his/her individual disease and treatment history. Our proposal is to provide leadership and statistical research support to this network.
