Optimizing Available Data Sources for Post-Marketing Surveillance: Real-World Use of Left Atrial Appendage Occlusion

项目摘要/摘要 房颤是全世界缺血性卒中和卒中相关死亡的主要原因。尽管存在以下风险 口服抗凝(OAC)可减少心房颤动患者的缺血性卒中，但许多患者并不是这样 由于出血风险高，适合长期接受OAC治疗。为了帮助治疗这类患者，经导管左 使用Watchman装置的心耳闭塞(LAAO)被开发并被批准为替代方案 致华侨城。然而，评估临床结果的有效方法与Watchman设备和 在监管部门批准后，还需要其他新的心血管技术来进行长期监测。在这 以患者为导向的职业发展奖，Kamil Faridi博士将使用LAAO的国家注册 与联邦医疗保险索赔数据相联系的程序，以评估不同的数据源和创新的统计 可以利用各种方法来评估Watchman设备的长期安全性和有效性 现实世界的实践。在目标1中，他将使用传统和先进的统计方法来比较缺血 由行政索赔数据确定的中风和出血事件到现场报告的全国事件 接受经导管LAAO治疗的患者登记。在目标2中，他将使用关联的药房索赔数据来 评估登记报告的OAC使用情况，并比较LAAO患者和医疗管理人员之间的结果 房颤患者在现实生活中的实践。补充营销后的量化策略 在目标3中，他将使用定性的方法来探索患者报告的观点和生活质量 在接受经导管LAAO治疗的患者中。完成这些研究目标将奠定基础 在未来的研究中检查其他心血管疗法的真实监测和其他临床 重要成果。该项目将由Kamil Faridi博士领导，他是一名早期职业调查员、心脏病专家和 耶鲁大学医学院教员，在心血管领域有成功的记录 结果研究。在这个为期5年的计划中，Faridi博士将继续进行机器学习方面的额外培训， 药物流行病学、比较有效性和定性方法在以患者为中心的应用 研究。这将通过一个全面的职业发展计划来实现，该计划旨在提供 法里迪博士拥有成为一名成功的心血管医学临床研究人员所需的技能。他的 长期的职业目标是成为一名独立的调查员，在事后调查领域拥有特定的专业知识 新的心血管疗法的市场监测和现实世界数据利用，最终目的 改善病人护理。在研究和培训计划的过程中，Faridi博士将由一名 卓越的指导团队和咨询委员会，由资深临床研究人员组成，具有 心血管医学、结果研究以及先进的生物统计学和定性方法方面的专业知识。 随着这一研究项目的实施以及为培训和职业发展提供的资源， 法里迪博士将为在获奖期结束前过渡到研究独立做好充分准备。

PROJECT SUMMARY/ABSTRACT Atrial fibrillation is a major cause of ischemic stroke and stroke-associated mortality worldwide. Though risk of ischemic stroke in patients with atrial fibrillation is reduced with oral anticoagulation (OAC), many are not suitable candidates for long-term OAC due to high bleeding risk. To help treat such patients, transcatheter left atrial appendage occlusion (LAAO) with the Watchman device was developed and approved as an alternative to OAC. However, efficient methods for evaluating clinical outcomes associated the Watchman device and other novel cardiovascular technologies are needed for long-term surveillance after regulatory approval. In this mentored patient-oriented career development award, Dr. Kamil Faridi will use a national registry of LAAO procedures linked to Medicare claims data to evaluate how different data sources and innovative statistical approaches can be utilized for evaluating the long-term safety and effectiveness of the Watchman device in real-world practice. In Aim 1, he will use traditional and advanced statistical methods to compare ischemic stroke and bleeding events identified with administrative claims data to site-reported events in a national registry of patients who undergo transcatheter LAAO. In Aim 2, he will use linked pharmacy claims data to evaluate registry-reported OAC use and compare outcomes between LAAO patients and medically managed patients with atrial fibrillation in real-world practice. To supplement quantitative strategies for post-marketing surveillance, in Aim 3 he will use qualitative methods to explore patient-reported perspectives and quality of life among patients referred for transcatheter LAAO. Accomplishing these research aims will lay the groundwork for future studies examining real-world surveillance of other cardiovascular therapies and other clinically important outcomes. This project will be led by Dr. Kamil Faridi, an early career investigator, cardiologist, and faculty member at the Yale School of Medicine with a track record of success in the field of cardiovascular outcomes research. During this 5-year program, Dr. Faridi will pursue additional training in machine learning, pharmacoepidemiology, comparative effectiveness and qualitative methods applied to patient-oriented research. This will be accomplished with a comprehensive career development program designed to provide Dr. Faridi with the skills needed to become a successful clinical researcher in cardiovascular medicine. His long-term career goal is to become an independent investigator with specific expertise in the areas of post- marketing surveillance and real-world data utilization for novel cardiovascular therapies, with the ultimate aim of improving patient care. Over the course of the research and training program, Dr. Faridi will be guided by an exceptional mentorship team and advisory committee consisting of established clinical investigators with expertise in cardiovascular medicine, outcomes research, and advanced biostatistical and qualitative methods. With implementation of this research project and the resources provided for training and career development, Dr. Faridi will be well prepared to transition to research independence by the end of the award period.