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

项目总结/摘要 房颤是全球缺血性卒中和卒中相关死亡率的主要原因。虽然风险 口服抗凝剂（OAC）可减少房颤患者的缺血性卒中，但许多患者不能 由于出血风险高，适合长期OAC。为了帮助治疗这些患者，经导管左 使用Watchman器械进行的心耳封堵术（LAAO）被开发并获批作为替代方案 到OAC。然而，用于评价临床结局的有效方法与Watchman器械和 在监管机构批准后，需要其他新的心血管技术进行长期监测。在这 指导以病人为导向的职业发展奖，Kamil Faridi博士将使用LAAO的国家登记处 与医疗保险索赔数据相关的程序，以评估不同的数据源和创新的统计 方法可用于评价Watchman器械在以下方面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性心脏病， 在一个国家中，通过行政索赔数据确定的卒中和出血事件与研究中心报告的事件 接受经导管LAAO的患者登记研究。在目标2中，他将使用关联的药房索赔数据， 评价登记处报告的OAC使用情况，并比较LAAO患者和药物治疗患者之间的结局 心房颤动患者在现实世界的实践。补充上市后定量策略 在目标3中，他将使用定性方法探索患者报告的观点和生活质量 在转诊接受经导管LAAO的患者中。这些研究目标的实现将为今后的研究奠定基础 未来的研究检查其他心血管治疗和其他临床 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， 耶鲁医学院的一名教员，在心血管领域取得了成功 成果研究。在这个为期5年的项目中，Faridi博士将继续接受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，适用于以患者为导向的 research.这将通过一个全面的职业发展计划来实现， 博士Faridi具备成为心血管医学成功临床研究者所需的技能。他 长期的职业目标是成为一名独立的调查员，在调查后的领域具有特定的专业知识， 新型心血管治疗的市场监测和真实世界数据利用，最终目的是 改善病人护理。在研究和培训计划的过程中，Faridi博士将由一位 卓越的指导团队和咨询委员会，由知名的临床研究者组成， 心血管医学、结果研究以及先进的生物统计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， 博士法里迪将做好充分准备，在奖励期结束前过渡到研究独立。

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.