Mentoring Patient-Oriented Pharmacoepidemiologic Research in Rheumatic Diseases

项目总结 类风湿性关节炎(RA)是一种慢性炎症性关节炎，目前尚无治愈方法。但是，有了 过去几十年的医学突破，十几种不同的生物和靶向合成(TS)疾病- 针对免疫系统特定成分的修改抗风湿药物(DMARD)是 目前适用于中至重度类风湿关节炎患者。虽然所有这些生物和TS-DMARD都高度 这些药物有效地控制了RA的炎症，具有广泛的安全性问题，包括感染、 心血管疾病、血栓形成、心力衰竭、低位肠穿孔和恶性肿瘤。给定 极大地扩大了RA的治疗范围，并日益重视对目标的治疗以实现缓解 或RA疾病活动性低，这对临床医生和患者都很重要，了解有关药物的数据 有效性和安全性。然而，在如何直接进行比较方面，存在着一个知识鸿沟 这些不同的DMARD在没有临床试验的情况下，关于它们的益处和风险。此外，几乎没有人 研究已经定量地评估了患者的偏好，并将其纳入到权衡 这些毒品。因此，申请者提出了一个为期5年的K24奖励项目，该项目有三个具体的研究目标 它将：1)模拟目标试验，以评估生物的相对有效性和安全性 和TS-DMARDS在RA患者中的应用；2)确定安全事件的重要预测因素 与使用生物或TS-DMARDS相关；以及3)进行以患者为中心的利益风险评估 这些药物包含了患者相对的治疗偏好。此外，申请者的目标是发展和 加强药物流行病学的研究培训计划和以患者为导向的结果研究 风湿病。申请人金秀英博士是一名风湿病专家，也是一名受过博士培训的医生。 药物流行病学家。她曾担任过30多年前和后的初级或次级研究导师。 过去十年的博士生研究实习生。申请者的许多正在进行的研究项目 药物流行病学和以患者为导向的研究，对指导的高度承诺，以及 优秀的机构资源将为青少年的发展提供良好的环境 调查人员。此外，申请者还组建了一支由高素质合作者组成的强大团队，他们可以 作为她的学员的联合导师，她将继续完善她的指导计划和技能，使用 学员在获奖期间的反馈。这项K24奖项研究将有助于产生基于真实世界的证据 结合患者治疗的生物性和TS-DMARDS类风湿关节炎的治疗建议 偏好和支持申请者建立强大的临床研究平台的能力，以指导下一步 一代药物流行病学的临床研究人员和风湿病患者为中心的研究人员 促进他们成功地过渡到独立。

PROJECT SUMMARY Rheumatoid arthritis (RA) is a chronic inflammatory arthritis for which there is no known cure. However, with medical breakthroughs in the past few decades, a dozen different biologic and targeted synthetic (ts) disease- modifying antirheumatic drugs (DMARDs) directed toward specific components of the immune system are currently available for patients with moderate-to-severe RA. While all these biologic and ts-DMARDs are highly effective in controlling inflammation in RA, these agents carry a wide range of safety concerns including infections, cardiovascular disease, thrombosis, heart failure, lower intestinal perforations, and malignancies. Given the significantly expanded armamentarium for RA and increasing emphasis on treating to target to achieve remission or low RA disease activity, it is important for both clinicians and patients to understand the data regarding drug effectiveness and safety. However, there is a knowledge gap in concerning how one can compare directly across these different DMARDs with regard to their benefits and risks in the absence of clinical trials. Moreover, few studies have quantitatively assessed and incorporated patient preferences into weighing benefits vs. harms of these drugs. The applicant, therefore, proposes a 5-year K24 award project with three specific research aims which will: 1) emulate a target trial to evaluate head-to-head comparative effectiveness and safety of biologic and ts-DMARDs in patients with RA using real-world data; 2) identify important predictors of safety events associated with use of biologic or ts-DMARDs; and 3) conduct a patient-centered benefit-risk assessment of these drugs incorporating patients’ relative treatment preferences. In addition, the applicant aims to grow and strengthen a research training program in pharmacoepidemiology and patient-oriented outcome research in rheumatic diseases. The applicant, Dr. Seoyoung C. Kim, is a rheumatologist and a doctoral trained pharmacoepidemiologist. She has served as primary or secondary research mentor for over 30 pre- and post- doctoral research trainees in the past decade. The applicant’s many ongoing research projects for pharmacoepidemiologic and patient-oriented research, high degree of commitment to mentoring, and the exceptional institutional resources will provide an outstanding environment for the development of junior investigators. Furthermore, the applicant has assembled a strong team of highly qualified collaborators who can serve as co-mentors for her trainees, and she will continue to refine her mentoring program and skills using trainee feedback during the award period. This K24 award study will help generate real-world evidence-based recommendations for the management of RA with biologic and ts-DMARDs, incorporating patients’ treatment preferences and support the applicant’s ability to build a strong clinical research platform to mentor the next generation of clinical researchers in pharmacoepidemiology and patient-oriented research in rheumatology and promote their successful transition to independence.