Mentoring Patient-Oriented Pharmacoepidemiologic Research in Rheumatic Diseases

指导以患者为导向的风湿性疾病药物流行病学研究

基本信息

批准号：
10187754
负责人：
Seoyoung Catherine Kim
金额：
$ 16.64万
依托单位：
BRIGHAM AND WOMEN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-04-01 至 2026-03-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10187754
关键词：
Affect Age Award Benefits and Risks Biological Biological Availability Biological Factors Cardiovascular Diseases Characteristics Chronic Client satisfaction Clinical Clinical Investigator Clinical Practice Guideline Clinical Research Clinical Trials Clinical effectiveness Computerized Medical Record Data Decision Making Development Diabetes Mellitus Disease Disease remission Disease-Modifying Second-Line Drugs Drug usage Effectiveness Enrollment Environment Event Evidence based treatment Feedback Future Future Generations Grant Head Heart failure Hospitals Immune system Infection Inflammation Inflammatory Arthritis Interleukins Intestinal Perforation Janus kinase Knowledge Link Malignant Neoplasms Medical Medicare Mentors Meta-Analysis Methods Methotrexate Mid-Career Clinical Scientist Award (K24)Midcareer Investigator Award in Patient-Oriented Research Opioid Outcome Outcomes Research Patient Preferences Patient risk Patients Pharmaceutical Preparations Pharmacoepidemiology Pharmacotherapy Physicians Population Postdoctoral Fellow Randomized Research Research Personnel Research Project Grants Research Training Resources Rheumatism Rheumatoid Arthritis Rheumatology Risk Risk-Benefit Assessment Safety Societies Socioeconomic Status Steroids T-Lymphocyte TNF gene Thrombosis Training Training Programs Woman career clinical development comparative effectiveness comparative safety compare effectiveness evidence base evidence based guidelines experimental study head-to-head comparison health plan illness length improved individual patient inhibitor/antagonist innovation male neglect next generation patient oriented patient oriented research pre-doctoral predictive modeling preference programs rheumatologist safety outcomes side effect skills subcutaneous

项目摘要

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.

项目概要类风湿性关节炎（RA）是一种慢性炎症性关节炎，目前尚无已知的治愈方法。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 - 针对免疫系统特定组成部分的改良抗风湿药物（DMARD）是目前可用于中度至重度 RA 患者，而所有这些生物制剂和 ts-DMARD 均具有很高的疗效。这些药物可有效控制 RA 炎症，但存在广泛的安全问题，包括感染、心血管疾病、血栓形成、心力衰竭、下肠穿孔和恶性肿瘤。显着扩大了 RA 的治疗范围，并更加重视治疗以实现缓解或低 RA 疾病活动度，对于大众和患者来说了解有关药物的数据都很重要然而，在如何直接进行比较方面存在知识差距。在没有临床试验的情况下，了解这些不同的 DMARD 的益处和风险。研究定量评估了患者的偏好并将其纳入权衡利弊的过程中因此，申请人提出了一个为期 5 年的 K24 奖励项目，该项目具有三个具体的研究目标。这将：1）模拟目标试验来评估生物制剂的头对头比较有效性和安全性和 ts-DMARDs 在 RA 患者中使用真实世界数据 2) 确定安全事件的重要预测因素；与生物制剂或 ts-DMARD 的使用相关；3) 进行以患者为中心的获益-风险评估此外，申请人的目标是成长和发展。加强药物流行病学和以患者为中心的结果研究方面的研究培训计划申请人 Seoyoung C. Kim 博士是一位风湿病学家和博士生。她曾担任 30 多名研究前和研究后的初级或次级研究导师。申请人在过去十年中正在进行的许多研究项目。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得天独厚的机构资源将为青少年的发展提供良好的环境此外，申请人还组建了一支强大的高素质合作团队。作为她的学员的共同导师，她将继续完善她的指导计划和技能获奖期间学员的反馈将有助于生成基于现实世界的证据。使用生物制剂和 ts-DMARD 治疗 RA 的建议，纳入患者的治疗偏好并支持申请人建立强大的临床研究平台来指导下一个的能力药物流行病学和风湿病学领域以患者为导向的研究方面的临床研究人员的产生促进他们成功过渡到独立。