Mentoring Patient-Oriented Pharmacoepidemiologic Research in Rheumatic Diseases

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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.