Clinical and Cost-effectiveness of biologics in Rheumatoid Arthritis

描述（由申请人提供）：本申请涉及比较有效性的广泛挑战领域（05）和自身免疫性风湿病和皮肤病生物制剂比较有效性的特定挑战主题（05- ar -101）。我们建议基于我们在类风湿性关节炎（RA）的荟萃分析、大规模流行病学和成本效益研究方面的经验，使用三个大型RA登记处、系统文献综述和计算机模拟建模来研究生物制剂的临床和成本效益，以确定个体患者的最佳治疗方案。RA是最常见的系统性自身免疫性疾病，约占总人口的1%。RA是一种慢性、进行性、破坏性关节疾病，伴有全身炎症，常导致严重残疾和过早死亡。生物制剂在治疗类风湿性关节炎方面非常有效，但也非常昂贵。这种显著的效益和高昂的成本相结合，在适当分配有限的资源和确定个体患者的最佳治疗方面带来了一个具有挑战性的困境。准确了解生物制剂在类风湿关节炎中的比较临床和成本效益是确定个体患者最佳治疗的重要一步。在这项研究中，我们将进行系统的文献综述并分析三个大型RA登记处（国家风湿病数据库[n=25,977]），瑞典RA登记处[n=40,000]和RA研究网络[n=2,000])，以比较替代生物制剂的临床有效性。我们的结局包括对疾病活动性、生活质量、终止率、缓解率、放射学结局和主要不良事件发生率（即死亡率、心血管结局、恶性肿瘤和感染）的影响。我们将确定生物制剂在按年龄、性别、病程、疾病修饰抗风湿药物（DMARD）使用史和合并症定义的亚人群中的临床有效性。此外，我们将从美国社会的角度估计替代生物制剂的长期成本效益，并确定生物制剂在这些亚人群中的成本效益。最后，我们将进行概率分析，以表征与RA中生物制剂的比较成本效益相关的不确定性，并确定附加信息的价值。这些特定的目标将全面表征生物制剂的比较临床和成本效益，以帮助确定个体RA患者的最佳治疗方法。该项目将以高成本效益的设计，为临床护理、公共卫生和未来研究经费分配提供全面的信息，这些信息对这一关键主题具有重要意义。

DESCRIPTION (provided by applicant): This application addresses the broad Challenge Area of comparative effectiveness (05) and specific Challenge Topic of Comparative Effectiveness of Biologics in Autoimmune Rheumatic and Skin Diseases (05-AR-101). We propose to build upon our experience in meta-analysis, large-scale epidemiology, and cost-effectiveness research in rheumatoid arthritis (RA) using three large RA registries, a systematic literature review, and computerized simulation modeling to study clinical and cost-effectiveness of biologics to determine the best therapy for individual patients. RA is the most common systemic autoimmune disease and affects approximately 1% of the general population. RA represents a chronic, progressive, and destructive joint disease associated with systemic inflammation, often leading to substantial disability and premature mortality. Biologics are very efficacious in treating RA, but are also very costly. This combination of marked benefits and high costs brings a challenging dilemma in appropriately allocating limited resources and determining the best therapy for individual patients. An accurate understanding of the comparative clinical and cost-effectiveness of biologics in RA is an important step in determining the best therapy for individual patients. In this study, we will perform a systematic literature review and analyze three large RA registries (National Data Bank for Rheumatic Diseases [n=25,977]), Swedish RA registry [n=40,000], and RA Investigational Network [n=2,000]) to compare the clinical effectiveness of alternative biologic agents. Our outcomes include effects on disease activity, quality of life, termination rate, remission rates, radiologic outcomes, and major adverse event rates (i.e. mortality, cardiovascular outcomes, malignancy, and infection). We will determine the clinical effectiveness of biologic agents among subpopulations defined by age, gender, disease duration, history of disease modifying anti-rheumatic drug (DMARD) use, and comorbidities. Furthermore, we will estimate the long-term cost-effectiveness of alternative biologic agents from a US societal perspective and determine the cost-effectiveness of biologic agents among these subpopulations. Lastly, we will perform a probabilistic analysis to characterize the uncertainty associated with comparative cost- effectiveness of biologics in RA and to establish the value of additional information. These specific aims will comprehensively characterize the comparative clinical and cost-effectiveness of biologics to help determine the best therapy for individual RA patients. This project will provide comprehensive information that has important implications for clinical care, public health, and future research funding allocations on this crucial topic in a highly cost-efficient design. PUBLIC HEALTH RELEVANCE: The proposed study will build upon our experience in meta-analysis, large-scale epidemiology, and cost-effectiveness research in rheumatoid arthritis (RA), using three large RA registries, a systematic literature review, and computerized projection modeling to study the clinical and cost-effectiveness of biologics to determine the best therapy for individual patients. This project will provide comprehensive information that has important implications for clinical care, public health, and future research funding allocations on this crucial topic, in a highly cost-efficient design.