VERITY: Value and Evidence in Rheumatology using bioInformaTics, and advanced analYtics

项目摘要 儿童和成人风湿性和肌肉骨骼(MSK)疾病向临床研究人员提出了挑战： 它们影响整个寿命的人群，影响以患者为中心的广泛结果，很少见 治愈，而且往往有多种可能的治疗策略。如果处理得当，这些因素就会出现 改善患者护理和患者结局的巨大机遇。的首要目标是 布里格姆妇女医院(BWH)CCCR旨在促进方法学科学的进步 支持NIAMS儿童和成人风湿和肌肉骨骼(MSK)的临床研究 疾病任务。 Verity围绕中心科学主题进行组织，这些主题可应用于多种风湿性和 MSK疾病。这些核心涉及以下交叉领域：1)将患者纳入临床试验 设计、结果选择、结果解释和传播；2)将行为经济学整合到 加强已知干预措施的益处，即坚持服药、参加体力活动和 减肥；3)从大数据中提取最大信息量；4)使用智能技术(移动 健康)以促进临床研究；以及5)传播知识和指导地方和国家 风湿病和MSK病临床研究人员通过分布式学习模型。这些科学主题 将通过以下具体目标中描述的几个核心进行接触。1.从战略上指导， 监督并向Verity提供财务责任，并培训广泛的地方和国家团队 儿童和成人风湿性疾病和MSK疾病以患者为中心的临床研究的研究人员。 (行政核心)2.开发和实施最先进的创新试验方法和 风湿病和MSK病研究的纵向比较有效性研究。(方法核心) 3.支持生物信息学方法，以加强利用大型数据库进行相关的表型分析 患者，并实施生物标志物、电子病历和比较效果分析。 (生物信息学资源核心)

PROJECT ABSTRACT Pediatric and adult rheumatic and musculoskeletal (MSK) diseases challenge the clinical researcher: they affect the population across the lifespan, impact a broad range of patient-centered outcomes, are rarely cured, and often have multiple possible treatment strategies. If properly addressed, these factors present tremendous opportunities for improving patient care and outcomes of patients. The overarching goal of the Brigham and Women’s Hospital (BWH) CCCR is to facilitate advancements in the methodologic sciences supporting clinical research within the NIAMS pediatric and adult rheumatic and musculoskeletal (MSK) disease missions. VERITY is organized around central scientific themes that can be applied to multiple rheumatic and MSK diseases. The Cores address the following cross-cutting areas: 1) including patients in clinical trial design, outcome selection, result interpretation, and dissemination; 2) integrating behavioral economics to enhance benefits of known interventions, i.e., adherence to medications, engagement in physical activity and weight loss; 3) extracting maximum information content from big data; 4) using smart technologies (mobile health) to advance clinical research; and 5) disseminating knowledge and mentoring of local and national rheumatic and MSK disease clinical researchers through distributed learning models. These scientific themes will be approached through several Cores described in the following Specific Aims. 1. To strategically direct, supervise, and provide financial accountability to VERITY and to train a broad group of local and national investigators for patient-oriented clinical research in pediatric and adult rheumatic and MSK diseases. (Administrative Core) 2. To develop and implement state of the art methods in innovative trials and longitudinal comparative effectiveness studies for rheumatic and MSK disease research. (Methodology Core) 3. To support bioinformatic approaches to enhance utilization of large databases for phenotyping relevant patients, and implementing biomarker, electronic medical record, and comparative effectiveness analyses. (Bioinformatics Resource Core)