Clinical Characterization and Biorepository Core

ORDRCC通过临床指导、临床指导提供风湿病研究服务的集中化 数据和来自我们临床管理集合中注册的约71,000名患者的生物标本 特征和生物信息库核心(CCBC)，包括与以下内容相关的全面临床评估 唯一已知的专注于风湿病的CAP认证生物储存库。到目前为止，我们已经批准 研究人员可以访问&gt；103,000个样本和4.2B个数据字段(临床、实验、免疫学、遗传学)。 1,963份样本/数据请求(过去4年中有571份)产生了445份出版物和19份新的 调查员飞行员利用6000万美元的外部资金帮助他们开创了独立的职业生涯。CCBC- 相关项目实现了对疾病过程的独特见解，开发了新的患者报告 结果并进行了创新的临床试验设计，为复杂的疾病状态量身定做 病理学。由我们的研究人员确定的自身免疫分子表型现在正在应用 在NIH资助的和药物试验中，在确定了独特的应答者群体后， 有针对性的治疗本来会失败的。CCBC的基本目标是帮助启动 并通过提供咨询临床人员和设施来支持独立研究事业 许多初级调查人员和基础科学家无法接触到。CCBC人员已指导或提供 151项IRB批准的研究方案，几乎所有新的JCI NIH人类受试者部分，患者招募 (n=1,282)，以及超过66,068个与全面临床信息有关的新样本等分。这个 目前的提案回应了先进表型科学的进展和不断变化的研究人员需求，具体如下 临床和转化性研究信息学建模以及收集和整合的新方法 分子表型数据与临床和患者报告数据相结合，以促进精准医学。我们会 继续增加我们在SLE，RA，UCTD和Sjogren‘s的收藏，同时增加新的收藏， 包括一名冠状病毒感染后自身抗体阳性的队列。目标1将继续风湿病临床 为ORDRCC初级调查人员、学者和具有导师和 临床/干预性研究设计中的监管协助和患者的临床研究服务单位 鉴定、招募和临床特征。目标2将扩展和增加复杂的 样本收集、管理、SOP和生物库基础设施，并支持大型风湿病 疾病基础/临床协作。在AIM 3中，一个新的CCBC创新中心将试验新的方法 多样化的患者招募、留住、鉴定、家庭监控和远程样本采集 在疾病爆发和改善时，优化对患者样本和数据的实时访问。目标4寻求 通过科学驱动的临床试验设计改变患者的生活，从而降低数据复杂性并提供信息 后来优化、个体化的治疗选择和指导，在临床上提供更精细的剂量策略。

The ORDRCC provides centralization of rheumatic disease research services through mentorship, clinical data, and biospecimens from ~71,000 patients registered in the managed collections of our Clinical Characterization and Biorepository Core (CCBC), comprised of comprehensive clinical evaluations linked to the only known rheumatic-disease-focused CAP-certified biorepository. To date we have granted investigator access to >103,000 samples and 4.2B data fields (clinical, experimental, immunologic, genetic). The 1,963 sample/data requests (571 in the past 4 years) have resulted in 445 publications and our 19 new investigator pilots have helped launch independent careers with their >$60M in external funding. CCBC- linked projects have achieved unique insights into disease processes, developed novel patient-reported outcomes and carried out innovative clinical trial designs, tailored for disease states with complex pathology. Autoimmune molecular phenotypes identified by our investigators are now being applied prospectively in NIH-funded and pharmaceutical trials, after identifying unique responder populations to targeted treatments that would have otherwise failed. The foundational goal of the CCBC is to help launch and support independent research careers by providing advisory clinical personnel and facilities otherwise inaccessible for many junior investigators and basic scientists. CCBC personnel have mentored or provided 151 IRB-approved study protocols, nearly all new JCI NIH Human Subject sections, patient recruitment (n=1,282), and more than 66,068 new sample aliquots linked to comprehensive clinical information. The current proposal responds to progress in advanced phenotyping science and evolving investigator needs by modeling clinical and translational research informatics and new methods for collecting and integrating molecular phenotype data with clinical and patient-reported data to facilitate precision medicine. We will continue to increase our collections in SLE, RA, UCTD, and Sjogren’s while adding new collections, including an autoantibody positive post-COVID cohort. Aim 1 will continue rheumatic disease clinical research support for ORDRCC Junior Investigators, Scholars, and Investigators with mentorship and regulatory assistance in clinical/interventional study design and a Clinical Research Service Unit for patient identification, recruitment, and clinical characterization. Aim 2 will expand and increase the sophistication of sample collection, management, SOPs, and Biorepository infrastructure, and support large rheumatic disease basic/clinical collaborations. In Aim 3 a new CCBC Innovation Hub will pilot new approaches for diverse patient recruitment, retention, characterization, home monitoring and remote sample collection to optimize real time access to patient samples and data as disease flares and improves. Aim 4 seeks to transform patient lives through science-driven clinical trial design that will reduce data complexity and inform later optimized, individualized treatment selection and guidance for more refined dosing strategies in clinics.