The UNC Core Center for Clinical Research: Phenotyping and Precision Medicine Resource Core

摘要-资源核心 该委员会的总体目标是加强临床研究的设计和实施， 风湿和肌肉骨骼疾病（RMD），重点是骨关节炎（OA）， 我们研究界的特殊力量。表型分析与精准医学 资源核心将为我们的研究社区提供关键服务， 包括：1）预测表型所需关键科学专业知识和分析资源 （通过特定亚组预测结果，例如“进展者”与“非进展者”）和 使用临床数据集的规定性表型分析（预测对特定干预的反应）， 和2）指导和建议，研究人员对表型的考虑（如选择 输入和结果变量），以加强设计、实施和解释 新的临床研究和试验。像许多慢性疾病一样，RMD，包括OA， 单一疾病，而是由多种表型组成的异质性疾病， 在其潜在的病理生物学机制上不同。如果不考虑这些因素， 差异导致对旨在解决特定机制的干预措施的测试， 在“错误”的病人身上。因此，RMD的成功治疗需要有针对性， 并在共享不同的潜在病理生物学的特定亚组或亚型中进行测试， 社会心理和疼痛机制，符合精准医疗的目标。的 表型分析和精准医学资源核心将与 方法核心提供服务，将大大提高研究设计和添加 对正在进行的、已完成的和计划的广泛临床研究的价值， 目标：目标1。提供咨询意见和分析服务， 和规定性表型，为有兴趣在新的或 正在进行的OA或其他RMD临床研究，包括辅助研究和 规划阶段。目标2.应用创新策略进行表型分析和精准医疗， 利用从我们的研究社区选择的临床研究中收集的数据， 这些研究，促进外部验证和通知未来的研究。目标3。鼓励 更广泛地实施RMD的表型分析和精准医学方法， 向地方、国家和国际研究提供教育和传播方法 社区.一个致力于表型分析和精准医学的资源核心将大大 使CCCR支持的研究团体受益，产生重大影响 在临床RMD研究领域，并最终对人们的生活患有这些 慢性病和致残性疾病。

ABSTRACT – RESOURCE CORE The overall goal of the UNC CCCR is to enhance the design and conduct of clinical studies in Rheumatic and Musculoskeletal Diseases (RMDs) with a focus on osteoarthritis (OA), a particular strength of our research community. The Phenotyping and Precision Medicine Resource Core will provide critical services to our research community that broadly include: 1) key scientific expertise and analytic resources needed for prognostic phenotyping (predicting outcomes by specific subgroups, e.g. “progressors” versus “non-progressors”) and prescriptive phenotyping (predicting response to specific interventions) using clinical datasets, and 2) guidance and advice to investigators on phenotypic considerations (such as choice of input and outcome variables) in order to enhance the design, implementation, and interpretation of new clinical studies and trials. Like many chronic conditions, RMDs, including OA, are not single diseases but rather heterogeneous conditions consisting of multiple phenotypes that differ in their underlying pathobiological mechanisms. If not taken into consideration, these differences result in the testing of interventions designed to address specific mechanisms of action in the “wrong” patients. Successful treatments for RMDs therefore need to be targeted to, and tested in, specific subgroups or subtypes that share distinct underlying pathobiological, psychosocial and pain mechanisms, consistent with the goal of precision medicine. The Phenotyping and Precision Medicine Resource Core will work in conjunction with the Methodology Core to provide services that will greatly enhance study design and add value to a wide spectrum of ongoing, completed, and planned clinical studies through the following aims: Aim 1. Provide consultative advice and analytical services for prognostic and prescriptive phenotyping for investigators interested in defining subgroups within new or ongoing clinical studies in OA or other RMDs, including ancillary studies and studies in the planning stages. Aim 2. Apply innovative strategies for phenotyping and precision medicine by leveraging data collected in selected clinical studies from our research community, adding value to those studies, facilitating external validation and informing future studies. Aim 3. Encourage broader implementation of phenotyping and precision medicine approaches for RMDs through education and dissemination of the methodologies to local, national, and international research communities. A resource core dedicated to phenotyping and precision medicine will greatly benefit the research community that will be supported by the CCCR, make a significant impact on the field of clinical RMD research, and ultimately on the lives of people suffering from these chronic and disabling conditions.