Clinical and Translational Science Institute at Childrens National

总体：项目摘要 尽管儿童占美国人口的四分之一，但只有10%的ClinicalTrials.gov注册研究 专注于与儿童健康相关的临床和转化研究(CTR)。临床与医疗服务中心的使命 国家儿童翻译科学研究所(CTSI-CN)将解决这些障碍，并充当枢纽 在CTSA网络内，重点是促进儿童健康CTR。成立于2010年， 首届CTSI-CN(CTSI-CN v1.0)是作为儿童国家卫生组织之间的合作组织的 美国国家卫生与公众服务部(CNHS)及其学术伙伴乔治·华盛顿大学(GW)。在过去的四年里， CTSI-CN v1.0围绕四个主要主题将自己定位为儿童健康CTR的有效催化剂：1) 改善儿童及其家庭的健康；2)开发罕见遗传疾病的新治疗策略 疾病；3)设计新的儿科护理设备；4)确定儿童疾病的特征 在整个生命周期中。我们已经接受了2013年7月医学研究所推荐的许多原则 (国际移民组织)报告，具体地说，发展多学科团队，提供具有成本效益的服务和支持 调查人员，精简影响CTR有效进行的程序。CTSI-CN是 当地公认为有效、高质量儿童健康CTR的枢纽，并被视为养育、综合 CTR团队科学之家。在全国范围内，CTSI-CN在儿童健康CTR方面发挥了领导作用，在这两方面 与CTSA和非CTSA有关联的财团和指导委员会。在目前的提案(CTSI-CN v2.0)中，我们 将继续通过创新的方法和增强的能力来推进我们的愿景和使命 我们的战略主题。为此，我们扩大了我们的合作伙伴关系，包括弗吉尼亚理工大学(VT)及其 临床合作伙伴Carilion诊所。CTSI-CN v2.0的目标将建立在我们现有优势的基础上， CTSI-CN v1.0的经验教训，以及我们与VT/Carilion(VTC)的新伙伴关系，也将解决 NCATS的四个战略主题：1)发展以儿童健康为重点的新一代多样化、高质量的 CTR调查人员通过创新的跨机构教育、培训和指导计划 (劳动力发展)；2)创造创新的方法和技术，以促进CTR， 特别是在儿童健康方面，通过新的跨学科和机构间合作，与 利益相关者社区(协作和参与)；3)促进发展， 传播和评价加强翻译科学的新方法，特别强调 儿童健康CTR谱(方法和过程)；4)提高质量和效率 通过a)整合一支高素质的多样化劳动力队伍，b)实施 新的方法和程序，以及c)机构伙伴和社区之间的合作 (整合)：和5)通过多站点协作和 教育方案，特别强调儿童健康的CTR和成果(CTSA网络)。

OVERALL: PROJECT ABSTRACT Although children comprise one-quarter of the US population, only 10% of ClinicalTrials.gov registered studies focus on child health-related clinical and translational research (CTR). The mission of the Clinical and Translational Science Institute at Children's National (CTSI-CN) is to address the barriers and serve as a hub within the CTSA Network that is focused on the promotion of child health CTR. Established in 2010, the inaugural CTSI-CN (CTSI-CN v1.0) was organized as a collaboration between Children's National Health System (CNHS) and its academic partner, The George Washington University (GW). Over the past four years, CTSI-CN v1.0 has positioned itself as an effective catalyst for child-health CTR around four major themes: 1) improving the health of children and their families; 2) developing novel treatment strategies for rare genetic diseases; 3) designing new devices for pediatric care; and 4) characterizing childhood precursors of disease along the lifespan. We have embraced many of the tenets recommended in the July 2013 Institute of Medicine (IOM) report, specifically, developing multidisciplinary teams, providing cost-effective services and support for investigators, and streamlining processes that impact the effective conduct of CTR. The CTSI-CN is acknowledged locally as the hub for effective, high quality child health CTR and as a nurturing, integrated home for CTR team science. Nationally, the CTSI-CN has provided leadership in child health CTR, in both CTSA- and non-CTSA-linked consortia and steering committees. In the current proposal (CTSI-CN v2.0), we will continue to advance our vision and mission through innovative approaches and enhanced capabilities in our strategic themes. To this end, we have expanded our partnership to include Virginia Tech (VT) and its clinical partner, the Carilion Clinic. The goals of CTSI-CN v2.0, which will build on our established strengths, the lessons learned in CTSI-CN v1.0, and our new partnership with VT/Carilion (VTC), will also address the four NCATS strategic themes by: 1) developing a new generation of diverse, high quality child health-focused CTR investigators through innovative, inter-institutional educational, training, and mentoring programs (WORKFORCE DEVELOPMENT); 2) creating innovative approaches and technologies to catalyze CTR, particularly in child-health, through novel cross-disciplinary and inter-institutional collaborations with multiple stakeholder communities (COLLABORATION AND ENGAGEMENT); 3) catalyzing the development, dissemination and evaluation of novel methods to enhance translational science, with a particular emphasis on the child-health CTR spectrum (METHODS AND PROCESSES); 4) enhancing quality and efficiency across the translational spectrum through a) the integration of a high quality diverse workforce, b) implementation of new methods and processes, and c) collaboration among the institutional partners and communities (INTEGRATION): and 5) providing leadership within the CTSA Network through multi-site collaborations and educational programs, with a particular emphasis on child-health CTR and outcomes (CTSA NETWORK).