Pediatric Preclinical Testing Consortium: Coordinating Center

 描述（由申请人提供）：儿科临床前测试联盟（PPTC）的主要目标是开发将转化为人类临床实践的模型，并有效地优先考虑治疗儿科癌症的疗法。一个有效的协调中心（CC）将通过提供广泛的组织，科学和分析专业知识，以支持NCI科学家和PPTC研究计划，对联盟的目标至关重要。CC还将负责制定、实施和评估一项全面的PPTC研究计划，在数据收集和持续的计划评估方面发挥领导作用，以确保研究计划朝着PPTC使命取得最佳进展。协调委员会的一项关键职能是提供必要的组织和行政领导，使联合会能够有效运作。RTI作为PPTC未来5年的拟议CC的目标是优化联盟在生成可靠数据方面的进展，通过实现以下具体目标来加速开发更有效的癌症儿童治疗方法：1.指导联盟成员和整个儿科肿瘤学社区之间的互动。通过有效的电话会议、网络研讨会、联盟会议和网站（包括公开和限制性网站），提供必要的后勤支持，以运行高效和富有成效的联盟。利用这些工具促进PPTC成员、儿科临床医生、制药公司和儿科肿瘤学倡导组织之间的互动，以确保有效利用有前途的新疗法。2.生成可用于儿科癌症治疗药物优先级的可靠数据。为数据收集、从研究项目中收集的数据的生物信息学分析以及测试结果的生物信息学分析提供有效和稳健的程序，这些程序可以将治疗效果与所用临床前模型的分子特征联系起来。3.为联盟提供一套全面而灵活的科学、药理学和分析资源，以开发创新研究计划，创建儿科临床前试验功能联盟。开发工具，通过临床前过程跟踪每个潜在候选药物的状态。将联盟端数据字典中的文档变量作为一个元素，以在REDCap中构建数据管理系统。

 DESCRIPTION (provided by applicant): The main goal of the Pediatric Preclinical Testing Consortium (PPTC) is to develop models that will translate to human clinical practice and effectively prioritize therapies for the treatment of pediatric cancers. An effective Coordinating Center (CC) will be critical to the Consortium's goals by providing broad organizational, scientific, and analytic expertise to support NCI scientists and the PPTC Research Programs. The CC will also be responsible for developing, implementing, and evaluating a comprehensive PPTC research program, providing leadership in data collection and ongoing program evaluation to ensure that the research program is making optimal progress toward the PPTC mission. Providing the organizational and administrative leadership necessary to enable the Consortium to function efficiently will also be a key function of the CC. RTI's goal as the proposed CC for the PPTC over the next 5 years is to optimize Consortium progress in generating reliable data to accelerate the development of more effective treatments for children with cancer by achieving the following specific aims: 1. Guide interactions between Consortium members and the pediatric oncology community at large. Provide the logistical support necessary to run an efficient and productive Consortium through effective teleconferences, webinars, Consortium meetings, and websites, both public and restricted. Employ these tools to facilitate interactions between PPTC members, pediatric clinicians, pharmaceutical companies, and pediatric oncology advocacy organizations to ensure that promising new therapies are utilized efficiently. 2. Generate reliable data that can be used for prioritization of the therapeuic agents in pediatric cancer. Provide efficient and robust procedures for data collection, statistica analysis of data collected from the Research Programs, and bioinformatic analysis of testing results that can relate treatment effects to molecular characteristics of the preclinical models utilized. 3. Provide the Consortium with a comprehensive, yet flexible set of scientific, statisticl and analytic resources needed to develop an innovative research program for creating a functional Consortium for pediatric preclinical testing. Develop tools to track the status of each potential drug candidate through the preclinical process. Document variables in a Consortium-side data dictionary as one element to build the data management system in REDCap.