I-ACT for Children, Global Pediatric Clinical Trials Network

I-ACT for Children，全球儿科临床试验网络

• 批准号: 9980852
• 负责人: CHRISTOPHER B FORREST
• 金额: $ 100万
• 依托单位: INSTITUTE FOR ADVANCED CLINICAL TRIALS FOR CHILDREN, INC.
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-25 至 2022-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9980852
• 关键词: ACT; Children; Global; Pediatric; Clinical

PROJECT SUMMARY The pediatric clinical trials enterprise is too slow and expensive and too disconnected from the needs of children. Nearly 60% of drugs used in children and 90% used in newborns are prescribed off-label. These problems persist despite a strong regulatory framework for advancing evidence on innovative medical products. Transformation of the system requires collaboration among public and private stakeholders and innovative reengineering of the pediatric trials system, including methods to integrate/catalyze existing expertise, resources, and infrastructure. The Institute for Advanced Clinical Trials for Children (I-ACT), an independent 501(c)3 non-profit, was launched as the instantiation of the Advisory Report from Critical Path Institute’s Pediatric Trials Consortium. I-ACT’s mission/vision are fully aligned with the goals of RFA-FD-17-014 and focus on creating an integrated resource for pediatric product development, with sustainable global infrastructure to support all phases of pediatric clinical trials. To address the FDA’s call for a Global Pediatric Clinical Trials Network (GPCTN), our specific aims are: Aim 1 (Strategy & Planning): Provide a multi-stakeholder forum for addressing existing and future challenges and opportunities in advancing pediatric clinical trials of innovative medicines and devices. Aim 2 (Tools, Best Practices & Education): Develop, curate, and disseminate tools, knowledge, and best practices and produce educational materials and programs that advance pediatric clinical trials on innovative medicines and devices. Aim 3 (Infrastructure & Trials Execution): Create and continuously improve a sustainable global infrastructure dedicated to rapid and successful execution of pediatric clinical trials of innovative medicines and devices. These aims subsume the 13 core functions for the GPCTN in the Advisory Report. I-ACT will lead the Administrative and Strategic Core of the GPCTN, ensuring successful execution of all network activities. PEDSnet will be the Data and Learning Core. PEDSnet is composed of the nation’s leading children’s research hospitals and condition-specific networks and provides hundreds of clinical sites and a resource (5.3M children) for data-driven feasibility and observational studies. Critical Path Institute, will lead the Regulatory Science Core and projects in key populations (e.g. newborns, rare diseases) and drug development tools. The National Capital Consortium for Pediatric Device Innovation, funded by FDA, is one of the strongest and successful pediatric device research networks in the world, and will lead the Device Core. I-ACT includes numerous global stakeholders whose commitment and expertise are essential GPCTN resources. The GPCTN will have the capacity to meet the demands of 50% of pediatric trials of innovative medicines/devices. Our approach will ensure that pediatric plans/protocols are created and conducted to enable timelier pediatric labeling, with a focus on outcomes. Our approach creates change that is inclusive of stakeholder perspectives but not limited by tradition. We will improve the child health value of projects to get it right the first time, producing a transformed and integrated global pediatric clinical trials infrastructure.

项目摘要 小儿临床试验企业太慢，昂贵，与儿童的需求脱节。 在儿童中使用的药物中有将近60％，新生儿中使用的90％是标签的规定。这些问题仍然存在 尽管有强大的监管框架来推进有关创新医疗产品的证据。转型 该系统需要在公共和私人利益相关者之间进行协作，并进行创新的重新设计 小儿试验系统，包括整合/催化现有专业知识，资源和基础设施的方法。 儿童晚期临床试验研究所（I-ACT），一个独立的501（c）3非营利组织，是 由Critical Path Institute的儿科试验联盟的咨询报告实例化。 I-Act的使命/愿景与RFA-FD-17-014的目标完全一致，并专注于创建一个集成的 儿科产品开发资源，并具有可持续的全球基础设施，以支持 小儿临床试验。为了解决FDA呼吁建立全球儿科临床试验网络（GPCTN）的呼吁，我们 具体目的是：目标1（战略与规划）：提供一个多利益相关者论坛，以解决现有和 未来的挑战和机遇，用于推进创新药物和设备的儿科临床试验。 目标2（工具，最佳实践和教育）：开发，策划和传播工具，知道和最佳 实践和制作教育材料和计划，以推进有关创新的小儿临床试验 药物和设备。目标3（基础架构和试验执行）：创建并不断改进 可持续的全球基础设施致力于快速，成功地执行儿科临床试验 创新的药物和设备。这些目标包含咨询中GPCTN的13个核心功能 报告。 I-Act将领导GPCTN的行政和战略核心，确保成功执行 所有网络活动。 PEDSNET将是数据和学习核心。 PEDSNET由国家的 领先儿童研究医院和特定条件的网络，并提供数百个临床站点和 用于数据驱动的可行性和观察性研究的资源（530万儿童）。关键路径学院将领导 关键人群（例如新生儿，稀有疾病）和药物的监管科学核心和项目 开发工具。由FDA资助的国家儿科设备创新联盟是一个 世界上强大而成功的小儿设备研究网络的属性，并将领导设备核心。 I-ACT包括许多全球利益相关者，他们的承诺和专业知识是必不可少的GPCTN 资源。 GPCTN将有能力满足50％的创新儿科试验的需求 药物/设备。我们的方法将确保创建并执行儿科计划/协议以实现 及时的儿科标签，重点是结果。我们的方法创造了包含的变化 利益相关者的观点，但不受传统的限制。我们将提高项目的儿童健康价值 就在第一次，产生了一个转化和综合的全球小儿临床试验基础设施。