I-ACT for Children, Global Pediatric Clinical Trials Network

项目摘要 儿科临床试验企业太慢，太昂贵，与儿童的需求脱节。 近60%用于儿童的药物和90%用于新生儿的药物是标签外处方。这些问题依然存在 尽管有一个强有力的监管框架来推进创新医疗产品的证据。转型 需要公共和私营利益攸关方之间的合作， 儿科试验系统，包括整合/催化现有专业知识、资源和基础设施的方法。 儿童高级临床试验研究所（I-ACT）是一家独立的501（c）3非营利组织， 作为关键路径研究所儿科试验联盟咨询报告的实例推出。 I-ACT的使命/愿景与RFA-FD-17-014的目标完全一致，并专注于创建一个综合的 儿科产品开发的资源，可持续的全球基础设施，以支持所有阶段， 儿科临床试验为了响应FDA对全球儿科临床试验网络（GPCTN）的呼吁，我们的 具体目标是：目标1（战略和规划）：提供一个多方利益攸关方论坛， 在推进创新药物和器械的儿科临床试验方面的未来挑战和机遇。 目标2（工具，最佳实践和教育）：开发，策划和传播工具，知识和最佳实践。 实践并制作教育材料和计划，以推进创新的儿科临床试验。 药品和器械。目标3（基础设施和试验执行）：创建并持续改进 可持续的全球基础设施，致力于快速和成功地执行儿科临床试验， 创新的药物和设备。这些目标支持了咨询委员会中GPCTN的13项核心职能。 次报告. 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将成为数据和学习的核心。PEDSnet由全国 领先的儿童研究医院和特定条件的网络，并提供数百个临床网站， 数据驱动的可行性和观察性研究资源（530万儿童）。关键路径研究所将领导 监管科学核心和重点人群（如新生儿、罕见病）和药物项目 开发工具。由FDA资助的国家儿科设备创新资本联盟就是其中之一。 我们是世界上最强大、最成功的儿科器械研究网络之一，并将领导Device Core。 I-ACT包括许多全球利益攸关方，他们的承诺和专业知识是GPCTN的关键 资源GPCTN将有能力满足50%的创新儿科试验的需求。 药品/器械。我们的方法将确保创建和执行儿科计划/方案， 更及时的儿科标签，重点是结果。我们的方法创造的变革包括 利益相关者的观点，但不受传统的限制。我们将提高儿童健康项目的价值，以获得它 第一次就成功了，建立了一个经过改造和整合的全球儿科临床试验基础设施。

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.