Global Pediatric Clinical Trials Network

全球儿科临床试验网络

• 批准号: 9980849
• 负责人: Michael Cohen-Wolkowiez
• 金额: $ 100万
• 依托单位: DUKE UNIVERSITY
• 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/9980849
• 关键词: Global; Pediatric; Clinical; Trials; Network

Failure to label devices and therapeutics for children threatens public health. Performing adequate studies for pediatric labeling, however, poses many challenges. These include a limited number of eligible participants with the disease; study design and operational inefficiencies; ineffective stakeholder engagement; and lack of a robust, pediatric-specific global infrastructure for enrollment. To address this critical unmet need, we will develop the Global Pediatric Clinical Trials Network (G-PCTN), which will support the infrastructure to plan and execute pediatric clinical trials through collaboration with academia, industry, parent/patient advocacy groups, and government agencies. G-PCTN will serve as the coordinating resource for pediatric product development to improve the public health of children. We will utilize the partnership of a fully functioning pediatric clinical trials network and a collaboration of FDA, academic medical centers, industry, patient and family advocacy groups, professional societies and other stakeholders. Under the leadership of Principal Investigators (PIs) Drs. Daniel K. Benjamin, Jr. and Michael Cohen-Wolkowiez, we will establish 3 cores (Network Operations, Patient Engagement, and Scientific Oversight) and 5 clinical study groups that will focus on design, dosing, regulatory/pharmacy, network partnerships, and rare diseases. Both PIs bring complementary skills to lead the network including vision, clinical trial expertise, chairing of large clinical trials networks (National Institute of Child Health and Human Development Pediatric Trials Network, Clinical and Translational Science Awards Trial Innovation Center), industry, and government therapeutics development experience, regulatory strategy, and PK/PD modeling. Together, we will leverage our experience, capacity, regulatory knowledge, and track- record to establish the scientific, operational, and stakeholder engagement infrastructure to plan and complete pediatric clinical studies. We will focus on operational efficiencies and network sustainability with early, systematic, and integrative approaches to pediatric trials. G-PCTN will solidify and expand our established global research site network, employing its infrastructure and resources to support investigators, sponsors, patients, and trainees to conduct clinical trials in children. We will focus on the following specific aims 1) provide strategy and content expertise to increase the success of pediatric product development; 2) provide education and training on best practices, design, and execution of pediatric trials; and 3) establish and manage a sustainable network of sites to execute clinical trials in children. As a result of the efforts of our collaborative G-PCTN, we will establish the required infrastructure to efficiently conduct scientifically sound global pediatric clinical trials. This new infrastructure will be leveraged by different sponsors to generate regulatory-grade quality data and improve pediatric public health.

未对儿童设备和治疗方法进行标签威胁公共健康。进行充分的研究， 然而，儿科标签提出了许多挑战。其中包括数量有限的合格参与者 研究设计和运营效率低下;利益相关者参与无效;缺乏 强大的、针对儿科的全球基础设施。为了满足这一关键的未满足需求，我们将 建立全球儿科临床试验网络（G-PCTN），该网络将支持基础设施， 通过与学术界、工业界、家长/患者倡导团体合作， 其他企事业单位提供G-PCTN将作为儿科产品开发的协调资源 改善儿童的公共健康。我们将利用一个功能齐全的儿科临床 试验网络和FDA、学术医疗中心、行业、患者和家庭倡导的合作 团体、专业协会和其他利益攸关方。在主要研究者（PI）的领导下 Drs.丹尼尔K.小本杰明和Michael Cohen-Wolkowiez，我们将建立3个核心（网络运营， 患者参与和科学监督）和5个临床研究组，将重点关注设计、给药、 监管/药房、网络伙伴关系和罕见疾病。两个PI带来互补的技能， 网络，包括愿景，临床试验专业知识，主持大型临床试验网络（国家研究所 儿童健康和人类发展儿科试验网络，临床和转化科学奖 试验创新中心）、行业和政府治疗开发经验、监管策略， 和PK/PD建模。我们将共同利用我们的经验、能力、监管知识，并跟踪- 记录，以建立科学、操作和利益相关者参与的基础设施， 儿科临床研究。我们将专注于运营效率和网络可持续性，尽早， 儿科试验的系统和综合方法。G-PCTN将巩固和扩大我们的既定 全球研究中心网络，利用其基础设施和资源支持研究者，申办者， 患者和受训人员在儿童中进行临床试验。我们将集中于以下具体目标1） 提供战略和内容专业知识，以提高儿科产品开发的成功率; 2）提供 关于儿科试验的最佳实践、设计和执行的教育和培训;以及3）建立和管理 一个可持续的网站网络，以执行儿童临床试验。由于我们的合作努力， G-PCTN，我们将建立必要的基础设施，以有效地进行科学合理的全球儿科 临床试验不同的赞助商将利用这一新的基础设施， 高质量的数据和改善儿科公共卫生。