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.

未能标记儿童的设备和治疗剂会威胁到公共卫生。进行足够的研究 然而，小儿标签构成了许多挑战。这些包括有限数量的合格参与者 疾病；研究设计和运营效率低下；无效的利益相关者参与；而且缺乏 强大的儿科特定全球基础设施用于入学。为了满足这个关键的未满足需求，我们将 开发全球儿科临床试验网络（G-PCTN），该试验将支持计划和 通过与学术界，行业，父母/患者倡导小组合作执行小儿临床试验， 和政府机构。 G-PCTN将作为儿科产品开发的协调资源 改善儿童的公共卫生。我们将利用功能齐全的儿科临床的合作伙伴关系 试验网络以及FDA，学术医疗中心，行业，患者和家庭倡导的合作 团体，专业社会和其他利益相关者。在首席研究人员（PI）的领导下 博士。小丹尼尔·K·本杰明（Daniel K. 患者参与和科学监督）和5个临床研究小组，将专注于设计，剂量， 监管/药房，网络伙伴关系和罕见疾病。两种PI都带来了互补技能，以领导 网络，包括视觉，临床试验专业知识，大型临床试验网络主席（国立国家研究所 儿童健康与人类发展儿科试验网络，临床和转化科学奖 试用创新中心），工业和政府治疗学发展经验，监管策略， 和PK/PD建模。我们将共同利用我们的经验，能力，监管知识和跟踪 - 记录以建立科学，运营和利益相关者参与基础设施以计划和完成 小儿临床研究。我们将重点介绍运营效率和网络可持续性， 小儿试验的系统和综合方法。 G-PCTN将巩固并扩大我们已建立的 全球研究站点网络采用其基础架构和资源来支持调查人员，赞助商， 患者和受训者在儿童中进行临床试验。我们将重点介绍以下特定目标1） 提供战略和内容专业知识，以增加小儿产品开发的成功； 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； 3）建立和管理 可持续的网站网络，以执行儿童的临床试验。由于我们的合作努力 G-PCTN，我们将建立所需的基础架构，以有效地进行科学地进行全球儿科 临床试验。不同的赞助商将利用这种新的基础设施来产生监管级 质量数据并改善小儿公共卫生。