Global Pediatric Clinical Trials Network

未能为儿童使用的设备和治疗药物贴上标签威胁着公共健康。对以下方面进行充分的研究 然而，儿科标签带来了许多挑战。其中包括数量有限的合格参与者。 与疾病有关；研究设计和操作效率低下；利益相关者参与不力；以及缺乏 强大的儿科专用全球注册基础设施。为了解决这一关键的未得到满足的需求，我们将 发展全球儿科临床试验网络(G-PCTN)，它将支持基础设施规划和 通过与学术界、产业界、家长/患者倡导团体合作执行儿科临床试验， 和政府机构。G-PCTN将作为儿科产品开发的协调资源 改善儿童的公共健康。我们将利用全功能儿科诊所的合作伙伴关系 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。在首席调查员的领导下 丹尼尔·K·本杰明博士。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个临床研究小组，他们将专注于设计、剂量、 监管/药房、网络合作伙伴关系和罕见疾病。两位PI都带来了互补的技能，以领导 网络包括愿景、临床试验专业知识、主持大型临床试验网络(美国国家临床试验研究所 儿童健康和人类发展儿科试验网络、临床和翻译科学奖 试验创新中心)、行业和政府治疗学开发经验、监管战略、 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- 记录以建立科学的、运营的和利益相关者参与的基础设施来规划和完成 儿科临床研究。我们将重点关注运营效率和网络可持续性， 系统的、综合的儿科试验方法。G-PCTN将巩固和扩大我们现有的 全球研究网站网络，利用其基础设施和资源来支持调查人员、赞助商、 患者和受训人员在儿童身上进行临床试验。我们将重点关注以下具体目标1) 提供战略和内容方面的专业知识，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做法、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，用于在儿童身上进行临床试验。由于我们合作伙伴的努力 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。这一新的基础设施将由不同的赞助商利用，以产生监管级别 高质量的数据和改善儿科公共卫生。

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.