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.
Failure to label devices and therapeutics for children threatens public health. We will use the partnership of a fully functioning pediatric clinical trials network and a collaboration of FDA; academia; industry; and patient engagement to 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.
