The Duke Clinical Research Institute and its strategic partners have been awarded a grant from the U.S. Food and Drug Administration (FDA) to establish a coordinating center for a Global Pediatric Clinical Trials Network (G-PCTN). The G-PCTN will support efficient pediatric clinical trials worldwide by developing scientific and operational infrastructure, fostering collaborative networks, sharing knowledge, and engaging stakeholders. The principal investigators for this program will be Drs. Danny Benjamin and Michael (Micky) Cohen-Wolkowiez.
“Although we’ve made a lot of progress in recent years, pediatric trials are still hard to complete successfully. There are perennial challenges – enrolling patients, finding access to supporting infrastructure, navigating a complex regulatory environment – that affect everyone working to advance pediatric research, but they can be especially challenging for research sites that don’t have access to resources or experience,” said Benjamin. “We’ve had great success with the Exclusivity Program, and NICHD’s off-patent program; but we need to work through some of the challenges that folks have had in meeting the requirements outlined by the Pediatric Research Equity Act. It’s crucial to have success across new therapeutics for children.”
Although the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) were both implemented to encourage research sponsors to conduct more pediatric clinical trials, many drugs used in children lack sufficient information to guide their safe and effective dosing. Further, 42% of pediatric trials done under BPCA have not successfully supported pediatric indications.
“Despite how vital it is for children’s health, proper dosing information is still sorely lacking for many important therapies used in children and infants,” Cohen-Wolkowiez said, noting that the relatively small numbers of patients eligible to enroll in pediatric trials of therapies for rare diseases adds an additional layer of difficulty. “This Network will provide a key opportunity to address inefficiencies and build capacity across the globe for conducting more effective pediatric clinical trials, which will help inform the decisions parents and healthcare providers make when caring for our youngest patients.”
The grant will be used to create a coordinating center comprising 3 cores for network operations, patient engagement, and scientific oversight and 5 clinical study groups devoted to study design, dosing, regulatory/pharmacy, network partnerships, and rare diseases. Benjamin, who also serves as Principal Investigator for the Eunice Kennedy Shriver National Institute of Child Health and Human Development’s Pediatric Trials Network (PTN) envisions the programs working synergistically: “There are natural interactions, or lessons learned, across the trials conducted in the older, off-patent medicines and the trials conducted for newer therapeutics.”
The creation of the Network reflects consensus findings from a multistakeholder group that included regulators, industry, academia, patient advocacy groups, disease networks, and parents. The group noted that no one set of stakeholders can address these problems in isolation; instead, a global network involving all stakeholders could better ensure successful pediatric trials, especially those being conducted in patients with rare diseases.
The G-PCTN will leverage and extend the extensive infrastructure, networks, and experience already in place via the PTN, whose administrative coordinating center is also located at the DCRI.