Researchers Stress the Need to Strengthen Pediatric Clinical Trials Infrastructure
Children's National Physician Edward Connor Authors Article in Science Translational Medicine
Washington, DC— Strengthening the infrastructure required to conduct pediatric research is essential for obtaining information needed for the medical care and treatment of children, wrote Children’s National Health System’s Director of the Office of Innovation Development, Edward M. Connor, MD, MBE, the lead author of the article “Meeting the Demand for Pediatric Clinical Trials,” published in the journal Science Translational Medicine.
In recent years, legislation and regulations around the need for high-quality data on medications used in pediatric patients have gotten stronger, increasing the demand for pediatric clinical trials. To meet this demand, the authors, who include researchers from the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA), highlight the need for an improved and standardized research infrastructure to better guide treatment and care for children.
“Many drugs used in children have not been adequately studied in this population,” says Dr. Connor. “While this situation is improving there is still a lot of work to do to fill the knowledge gap, particularly in newborn infants and young children.”
The authors suggest that collaboration among the public and private sectors is needed to ensure that the proper expertise and infrastructure are in place.
“In the past, the required trials networks were created as the need arose then dismantled when it waned,” says Dr. Connor. “The increasing demand for data that meet regulatory standards for labeling means that we need to find ways to sustain infrastructure for pediatric trials. The pediatric research community, federal agencies, advocates, and industry are all making progress but a comprehensive and urgent solution is needed.”
According to Dr. Connor, pediatric trials that are used for drug labeling demand a level of quality standard that is higher than the average study. Having a network of experts in pediatric clinical trials and an enhanced infrastructure in place would ensure consistent, high-quality data are produced to guide therapeutic decisions in pediatric clinical practice.
“Establishing such an enhanced infrastructure has been a major goal of the child health platform within the Clinical and Translational Science Award (CTSA) National Consortium,” says Lisa Guay-Woodford, MD, Director of the CTSA program, a partnership between Children’s National and George Washington University. “This report provides a clear roadmap for our continued efforts.”
“There is a need for high-quality, compliant pediatric clinical trials infrastructure,” said study co-author Steven Hirschfeld, MD, PhD, Associate Director for Clinical Research at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). “The NICHD’s Pediatric Trials Network provides needed infrastructure for evaluating drugs commonly prescribed off label for the express purpose of obtaining information needed for pediatric labeling.
Co-authors of the article were William E. Smoyer, MD, Nationwide Children’s Hospital, Jonathan M. Davis, MD, Tufts Medical Center, Anne Zajicek, MD, PharmD, and Steven Hirschfeld, MD, PhD, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, Linda Ulrich, MD, Office of Orphan Products Development at the FDA, and Mary Purucker, MD, PhD, the National Center for Advancing Translational Sciences at the NIH.
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