The American Academy of Pediatrics (AAP) posted an article on the AAP News website to spotlight the work done by the Pediatric Trials Network (PTN) to provide much-needed dosing, safety and efficacy information for off-patent drugs used in children. The article was written by Perdita Taylor-Zapata of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and P. Brian Smith of the PTN and the Duke Department of Pediatrics.
Despite pediatric medicine’s long history of catastrophic events resulting from inadequate study of drugs prior to their widespread use, the majority of drugs used in children have undergone insufficient study to receive pediatric-specific labeling from the FDA. To fill this troubling information gap, the NICHD established the PTN at the Duke Clinical Research Institute. The PTN generates data to help regulators update drug labels for safer and more effective use of medications in infants and children, in keeping with the goals of the Best Pharmaceuticals for Children Act (BPCA).
BPCA became law in 2002 and provides a mechanism to study off-patent drugs through a collaborative effort of the National Institutes of Health (NIH) and the FDA. This effort includes identifying drugs and therapeutic areas that lack pediatric dosing, safety or efficacy data; sponsoring clinical trials for prioritized drugs; and submitting study results to the FDA for consideration of label modification.
PTN trials are conducted across the U.S. and other countries in partnership with the NIH, and eight clinical trials are ongoing. More than 100 clinical sites are enrolling children in PTN trials, and more than 7,000 children have been enrolled to date.
The PTN has submitted data to the FDA for 21 drugs and devices. To date, eight label changes have been made based on clinical trials sponsored by the NIH BPCA program, including recent label changes to lisinopril and meropenem.