Duke Medicine: Program to study pediatric dosing, prescription medicine safety

Duke Medicine is part of a groundbreaking initiative to reduce the risk and dangers faced everyday by millions of children who are prescribed medications that lack definitive data about pediatric dosing, efficacy and safety.

"It's staggering to consider that less than 10 percent of licensed therapeutics have been adequately studied in children, so much of pediatric drug use is based on an educated guess by a pediatrician using studies conducted in adults, who often absorb drugs differently or experience different side effects than children," said Daniel K. Benjamin, Jr., MD, PhD, professor of pediatrics at Duke and associate director of the Duke research institute, who will lead the initiative.

The program was conceived to create a scientific, technical, and administrative infrastructure for conducting pediatric clinical trials in an environment that provides optimal safety for the research participants and sophisticated expertise to determine the balance of benefits and risks of drug and device treatment of medical illnesses in children.

A network of hospitals will conduct 16 trials spanning a variety of areas, including cardiovascular diseases, cancer, infectious diseases, gastroenterology, respiratory diseases, neonatology, and medical devices. The drugs and devices have yet to be determined. The compounds studied will be those which have already been on the market long enough that their patents have expired, otherwise known as generic drugs.

The first trial in this project will likely be conducted to determine optimal dosing of medications used to treat high blood pressure in children, Benjamin said. Most of the trials will enroll between 100 to 200 children. Aspects of this network will include product formulation, drug distribution and device development. "In effect, each trial will become a 'mini' drug development program," Benjamin said.

Another objective for this project is to use the data collected from the initiative to help inform pediatric drug labeling, and to provide regulators, pediatricians, and researchers with new information on medication response in children.

"The Pediatric Trials Network has the potential to correct a problem that continues to surprise people—we have inadequate information about the proper treatment of children, even for commonly used treatments that have been on the market for years," said Robert M. Califf, MD, director of the Duke Translational Medicine Institute and vice chancellor for clinical research at Duke University. "This team composed of the nation's experts in therapeutics in children should provide information that will be critical to pediatricians and the children they treat."