Federal policy that encourages the study of drugs in pediatric patients has led to few pharmaceutical label changes that include new information on drug effects in neonates, in a study published Dec. 9 in JAMA Pediatrics.
Dr. Matthew M. Laughon of the department of pediatrics, University of North Carolina at Chapel Hill, and his colleagues looked at drug studies between 1997 and 2010 that included neonates as a result of legislation encouraging this type of research. They then examined the use of all drugs in neonates admitted to 290 neonatal intensive care units between 2005 and 2010 (JAMA Pediatr. 2013 Dec. 9 [doi:10.1001/jamapediatrics.2013.4208]).
Few labeling changes resulted from neonate studies, and even the drugs that did receive labeling changes following studies in neonates were used infrequently, the researchers found.
They identified 28 drugs studied in neonates and 24 related labeling changes. Eleven of those labeling changes established safety and effectiveness of the drugs in neonates.
Meanwhile, in their review of 446,335 hospitalized infants, the researchers identified 399 drugs used and more than 1.5 million drug exposures in the first 28 postnatal days. Close to half of the 28 drugs studied in neonates were not used in any of these patients, and almost another third of the drugs were used in fewer than 60 neonates each.
Since 1997, a combination of federal incentives and requirements has significantly increased pediatric drug research and development and stimulated an increase in pediatric labeling, the study showed. Federal lawmakers permanently reauthorized these incentives in 2012.
Although many label changes have resulted from these policies, "approximately 50% of drug product labeling has insufficient information on the safety, efficacy, or dosing appropriate for use in children," the researchers said.
In addition, "few labeling changes have included infant-specific information," the researchers concluded. "Novel trial designs need to be developed and appropriate study endpoints must be identified and validated. Education of parents and caregivers regarding the need for studies of drugs being given to neonates will also increase trial success. The scientific and clinical research community will need to work together with the [Food and Drug Administration] to conduct essential neonatal studies."
The study used NIH Clinical and Translational Science Award biostatistical services through the division of pediatric quantitative sciences. Dr. Laughon receives support from the U.S. government for work in pediatric and neonatal clinical pharmacology and from the National Institute of Child Health and Human Development. His colleagues disclosed support from the NIH, the Department of Health and Human Services, Thrasher Research Fund, and industry sources.