Children are underrepresented in new drug studies, raising questions about how effective and safe new medications – tested only in adult patients – may be for kids, new research suggests.
The findings, based on analysis of public-access data conducted by researchers at Duke University, suggest more needs to be done to improve the quality and efficiency of clinical trials.
"Although children comprise one-quarter of the population in the United States, they are greatly underrepresented in the clinical trial process that is designed to lead to new and better therapies, determine appropriate drug dosages, and establish standards of practice," said Dr. Sara K. Pasquali, who headed up the study, published in the journal Pediatrics.
The analysis was undertaken as part of a public-private partnership involving Duke and the U.S. Food and Drug Administration to identify practices that will improve clinical trials. Pasquali, co-director of the Michigan Congenital Heart Outcomes Research and Discovery Program at the University of Michigan, said researchers examined more than 60,000 trials from 2005 to 2010 using information from the ClinicalTrials.gov registry. The on-line registry aims to improve patient access to clinical trials and provide greater transparency of trial results and data.
The researchers found only about 5,000 of those trials were specifically designed to enroll children under the age of 18. Enrollments tended to be small in such studies, making it difficult to obtain meaningful information.
"With fewer studies to guide therapeutic decisions, treatments and outcomes for young patients often vary widely from center to center,” Pasquali said.
"For the vast majority of therapies used on children every day in United States and around the world, clinicians lack basic data to support decisions about the correct dosage, the best type of medication to use, and the appropriate situations to provide treatment. Without that information, it really puts physicians and the children we're treating at a significant disadvantage."