Children Underrepresented in Clinical Drug Trials: Study

MONDAY, Oct. 1 (HealthDay News) -- Drug studies enrolling adults far outnumber clinical trials involving children, and the scope of research on children is also more limited than it is for adults, new research from Duke University finds.

This puts children and their physicians at a significant disadvantage, the researchers concluded.

"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 study first author Dr. Sara Pasquali, who is now co-director of the Michigan Congenital Heart Outcomes Research and Discovery Program at the University of Michigan in Ann Arbor.

"For the vast majority of therapies used on children every day in the 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," she said in a Duke news release.

In conducting the study, which was published online Oct. 1 in the journal Pediatrics, the researchers examined more than 60,000 research trials from 2005 to 2010 using data entered into the ClinicalTrials.gov registry. The analysis revealed that slightly more than 5,000 of those trials (about 8 percent) were designed for children younger than 18 years old.

The researchers also found that 23 percent of the pediatric trials included infectious diseases or vaccine studies and 13 percent involved psychiatric or mental health studies.

The fact that many pediatric diseases are uncommon and diverse could play a role in the small number of drug studies involving children, the researchers suggested.

"Many pediatric diseases are relatively rare, as opposed to something like adult coronary artery disease. As a result, it can take much more time to build a research infrastructure, often involving multiple hospitals, to enroll enough patients in a study," Pasquali said. "But with fewer studies to guide therapeutic decisions, treatments and outcomes for young patients often vary widely from center to center."

The study also revealed that drug studies involving children often were small. As a result, it's harder to draw conclusions that could be applied to a large population of people. The researchers said larger pediatric trials that focused on the most pressing questions would be more helpful than many small trials.

They noted that their findings should provide insight for researchers, funders and policymakers who have addressed this issue in recent years.

"Conducting clinical trials with children is a complex issue -- they are not volunteers, they are dissimilar in terms of size and disease condition, and the number of patients is a lot less than what we would find among adults," study senior author Dr. Jennifer Li, a member of the Duke Clinical Research Institute, said in the news release. "This analysis provides one snapshot in time, and it's good to know what the research landscape is so that we can address where we should focus our efforts."

The study was part of the Clinical Trials Transformation Initiative, a public-private partnership founded by the U.S. Food and Drug Administration and Duke University to identify ways to improve the quality and efficiency of clinical trials.

More information

The U.S. Food and Drug Administration provides more information on drug research and children.

SOURCE: Duke University, news release, Oct. 1, 2012

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