Encourage clinical evidence for pediatric medication use

March 1, 2011

Studying drugs in younger populations is challenging, and the scarcity of pediatric studies limits the ability of doctors and scientists to accurately predict drug dosing, safety and efficacy in children.

Key Points

STUDYING DRUGS IN younger populations is challenging, and the scarcity of pediatric studies limits the ability of doctors and scientists to accurately predict drug dosing, safety and efficacy in children. Nearly 50% of products are labeled with pediatric information, indicating positive, negative or inconclusive results but not necessarily data supporting a specific indication for the drug, according to the U.S. Food & Drug Administration (FDA).

"Children with medical conditions require treatment of those conditions just as adults require treatment," says Lisa L. Mathis, MD, associate director, Office of New Drugs, FDA. "Clinicians are often forced to treat patients who require medical care without the benefit of data. While this may not be optimal, the lack of treatment may be worse."

To address this gap, last year the National Institutes of Health (NIH) developed an initiative, granting up to $95 million over the next seven years to conduct pediatric clinical trials in a safe environment and to weigh the benefits and risks of drug and device treatment in children. Danny Benjamin, MD, and his team at the Duke Clinical Research Institute, are the recipients of NIH's large grant, enabling trials to test drugs for life-threatening conditions and those for more common ailments among children.

The PREA allows the FDA to require studies when an application is submitted for a product with a new indication, active ingredient, formulation, dosing regimen or route of administration, Dr. Mathis says. Companies can receive waivers for the studies if the condition, targeted by the drug, does not occur or is rare in the pediatric population. The last two pieces of legislation sunset in 2012 but could be renewed.