Federal incentives have driven up the number of new biologic drugs that reference pediatric uses in their labels, as well as boosted related pediatric drug trials, researchers reported.
Almost 90% of the biologics developed since 1997 in this analysis have pediatric information in their labeling, have at least one registered pediatric study (84%), or both, Marilyn Field, PhD, of the Institute of Medicine (IOM), and colleagues reported online in Pediatrics.
There's been "substantial investigation of pediatric uses of biologics," they wrote.
In the 1990s, policymakers developed incentives for pediatric studies of drugs, which were incorporated into the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA).
The contributions of these policies to pediatric labeling of drugs in general have been assessed, but the specific impact on biologics in children hasn't been looked at systematically, Field and colleagues wrote.
To do so, they assessed 96 biologics -- not including vaccines -- that were first licensed by the FDA between 1997 and 2010. These included antitoxins, blood and blood components, tissues, allergenics, proteins, and other molecules.
They conducted a separate analysis of the 55 vaccines licensed during that time.
Overall, Field and colleagues found that 61% of those 96 biologics have some pediatric information in their labeling, and about half (49%) are actually labeled for pediatric use.
About 10% aren't labeled for pediatric use but include pediatric study information, and 2% contain only a pediatric safety warning based on adverse event data.
The authors noted that the majority (89%) are ultimately labeled for pediatric use, contain pediatric information in their labels, have a registered pediatric study, or a combination of these factors.
For the 11 products with no label information or registered studies, four are similar to other products that have pediatric labeling, and most of the rest are approved for indications that are rarely or never diagnosed in children, such as prostate cancer or chronic refractory gout, they noted.
For the 55 vaccines that were analyzed, only 5% were neither labeled for pediatric use or were without registered pediatric studies.
There were several limitations in trying to determine whether PREA has contributed to pediatric studies of biologics. First, Congress doesn't require the FDA to post relevant documents for all PREA-related labeling changes involving biologics, nor do the agency's approval letters routinely mention PREA requirements.
Also, the FDA didn't initially require that such study results be submitted via licensing applications, which are easier to retrieve then less formal submissions, they wrote.
They also cautioned that the incentives of Orphan Drug Act, which provides 7 years of marketing protection, are stronger than the incentives of BCPA. That likely encouraged some studies of products for rare conditions that affect children, which would be exempt from PREA requirements, they wrote.
Still, they concluded, it "seems reasonable that the requirements of PREA and its predecessor policies have prompted pediatric studies that would not otherwise have been undertaken for products aimed at conditions most often found in adults but also affecting children."
The study was supported by the FDA via the National Academy of Sciences.
The researchers reported no conflicts of interest.
- Reviewed by Robert Jasmer, MD Associate Clinical Professor of Medicine, University of California, San Francisco and Dorothy Caputo, MA, BSN, RN, Nurse Planner