July 23, 2014
Effects of FDA Program in Increasing Pediatric Indications
for Medications

Silver Spring, MD—Some therapeutic areas were more likely than others to obtain pediatric indications for medications, according to a new study.

Under the pediatric exclusivity (PE) program, oncology, cardiovascular, and endocrine drugs were less likely to demonstrate efficacy compared with gastrointestinal and pain/anesthesia drugs, according to researchers from the FDA.

The report, published online early by the journal Pediatrics, also found that drugs studied later in the program were less likely to demonstrate efficacy, but that sales, initiation process, and small disease population appeared to have little influence on the outcomes.

The article’s background notes that other than vaccines and otitis media medications, most products prescribed for children have not been studied in the pediatric population. In 1997, Congress enacted the PE legislation, which provides 6 months of additional market protection to drug sponsors in exchange for studying their products in children.

For the study, researchers reviewed requests for pediatric studies and subsequent labeling for drugs granted PE from 1998 through 2012, looking at therapeutic group, year of exclusivity, product sales, initiation process, and small disease population.

Results indicate that, from 1998 through 2012, the FDA issued 401 pediatric study requests, with studies completed and exclusivity granted for 189 drugs. New pediatric labeling was awarded to 173 drugs, 92%, with 108, 57%, also receiving a new or expanded pediatric indication.

Efficacy was not established for 78 drugs, and three drugs had nonefficacy trials.

“Therapeutic area and year of exclusivity influenced the likelihood of obtaining a pediatric indication,” the authors conclude. “Positive and negative outcomes continue to inform the construct of future pediatric trials.”

A study late last year reported in JAMA Pediatrics that “federal legislation has led to a notable increase in pediatric studies submitted to the [FDA], resulting in new pediatric information in product labeling. However, approximately 50% of drug labels still have insufficient information on safety, efficacy, or dosing in children.”

That study also suggested that the benefit was greater for older children, especially bemoaning the lack of neonatal information in labeling. “Few drug labeling changes made under pediatric legislation include neonates. Most drugs studied are either not used or rarely used in U.S. [neonatal intensive care units]. Strategies to increase the study of safe and effective drugs for neonates are needed.”

U.S. Pharmacist Social Connect