Disparity between children and neonatal subpopulations: A review of drugs and biologics approved by the FDA in the period 1980-2012

Background:  Recent regulatory policy changes implemented in the United States to improve drug labelling data for children and incentivizing pediatric research and development have been considered successful. Despite the success of these policies, many drugs currently administered to newborns are prescribed for off-label use and drug label information pertinent to neonatal use is absent or incomplete.

Objectives: We conducted an assessment of pediatric exclusivity and neonate information available in the labels of the drugs approved by the FDA in the period 1980-2012.

Methods: Analysis data retrieved from the FDA website Drugs@FDA included a review of drug labels for NDA/BLA approvals by the FDA between January 1, 1980 and December 31, 2012. Three sections were analyzed; “Indication and Usage”, “Dosage and Administration” and “Pediatric Use” for mention of “neonates” (defined as infants age 0-28 days and/or “neonates” or “newborn”). We collected information about pediatric exclusivity granted by the FDA. Drug classifications were obtained via the Anatomical Therapeutic Chemical Classification, WHO 2013. We identified drugs that included neonatal data and enumerated them by ATC class, employing descriptive statistics.

Results: Of the FDA approved and marketed new drugs and biologics (n=655), 128 (19.5%) had pediatric exclusivity and 67 (10.2%) had neonatal information contained in the label.  Pediatric exclusivity was granted to 12.8% of the products approved in the 1980s, 35.4% in the 1990s, and 9.0% in the period 2000-2012. The therapeutic classes with the largest number of pediatric exclusivities were nervous system (20.3% of all the pediatric exclusivities), cardiovascular system (18.8%), antiinfectives for systemic use (13.3%), antineoplastic and immunomodulating agents (11.7%), and alimentary tract and metabolism (10.9%). The therapeutic classes with the largest number of neonatal label information were antiinfectives for systemic use (28.4%), cardiovascular system (17.9%), alimentary tract and metabolism (17.9%), antineoplastic and immunomodulating agents (7.5%), and nervous system (6.0%).

Conclusions: Despite successful efforts to improve child mortality and drug development for children, neonates persist as an understudied and underserved population. These data indicate that neonate drug development should be prioritized in regulatory and public health policies to reduce disparity within the broad category of “pediatrics”.