Background and Objectives: According to the Food and Drug Administration (FDA), prior to 2007 more than 80% of drugs approved in adults were being used off-label in pediatrics. In order to improve data on the use of drugs in children, Congress authorized two acts, the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). The impact of these acts on the age groups for which new drugs are approved has not been evaluated. The objective of this study was to evaluate the age groups for which novel drugs are approved and studied after approval. Methods: We collected information on all drugs approved by the FDA between January 2013 and December 2017. Radiopharmaceuticals and diagnostic drugs were excluded. The first approved U.S. package inserts (USPI) were reviewed. Five pediatric-trained clinical pharmacists further analyzed the list of drugs and determined the potential age ranges that could benefit from each drug. Pediatric age groups were divided into neonate (birth - <1 month), infant (1 month - <2 years), child (2 years- <12 years) and adolescent (12-17 years). The primary endpoint of this study was to determine the number of drugs approved in each pediatric age group as a percent of the total drugs approved for that time period. Secondary endpoints included the number of drugs approved with the potential to be used in younger ages than those indicated in the USPI, and the number of drugs with industry-sponsored clinical trials in these younger populations Results: A total of 173 novel drugs approved from 2013-2017 met the criteria for analysis. The number of medications approved in each age group is listed in Table 1. Analysis indicated that 51% (89/173) of novel drugs had the potential for use in younger age groups than those indicated in the USPI, which was similar in all years studied (Table 2). Based on the ages these drugs are appropriate for, only 28% (25/89) of these novel drug products were adequately studied down to the youngest appropriate age (Table 2). Conclusion: A large gap was identified between the age for which drugs are approved and the age for which drugs would be needed. While the increase in pediatric drug approvals is positive, there is still a need to study these drugs in younger age groups. In particular, there is a significant data gap due to a lack of studies in neonates and infants.

Table 1

Age Groups for Which New Drugs were Approved.

Table 1

Age Groups for Which New Drugs were Approved.

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*Age ranges for use were determined by five pediatric-trained pharmacists **Based on age guidelines from pediatric-trained pharmacists and industry-sponsored trials listed on the clinicaltrials.gov website

*Age ranges for use were determined by five pediatric-trained pharmacists **Based on age guidelines from pediatric-trained pharmacists and industry-sponsored trials listed on the clinicaltrials.gov website

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