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A Review of Clinical Studies Supporting Pediatric Labeling Changes Approved by the US Food and Drug Administration Leveraging Existing Clinical Data (301196)

*Jaejoon Song, US Food and Drug Administration, Center for Drug Evaluation Research, Office of Translational Scienc 

Keywords: pediatric labeling, historical clinical data, US Food and Drug Administration

The majority of new drugs approved by the US Food and Drug Administration (FDA) have been approved without pediatric labeling information. The Best Pharmaceuticals with Children Act (BPCA) in 2002 and the Pediatric Research Equity Act (PREA) in 2003 gave FDA authority to require drug manufacturers to submit pediatric study data for a new drug. Under this new standard, an increasing number of drugs were approved with pediatric labeling. However, challenges remain in conducting clinical trials in children, often triggered by difficulties in recruiting pediatric patients. Recently, there has been methodological developments for leveraging historical clinical data for regulatory approval. Such approaches can be particularly relevant for clinical investigations in pediatric population. We review the study designs and statistical analyses performed in safety and efficacy clinical trials conducted under BPCA or PREA using the FDA’s New Pediatric Labeling Information Database. This database, launched in 2012, houses results of clinical studies conducted for new pediatric labeling changes from 1998 to December 2019. We characterize clinical studies that leverage existing clinical data in studies supporting pediatric labeling changes.