Labeling Pediatric Products: BPCA Efforts and Orphan Drug Opportunities.

Objective: Although the Orphan Drug Act (ODA) has substantially increased the number of therapies available for rare diseases, there are still unmet needs for drug development in pediatric patients. The current study represents an exploratory collaborative effort from the National Institutes of Health (NIH) and the US Food and Drug Administration (FDA) to use publicly available data to advance the assessment of orphan products not fully labeled for pediatric use. We first document the efforts to label drugs for pediatric use by the NIH under the Best Pharmaceuticals for Children Act (BPCA). Then, we explore whether publicly available pediatric data exist for orphan drugs previously identified as not fully labeled for pediatric use.

Methods: The NIH BPCA efforts have labeled 20 drugs for pediatric use. We conducted additional analyses of drugs not fully labeled for pediatric use under the ODA for assessment in the relevant pediatric age range using data from published studies.

Results: Tafenoquine and miltefosine were identified, and noncompartmental analyses were conducted using published data. Tafenoquine exposures of pediatric patients (2-15 years old) with body weight-based dosing were comparable to the approved patient population with 300 mg daily, along with acceptable variability within the observed subjects. High similarity was also found between studied pediatric patients (2-12 years old) and the approved patient population with the same dose of 2.5 mg/kg/day in the miltefosine exposure-response analysis.

Conclusion: Tafenoquine tablets and miltefosine capsules could be considered as additional examples of the BPCA effort to fully label drug products for the pediatric patient population.