The US Food and Drug Administration (“FDA”) provided a report to Congress, entitled Pediatric Labeling of Orphan Drugs, detailing the results of a study that examined drugs that have been approved for an orphan indication, but which lac02k pediatric labeling information for that orphan indication. The report concluded that “there is a public health need for additional pediatric information in labeling for over one-third of approved orphan indications that are relevant in the pediatric population.”
This study, which was required by the FDA Reauthorization Act (“FDARA”) of 2017, examined drugs approved for an orphan indication between April 1, 1999 and August 31, 2018. The study results are summarized, as follows.
548 orphan indications were approved during the relevant period.
200 orphan indication do not warrant pediatric labeling, because, for example, the indications were for adults only, or pediatric studies were “impossible of highly impracticable.”
348 orphan indications are relevant to children and warrant pediatric use labeling.
64% (221 of 348) of the orphan indications relevant to children are fully labeled for pediatric use.
The remaining 35% (127 of 348) are not fully labeled for pediatric use, having either none (81) or some (46) pediatric labeling. The latter group typically included labeling related to efficacy and safety, but lacked information for use in specific populations, generally the youngest populations. Of the 127 not fully labeled, there are ongoing studies to address pediatric labeling in connection with 29 of those.
The report also noted that the drugs that were most likely to be fully labeled for pediatrics were those that treat exclusively pediatric disease, or diseases for which pediatric patients comprise a majority of the overall disease population. However, indications for diseases where pediatric patients comprise a low percentage of the overall disease population were less likely to be fully labeled for pediatrics.
The drugs identified in the report as having either no or incomplete pediatric labeling span a broad range of indications, including for example, Abilify to treat Tourette’s disorder, Avastin to treat malignant glioma, Adcetris and Opdivo to treat Hodgkin’s lymphoma, Repatha to treat familial hypercholesterolemia, Kalydeco to treat cystic fibrosis, and Harvoni to treat chronic hepatitis C infection.
The FDA included a policy recommendation to label appropriately drugs with orphan designations for children, since over one-third of orphan indications that are relevant to pediatric patients lacks relevant labeling information. The FDA also noted that it will “continue to work with stakeholders to achieve this goal.”