FDA to Congress: Pediatric Information Lacking in 36% of Relevant Orphan Drug Labels

Regulatory NewsRegulatory News | 26 August 2019 |  By 

In a report to Congress, the US Food and Drug Administration said 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.

FDA research, conducted as part of the FDA Reauthorization Act of 2017 (FDARA), found that of all drugs that were approved for an orphan indication between 1 April 1999 and 31 August 2018, a total of 548 orphan indications were approved. Of those 548, FDA identified 348 approved orphan indications for which pediatric labeling was warranted.

And of those 348, “127 indications [for 98 drugs] relevant to the pediatric population lacked pediatric information, either having no pediatric information at all (81 indications; 23 percent of all indications relevant in the pediatric population) or missing information for some relevant pediatric age ranges (46 indications; 13 percent of all indications relevant in the pediatric population), especially for the youngest populations.”

Examples of drugs listed in the report that lack any pediatric information include Roche’s Avastin (bevacizumab), Amgen’s Sensipar (cinacalcet), J&J’s Imbruvica (ibrutinib) and Bristol-Myers Squibb’s Yervoy (ipilimumab) and Opdivo (nivolumab).

But the report also found that FDA has seen an increase since 1999 in the number of approved orphan products with indications that are fully labeled for pediatric use.

“During the review period for this report, 221 indications relevant to the pediatric population were fully labeled for pediatric use; most of these indications (90 percent) included appropriate pediatric labeling at the time of the original orphan product approval. This increase is most notable over the last 3 years,” FDA said.

FDA said it supports ensuring that companies work to obtain labeling for all appropriate pediatric age groups and that it will continue to work to achieve this goal.

The report comes as FDA last summer closed a loophole in the Pediatric Research Equity Act (PREA) that exempted companies from conducting pediatric study requirements for orphan designated drugs.

The exemption allowed sponsors to get a pediatric subpopulation designation for a non-orphan disease in adult populations and be exempted from conducting pediatric studies normally required under PREA when applying for an adult indication for that disease. The language on closing the loophole was initially going to be included in FDARA. 

Report to Congress


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