Drugs

FDA Report on Pediatric Labeling in Orphan Drugs

A new report from FDA says that from 4/1/1999 to 8/31/2018 there were 548 orphan indications approved. Of these, FDA identified 200 orphan indications that did not warrant pediatric labeling (adult only indications or indications for which pediatric studies were impossible or highly impracticable). “Of the total of 548 orphan indications , FDA deemed 348 indications (64%) to be relevant to children, thus warranting pediatric use information in the labeling for the indication,” it says.

The report further found that for the 348 child-relevant indications , 221 (64%) included pediatric use information and were considered to be appropriately labeled (i.e., the labeling contained adequate efficacy, safety, dosing, and age-appropriate formulation information to support its use in the full range of affected pediatric patients) for pediatric use. “Information used to support appropriate pediatric labeling was derived from a variety of sources," the report says. "In addition to data from pediatric trials conducted by the sponsor for the orphan indication, other sources that contributed to pediatric labeling information included, where appropriate, published literature, extrapolation of efficacy from adults or other pediatric populations, safety data obtained from pediatric trials for other indications, pharmacokinetic modeling and simulation data, and studies conducted under the Animal Rule.”

Additionally, FDA’s report identifies 127 orphan indications that were not fully labeled for the pediatric population. "The 127 orphan indications missing pediatric information in labeling corresponded to 98 drugs approved for one or more orphan indications (19 of the 98 drugs were approved for more than one orphan indication),” it says. “Approximately two-thirds (81) of these indications had no pediatric information at all, while the remainder had some pediatric labeling.”

The report was mandated by the FDA Reauthorization Act of 2017 and called on FDA to examine the lack of pediatric information in the labeling of drugs for indications that have received an orphan designation.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome