The Food and Drug Administration moved Friday to close a loophole that had inadvertently excused some drugmakers from having to conduct clinical trials in children for drugs to treat diseases that commonly affect adults.
In a Federal Register notice, the FDA announced that it had finalized a draft guidance originally issued in December intended to close a loophole in the Pediatric Research Equity Act that allowed drug companies making medicines for non-orphan diseases in adults – meaning those affecting more than 200,000 patients – to get pediatric subpopulation designation. Consequently, companies were exempt from conducting the clinical trials in children that PREA would normally have required.
In the December announcement of the draft guidance, the agency said that in cases of diseases affecting more than 200,000 adults, even if the pediatric population was fewer than 200,000, it would only give orphan designation if the pediatric population “constitutes a valid orphan subset” or if the company could adequately demonstrate that the pediatric disease was sufficiently different from its adult counterpart.
“Addressing the inadequate testing of drugs in pediatric populations has been a priority for the FDA, the medical community and Congress and has led to important laws to ensure this important, vulnerable population is not overlooked,” FDA Commissioner Scott Gottlieb said in a statement in December. “Unfortunately, some of the efforts intended to encourage pediatric drug development have been used by some drug developers to avoid their obligations to study drugs in pediatric populations. One such area is the longstanding practice of designating pediatric subpopulations of common diseases as orphan conditions.”
Under FDA regulations, a drug qualifies for orphan designation if it targets a population of fewer than 200,000 patients or, for more common diseases, would leave a drugmaker unlikely to recoup the expenses of developing and marketing the drug. The guidance was part of the agency’s broader Orphan Drug Modernization Plan, which was announced in June 2017. The policy was issued in response to a significant uptick in the number of requests the FDA has received for orphan drug designations, which rose from 267 in 2012 to 568 in 2016.
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