FDA: Drug Companies Can’t Game Pediatric Drug Laws Anymore

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By Jacquie Lee

Drug companies can’t avoid including kids in certain clinical studies now that the FDA has finally closed a loophole within a pediatric research law.

The Food and Drug Administration has been emphasizing the need for kids to be included in drug trials since Scott Gottlieb took over as commissioner of the agency in May 2017. The Pediatric Research Equity Act (PREA), passed in 2003, requires drug companies to study some products in children. Drug companies have been skirting the requirement using certain exemptions. The biggest exemption revolves around so-called orphan drugs that treat small patient populations and rare diseases.

The agency gives special incentives to manufacturers—like tax credits and seven extra years of marketing exclusivity—for producing drugs that treat diseases affecting 200,000 or fewer people in the U.S. Drugmakers that create an orphan drug don’t need to conduct research for that drug on pediatric populations. Consequently, drug companies have been designating more and more drugs as orphan drugs in order to skirt the pediatric research responsibility.

The FDA will now be cracking down on what it considers an orphan drug. The agency “does not expect to grant any additional orphan drug designation to drugs for pediatric subpopulations of common diseases,” as it had in the past.

This latest industry advice, a notice for which is set to be published in the Federal Register July 27, closes that loophole. It also helps fulfill the agency’s goal of beefing up research on treatment for children.

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