FDA Closes Pediatric Study Loophole

The U.S. Food and Drug Administration (FDA) has finalized guidelines eliminating a loophole that allowed drug manufacturers to avoid pediatric trials of its products designated as “orphan drugs.” This action was a top goal of FDA Commissioner Scott Gottlieb, MD.

The FDA encourages the production of orphan drugs – those indicated for the treatment of diseases that affect fewer than 200,000 individuals within the U.S. and, therefore, are unlikely to be profitable – by offering drug manufacturers incentives, including tax breaks and marketing exclusivity.

Since the passage of the Pediatric Research Equity Act (PREA) in 2003, drug companies have been required to include children in clinical studies to receive FDA approval on their products, but the rule has not been applied to orphan drugs. Manufacturers have exploited this exemption, seeking to designate their products as orphan drugs to avoid the pediatric study requirement.

To close this loophole, the agency will tighten restrictions on which products are granted orphan drug designation. However, the agency said that it may still grant the designation in specific cases, including for pediatric subsets of common diseases, if it would be inappropriate to treat adults with the same drug or when the pediatric and adult subsets represent distinct diseases.

Sources: Bloomberg, July 26, 2018; Endpoints News, July 27, 2018.