News & Events

FDA In Brief: FDA takes step to close orphan drug loophole that let drug developers sidestep pediatric studies

For Immediate Release: Dec. 19, 2017

Media Inquiries

  Sandy Walsh
  301-796-4669

"The scientific and medical communities recognize that treating children safely and effectively requires data from pediatric studies. Children are different from adults in the way they may metabolize a given drug and in the side effects they may experience. 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,” said FDA Commissioner Scott Gottlieb, M.D. “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. By making this designation, drug developers took advantage of an unintended loophole in a law that was, to the contrary, specifically designed to ensure that drugs are studied for pediatric indications. Today, we’re closing that loophole and announcing that we no longer intend to grant pediatric-subpopulation designations through the orphan drug program."

Today the U.S. Food and Drug Administration issued a draft guidance for industry, Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases to provide clarity for industry on orphan drug designation requirements, noting that if a sponsor requests orphan drug designation for a pediatric subpopulation of a common disease, and even if the pediatric subpopulation prevalence is below 200,000, if the prevalence of the disease as a whole is greater than 200,000 the FDA will not grant orphan drug designation to that pediatric subpopulation unless:

  1. The disease in the pediatric population constitutes a valid orphan subset, and the drug meets all the other criteria for orphan designation; or
  2. The sponsor can adequately demonstrate that the disease in the pediatric subpopulation is a different disease from the disease in the adult population, and the drug meets all other criteria for orphan designation. For example, if, as a scientific matter, efficacy from clinical studies in the adult population could not be extrapolated to the pediatric subpopulation, such information may help demonstrate that the disease in the pediatric and adult populations may be considered different diseases.

The FDA will continue to make full use of tools provided by Congress to apply incentives for the efficient development of rare disease therapies and for stimulating more pediatric research. These steps will help us achieve the agency’s ultimate goal of facilitating a more efficient, scientifically advanced, predictable, and modern approach to the approval of safe and effective treatments for rare diseases.

The agency welcomes public comment on the draft guidance, which is part of the Orphan Drug Modernization Plan announced in June 2017.

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