Rare Disease Report

FDA Closes Loophole, Enforces Pediatric Studies for Drug Developers

DECEMBER 19, 2017
Mathew Shanley
Today, the U.S. Food and Drug Administration (FDA) took steps toward preventing drug developers from sidestepping pediatric studies.

A draft guidance was issued to sponsors of drugs and biological products that submit orphan drug designation requests. It provides clarity on requirements for orphan drug designation, noting that the designation will not be granted unless the pediatric subpopulation meets 2 criteria.

The Clarification of Orphan Drug Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases states that if orphan drug designation for a pediatric subpopulation of a common disease is requested by a sponsor, it will not be granted if the prevalence of the disease is greater than 200,000, even if the pediatric subpopulation prevalence is below 200,000, unless:
  • The disease in the pediatric population constitutes a valid orphan subset, and the drug meets all other criteria for orphan designation; or
  • 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 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. in the official FDA press release.

An example given in the draft guidance suggests that, previously, if the FDA grants pediatric-subpopulation designation for a sponsor’s drug for pediatric ulcerative colitis (UC) and the sponsor submits a New Drug Application (NDA) and Biologics Drug Application (BLA) for its drug to treat UC in adults, the sponsor would be exempt from having to conduct pediatric studies by having the pediatric-subpopulation designation for pediatric UC. Obtaining pediatric-subpopulation designation provides orphan incentives to study the drug in the pediatric population with the disease but does not mandate the sponsor to conduct those studies.

The FDA intends to discontinue the granting of pediatric-subpopulation designation to close this loophole.

“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,” said Gottlieb. “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."

The agency is encouraging public comment on the draft guidance.

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