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# No Orphan Drug Designation for Some Pediatric Subpopulations
- URL: https://www.fdaweb.com/no-orphan-drug-designation-for-some-pediatric-subpopulations/
- Published: 2018-01-02T12:00:00.000Z
- Updated: 2026-09-14T23:09:45.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5140378

In a draft guidance, *Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases*, FDA says it no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases unless use of the drug in the pediatric subpopulation meets the regulatory for an orphan subset, or unless the disease in the pediatric populations is considered a different disease from the disease in the adult population. The [document](http://www.fda.gov/downloads/regulatoryinformation/guidances/UCM589710.pdf?ref=fdaweb.com) says that historically, FDA granted orphan drug designation to drugs for use in pediatric subpopulations of common diseases or conditions if the prevalence in the pediatric subpopulation in the U.S. was below 200,000\. The agency says it began that practice before there was pediatric-specific legislation to promote the study of drugs in the pediatric population.

“Since the time that FDA began granting pediatric-subpopulation designation to incentivize pediatric studies,” it says, “Congress created several programs that promote pediatric studies and changed the regulatory landscape…. The introduction of these statutory provisions has not only rendered pediatric-subpopulation designation no longer necessary to promote pediatric studies, but has inadvertently introduced complications that have the potential to actively inhibit the achievement of that goal.”