Comments to the FDA Supporting Draft Guidance Restricting Orphan Drug Designations for Pediatric Uses

January 11, 2018

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Public Citizen filed comments supporting a Food and Drug Administration (FDA) draft guidance, in which the FDA declared that it would no longer grant orphan drug designation to drugs for use in pediatric subpopulations of common diseases or conditions unless: 1) The disease in the pediatric population constitutes a valid orphan subset, or 2) the disease in the pediatric subpopulation is a different disease from the disease in the adult population.

View Public Citizen’s 2015 report on orphan drugs.