Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases; Draft Guidance for Industry; Availability

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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases." FDA intends to no longer grant orphan drug designation to drugs for pediatric subpopulations of common diseases (i.e., diseases or conditions with an overall prevalence of over 200,000 in the United States), unless the use of the drug in the pediatric subpopulation meets the regulatory criteria for an orphan subset, or unless the disease in the pediatric subpopulation is considered a different disease from the disease in the adult population. This will help resolve an unintended loophole in the Pediatric Research Equity Act (PREA) orphan exemption process where a sponsor holding a pediatric-subpopulation designation can submit a marketing application for use of its drug in the non-orphan adult population of that disease, get a pediatric-subpopulation designation for the pediatric subset of the disease, and, due to this designation, be exempt from conducting the pediatric studies normally required under PREA when seeking approval of the adult indication.