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federal_register: 2018-16027

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document_number title type abstract publication_date pub_year pub_month html_url pdf_url agency_names agency_ids excerpts regulation_id_numbers
2018-16027 Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases; Guidance for Industry; Availability Notice The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases." FDA does not expect to grant any additional orphan-drug designation to drugs for pediatric subpopulations of common diseases (i.e., diseases or conditions with an overall prevalence of 200,000 or greater). 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. 2018-07-27 2018 7 https://www.federalregister.gov/documents/2018/07/27/2018-16027/clarification-of-orphan-designation-of-drugs-and-biologics-for-pediatric-subpopulations-of-common https://www.govinfo.gov/content/pkg/FR-2018-07-27/pdf/2018-16027.pdf Health and Human Services Department; Food and Drug Administration 221,199 The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases." FDA does not expect...  

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