1. Clarification of orphan designation of drugs and biologics for pediatric subpopulations of common diseases Publication: Silver Spring, MD : Office of Orphan Products Development, Food and Drug Administration, July 2018 Subject(s): Drug ApprovalOrphan Drug ProductionPediatricsAdolescentBiological ProductsChildClinical Trials as TopicColitis, Ulcerative -- drug therapyHIV Infections -- drug therapyInfantPrevalenceHumansUnited StatesUnited States. Department of Health and Human Services.United States. Food and Drug Administration.
2. Orphan drugs: FDA could improve designation review consistency; rare disease drug development challenges continue : report to Congressional requesters Publication: Washington, DC : United States Government Accountability Office, November 2018 Subject(s): Drug ApprovalOrphan Drug ProductionUnited StatesUnited States. Food and Drug Administration.
