title: The Orphan Drug Act and the development of stem cell-based products for rare diseases.
abstract: The Orphan Drug Act encourages the development of products for rare diseases and conditions. Many conditions that stand to benefit from stem cell-based products are rare diseases. We address the Orphan Drug Act in relation to the development of stem cell-based products.
authors.affiliation: Office of Orphan Products Development, Office of Special Medical Programs, Office of the Commissioner, United States Food and Drug Administration, Silver Spring, MD 20993, USA.
authors.forename: Scott N
authors.initials: SN
authors.lastname: Freeman
authors.forename: Kathryn A
authors.initials: KA
authors.lastname: Burke
authors.forename: Menfo A
authors.initials: MA
authors.lastname: Imoisili
authors.forename: Timothy R
authors.initials: TR
authors.lastname: Coté
mesh_terms: D004345:Drug Industry; D006078:Government Programs; D009965:Orphan Drug Production; D035583:Rare Diseases / Q000628:therapy*; D033581:Stem Cell Transplantation; D013234:Stem Cells / Q000166:cytology*; D014481:United States; D014486:United States Food and Drug Administration