title: The impact of the Orphan Drug Act on the development and advancement of neurological products for rare diseases: a descriptive review.
abstract: Many neurological diseases or conditions are rare disorders. The Orphan Drug Act (ODA) of 1983 was promulgated to promote the development of products for such conditions. In this Opinion piece, we discuss how the ODA has affected neurological diseases, note how current and future sponsors (any person(s) or entity (i.e., academic, corporate body, individual, manufacturer) that applies for an official regulatory action) of products for rare neurological diseases can take advantage of ODA incentives, identify areas of success and continuing needs, and review data that can help drive the future development of products for rare neurological conditions.
authors.affiliation: Office of Orphan Products Development, US Food and Drug Administration, Silver Spring, Maryland, USA.
authors.forename: K A
authors.initials: KA
authors.lastname: Burke
authors.forename: S N
authors.initials: SN
authors.lastname: Freeman
authors.forename: M A
authors.initials: MA
authors.lastname: Imoisili
authors.forename: T R
authors.initials: TR
authors.lastname: Coté
mesh_terms: D017277:Drug Approval / Q000331:legislation & jurisprudence*; D004345:Drug Industry / Q000331:legislation & jurisprudence; D006801:Humans; D009422:Nervous System Diseases / Q000188:drug therapy*; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D035583:Rare Diseases / Q000188:drug therapy*; D014481:United States; D014486:United States Food and Drug Administration / Q000331:legislation & jurisprudence*