Accelerating orphan drug development.
Interest in developing drugs for rare diseases has increased substantially in recent years. This article from the US Food and Drug Administration highlights the role of regulators in catalysing further progress in this field.
Office of the Commissioner, Office of Orphan Products Development, Silver Spring, Maryland 20993, USA. timothy.cote@fda.hhs.gov
Timothy R
TR
Coté
Kui
K
Xu
Anne R
AR
Pariser
D000818:Animals; D017277:Drug Approval / Q000379:methods; D055808:Drug Discovery / Q000379:methods / Q000639:trends*; D006801:Humans; D009965:Orphan Drug Production / Q000379:methods; D035583:Rare Diseases / Q000188:drug therapy; D014481:United States; D014486:United States Food and Drug Administration / Q000639:trends*