title: Accelerating access to treatments for rare diseases.
abstract: Changes in regulatory policy and legislative incentives to promote the development of drugs for rare diseases - orphan drugs - have led to increases in the number of orphan drug designations, but the rate of such products reaching the market remains frustratingly flat. This article highlights areas in which novel approaches could facilitate regulatory approval and access to treatments for rare diseases.
authors.affiliation: Global Head of GSK Rare Diseases, GSK House, 980 Great West Road, Brentford, TW8 9GS, UK. marc.dunoyer@gsk.com
authors.forename: Marc
authors.initials: M
authors.lastname: Dunoyer
mesh_terms: D002986:Clinical Trials as Topic; D017277:Drug Approval; D004345:Drug Industry; D006801:Humans; D009042:Motivation; D009965:Orphan Drug Production; D057766:Policy; D035583:Rare Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology*; D014481:United States; D014486:United States Food and Drug Administration