title: Characteristics of orphan drug applications that fail to achieve marketing approval in the USA.
abstract: The US Orphan Drug Act has fostered the development of drugs for patients with rare diseases by granting 'orphan designations', although several orphan drugs for which a marketing application has been submitted to the FDA have failed to obtain approval. This study identified the clinical trial design, the level of experience of the sponsor and the level of interaction with the FDA to be associated with non-approval. Sponsors, therefore, should engage in dialogue with the FDA and thoughtfully design pivotal clinical trials in accordance with FDA guidance documents.
authors.affiliation: Medicines Evaluations Board, PO Box 16229, 2500 BE Den Haag, The Netherlands.
authors.forename: Harald E
authors.initials: HE
authors.lastname: Heemstra
authors.forename: Hubert G M
authors.initials: HG
authors.lastname: Leufkens
authors.forename: R P Channing
authors.initials: RP
authors.lastname: Rodgers
authors.forename: Kui
authors.initials: K
authors.lastname: Xu
authors.forename: Bettie C G
authors.initials: BC
authors.lastname: Voordouw
authors.forename: M Miles
authors.initials: MM
authors.lastname: Braun
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic / Q000379:methods*; D017277:Drug Approval / Q000331:legislation & jurisprudence* / Q000379:methods*; D006801:Humans; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D014481:United States; D014486:United States Food and Drug Administration