title: Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010.
abstract: New drug and biologic product marketing applications submitted to FDA's Center for Drug Evaluation and Research (CDER) between 2006 and 2010 were analyzed to identify rare disease application characteristics associated with higher approval rates. The results show that approval rates were similar for rare and common disease applications. Larger company size, prior regulatory experience and priority review designation were associated with higher approval rates. The study findings show that rare disease product development is feasible, and increased interactions between product developers and FDA in early investigational phases can facilitate product development.
authors.affiliation: Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD 20993, USA. anne.pariser@fda.hhs.gov
authors.forename: Anne R
authors.initials: AR
authors.lastname: Pariser
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Slack
authors.forename: Larry J
authors.initials: LJ
authors.lastname: Bauer
authors.forename: Catherine A
authors.initials: CA
authors.lastname: Warner
authors.forename: LaRee A
authors.initials: LA
authors.lastname: Tracy
mesh_terms: D017277:Drug Approval / Q000706:statistics & numerical data*; D006801:Humans; D040541:Marketing; D035583:Rare Diseases / Q000188:drug therapy*; D014481:United States; D014486:United States Food and Drug Administration / Q000706:statistics & numerical data*