title: Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012.
abstract: Orphan designated medicinal products benefit from regulatory and economic incentives for orphan drug development. Approximately 40% of orphan designations target rare neoplastic disorders, referring to rare cancers. In order to provide more insights in drugs for rare neoplastic disorders that are under development and to better understand the role of orphan designation in the development of oncology drugs, this study investigates the characteristics of the product, the indication and the applicants as well as the stage of development of products with an orphan designation for rare neoplastic disorders and compares them with products with an orphan designation for other rare indications. Therefore, orphan designation application files and annual reports submitted by the applicant were reviewed at the premises of the European Medicines Agency.
  
  
  
  At the time of application, 41.6% of products with orphan designation for rare neoplastic disorders were in pre-clinical phase; this was 65.1% for other rare conditions (p < 0.05). Thirty percent of orphan designations for rare neoplastic disorders had reached phase 1; compared to 19.3% of orphan designations targeting other rare conditions (p < 0.05). The same trend was observed for the stage of development at the time of the latest annual report. Significant benefit was more often considered for orphan designations for rare neoplastic disorders compared to orphan designations for other rare conditions.
  
  
  
  Orphan designations for rare neoplastic disorders involve products that are in a more advanced stages of development compared to orphan designations for other (non-oncology) rare conditions.
authors.affiliation: Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000, Leuven, Belgium. Kim.Pauwels@kuleuven.be.
authors.forename: Kim
authors.initials: K
authors.lastname: Pauwels
authors.affiliation: Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000, Leuven, Belgium.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Huys
authors.affiliation: Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000, Leuven, Belgium.
authors.forename: Minne
authors.initials: M
authors.lastname: Casteels
authors.affiliation: European Medicines Agency, London, UK.
authors.forename: Kristina
authors.initials: K
authors.lastname: Larsson
authors.affiliation: European Medicines Agency, London, UK.
authors.forename: Caroline
authors.initials: C
authors.lastname: Voltz
authors.affiliation: Finnish Medicines Agency, Helsinki, Finland.|Savonlinna Central Hospital, Savonlinna, Finland.
authors.forename: Karri
authors.initials: K
authors.lastname: Penttila
authors.affiliation: Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000, Leuven, Belgium.
authors.forename: Thomas
authors.initials: T
authors.lastname: Morel
authors.affiliation: Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000, Leuven, Belgium.
authors.forename: Steven
authors.initials: S
authors.lastname: Simoens
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D017277:Drug Approval; D004345:Drug Industry; D005060:Europe; D006801:Humans; D009369:Neoplasms / Q000188:drug therapy*; D009965:Orphan Drug Production; D035583:Rare Diseases / Q000188:drug therapy*; D012189:Retrospective Studies
chemical_list: D000970:Antineoplastic Agents
keywords: Cancer; EMA; Neoplastic disorders; Orphan designation; Orphan medicinal product