title: Determinants for successful marketing authorisation of orphan medicinal products in the EU.
abstract: In 2010, the European Regulation for Orphan Medicinal Products (OMPs) was in force for ten years. In this study we assessed possible determinants of applications for OMPs in the EU since 2000 that are associated with a successful marketing authorisation. Our analysis shows that clinical trial characteristics such as demonstrating convincing evidence of a beneficial effect on the primary endpoint, the selection of a clinically relevant endpoint, providing RCT data as pivotal study evidence and the submission of sound dose finding data are critical success factors. In addition, high medical need seems to counterweigh uncertainties about the scientific evidence in the benefit-risk assessment of OMPs.
authors.affiliation: Utrecht Institute for Pharmaceutical Sciences, Division of Pharmacoepidemiology & Clinical Pharmacology, Utrecht University, The Netherlands.
authors.forename: Michelle
authors.initials: M
authors.lastname: Putzeist
authors.forename: Harald E
authors.initials: HE
authors.lastname: Heemstra
authors.forename: Jordi Llinares
authors.initials: JL
authors.lastname: Garcia
authors.forename: Aukje K
authors.initials: AK
authors.lastname: Mantel-Teeuwisse
authors.forename: Christine C
authors.initials: CC
authors.lastname: Gispen-De Wied
authors.forename: Arno W
authors.initials: AW
authors.lastname: Hoes
authors.forename: Hubert G M
authors.initials: HG
authors.lastname: Leufkens
mesh_terms: D017277:Drug Approval; D004345:Drug Industry; D005060:Europe; D006801:Humans; D040541:Marketing; D004364:Pharmaceutical Preparations; D018570:Risk Assessment
chemical_list: D004364:Pharmaceutical Preparations