Systematic review of available evidence on 11 high-priced inpatient orphan drugs

• Main Authors: Kanters, T.A, De Sonneville-Koedoot, C, Redekop, W.K, Hakkaart, L
• Other Authors: SAW SWEE HOCK SCHOOL OF PUBLIC HEALTH
• Format: Article
• Published: 2020
• Subjects: agalsidase alfa; agalsidase beta; alemtuzumab; canakinumab; clofarabine; eculizumab; fludarabine; galsulfase; iduronate 2 sulfatase; laronidase; ofatumumab; orphan drug; recombinant glucan 1,4 alpha glucosidase; trabectedin; acute lymphoblastic leukemia; article; case study; chronic lymphatic leukemia; CINCA syndrome; clinical effectiveness; cost effectiveness analysis; cost utility analysis; drug approval; drug cost; evidence based medicine; Fabry disease; hospital patient; human; mucopolysaccharidosis; observational study; paroxysmal nocturnal hemoglobinuria; quality adjusted life year; quasi experimental study; soft tissue sarcoma; systematic review; cost benefit analysis; drug manufacture; economics; Netherlands; randomized controlled trial (topic); Rare Diseases; standards; Cost-Benefit Analysis; Humans; Inpatients; Orphan Drug Production; Randomized Controlled Trials as Topic
• Online Access: https://scholarbank.nus.edu.sg/handle/10635/181805
• Institution: National University of Singapore

10.1186/1750-1172-8-124