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https://doi.org/10.1186/1750-1172-8-124
Title: | Systematic review of available evidence on 11 high-priced inpatient orphan drugs | Authors: | Kanters, T.A De Sonneville-Koedoot, C Redekop, W.K Hakkaart, L |
Keywords: | 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 Netherlands Orphan Drug Production Randomized Controlled Trials as Topic Rare Diseases |
Issue Date: | 2013 | Citation: | Kanters, T.A, De Sonneville-Koedoot, C, Redekop, W.K, Hakkaart, L (2013). Systematic review of available evidence on 11 high-priced inpatient orphan drugs. Orphanet Journal of Rare Diseases 8 (1) : 124. ScholarBank@NUS Repository. https://doi.org/10.1186/1750-1172-8-124 | Rights: | Attribution 4.0 International | Abstract: | Background: Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, cost-effectiveness and budget impact for orphan drugs. Methods. A systematic review was performed in PubMed, Embase, NHS EED and HTA databases for 11 inpatient orphan drugs listed on the Dutch policy rule on orphan drugs. For included studies, we determined the type of study and various study characteristics. Results: A total of 338 studies met all inclusion criteria. Almost all studies (96%) focused on clinical effectiveness of the drug. Of these studies, most studies were case studies (41%) or observational studies (39%). However, for all orphan diseases at least one experimental or quasi-experimental study was found, and a randomized clinical trial was available for 60% of the orphan drugs. Eight studies described the cost-effectiveness of an orphan drug; an equal number described an orphan drug's budget impact. Conclusions: Despite the often heard claim that RCTs are not feasible for orphan drugs, we found that an RCT was available in 60% of orphan drugs investigated. Cost-effectiveness and budget impact analyses for orphan drugs are seldom published. © 2013 Kanters et al.; licensee BioMed Central Ltd. | Source Title: | Orphanet Journal of Rare Diseases | URI: | https://scholarbank.nus.edu.sg/handle/10635/181805 | ISSN: | 17501172 | DOI: | 10.1186/1750-1172-8-124 | Rights: | Attribution 4.0 International |
Appears in Collections: | Staff Publications Elements |
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