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, costeffectiveness 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 or

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Keywords Rare diseases, Orphan drugs, Evidence based medicine, Clinical effectiveness, Cost-effectiveness, Budget impact
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Journal Orphanet Journal of Rare Diseases
Kanters, T.A, de Sonneville, C, Redekop, W.K, & van Hakkaart-van Roijen, L. (2013). Systematic review of available evidence on 11 high-priced inpatient orphan drugs. Orphanet Journal of Rare Diseases, 2013(8), 124–130. doi:10.1186/1750-1172-8-124