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9 p, 899.9 KB Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut) / Guarga, Laura (Servei Català de la Salut) ; Badia, Xavier (Omakase Consulting S.L.) ; Obach, Mercè (Servei Català de la Salut) ; Fontanet, Manel (Universitat Autònoma de Barcelona. Departament de Farmacologia, de Terapèutica i de Toxicologia) ; Prat, Alba (Servei Català de la Salut) ; Vallano, Antonio (Servei Català de la Salut) ; Torrent, Josep (Servei Català de la Salut) ; Pontes García, Caridad (Universitat Autònoma de Barcelona. Departament de Farmacologia, de Terapèutica i de Toxicologia)
Background: Orphan medicines show some characteristics that hinder the evaluation of their clinical added value. The often low level of evidence available for orphan drugs, together with a high budget impact and an incremental cost-effectiveness ratio many times higher than drugs used for non-orphan diseases, represent challenges in their appraisal and effective access to clinical use. [...]
2019 - 10.1186/s13023-019-1121-6
Orphanet Journal of Rare Diseases, Vol. 14 Núm. 1 (27 2019) , p. 157  
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