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The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review

An orphan disease is defined in the EU as a disorder affecting less than 1 in 2 000 individuals. The concept of ultra-orphan has been proposed for diseases with a prevalence of less than 1:50 000. Drugs for ultra-orphan diseases are amongst the most expensive medicines on a cost-per-patient basis. T...

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Pubblicato in:Orphanet J Rare Dis
Autori principali: Schuller, Y., Hollak, C. E. M., Biegstraaten, M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2015
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4520069/
https://ncbi.nlm.nih.gov/pubmed/26223689
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0305-y
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