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Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases

INTRODUCTION: Authorization of orphan medicinal products (OMPs) is often based on studies with several methodological shortcomings. Hence, data are difficult to interpret and efficacy does not always correspond to real-world effectiveness. We investigated to what extent an efficacy-effectiveness gap...

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Publicat a:Drugs
Autors principals: Schuller, Y., Hollak, C. E. M., Gispen-de Wied, C. C., Stoyanova-Beninska, V., Biegstraaten, M.
Format: Artigo
Idioma:Inglês
Publicat: Springer International Publishing 2017
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5577060/
https://ncbi.nlm.nih.gov/pubmed/28752290
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40265-017-0788-z
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