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Effectiveness, safety and costs of orphan drugs: an evidence-based review

INTRODUCTION: Several orphan drugs have been approved by the European Medicines Agency (EMA) over the past two decades. However, the drugs are expensive, and in some instances, the evidence for effectiveness is not convincing at the time of regulatory approval. Our objective was to evaluate the clin...

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Dades bibliogràfiques
Publicat a:BMJ Open
Autors principals: Onakpoya, Igho J, Spencer, Elizabeth A, Thompson, Matthew J, Heneghan, Carl J
Format: Artigo
Idioma:Inglês
Publicat: BMJ Publishing Group 2015
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4480037/
https://ncbi.nlm.nih.gov/pubmed/26109112
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/bmjopen-2014-007199
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