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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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Detalhes bibliográficos
Publicado no:BMJ Open
Main Authors: Onakpoya, Igho J, Spencer, Elizabeth A, Thompson, Matthew J, Heneghan, Carl J
Formato: Artigo
Idioma:Inglês
Publicado em: BMJ Publishing Group 2015
Assuntos:
Acesso em linha: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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