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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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Detalles Bibliográficos
Autores principales: Onakpoya, Igho J, Spencer, Elizabeth A, Thompson, Matthew J, Heneghan, Carl J
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4480037/
https://www.ncbi.nlm.nih.gov/pubmed/26109112
http://dx.doi.org/10.1136/bmjopen-2014-007199