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...

Descripción completa

Detalles Bibliográficos
Autores principales: Schuller, Y., Hollak, C. E. M., Gispen-de Wied, C. C., Stoyanova-Beninska, V., Biegstraaten, M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2017
Materias:
Original Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5577060/
https://www.ncbi.nlm.nih.gov/pubmed/28752290
http://dx.doi.org/10.1007/s40265-017-0788-z

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5577060/
https://www.ncbi.nlm.nih.gov/pubmed/28752290
http://dx.doi.org/10.1007/s40265-017-0788-z

Ejemplares similares