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Oncologic orphan drugs approved in the EU – do clinical trial data correspond with real-world effectiveness?

BACKGROUND: Evaluation of evidence for efficacy of orphan medicinal products (OMPs) for rare malignancies may be hampered by the use of tumor measurements instead of clinical endpoints. This may cause efficacy data to not always match effectiveness in the real-world. We investigated whether an effic...

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Detalles Bibliográficos
Autores principales: Schuller, Yvonne, Biegstraaten, Marieke, Hollak, Carla E. M., Klümpen, Heinz-Josef, Gispen-de Wied, Christine C., Stoyanova-Beninska, Violeta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6263065/
https://www.ncbi.nlm.nih.gov/pubmed/30486835
http://dx.doi.org/10.1186/s13023-018-0900-9