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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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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Schuller, Yvonne, Biegstraaten, Marieke, Hollak, Carla E. M., Klümpen, Heinz-Josef, Gispen-de Wied, Christine C., Stoyanova-Beninska, Violeta
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6263065/
https://ncbi.nlm.nih.gov/pubmed/30486835
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0900-9
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