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

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

Abstract 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...

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Bibliographic Details
Main Authors: Yvonne Schuller, Marieke Biegstraaten, Carla E. M. Hollak, Heinz-Josef Klümpen, Christine C. Gispen-de Wied, Violeta Stoyanova-Beninska
Format: Article
Language:English
Published: BMC 2018-11-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Oncology
Orphan drugs
Efficacy
Effectiveness
Endpoints
Online Access:http://link.springer.com/article/10.1186/s13023-018-0900-9

Internet

http://link.springer.com/article/10.1186/s13023-018-0900-9

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