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 an efficacy-effectiveness gap exists for oncologic OMPs and aimed to identify which factors contribute to it. Also, the magnitude of the clinical efficacy of oncologic OMPs was evaluated.

Methods

We included all oncologic OMPs authorized in the European Union from 2000 to 2017. Pivotal studies were evaluated by means of the European Society for Medical Oncology - Magnitude of Clinical Benefit Scale (ESMO-MCBS). To estimate real-world effectiveness, a literature search was performed to iden

SUBMITTER: Schuller Y 

PROVIDER: S-EPMC6263065 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature