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

Autor: Christine C. Gispen-de Wied, Carla E. M. Hollak, Yvonne Schuller, Violeta Stoyanova-Beninska, Heinz-Josef Klümpen, Marieke Biegstraaten
Přispěvatelé: Endocrinology, AGEM - Inborn errors of metabolism, Graduate School, CCA - Cancer Treatment and Quality of Life, Oncology, AGEM - Digestive immunity, AGEM - Endocrinology, metabolism and nutrition, VU University medical center, CCA - Cancer Treatment and quality of life
Rok vydání: 2017
Předmět:
Zdroj: Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, Vol 13, Iss 1, Pp 1-11 (2018)
Orphanet journal of rare diseases, 13(1):214. BioMed Central
Orphanet Journal of Rare Diseases, 13(1):214. BioMed Central
Schuller, Y, Biegstraaten, M, Hollak, C E M, Klümpen, H-J, Gispen-de Wied, C C & Stoyanova-Beninska, V 2018, ' Oncologic orphan drugs approved in the EU-Do clinical trial data correspond with real-world effectiveness? ', Orphanet Journal of Rare Diseases, vol. 13, no. 1, 214 . https://doi.org/10.1186/s13023-018-0900-9
ISSN: 1750-1172
Popis: 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 identify post-marketing studies, of which data on overall survival (OS) were extracted. OS of the new OMP was compared with OS data of standard of care. An OS gain of ≥3 months compared to pre-marketing data was considered clinically relevant. Results Twenty OMPs were included, of which 5 were authorized based on OS as a primary endpoint. 10 OMPs had post-marketing data available, of which 40% did not show a clinically relevant OS gain in the real world. All OMPs that were studied with OS as primary endpoint in the pivotal study had a clinically relevant OS gain in the real world. Furthermore, all OMPs that had a high ESMO-MCBS score and post-marketing data available, resulted in a clinically relevant OS gain in the real world. Conclusions Although the sample size is small, our results indicate an efficacy-effectiveness gap for oncologic OMPs exists. Significant changes in PFS do not always lead to an increased OS. The use of PFS may be justified, but validation of surrogate endpoints is needed. Electronic supplementary material The online version of this article (10.1186/s13023-018-0900-9) contains supplementary material, which is available to authorized users.
Databáze: OpenAIRE
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