Bloem, L.T., Schelhaas, J., López-Anglada, L., Herberts, C., van Hennik, P.B. and Tenhunen, O. (2023), European Conditional Marketing Authorization in a Rapidly Evolving Treatment Landscape: A Comprehensive Study of Anticancer Medicinal Products in 2006–2020. Clin Pharmacol Ther, 114: 148-160. https://doi.org/10.1002/cpt.2906
European conditional marketing authorization in a rapidly evolving treatment landscape : a comprehensive study of anticancer medicinal products in 2006–2020
|Author:||Bloem, Lourens T.1; Schelhaas, Jasmin1,2; López-Anglada, Lucía3;|
1Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, The Netherlands
2Dutch Medicines Evaluation Board (CBG-MEB), Utrecht, The Netherlands
3Pharmacology and Clinical Assessment Division, Spanish Medicines Agency (AEMPS), Madrid, Spain
4Medical Research Center Oulu, University of Oulu and Oulu University Hospital, Oulu, Finland
5Finnish Medicines Agency (Fimea), Helsinki, Finland
|Online Access:||PDF Full Text (PDF, 1 MB)|
|Persistent link:|| http://urn.fi/urn:nbn:fi-fe20230907121610
John Wiley & Sons,
|Publish Date:|| 2023-09-07
Since 2006, the European conditional marketing authorization (CMA) aims to facilitate timely patient access to medicinal products for which there is an unmet medical need by accepting less comprehensive data than normally required. The granting of CMA requires a positive benefit–risk balance, unmet medical needs to be fulfilled, likely submission of comprehensive data postauthorization, and the benefit of immediate availability to outweigh the risks of data noncomprehensiveness. Since its first use, more than half of all CMAs represent (hemato-)oncology indications. Therefore, we aimed to investigate the conditions in which CMA has been applied for anticancer medicinal products and whether they have changed over time. We retrospectively assessed the European public assessment reports of the 30 anticancer medicinal products granted CMA in 2006–2020 (51% of all 59 CMAs). Comparison of 2006–2013 to 2014–2020 highlighted increased proportions of proactively requested CMAs (+40%), medicinal products that addressed unmet medical needs by providing a major therapeutic advantage over authorized treatments (+38%), and orphan designated indications (+32%). In contrast, it showed decreased proportions of medicinal products for which a scientific advisory group was consulted (−55%) and phase III randomized controlled trial data were available (−38%). This suggests that applicants and the European Medicines Agency have learned how to use the CMA as a regulatory tool, among others, through better planning and proactive interaction. However, the increasing number of granted CMAs complicates the establishment of unmet medical need and the benefit–risk balance, especially in crowded indications and when only phase II uncontrolled trials are available.
Clinical pharmacology & therapeutics
|Pages:||148 - 160|
|Type of Publication:||
A1 Journal article – refereed
|Field of Science:||
No funding was received for this work.
© 2023 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.