Frontiers in pharmacology
Conditional approval of oncology medicines in Europe (2017-2022): evidence maturity, regulatory outcomes, clinical benefit and downstream access
By Urroz-Elizalde M, Moraga-Alapont P, Jiménez-González M and 2 others.
Abstract
Background
Conditional marketing authorisation (CMA) is a European regulatory pathway that enables earlier access to medicines for serious conditions while allowing additional evidence generation after approval. Important questions remain regarding the maturity of supporting evidence, subsequent regulatory outcomes, clinical benefit, and patient access. We characterised the evidentiary basis, regulatory outcomes, clinical benefit, clinical practice guideline incorporation, and downstream access of oncology medicines granted CMA between 2017 and 2022.
Methods
We analysed all antineoplastic medicines granted CMA by the European Commission between 1 January 2017 and 31 December 2022. European Medicines Agency documentation was reviewed to assess regulatory characteristics, post-authorisation obligations, pivotal studies, and outcomes through 31 March 2025. Clinical benefit was evaluated using ESMO-MCBS, while reimbursement was assessed in Spain as an illustrative case of downstream access as of 15 December 2025. Kaplan-Meier analysis estimated time to regulatory resolution and reimbursement.