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Lookup NU author(s): Professor Graham Jackson
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
© The Author(s) 2024.Measurement of overall survival (OS) remains the gold standard for interpreting the impact of new therapies for multiple myeloma in phase 3 trials. However, as outcomes have improved, it is increasingly challenging to use OS as the primary endpoint if timely approval of novel agents is to be ensured to enable maximum benefit for patients. Surrogate endpoints of OS, such as progression-free survival (PFS) and response to treatment, have contributed to approval decisions by the Food and Drug Administration (FDA) and European Medicines Agency as endpoints demonstrating clinical benefit, and the FDA has recently supported the use of minimal residual disease (MRD) as an accelerated approval endpoint in multiple myeloma. This review aims to address situations in which the use of PFS as a surrogate endpoint warrants careful interpretation especially for specific subgroups of patients and considers ways to ensure that studies can be designed to account for possible discordance between PFS and OS. The utility of subgroup analyses, including the potential for those not pre-specified, to identify target populations for new agents is also discussed.
Author(s): Pawlyn C, Schjesvold FH, Cairns DA, Wei LJ, Davies F, Nadeem O, Abdulhaq H, Mateos M-V, Laubach J, Weisel K, Ludwig H, Rajkumar SV, Sonneveld P, Jackson G, Morgan G, Richardson PG
Publication type: Review
Publication status: Published
Journal: Blood Cancer Journal
Year: 2024
Volume: 14
Issue: 1
Online publication date: 12/08/2024
Acceptance date: 23/07/2024
ISSN (electronic): 2044-5385
Publisher: Springer Nature
URL: https://doi.org/10.1038/s41408-024-01109-4
DOI: 10.1038/s41408-024-01109-4
PubMed id: 39134544
Data Access Statement: All relevant data related to the study are already published, included in the article, or uploaded as supplementary information.
