Pawlyn, C. orcid.org/0000-0002-7190-0028, Schjesvold, F.H. orcid.org/0000-0003-1096-0569, Cairns, D.A. orcid.org/0000-0002-2338-0179 et al. (13 more authors) (2024) Progression-free survival as a surrogate endpoint in myeloma clinical trials: an evolving paradigm. Blood Cancer Journal, 14. 134.
Abstract
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.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © The Author(s) 2024. This is an open access article under the terms of the Creative Commons Attribution License (CC-BY-NC-ND 4.0), which permits unrestricted use, distribution and reproduction in any medium, provided the original work is properly cited. |
Dates: |
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Institution: | The University of Leeds |
Academic Units: | The University of Leeds > Faculty of Medicine and Health (Leeds) > School of Medicine (Leeds) > Inst of Clinical Trials Research (LICTR) (Leeds) |
Funding Information: | Funder Grant number Cancer Research UK Supplier No: 138573 CTUQQR-Dec22/100002 |
Depositing User: | Symplectic Publications |
Date Deposited: | 14 Aug 2024 14:14 |
Last Modified: | 14 Aug 2024 14:14 |
Status: | Published |
Publisher: | Springer Nature |
Identification Number: | 10.1038/s41408-024-01109-4 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:216113 |