AI Article Synopsis

  • Measuring overall survival (OS) is the best way to see how well new treatments for multiple myeloma work in big studies, but it's getting harder to use OS since treatments are improving and they want to approve new medicines faster.
  • Other measures like progression-free survival (PFS) and response to treatment are being used to help decide if a treatment is good enough for approval, and the FDA even thinks minimal residual disease (MRD) can be used for quick approval.
  • This review talks about how PFS can sometimes be tricky to understand for different groups of patients, and it looks at how to design studies that take these differences into account so doctors can find the right treatments for the right people.

Article 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.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11319634PMC
http://dx.doi.org/10.1038/s41408-024-01109-4DOI Listing

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