The Value of Progression-Free Survival in the Perspective of Patients with Multiple Myeloma and Treating Physicians: Research Findings from the US, EU4, UK, Brazil, and Japan.

BACKGROUND

Treatment advancements in multiple myeloma (MM) have improved OS, but OS is a challenging endpoint for regulatory approvals as it both requires a prolonged follow-up period and is affected by subsequent therapies. While PFS is increasingly considered a surrogate endpoint in clinical trials, it is not considered to be patient relevant when assessing treatment benefit in some countries. Therefore, the main objective of this study was to assess the importance of PFS relative to other treatment outcomes for MM, from the patients' and physicians' perspectives.

METHODS

This cross-sectional observational study included adult patients with MM and physicians treating patients with MM across the EU4, Brazil, and Japan. After an initial qualitative phase for survey development, patients and physicians responded to a quantitative online survey using best-worst scaling and questions.

RESULTS

Overall, 237 patients and 267 physicians participated in the quantitative phase. Both groups valued OS and PFS when selecting new MM treatments, which was consistent across lines of therapy. This was especially relevant for physicians treating patients experiencing relapse and at-risk patient populations. Additionally, physicians supported PFS as a valuable endpoint for healthcare access and coverage decisions.

CONCLUSION

This study showed that PFS is an important endpoint for patients and physicians when making treatment decisions. These findings indicate that PFS should be incorporated into treatment-related and regulatory decisions.