An interesting paper from my colleagues at Precision in Medical Decision Making looks at how patients, physicians and nurses value different treatment attributes related to survival and treatment toxicity. The authors surveyed patients, oncologists, and oncology nurses using a combined conjoint analysis and discrete choice experiment survey approach. They found that:
Virtually all patients preferred health state sequences with contiguous periods of PFS [progression free survival] , compared with approximately 85% and 75% of nurses and oncologists, respectively. On average, longer OS [overall survival] was significantly (P < 0.01) preferred by the majority (75%) patients, but only 15% of nurses preferred longer OS, and OS did not significantly affect oncologists’ preferred health state…The strong preference for observed PFS suggests more research is warranted to better understand the reasons for PFS having positive value to patients. The results also suggest a range of endpoints in clinical trials may have importance to patients.
A paper Gyawali et al. (2019) in JAMA Internal Medicine calls into question the extensive use of surrogate outcomes in oncology clinical trials. However, it appears that patients do value PFS independently of its ability to predict long-term OS.
- MacEwan, Joanna P., Jason Doctor, Karen Mulligan, Suepattra G. May, Katharine Batt, Christopher Zacker, Darius Lakdawalla, and Dana Goldman. “The Value of Progression-Free Survival in Metastatic Breast Cancer: Results From a Survey of Patients and Providers.” MDM Policy & Practice, (January 2019). doi:10.1177/2381468319855386.