Innovation and Role of Alternative Markers of Efficacy - Episode 6

Segment 6 - Patient-Reported Outcomes and Other Important Measures

Beyond progression-free survival and overall survival, other measures that are often analyzed in clinical trials are objective response rates and disease-free survival. Richard W. Joseph, MD, explains why these measures are useful when looking at efficacy of a therapeutic agent.

Disease-free survival is used moreover in the adjuvant treatment setting. Joseph explains that he likes using progression-free survival, but is cautious about basing response assessment on radiographic findings with immunotherapies, as some of these agents can cause initial tumor swelling, and some patients will then get tumor shrinkage later.

Joseph describes a 3-metric approach to assessing response:

  • 70% is the clinician metric: how does the patient appear; eg, better or worse than last visit
  • 25% is the imaging metric: how do the scans look
  • 5% is the laboratory metric: how do the test results look; eg, liver enzymes

Should patient-reported outcomes be part of the assessment metric? Joseph describes the COMPARZ trial, a double-blind study of sunitinib and pazopanib that collected quality-of-life metrics. It examined both patient and physician reports, and found that pazopanib was better tolerated.

Ira M. Klein, MD, MBA, FACP, describes ways of integrating patient surveys to collect patient-reported outcomes into the delivery system.

Watch our related Peer Exchange, Oncology Stakeholder Summit 2014: Evidence-Based Decisions to Improve Quality and Regulate Costs