Value frameworks for cancer care: Do they account for potential durable survival with immunotherapies?

Abstract

6601 Background: Modern immuno-oncologic agents may have the potential to provide durable survival for some patients. Simultaneously, there is growing concern regarding the cost of cancer care, and multiple frameworks have been developed to assess value. The points based framework created by the American Society of Clinical Oncology (ASCO) has a specific threshold, and more points are awarded if substantial durable survival is demonstrated. The objective of this study was to assess if modern immuno-oncologic agents reach defined efficacy thresholds in this framework. Methods: We reviewedall FDA approvals for immuno-oncologic agents since the first approval in 2011. We collected data regarding study endpoints, specifically the parameters defined in the updated ASCO value framework: improvement in proportion of patients alive with the test regimen, and survival rate with standard. We assessed whether bonus points would be rewarded as per the updated ASCO value framework for durable responses demonstrated on the tails of the Kaplan-Meier curves. These parameters were used as an input to check whether each drug reaches the required achievement defined in the framework. Results: 17 indications for four immunotherapy active ingredients were approved by FDA between 2011-2016: ipilimumab, pembrolizumab, nivolumab and atezolizumab. 59% of the approvals rely on survival endpoints such as overall, progression-free, or relapse-free survival. 41% are based on objective response rates. Only one drug (nivolumab indicated for melanoma first-line treatment) was found to fulfill the threshold defined for the survival rate of standard care (above 20%). Six drugs achieved the required level of improvement in proportion of patients alive in the test regimen compared to the standard (above 50%). As there was no overlap between these two groups of drugs, no drug was found to gain the durable survival bonus points defined by the ASCO framework. Conclusions: Durable survival and responses of modern immuno-oncology agents are currently not recognized to be significant by the ASCO value framework. This may be due to insufficient demonstration of efficacy of such agents, or may be due to an inappropriately calibrated value framework.