RW2 Real-World Evidence of Option Value in Metastatic Melanoma

Abstract

There is little real-world evidence of realized real option value (ROV) - the value a drug provides by extending survival to receive benefits from future innovations. This analysis aims to measure ROV with real-world clinical outcomes data, using ipilimumab in metastatic melanoma (mM). We conducted a retrospective analysis using the nationwide Flatiron Health electronic health record-derived de-identified database. Patients with mM initiating treatment before 4/19/15 (1st line (1L) pembrolizumab data available) were included in the study. The next innovation date was defined as 9/4/2014 (the FDA approval date for 2nd line (2L) pembrolizumab). We estimated proportions of patients surviving to the next innovation date and receiving a cancer immunotherapy (CIT). Inverse probability of treatment weighted Kaplan-Meier estimates for real-world overall survival (OS) were estimated by treatment cohorts (Ipilimumab followed by CIT or no CIT) and line of therapy. Observed ROV was estimated using truncated mean OS and proportions in each treatment cohort. Among 721 mM patients, 50% of 1L ipilimumab users and 18% of 1L chemotherapy users survived to the next innovation date. Among survivors to the next innovation date, 45% (1L Ipilimumab), 26% (1L chemotherapy), 52% (2L Ipilimumab), and 50% (2L Chemotherapy) received CITs. Mean OS for ipilimumab followed by a CIT was 38.3 months (1L) and 38.9 months (2L), while those who didn’t receive a CIT was 21.7 months (1L) and 13.9 months (2L). Estimated ROV for ipilimumab was an additional 3.7 months survival (32.2% of conventional value) in 1L, and 5.1 months (60% of conventional value) in 2L ipilimumab, and 4.0 months across lines of therapy. This study provides real-world evidence of clinically meaningful ROV and may support consideration of ROV when assessing the value of life-extending drugs in areas with likely future innovation.