Real option value (ROV) is created when a drug enables a patient to live long enough to benefit from a future innovation, but few if any studies have quantified ROV observed in the real world. We aimed to estimate the realized ROV using real-world data (RWD) using ipilimumab in melanoma as a case study.
We developed a framework for calculating ROV using RWD that accounted for the health gain in the standard therapy arm as well as the actual uptake of future innovative drugs. We developed a Markov model to estimate the quality-adjusted life-years (QALYs) gained with ipilimumab compared to chemotherapy for patients with or without subsequent cancer immunotherapy (CIT). The realized ROV of ipilimumab and chemotherapy was the additional health gain due to the CIT. To inform the model parameters, we used the nationwide Flatiron Health electronic health record-derived de-identified database to estimate real-world progression-free survival (PFS) and overall survival (OS) curves for patients stratified by the first- and subsequent-lines of therapy. Patients diagnosed with metastatic or advanced unresectable melanoma and started treatment between 1/1/2011 and date of 1st CIT approval (9/4/2014) were included.
The incremental QALYs gained for ipilimumab vs. chemotherapy without subsequent CIT were 1.74. With subsequent CIT, the incremental QALYs increased by 0.92, 0.60, 0.33, 0.18, 0.10, and 0.02 when CIT became available 0, 3, 6, 9, 12, and 24 months after the initiation of first-line ipilimumab, respectively. The results were sensitive to the survival benefit of ipilimumab, the survival benefit of subsequent CIT, and the uptake of CIT.
This is the first study to demonstrate the realized ROV using RWD. The realized ROV was between 1%–54% of conventional value for patients diagnosed within 2 years before CIT availability. Further studies are needed to understand ROV in other diseases, particularly those with longer survival times.
© 2021 Published by Elsevier Inc.