Cost-effectiveness of regorafenib for pretreated metastatic colorectal cancer patients in the United States.

Abstract

578 Background: There is an unmet need for treatments that provide a survival benefit for patients with heavily pretreated metastatic colorectal cancer (mCRC). In a randomized, double-blind, placebo-controlled trial, patients treated with regorafenib plus best supportive care (BSC) had significant improvement in overall survival (OS) versus patients in the placebo arm receiving BSC alone. The aim of this study was to estimate the cost-effectiveness of regorafenib for the treatment of patients with mCRC after failure or intolerance to irinotecan-, oxaliplatin-, and fluorouracil-based regimens, from the perspective of a U.S. health care payer. Methods: A cohort-partition model was developed to simulate treatment costs and survival for patients treated with regorafenib or BSC alone. Survival was projected by fitting Weibull distributions to trial data. Mean duration of treatment (3.0 months regorafenib, 2.1 months BSC), utilities (0.71 on treatment, 0.59 post-treatment) and serious adverse event rates were also based on trial data. Monthly drug monitoring and physician fee costs were based on Medicare fee schedules. Routine mCRC care and adverse event unit costs were obtained from an analysis of administrative claims in two large managed care databases. Costs were estimated in 2012 USD. Results: Patients treated with regorafenib had prolonged survival versus BSC alone (0.75 versus 0.60 years) and also had higher lifetime costs ($97,700 versus $59,494). The improvement in quality-adjusted life years (0.47 versus 0.37) was small due to the heavily pretreated nature of the patient population. The increase in costs was attributable to increases in costs for drug acquisition, adverse events , and routine care from increased survival. Conclusions: Regorafenib increases life expectancy for patients with pretreated metastatic colorectal cancer. The cost of this benefit could be considered high; however, it could be justifiable given high unmet need for these patients. The increase in cost is primarily due to drug acquisition costs, similar to other recent treatments for metastatic cancers.

Evolution of regorafenib from bench to bedside in colorectal cancer: Is it an attractive option or merely a “me too” drug?

“…Their analysis showed that patients treated with regorafenib had improved OS in comparison with those on BSC alone (0.75 versus 0.60 years), but regorafenib treatment was also associated with higher lifetime costs than BSC alone ($97,700 versus $59,494). 36 Gold-stein et al also studied the cost-effectiveness of regorafenib in the third-line setting for mCRC patients, using a Markov model. 37 Treatment with regorafenib provided an additional 0.13 life-years at a cost of $40,000, resulting in an incremental cost-effectiveness ratio of $900,000 per quality-adjusted life-years.…”

Propensity Score Weighting Using Overlap Weights: A New Method Applied to Regorafenib Clinical Data and a Cost-Effectiveness Analysis

“…The model was also previously described in 2 posters. 20,21 The model structure is shown in see Appendix Figure 1 in Supplemental Materials found at https://doi.org/10.1016/j.jval.2019.06.010.…”