Department of Medicine D and the Liver Institute, Rabin Medical Center, Petach-Tikva, Israel.
The Sackler Faculty of Medicine, Tel-Aviv University, Ramat Aviv, Israel.
Coller School of Management, Tel Aviv University, Tel Aviv, Israel.
Institute of Oncology, Davidoff Cancer Center, Rabin Medical Center, Petach-Tikva, Israel.
Department of Health Policy and Management, Gillings School of Global Public Health, University of North Carolina at Chapel Hill, United States of America.
BACKGROUND AND AIMS:
Hepatocellular carcinoma (HCC) is one of the leading causes of cancer related deaths. Patients with advanced HCC are treated with sorafenib. A recent randomized controlled trial demonstrated a survival benefit for regorafenibtreatment in patients with advanced HCC who had progressed on sorafenib. We aimed to evaluate the cost-effectiveness of this approach.
To evaluate the cost effectiveness of regorafenib, we used a Markov model that incorporates health outcomes, measured by life-years and quality-adjusted life-years (QALYs). Drug costs were based on 2017 discounted prices. Model robustness was validated by probabilistic sensitivity analyses using Monte Carlo simulations.
The use of regorafenib results in a gain of 19.76 weeks of life (0.38 Life Years) as compared to placebo. When adjusted for quality of life, using regorafenib produced a gain of 0.25 quality adjusted life years (QALYs). The incremental cost-effectiveness ratio for regorafenib compared with best supportive care was between $201,797 and $268,506 per QALY.
The modest incremental benefit at a relatively high incremental cost of regorafenib treatment suggests that it is not cost-effective at commonly accepted willingness to pay thresholds.