Cost-Effectiveness of Sorafenib Monotherapy and Selected Combination Therapy with Sorafenib in Patients with Advanced Hepatocellular Carcinoma.

Objectives: To evaluate the cost-effectiveness of sorafenib treatment in combination with other therapies versus sorafenib monotherapy among patients with advanced hepatocellular carcinoma (HCC) who are enrolled in Taiwan's National Health Insurance.

Methods: A Markov model was constructed to simulate treatment outcomes and direct medical costs of sorafenib combination therapy and monotherapy from the perspective of the healthcare payer in Taiwan. Both life-years (LYs) and quality-adjusted life-years (QALYs) were used to measure treatment outcomes, and all costs were expressed in 2014 New Taiwan dollars (NT$). Model parameters were acquired primarily using data from population-based administrative databases: the Cancer Registry, National Health Insurance Research Database, and the Death Registry. Willingness-to-pay (WTP) threshold was set at three times the per capita gross domestic product at NT$2,133,930. Deterministic and probabilistic sensitivity analyses were conducted.

Results: For advanced HCC patients, sorafenib combined with other treatments might not be a cost-effective option when compared with sorafenib therapy alone. In the base-case analysis, combination treatment with sorafenib was estimated to increase costs by NT$434,788 compared with monotherapy, with a gain of 0.1595 QALYs. The resulting incremental cost-effectiveness ratio (ICER) was NT$2,725,943 per QALY gained. Results were sensitive to health utility values and monthly costs accrued in the progression-free survival state of the combination therapy group.

Conclusions: Our evidence from Taiwan demonstrated that while sorafenib in combination with other therapeutic approaches might improve treatment outcome when compared with sorafenib monotherapy, its ICER exceeded the WTP threshold and was considered not cost-effective.