OBJECTIVES: According to the Japanese clinical guidelines, sorafenib is used as first-line therapy for patients with advanced hepatocellular carcinoma (HCC) when not applicable to atezolizumab plus bevacizumab. Although donafenib showed superiority over sorafenib in improving overall survival, it is not approved in Japan and its cost-effectiveness is not necessarily clear. This study aimed to evaluate the cost-effectiveness of donafenib from the payer’s perspective in Japan.

METHODS: A partitioned survival analysis model was developed to predict costs and quality-adjusted life years (QALYs) in both groups. Survival data were derived from the phase II/III trial. Drug prices for donafenib were estimated based on foreign prices. Other cost parameters were estimated by using the JMDC calims database. Lifetime horizon and discount rate of 2% was applied. Utility weights were estimated based on previous studies. The incremental cost-effectiveness ratio (ICER) of donafemnib compared with sorafenib was estimated. Sensitivity analysis (SA) was performed to assess parameter uncertainty.

RESULTS: Compared with sorafenib, donafenib incurred an additional cost of JPY5,253,716 and conferred an additional 0.211 QALY. This resulted in the ICER of JPY24,864,390/QALY gained. SA showed that the parameter with the largest fluctuation range was utility of donafenib group in progression state, and the minimum value of ICER was JPY22,585,521/QALY and the maximum value was JPY27,654,741/QALY.

CONCLUSIONS: Applying the willingness to pay threshold of JPY15 million/QALY and foreign drug price, donafenib was not cost-effective compared to sorafenib. In the future, it would be necessary to incorporate a cost-effectiveness perspective in the pricing of cancer treatment in Japan.