Sorafenib for advanced hepatocellular carcinoma (HCC) in the public health setting in Brazil: a cost-effectiveness analysis

Abstract

Introduction: Hepatocellular carcinoma (HCC) has a high incidence andmortality worldwide. Until 2007, there were no drugs that could increase the overall survival in advancedHCC patients. Sorafenib remains the standard-of-care in first-line setting since then. The survival benefit of sorafenib is accompanied by its high cost. All over the world, there has been an increasing discussion on treatment costs and the sustainability of health care systems. In Brazil, the government health expenditures for the public health system have not been sufficient to ensure adequate service for the population and sorafenib is not available in this setting. Therefore, this study aimed to assess the cost-effectiveness of sorafenib for advancedHCC in the public health system in Brazil. Method(s): A systematic review of the literature was performed in order to identify the best evidence of sorafenib against placebo and/or best supportive care in advanced HCC. The search strategy retrieved 443 articles in Pubmed and EMBASE platforms and 441 were excluded after thourough analysis. The remaining two studies were SHARP trial and ASIA-PACIFIC trial. As the Brazilian population is more similar to the one of SHARP and also because this study is larger, it was selected for the analysis. The data on efficacy and safety were extracted. Cost of sorafenib and costs related to usual medical care were taken from governmental sources. After all the data were gathered, the analysis was performed with the software Treeage Pro Suite 2017 using the Markov model for simulating the possible health states of HCC patients (Sorafenib, best supportive care or death). The primary outcome was the Incremental Cost-Effectiveness Ratio (ICER). As the Brazilian government does not define a cost-effectiveness threshold, we used the OMS-suggested 3 times the Gross Domestic Product (GDP), that is, US$29.464,23. The discount rate was set to 5% and the time horizon, 10 years. Afterwards, a sensitivity analysis was conducted to determine the most contributable factors to ICER value. Result(s): The estimated effectiveness of sorafenib yielded a median overall survival of 14,4 months, while the best supportive care (BSC) group was 9,24 months, leading to an incremental effectiveness of 5,16 months. The average cost per patient who received sorafenib was US$14.013,76, while the cost for BSC group was US$447,10. The ICER was US$31.793,21, which surpassed the threshold in 8%. In the sensitivity analysis, the most important factor that contributed to the ICER was the cost of sorafenib. Conclusion(s): Sorafenib was not considered cost-effective for advanced HCC patients in the Brazilian public health systemwhen compared to BSC. Nevertheless, the difference was small and the drug could be considered for incorporation in this setting after a price negotiation with the healthcare industry, once the latter has already offered a 26% discount in another study. Moreover, at this time, these advanced HCC patients have no treatment available for their disease.