Noemi Muszbek

(2012). Modelling survival in hepatocellular carcinoma. Current Medical Research and Opinion: Vol. 28, No. 7, pp. 1141-1153. doi: 10.1185/03007995.2012.691422

A randomized phase III trial of sorafenib vs. placebo in hepatocellular carcinoma (HCC) demonstrated that sorafenib significantly prolonged overall survival (OS) compared to placebo. A Markov model was developed to evaluate the cost-effectiveness of sorafenib vs. best supportive care (BSC) in HCC from the perspective of the Canadian provincial Ministry of Health. The model followed survival and time to progression (TTP) in monthly cycles based on the extrapolation of patient level trial data. Health effects were expressed as life-years gained (LYG). Resource use included drugs, physician visits, laboratory tests, scans, and hospitalizations. Unit costs were gathered from public sources and were expressed in 2007 Canadian Dollars. Costs and effects were evaluated over a lifetime and discounted at 5%. Results were presented as mean +/- standard deviation. Deterministic and probabilistic sensitivity analyses were conducted. LYG was longer for sorafenib (1.52 +/- 0.16 vs. 1.03 +/- 0.09 LYG/patient for sorafenib and BSC, respectively). The lifetime total costs were $47,511 +/- 3 656 for sorafenib and $10,376 +/- 1 649 for BSC, resulting in an incremental cost-effectiveness ratio (ICER) of $75,821/LYG, and deterministic ICER of $75,759/LYG. The results were most sensitive to OS, TTP and BSC costs after progression. Sensitivity analyses results showed that the model was robust. The economic evaluation indicates that sorafenib is cost-effective as compared to BSC in HCC. Limitations include multiple data sources, use of expert opinion for resource use, and the lack of utility data.