Liver cancer is a leading cause of cancer-related death worldwide. It is the fifth most common cancer in men (554,000 cases, 7.5% of the total) and the ninth in women (228,000 cases, 3.4%). In men, the regions of high incidence are Eastern and South-Eastern Asia (ASRs 31.9 and 22.2 respectively). In women, the rates are generally much lower. Liver cancer is the second most common cause of death from cancer worldwide, estimated to be responsible for nearly 746,000 deaths in 2012 (9.1% of the total) and prognosis is very poor.

Hepatocellular carcinoma (HCC) is the most common type of liver cancer. The incidence of HCC is expected to rise in the United States and across the world, given the increasing prevalence of hepatitis C and B infections, alcohol consumption, diabetes, toxin exposure, and obesity. Various interventions, such as resection, chemoembolisation, radiofrequency ablation, cryotherapy, or liver transplantation are often not feasible.

Sorafenib remains the only approved systemic treatment for advanced tumors and the need for safer and more effective treatment remains. Considering that no biomarkers are able to predict response to sorafenib and recent trials in first and second line have not provided treatment alternatives, the development of novel and innovative therapies is crucial.

This white paper provided a detailed analysis of HCC from the scientific and clinical trial management point of view.