Role of reimbursement and Physicians' awareness in the survival of sorafenib-eligible advanced hepatocellular carcinoma patients

Abstract

In 2008, sorafenib became the first approved systemic therapeutic agent for advanced HCC. Although its pharmacological efficacy has been established, reimbursement for such a new, high-cost drug, as well as physicians' awareness and prescription practice, likewise contribute to its clinical effectiveness. We therefore conducted a retrospective study using 38 sorafenib-eligible, advanced HCC patients when sorafenib was approved but not yet reimbursed as a control and 216 patients during the reimbursed era. Study group showed longer survival at 8.2 months versus the control's 4.9 months (p = 0.0063 hazard ratio: 0.612 [0.431 ~ 0.868], p = 0.0059). Among the 42 (19.4%) patients who survived more than 2 years, 50% had tumor rupture, and all 32 patients with portal vein tumor thrombus and/or extrahepatic metastasis received sorafenib (p = 0.003). Furthermore, during their first 2 years of HCC management, sorafenib had been given in 29.1% of the treatment courses among survivors between 2 and 5 years while it was prescribed in 55.8% among the more than 5 years survivor group (p < 0.001). In conclusion, survival of sorafenib-eligible HCC patients significantly improved after reimbursement. Patients who underwent longer sorafenib treatment had a survival advantage, except for those with tumor rupture. Reimbursement and awareness of prescriptions for a newly introduced medication therefore improve clinical effectiveness.

Keywords: clinical effectiveness; hepatocellular carcinoma; pharmacological efficacy; reimbursement; sorafenib.

FIGURE 1

Patient enrollment flow diagram. BCLC, Barcelona clinic liver cancer; HCC, Hepatocellular carcinoma.

FIGURE 2

Kaplan–Meier analysis of overall survival between the control and study groups showing that 216 patients during the era of sorafenib reimbursement (study group) had a higher OS at 8.2 months (red dashed line) compared to the control (blue line) (p = 0.0063).

FIGURE 3

Swimmer plot presenting the different treatment modalities undergone by the 42 advanced HCC patients who survived for more than 2 years, including 15 who survived longer than 5 years. The patients were classified as BCLC stage C due to either having portal vein tumor thrombosis (PVT), extrahepatic spread (EHS), tumor rupture, or their combinations. Five out of the 10 patients with tumor rupture and all of the other 32 patients underwent sorafenib treatment.