The Cancer Centre, University Hospital Birmingham NHS Foundation Trust, Edgbaston, UK.
Expert Rev Pharmacoecon Outcomes Res. 2012 Aug;12(4):465-73. doi: 10.1586/erp.12.33.
Hepatocellular carcinoma (HCC) is a leading cause of cancer death globally, and its incidence is increasing in the West, including the UK, with the increasing burden of chronic liver disease. Until recently, systemic treatment options for advanced disease were limited. However, randomized clinical trials have demonstrated that the multikinase inhibitor sorafenib prolongs survival in appropriately selected patients, and this drug has become the standard of care for patients with advanced HCC. However, a single-technology appraisal by the NICE recommended that the UK National Health Service should not fund sorafenib on the grounds of cost-effectiveness. A number of other novel agents and combinations are currently in clinical trials, the results of which may further expand the treatment options and indications for systemic therapy in HCC. This review discusses the impact of restricting access to high-cost medications for patients with HCC in the UK, and describes potential strategies and future directions that may improve the cost-effectiveness of such drugs. It also describes the potential impact, pending national guidance, of variations in local funding decision-making on patient outcomes.
肝细胞癌（HCC）是全球癌症死亡的主要原因，其发病率在包括英国在内的西方国家正在上升，这与慢性肝病的负担增加有关。直到最近，晚期疾病的系统治疗选择还很有限。然而，随机临床试验表明，多激酶抑制剂索拉非尼可延长适当选择的患者的生存期，因此该药物已成为晚期 HCC 患者的标准治疗方法。然而，NICE 的一项单一技术评估建议，英国国民保健制度（NHS）不应出于成本效益的考虑而资助索拉非尼。目前有许多其他新型药物和联合用药正在临床试验中，其结果可能会进一步扩大 HCC 的系统治疗选择和适应证。本文讨论了限制英国 HCC 患者获得高成本药物的途径所产生的影响，并描述了可能改善此类药物成本效益的潜在策略和未来方向。它还描述了在等待国家指导的情况下，当地资金决策的变化对患者结局可能产生的潜在影响。
