Under current pricing, pembrolizumab may not be a cost-effective second-line therapy for hepatocellular carcinoma (HCC), according to an analysis published in JAMA Network Open (2021;[1]:e2033761. doi:10.1001/jamanetworkopen.2020.33761).
“Immune checkpoint inhibitors have been approved for use as a second-line therapy for [HCC] in patients who previously received sorafenib. Pembrolizumab has shown substantial antitumor activity and a favorable toxicity profile as a second-line treatment of HCC,” wrote Chi-Leung Chiang, Department of Clinical Oncology, University of Hong Kong (China), and colleagues.
“However, considering the high cost of pembrolizumab, there is a need to assess its value by considering both the clinical efficacy and cost,” they continued.
This lead Dr Chiang and colleagues to conduct this economic evaluation to assess the cost-effectiveness of pembrolizumab from the US payer perspective, using data from the KEYNOTE-240 trial.
The KEYNOTE-240 trial randomized 413 patients with advanced HCC in a 2:1 ratio to pembrolizumab plus best supportive care or placebo plus supportive care.
A Markov model was developed to compare the lifetime cost and efficacy of pembrolizumab compared with placebo. Life-years, quality-adjusted life-years (QALYs), lifetime costs, and incremental cost-effectiveness ratio (ICER) were estimated at a willingness-to-pay threshold of $150,000 per QALY.
Compared with placebo, pembrolizumab increased overall cost by $47,057, and improved QALYs by 0.138, resulting in an ICER of $340,409 per QALY. The ICER of pembrolizumab was larger than $150,000 per QALY in most of the sensitivity and subgroup analyses.
In order to be considered cost-effective, Dr Chiang and colleagues determined that the price of pembrolizumab would need to be reduced by 57.7% to $2925 per cycle
“The findings of this cost-effectiveness analysis suggest that, at its current price, pembrolizumab is not a cost-effective second-line therapy for HCC in the US, with a willingness-to-pay threshold of $150 000 per QALY,” Dr Chiang and colleagues concluded.—Marta Rybczynski
