Cost-effectiveness of Pembrolizumab as a Second-Line Therapy for Hepatocellular Carcinoma

Abstract

IMPORTANCE Immune checkpoint inhibitors have been approved for use as a second-line therapy for hepatocellular carcinoma (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. However, considering the high cost of pembrolizumab, there is a need to assess its value by considering both the clinical efficacy and cost.OBJECTIVE To evaluate the cost-effectiveness of pembrolizumab vs placebo as second-line therapy in patients with HCC from the US payer perspective. DESIGN, SETTING, AND PARTICIPANTSA Markov model was developed to compare the lifetime cost and efficacy of pembrolizumab as a second-line treatment of HCC with those of placebo using outcome data from the KEYNOTE-240 randomized placebo-controlled trial, which included 413 patients with advanced HCC previously treated with sorafenib and randomized patients to receive pembrolizumab plus best supportive care or placebo plus best supportive care in a 2:1 ratio. MAIN OUTCOMES AND MEASURESLife-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. One-way and probabilistic sensitivity analyses were performed to account for the parameter of uncertainty. A cost-threshold analysis was also performed. The study was conducted from January 31 to July 29, 2020.

Sintilimab Plus Bevacizumab Biosimilar Versus Sorafenib as First-Line Treatment for Unresectable Hepatocellular Carcinoma: A Cost-Effectiveness Analysis

“…A China-based study showed that atezolizumab plus bevacizumab was unlikely to be cost-effective compared with sorafenib as a frontline treatment for patients with unresectable HCC, with an ICER was $64,613/QALY ( Hou and Wu 2020 ). Based on the clinical data of the KEYNOTE-240 trial, the study by Chiang et al (2021b) conducted a cost-effectiveness analysis of pembrolizumab as a second-line treatment for HCC in the United States. The results indicated that pembrolizumab was unlikely to be cost-effective.…”

Clinical Characteristics, Treatment Patterns, and Healthcare Costs and Utilization for Hepatocellular Carcinoma (HCC) Patients Treated at a Large Referral Center in Washington State 2007–2018

“…Indeed, recent studies have concluded that many newer therapies for advanced HCC are not cost-effective by accepted thresholds given the high price of these agents relative to the modest survival benefits. [12][13][14][15][16][17][18] Our study provides a baseline cost estimate for future real-world cost analyses as newer therapies are increasingly taken up in clinical practice. If newer therapies for advanced BCLC stage HCC have improved toxicity profiles and result in decreased inpatient and ED utilization and outpatient care, the higher costs of the treatments may be offset by savings in other areas.…”

Systemic Therapy for Hepatocellular Carcinoma: Current Updates and Outlook

“…However, pembrolizumab was shown not to be a cost-effective therapy for HCC. 157 Moreover, the clinical study (NCT04442581) comparing cabozantinib with pembrolizumab in the first-line setting is ongoing.…”