Abstract / Summary
To systematically evaluate the clinical efficacy, cost-effectiveness and impact on medical insurance budgets of transarterial chemoembolisation (TACE) combined with apatinib versus TACE alone for the treatment of unresectable hepatocellular carcinoma (uHCC) in China, from an integrated health technology assessment (HTA) perspective. An HTA incorporating evidence from a randomised controlled trial, a Markov model with parametric extrapolation of survival data over a lifetime horizon and a static 5-year budget impact model from the Chinese healthcare payer perspective. Analysis was conducted from the perspective of the Chinese public healthcare payer. Clinical evidence was derived from a multicentre, randomised, open-label, prospective, phase III trial conducted at 15 hospitals in China. The published trial included 196 patients with uHCC. Participants were predominantly male (overall 85%), with a mean age of 52.7 years in the combination group and 52.9 years in the TACE-alone group. All had an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 and were of Chinese ethnicity. The present analysis used the de-identified, aggregated data from this published trial. The experimental regimen comprised TACE (intra-arterial injection of lipiodol and epirubicin) combined with oral apatinib (500 mg/day, initiated 4 days after the first TACE). The control regimen was TACE alone. Clinical outcomes included median progression-free survival (PFS) and overall survival (OS). The primary economic outcome was the incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained. The primary budgetary outcome was the 5-year cumulative incremental expenditure on the national basic medical insurance fund. Clinical evidence showed that the combination therapy significantly prolonged median PFS (6.1 vs 3.4 months; HR 0.45, 95% CI 0.33 to 0.63; p<0.0001) and OS (28.9 vs 24.0 months; HR 0.55, 95% CI 0.40 to 0.77; p=0.0005). The economic evaluation indicated that at the 2021 centralised procurement price, the ICER for the combination regimen was US$42 864/QALY, exceeding the willingness-to-pay threshold (US$37 653/QALY); however, when applying the 2025 national centralised procurement price, the ICER decreased to US$30 650/QALY, rendering it cost-effective. Budget impact analysis results showed that under the base-case penetration rate (reaching 50% by year 5), the cumulative incremental expenditure over 5 years would be approximately US$365 million, a finding highly sensitive to both the penetration rate and drug price. The TACE-apatinib regimen provides clear clinical benefit for uHCC in China, but its cost-effectiveness and budget feasibility are contingent on drug price. Successful national centralised procurement is the key policy lever to enable value-based access, as demonstrated by this integrated evidence framework. ChiCTR1800018621.