Abstract
Cabozantinib has demonstrated a notable benefit in prolonging progression-free survival (PFS) compared to placebo in patients with advanced neuroendocrine tumors (NETs). This study provides the first cost-effectiveness assessment of cabozantinib from the perspectives of both the U.S. healthcare payer and the Chinese healthcare system. A Markov state-transition model was developed to evaluate the economic impact of cabozantinib, incorporating both U.S. and Chinese healthcare perspectives. The key outcomes included quality-adjusted life-years (QALYs), incremental cost-effectiveness ratio (ICER), incremental net health benefit (INHB), and incremental net monetary benefit (INMB). Given the high crossover rate in the control group, its impact on the results was carefully analyzed. In the extrapancreatic NET (EPNET) cohort, where 33.00% of patients crossed over, cabozantinib resulted in an ICER of $78,705.52 per QALY in China and $412,189.62 per QALY in the U.S., both exceeding the willingness-to-pay thresholds of $40,354.27 per QALY in China and $150,000.00 per QALY in the U.S. In the pancreatic NET (PNET) cohort, with a 41.00% crossover, cabozantinib demonstrated greater cost-effectiveness. In China, it achieved an ICER of $11,095.91 per QALY, with an INHB of 0.06 QALYs, an INMB of $2,497.95, and a probabilistic sensitivity analysis (PSA) probability of 52.39%. In the U.S., cabozantinib provided an incremental gain of 0.09 QALYs at an additional cost of $2,334.04, yielding an ICER of $24,571.17 per QALY, with an INHB of 0.08 QALYs, an INMB of $11,914.62, and a PSA probability of 52.48%. Cabozantinib is cost-effective for PNETs in both China and the U.S., but not in the EPNET cohort. Due to the high crossover rate, discrepancies may arise when comparing ICER values with other economic evaluation metrics, such as INHB, INMB, one-way sensitivity analysis, and PSA outcomes.