Abstract
226 Background: Everolimus and sunitinib were recently approved to treat patients with advanced, progressive pancreatic neuroendocrine tumors (pNETs). This analysis examines the projected cost-effectiveness of everolimus versus sunitinib in this setting from a US payer perspective. Methods: A lifetime Markov model was developed to simulate a cohort of advanced, progressive pNET patients and to estimate the cost per incremental life-years gained (LYG) and quality-adjusted life years (QALYs) gained when treating with everolimus as compared to sunitinib. Absent head-to-head trials, efficacy data were based on a weight-adjusted indirect comparison of the two agents using the respective phase 3 trial data. Disease or health states considered in the model included: stable disease without adverse events, stable disease with adverse events, disease progression, and death. Costs of anti-tumor therapies, symptomatic care drugs, and post-progression therapy were based on wholesale acquisition cost. Other costs including physician visits, tests, infusions, hospitalizations, adverse event costs, and end-of-life care costs were obtained from literature and/or standard sources such as the Healthcare Cost and Utilization Project and Medicare reimbursement rates. Utility inputs were based on a UK time trade-off study. Sensitivity analyses were conducted to test the model’s robustness. Results: In the base case analysis, the estimated gain of everolimus over sunitinib was 0.448 LYs (0.304 QALYs) at an incremental cost of $12,673, resulting in an incremental cost-effectiveness ratio (ICER) of $28,281/LYG ($41,702/QALY gained), which fell within the cost per QALY range for many other widely used oncology drugs. The analysis was sensitive to the uncertainty of the sunitinib trial results; however, a probabilistic sensitivity analysis showed the results were consistent across simulations. Conclusions: While the analysis is limited by its reliance on an indirect comparison of two phase 3 studies rather than a single head-to-head trial, everolimus is expected to be cost-effective relative to sunitinib in advanced pNET.
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