Reducing the price of ipilimumab by 100% resulted in an ICER of $111,728/QALY. Adjusting for quality of life using utility weights resulted in an ICER of $225,885/QALY, confirming the original HTA estimate prior to public reimbursement. ICERs were not sensitive to discounting rate. Ipilimumab was associated with an incremental effectiveness of 0.59 LYG, incremental cost of $91,233, and ICER of $153,778/LYG. In total, 329 MM were identified (Treated: 189 Controls: 140). Varying the discount rate and reducing the price of ipilimumab were done as sensitivity analyses. Using a 5-year time horizon, censor-adjusted and discounted (1.5%) costs (from the public payer's perspective in Canadian dollars) and effectiveness were used to calculate incremental cost-effectiveness ratios (ICERs) in life-years gained (LYGs) and quality-adjusted life years (QALYs), with bootstrapping to capture uncertainty. This was a population-based retrospective cohort study of patients who received second-line non-ipilimumab therapies between 20 versus ipilimumab treatment between 20 (after public reimbursement) for MM in Ontario. As these differences can significantly impact cost-effectiveness, it is critical to assess the real-world cost-effectiveness of second-line ipilimumab versus non-ipilimumab treatments for MM. The efficacy-effectiveness gap between randomized trial and real-world evidence regarding the clinical benefit of ipilimumab for metastatic melanoma (MM) has been well characterized by previous literature, consistent with initial concerns raised by health technology assessment agencies (HTAs).
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