Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada.
Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada.
Cancer Med. 2023 May;12(10):11451-11461. doi: 10.1002/cam4.5862. Epub 2023 Mar 31.
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). 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.
This was a population-based retrospective cohort study of patients who received second-line non-ipilimumab therapies between 2008 and 2012 versus ipilimumab treatment between 2012 and 2015 (after public reimbursement) for MM in Ontario. 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. Varying the discount rate and reducing the price of ipilimumab were done as sensitivity analyses.
In total, 329 MM were identified (Treated: 189; Controls: 140). Ipilimumab was associated with an incremental effectiveness of 0.59 LYG, incremental cost of $91,233, and ICER of $153,778/LYG. ICERs were not sensitive to discounting rate. 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. Reducing the price of ipilimumab by 100% resulted in an ICER of $111,728/QALY.
Despite its clinical benefit, ipilimumab as second-line monotherapy for MM patients is not cost-effective in the real world as projected by HTA under conventional willingness-to-pay thresholds.
先前的文献充分描述了关于转移性黑色素瘤（MM）临床获益的随机试验与真实世界证据之间的疗效-效果差距，这与健康技术评估机构（HTA）最初提出的担忧一致。由于这些差异可能会对成本效益产生重大影响，因此评估 MM 二线伊匹单抗与非伊匹单抗治疗的真实世界成本效益至关重要。
这是一项基于人群的回顾性队列研究，纳入了 2008 年至 2012 年间接受二线非伊匹单抗治疗的 MM 患者与 2012 年至 2015 年（公共报销后）接受伊匹单抗治疗的患者。使用 5 年时间范围，从加拿大元的公共支付者角度计算调整后的censored 和贴现（1.5%）成本以及疗效，以计算增量成本效益比（ICER）在获得的生命年（LYG）和质量调整生命年（QALY）中，使用bootstrapping 来捕获不确定性。作为敏感性分析，还对贴现率和降低伊匹单抗价格进行了研究。
共确定了 329 例 MM（治疗组：189 例；对照组：140 例）。伊匹单抗治疗与 0.59 LYG 的增量有效性、91233 美元的增量成本和 153778 美元/LYG 的增量成本效益比相关。ICER 对贴现率不敏感。使用效用权重调整生活质量后，ICER 为 225885 美元/QALY，确认了公共报销前 HTA 的原始估计。将伊匹单抗的价格降低 100%，ICER 为 111728 美元/QALY。
尽管具有临床获益，但从 HTA 在传统意愿支付阈值下的预测来看，作为 MM 二线单药治疗的伊匹单抗在真实世界中并不具有成本效益。
