Pritzker School of Medicine, Division of Biological Sciences, University of Chicago, Chicago, IL, USA.
Center for Health and the Social Sciences, University of Chicago, Chicago, IL, USA.
Ann Surg Oncol. 2021 Dec;28(13):9039-9047. doi: 10.1245/s10434-021-10288-4. Epub 2021 Jun 15.
Adjuvant therapy for stage III melanoma improves several measures of patient survival. However, decisions regarding inclusion of adjuvant therapies in the formularies of public payers necessarily consider the cost-effectiveness of those treatments. The objective of this study is to evaluate the cost-effectiveness of four recently approved adjuvant therapies for BRAF-mutant stage III melanoma in the Medicare patient population.
In this cost-effectiveness analysis, a Markov microsimulation model was used to simulate the healthcare trajectory of patients randomized to receive either first-line targeted therapy (dabrafenib-trametinib) or immunotherapy (ipilimumab, nivolumab, or pembrolizumab). The base case was a 65-year-old Medicare patient with BRAF V600E-mutant resected stage III melanoma. Possible health states included recurrence-free survival, adverse events, local recurrence, distant metastases, and death. Transition probabilities were determined from published clinical trials. Costs were estimated from reimbursement rates reported by CMS and the Red Book drug price database. Primary outcomes were costs (US$), life years, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs). Model robustness was evaluated using one-way and probabilistic sensitivity analyses.
Dabrafenib-trametinib provided 1.83 QALYs over no treatment and 0.23 QALYs over the most effective immunotherapy, pembrolizumab. Dabrafenib-trametinib was associated with an ICER of $95,758/QALY over no treatment and $285,863/QALY over pembrolizumab. Pembrolizumab yielded an ICER of $68,396/QALY over no treatment and dominated other immunotherapies.
Pembrolizumab is cost-effective at a conventional willingness-to-pay (WTP) threshold, but dabrafenib-trametinib is not. Though dabrafenib-trametinib offers incremental QALYs, optimization of drug pricing is necessary to ensure dabrafenib-trametinib is accessible at an acceptable WTP threshold.
辅助治疗 III 期黑色素瘤可改善患者生存的多个指标。然而,公共支付者在考虑纳入辅助治疗方案时,必然会考虑这些治疗方法的成本效益。本研究旨在评估四项最近批准的 BRAF 突变型 III 期黑色素瘤辅助治疗药物在 Medicare 患者人群中的成本效益。
在这项成本效益分析中,采用马尔可夫微模拟模型来模拟接受一线靶向治疗(dabrafenib-trametinib)或免疫治疗(ipilimumab、nivolumab 或 pembrolizumab)的随机分组患者的医疗轨迹。基础病例是一位 65 岁的 Medicare 患者,患有 BRAF V600E 突变的 III 期黑色素瘤。可能的健康状态包括无复发生存、不良事件、局部复发、远处转移和死亡。转移概率来自已发表的临床试验。成本根据 CMS 和 Red Book 药物价格数据库报告的报销率进行估算。主要结果是成本(美元)、生命年、质量调整生命年(QALY)和增量成本效益比(ICER)。使用单因素和概率敏感性分析评估模型的稳健性。
与不治疗相比,dabrafenib-trametinib 提供了 1.83 个 QALY,与最有效的免疫治疗药物 pembrolizumab 相比,提供了 0.23 个 QALY。与不治疗相比,dabrafenib-trametinib 的 ICER 为 95758 美元/QALY,与 pembrolizumab 相比为 285863 美元/QALY。与不治疗相比,pembrolizumab 的 ICER 为 68396 美元/QALY,且优于其他免疫疗法。
pembrolizumab 在常规意愿支付(WTP)阈值下具有成本效益,但 dabrafenib-trametinib 则不然。虽然 dabrafenib-trametinib 提供了增量 QALY,但需要优化药物定价,以确保 dabrafenib-trametinib在可接受的 WTP 阈值下可获得。