Rosa Daniela Dornelles, Magliano Carlos Alberto da Silva, Simon Sergio D, Amorim Gilberto, Reinert Tomás, Landeiro Luciana, Gagliato Débora de Melo, Exman Pedro, Argolo Daniel, Guilgen Gisah, Mano Max, Testa Laura, Liedke Pedro, Barroso Romualdo, Sasse Mariana, Buehler Anna Maria
Oncology, Grupo Brasileiro de Estudos do Câncer de Mama (GBECAM), São Paulo, BrazilOncology, Hospital Moinhos de Vento (HMV), Porto Alegre, Brazil.
Oncology, Asas Avaliações Econômicas em Saúde Ltda, Rio de Janeiro, Brazil.
Ther Adv Med Oncol. 2022 Jun 20;14:17588359221100865. doi: 10.1177/17588359221100865. eCollection 2022.
The MONALEESA-7 trial compared ribociclib plus endocrine therapy (ET) with placebo as first-line treatment of advanced luminal/HER2-negative breast cancer (ABC) in premenopausal and perimenopausal women (age <50 years) and showed significant benefits to progression-free survival and overall survival. This study aimed to compare the cost-effectiveness of ribociclib + ET ET alone in patients with ABC from the perspective of the Brazilian public national health system.
We calculated the incremental cost-effectiveness ratio (ICER) using a Markov model with progression-free survival, post-progression survival, and death states. We expressed ICER as incremental costs per progression-free life-year (PFLY) and quality-adjusted life-year (QALY) gained in a 10-year time horizon. We used parametric survival distributions fit to MONALEESA-7 data to generate survival distributions for progression-free and post-progression survival. The largest British preference study in breast cancer served as the basis to estimate health-state utilities. We estimated direct costs (ABC treatment, follow-up, monitoring, and adverse events) using Brazilian-specific values from public sources. An expert consensus panel determined the resource patterns required. We applied annual discounts of 5% to costs and QALYs.
Ribociclib + ET resulted in an incremental gain of 1.03 PFLYs and 0.80 QALYs at a cost of $37,319.31. The ICER of ribociclib + ET ET was $36,379.41 per PFLY gained and $46,590.79 per QALY gained. In deterministic sensitivity analysis, results were primarily affected by the annual discount rate, followed by the cost of ribociclib. In probabilistic sensitivity analysis, simulations agreed with the base-case.
Ribociclib increased PFLYs and QALYs in patients with HR+/HER2- ABC when added to ET. Because Brazil does not have a formally defined cost-effectiveness threshold, other domains need to be considered for incorporation decisions, such as disease burden and humanistic impact on this young, economically active population. These findings may be useful in discussions for incorporation of ribociclib into the Brazilian public health system.
MONALEESA - 7试验比较了瑞博西尼联合内分泌治疗（ET）与安慰剂作为绝经前和围绝经期女性（年龄<50岁）晚期腔面型/人表皮生长因子受体2阴性乳腺癌（ABC）一线治疗的疗效，结果显示其对无进展生存期和总生存期有显著益处。本研究旨在从巴西国家公共卫生系统的角度比较瑞博西尼联合ET与单纯ET治疗ABC患者的成本效益。
我们使用马尔可夫模型，结合无进展生存期、进展后生存期和死亡状态，计算增量成本效益比（ICER）。我们将ICER表示为每获得一个无进展生命年（PFLY）和质量调整生命年（QALY）所增加的成本，时间跨度为10年。我们使用拟合MONALEESA - 7数据的参数生存分布来生成无进展生存期和进展后生存期的生存分布。英国规模最大的乳腺癌偏好研究作为估计健康状态效用的基础。我们使用来自公共来源的巴西特定值估计直接成本（ABC治疗、随访、监测和不良事件）。一个专家共识小组确定了所需的资源模式。我们对成本和QALY应用5%的年度贴现率。
瑞博西尼联合ET可增加1.03个PFLY和0.80个QALY，成本为37,319.31美元。瑞博西尼联合ET相对于单纯ET的ICER为每获得一个PFLY增加36,379.41美元，每获得一个QALY增加46,590.79美元。在确定性敏感性分析中，结果主要受年度贴现率影响，其次是瑞博西尼的成本。在概率敏感性分析中，模拟结果与基础情况一致。
对于HR + /HER2 - ABC患者，在ET基础上加用瑞博西尼可增加PFLY和QALY。由于巴西没有正式定义的成本效益阈值，纳入决策时需要考虑其他领域，如疾病负担以及对这个年轻且经济活跃人群的人文影响。这些发现可能有助于在将瑞博西尼纳入巴西公共卫生系统的讨论中发挥作用。
