Graduate Program in Pharmaceutical Sciences, Universidade Federal do Paraná, Curitiba, Brazil.
Toronto Health Economics and Technology Assessment Collaborative (THETA), Toronto, Ontario, Canada.
Neuroendocrinology. 2021;111(4):388-402. doi: 10.1159/000507890. Epub 2020 Apr 16.
To conduct a cost-utility analysis comparing drug strategies involving octreotide, lanreotide, pasireotide, and pegvisomant for the treatment of patients with acromegaly who have failed surgery, from a Brazilian public payer perspective.
A probabilistic cohort Markov model was developed. One-year cycles were employed. The patients started at 45 years of age and were followed lifelong. Costs, efficacy, and quality of life parameters were retrieved from the literature. A discount rate (5%) was applied to both costs and efficacy. The results were reported as costs per quality-adjusted life year (QALY), and incremental cost-effectiveness ratios (ICERs) were calculated when applicable. Scenario analyses considered alternative dosages, discount rate, tax exemption, and continued use of treatment despite lack of response. Value of information (VOI) analysis was conducted to explore uncertainty and to estimate the costs to be spent in future research.
Only lanreotide showed an ICER reasonable for having its use considered in clinical practice (R$ 112,138/US$ 28,389 per QALY compared to no treatment). Scenario analyses corroborated the base-case result. VOI analysis showed that much uncertainty surrounds the parameters, and future clinical research should cost less than R$ 43,230,000/US$ 10,944,304 per year. VOI also showed that almost all uncertainty that precludes an optimal strategy choice involves quality of life.
With current information, the only strategy that can be considered cost-effective in Brazil is lanreotide treatment. No second-line treatment is recommended. Significant uncertainty of parameters impairs optimal decision-making, and this conclusion can be generalized to other countries. Future research should focus on acquiring utility data.
从巴西公共支付方的角度出发，比较奥曲肽、兰瑞肽、帕瑞肽和培维索孟治疗手术失败的肢端肥大症患者的药物策略的成本-效用分析。
开发了一个概率队列马尔可夫模型。采用一年周期。患者从 45 岁开始，终身随访。从文献中检索成本、疗效和生活质量参数。对成本和疗效均应用 5%的折扣率。结果以每质量调整生命年（QALY）的成本表示，在适用的情况下计算增量成本效益比（ICER）。情景分析考虑了替代剂量、折扣率、免税以及尽管缺乏反应但继续使用治疗。进行价值信息（VOI）分析以探索不确定性并估计未来研究的成本。
只有兰瑞肽的 ICER 合理，可考虑在临床实践中使用（与不治疗相比，每 QALY 的成本为 112,138 雷亚尔/28,389 美元）。情景分析证实了基础案例的结果。VOI 分析表明，参数存在很大的不确定性，未来的临床研究每年应花费少于 43,230,000 雷亚尔/10,944,304 美元。VOI 还表明，几乎所有排除最佳策略选择的不确定性都涉及生活质量。
根据现有信息，巴西唯一具有成本效益的策略是兰瑞肽治疗。不推荐二线治疗。参数的显著不确定性会影响最佳决策制定，这一结论可以推广到其他国家。未来的研究应侧重于获取效用数据。
