Assistance Publique-Hôpitaux de Marseille (AP-HM), Department of Endocrinology, Hôpital de la Conception, Centre de Référence des Maladies Rares de l'hypophyse HYPO, Marseille, France.
Aix-Marseille Université, Institut National de la Santé et de la Recherche Médicale (INSERM), U1251, Marseille Medical Genetics (MMG), Institut Marseille Maladies Rares (MarMaRa), Marseille, France.
Front Endocrinol (Lausanne). 2021 Oct 7;12:745843. doi: 10.3389/fendo.2021.745843. eCollection 2021.
Efficacy of pharmacological treatments for acromegaly has been assessed in many clinical or real-world studies but no study was interested in economics evaluation of these treatments in France. Therefore, the objective of this study was to estimate the cost-utility of second-line pharmacological treatments in acromegaly patients.
A Markov model was developed to follow a cohort of 1,000 patients for a lifetime horizon. First-generation somatostatin analogues (FGSA), pegvisomant, pasireotide and pegvisomant combined with FGSA (off label) were compared. Efficacy was defined as the normalization of insulin-like growth factor-1 (IGF-1) concentration and was obtained from pivotal trials and adjusted by a network meta-analysis. Costs data were obtained from French databases and literature. Utilities from the literature were used to estimate quality-adjusted life year (QALY).
The incremental cost-utility ratios (ICUR) of treatments compared to FGSA were estimated to be 562,463 € per QALY gained for pasireotide, 171,332 € per QALY gained for pegvisomant, and 186,242 € per QALY gained for pegvisomant + FGSA. Pasireotide seems to be the least cost-efficient treatment. Sensitivity analyses showed the robustness of the results.
FGSA, pegvisomant and pegvisomant + FGSA were on the cost-effective frontier, therefore, depending on the willingness-to-pay for an additional QALY, they are the most cost-effective treatments. This medico-economic analysis highlighted the consistency of the efficiency results with the efficacy results assessed in the pivotal trials. However, most recent treatment guidelines recommend an individualized treatment strategy based on the patient and disease profile.
许多临床或真实世界研究已经评估了药物治疗肢端肥大症的疗效，但没有研究关注这些治疗方法在法国的经济学评价。因此，本研究的目的是评估肢端肥大症二线药物治疗的成本效用。
开发了一个马尔可夫模型来跟踪 1000 名患者的终生情况。第一代生长抑素类似物（FGSA）、培维索孟、帕瑞肽和 FGSA 联合培维索孟（超适应证）进行了比较。疗效通过关键试验确定，并通过网络荟萃分析进行了调整。成本数据来自法国数据库和文献。效用来自文献，用于估计质量调整生命年（QALY）。
与 FGSA 相比，培维索孟、帕瑞肽和培维索孟+FGSA 的增量成本效用比（ICUR）分别为每获得 1 个 QALY 需花费 562463 欧元、171332 欧元和 186242 欧元。帕瑞肽似乎是最不具有成本效益的治疗方法。敏感性分析显示结果具有稳健性。
FGSA、培维索孟和培维索孟+FGSA 均位于成本效益边界内，因此，根据对额外 QALY 的支付意愿，它们是最具成本效益的治疗方法。这项医疗经济分析强调了效率结果与关键试验中评估的疗效结果的一致性。然而，最新的治疗指南建议根据患者和疾病特征制定个体化的治疗策略。
