Astellas Pharma Europe Ltd, Addlestone, UK.
York Health Economics Consortium, University of York, York, UK.
J Med Econ. 2023 Jan-Dec;26(1):1250-1260. doi: 10.1080/13696998.2023.2263263. Epub 2023 Oct 10.
Treatment for anemia of chronic kidney disease (CKD) largely consists of erythropoiesis-stimulating agents (ESAs) with iron supplementation. Although ESAs are well-established and efficacious, their use has been associated with considerable economic and humanistic burdens. Roxadustat, an oral medication, is a hypoxia-inducible factor prolyl hydroxylase inhibitor that targets multiple causes of CKD and has a similar efficacy and safety profile to ESAs. The cost-effectiveness of this treatment, however, has yet to be investigated.
The study objective was to develop a health economic model to evaluate the cost-effectiveness of roxadustat compared with ESAs for treating anemia of non-dialysis-dependent (NDD) CKD.
A cohort-based model was developed for a hypothetical cohort of 1,000 patients with anemia of NDD CKD, incorporating eight health states, representing the hemoglobin level of each patient. The model was informed by individual patient-level data from the roxadustat global phase 3 clinical trial program. Total and incremental costs as well as quality-adjusted life-years (QALYs) associated with roxadustat versus ESAs were estimated from the perspective of the UK National Health Service. Sensitivity analyses were performed to assess the robustness of the model. Analyses exploring alternative scenarios were also conducted.
On a per-person basis, over 1,000 simulations, roxadustat was found to be on average less costly (-£32) and more effective (+0.01 QALYs) than ESAs, with a dominant incremental cost-effectiveness ratio. The probability of cost-effectiveness at a £20,000 per QALY willingness-to-pay threshold from the UK perspective was 67%.
The model developed may be a useful instrument that, alongside expert clinical opinion, can inform clinical and policy decision-making regarding treatment of anemia of NDD CKD. The model highlights the cost-effectiveness of roxadustat, as well as its potential to have a meaningful impact in reducing the burden of anemia of NDD CKD.
慢性肾脏病（CKD）贫血的治疗主要包括促红细胞生成素刺激剂（ESA）和铁剂补充。虽然 ESA 已经得到广泛认可并且有效，但它们的使用也带来了相当大的经济和人文负担。罗沙司他是一种口服药物，是一种低氧诱导因子脯氨酰羟化酶抑制剂，针对 CKD 的多种病因，其疗效和安全性与 ESA 相似。然而，这种治疗的成本效益尚未得到研究。
本研究旨在开发一种健康经济学模型，以评估罗沙司他治疗非透析依赖性（NDD）CKD 贫血与 ESA 相比的成本效益。
为 NDD CKD 贫血的 1000 名患者队列建立了一个基于队列的模型，纳入了 8 种健康状态，代表了每位患者的血红蛋白水平。该模型的信息来自罗沙司他全球 3 期临床试验计划的个体患者水平数据。从英国国家医疗服务体系的角度估算了罗沙司他与 ESA 相比的总费用和增量成本以及质量调整生命年（QALY）。进行了敏感性分析以评估模型的稳健性。还进行了探索替代方案的分析。
在 1000 次模拟中，罗沙司他的人均成本平均降低了 32 英镑（-£32），且效果更好（增加了 0.01 个 QALY），具有优势增量成本效益比。从英国的角度来看，在 £20,000 每 QALY 的意愿支付阈值下，具有成本效益的概率为 67%。
从英国的角度来看，所开发的模型可能是一种有用的工具，可以结合专家临床意见，为治疗 NDD CKD 贫血的临床和政策决策提供信息。该模型突出了罗沙司他的成本效益，以及它在减轻 NDD CKD 贫血负担方面的潜在意义。
