Cost-effectiveness analysis of targeted-release formulation of budesonide (Tarpeyo) in conjunction with optimized renin-angiotensin system inhibitor (RASi) therapy relative to optimized RASi therapy alone for adults with primary immunoglobulin A nephropathy in the United States.

IF 2.3 4区 医学 Q2 HEALTH CARE SCIENCES & SERVICES
Mohsen Yaghoubi, Heng Jiang, Roman Casciano, Christopher Ngai, Mit Patel
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引用次数: 0

Abstract

Background: Immunoglobulin A nephropathy (IgAN) is a rare autoimmune disease that often leads to end-stage renal disease. The goal of treatment is to reduce disease progression so that patients are less likely to develop kidney failure in their natural lifetime. Recent clinical trial results show that Tarpeyo, a targeted-release formulation of budesonide designed to deliver the drug directly to gut-associated lymphoid tissue, reduces estimated glomerular filtration rate loss, potentially modifying the disease and thus prolonging the time to kidney failure.

Objective: To assess the cost-effectiveness of Tarpeyo in conjunction with optimized renin-angiotensin system inhibitor (RASi) therapy relative to optimized RASi therapy alone in US adult patients with primary IgAN.

Methods: A cost-utility approach is taken based on the full dataset from the phase 3 NefIgArd clinical trial. A semi-Markov model was developed with a lifetime horizon, encompassing both the US commercial payer and societal perspectives. The model architecture incorporated 9 health states, reflecting varying degrees of disease severity and mortality. Transition probabilities between health states were determined by a robust regression analysis of individual patient-level data obtained from the NefIgArd clinical trial and supplemented with data from literature. In the base-case analysis, treatment effect was assumed to be continuously maintained over the model time horizon (lifetime) and treatment was reapplied every 2 years. Treatment cost, adverse event management, dialysis, transplantation, mortality costs, and indirect costs were considered.

Results: Tarpeyo + optimized RASi was found to be dominant compared with optimized RASi alone from the perspective of a US third-party commercial payer, ie, cost saving ($105 729) with concurrent quality-adjusted life-year (QALY) gains of 1.12. The base-case results show that Tarpeyo is dominant when retreatment occurs every 2 years, with the treatment benefit assumed to be maintained over the same period throughout the model. Sensitivity analyses confirmed the robustness of the base-case results, showing that Tarpeyo plus optimized RASi is cost saving if benefits are sustained for at least 3 years. The treatment demonstrated high probabilities of cost-effectiveness at willingness-to-pay thresholds of less than $100K and less than $150K per QALY.

Conclusions: Clinical trials suggest that adding Tarpeyo to optimized RASi can help preserve kidney function by reducing estimated glomerular filtration rate loss in patients with IgAN. This addition was estimated to produce a greater QALY gain and reduced overall net costs from the payer and societal perspective in the United States.

布地奈德(Tarpeyo)靶向释放制剂联合优化肾素-血管紧张素系统抑制剂(RASi)治疗相对于优化的RASi单独治疗在美国原发性免疫球蛋白A肾病成人的成本-效果分析。
背景:免疫球蛋白A肾病(IgAN)是一种罕见的自身免疫性疾病,常导致终末期肾脏疾病。治疗的目标是减少疾病进展,使患者在其自然寿命中不太可能发生肾衰竭。最近的临床试验结果显示,Tarpeyo是一种布地奈德的靶向释放制剂,旨在将药物直接递送到肠道相关淋巴组织,可减少肾小球滤过率的估计损失,可能改善疾病,从而延长肾功能衰竭的时间。目的:评估Tarpeyo联合优化的肾素-血管紧张素系统抑制剂(RASi)治疗相对于优化的RASi单独治疗在美国成人原发性IgAN患者中的成本效益。方法:基于3期NefIgArd临床试验的完整数据集,采用成本-效用方法。一个包含美国商业付款人和社会观点的半马尔可夫模型被开发出来。模型架构包含9种健康状态,反映了不同程度的疾病严重程度和死亡率。通过对NefIgArd临床试验中获得的个体患者水平数据进行稳健回归分析,并辅以文献数据,确定了健康状态之间的转换概率。在基本情况分析中,假设治疗效果在模型时间范围(生命周期)内持续保持,并且每2年重新应用治疗。考虑了治疗费用、不良事件管理、透析、移植、死亡费用和间接费用。结果:从美国第三方商业付款人的角度来看,与单独优化的RASi相比,Tarpeyo +优化的RASi占主导地位,即节省成本(105729美元),同时质量调整生命年(QALY)收益为1.12。基本情况结果表明,当每2年进行一次再处理时,Tarpeyo占主导地位,并假设在整个模型中保持同一时期的处理效益。敏感性分析证实了基本情况结果的稳健性,表明如果效益持续至少3年,则Tarpeyo加上优化的RASi可以节省成本。在每个QALY的支付意愿阈值低于10万美元和15万美元时,该治疗显示出较高的成本效益概率。结论:临床试验表明,在优化的RASi中加入Tarpeyo可以通过减少IgAN患者的肾小球滤过率损失来帮助保护肾功能。据估计,从美国的付款人和社会角度来看,这一增加将产生更大的QALY收益,并降低总体净成本。
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来源期刊
Journal of managed care & specialty pharmacy
Journal of managed care & specialty pharmacy Health Professions-Pharmacy
CiteScore
3.50
自引率
4.80%
发文量
131
期刊介绍: JMCP welcomes research studies conducted outside of the United States that are relevant to our readership. Our audience is primarily concerned with designing policies of formulary coverage, health benefit design, and pharmaceutical programs that are based on evidence from large populations of people. Studies of pharmacist interventions conducted outside the United States that have already been extensively studied within the United States and studies of small sample sizes in non-managed care environments outside of the United States (e.g., hospitals or community pharmacies) are generally of low interest to our readership. However, studies of health outcomes and costs assessed in large populations that provide evidence for formulary coverage, health benefit design, and pharmaceutical programs are of high interest to JMCP’s readership.
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