Yaghoubi Mohsen, Jiang Heng, Casciano Roman, Ngai Christopher, Patel Mit
Certara US, Inc., Radnor, PA.
Calliditas NA Enterprises, Inc., New York, NY.
J Manag Care Spec Pharm. 2025 May;31(5):499-509. doi: 10.18553/jmcp.2025.31.5.499.
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.
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.
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.
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.
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.
免疫球蛋白A肾病（IgAN）是一种罕见的自身免疫性疾病，常导致终末期肾病。治疗的目标是减缓疾病进展，使患者在自然寿命期内发生肾衰竭的可能性降低。近期临床试验结果显示，替泊尤（Tarpeyo）是一种布地奈德的靶向释放制剂，旨在将药物直接递送至肠道相关淋巴组织，可降低估计肾小球滤过率损失，有可能改变疾病进程，从而延长至肾衰竭的时间。
评估在美国原发性IgAN成年患者中，替泊尤联合优化的肾素 - 血管紧张素系统抑制剂（RASi）治疗相对于单纯优化的RASi治疗的成本效益。
基于3期NefIgArd临床试验的完整数据集采用成本效用方法。开发了一个具有终身期限的半马尔可夫模型，涵盖美国商业支付方和社会视角。该模型架构纳入了9种健康状态，反映了不同程度的疾病严重程度和死亡率。健康状态之间的转移概率通过对从NefIgArd临床试验获得的个体患者水平数据进行稳健回归分析确定，并辅以文献数据。在基础案例分析中，假设治疗效果在模型时间范围（终身）内持续维持，且每2年重新进行治疗。考虑了治疗成本、不良事件管理、透析、移植、死亡率成本和间接成本。
从美国第三方商业支付方的角度来看，与单纯优化的RASi相比，替泊尤 + 优化的RASi具有优势，即节省成本（105729美元），同时质量调整生命年（QALY）增加1.12。基础案例结果表明，每2年重新治疗时，替泊尤具有优势，假设在整个模型期间治疗益处持续存在。敏感性分析证实了基础案例结果的稳健性，表明如果益处持续至少3年，替泊尤加优化的RASi可节省成本。在每QALY支付意愿阈值低于10万美元和低于15万美元时，该治疗显示出高成本效益概率。
临床试验表明，在优化的RASi基础上加用替泊尤可通过降低IgAN患者的估计肾小球滤过率损失来帮助保护肾功能。从美国支付方和社会角度估计，这种联合用药可带来更大的QALY增益并降低总体净成本。
