Mohsen Yaghoubi, Heng Jiang, Roman Casciano, Christopher Ngai, Mit Patel
{"title":"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。","authors":"Mohsen Yaghoubi, Heng Jiang, Roman Casciano, Christopher Ngai, Mit Patel","doi":"10.18553/jmcp.2025.31.5.499","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":16170,"journal":{"name":"Journal of managed care & specialty pharmacy","volume":"31 5","pages":"499-509"},"PeriodicalIF":2.3000,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12041922/pdf/","citationCount":"0","resultStr":"{\"title\":\"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.\",\"authors\":\"Mohsen Yaghoubi, Heng Jiang, Roman Casciano, Christopher Ngai, Mit Patel\",\"doi\":\"10.18553/jmcp.2025.31.5.499\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>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.</p><p><strong>Objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>\",\"PeriodicalId\":16170,\"journal\":{\"name\":\"Journal of managed care & specialty pharmacy\",\"volume\":\"31 5\",\"pages\":\"499-509\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2025-05-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12041922/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of managed care & specialty pharmacy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.18553/jmcp.2025.31.5.499\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of managed care & specialty pharmacy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.18553/jmcp.2025.31.5.499","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
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.
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.
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.