Daniel Lindsay, Andrea Henden, Ricky Nelles, Thomas M Elliott, Louisa G Collins
{"title":"为指导中危髓系恶性肿瘤患者的治疗而进行的 37 基因小组与常规护理的成本效用探索性分析。","authors":"Daniel Lindsay, Andrea Henden, Ricky Nelles, Thomas M Elliott, Louisa G Collins","doi":"10.1007/s40258-024-00927-8","DOIUrl":null,"url":null,"abstract":"<p><strong>Objective: </strong>Genomic risk stratification methods for myeloid malignancies have moved beyond conventional karyotyping and single gene approaches to better define disease behaviour. Next-generation sequencing has been established as the new standard-of-care tool to accurately define prognosis at diagnosis and guide therapy decisions. We aimed to determine the economic value of a 37-gene panel test for informing subsequent care for patients with intermediate-risk myeloid malignancies.</p><p><strong>Method: </strong>We performed an exploratory cost-utility analysis of a 37-gene panel test to inform stem cell transplantation therapy in patients with myeloid malignancies in Queensland, Australia. Clinician surveys provided data on management choice with and without genomics information while both published and individual-level data were used for healthcare costs, quality of life, relapse rates and survival data. We used a decision-analytic cohort model with Markov chains and 5000 simulations to derive the incremental cost per quality-adjusted life year (QALY) gained. Scenario, one-way and probabilistic sensitivity analyses were undertaken to test input variation on the stability of the main findings.</p><p><strong>Results: </strong>Over 10 years, the model predicted mean costs of AU$125,561 for the panel testing strategy and AU$117,045 for usual care, indicating an incremental cost of AU$8516 for panel testing. The corresponding mean QALYs were 4.52 for panel testing and 4.46 for usual care, producing a cost of AU$153,854 per QALY gained. In the Australian system, the likelihood that panel testing would be cost effective was <1 % and would have a more favourable cost-effective profile at a willingness-to-pay of AU$140,000 per QALY gained.</p><p><strong>Conclusions: </strong>Driven by small gains in survival and relapse rates following therapies, genomic panel sequencing for myeloid malignancies in people with intermediate-risk disease is unlikely to be cost effective in Australia.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.1000,"publicationDate":"2024-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Exploratory Cost-Utility Analysis of a 37-Gene Panel Versus Usual Care to Guide Therapy for Patients with Intermediate-Risk Myeloid Malignancies.\",\"authors\":\"Daniel Lindsay, Andrea Henden, Ricky Nelles, Thomas M Elliott, Louisa G Collins\",\"doi\":\"10.1007/s40258-024-00927-8\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Objective: </strong>Genomic risk stratification methods for myeloid malignancies have moved beyond conventional karyotyping and single gene approaches to better define disease behaviour. Next-generation sequencing has been established as the new standard-of-care tool to accurately define prognosis at diagnosis and guide therapy decisions. We aimed to determine the economic value of a 37-gene panel test for informing subsequent care for patients with intermediate-risk myeloid malignancies.</p><p><strong>Method: </strong>We performed an exploratory cost-utility analysis of a 37-gene panel test to inform stem cell transplantation therapy in patients with myeloid malignancies in Queensland, Australia. Clinician surveys provided data on management choice with and without genomics information while both published and individual-level data were used for healthcare costs, quality of life, relapse rates and survival data. We used a decision-analytic cohort model with Markov chains and 5000 simulations to derive the incremental cost per quality-adjusted life year (QALY) gained. Scenario, one-way and probabilistic sensitivity analyses were undertaken to test input variation on the stability of the main findings.</p><p><strong>Results: </strong>Over 10 years, the model predicted mean costs of AU$125,561 for the panel testing strategy and AU$117,045 for usual care, indicating an incremental cost of AU$8516 for panel testing. The corresponding mean QALYs were 4.52 for panel testing and 4.46 for usual care, producing a cost of AU$153,854 per QALY gained. In the Australian system, the likelihood that panel testing would be cost effective was <1 % and would have a more favourable cost-effective profile at a willingness-to-pay of AU$140,000 per QALY gained.</p><p><strong>Conclusions: </strong>Driven by small gains in survival and relapse rates following therapies, genomic panel sequencing for myeloid malignancies in people with intermediate-risk disease is unlikely to be cost effective in Australia.</p>\",\"PeriodicalId\":8065,\"journal\":{\"name\":\"Applied Health Economics and Health Policy\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":3.1000,\"publicationDate\":\"2024-11-13\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Applied Health Economics and Health Policy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s40258-024-00927-8\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"ECONOMICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Applied Health Economics and Health Policy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40258-024-00927-8","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ECONOMICS","Score":null,"Total":0}
Exploratory Cost-Utility Analysis of a 37-Gene Panel Versus Usual Care to Guide Therapy for Patients with Intermediate-Risk Myeloid Malignancies.
Objective: Genomic risk stratification methods for myeloid malignancies have moved beyond conventional karyotyping and single gene approaches to better define disease behaviour. Next-generation sequencing has been established as the new standard-of-care tool to accurately define prognosis at diagnosis and guide therapy decisions. We aimed to determine the economic value of a 37-gene panel test for informing subsequent care for patients with intermediate-risk myeloid malignancies.
Method: We performed an exploratory cost-utility analysis of a 37-gene panel test to inform stem cell transplantation therapy in patients with myeloid malignancies in Queensland, Australia. Clinician surveys provided data on management choice with and without genomics information while both published and individual-level data were used for healthcare costs, quality of life, relapse rates and survival data. We used a decision-analytic cohort model with Markov chains and 5000 simulations to derive the incremental cost per quality-adjusted life year (QALY) gained. Scenario, one-way and probabilistic sensitivity analyses were undertaken to test input variation on the stability of the main findings.
Results: Over 10 years, the model predicted mean costs of AU$125,561 for the panel testing strategy and AU$117,045 for usual care, indicating an incremental cost of AU$8516 for panel testing. The corresponding mean QALYs were 4.52 for panel testing and 4.46 for usual care, producing a cost of AU$153,854 per QALY gained. In the Australian system, the likelihood that panel testing would be cost effective was <1 % and would have a more favourable cost-effective profile at a willingness-to-pay of AU$140,000 per QALY gained.
Conclusions: Driven by small gains in survival and relapse rates following therapies, genomic panel sequencing for myeloid malignancies in people with intermediate-risk disease is unlikely to be cost effective in Australia.
期刊介绍:
Applied Health Economics and Health Policy provides timely publication of cutting-edge research and expert opinion from this increasingly important field, making it a vital resource for payers, providers and researchers alike. The journal includes high quality economic research and reviews of all aspects of healthcare from various perspectives and countries, designed to communicate the latest applied information in health economics and health policy.
While emphasis is placed on information with practical applications, a strong basis of underlying scientific rigor is maintained.