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Acceptance of Evidence Transfer Within German Early Benefit Assessment of New Drugs for Pediatric and Adolescents Target Populations. 在德国儿科和青少年目标人群新药早期效益评估中的证据转移接受。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 Epub Date: 2025-01-17 DOI: 10.1007/s40273-024-01467-8
Georgia Pick, Dirk Müller, Charalabos-Markos Dintsios
{"title":"Acceptance of Evidence Transfer Within German Early Benefit Assessment of New Drugs for Pediatric and Adolescents Target Populations.","authors":"Georgia Pick, Dirk Müller, Charalabos-Markos Dintsios","doi":"10.1007/s40273-024-01467-8","DOIUrl":"10.1007/s40273-024-01467-8","url":null,"abstract":"<p><strong>Background and objective: </strong>In Germany, all new drugs undergo an early benefit assessment (EBA) by the decision-making body (G-BA). Due to limited access to clinical data in pediatric healthcare since 2017, evidence transfer has allowed for data from adult studies to be used in the EBA of pediatric drugs. This study examines the acceptance of evidence transfer, aiming to understand its correlation with granted added benefit.</p><p><strong>Methods: </strong>By searching the G-BA database, relevant EBAs were identified. In addition to descriptive statistics, agreement statistics regarding binary and ordinal extent of added benefit and binary logistic regression with and without intercept were performed to investigate acceptance of evidence transfer, juxtaposing it with manufacturers' claims, and to evaluate the impact of identified factors on evidence transfer.</p><p><strong>Results: </strong>In 14 of 36 identified EBAs, the evidence transfer was accepted by the G-BA. They referred to four therapeutic areas, received a non-quantifiable added benefit and were subject to a pediatric investigation program. Non-quantifiable added benefit implies an added value in itself which can range from minor to major added benefit and is considering the genuine uncertainty mainly induced in theese EBAs due to evidence transfer, which is not allowing a quantification of an added benefit. The binary agreement between manufacturers' claims and G-BA's appraisals was less than by chance [kappa - 0.054 (- 0.158 to 0.050)] whereas the ordinal agreement became fair [kappa 0.333 (0.261-0.406)]. Congruence of the mechanism of action, alignment of disease pattern, transferability of efficacy and safety, and same comparator were fundamental for evidence transfer. Additionally, supportive evidence, therapeutic breakthroughs, and small-scale approval enhanced the acceptance of evidence transfer. The regression models yielded similar results showing different model fit and explained variance.</p><p><strong>Conclusions: </strong>Evidence transfer hinges upon fulfilling various minimum criteria and additional supportive evidence. Availability of study data from adult or older patients and the pediatric group under evaluation is crucial.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"521-553"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011912/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009453","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI's Gene Therapy Access Model. 医疗补助中镰状细胞病基因治疗的创新支付模式:利用CMMI基因治疗获取模型的真实世界数据和见解。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 Epub Date: 2025-02-21 DOI: 10.1007/s40273-025-01474-3
Antal Zemplenyi, Jim Leonard, Garth C Wright, Michael J DiStefano, Kavita Nair, Kelly E Anderson, R Brett McQueen
{"title":"Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI's Gene Therapy Access Model.","authors":"Antal Zemplenyi, Jim Leonard, Garth C Wright, Michael J DiStefano, Kavita Nair, Kelly E Anderson, R Brett McQueen","doi":"10.1007/s40273-025-01474-3","DOIUrl":"10.1007/s40273-025-01474-3","url":null,"abstract":"<p><strong>Objective: </strong>This study aims to evaluate the financial implications of implementing various payment models, including outcome-based agreements (OBAs), volume-based rebates, and guaranteed rebates, for the newly approved gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), in the treatment of sickle-cell disease (SCD) from the perspective of Colorado Medicaid. The analysis specifically examines the cost of standard of care (SoC) for severe SCD, the impact of different eligibility criteria based on vaso-occlusive events (VOEs), and the potential financial impacts associated with rebate structures.</p><p><strong>Methods: </strong>Data from the Colorado Department of Health Care Policy & Financing (HCPF) database was used to estimate the annual costs for Medicaid-enrolled patients with severe SCD from 2018 to 2023. Patients were selected based on various eligibility criteria, including the number of VOEs, acute chest syndrome events, and stroke diagnoses. Three-state Markov models (SCD, stable, and dead) were constructed to compare the costs of SoC and gene therapies. The durability of gene therapy effectiveness and the financial impact of OBAs, volume-based rebates, and guaranteed rebates were evaluated over a 6-year contract period, with scenarios reflecting different VOE criteria and treatment durability.</p><p><strong>Results: </strong>The average annual SoC cost for severe SCD patients (N = 138) was US$45,941 (SD US$59,653), with higher costs associated with more frequent VOEs. Gene therapies exa-cel and lovo-cel, with one-off list prices of US$2.2 million and US$3.1 million, respectively, exhibited high upfront costs, resulting in a negative cumulative balance averaging - US$2.11 million for exa-cel and - US$3.00 million for lovo-cel per patient over 6 years compared with SoC. Outcome-based rebates could potentially save Medicaid approximately US$260K (uncertainty interval 88K-772K) per patient on average for exa-cel and US$367K (uncertainty interval 122K-1111K) for lovo-cel after they pay the full up-front cost. Volume-based and guaranteed rebates also offered potential savings but varied in impact based on contract duration and effectiveness of gene therapy.</p><p><strong>Conclusions: </strong>The study highlights critical considerations for Medicaid in negotiating OBAs for SCD gene therapies. Achieving budget neutrality over 6 years is unlikely due to low SoC costs. However, payment models can enhance value-based spending by linking high therapy costs and potential rebates to the health gains these treatments may offer. OBAs offer offsets contingent on therapy effectiveness durability and contract terms (such as length and price), while varying eligibility criteria impact budgets and outcomes. Medicaid real-world data is crucial for navigating complexities in defining eligible populations and structuring OBAs.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"583-594"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011968/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143468544","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Correction: De Novo Cost‑Effectiveness Model Framework for Nonalcoholic Steatohepatitis-Modeling Approach and Validation. 更正:非酒精性脂肪性肝炎的全新成本-效果模型框架-建模方法和验证。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 DOI: 10.1007/s40273-025-01475-2
Peter Gal, Gyorgyi Feldmajer, Margarida Augusto, Ray Gani, Emma Hook, Ash Bullement, Zoe Philips, Inger Smith
{"title":"Correction: De Novo Cost‑Effectiveness Model Framework for Nonalcoholic Steatohepatitis-Modeling Approach and Validation.","authors":"Peter Gal, Gyorgyi Feldmajer, Margarida Augusto, Ray Gani, Emma Hook, Ash Bullement, Zoe Philips, Inger Smith","doi":"10.1007/s40273-025-01475-2","DOIUrl":"10.1007/s40273-025-01475-2","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"595-596"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011896/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143616740","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
EQ-5D-5L or EQ-HWB-S: Which is the Better Instrument for Capturing Spillover Effects in Parental Carers of Children with COVID-19? EQ-5D-5L与EQ-HWB-S:哪种工具更能捕捉COVID-19患儿父母照顾者的溢出效应?
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 Epub Date: 2025-02-05 DOI: 10.1007/s40273-025-01473-4
Wenjing Zhou, Bo Ding, Jan Busschbach, Michael Herdman, Zhihao Yang, Yanming Lu
{"title":"EQ-5D-5L or EQ-HWB-S: Which is the Better Instrument for Capturing Spillover Effects in Parental Carers of Children with COVID-19?","authors":"Wenjing Zhou, Bo Ding, Jan Busschbach, Michael Herdman, Zhihao Yang, Yanming Lu","doi":"10.1007/s40273-025-01473-4","DOIUrl":"10.1007/s40273-025-01473-4","url":null,"abstract":"<p><strong>Background and objectives: </strong>'Caregiver health spillovers' refer to the broader impacts of an individual's illness and interventions on informal caregivers' health and well-being. This study focuses on the spillover effects experienced by parental carers of children with coronavirus disease 2019 (COVID-19), aiming to compare the psychometric properties of the EQ-5D-5L and the experimental EQ Health and Wellbeing Short version (EQ-HWB-S) in capturing these effects.</p><p><strong>Methods: </strong>A longitudinal study was conducted with 861 parental carers of children aged 0-18 years with COVID-19 and 231 parents of healthy children as the control group. The EQ-5D-5L and EQ-HWB-S were used to assess parental health and well-being. Analyses included known-groups validity (multivariable regression), test-retest reliability (Gwet's AC1, intraclass correlation coefficient) and responsiveness to health improvement (Glass' Δ effect size).</p><p><strong>Results: </strong>Parents of infected children reported more problems than those of healthy controls. The EQ-HWB-S better discriminated between sub-groups defined by the child's COVID-19 presence, caring time and work impact. Test-retest reliability was fair to good for EQ-HWB-S dimensions (Gwet's AC1: 0.33-0.79), moderate to good for EQ-5D-5L (Gwet's AC1: 0.40-0.76), and good for index scores and EQ VAS (intraclass correlation coefficient: 0.70-0.77). Parental health and well-being improved as children recovered, with the EQ-5D-5L showing slightly higher responsiveness (effect size: 0.77-0.87) than EQ-HWB-S (effect size: 0.62-0.74).</p><p><strong>Conclusions: </strong>Both EQ-HWB-S and EQ-5D-5L are valid, reliable and responsive for measuring parental spillover effects related to a child's COVID-19 infection. EQ-HWB-S outperformed in distinguishing social and emotional impacts of caregiving, while EQ-5D-5L better captured physical health improvements. The choice between tools may depend on study objectives.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"555-567"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011911/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143188709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Taxonomy for Assessing Whether HRQoL Value Sets Are Obsolete. 评估HRQoL值集是否过时的分类法。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 Epub Date: 2025-02-17 DOI: 10.1007/s40273-025-01476-1
Richard Norman, Bram Roudijk, Marcel Jonker, Elly Stolk, Saskia Knies, Raoh-Fang Pwu, Ciaran O'Neill, Kirsten Howard, Nancy Devlin
{"title":"A Taxonomy for Assessing Whether HRQoL Value Sets Are Obsolete.","authors":"Richard Norman, Bram Roudijk, Marcel Jonker, Elly Stolk, Saskia Knies, Raoh-Fang Pwu, Ciaran O'Neill, Kirsten Howard, Nancy Devlin","doi":"10.1007/s40273-025-01476-1","DOIUrl":"10.1007/s40273-025-01476-1","url":null,"abstract":"<p><p>Providing health-related quality of life (HRQoL) value sets to enable estimation of quality adjusted life years (QALYs) is important in facilitating economic evaluation and in supporting reliable decision-making about healthcare. However, as the field matures, many value sets across a range of HRQoL instruments are now old, based on potentially outdated valuation methodologies and preference data from samples that no longer represent the contemporary population. Having a clear strategy for identification and mitigation of obsolescence is important to ensure policy makers retain confidence in their country-specific value sets. In this Current Opinion, we develop a taxonomy of value set obsolescence. We then explore how the different types of obsolescence might be identified and how methodologists might work with local policymakers to address obsolescence and therefore ensure HRQoL instruments remain relevant for use. The taxonomy of obsolescence consists of four main areas: (1) the value set no longer aligns with current normative health technology assessment (HTA) requirements; (2) the methods used to generate it are no longer considered robust or adequately close to best practice; (3) the population composition has moved too far from the characteristics of the sample in which the original value set was derived; and (4) even after controlling for population differences, preferences are likely to have changed since the original data collection. Through identification of the type of obsolescence that applies in a particular setting, we then suggest a range of possible solutions to each, ranging from recommending particular sensitivity analyses, through reweighting of existing data to better account for population differences, to collecting new data for an updated value set. Obsolescence of existing value sets is driven by more than just time since data collection is often a matter of judgment rather than based on a clear definition. The taxonomy presented here provides a tool for assessing whether value sets are obsolete and what the appropriate response to this obsolescence should be. Working closely with local policymakers and involving discussions regarding the ongoing appropriateness of existing value sets should form an important part of future activities. This should include the consideration of updating value sets in contemporary populations using current best-practice methods. However, the benefits of updating value sets have to be balanced against the cost of doing so, including the challenges faced by policymakers when new values sets require a transition to new local decision-making processes.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"473-481"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011929/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Uncertainty in Matching-Adjusted Indirect Comparisons Using Real-World Evidence: Evidence Assessment Group Perspective. 使用真实世界证据的匹配调整间接比较的不确定性:证据评估组的观点。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 Epub Date: 2025-03-21 DOI: 10.1007/s40273-025-01480-5
Cyril Onwuelazu Uteh, Eugenie Evelynne Johnson, Tomos Robinson, Najmeh Moradi, Alex Inskip, Fiona R Beyer, Katie Thomson, Gurdeep S Sagoo
{"title":"Uncertainty in Matching-Adjusted Indirect Comparisons Using Real-World Evidence: Evidence Assessment Group Perspective.","authors":"Cyril Onwuelazu Uteh, Eugenie Evelynne Johnson, Tomos Robinson, Najmeh Moradi, Alex Inskip, Fiona R Beyer, Katie Thomson, Gurdeep S Sagoo","doi":"10.1007/s40273-025-01480-5","DOIUrl":"10.1007/s40273-025-01480-5","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"469-472"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143674450","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Towards Recommendations for Cost-Effectiveness Analysis of Predictive, Prognostic, and Serial Biomarker Tests in Oncology. 肿瘤学预测、预后和系列生物标志物测试的成本-效果分析建议
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 Epub Date: 2025-02-08 DOI: 10.1007/s40273-025-01470-7
Astrid Kramer, Lucas F van Schaik, Daan van den Broek, Gerrit A Meijer, Iñaki Gutierrez Ibarluzea, Lorea Galnares Cordero, Remond J A Fijneman, Marjolijn J L Ligtenberg, Ed Schuuring, Wim H van Harten, Veerle M H Coupé, Valesca P Retèl
{"title":"Towards Recommendations for Cost-Effectiveness Analysis of Predictive, Prognostic, and Serial Biomarker Tests in Oncology.","authors":"Astrid Kramer, Lucas F van Schaik, Daan van den Broek, Gerrit A Meijer, Iñaki Gutierrez Ibarluzea, Lorea Galnares Cordero, Remond J A Fijneman, Marjolijn J L Ligtenberg, Ed Schuuring, Wim H van Harten, Veerle M H Coupé, Valesca P Retèl","doi":"10.1007/s40273-025-01470-7","DOIUrl":"10.1007/s40273-025-01470-7","url":null,"abstract":"<p><strong>Background: </strong>Cost-effectiveness analysis (CEA) of biomarkers is challenging due to the indirect impact on health outcomes and the lack of sufficient fit-for-purpose data. Hands-on guidance is lacking.</p><p><strong>Objective: </strong>We aimed firstly to explore how CEAs in the context of three different types of biomarker applications have addressed these challenges, and secondly to develop recommendations for future CEAs.</p><p><strong>Methods: </strong>A scoping review was performed for three biomarker applications: predictive, prognostic, and serial testing, in advanced non-small cell lung cancer, early-stage colorectal cancer, and all-stage colorectal cancer, respectively. Information was extracted on the model assumptions and uncertainty, and the reported outcomes. An in-depth analysis of the literature was performed describing the impact of model assumptions in the included studies.</p><p><strong>Results: </strong>A total of 43 CEAs were included (31 predictive, 6 prognostic, and 6 serial testing). Of these, 40 utilized different sources for test and treatment parameters, and three studies utilized a single source. Test performance was included in 78% of these studies utilizing different sources, but this parameter was differently expressed across biomarker applications. Sensitivity analyses for test performance was only performed in half of these studies. For the linkage of test results to treatments outcomes, a minority of the studies explored the impact of suboptimal adherence to test results, and/or explored potential differences in treatment effects for different biomarker subgroups. Intermediate outcomes were reported by 67% of studies.</p><p><strong>Conclusions: </strong>We identified various approaches for dealing with challenges in CEAs of biomarker tests for three different biomarker applications. Recommendations on assumptions, handling uncertainty, and reported outcomes were drafted to enhance modeling practices for future biomarker cost-effectiveness evaluations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"483-497"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011951/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143370696","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria. 检查NICE单一技术评价的一致性:阵发性夜间血红蛋白尿评价综述。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 Epub Date: 2025-02-12 DOI: 10.1007/s40273-025-01472-5
Jeremiah Donoghue, Matthew Youngs, Alex Reeve, Krishna Vydyula, Natalia Kunst, Roochi Trikha, Daniel Gallacher
{"title":"Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria.","authors":"Jeremiah Donoghue, Matthew Youngs, Alex Reeve, Krishna Vydyula, Natalia Kunst, Roochi Trikha, Daniel Gallacher","doi":"10.1007/s40273-025-01472-5","DOIUrl":"10.1007/s40273-025-01472-5","url":null,"abstract":"<p><p>In 2024, the National Institute for Health and Care Excellence (NICE) recommended two new health technologies for paroxysmal nocturnal haemoglobinuria. This review systematically compares the clinical and cost-effectiveness evidence considered within the NICE single technology appraisals of iptacopan, danicopan and pegcetacoplan, examines the consistency of the clinical evidence and economic modelling, and considers whether single technology appraisals are a suitable apparatus for consistent decision making. The studies used different follow-up lengths and used different definitions for reporting breakthrough haemolysis (BTH), but otherwise reported similar outcomes and found a significant benefit for their interventions. A lack of direct evidence and unreliable indirect comparisons meant that naïve comparisons across trials were carried into the economic modelling despite differences in their control arms. Approaches to modelling BTH and associated dose escalation differed across appraisals, despite information for pegcetacoplan coming from the same source in each appraisal, which had a large impact on the economic results. This review raises the question of whether NICE should implement multiple technology appraisals more frequently to reduce these inconsistences. Additionally, we recommend the development of a framework for revisiting positive recommendations when the implementation of health technologies deviates from assumptions made in the economic modelling to ensure cost-effective healthcare is preserved.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"499-508"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011644/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Prognostic Testing for Prostate Cancer-A Cost-Effectiveness Analysis Comparing a Prostatype P-Score Biomarker Approach to Standard Clinical Practice. 前列腺癌的预后检测:比较前列腺型P-Score生物标志物方法与标准临床实践的成本-效果分析
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-05-01 Epub Date: 2025-01-11 DOI: 10.1007/s40273-024-01466-9
Adam Fridhammar, Oskar Frisell, Karin Wahlberg, Emelie Berglund, Pontus Röbeck, Sofie Persson
{"title":"Prognostic Testing for Prostate Cancer-A Cost-Effectiveness Analysis Comparing a Prostatype P-Score Biomarker Approach to Standard Clinical Practice.","authors":"Adam Fridhammar, Oskar Frisell, Karin Wahlberg, Emelie Berglund, Pontus Röbeck, Sofie Persson","doi":"10.1007/s40273-024-01466-9","DOIUrl":"10.1007/s40273-024-01466-9","url":null,"abstract":"<p><strong>Background: </strong>The Prostatype score (P-score) is a prognostic biomarker that integrates a three-gene (IGFBP3, F3, and VGLL3) signature derived from prostate biopsy samples, with key clinical parameters, including prostate-specific antigen (PSA) levels, Gleason grade, and tumor stage at diagnosis. The test has demonstrated superior predictive accuracy for prostate cancer outcomes compared with traditional risk categorization systems such as D'Amico. Notably, it reclassifies a higher proportion of patients into the low-risk category, making them eligible for active surveillance. This study assessed the cost-effectiveness of the P-score in comparison with D'Amico and the Swedish National Prostate Cancer Register (NPCR) risk categorization systems.</p><p><strong>Methods: </strong>A two-step decision analytic model was developed. The model consisted of a decision tree-informed Markov structure estimating the lifetime outcomes of 60-year-old men with diagnosed prostate cancer. Prostate cancer was classified as low-risk, intermediate-risk, or high-risk using either the P-score or D'Amico. Initial therapy was based on observed treatment patterns from the Swedish NPCR. Costs (SEK, year 2022) and quality-adjusted life years (QALYs) were estimated from a healthcare perspective and discounted at 3% per year; incremental cost-effectiveness ratio (ICER) was the primary outcome.</p><p><strong>Results: </strong>The P-score led to cost savings and generated an additional 0.19 QALYs compared with D'Amico. The added costs of the genetic test and higher costs of active surveillance and radiotherapy were counterbalanced by savings from reduced costs of surgery, treatment-related side-effects, and metastatic disease. The gain in QALYs was primarily due to the avoidance of metastatic disease and a reduction in treatment-related side-effects.</p><p><strong>Conclusions: </strong>The results of this study suggest that the P-score is likely to be a cost-effective alternative to D'Amico for prognostic evaluation of newly diagnosed prostate cancer in Sweden and compared with NPCR when health-related quality of life was included.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"509-520"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011948/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966116","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Value of Innovative Multiple Myeloma Treatments from Patient and Healthcare Provider Perspectives: Evidence from a Discrete Choice Experiment. 从患者和医疗保健提供者的角度来看,创新多发性骨髓瘤治疗的价值:来自离散选择实验的证据。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-04-01 Epub Date: 2024-12-06 DOI: 10.1007/s40273-024-01459-8
Sakil Syeed, Chia Jie Tan, Amandeep Godara, Kyna Gooden, Derek Tang, Samantha Slaff, Yu-Hsuan Shih, Surachat Ngorsuraches, Nathorn Chaiyakunapruk
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