PharmacoEconomics最新文献

{"title":"Cost-Effectiveness of a Digital Leakage Notification System (Heylo™) for People with Ileostomies or Colostomies in the United Kingdom.","authors":"Esben Bo Boisen, Matthew Cawson, Lasse de Fries Jensen, Stuart Mealing, Naomi van Hest","doi":"10.1007/s40273-025-01498-9","DOIUrl":"https://doi.org/10.1007/s40273-025-01498-9","url":null,"abstract":"<p><strong>Background: </strong>People with stomas report a substantial negative impact of stomal effluent leaking outside the ostomy baseplate and subsequent complications in their professional and social lives, causing immense worry. However, many patients are not able to recognize leakages in a timely manner. We conducted a cost-effectiveness study to evaluate the impact of a digital leakage notification system (DLNS) to reduce leakages outside the baseplate (LOB) and worry about leakage for people with intestinal stomas from a UK National Health Service and Personal Social Services perspective.</p><p><strong>Methods: </strong>A Markov model for ostomy care was used to compare health-related quality of life and costs for adults with ileostomies or colostomies using UK standard of care ostomy products with the DLNS (intervention) or without the DLNS (comparator). The base case model used a 3-year time horizon with 1-week cycles and an annual 3.5% discounting of utilities and costs. Patients in all health states experience LOB events and/or worry about leakage as events associated with one-time utility decrements and costs of additional healthcare provider visits and ostomy product use. Probabilities of LOB (DLNS, 46.5%; comparator, 78.6%) and worry about leakage (DLNS, 39.1%; comparator, 78.6%) were based on clinical trial results. Peristomal skin complications were included in the model with the same probabilities of occurrence in the intervention and comparator arms. Sensitivity and scenario analyses were performed to test the robustness of the base case model assumptions.</p><p><strong>Results: </strong>In the base case analysis, the DLNS arm had 49.81 fewer LOB events per person than the comparator arm, resulting in 0.309 incremental quality-adjusted life-years (QALYs) and cost savings of British Pound Sterling (GBP) £1703 per person over 3 years (2023/2024 costing year). The DLNS arm had 56.98 fewer worry about leakage events per person, resulting in cost savings of £403 per person. Total costs and QALYs were £18,600 and 1.818 for the intervention arm, respectively, and £18,566 and 1.509 for the comparator arm. Overall, the DLNS provided 0.309 incremental QALYs at an incremental cost of £34 versus the comparator arm for an incremental cost-effectiveness ratio of £110/QALY gained, well below a willingness-to-pay threshold of £20,000/QALY. Sensitivity analyses showed the DLNS was cost-effective in 97.6% of simulations.</p><p><strong>Conclusions: </strong>This analysis suggests that the DLNS added to standard of care ostomy products is a cost-effective intervention to help prevent LOB events and reduce worry about leakage for people with stomas in the UK. Results of the present study suggest that timely awareness of leakage has a positive impact on the health-related quality of life of people with stomas and on the direct costs of stoma management in the UK.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143986686","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}

{"title":"A Framework for Reliable, Transparent, and Reproducible Population-Adjusted Indirect Comparisons.","authors":"K Jack Ishak, Conor Chandler, Fei Fei Liu, Sven Klijn","doi":"10.1007/s40273-025-01503-1","DOIUrl":"https://doi.org/10.1007/s40273-025-01503-1","url":null,"abstract":"<p><p>Population-adjusted indirect comparison (PAIC) methods aim to address some of the potential shortcomings of conventional approaches to indirect treatment comparisons by adjusting for imbalances in effect modifiers or prognostic factors and allowing for unanchored indirect treatment comparisons from disconnected networks of evidence. Health technology assessment bodies have published guidance and best practice recommendations for PAICs. However, recently published reviews of published PAICs have highlighted notable variability in implementation and a lack of transparency in the decision-making process in analyses and reporting; this hinders the interpretation and reproducibility of analyses, which, in turn, could affect reimbursement decision-making. We propose a systematic framework to address these challenges by describing considerations on six key elements of analyses: (1) definition of the comparison of interest (e.g., in terms of an estimand), (2) selection of the PAIC method, (3) selection of adjustment variables, (4) application of adjustment method, (5) risk-of-bias assessment, and (6) comprehensive reporting.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015053","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}

{"title":"Cost-Effectiveness Analysis of Nirsevimab for Preventing Respiratory Syncytial Virus-Related Lower Respiratory Tract Disease in Dutch Infants: An Analysis Including All-Infant Protection.","authors":"Florian Zeevat, Simon van der Pol, Alexia Kieffer, Maarten J Postma, Cornelis Boersma","doi":"10.1007/s40273-025-01469-0","DOIUrl":"10.1007/s40273-025-01469-0","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to assess the cost effectiveness of nirsevimab, a recently authorized monoclonal antibody (mAb) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV), in comparison with the standard practice involving palivizumab for high-risk infants during their first RSV season in the Netherlands.</p><p><strong>Methods: </strong>A static cost-effectiveness model was populated for the Netherlands to evaluate different immunization strategies for nirsevimab over a single RSV season from a societal perspective. The model considered the most recently published RSV incidence data (average incidence from 2006 to2018), costs (adjusted to the 2023 price year), and associated health effects. Extensive scenario analyses were conducted to explore various strategies, and sensitivity analysis was performed to assess the model's robustness.</p><p><strong>Results: </strong>In the base-case scenario, all-infant protection-a strategy of in-season with catch-up immunization for all infants-nirsevimab has the potential to prevent numerous RSV-related cases, including 2333 hospitalizations and 150 intensive-care admissions, in the overall population compared with the standard of care. Nirsevimab appears to be cost effective under this strategy with an economically justifiable acquisition price for nirsevimab of €220 at a willingness-to-pay threshold of €50,000 per quality-adjusted life-year. Sensitivity analyses indicate a 52% probability that nirsevimab is cost effective at this threshold. Comparison of different vaccination strategies revealed that the all-infant protection approach was the one that prevented the higher number of cases.</p><p><strong>Conclusions: </strong>This study indicates that universal infant immunization with nirsevimab has the potential to be cost effective and significantly reduces the burden of RSV among Dutch infants. These findings underscore the importance of implementing effective protective measures against RSV-LRTD, reducing the pressure on the healthcare system during the RSV season.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"569-582"},"PeriodicalIF":4.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12011648/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143458849","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}

{"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}

{"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}

{"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}

{"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}

{"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}

{"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}

{"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}