PharmacoEconomics最新文献

{"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}

{"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}

{"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}

{"title":"Value of Innovative Multiple Myeloma Treatments from Patient and Healthcare Provider Perspectives: Evidence from a Discrete Choice Experiment.","authors":"Sakil Syeed, Chia Jie Tan, Amandeep Godara, Kyna Gooden, Derek Tang, Samantha Slaff, Yu-Hsuan Shih, Surachat Ngorsuraches, Nathorn Chaiyakunapruk","doi":"10.1007/s40273-024-01459-8","DOIUrl":"10.1007/s40273-024-01459-8","url":null,"abstract":"<p><strong>Background: </strong>Although innovation generally provides measurable improvements in disease characteristics and patient survival, some benefits can remain unclear. This study aimed to investigate patient and healthcare provider (HCP) preferences for the innovative attributes of multiple myeloma (MM) treatments.</p><p><strong>Methods: </strong>A cross-sectional, web-based, discrete choice experiment (DCE) survey was conducted among 200 patients with MM and 30 HCPs of patients with MM in the USA. A literature review, followed by interviews with patients with MM and HCPs, was undertaken to select five attributes (progression-free survival [PFS], chance of severe side effects, how patients live with MM treatments, scientific innovation, and monthly out-of-pocket [OOP] cost) and their levels. A Bayesian efficient design was used to generate DCE choice sets. Each choice set comprised two hypothetical MM treatment alternatives described by the selected attributes and their levels. Each patient and HCP was asked to choose a preferred alternative from each of the 11 choice sets. Mixed logit and latent class models were developed to estimate patient and HCP preferences for the treatment attributes.</p><p><strong>Results: </strong>Overall, patients and HCPs preferred increased PFS, less chance of severe side effects, a treatment that offered life without treatment, scientific innovation, and lower OOP cost. From patients' perspectives, PFS had the highest conditional relative importance (44.7%), followed by how patients live with MM treatments (21.6%) and scientific innovation (16.0%).</p><p><strong>Conclusions: </strong>In addition to PFS, patients and HCPs also valued innovative MM treatments that allowed them to live without treatments and/or offered scientific innovation. These attributes should be considered when evaluating MM treatments.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"403-414"},"PeriodicalIF":4.4,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11929691/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142792203","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":"Home Urine Dipstick Screening for Bladder and Kidney Cancer in High-Risk Populations in England: A Microsimulation Study of Long-Term Impact and Cost-Effectiveness.","authors":"Olena Mandrik, Chloe Thomas, Edifofon Akpan, James W F Catto, Jim Chilcott","doi":"10.1007/s40273-024-01463-y","DOIUrl":"10.1007/s40273-024-01463-y","url":null,"abstract":"<p><strong>Background: </strong>Testing high-risk populations for non-visible haematuria may enable earlier detection of bladder cancer, potentially decreasing mortality. This research aimed to assess the cost-effectiveness of urine dipstick screening for bladder cancer in high-risk populations in England.</p><p><strong>Methods: </strong> A microsimulation model developed in R software was calibrated to national incidence data by age, sex and stage, and validated against mortality data. Individual risk factors included age, sex, smoking status and factory employment. We evaluated three one-time screening scenarios: (1) current and former smokers of different ages within the 55-70 years range, (2) a mixed-age cohort of smokers aged 55-80 years and (3) individuals aged 65-79 years from high-risk regions. Probabilistic and scenario analyses evaluated uncertainty. The incremental cost-effectiveness ratio (ICER) was calculated and compared with the standard £20,000/quality-adjusted life year (QALY) threshold using payer's perspective and 2022 year of evaluation with 3.5% discounting for both costs and effects.</p><p><strong>Results: </strong> Screening all current and former smokers (scenario 1) and both mixed-age cohorts (scenarios 2 and 3) was not cost-effective at the threshold of £20,000/QALY. Screening at age 58 years had a 33% probability of being cost-effective at £20,000/QALY threshold and a 64% probability at £30,000/QALY threshold. Screening current and former smoking men aged 58 and 60 years was cost-effective, with ICERs of £18,181 and £18,425 per QALY, respectively. Scenario results demonstrated the high impact of assumptions on lead time, diagnostic pathway, and screening efficacy on predictions.</p><p><strong>Conclusions: </strong> Screening smoking men aged 58 or 60 years for bladder cancer using urine dipstick tests may be cost-effective.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"441-452"},"PeriodicalIF":4.4,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142927656","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 Using Cost-effectiveness Analysis to Support Pricing and Reimbursement Decisions for New Pharmaceuticals in a Context of Evolving Treatments, Prices, and Evidence.","authors":"Beth Woods, Alfredo Palacios, Mark Sculpher","doi":"10.1007/s40273-024-01450-3","DOIUrl":"10.1007/s40273-024-01450-3","url":null,"abstract":"<p><p>Current approaches to the pricing and funding of new pharmaceuticals often focus on a one-time decision about a product for each clinical indication. This can result in multiple options being available to health systems without a clear signal about how to prioritise between them. This runs the risk that, as available treatments, evidence, and drug prices evolve, clinical and patient choices may not be aligned with the objective of allocating resources to promote population health. We propose a framework for using cost-effectiveness analysis to support pricing and funding policies for new pharmaceuticals in multi-comparator indications, some of the key aspects of which evolve over time. The framework comprises three core considerations: (1) designing proportionate processes, (2) assessing the costs and benefits of recommending multiple treatment options, and (3) appropriate application of cost-effectiveness analysis 'decision rules' to support recommendations and price negotiations. We highlight that proportionate processes require prioritisation of topics for reassessment to be aligned with clear objectives, the need for full flexibility of decision making at the point of reassessment, and that in some contexts contractual re-specification rather than typical deliberative health technology assessment processes may be more appropriate. We discuss reasons why the recommendation of multiple treatment options rather than a single cost-effective treatment may be appropriate and urge health technology assessment bodies to explicitly address the trade-offs that may be associated with recommending multiple treatments. Finally, we discuss how value-based pricing could be achieved when multiple competitor manufacturers offer confidential discounts.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"363-373"},"PeriodicalIF":4.4,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12227514/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142910132","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":"Examination of Methods to Estimate Productivity Losses in an Economic Evaluation: Using Foodborne Illness as a Case Study.","authors":"Kathleen Manipis, Paula Cronin, Deborah Street, Jody Church, Rosalie Viney, Stephen Goodall","doi":"10.1007/s40273-024-01458-9","DOIUrl":"10.1007/s40273-024-01458-9","url":null,"abstract":"<p><strong>Background: </strong>Cost-utility analyses commonly use two primary methods to value productivity: the human capital approach (HCA) and the friction cost approach (FCA). Another less frequently used method is the willingness-to-pay (WTP) approach, which estimates the monetary value individuals assign to avoiding an illness. In the context of foodborne illnesses (FBI), productivity loss represents one of the most significant economic impacts, particularly in developed nations. These losses arise from factors such as missed workdays, reduced workplace efficiency due to illness, and long-term health complications that can limit an individual's ability to work. As a result, accurately quantifying productivity loss is critical in understanding the broader economic burden of FBI.</p><p><strong>Aim: </strong>Our aim was to compare the impact of valuation methods used to measure productivity loss in an economic evaluation, using a hypothetical intervention for FBI caused by campylobacter as a case study. Cost effectiveness from three perspectives is examined: health care system, employee, and employer.</p><p><strong>Method: </strong>A Markov model with a 10-year time horizon was developed to evaluate the morbidity and productivity impacts of FBI caused by campylobacter. The model included four health states: 'healthy', 'acute gastroenteritis', 'irritable bowel syndrome and being unable to work some of the time', and 'irritable bowel syndrome and unable to work'. Five approaches to valuing productivity loss were compared: model 1 (cost-utility analysis), model 2 (HCA), model 3 (FCA), model 4 (FCA+WTP to avoid illness with paid sick leave), and model 5 (WTP to avoid illness without paid sick leave). Health outcomes and costs were discounted using a 5% discount rate. Costs were reported in 2024 Australian dollars ($AUD).</p><p><strong>Results: </strong>Model 1, which did not include productivity losses, yielded the highest incremental cost-effectiveness ratio (ICER) at $56,467 per quality-adjusted life-year (QALY) gained. The inclusion of productivity costs (models 2-5) significantly increased the total costs in both arms of the models but led to a marked reduction in the ICERs. For example, model 2 (HCA) resulted in an ICER of $11,174/QALY gained, whereas model 3 (FCA) resulted in $21,136/QALY gained. Models 4 and 5, which included WTP approaches, had ICERs of $19,661/QALY gained and $24,773/QALY gained, respectively.</p><p><strong>Conclusion: </strong>These findings underscore the significant impact of different modelling approaches to productivity loss on ICER estimates and consequently the decision to adopt a new policy or intervention. The choice of perspective in the analysis is critical, as it determines how the short-term and long-term productivity losses are accounted for and valued. This highlights the importance of carefully selecting and justifying the perspective and valuation methods used in economic evaluations to ensure informed a","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"453-467"},"PeriodicalIF":4.4,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142927650","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":"Projections of Public Spending on Pharmaceuticals: A Review of Methods.","authors":"Irina Odnoletkova, Patrice X Chalon, Stephan Devriese, Irina Cleemput","doi":"10.1007/s40273-024-01465-w","DOIUrl":"10.1007/s40273-024-01465-w","url":null,"abstract":"<p><strong>Background: </strong>Forecasting future public pharmaceutical expenditure is a challenge for healthcare payers, particularly owing to the unpredictability of new market introductions and their economic impact. No best-practice forecasting methods have been established so far. The literature distinguishes between the top-down approach, based on historical trends, and the bottom-up approach, using a combination of historical and horizon scanning data. The objective of this review is to describe the methods for projections of pharmaceutical expenditure that apply the \"bottom-up\" approach and to synthesize the knowledge of their predictive accuracy.</p><p><strong>Methods: </strong>Projections of public pharmaceutical expenditure applicable to Western economies including a comprehensive method description and published 2000-2024 were searched in scientific databases (MEDLINE, EMBASE, and EconLit) and in gray literature (websites of international health organizations and national healthcare authorities). The data sources, assumptions about the future market dynamics, analytical approaches, and the projection results are summarized.</p><p><strong>Results: </strong>Twenty-four out of 3492 screened publications were included, associated with nine expenditure projection models. Four models were developed for all reimbursable drugs in the USA, the UK, the Stockholm region (Sweden), and seven European Union (EU) countries: France, Germany, Greece, Hungary, Poland, Portugal, and the UK, respectively. The other five models concerned specific groups of medicines: orphan drugs in Belgium, the Eurozone plus the UK, and Canada, respectively; psychotropic medications in the USA; and outpatient intravenous cancer medicines in the Province of Ontario (Canada). For trend analysis, drug coverage claims or sales data were used, applying linear and/or nonlinear models. The budget impact of new launches and patent expirations was estimated through (a form of) horizon scanning, i.e., a systematic monitoring of the pharmaceutical pipeline, with engagement of clinical expert judgment. Projections with a predictive time window greater than 3 years largely relied on previously observed trends to model new market introductions. Four models were validated through an ex post comparison of projected and observed expenditure. The absolute difference between the forecasted and actual percentual change in pharmaceutical expenditure was: 0.3% (\"UK model\"), 1.9% (\"Stockholm model\"), and 2% (nonfederal hospitals, \"US model\"). The \"Ontario cancer drug model\" overestimated the actual expenditure by 1%. Overall, the largest errors were attributable to new market launches and unforeseen policy reforms. Prediction accuracy decreased substantially for forecasts beyond 1 year in the future. For two not validated projections, a face validity check was feasible. One of the models forecasted a decrease in pharmaceutical expenditure from 2012 to 2016 in six European countries, contrasti","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"375-388"},"PeriodicalIF":4.6,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11929675/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966121","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":"Cost Effectiveness of Exclusionary EGFR Testing for Taiwanese Patients Newly Diagnosed with Advanced Lung Adenocarcinoma.","authors":"Huang-Tz Ou, Jui-Hung Tsai, Yi-Lin Chen, Tzu-I Wu, Li-Jun Chen, Szu-Chun Yang","doi":"10.1007/s40273-024-01462-z","DOIUrl":"10.1007/s40273-024-01462-z","url":null,"abstract":"<p><strong>Background and objective: </strong>Approximately half of lung adenocarcinomas in East Asia harbor epidermal growth factor receptor (EGFR) mutations. EGFR testing followed by tissue-based next-generation sequencing (NGS), upfront tissue-based NGS, and complementary NGS approaches have emerged on the front line to guide personalized therapy. We study the cost effectiveness of exclusionary EGFR testing for Taiwanese patients newly diagnosed with advanced lung adenocarcinoma.</p><p><strong>Methods: </strong>This economic evaluation was conducted from the perspective of the healthcare sector with a lifetime horizon. Simulated patients were entered into a joint model combining decision trees and partitioned survival models upon diagnosis of advanced lung adenocarcinoma. We compared exclusionary EGFR testing with upfront tissue-based NGS and complementary NGS approaches. The model inputs were derived from regional estimates (prevalence of targetable gene alterations), trials (testing accuracy, survival outcomes, and adverse events), ACT Genomics (testing costs), National Health Insurance payments, retail prices (drug costs), and hospital cohorts (utility values). All costs were made equivalent to 2023 US dollars. An annual discount rate of 3% was applied. We adopted a willingness-to-pay threshold of US$70,000 per quality-adjusted life-year. One-way deterministic and probabilistic analyses were performed.</p><p><strong>Results: </strong>The incremental cost-effectiveness ratio of exclusionary EGFR testing versus upfront tissue-based NGS was US$15,521 per quality-adjusted life-year, whereas the incremental net monetary benefit was US$2530. The costs of osimertinib and pembrolizumab were the major determinants. The incremental net monetary benefit of exclusionary EGFR testing versus complementary NGS approach was US$2174, and its major determinants included the true-negative rate of EGFR testing and the prevalence rate of an EGFR mutation. Given the willingness-to-pay thresholds of US$35,000, US$70,000, and US$105,000 (1, 2, and 3 per capita gross domestic product) per quality-adjusted life-year, the probabilities that exclusionary EGFR testing would be cost effective were 79.1%, 95.6%, and 91.2%, respectively.</p><p><strong>Conclusions: </strong>Our analysis suggests that exclusionary EGFR testing is a cost-effective strategy for Taiwanese patients newly diagnosed with advanced lung adenocarcinoma.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"429-440"},"PeriodicalIF":4.4,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142922433","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}