PharmacoEconomics Open最新文献

{"title":"The Economic and Clinical Impact of Recurring Automated Red Blood Cell Exchange to Manage Sickle Cell Disease in the UK.","authors":"Sarah M Medland, Jamie Bainbridge, Matthew Cawson, Stuart J Mealing, Anna Yudina, Isabel Eastwood, Arne de Kreuk, Martin Besser, Eva Tsouana","doi":"10.1007/s41669-025-00605-y","DOIUrl":"https://doi.org/10.1007/s41669-025-00605-y","url":null,"abstract":"<p><strong>Background/objective: </strong>Sickle cell disease (SCD) is a group of inherited health conditions affecting 7.74 million people worldwide. Regular automated red blood cell exchange (aRBCX) transfusions have been shown to improve control and management of SCD compared with manual RBCX (mRBCX). The aim of this study was to estimate the lifetime clinical and economic impact of aRBCX versus mRBCX in two United Kingdom-based populations with SCD (paediatrics initiated aged 5 years and adults initiated aged 38 years) that were clinically indicated for chronic disease-modifying transfusions (DMTs).</p><p><strong>Methods: </strong>An individual patient-level simulation model was developed to estimate lifetime quality-adjusted life years (QALYs) and healthcare costs. DMT administration programmes aligned with recommended treatment schedules. Monte Carlo methods determined baseline characteristics and clinical event occurrence. Pragmatic review findings and expert opinion informed model parameters and assumptions. Second-order probabilistic sensitivity analysis (PSA) was performed for 1000 individuals' lifetimes over 500 iterations.</p><p><strong>Results: </strong>Per individual, aRBCX reduced acute clinical events by 19% in both populations versus mRBCX. The time spent receiving chelation therapy reduced by 63 and 32 months for paediatric-initiated and adult-initiated individuals, respectively. Total lifetime DMT costs were reduced by £71,217 and £30,740 for paediatric-initiated and adult-initiated individuals, respectively. Overall, aRBCX increased QALYs and reduced costs by 0.29 and £112,811 in paediatric-initiated individuals and 0.24 and £61,895 in adult-initiated individuals. aRBCX was cost-effective in 100% of PSA iterations for both populations.</p><p><strong>Conclusion: </strong>aRBCX shows potential to improve health outcomes and reduce healthcare costs for individuals with SCD initiating a chronic DMT programme.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145252282","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimating the Value of Adding 30 Days to the 180-Day Market Exclusivity of the First-to-File Generic Drug Manufacturer.","authors":"Aylin Sertkaya, Zeid El-Kilani, Sean Klein, Andreas Lord, Ruben Jacobo-Rubio, Sonal Parasrampuria","doi":"10.1007/s41669-025-00607-w","DOIUrl":"https://doi.org/10.1007/s41669-025-00607-w","url":null,"abstract":"<p><strong>Objectives: </strong>As an additional incentive to bringing more generic drugs to more markets sooner, our aim was to estimate the value of adding 30 days to the 180 days of market exclusivity currently granted to a qualifying first-to-file (FTF) generic drug entrant after its manufacturer successfully challenges a brand drug's patents.</p><p><strong>Methods: </strong>Using IQVIA's monthly data on drug sales in the U.S. from January 2014 through June 2021, we identified 37 generic drug markets in which one or more generic companies successfully challenged the relevant brand drug patents and began commercial marketing within 75 days of receiving final approval of their Abbreviated New Drug Application (ANDA). We compared each generic drug's sales during the last month of its 180-day exclusive market access (Month 6) with its sales during the first month after their exclusivity expired (Month 7). Our calculation of this change in sales accounted for confounding factors such as the presence of an authorized generic in the market during the exclusivity period and the number of new generic entrants in Month 7. This calculated change represents the value to the FTF manufacturer of extending the 180-day market exclusivity to 210 days.</p><p><strong>Results: </strong>Our analysis shows that an FTF generic experiences a 13.0% reduction in sales from Month 6 to Month 7, on average. Extending the 180-day exclusivity by 30 days could lower the magnitude of this reduction to 3.1%, resulting in a 9.8% gain in sales by the FTF generic. This gain represents about $870,000 in additional sales during Month 7, on average.</p><p><strong>Conclusions: </strong>The value of an additional month of exclusivity to a generic firm is sizable and could entice more generic companies on the margin to take on the risk of patent challenges, especially in large markets.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145150472","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Budget Impact Model of Treatment of Primary Immune Deficiency with Immune Globulin Subcutaneous (Human), 20% (IgPro20) (Hizentra).","authors":"Rajiv Mallick, Rashad Carlton","doi":"10.1007/s41669-025-00594-y","DOIUrl":"https://doi.org/10.1007/s41669-025-00594-y","url":null,"abstract":"<p><strong>Background: </strong>Primary immune deficiency (PID) comprises over 400 inborn errors of immunity. Immune globulin replacement therapy (IGRT) is the standard of care for many PID subtypes. Intravenous immunoglobulin (IVIG) is the historic standard of care, but subcutaneous immunoglobulin (SCIG) offers several advantages for IGRT. Further, a novel pre-filled syringe (PFS) drug packaging form of SCIG is expected to facilitate more efficient patient infusions.</p><p><strong>Objective: </strong>The aim of this study was to quantify the projected budget impact associated with adding the IgPro20 PFS as an SCIG drug packaging option to a health plan formulary for the treatment of PID.</p><p><strong>Methods: </strong>A budget impact model (BIM) was developed across the United States (US) integrated delivery networks (IDNs)' perspective over a 3-year time horizon to quantify the projected budget impact of adding the IgPro20 PFS as an SCIG packaging option for the treatment of PID. Model comparators included six IVIGs and seven SCIGs. A one-way sensitivity analysis was conducted to assess the impact of model parameters on the budget impact. Scenario analyses of alternative market share shifts and pricing were conducted to test the robustness of the model results.</p><p><strong>Results: </strong>From the perspective of an IDN with 25 million members, the average annual expected cost per PID patient was US$73,343 for IgPro20 vials w/pump, US$60,892 for IgPro20 PFS w/pump, US$72,179 for IVIG, and US$96,581 for other SCIGs. Assuming market share uptake of 6.0% in the first year for IgPro20 PFS w/pump, rising to 8.4% by year 3, and offset by corresponding proportionate reductions in shares of the non-PFS SCIG IgPro20 vials w/pump only, the expected incremental budget impact over the 3-year horizon was savings of US$10.5 million.</p><p><strong>Conclusion: </strong>This BIM demonstrates that uptake of subcutaneous IgPro20 PFS is expected to be associated with substantial savings for a US IDN, including drug cost savings if offset by reduced non-PFS SCIG-packaging use.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145081168","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost Effectiveness of Recombinant Zoster Vaccine and Live-Attenuated Vaccine Against Herpes Zoster for Adults Aged 50 and Over in China.","authors":"Lulu Liu, Qilin Zhang, Yamin Shu, Lei Wang, Zhenqingyun Shuai, Mingming Chu, Zhe Zhang, Rong Zhang","doi":"10.1007/s41669-025-00604-z","DOIUrl":"https://doi.org/10.1007/s41669-025-00604-z","url":null,"abstract":"<p><strong>Objective: </strong>Two vaccines against herpes zoster (HZ) are currently authorized for use in China: the adjuvanted recombinant zoster vaccine (RZV) and live-attenuated Zoster Vaccine Live (ZVL). The significant disparities in prices and efficacy between the two vaccines necessitate an evaluation of their relative value in order to make an informed choice. This study aimed to evaluate the comparative cost effectiveness of RZV, ZVL, and no vaccination for older adults at different ages from the societal perspective.</p><p><strong>Methods: </strong>The Markov decision model was parameterized using vaccine efficacy data, the latest incidence rates, costs, and health-related quality-of-life data. Participants consisted of a cohort comprising adults aged ≥ 50 years in China. The lifetime health impacts, cost, life-year (LY), quality-adjusted life-year (QALY) gained and the incremental cost-effectiveness ratio (ICER), net monetary benefits (NMB), net health benefits (NHB), and expected value of perfect information (EVPI) were calculated. Sensitivity analyses, scenario analyses, and subgroup analyses were performed.</p><p><strong>Results: </strong>Compared with no vaccination, RZV and ZVL could avoid 2,009,292 versus 510,192 HZ cases, and 334,637 versus 90,996 post-herpetic neuralgia (PHN) cases in the overall cohort, respectively. RZV and ZVL were more costly by US$1624 million versus US$896 million compared with no vaccination, with an additional 58,180 versus 23,390 QALYs. At threefold gross domestic product per capita, RZV had cost-effectiveness probabilities of 47.37% for ages 50-59 years, 83.24% for ages 60-69 years, 28.28% for ages 70-79 years, and 51.93% for ages ≥ 80 years, while ZVL had 7.07%, 12.79%, 69.55%, 2.82% probability, and no vaccination had 45.56%, 3.97%, 2.17%, 45.25% probability of being cost effective for the corresponding age cohorts.</p><p><strong>Conclusions: </strong>The strategy utilizing RZV demonstrated superior efficacy in reducing the burden of HZ compared with ZVL. RZV was most cost effective in the 50-59, 60-69 and ≥ 80 years age groups, and ZVL was most cost effective in the 70-79 years group.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145023822","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimating Health State Utilities for IDH-Mutant Diffuse Glioma.","authors":"Ellen M Howard, Marc Massetti, Gin Nie Chua, Millie Gaydon, Sara Savar, Andrew Lloyd","doi":"10.1007/s41669-025-00603-0","DOIUrl":"https://doi.org/10.1007/s41669-025-00603-0","url":null,"abstract":"<p><strong>Background: </strong>Isocitrate dehydrogenase-mutant (mIDH) gliomas are malignant central nervous system tumours. After initial resection, patients with mIDH gliomas with favourable prognosis may live without receiving oncologic treatment for years, but ultimately patients will experience recurrence and require radio- and/or chemotherapy (RT/CT). Cost-utility analyses (CUA) can explore the value of treatments that delay recurrence and initiation of RT/CT. This study aimed to estimate health state utilities for mIDH glioma over the full disease course.</p><p><strong>Method: </strong>Vignettes were developed to describe states from post-initial resection with no immediate need for RT/CT through to RT/CT and palliative care after recurrence. These were developed on the basis of literature review, patient-reported outcomes data from a clinical trial and qualitative interviews with clinicians and patients. The health state vignettes were valued by members of the UK public using the EQ-5D-5L and time trade-off methods. Subsequently, the vignette-derived utilities were re-weighted with the utility data from a clinical trial to estimate health state utilities for CUA.</p><p><strong>Results: </strong>Health state utilities were similar following the initial resection for targeted therapy or active observation (0.74) but lower in later stages of disease. Mean utilities for receiving first- and second-line RT/CT ranged from 0.34 to 0.55. Mean utilities ranged from 0.21 to 0.42 for recovery after tumour resection following recurrence and from -0.05 to 0.14 for palliative care.</p><p><strong>Conclusion: </strong>This study estimates health state utilities in glioma from after the initial resection through to palliative care and suggests that new treatments capable of delaying time to disease recurrence and initiation of RT/CT could produce a net gain in quality of life for individuals with mIDH glioma.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145008317","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Individual Preference for Fecal Immunochemical Test Options among Younger Adults: A Discrete Choice Experiment.","authors":"Kailu Wang, Ho-Man Shum, Carrie Ho-Kwan Yam, Shangfeng Tang, Chunyan Li, Yushan Wu, Eliza Lai-Yi Wong, Eng-Kiong Yeoh","doi":"10.1007/s41669-025-00598-8","DOIUrl":"https://doi.org/10.1007/s41669-025-00598-8","url":null,"abstract":"<p><strong>Objectives: </strong>This study aims to quantify the preference of adults below 50 years of age for fecal immunochemical test (FIT) options as screening for colorectal cancer (CRC) and its disparities across different subgroups.</p><p><strong>Methods: </strong>A discrete choice experiment (DCE) was conducted among adults aged < 50 years old in Hong Kong. A mixed logit model and latent class model were used to estimate their preference, taking into account their preference heterogeneity. Marginal willingness-to-pay for changes in the attribute levels was also estimated.</p><p><strong>Results: </strong>A total of 408 participants with valid responses were included for analysis. The outcomes showed 5.2% of participants would consistently reject all the 20 FIT alternatives presented to them in the DCE survey. Mortality reduction in FIT programs was deemed as the most important attribute, followed by testing frequency, early detection likelihood, familiar physician, and reduction of false positive rate. The FIT acceptance was sensitive to the change of out-of-pocket payment amount. The latent class model showed participants with different ages, educational level, and self-reported health status would have different preferences for FIT attribute levels.</p><p><strong>Conclusions: </strong>The study showed that most adults below 50 years old would be willing to consider receiving FIT if suitable testing options are available. They are more sensitive to changes in mortality reduction than changes in CRC early detection likelihood, suggesting the former is more important in information dissemination. The impact of out-of-pocket payments for FIT on its acceptance suggests that the introduction of financial subsidies can be considered to improve screening acceptance. The preference heterogeneity highlights the importance of healthcare professionals' understanding of people's values and preferences for decision-making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992902","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An Enhanced Retention Strategy to Prevent the Vertical Transmission of HIV in Uganda: A Budget Impact Analysis.","authors":"Elly Nuwamanya, Benjamin C Johnson, Stephen Okoboi, Ronald Galiwango, Diana Namuddu, Tabitha Ayabo, Joseph B Babigumira, Mohammed Lamorde","doi":"10.1007/s41669-025-00587-x","DOIUrl":"10.1007/s41669-025-00587-x","url":null,"abstract":"<p><strong>Background: </strong>Novel retention strategies have the potential to reduce vertical transmission of HIV and improve patient outcomes for women living with HIV (WLHIV) and their infants. We estimated the budget impact of the enhanced retention strategy (ERS) compared with the Ministry of Health strategy/standard of care (SOC) approach for preventing vertical transmission of HIV among women initiating antiretroviral therapy (ART) in late pregnancy in Uganda.</p><p><strong>Methods: </strong>A budget impact analysis (BIA) was conducted from the payer (Uganda's Ministry of Health) perspective with a 5-year time horizon. A Microsoft Excel-based BIA model was populated with HIV epidemiological data and expenditures from the literature and the clinical trial of dolutegravir in pregnant HIV mothers and their neonates. These cost projections accounted for various programmatic inputs, disease progression, differences in mortality based on treatment status, and subsequent pregnancies. The eligible population included all HIV-positive pregnant women currently receiving prevention of vertical transmission services in Uganda. The primary outcomes of the analysis were incremental budget costs, and infections averted over 5 years.</p><p><strong>Results: </strong>Adopting the ERS would lead to a net cost increase of US$63.8 million over the next 5 years, or a net cost increase of US$12.7 million per year compared with the SOC. Newly enrolled WLHIV accounts for US$39.5 million of these marginal costs, while in-system patients account for US$24.2 million. Direct programmatic costs of the ERS only account for 13% of this additional cost, with 87% of the marginal increase coming from the cost of providing ART for WLHIV who would otherwise be lost to follow-up. The ERS would avert an additional 6933 infant infections compared with the SOC.</p><p><strong>Conclusion: </strong>Implementing the ERS would significantly increase the Ugandan Ministry of Health's budget, but most additional costs would be accrued from the resulting expansion of ART client volume. The ERS is a relatively low-cost intervention to reduce loss to follow-up rates among marginalized and hard-to-reach populations.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"815-825"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12401848/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248986","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Importance of a Methodological Manual for Economic Evaluations in Healthcare Decision-Making in Ecuador.","authors":"Ricardo Yajamín-Villamarín","doi":"10.1007/s41669-025-00585-z","DOIUrl":"10.1007/s41669-025-00585-z","url":null,"abstract":"<p><p>This article provides an overview of Ecuador's Methodological Manual for Economic Evaluations of Health Technologies, emphasizing its importance, key methodological aspects, and relevance within the country's health technology assessment (HTA) framework. The manual establishes standardized guidelines for conducting economic evaluations, incorporating international best practices while adapting them to the national context. It outlines essential methodological components, including decision problem definition, economic evaluation types, cost estimation, use of decision models, and sensitivity analysis. Additionally, it highlights the role of quality-adjusted life years (QALYs) and cost-effectiveness thresholds in supporting evidence-based decision-making. Beyond its methodological contributions, the manual plays a crucial role in institutionalizing economic evaluations in Ecuador's healthcare system. By promoting transparency, consistency, and technical rigor, it strengthens the integration of economic evidence into policy decisions, ensuring efficient resource allocation. However, challenges persist, including limitations in technical capacity, data availability, and the need for further refinement of local cost-effectiveness thresholds and indirect cost valuation. This article also explores the broader policy implications of adopting the manual within Ecuador's HTA framework. Strengthening institutional capacity, improving data infrastructure, and fostering international collaboration will be essential to its successful implementation. By embedding economic evaluations into healthcare decision-making, Ecuador can enhance the sustainability of its health system, optimize expenditures, and improve access to cost-effective health technologies.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"713-722"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12401847/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199803","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Missing Pieces of the Puzzle to Address Market Failures for Antibiotics: Delinked Payment Systems and Insurance Value.","authors":"Neil Hawkins, Adrian Towse, Amanda Adler","doi":"10.1007/s41669-025-00591-1","DOIUrl":"10.1007/s41669-025-00591-1","url":null,"abstract":"<p><p>Too few companies develop new antibiotics because of the threat of market failure. To address this, a 'delinked' payment, distinct from the usual payment model for drugs, makes payments to manufacturers that do not depend on the volume of antibiotic prescribed. A delinked system removes incentives to overpromote antibiotic use. If total payments are high enough, this system should provide incentives for manufacturers to develop new antibiotics. This assumes that using antibiotics remains consistent with antimicrobial stewardship, a coordinated approach to prescribing antimicrobials responsibly. A delinked system can address market failure that occurs when a disproportionate degree of clinical benefit from a new antibiotic occurs after patent protection ends, reducing the 'reward' to the innovator. Determining value in a delinked payment system requires that the health service estimates the lifetime value of an antimicrobial product, and then decides what proportion of that value to include. These values depend in part on 'STEDI' values including the 'insurance' value new antibiotics would offer in reducing society's risk of major health loss from possible future major episodes of antibiotic resistance. Estimating insurance value requires estimating the health consequences of catastrophic outcomes. Payments in a delinked system can incorporate an 'insurance premium'. We use the example of the UK's delinked payment scheme to illustrate issues and solutions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"707-712"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12401795/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144554130","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Economic Evaluation of Lecanemab for Early Symptomatic Alzheimer's Disease in South Korea.","authors":"Seungyeon Shin, Maryanne Kim, Song Hee Hong","doi":"10.1007/s41669-025-00593-z","DOIUrl":"10.1007/s41669-025-00593-z","url":null,"abstract":"<p><strong>Background/objectives: </strong>Alzheimer's disease (AD) exerts a considerable economic burden on South Korea's aging population. Lecanemab, an amyloid-targeting therapy, has demonstrated efficacy in mitigating cognitive decline in early-stage AD but remains non-reimbursed in South Korea due to concerns over its economic feasibility. This study aimed to examine the cost-effectiveness of lecanemab using nationwide claims data for cost estimation within the South Korean healthcare system. Considering the substantial societal burden of AD, we also evaluated the cost-effectiveness of lecanemab from a limited societal perspective.</p><p><strong>Methods: </strong>A Markov state transition cohort model was developed to compare costs and outcomes of lecanemab combined with standard of care (SoC) versus SoC alone. The model simulated five stages of AD progression: mild cognitive impairment, mild AD, moderate AD, severe AD, and death. Transition probabilities between health states were derived from data provided by the National Alzheimer's Coordinating Center. Formal medical costs and long-term care costs were obtained from the national claims database, while drug cost and other medical expenses were derived from previous studies. Additional cost components such as opportunity cost of caregiver time, out-of-pocket expenses, and time and travel costs for hospital visits were included in the limited societal perspective. Korean-specific utilities for patients and caregivers differentiated by states of AD progression and care settings were obtained from the published literature. Effectiveness was measured in quality-adjusted life years (QALYs) over a lifetime horizon. Scenario analyses were conducted by varying compositions of the cohort, age of onset, and drug pricing.</p><p><strong>Results: </strong>The incremental cost-effectiveness ratio (ICER) of lecanemab combined with SoC was 198,171,820 Korean Won (KRW)/QALY from the healthcare payer perspective and 181,185,820 KRW/QALY from the limited societal perspective, which significantly exceeded South Korea's willingness-to-pay (WTP) threshold of 30 million KRW/QALY. Sensitivity analyses revealed that the ICER was highly influenced by variations in treatment effect and discount rates. The result of scenario analyses suggested that targeting lecanemab to patients with mild AD or implementing price reductions could substantially improve its cost-effectiveness.</p><p><strong>Conclusions: </strong>Lecanemab's high cost poses a challenge to its inclusion in the National Health Insurance formulary under South Korea's current WTP thresholds. Strategic price adjustments and patient targeting are essential to enhance its economic viability. These findings provide valuable insights for policymakers and stakeholders in balancing treatment outcomes and resource allocation for AD management.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"793-804"},"PeriodicalIF":2.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12401850/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144675469","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}