PharmacoEconomics最新文献

{"title":"Market and Non-Market Productivity Losses Associated with Invasive Meningococcal Disease in the USA.","authors":"Hiral Anil Shah, Ginita Jutlla, Oscar Herrera-Restrepo, Jonathan Graham, Mei Grace, Shah Alam Khan, Elise Kuylen, Shahina Begum, Frederik Verelst, Zeki Kocaata","doi":"10.1007/s40273-025-01477-0","DOIUrl":"https://doi.org/10.1007/s40273-025-01477-0","url":null,"abstract":"<p><strong>Background and objective: </strong>Invasive meningococcal disease (IMD) is an uncommon but serious disease associated with a risk of death and severe long-term sequelae, impacting both patients and their caregivers. Productivity losses due to IMD have not previously been comprehensively evaluated in the USA. This study evaluated both market and non-market productivity losses to better estimate the economic burden of IMD in the USA.</p><p><strong>Methods: </strong>An economic model estimated lifetime market (labour) and non-market (unpaid household, caring and voluntary services) productivity losses due to acute IMD, premature death due to IMD, reduced life expectancy in IMD survivors and IMD-related sequelae for patients 16 years of age or older and their caregivers based on IMD cases diagnosed in the USA in 2021 (due to data availability). Time use data were used to characterise IMD-incurred disruptions as market or non-market productivity losses. Time lost during the acute phase (assumed equal for patients and caregivers) was estimated based on hospital length-of-stay data. Time lost due to premature death from acute IMD or reduced remaining life expectancy (only calculated for patients) was estimated by subtracting the age at IMD acquisition or life expectancy of IMD survivors from average life expectancy. Time lost due to IMD-related sequelae was estimated based on sequelae event rates. Time lost was multiplied by earnings per hour (derived from median salary) to estimate productivity losses. Assumptions about sequelae impact on productivity were derived from the literature and expert clinical opinion. Scenario and sensitivity analyses assessed the impact of different inputs and assumptions on the results. Costs were inflated to 2023 US dollars.</p><p><strong>Results: </strong>Lifetime productivity losses for IMD cases diagnosed in the USA in 2021 (N = 121) totalled $87.4 million ($722,458 per case) for patients 16 years of age and older and their caregivers, with market and non-market losses accounting for approximately 72% and 28%, respectively. Premature death, reduced life expectancy and long-term sequelae were responsible for the majority of total productivity losses for patients and caregivers ($87.1 million); the acute phase accounted for $314,850. Results were most sensitive to the ratio of total benefits, median salary, case-fatality rates and specific sequelae included.</p><p><strong>Conclusions: </strong>Despite being an uncommon disease, the high mortality rate and severe long-term consequences of IMD result in a substantial economic impact. Comprehensive market and non-market productivity losses for both patients and caregivers should be considered when evaluating and communicating the true burden of IMD.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143616743","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":"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":"https://doi.org/10.1007/s40273-025-01475-2","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Review of Heterogeneity in Comparative Economic Analysis, with Specific Considerations for the Decentralized US Setting and Patient-Centered Care.","authors":"Michael S Willis, Andreas Nilsson, Cheryl A Neslusan","doi":"10.1007/s40273-025-01478-z","DOIUrl":"https://doi.org/10.1007/s40273-025-01478-z","url":null,"abstract":"&lt;p&gt;&lt;p&gt;Patient-centered care emphasizes individual preferences, but insurer coverage decisions-based on population-level evidence-may restrict treatment options for patients who differ from the average. This highlights the importance of considering heterogeneity, which refers to differences in health and cost outcomes that are systematically linked to variations in factors like patient characteristics, insurer policies, and provider practices. Failing to account for heterogeneity in economic evaluations can lead to suboptimal decisions, inferior outcomes, and inefficiency. This study aimed to assess the tools and methods for addressing heterogeneity in economic evaluations, examine the extent to which, and how, heterogeneity has been addressed in US cost-utility studies, and provide insights and recommendations to promote more fuller consideration of heterogeneity in US economic evaluations. We reviewed and adapted a seminal taxonomy of heterogeneity to the US setting, highlighting key drivers like patient preferences and insurance design. Methods for addressing heterogeneity in economic evaluations were also reviewed and summarized. We used data from the Tufts Medical Center Cost-Effectiveness Analysis Registry to assess empirical practices in US cost-utility applications, specifically the frequency, types, and impact of a subgroup analysis, and whether rationales for including or excluding subgroups were provided. The revised taxonomy highlights key drivers of heterogeneity in the diverse and decentralized US healthcare ecosystem, such as the diversity of patient preferences and in non-patient factors like access to healthcare providers and insurance coverage. Methods to explore, confirm, and incorporate heterogeneity into a comparative economic analysis exist, but are often challenged by data availability. In addition to the trade-off between potential efficiency gains and increasing uncertainty in comparative value estimates, ethical implications of stratified decisions were highlighted in the literature. We found that a subgroup analysis was rare, and primarily performed for clinical factors like age and disease severity. Only 2 of the 85 studies published between 2015 and 2022 with subgroup-level results were found to consider non-patient factors, and none considered preferences. One-third of studies reported incremental cost-effectiveness ratios differing by more than 50% from the unstratified estimate. No studies provided a rationale for omitting a subgroup analysis, and only two motivated inclusion of a subgroup analysis, limiting our ability to assess the appropriateness of these decisions. Despite well-documented methods to address heterogeneity, its application is limited in US cost-utility studies, especially regarding patient preferences and non-patient factors. As these factors often drive real-world health outcomes and costs in the USA, proper consideration of, and reporting on, heterogeneity is essential to avoid erroneous market ","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586635","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":"Economic Evaluation of Interventions to Reduce Antimicrobial Resistance: A Systematic Literature Review of Methods.","authors":"Kristina Aluzaite, Marta O Soares, Catherine Hewitt, Julie Robotham, Chris Painter, Beth Woods","doi":"10.1007/s40273-024-01468-7","DOIUrl":"https://doi.org/10.1007/s40273-024-01468-7","url":null,"abstract":"<p><strong>Background and objective: </strong>Economic evaluation of antimicrobial resistance (AMR) interventions is complicated by the multisectoral, inter-temporal and international aspects of the problem, further hindered by a lack of available data and theoretical understanding of the emergence and transmission of AMR. Despite the substantial global focus on the problem, there is a lack of comprehensive economic evaluation literature on AMR policies. The goal of this work is to review the available literature on the economic evaluation of AMR interventions focusing on methods used to quantify the effects on AMR and the associated health consequences and costs.</p><p><strong>Methods: </strong>The studies included in the review were identified by a previous study by Painter et al. that included all full economic evaluations of AMR policies in the peer-reviewed and grey literature published between 2000 and 2020. The current review extracted additional information to (1) summarise the types and the key features of the AMR intervention economic evaluation literature available; (2) systemise the types of intervention effects on AMR quantified and describe these across the dimensions of AMR burden: time, space, wider pathogen pool and different sectors (One Health framework); and (3) categorise the methods used to derive these outcomes and how were these linked to health consequences and costs.</p><p><strong>Results: </strong>Thirty-one studies were included within this review, of which 18 evaluated interventions that aimed to reduce infection rates and 11 evaluated interventions that aimed to optimise antimicrobial use. Almost all were conducted with a high-income and/or upper-middle income country perspective and focused on human health. Thirteen of 31 studies were cost-utility analyses. Fifteen of 31 and 7/31 studies estimated the AMR effects through decision tree and/or Markov models and transmission models, respectively. Transmission models and linkage of AMR outcomes to quality-adjusted life-years and costs were more common in evaluations of interventions aimed at reducing infection rates. Most of the included studies restricted the scope of evaluation to a short time horizon and a narrow geographical scope and did not consider the wider impact on other pathogens and other settings, potentially resulting in an incomplete capture of the effects of interventions.</p><p><strong>Conclusions: </strong>This review found limited available literature that mainly focused on high-income countries and infection prevention/reduction strategies. Most evaluations used a narrow study scope, which might have prevented the full capture of the costs and outcomes associated with interventions. Finally, despite the known complexities associated with quantifying AMR effects, and the corresponding methodological challenges, the implications of these choices were rarely discussed explicitly.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143567844","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":"Economic Evaluations of Non-Pharmacological Interventions for Treating Disorders of Gut-Brain Interaction: A Scoping Review.","authors":"Anton Pak, Madeline O'Grady, Gerald Holtmann, Ayesha Shah, Haitham Tuffaha","doi":"10.1007/s40273-024-01455-y","DOIUrl":"10.1007/s40273-024-01455-y","url":null,"abstract":"<p><strong>Background and objectives: </strong>Disorders of gut-brain interaction are highly prevalent and burdensome conditions for both patients and healthcare systems. Given the limited effectiveness of pharmacotherapy in treating disorders of gut-brain interaction, non-pharmacological interventions are increasingly used; however, the value for money of non-pharmacological treatments is uncertain. This is the first review to assess the economic evaluation evidence of non-pharmacological interventions for disorders of gut-brain interaction.</p><p><strong>Methods: </strong>A scoping review was conducted in line with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews (PRISMA-ScR) guidelines. Reporting adhered to ISPOR's good practices for systematic reviews with cost and cost-effectiveness outcomes. Comprehensive searches were performed on 24 October, 2023, and an updated search was run on 18 May, 2024 in PubMed/MEDLINE, Embase, Web of Science, Scopus and the International HTA database, with two reviewers screening studies in parallel. The novel Criteria for Health Economic Quality Evaluation (CHEQUE) framework was used to assess methodological and reporting quality. Reporting quality was further assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022.</p><p><strong>Results: </strong>Fifteen studies were included. Most studies examined treatments for irritable bowel syndrome. Cognitive behavioural therapy, dietary interventions and sacral neuromodulation were cost effective. Acupuncture and physiotherapy were not. CHEQUE assessment showed 12 studies met at least 70% of the methodological criteria, and 14 studies achieved 70% or more for reporting quality.</p><p><strong>Conclusions: </strong>This review highlights gaps in the current evidence base, particularly in the robustness and generalisability of results due to methodological inconsistencies. Future research should incorporate longer follow-ups, comprehensive cost assessments, subgroup analyses, equity considerations and clearer justifications for modelling assumptions.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"249-269"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681671","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 of Capivasertib as a Second-Line Therapy for Advanced Breast Cancer.","authors":"Trang T H Nguyen, Shweta Mital","doi":"10.1007/s40273-024-01456-x","DOIUrl":"10.1007/s40273-024-01456-x","url":null,"abstract":"<p><strong>Background: </strong>Capivasertib, a first-in-class AKT inhibitor, was recently approved as a second-line treatment for advanced breast cancer. However, capivasertib is expensive, raising questions over its economic value. This study provides the first evidence on the cost effectiveness of adding capivasertib to endocrine therapy (fulvestrant) for patients with PIK3CA/AKT1/PTEN-altered, hormone receptor-positive (HR⁺) human epidermal growth factor receptor 2-negative (HER2^-) advanced breast cancer.</p><p><strong>Methods: </strong>A Markov model was built to compare the costs and effectiveness of three treatment strategies. The first strategy involved adding capivasertib to fulvestrant for all patients, while the second strategy involved adding it for only postmenopausal women. The third strategy involved treatment with fulvestrant alone. Analyses were conducted from a US payer perspective over a lifetime horizon. Costs were measured in 2023 US dollars, and effectiveness was measured in life years (LYs) and quality adjusted life years (QALYs), discounted at 3% per year. One-way sensitivity analyses, probabilistic sensitivity analyses, and scenario analyses were conducted to assess the robustness of results.</p><p><strong>Results: </strong>The addition of capivasertib to fulvestrant for all patients was associated with $410,765 higher costs and 1.46 additional quality adjusted life years (QALYs) compared with fulvestrant alone, resulting in an incremental cost effectiveness ratio of $280,854/QALY. The strategy of adding capivasertib for only patients who are postmenopausal was extended dominated, i.e., yielded fewer QALYs at a higher cost per QALY than if capivasertib was added for all patients. These results were found to be robust in sensitivity and scenario analyses.</p><p><strong>Conclusions: </strong>At its current price, our analysis suggests that the addition of capivasertib to fulvestrant as a second line treatment is not cost effective versus fulvestrant alone at a willingness-to-pay threshold of $100,000/QALY. The price of capivasertib will need to be reduced by nearly 70% (to $7000 per cycle) for it to become cost effective.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"351-361"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771141","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":"Unravelling the Association Between Uncertainties in Model-based Economic Analysis and Funding Recommendations of Medicines in Australia.","authors":"Qunfei Chen, Martin Hoyle, Varinder Jeet, Yuanyuan Gu, Kompal Sinha, Bonny Parkinson","doi":"10.1007/s40273-024-01446-z","DOIUrl":"10.1007/s40273-024-01446-z","url":null,"abstract":"<p><strong>Objective: </strong>Health technology assessment is used extensively by the Pharmaceutical Benefits Advisory Committee (PBAC) to inform medicine funding recommendations in Australia. The PBAC often does not recommend medicines due to uncertainties in economic modelling that result in delaying access to medicines for patients. The systematic identification of which uncertainties can be reduced with alternative evidence or the collection of additional data can help inform recommendations. This study aims to characterise different types of uncertainty in economic models and empirically assess their association with the PBAC recommendations.</p><p><strong>Methods: </strong>A framework was developed to characterise four types of uncertainties: methodological, structural, generalisability and parameter uncertainty. The first two types were further subcategorised into parameterisable and unparameterisable uncertainty. Data on uncertainty and other factors were extracted from PBAC's Public Summary Documents of first submissions for 193 medicine (vaccine)-indication pairs including economic modelling between 2014 and 2021. Logistic regression was used to estimate the average marginal effect of each type of uncertainty on the probability of a positive recommendation.</p><p><strong>Results: </strong>The PBAC more often raised issues regarding parameter uncertainty (95%) and parameterisable structural uncertainty (83%) than generalisability uncertainty (48%) and unparameterisable methodological uncertainty (56%). The logistic regression results suggested that the PBAC was more likely to recommend a medicine without unparameterisable methodological, generalisability, and parameterisable structural uncertainty by 15.0%, 10.2 %, and 17.6%, respectively. Parameterisable methodological, unparameterisable structural and parameter uncertainty were not significantly associated with the PBAC recommendations.</p><p><strong>Conclusions: </strong>This study identified the uncertainties that had significant associations with PBAC recommendations based on the first submission. This may help improve model quality and reduce resubmissions in the future, thus improving patients' access to medicines.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"283-296"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825629/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638770","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":"Public Health Impact of Introducing a Pentavalent Vaccine Against Invasive Meningococcal Disease in the United States.","authors":"Hiral Anil Shah, Ginita Jutlla, Oscar Herrera-Restrepo, Jonathan Graham, Katherine A Hicks, Justin Carrico, Mei Grace, Diana E Clements, Cindy Burman, Woo-Yun Sohn, Elise Kuylen, Shahina Begum, Zeki Kocaata","doi":"10.1007/s40273-024-01439-y","DOIUrl":"10.1007/s40273-024-01439-y","url":null,"abstract":"<p><strong>Background: </strong>Invasive meningococcal disease (IMD) is primarily associated with five Neisseria meningitidis serogroups: A, B, C, W, or Y. In the United States (US), available vaccines protect against serogroups B (MenB), A, C, W, and Y (MenACWY), and A, B, C, W, and Y (MenABCWY). The Advisory Committee on Immunization Practices is re-evaluating the adolescent meningococcal vaccination schedule with varying recommendation formats. This analysis aimed to predict which schedule could avert the most IMD cases and have the most positive public health impact (PHI).</p><p><strong>Methods: </strong>An epidemiological model compared the 15-year PHI of vaccination schedules using MenB, MenACWY, and/or MenABCWY vaccines versus current US standard of care (SoC). Varying coverage rates reflected routine, shared clinical decision making, and risk-based recommendations. Sensitivity analyses assessed robustness of the results to different inputs/assumptions.</p><p><strong>Results: </strong>The most positive PHI compared with SoC was observed with one dose of MenACWY at 11 years of age and two doses of MenABCWY (6 months apart) at 16 years of age, assuming routine recommendation and coverage reflecting real-world uptake of MenACWY. This strategy resulted in 123 IMD cases averted (MenB: 59, MenACWY: 64), 17 deaths prevented, 574 life-years saved, and 757 quality-adjusted life-years gained versus SoC. Eliminating MenACWY vaccination at 11 years was found to result in an additional IMD burden.</p><p><strong>Conclusion: </strong>A routinely recommended two-dose pentavalent vaccine, with doses administered 6 months apart at 16 years of age, alongside the routinely recommended MenACWY vaccine at 11 years of age, would improve the PHI and benefits of IMD vaccination to society.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"311-329"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825582/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142710883","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 Tremelimumab Plus Durvalumab for Unresectable Hepatocellular Carcinoma in the USA.","authors":"Xiaomo Xiong, Jeff Jianfei Guo","doi":"10.1007/s40273-024-01453-0","DOIUrl":"10.1007/s40273-024-01453-0","url":null,"abstract":"<p><strong>Background: </strong>Treating unresectable hepatocellular carcinoma (uHCC) is challenging. Clinical trials have shown that Single Tremelimumab Regular Interval Durvalumab (STRIDE) offers clinical benefits as a first-line treatment for uHCC, but its cost effectiveness remains unknown in the USA.</p><p><strong>Objective: </strong>We aimed to assess the cost effectiveness of STRIDE (tremelimumab plus durvalumab) versus sorafenib and durvalumab monotherapy as the first-line treatment for uHCC in the USA.</p><p><strong>Methods: </strong>A partitioned survival model was constructed to assess the cost effectiveness of STRIDE compared to sorafenib and durvalumab monotherapy as the first-line treatment for uHCC from the US societal perspective. The time horizon was 48 months with 1-month cycles. Seven parametric survival functions replicated survival curves from clinical trials, with the best-fitting model used to calculate survival probabilities. Costs, health utilities, and adverse events were included, with quality-adjusted life-years (QALYs) as the primary effectiveness measure. Both costs and effectiveness were discounted at 3%. In the base-case analysis, the incremental cost-effectiveness ratio was compared to a willingness-to-pay threshold of $150,000 per QALY gained. Deterministic and probabilistic sensitivity analyses were conducted to examine the uncertainty of the model.</p><p><strong>Results: </strong>In the base-case analysis, STRIDE was cost effective compared to sorafenib, with an incremental cost-effectiveness ratio of $97,995.51 per QALY gained, based on a willingness-to-pay threshold of $150,000 per QALY gained. However, STRIDE was not cost effective compared to durvalumab monotherapy at the same threshold, with an incremental cost-effectiveness ratio of $754,408.92 per QALY gained. Deterministic sensitivity analyses were consistent with the base-case analysis. A probabilistic sensitivity analysis indicated that STRIDE was more likely to be cost effective than sorafenib and durvalumab monotherapy when the willingness-to-pay exceeded $101,000 and $713,000, respectively.</p><p><strong>Conclusions: </strong>The STRIDE regimen appears to be cost effective compared to sorafenib but not compared to durvalumab for first-line uHCC treatment in the USA. However, durvalumab has not yet been approved for uHCC in the USA. Future research should focus on long-term data and economic evaluations of other recommended biologics.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"271-282"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638768","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":"Immediate Versus 5-Year Risk-Guided Initiation of Treatment for Primary Prevention of Cardiovascular Disease for Australians Aged 40 Years: A Health Economic Analysis.","authors":"Jedidiah I Morton, Danny Liew, Gerald F Watts, Sophia Zoungas, Stephen J Nicholls, Christopher M Reid, Zanfina Ademi","doi":"10.1007/s40273-024-01454-z","DOIUrl":"10.1007/s40273-024-01454-z","url":null,"abstract":"<p><strong>Background and objective: </strong>Current Australian cardiovascular disease (CVD) prevention guidelines calculate 5-year CVD risk and recommend treatment when risk crosses specific thresholds. This may leave risk factors untreated for people with a low short-term (i.e. 5 years), but high long-term (i.e. lifetime), risk of CVD. We aimed to evaluate the cost effectiveness of intervention for risk factor control at age 40 years (regardless of calculated risk) compared to intervention for risk factor control at the age recommended by contemporary Australian CVD prevention guidelines (when the 5-year CVD risk reaches 10%) across a range of individual risk factor profiles.</p><p><strong>Methods: </strong>We used a causal microsimulation model populated with 108 different risk factor profiles, each replicated 10,000 times. Model data were derived from the UK Biobank study and published sources. The primary causal relationships factored in were those of low-density lipoprotein-cholesterol and systolic blood pressure with CVD (defined as myocardial infarction or stroke). The model simulated the ageing of individuals from 40 to 85 years. We calculated years of life lived, quality-adjusted life-years gained, incremental healthcare costs and the incremental cost-effectiveness ratio when low-density lipoprotein-cholesterol and blood pressure were controlled from age 40 years compared to initiation of treatment as recommended by Australian guidelines. The main side effect in the model was an increased risk of type 2 diabetes mellitus from statin use. The trade-off between reduced CVD and increased type 2 diabetes was summarised via quality-adjust life-years. Incremental cost-effectiveness ratios were compared to the Australian willingness-to-pay threshold of AU$28,000 per quality-adjust life-year gained. We adopted a healthcare perspective (2022 AUD) and discounted results at 3% annually.</p><p><strong>Results: </strong>An earlier intervention meaningfully prevented CVD in all but the lowest risk individuals. Intervention at age 40 years versus age when the 5-year CVD risk reaches 10% led to an increase in quality-adjust life-years for 37/54 female individuals and 44/54 male individuals simulated and an increase in years of life lived (i.e. life expectancy) for 46/54 female individuals and 47/54 male individuals simulated. Earlier intervention was also cost effective in 5/54 female individuals and 17/54 male individuals.</p><p><strong>Conclusions: </strong>Current guidelines may result in certain individuals with a lower 5-year, but higher lifetime, risk of CVD being overlooked for earlier cost-effective interventions to prevent CVD.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"331-349"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771142","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}