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The Monetary Value of a Statistical Life in the Context of Atherosclerotic Cardiovascular Disease.
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-29 DOI: 10.1007/s40273-025-01482-3
Jorge-Eduardo Martínez-Pérez, Fernando-Ignacio Sánchez-Martínez, José-María Abellán-Perpiñán, Domingo Pascual-Figal
{"title":"The Monetary Value of a Statistical Life in the Context of Atherosclerotic Cardiovascular Disease.","authors":"Jorge-Eduardo Martínez-Pérez, Fernando-Ignacio Sánchez-Martínez, José-María Abellán-Perpiñán, Domingo Pascual-Figal","doi":"10.1007/s40273-025-01482-3","DOIUrl":"https://doi.org/10.1007/s40273-025-01482-3","url":null,"abstract":"<p><strong>Aim: </strong>This study aims to estimate the value of a statistical life (VSL) in the context of atherosclerotic cardiovascular disease (ASCVD) in Spain using a contingent valuation/standard gamble (CV/SG) chained approach.</p><p><strong>Methods: </strong>The study employed a two-stage preference elicitation method that combined contingent valuation and a modified standard gamble technique. Specifically, willingness-to-pay and willingness-to-accept values were obtained for two health states depicting hypothetical outcomes following cardiovascular events. Subsequently, relative utility losses for the health states were derived using a modified standard gamble framing two risky choices. Chaining these elicited values allowed for VSL calculation without requiring direct valuation of small mortality risk reductions. The study was conducted through in-person interviews with a representative sample of 412 Spanish adults selected by stratified quotas.</p><p><strong>Results: </strong>The estimated VSL range is from 1.59 to 2.06 million euros. Minor differences emerge between VSL figures on the basis of each of the two health states. These VSL estimates for ASCVD are congruent with the recent update of the official VSL estimated for Spain in the context of road traffic accidents, though the upper limit of the range is slightly higher (almost 9%).</p><p><strong>Conclusions: </strong>VSL estimates align with existing ranges in other European countries, particularly in the context of road safety, where a significant portion of existing studies is concentrated. Comparisons with other contexts, involving cardiovascular diseases, also lend support to the estimates presented here.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743551","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Uncertainty in Matching-Adjusted Indirect Comparisons Using Real-World Evidence: Evidence Assessment Group Perspective.
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-21 DOI: 10.1007/s40273-025-01480-5
Cyril Onwuelazu Uteh, Eugenie Evelynne Johnson, Tomos Robinson, Najmeh Moradi, Alex Inskip, Fiona R Beyer, Katie Thomson, Gurdeep S Sagoo
{"title":"Uncertainty in Matching-Adjusted Indirect Comparisons Using Real-World Evidence: Evidence Assessment Group Perspective.","authors":"Cyril Onwuelazu Uteh, Eugenie Evelynne Johnson, Tomos Robinson, Najmeh Moradi, Alex Inskip, Fiona R Beyer, Katie Thomson, Gurdeep S Sagoo","doi":"10.1007/s40273-025-01480-5","DOIUrl":"https://doi.org/10.1007/s40273-025-01480-5","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143674450","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
What are the Revealed and Stated Population Preferences for Environmental Sustainability in Healthcare? A Scoping Review.
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-19 DOI: 10.1007/s40273-025-01479-y
Charlotte Desterbecq, Mark Harrison, Sandy Tubeuf
{"title":"What are the Revealed and Stated Population Preferences for Environmental Sustainability in Healthcare? A Scoping Review.","authors":"Charlotte Desterbecq, Mark Harrison, Sandy Tubeuf","doi":"10.1007/s40273-025-01479-y","DOIUrl":"https://doi.org/10.1007/s40273-025-01479-y","url":null,"abstract":"<p><strong>Objective: </strong>Collective changes in healthcare practices are required to ensure real environmental gains. As patient-centred care is increasingly considered to enhance the ability of health systems to meet the expectations of the population, it is crucial for policymakers and health professionals to account for the preferences of the wider public regarding environmentally friendly healthcare. This article synthesises and appraises evidence from empirical studies to understand how people value environmental concerns when making decisions within medical-related or pharmaceutical sectors.</p><p><strong>Methods: </strong>We conducted electronic searches of the PubMed, Scopus, and Embase literature databases. Studies were eligible if they conducted a quantitative experiment to understand participants' preferences regarding sustainability and green initiatives in the medical sector or for pharmaceuticals.</p><p><strong>Results: </strong>Of the 1138 documents identified, 32 studies were deemed eligible. More than 60% were published since 2020. Different methods were used to elicit the revealed and/or stated preferences of participants. In most studies, respondents valued the environment positively and were willing to change their behaviour or practices to support sustainability. However, concerns such as disease severity or clinical effectiveness of medicines or medical interventions were often prioritised over environmental considerations. The wide heterogeneity in study participants emphasises the need to involve all stakeholders to achieve the transition to a greener and sustainable healthcare system.</p><p><strong>Conclusion: </strong>The identified studies used various methods but were consistent in finding broad support for environmental considerations within the healthcare sector.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664152","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Market and Non-Market Productivity Losses Associated with Invasive Meningococcal Disease in the USA.
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-12 DOI: 10.1007/s40273-025-01477-0
Hiral Anil Shah, Ginita Jutlla, Oscar Herrera-Restrepo, Jonathan Graham, Mei Grace, Shah Alam Khan, Elise Kuylen, Shahina Begum, Frederik Verelst, Zeki Kocaata
{"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}
引用次数: 0
Correction: De Novo Cost‑Effectiveness Model Framework for Nonalcoholic Steatohepatitis-Modeling Approach and Validation.
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-12 DOI: 10.1007/s40273-025-01475-2
Peter Gal, Gyorgyi Feldmajer, Margarida Augusto, Ray Gani, Emma Hook, Ash Bullement, Zoe Philips, Inger Smith
{"title":"Correction: De Novo Cost‑Effectiveness Model Framework for Nonalcoholic Steatohepatitis-Modeling Approach and Validation.","authors":"Peter Gal, Gyorgyi Feldmajer, Margarida Augusto, Ray Gani, Emma Hook, Ash Bullement, Zoe Philips, Inger Smith","doi":"10.1007/s40273-025-01475-2","DOIUrl":"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}
引用次数: 0
A Review of Heterogeneity in Comparative Economic Analysis, with Specific Considerations for the Decentralized US Setting and Patient-Centered Care.
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-08 DOI: 10.1007/s40273-025-01478-z
Michael S Willis, Andreas Nilsson, Cheryl A Neslusan
{"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}
引用次数: 0
Economic Evaluation of Interventions to Reduce Antimicrobial Resistance: A Systematic Literature Review of Methods.
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-06 DOI: 10.1007/s40273-024-01468-7
Kristina Aluzaite, Marta O Soares, Catherine Hewitt, Julie Robotham, Chris Painter, Beth Woods
{"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}
引用次数: 0
Economic Evaluations of Non-Pharmacological Interventions for Treating Disorders of Gut-Brain Interaction: A Scoping Review. 治疗肠脑互动障碍的非药物干预措施的经济评估:范围综述》。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-01 Epub Date: 2024-11-21 DOI: 10.1007/s40273-024-01455-y
Anton Pak, Madeline O'Grady, Gerald Holtmann, Ayesha Shah, Haitham Tuffaha
{"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}
引用次数: 0
Cost-Effectiveness of Capivasertib as a Second-Line Therapy for Advanced Breast Cancer. Capivasertib作为晚期乳腺癌二线治疗的成本-效果
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-01 Epub Date: 2024-12-04 DOI: 10.1007/s40273-024-01456-x
Trang T H Nguyen, Shweta Mital
{"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<sup>+</sup>) human epidermal growth factor receptor 2-negative (HER2<sup>-</sup>) 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}
引用次数: 0
Unravelling the Association Between Uncertainties in Model-based Economic Analysis and Funding Recommendations of Medicines in Australia. 揭示基于模型的经济分析中的不确定性与澳大利亚药品资助建议之间的关联。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-03-01 Epub Date: 2024-11-15 DOI: 10.1007/s40273-024-01446-z
Qunfei Chen, Martin Hoyle, Varinder Jeet, Yuanyuan Gu, Kompal Sinha, Bonny Parkinson
{"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}
引用次数: 0
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