PharmacoEconomics最新文献

{"title":"Application of a Prevalence-Adjusted Cost-Effectiveness Threshold Framework for Pulmonary Arterial Hypertension.","authors":"Adnan Alsumali, Vasco M Pontinha, Benjamin G Cohen, Meghan Levy, William V Padula","doi":"10.1007/s40273-026-01616-1","DOIUrl":"https://doi.org/10.1007/s40273-026-01616-1","url":null,"abstract":"<p><strong>Background: </strong>Pulmonary arterial hypertension (PAH) is a rare, progressive condition associated with high per-patient treatment costs and substantial clinical burden. Interpreting cost-effectiveness evidence for rare diseases remains challenging when uniform thresholds are applied across conditions that differ markedly in prevalence and scale of healthcare production.</p><p><strong>Objective: </strong>The aim of this study was to estimate a prevalence-adjusted effective cost-effectiveness threshold for PAH and illustrate how disease prevalence and production scale influence the interpretation of cost-effectiveness evidence under a fixed health care budget.</p><p><strong>Methods: </strong>We applied a previously developed prevalence-adjusted cost-effectiveness threshold framework, referred to as a generalized dynamic prevalence (GeDP) approach, to PAH using nationally representative disease prevalence data from the Medical Expenditure Panel Survey. A flexible statistical transformation was used to accommodate the highly right skewed distribution of disease prevalence and to estimate the relationship between prevalence and effective thresholds. The analysis was anchored to an empirically estimated US health opportunity cost benchmark and applied to PAH, with comparisons to selected common and rare diseases. Illustrative scenarios examined how effective thresholds evolve under changes in disease prevalence over time.</p><p><strong>Results: </strong>Effective cost-effectiveness thresholds increased as disease prevalence declined, reflecting production-side scale effects rather than differences in societal preferences. For PAH (prevalence ≈ 0.013%), the prevalence-adjusted effective threshold was estimated at US$582,270 per QALY (95% CI 581,319-583,163), representing more than a five-fold increase relative to the reference opportunity-cost benchmark of US$104,000 per QALY applied to highly prevalent conditions. Dynamic scenarios showed that effective cost-effectiveness thresholds decline as prevalence changes over time, with larger adjustments observed for initially rare conditions.</p><p><strong>Conclusion: </strong>Applying a uniform cost-effectiveness threshold across diseases implicitly assumes homogeneous production conditions and opportunity costs. Prevalence-adjusted effective thresholds derived under the GeDP framework provide a quantitative, descriptive approach for contextualizing cost-effectiveness evidence for rare diseases such as PAH without redefining societal willingness to pay or prescribing decision rules. This approach can complement existing pharmacoeconomic evaluations by improving transparency around the role of prevalence and scale in shaping opportunity costs.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2026-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147840269","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":"The Cost Effectiveness of Upgrading Cochlear Implant Sound Processors in Australia: A Novel Application of Individual-Level Microsimulation Models.","authors":"Bonny Parkinson, Elizabeth Seil, Rajan Sharma, Paola Incerti, Jason Gavrilis, Antonio Ahumada-Canale, Padraig Kitterick","doi":"10.1007/s40273-026-01588-2","DOIUrl":"10.1007/s40273-026-01588-2","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Cochlear implants (CIs) can be used to address severe to profound sensorineural hearing loss, which is more prevalent in older adults. CI system reliability depends on both internal implants and external sound processors. Over time, sound processors age and may experience failure or faults and need repairing. However, spare parts for obsolete sound processors are harder to access. Upgrading sound processors could help CI users benefit from technology improvements; however, this may be prohibitively expensive for users. This study assessed the cost effectiveness of upgrading obsolete or soon-to-be obsolete sound processors compared with not upgrading them in adults aged 65 years and over with existing CIs from the Australian health system perspective, including whether and when to upgrade sound processors. In doing so, we present a novel application of individual-level microsimulation models that track not only individuals, but ears fitted with CIs.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We developed two state-transition microsimulation models reflecting adults with (i) unilateral CIs, and (ii) bilateral CIs. We applied a microsimulation approach to track CI users' age and each sound processor's age (which affects failure and fault rates, and health-related quality of life [HRQoL]) over 35 years. Two models were developed as bilateral CI users may have two sound processors of differing ages, and because sound processors beyond replacement or repair result in no access to sound in that ear-although unilateral CI users may have residual hearing in the non-implanted ear. Inputs were based on the Australian Cochlear Upgrade Sound Processor (CUSP) study (N = 200), a survey of four clinics involved in the study, the published literature and administrative sources. We reported the incremental cost-effectiveness ratio (ICER) in terms of cost per quality-adjusted life year (QALY) gained and conducted extensive threshold, univariate, scenario and probabilistic sensitivity analyses. All costs were reported in 2022 Australian dollars (AUD).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;For unilateral CI users, if sound processors were beyond replacement or repair at 20 (15) years then upgrading sound processors when they reach the age of around 11 years (3 years) resulted in an ICER of AUD$50,000 per QALY gained when compared with not upgrading sound processors. When the scenarios were compared incrementally to each other, upgrading sound processors when they reach the age of 20 (15) years resulted in an ICER below AUD$50,000 per QALY gained. For bilateral CI users, if sound processors were beyond replacement or repair at 15 or 20 years then upgrading sound processors when they reach the end of warranty (3 years) would result in an ICER of AUD$50,000 per QALY gained when compared with not upgrading sound processors. When the scenarios were compared incrementally to each other, then upgrading sound processors when they reach 9 years resulted in an I","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"615-638"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110226/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146202358","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":"Estimating the Value of Combination Vaccines: A Methodological Framework.","authors":"Mark Jit, Allison Portnoy, Clint Pecenka, William P Hausdorff, Christopher Gill","doi":"10.1007/s40273-025-01575-z","DOIUrl":"10.1007/s40273-025-01575-z","url":null,"abstract":"<p><p>Combination vaccines combine several components in a single dose administration. They offer programmatic and public health advantages, particularly as vaccine schedules become increasingly crowded. They are often more expensive to develop and produce, which discourages manufacturer investment without clear market signals. Hence their benefits need to be captured with existing health economic evaluation reference cases used by decision-makers to guide vaccine investments. We propose that the value of combination vaccines can be captured through at least four domains: (1) reductions in tangible and intangible costs to caregivers, (2) operational efficiencies to the health system, (3) opportunity costs of vaccine schedule slots, and (4) more streamlined vaccine schedules. We demonstrate the practicality of our framework by comparing the value of introducing a hypothetical vaccine to a crowded schedule as a standalone formulation, a replacement for a vaccine already in the schedule, or a combination product. The framework could also be applied to estimate the value of reducing the number of separate administrations needed for a standalone vaccine. Applying it in real-world situations could be facilitated by further data collection, particularly on collating results on the value of existing vaccines in the schedule and estimating willingness-to-pay for fewer vaccine administrations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"517-526"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110238/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145900786","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Innovative Contracting for Gene Therapies: Current Landscape and Perspectives on the Future of Gene Therapy Financing in the USA.","authors":"Tyler D Wagner, Jacqlyn W Riposo, Kendra M Gould, Jonathan D Campbell, James T Kenney, Claire M Csenge, Theresa Schmidt","doi":"10.1007/s40273-025-01563-3","DOIUrl":"10.1007/s40273-025-01563-3","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background and objective: &lt;/strong&gt;Over the last decade, payers in the USA have been exploring novel financing mechanisms for gene therapies (GTs). Our research objective was to assess the landscape of innovative contracts (ICs) between payers and manufacturers for GTs and identify barriers and opportunities for future contract development and implementation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We used a multi-method approach including a targeted literature review and interviews. We developed a framework defining 'innovative contracts' as agreements using real-world outcomes that link to the total price paid for gene therapy, encompassing value-based pricing, outcome-based payments, and performance-based models between payers and manufacturers. We searched for published information about implementation of ICs for GTs in PubMed and government, industry, and research institutions from January 2014 to January 2025. We excluded any insights specific to ICs for non-GTs as well as those relevant to ex-US markets. We supplemented these findings with bibliographic searches. Semi-structured interviews with payers, manufacturers, and other diverse representatives from the GT financing ecosystem were conducted to validate and enrich the literature findings.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The PubMed search yielded ten studies relevant to implementation of ICs. Gray literature included over 50 publications referencing active contracts, policy solutions, payer budget impact, and state Medicaid programs' innovative GT contracting. Information on manufacturer and payer contracts was publicly available for 10 of 14 gene therapies (71%). Of 16 identified GT contracts, eight used upfront payments with milestone-based rebates, two used performance-based installment payments, one offered upfront payment with a rebate or payment over 5 years, and five do not have publicly available details on the type of financial arrangement. Interviews (N = 15) suggested that barriers to ICs include a lack of mutual trust between payers and manufacturers, lack of data conveying the return on investment for innovative contracts, lack of a sufficient incentive for stakeholders to engage in contracting, perceived regulatory limitations (e.g., implications of Medicaid Best Price), and patient portability challenges. Some interviewees believed that ICs should be the standard for GTs, while others stated that ICs should only be pursued when they are expected to have a significant impact on timely patient access in the early launch period when payers are considering limited or no coverage. Interviewees indicated that policy changes may encourage future contracting negotiation and implementation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Widespread uptake of ICs will require a multi-stakeholder collaboration to overcome common barriers, as a one-size-fits-all approach is insufficient for diverse stakeholder needs. Establishing industry-wide contracting principles and practices may help br","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"585-598"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110221/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145637199","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":"The Value of Combination Vaccines in Children and Adults: More than the Sum of Their Parts.","authors":"Anthony T Newall","doi":"10.1007/s40273-026-01603-6","DOIUrl":"10.1007/s40273-026-01603-6","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"515-516"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146213656","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":"Comment on \"The Importance of Structured Expert Elicitation to Inform Outcomes Following Treatment Discontinuation: Evidence Assessment Group Perspective\".","authors":"Dyfrig A Hughes","doi":"10.1007/s40273-026-01605-4","DOIUrl":"10.1007/s40273-026-01605-4","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"639-640"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147356008","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: Estimating the Value of Combination Vaccines: A Methodological Framework.","authors":"Mark Jit, Allison Portnoy, Clint Pecenka, William P Hausdorff, Christopher Gill","doi":"10.1007/s40273-026-01598-0","DOIUrl":"10.1007/s40273-026-01598-0","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"643-646"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110219/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147373046","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":"Are Health Gains to Children and Adolescents More Important Than Health Gains to Adults? A Person Trade-Off Study.","authors":"Tessa Peasgood, Udeni De Silva Perera, Gang Chen, Cate Bailey, Richard Norman, Rosalie Viney, Koonal Shah, Nancy Devlin","doi":"10.1007/s40273-025-01574-0","DOIUrl":"10.1007/s40273-025-01574-0","url":null,"abstract":"<p><strong>Purpose: </strong>Healthcare decision-making often assumes equal value for quality-adjusted life years (QALYs) across patient groups, yet societal preferences suggest that the value of a QALY may vary with characteristics such as age. Evidence indicates some willingness to prioritise child health gains, though findings are inconsistent. This study used person trade-off (PTO) to estimate the relative social value of different types of health gains for children and adolescents (aged 0-24 years) compared with adults.</p><p><strong>Methods: </strong>A representative Australian sample aged 16 years and above (n = 2098) completed an online survey comparing life extension and quality-of-life improvements for different ages. A 'chaining' approach tested response consistency, and logistic regression explored associations between PTO choices and respondent characteristics. Attitudinal questions and open text responses provided additional insights.</p><p><strong>Results: </strong>PTO responses show that health gains for children and adolescents (4-24 years) are generally valued more highly than those for adults (age 40 or 55 years), with weights ranging from 1 to 1.3. For very young children, findings vary by health gain type: life extensions for infants (1 month or 2 years) are weighted lower, but pain alleviation is higher (weights ≥ 1.2). Qualitative and attitudinal data reveal diverse views, with many opposing age-weighting. Younger respondents and those with young children prioritise children more, while older and female participants preferred equal treatment.</p><p><strong>Conclusions: </strong>The relative value of child QALY gains varies by age of the child, by health gain type, and by adult comparison age. While alleviating children's pain is strongly supported (weights ≥ 1.2), overall views are polarised, highlighting the complexity of age-based prioritisation.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"599-613"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110227/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146132605","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Modern Approach for Constructing Decision Analytic Models in Microsoft Excel.","authors":"Mike Paulden","doi":"10.1007/s40273-025-01582-0","DOIUrl":"10.1007/s40273-025-01582-0","url":null,"abstract":"<p><p>The majority of decision analytic models submitted to health technology assessment (HTA) agencies are developed using Microsoft Excel. The approaches commonly used to construct these models have not been substantially updated in decades, and studies have found that spreadsheet models are often slower and more difficult to validate than models built using R. However, Excel and Google Sheets were recently upgraded to add support for dynamic array functions. This allows for many of the techniques used in R modeling to be applied to spreadsheet models. This paper provides a tutorial on how these new functions can be leveraged to build efficient Markov cohort models using modern spreadsheet software. A novel approach is presented for conducting Monte Carlo simulation using a single formula in one cell, without the need for Visual Basic for Applications (VBA) macros. A number of template formulas are also provided that can be used to assist in common modeling tasks, including constructing a Markov trace and calculating the table of probabilities needed to plot cost-effectiveness acceptability curves (CEACs). These template formulas may be directly copied and pasted into any spreadsheet model, with no add-ons, plug-ins, or additional packages required. These advancements have the potential to modernize how spreadsheet models are developed, simplifying their construction, improving their calculation speed, and reducing the time needed for validation. They will also aid in teaching more efficient approaches for decision analytic modeling to a new generation of students.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"539-558"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147481297","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 Systematic Review of the Economic Burden of Prostate Cancer: Direct and Indirect Cost Perspectives.","authors":"Josep Darbà, Meritxell Ascanio, Antonio Rodríguez","doi":"10.1007/s40273-026-01594-4","DOIUrl":"10.1007/s40273-026-01594-4","url":null,"abstract":"<p><strong>Introduction: </strong>Prostate cancer (PC) is the second most common cancer in men. Although many studies have assessed its economic burden, no recent reviews have focused on studies conducted under current clinical guidelines. This study systematically reviews recent cost-of-illness studies evaluating direct and indirect costs associated with PC.</p><p><strong>Methods: </strong>A systematic search was conducted using the PICOS framework and a combination of free-text and MeSH terms in PubMed and the Cochrane Library, and only free-text terms in EconLit. The search included articles published between January 2015 and October 2025. Data on total, direct, and indirect costs were extracted and synthesized. All costs were converted to 2025 USD, and quality of studies was assessed with a simplified version of the CHEERS checklist.</p><p><strong>Results: </strong>Ninety-five studies met the inclusion criteria. Direct medical costs for non-metastatic prostate cancer (nmPC) varied widely by disease stage, treatment, and country, ranging from approximately US$1200 to US$280,000 per patient-year, with higher costs observed in advanced stages and in patients experiencing treatment-related adverse events (AEs). Progression to metastatic disease was associated with a marked cost escalation, with annual costs largely driven by systemic therapies and skeletal-related events. Indirect costs ranged from US$666 to US$12,900 per patient-year and accounted for up to 30% of total PC-related costs, primarily due to productivity losses from premature mortality.</p><p><strong>Conclusions: </strong>PC imposes a substantial economic burden on healthcare systems and society, particularly in advanced stages. Policy promoting early detection, risk-adapted treatment, and equitable therapy access may help contain costs. Further research should address the economic impact of emerging diagnostics and minimally invasive interventions.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"559-583"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110197/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146132689","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}