PharmacoEconomics最新文献

{"title":"Response to Comment on \"The Importance of Structured Expert Elicitation to Inform Outcomes Following Treatment Discontinuation: Evidence Assessment Group Perspective\".","authors":"Hangjian Wu, Cristina Fernandez-Garcia, Stephen Rice","doi":"10.1007/s40273-026-01606-3","DOIUrl":"10.1007/s40273-026-01606-3","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"641-642"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147356072","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":"Patient-Level Health Economic Modeling in Excel Without VBA: A Tutorial.","authors":"Rob Blissett, Will Sullivan, Inola Subban, Adam Igloi-Nagy","doi":"10.1007/s40273-025-01543-7","DOIUrl":"10.1007/s40273-025-01543-7","url":null,"abstract":"<p><p>Cohort-level models in Microsoft Excel^® remain the standard for cost-effectiveness modeling to inform health technology assessment (HTA), despite calls and rationale for more flexible approaches. Their limited ability to capture patient-level characteristics can, in the presence of patient heterogeneity or the need to track patient characteristics to accurately capture a technology's implications, introduce bias. Their continued prevalence is explained by key stakeholders' familiarity with spreadsheet software, and the lower computational burden of cohort-level versus patient-level models. However, contemporary Excel functions have opened up possibilities for calculations within native Excel that enable more flexible, patient-level approaches to be implemented in familiar spreadsheet-based software, without use of any Visual Basic for Applications (VBA) code. Therefore, this tutorial aims to provide step-by-step guidance on how to implement a previously published and freely available individual-level discrete event simulation (DES) in Excel, using contemporary Excel functions and without any VBA code.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"527-538"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110215/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145252257","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":"Subgroup Modelling and Use of External Evidence in Precision Oncology Appraisals: An External Assessment Group Perspective on the NICE Single Technology Appraisal of Zolbetuximab for Untreated CLDN18.2-Positive HER2-Negative Unresectable Advanced Gastric or Gastro-oesophageal Junction Adenocarcinoma.","authors":"Mirre Scholte, Andrea Fernández Coves, Huiqin Yang, Nigel Armstrong, Kevin McDermott, Xiaoyu Tian, Lisa Stirk, Robert Wolff, Manuela A Joore, Sabine E Grimm","doi":"10.1007/s40273-026-01591-7","DOIUrl":"10.1007/s40273-026-01591-7","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"509-513"},"PeriodicalIF":4.6,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110199/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146046917","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":"Accounting for Oral Corticosteroids Tapering and Reduction in Adverse Effect Burden in Health Economic Evaluations: External Assessment Group Perspective.","authors":"Julia Whitehall, Lakshmi Jayachandran, Najmeh Moradi, Katie Thomson, Madeleine Still, Sheila A Wallace, Ayomikun Ogunyemi, Gurdeep S Sagoo, Gill Norman, Stephen Rice","doi":"10.1007/s40273-026-01617-0","DOIUrl":"https://doi.org/10.1007/s40273-026-01617-0","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2026-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147777788","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":"Developing French Population Tariffs for the ICECAP-A Using Best-Worst Scaling.","authors":"Zexuan Wang, Jonathan Sicsic, Pauline Chauvin","doi":"10.1007/s40273-026-01614-3","DOIUrl":"https://doi.org/10.1007/s40273-026-01614-3","url":null,"abstract":"<p><strong>Context: </strong>There is growing recognition of the importance of measuring well-being on the basis of individuals' capabilities, rather than focusing solely on health outcomes when evaluating health and social care interventions. The ICEpop Capability Measure for Adults (ICECAP-A) operationalizes this approach by assessing five core dimensions of capability well-being. Capability value sets for ICECAP-A have already been developed in the UK, the Netherlands, and Hungary.</p><p><strong>Methods: </strong>This study aims to establish the first French value set for the ICECAP-A. We conducted an online, self-administered survey with a representative sample of the French general population (N = 3,546). Respondents completed a series of profile-case best-worst scaling tasks. Preferences for ICECAP-A attributes were analyzed using a scale-adjusted latent class best-worst choice model, which accounts for heterogeneity in choice behavior.</p><p><strong>Results: </strong>A latent class model with four classes provided the best fit to the data. The first class displayed a strong preference for Autonomy. The second class expressed relatively balanced preferences across all capabilities. The third and fourth classes prioritized enjoyment and attachment, respectively; however, the fourth class did not value Stability. On the basis of these findings, a capability value set was estimated for the French population. At the aggregate level, Attachment and Enjoyment emerged as the most important capabilities, followed by Autonomy, Stability, and Achievement.</p><p><strong>Conclusions: </strong>This study presents the first ICECAP-A value set for France. It can be applied in economic evaluations and policy decision-making to better reflect well-being beyond traditional health-related outcomes.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2026-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147717748","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":"Balancing Innovation and Access: Insights from AIM's Fair Pricing Model and Its Current and Future Applications.","authors":"Jocelijn Stokx, Anne Hendrickx, Kärt Veliste, Chiara Brouns, Sandra Scheffler, Goentje-Gesine Schoch, Maja Slapsak, Evelyn Macken, Patrick Walter, Thomas Kanga-Tona, Sahar Barjesteh van Waalwijk van Doorn-Khosrovani","doi":"10.1007/s40273-026-01615-2","DOIUrl":"https://doi.org/10.1007/s40273-026-01615-2","url":null,"abstract":"<p><strong>Objective: </strong>Access to affordable medicines is a cornerstone of equitable healthcare, yet escalating drug prices increasingly strain national healthcare systems. Cost-based pricing can be a useful complement to value-based pricing, provided that relevant variables are considered.</p><p><strong>Methods: </strong>We applied the International Association of Mutual Benefit Societies (AIM) Fair Pricing Model to ten market-protected medicines authorized by the European Medicines Agency between 2015 and 2018, covering oncology, rare diseases, and chronic conditions. Analyses were conducted across six European countries-Belgium, Estonia, Germany, the Netherlands, Slovenia, and Switzerland-to assess discrepancies between calculated fair prices and actual market prices. Fair prices were calculated using a standardized research and development (R&D) lump sum of €800 million per medicine with sensitivity analyses at €250 million and €2.5 billion. Fair prices were compared with 2020 list prices, and where available, estimated net prices. Potential budgetary savings were estimated by deriving a weighted reduction percentage from the expenditure gap between actual prices and fair prices and applying this to national spending on newly marketed medicines.</p><p><strong>Results: </strong>The analysis demonstrated that fair prices were up to 97% lower than list prices, and on average, 53% lower. When confidential discounts were considered, fair prices remained on average 33% below net prices. Applying fair prices to the ten selected medicines resulted in an average weighted reduction of 77% in gross expenditures and 73% in net expenditures of the six participating countries. When extrapolated to the European level, this corresponds to potential annual net savings of €27 billion for new medicines.</p><p><strong>Conclusions: </strong>These findings align with previous studies in Germany and Belgium, which reported potential savings of more than €13 billion and €1 billion, respectively (with R&D costs of €250 million). Currently, the model supports price negotiations of Dutch insurers and is being integrated into the Horizon Europe funded project ASCERTAIN. Overall, AIM's model offers a useful framework for both innovative and repurposed medicines, improving transparency and fairness in pricing. Current prices paid for new medicines are not justified by the underlying costs of research or their therapeutic value. Therefore, applying this model can generate substantial savings for European health systems.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147699425","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":"What is the Value of Developing an HTA Process?","authors":"Peter Murphy, Simon Walker, Septiara Putri, Mark Sculpher, Susan Griffin","doi":"10.1007/s40273-026-01613-4","DOIUrl":"https://doi.org/10.1007/s40273-026-01613-4","url":null,"abstract":"<p><p>Health technology assessment (HTA) processes are becoming increasingly established and embedded in healthcare decision making around the world. Yet, there is limited evidence on whether HTA processes at the system level offer value for money to those presiding over their creation, such as governments and ministries of health. That is, whether the value of improvements in the health system exceed the costs. To address this issue, we outline the approaches adopted in a sample of recent evaluations of HTA systems and propose a set of considerations for measuring and valuing HTA processes. The scale and remit of the process can help identify the relevant system-level impacts of HTA such as those on productive efficiency and population health, health equity, guiding innovation and care pathways, and broader impacts. We further describe the methodological challenges and potential approaches to evaluating HTA including the appropriateness of approaches. The considerations discussed can reveal the potential for ill-designed HTA processes to generate less social value than intended.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2026-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593784","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":"Deriving an Analytical Solution to Inversion of Royston/Parmar Restricted Cubic Spline Parametric Survival Models for Discrete Event Simulation.","authors":"George Bungey, Jorgen Moller, James Saunders, Venediktos Kapetanakis","doi":"10.1007/s40273-025-01569-x","DOIUrl":"10.1007/s40273-025-01569-x","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background and objective: &lt;/strong&gt;Discrete event simulation models simulate times to events rather than using the cumulative survival probabilities provided by parametric survival models. This requires inversion of the survival functions to produce analytical solutions to derive these event times from given survival estimates. While numerical methods can approximate event times for more complex survival models, this process may be computationally expensive, especially when repeated over large numbers of simulations. We aimed to derive an analytical solution to inverse functions for Royston/Parmar restricted cubic spline parametric survival models and test the execution speed when implemented in Microsoft Excel against numerical approximation methods (Goal Seek) and a hybrid approach using Brent's root-solving algorithm.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Three case types were classified according to the positioning of the given cumulative survival estimate \" &lt;math&gt;&lt;mmultiscripts&gt;&lt;mi&gt;S&lt;/mi&gt; &lt;mrow&gt;&lt;mrow&gt;&lt;/mrow&gt; &lt;mo&gt;∗&lt;/mo&gt;&lt;/mrow&gt; &lt;mrow&gt;&lt;/mrow&gt;&lt;/mmultiscripts&gt; &lt;/math&gt; \" between cumulative survival probabilities corresponding to the boundary knots from the Royston/Parmar restricted cubic spline model to determine the positioning of the solution \"t\" between knot values. For Case 1 (t before first knot) and Case 3 (t after last knot), a linear equation for &lt;math&gt;&lt;mrow&gt;&lt;mi&gt;x&lt;/mi&gt; &lt;mo&gt;=&lt;/mo&gt;&lt;/mrow&gt; &lt;/math&gt; ln(t) is produced, and single solutions are derived for t as a function of &lt;math&gt;&lt;mmultiscripts&gt;&lt;mi&gt;S&lt;/mi&gt; &lt;mrow&gt;&lt;mrow&gt;&lt;/mrow&gt; &lt;mo&gt;∗&lt;/mo&gt;&lt;/mrow&gt; &lt;mrow&gt;&lt;/mrow&gt;&lt;/mmultiscripts&gt; &lt;/math&gt; . For Case 2 (between boundary knots), a cubic equation of the form a &lt;math&gt;&lt;mi&gt;x&lt;/mi&gt;&lt;/math&gt; &lt;sup&gt;3&lt;/sup&gt; + b &lt;math&gt;&lt;mi&gt;x&lt;/mi&gt;&lt;/math&gt; &lt;sup&gt;2&lt;/sup&gt; + c &lt;math&gt;&lt;mi&gt;x&lt;/mi&gt;&lt;/math&gt; + d = 0 is derived, with a published cubic equation-solving algorithm used to obtain the correct solution for t. Royston/Parmar restricted cubic spline models were then fitted to published colon cancer data, and used to test the average execution speed of a user-defined function coded in Visual Basic for Applications (VBA) based on the analytical inversion solution compared to two Goal Seek approaches (default and increased precision) and a hybrid approach using Brent's method in Microsoft Excel over 100 replications of event time simulations, for a range of given survival estimates between 1% and 99% for all fitted models.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The mean (standard deviation) execution speed for the spline inversion user-defined function across 100 replications was 0.612 (0.029) seconds compared with 10.567 (0.175) seconds for the default Goal Seek approach, 12.230 (0.265) seconds for the increased precision Goal Seek approach and 1.140 (0.114) seconds for the hybrid Brent method, corresponding to 94.2%, 95.0%, and 46.3% reductions in average execution time, respectively.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Analytical solutions to inverse functions of Royston/Parmar restricted cubic ","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"451-459"},"PeriodicalIF":4.6,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145677863","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":"Developing and Calibrating a Colorectal Cancer Microsimulation Model for Northern Ireland.","authors":"Olivia Adair, Ethna McFerran, Mark Lawler, Luuk A van Duuren, Felicity Lamrock","doi":"10.1007/s40273-025-01572-2","DOIUrl":"10.1007/s40273-025-01572-2","url":null,"abstract":"<p><strong>Background: </strong>Individual-level microsimulation models are essential for evaluating colorectal cancer (CRC) screening programmes to capture the heterogeneity in disease progression. To ensure regional relevance, such models require detailed natural history structures and robust calibration to population-specific data. This study presents the development of the first CRC natural history microsimulation model tailored to Northern Ireland (NI) for evaluating the NI Bowel Cancer Screening Programme (NI BCSP).</p><p><strong>Method: </strong>The model simulates individual trajectories from adenoma onset to CRC diagnosis. Eight natural history parameters were calibrated to sex-specific CRC incidence data, initially using empirical (frequentist) calibration and Approximate Bayesian Computation (ABC) rejection, followed by the ABC-Markov Chain Monte Carlo (ABC-MCMC) algorithm. Other parameters were informed by NI-specific data sources.</p><p><strong>Results: </strong>The frequentist and ABC rejection calibration approach's posterior distributions informed the prior distribution for the ABC-MCMC approach. ABC-MCMC was informative, yielding 55 parameter sets, but results were constrained by limited calibration targets and parameter identifiability.</p><p><strong>Conclusion: </strong>This is the first NI-specific CRC microsimulation model, providing a regionally tailored platform for evaluating screening strategies and supporting policy. Calibration was feasible in a data-limited context, but further refinement and additional targets are needed to improve parameter estimation.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"461-475"},"PeriodicalIF":4.6,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145896193","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":"Utilization and Lifetime Costs of Formal Care for Alzheimer's Disease Dementia in Sweden.","authors":"Xin Xia, Maria Eriksdotter, Alice Clark, Niels Juul Brogaard, Jens Gundgaard, Pepa Polavieja, Nils Skajaa, Henrik Zetterberg, Silke Kern, Tobias Borgh Skillbäck, Linus Jönsson","doi":"10.1007/s40273-026-01612-5","DOIUrl":"https://doi.org/10.1007/s40273-026-01612-5","url":null,"abstract":"<p><strong>Introduction: </strong>The study aimed to estimate the utilization and lifetime costs of formal care for individuals with Alzheimer's disease (AD) dementia in Sweden.</p><p><strong>Methods: </strong>We used longitudinal data from 20,366 individuals (mean age: 78.54 years) diagnosed with AD dementia from the Swedish Register of Cognitive/Dementia Disorders linked to other registers to derive data on formal care utilization, including inpatient and outpatient specialist care, prescribed drugs, and social care. We estimated formal care utilization across AD dementia stages defined by the Mini-Mental State Examination and the lifetime formal care costs (in 2023 Swedish krona, SEK) in patients with AD dementia using the Zhao and Tian estimator, which uses the inverse probability weighting technique to account for censoring due to loss to follow-up. The estimates of lifetime costs were compared with those of controls matched to patients with AD dementia by birth year, sex, and region of residence at the time of diagnosis.</p><p><strong>Results: </strong>The number of outpatient specialist visits decreased as AD severity increased, while the number of drug prescriptions and social care utilization increased with advancing AD stages. The estimated lifetime costs of formal care for AD dementia were 2,440,000 SEK (€212,174; $US230,189; 95% CI 2,088,000-2,793,000 SEK), compared with 510,000 SEK (€44,348; $US48,113; 95% CI 482,000-539,000 SEK) for matched controls.</p><p><strong>Conclusions: </strong>This study estimated the utilization and lifetime costs of formal care for AD dementia using longitudinal register data. These findings will provide inputs for future economic evaluations of treatments and preventive interventions for AD dementia.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593699","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}