PharmacoEconomics最新文献

{"title":"Delay and Pay? Healthcare Costs Associated with Late Oral Anti-coagulant Prescribing in People with Atrial Fibrillation.","authors":"Ryan J Mulholland, Giorgio Ciminata, Terry J Quinn, Kevin G Pollock, Steven Lister, Claudia Geue","doi":"10.1007/s40273-024-01419-2","DOIUrl":"10.1007/s40273-024-01419-2","url":null,"abstract":"<p><strong>Background: </strong>Atrial fibrillation (AF) is associated with increased morbidity and mortality and exerts an increasingly significant burden on global healthcare resources, with its prevalence rising with an ageing population. Despite a substantial thromboembolic risk, particularly in the period immediately following diagnosis, oral anti-coagulation is frequently not initiated or is delayed. The aim of this study was to evaluate healthcare costs in people with AF, comparing those who were commenced on oral anti-coagulation in the immediate period following the index diagnosis date with those in whom initiation was late and those who never started anti-coagulation.</p><p><strong>Methods: </strong>This retrospective cost analysis used linked Scottish health data to identify adults newly diagnosed with AF between January 1st 2012 and April 30th 2019 with a baseline CHA₂DS₂-VASc score of ≥ 2. This AF population was sub-divided according to timing of the first prescription of oral anti-coagulant (OAC) during a 2-year follow-up period: never started (OAC never initiated), immediate OAC (OAC prescribed within 60 days of incident AF diagnosis), and delayed OAC (OAC prescribed more than 60 days after incident AF diagnosis). A two-part model was developed, adjusted for key covariates, including age, sex, and frailty, to estimate costs for inpatient admissions, outpatient care, prescriptions, and care home admissions, and overall costs.</p><p><strong>Results: </strong>Of an overall AF population of 54,385, 26,805 (49.3%) never commenced OAC, 7654 (14.1%) initiated an OAC late, and 19,926 (36.6%) were prescribed anti-coagulation immediately. The mean adjusted cost for the overall AF population was £7807 per person per year (unadjusted: £8491). Delayed OAC initiation was associated with the greatest adjusted estimated mean annual cost (unadjusted: £13,983; adjusted: £9763), compared to those that never started (unadjusted: £10,433; adjusted: £7981) and those that received an immediate OAC prescription (unadjusted: £3976; adjusted: £6621). Increasing frailty, mortality, and female sex were associated with greater healthcare costs.</p><p><strong>Conclusion: </strong>AF is associated with significant healthcare resource utilisation and costs, particularly in the context of delayed or non-initiation of anti-coagulation. Indeed, there exists substantial opportunity to improve the utilisation and prompt initiation in people newly diagnosed with AF in Scotland. Interventions to mitigate against the growing economic burden of AF should focus on reducing admissions to hospitals and care homes, which are the principal drivers of costs; prescriptions and outpatient appointments account for a relatively small proportion of overall costs for AF.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1241-1253"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499361/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141875586","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":"Improving Transparency of Decision Models Through the Application of Decision Analytic Models with Omitted Objects Displayed (DAMWOOD).","authors":"Jeff Round, Erin Kirwin, Sasha van Katwyk, Christopher McCabe","doi":"10.1007/s40273-024-01401-y","DOIUrl":"10.1007/s40273-024-01401-y","url":null,"abstract":"<p><p>The coronavirus disease 2019 (COVID-19) pandemic has increased public awareness of the influence of epidemiological and economic decision models on public policy decisions. Alongside this is an increased scrutiny on the development, analysis, reporting and utilisation of decision models for public policy making. Therefore, it is important that model developers can clearly explain and justify to all stakeholders what is included and excluded from a model developed to support decision-making, to both improve transparency and trust in decision-making. Our aim is to provide tools for improving communication between modellers and decision-makers, leading to improved transparency in decision-making. To do so, we extend the recently described directed acyclic graphs with omitted objects displayed (DAGWOOD) approach from Haber et al. (Ann Epidemiol 68:64-71, 2022) to decision analytic models, giving the decision analytic models with omitted objects displayed (DAMWOOD) approach. DAMWOOD is a framework for the identification of objects omitted from a decision model, as well as for consideration of the effects of omissions on model outcomes. Objects omitted from a decision model are classed as either an exclusion (known and unknown confounders), misdirection (alternative model pathways) or structure (e.g. model type, methods for estimating relationships between objects). DAMWOOD requires model developers to use explicit statements and provide illustration of included and omitted objects, supporting communication with model users and stakeholders, allowing them to provide input and feedback to modellers about which objects to include or omit in a model. In developing DAMWOOD, we considered two challenges we encountered in modelling for pandemic policy response. First, the scope of the decision problem is not always made sufficiently explicit by decision-makers, requiring modellers to intuit which policy options should be considered, and/or which outcomes should be considered in their evaluation. Second, there is rarely sufficient transparency to ensure stakeholders can see what is included in models and why. This limits stakeholders' ability to advocate to decision-makers for the prioritisation of specific outcomes and challenge the model results. To illustrate the application of DAMWOOD, we apply it to a previously published COVID-19 vaccine allocation optimisation model. The DAMWOOD diagrams illustrate the ways in which it is possible to improve the communication of model assumptions. The diagrams make explicit which outcomes are omitted and provide information on the expected impact of the omissions on model results. We discuss the usefulness of DAMWOOD for framing the decision problem, communicating the model structure and results and engaging with those making and affected by the decisions the model is developed to inform.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1197-1208"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897962","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":"Using Cure Modelling for Cost Effectiveness in the NICE Technology Appraisal of Polatuzumab Vedotin in Combination for Untreated Diffuse Large B Cell Lymphoma: An External Assessment Group Perspective.","authors":"Keith Cooper, Emma Maund, Marcia Tomie Takahashi, Jonathan Shepherd","doi":"10.1007/s40273-024-01421-8","DOIUrl":"10.1007/s40273-024-01421-8","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1177-1179"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141907348","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":"Drug Pricing Stewardship from Mark Cuban's Cost Plus Generic Drug Program.","authors":"Snigdha Gulati, Mohak Gupta, TingTing Yan, Sneha Yelamanchili, Lucy Qinghua Xu, Tina Bharani, Ali Naji, Divyansh Agarwal","doi":"10.1007/s40273-024-01426-3","DOIUrl":"10.1007/s40273-024-01426-3","url":null,"abstract":"<p><strong>Importance: </strong>The exceedingly high US spending per capita on prescription medications is mediated, at least in part, by the inefficiencies of existing generic pharmaceutical distribution and reimbursement systems; yet, the extent of potential savings and areas for targeted interventions for generic drug prescribers remains underexplored.</p><p><strong>Objective: </strong>We aimed to analyze 2021 Medicare Part D spending on generic drugs in comparison with pricing of a low-cost generic drug program, the Mark Cuban Cost Plus Drug Company (MCCPDC), to gauge the extent of achievable potential savings.</p><p><strong>Design, setting, and participants: </strong>In this retrospective, observational study, we performed a systematic analysis of potential Medicare Part D savings when using MCCPDC generic pricing. The 2023 MCCPDC data, as of August 2023, were obtained from the provider's publicly available database. The 2021 Medicare Part D data and prescriber datasets were obtained from the US Centers for Medicare and Medicaid Services.</p><p><strong>Main outcomes and measures: </strong>Outcomes included total prescription volume, proportion of drugs with savings, total US dollar Medicare savings, and average weighted price reduction per unit drug. Results were stratified by medical and surgical subspecialties to identify areas for targeted interventions. Subspecialty-wise contribution to total savings versus contribution to total prescription volume was characterized.</p><p><strong>Results: </strong>Total estimated Medicare Part D savings were $8.6 billion using 90-day MCCPDC pricing, with surgical drugs accounting for over $900 million. Nearly 80% of the examined drugs were more price effective through MCCPDC using 90-day supply. Commonly prescribed drugs in cardiology, psychiatry, neurology, transplant surgery, and urology demonstrated the highest estimated absolute savings. The most disproportionate savings relative to prescription volume were observed for drugs in oncology, gynecology, infectious disease, transplant surgery, and colorectal surgery.</p><p><strong>Conclusions and relevance: </strong>This study underscores the significant potential for Medicare Part D savings through strategies that address the systemic overpayment for generic medications. We identified key areas for reform as well as specific medical and surgical subspecialties where targeted interventions could yield substantial savings.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1279-1286"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142018269","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":"Measuring the Direct Medical Costs of Hospital-Onset Infections Using an Analogy Costing Framework.","authors":"R Douglas Scott, Steven D Culler, James Baggs, Sujan C Reddy, Kara Jacobs Slifka, Shelley S Magill, Sophia V Kazakova, John A Jernigan, Richard E Nelson, Robert E Rosenman, Philip R Wandschneider","doi":"10.1007/s40273-024-01400-z","DOIUrl":"10.1007/s40273-024-01400-z","url":null,"abstract":"<p><strong>Background: </strong>The majority of recent estimates on the direct medical cost attributable to hospital-onset infections (HOIs) has focused on device- or procedure-associated HOIs. The attributable costs of HOIs that are not associated with device use or procedures have not been extensively studied.</p><p><strong>Objective: </strong>We developed simulation models of attributable cost for 16 HOIs and estimated the total direct medical cost, including nondevice-related HOIs in the USA for 2011 and 2015.</p><p><strong>Data and methods: </strong>We used total discharge costs associated with HOI-related hospitalization from the National Inpatient Sample and applied an analogy costing methodology to develop simulation models of the costs attributable to HOIs. The mean attributable cost estimate from the simulation analysis was then multiplied by previously published estimates of the number of HOIs for 2011 and 2015 to generate national estimates of direct medical costs.</p><p><strong>Results: </strong>After adjusting all estimates to 2017 US dollars, attributable cost estimates for select nondevice-related infections attributable cost estimates ranged from $7661 for ear, eye, nose, throat, and mouth (EENTM) infections to $27,709 for cardiovascular system infections in 2011; and from $8394 for EENTM to $26,445 for central nervous system infections in 2016 (based on 2015 incidence data). The national direct medical costs for all HOIs were $14.6 billion in 2011 and $12.1 billion in 2016. Nondevice- and nonprocedure-associated HOIs comprise approximately 26-28% of total HOI costs.</p><p><strong>Conclusion: </strong>Results suggest that nondevice- and nonprocedure-related HOIs result in considerable costs to the healthcare system.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1127-1144"},"PeriodicalIF":4.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11405445/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141535057","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":"Evaluating the Public Health and Health Economic Impacts of Baloxavir Marboxil and Oseltamivir for Influenza Pandemic Control in China: A Cost-Effectiveness Analysis Using a Linked Dynamic Transmission-Economic Evaluation Model.","authors":"Yawen Jiang, Jiaxin Wen, Jiatong Sun, Yuelong Shu","doi":"10.1007/s40273-024-01412-9","DOIUrl":"10.1007/s40273-024-01412-9","url":null,"abstract":"<p><strong>Background: </strong>Pandemic influenza poses a recurring threat to public health. Antiviral drugs are vital in combating influenza pandemics. Baloxavir marboxil (BXM) is a novel agent that provides clinical and public health benefits in influenza treatment.</p><p><strong>Methods: </strong>We constructed a linked dynamic transmission-economic evaluation model combining a modified susceptible-exposed-infected-recovered (SEIR) model and a decision tree model to evaluate the cost-effectiveness of adding BXM to oseltamivir in China's influenza pandemic scenario. The cost-effectiveness was evaluated for the general population from the Chinese healthcare system perspective, although the users of BXM and oseltamivir were influenza-infected persons. The SEIR model simulated the transmission dynamics, dividing the population into four compartments: susceptible, exposed, infected, and recovered, while the decision tree model assessed disease severity and costs. We utilized data from clinical trials and observational studies in the literature to parameterize the models. Costs were based on 2021 CN¥ and not discounted due to a short time-frame of one year in the model. One-way, two-way, and probabilistic sensitivity analyses were also conducted.</p><p><strong>Results: </strong>The integrated model demonstrated that adding BXM to treatment choices reduced the cumulative incidence of infection from 49.49% to 43.26% and increased quality-adjusted life years (QALYs) by 0.00021 per person compared with oseltamivir alone in the base-case scenario. The intervention also amounted to a positive net monetary benefit of CN¥77.85 per person at the willingness to pay of CN¥80,976 per QALY. Sensitivity analysis confirmed the robustness of these findings, with consistent results across varied key parameters and assumptions.</p><p><strong>Conclusions: </strong>Adding BXM to treatment choices instead of only treating with oseltamivir for influenza pandemic control in China appears to be cost-effective compared with oseltamivir alone. The dual-agent strategy not only enhances population health outcomes and conserves resources, but also mitigates influenza transmission and alleviates healthcare burden.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1111-1125"},"PeriodicalIF":4.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141493001","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":"Proxy Preferences and the Values of Children's Health States.","authors":"Daniel M Hausman","doi":"10.1007/s40273-024-01415-6","DOIUrl":"10.1007/s40273-024-01415-6","url":null,"abstract":"<p><p>To assign values to the health states of children, some health economists have suggested relying on the 'proxy' preferences among the health states of children expressed by a random sample of the adult population. These preferences have been elicited in several ways, with respondents sometimes asked to express their (adult) preferences among the health states of children, and sometimes asked to imagine themselves as children and to express what they think their preferences would be. This essay discusses three grounds for eliciting the preferences of a random sample of adults that have been suggested as ways to assign values to the health states in the EQ-5D-Y, and criticizes the first two: (1) the evidential ground: the preferences of the population sample are good evidence of how good or bad the health states of children are; (2) the 'taxpayer' ground: the adult population has the authority to assign values to health states, therefore their preferences are determinative; and (3) the pragmatic grounds: surveying is straightforward and shifts the responsibility from health economists to the population. I argue that instead of surveying a random sample of the population, health economists should rely on deliberative groups that include older children, experts on children's health and development, as well as members of the population at large. These groups should engage with the reasons that lie behind preferences among health states.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1065-1072"},"PeriodicalIF":4.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11405500/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141748763","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":"Systematic Review of Economic Evaluations of Systemic Treatments for Advanced and Metastatic Gastric Cancer.","authors":"Shikha Sharma, Niamh Carey, David McConnell, Maeve Lowery, Jacintha O'Sullivan, Laura McCullagh","doi":"10.1007/s40273-024-01413-8","DOIUrl":"10.1007/s40273-024-01413-8","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Recent advances in the development of biomarker-directed therapy and immunotherapy, for advanced and metastatic gastric cancers, have the potential to improve survival and quality of life. Much attention has been directed towards second- and later-line treatments, and the landscape here is evolving rapidly. However, uncertainty in relative effectiveness, high costs and uncertainty in cost effectiveness represent challenges for decision makers.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To identify economic evaluations for the second-line or later-line treatment of advanced and metastatic gastric cancer. Also, to assess key criteria (including model assumptions, inputs and outcomes), reporting completeness and methodological quality to inform future cost-effectiveness evaluations.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A systematic literature search (from database inception to 5 March 2023) of EconLit via EBSCOhost, Cochrane Library (restricted to National Health Service [NHS] Economic Evaluation Database and Health Technology Assessment [HTA] Database), Embase, MEDLINE and of grey literature was conducted. This aimed to identify systemic treatments that align with National Comprehensive Cancer Network (NCCN) and European Society for Medical Oncology (ESMO) Clinical Practice Guidelines. Data were collected on key criteria and on reporting completeness and methodological quality. A narrative synthesis focussed on cost-effectiveness and cost-of-illness studies. Outcomes of interest included total and incremental costs and outcomes (life-years and quality-adjusted life-years), ratios of incremental costs per unit outcome and other summary cost and outcome measures. Also, for cost-effectiveness studies, reporting completeness and the methodological quality were assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) and the Philips Checklist, respectively.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 19 eligible economic evaluations were identified (cost-effectiveness studies [n = 15] and cost-of-illness studies [n = 4]). There was a general lack of consistency in the methodological approaches taken across studies. In the main, the cost-effectiveness studies indicated that the intervention under consideration was more effective and more costly than the comparator(s). However, most interventions were not cost effective. No studies were fully compliant with reporting-completeness and methodological-quality requirements. Given the lack of consistency in the approaches taken across cost-of-illness studies, outcomes could not be directly compared.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;To our knowledge, this is the first published systematic literature review that has qualitatively synthesised economic evaluations for advanced and metastatic gastric cancer. There were differences in the approaches taken across the cost-effectiveness studies and the cost-of-illness studies. The conclusions of most of the cost-effec","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1091-1110"},"PeriodicalIF":4.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11405472/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141766938","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 Reporting Checklist for Discrete Choice Experiments in Health: The DIRECT Checklist.","authors":"Jemimah Ride, Ilias Goranitis, Yan Meng, Christine LaBond, Emily Lancsar","doi":"10.1007/s40273-024-01431-6","DOIUrl":"10.1007/s40273-024-01431-6","url":null,"abstract":"<p><strong>Background: </strong>Reporting standards of discrete choice experiments (DCEs) in health have not kept pace with the growth of this method, with multiple reviews calling for better reporting to improve transparency, assessment of validity and translation. A key missing piece has been the absence of a reporting checklist that details minimum standards of what should be reported, as exists for many other methods used in health economics.</p><p><strong>Methods: </strong>This paper reports the development of a reporting checklist for DCEs in health, which involved a scoping review to identify potential items and a Delphi consensus study among 45 DCE experts internationally to select items and guide the wording and structure of the checklist. The Delphi study included a best-worst scaling study for prioritisation.</p><p><strong>Conclusions: </strong>The final checklist is presented along with guidance on how to apply it. This checklist can be used by authors to ensure that sufficient detail of a DCE's methods are reported, providing reviewers and readers with the information they need to assess the quality of the study for themselves. Embedding this reporting checklist into standard practice for health DCEs offers an opportunity to improve consistency of reporting standards, thereby enabling transparency of review and facilitating comparison of studies and their translation into policy and practice.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1161-1175"},"PeriodicalIF":4.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11405421/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142126334","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":"Mixture and Non-mixture Cure Models for Health Technology Assessment: What You Need to Know.","authors":"Nicholas R Latimer, Mark J Rutherford","doi":"10.1007/s40273-024-01406-7","DOIUrl":"10.1007/s40273-024-01406-7","url":null,"abstract":"<p><p>There is increasing interest in the use of cure modelling to inform health technology assessment (HTA) due to the development of new treatments that appear to offer the potential for cure in some patients. However, cure models are often not included in evidence dossiers submitted to HTA agencies, and they are relatively rarely relied upon to inform decision-making. This is likely due to a lack of understanding of how cure models work, what they assume, and how reliable they are. In this tutorial we explain why and when cure models may be useful for HTA, describe the key characteristics of mixture and non-mixture cure models, and demonstrate their use in a range of scenarios, providing Stata code. We highlight key issues that must be taken into account by analysts when fitting these models and by reviewers and decision-makers when interpreting their predictions. In particular, we note that flexible parametric non-mixture cure models have not been used in HTA, but they offer advantages that make them well suited to an HTA context when a cure assumption is valid but follow-up is limited.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1073-1090"},"PeriodicalIF":4.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11405446/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141535058","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}