PharmacoEconomics最新文献

{"title":"Measuring Effectiveness Based on Patient Experience (Instead of QALYs) in US Value Assessments.","authors":"Maksat Jumamyradov, Benjamin M Craig","doi":"10.1007/s40273-024-01444-1","DOIUrl":"10.1007/s40273-024-01444-1","url":null,"abstract":"<p><strong>Background: </strong>A key challenge in value assessment is how to summarize effectiveness, particularly the impact of interventions on patient health-related quality of life (HRQoL). One approach is to quantify the gains in HRQoL and life expectancy together as quality-adjusted life years (QALYs); however, this approach has faced various criticisms regarding its potential discriminatory aspects toward persons with disabilities, older adults, and the most vulnerable individuals in society.</p><p><strong>Methods: </strong>Instead of QALYs, we provide an alternative approach that summarizes HRQoL gains from the perspective of its stakeholders (e.g., patients, parents, and caregivers) using an \"experience\" scale. On an experience scale, a positive value signifies an experience better than having no experience at all, while a negative value indicates an experience worse than having no experience. To illustrate the merits of this approach, we examine US preferences on the relief of child health problems, namely a discrete choice experiment (DCE) with kaizen tasks and alternatives described using the EQ-5D-Y-3L.</p><p><strong>Results: </strong>Using this approach, we demonstrate the differences in perspectives between parents (N = 179), mothers (N = 99), and fathers (N = 80) of children younger than 18 years of age, as well as the feasibility of this patient-centered approach using a brief DCE survey of less than 100 respondents each (and without QALYs). Specifically, we found that mothers place a higher value on the child's feelings than fathers. The results also suggest other differences between the perspectives of mothers and fathers, but these differences were not statistically significant (p-values < .05).</p><p><strong>Conclusions: </strong>We put forth that future value assessments may summarize gains in HRQoL on a patient experience scale (i.e., experience scale from the patient perspective) to inform decision-making.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"171-176"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782394/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142564477","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":"Estimation of Transition Probabilities from a Large Cohort (> 6000) of Australians Living with Multiple Sclerosis (MS) for Changing Disability Severity Classifications, MS Phenotype, and Disease-Modifying Therapy Classifications.","authors":"Julie A Campbell, Glen J Henson, Valery Fuh Ngwa, Hasnat Ahmad, Bruce V Taylor, Ingrid van der Mei, Andrew J Palmer","doi":"10.1007/s40273-024-01417-4","DOIUrl":"10.1007/s40273-024-01417-4","url":null,"abstract":"<p><strong>Background: </strong>Multiple sclerosis (MS) is a chronic autoimmune/neurodegenerative disease associated with progressing disability affecting mostly women. We aim to estimate transition probabilities describing MS-related disability progression from no disability to severe disability. Transition probabilities are a vital input for health economics models. In MS, this is particularly relevant for pharmaceutical agency reimbursement decisions for disease-modifying therapies (DMTs).</p><p><strong>Methods: </strong>Data were obtained from Australian participants of the MSBase registry. We used a four-state continuous-time Markov model to describe how people with MS transition between disability milestones defined by the Expanded Disability Status Scale (scale 0-10): no disability (EDSS of 0.0), mild (EDSS of 1.0-3.5), moderate (EDSS of 4.0-6.0), and severe (EDSS of 6.5-9.5). Model covariates included sex, DMT usage, MS-phenotype, and disease duration, and analysis of covariate groups were also conducted. All data were recorded by the treating neurologist.</p><p><strong>Results: </strong>A total of N = 6369 participants (mean age 42.5 years, 75.00% female) with 38,837 person-years of follow-up and 54,570 clinical reviews were identified for the study. Annual transition probabilities included: remaining in the no, mild, moderate, and severe states (54.24%, 82.02%, 69.86%, 77.83% respectively) and transitioning from no to mild (42.31%), mild to moderate (11.38%), and moderate to severe (9.41%). Secondary-progressive MS was associated with a 150.9% increase in the hazard of disability progression versus relapsing-remitting MS.</p><p><strong>Conclusions: </strong>People with MS have an approximately 45% probability of transitioning from the no disability state after one year, with people with progressive MS transitioning from this health state at a much higher rate. These transition probabilities will be applied in a publicly available health economics simulation model for Australia and similar populations, intended to support reimbursement of a plethora of existing and upcoming interventions including medications to reduce progression of MS.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"223-239"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782298/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879169","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":"Recurrence of Cardiovascular Events After an Acute Myocardial Infarction in Patients with Multivessel Disease and Associated Healthcare Costs: A German Claims Data Analysis.","authors":"Alexandra Starry, Nils Picker, Jonathan Galduf, Ulf Maywald, Axel Dittmar, Stefan G Spitzer","doi":"10.1007/s40273-024-01440-5","DOIUrl":"10.1007/s40273-024-01440-5","url":null,"abstract":"<p><strong>Aim: </strong>This study sought to quantify the healthcare costs of multivessel disease (MVD) and determine the prevalence and incidence of recurrent major adverse cardiovascular events (MACE) in high-risk patients diagnosed with MVD following an acute myocardial infarction (MI).</p><p><strong>Methods: </strong>This retrospective study utilized German claims data (AOK PLUS), between 01/01/2010 and 31/12/2020. Patients were included if they (1) had an inpatient diagnosis of an MI between 01/01/2012 and 31/12/2019 (index date), (2) were ≥ 18 years of age at date of MI diagnosis, and (3) had diabetes or met two of the following criteria: ≥ 65 years old, prior MI, peripheral arterial disease. MACE was defined as (1) MI, (2) stroke, or (3) death with a cardiovascular diagnosis within 30 days prior. To measure the burden of MVD, patients were identified during the index hospitalization by presence of MVD. Healthcare resource use and costs were compared after adjustment based on propensity score matching (PSM).</p><p><strong>Results: </strong>A total of 5158 patients with evidence for MVD were included in the main analysis. 31.17% experienced a MACE within 365 days following the incident MI. After PSM adjustment, 33.22% of the MVD cohort experienced a MACE versus 36.48% of non-MVD patients. MVD patients had a higher rate of recurrent MI (14.22% vs. 9.81%). Additionally, public healthcare costs were about €4 million higher in the total MVD cohort than in the non-MVD cohort in the first year after an MI (€47,896,012.32 vs. €43,718,713.75, respectively), reflecting the MVD cohort's higher use of the public healthcare system. More MVD patients were prescribed guideline-recommended medication (61.4% vs. 46.0%).</p><p><strong>Conclusion: </strong>This study found that presence of MVD contributed to higher rates of recurrent MI. Patients with MVD experienced higher rates of recurrent MI despite a higher proportion of patients receiving guideline-directed medication therapy compared to non-MVD patients. Conversely, there was a higher mortality rate observed in the non-MVD cohort.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"177-189"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142575779","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 Literature Review of Modelling Approaches to Evaluate the Cost Effectiveness of PET/CT for Therapy Response Monitoring in Oncology.","authors":"Sietse van Mossel, Rafael Emilio de Feria Cardet, Lioe-Fee de Geus-Oei, Dennis Vriens, Hendrik Koffijberg, Sopany Saing","doi":"10.1007/s40273-024-01447-y","DOIUrl":"10.1007/s40273-024-01447-y","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background and objective: &lt;/strong&gt;This systematic literature review addresses model-based cost-effectiveness studies for therapy response monitoring with positron emission tomography (PET) generally combined with low-dose computed tomography (CT) for various cancer types. Given the known heterogeneity in therapy response events, studies should consider patient-level modelling rather than cohort-based modelling because of its flexibility in handling these events and the time to events. This review aims to identify the modelling methods used and includes a systematic assessment of the assumptions made in the current literature.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This study was conducted and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 statement. Information sources included electronic bibliographic databases, reference lists of review articles and contact with experts in the fields of nuclear medicine, health technology assessment and health economics. Eligibility criteria included peer-reviewed scientific publications and published grey literature. Literature searches, screening and critical appraisal were conducted by two reviewers independently. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) were used to assess the methodological quality. The Bias in Economic Evaluation (ECOBIAS) checklist was used to determine the risk of bias in the included publications.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The search results included 2959 publications. The number of publications included for data extraction and synthesis was ten, representing eight unique studies. These studies addressed patients with lymphoma, advanced head and neck cancers, brain tumours, non-small cell lung cancer and cervical cancer. All studies addressed response to chemotherapy. No study evaluated response to immunotherapy. Most studies positioned PET/CT as an add-on modality and one study positioned PET/CT as a replacement for conventional imaging (X-ray and contrast-enhanced CT). Three studies reported decision-tree structures, four studies reported cohort-level state-transition models and one study reported a partitioned survival model. No patient-level models were reported. The simulation horizons adopted ranged from 1 year to lifetime. Most studies reported a probabilistic analysis, whereas two studies reported a deterministic analysis only. Two studies conducted a value of information analysis. Multiple studies did not adequately discuss model-specific aspects of bias. Most importantly and regularly observed were a high risk of structural assumptions bias, limited simulation horizon bias and wrong model bias.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Model-based cost-effectiveness analysis for therapy response monitoring with PET/CT was based on cohorts of patients instead of individual patients in the current literature. Therefore, the heterogeneity in therapy response events was commonly not addr","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"133-151"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782410/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142564433","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 Use of Real-World Data for Estimating Relative Treatment Effects in NICE Health Technology Assessment Submissions: A Review.","authors":"Yoojung Che, Stephen Duffield, Manuel Gomes","doi":"10.1007/s40273-024-01449-w","DOIUrl":"10.1007/s40273-024-01449-w","url":null,"abstract":"<p><p>This paper investigates the current use of real-world data (RWD) for estimating relative treatment effects in National Institute for Health and Care Excellence (NICE) health technology assessment (HTA) submissions. This review included 64 HTA submissions, which accounted for approximately 11% of the total NICE HTA submissions between January 2016 and December 2023. The main sources of RWD considered in the submissions were disease registries and electronic health records. RWD were primarily used to provide an external control arm to enable comparisons within single-arm trials and to inform long-term treatment effects when extrapolating survival data beyond the trial follow-up. Adjustments for potential systematic differences between treatment groups have improved over time; however, approximately one-third of the submissions still relied on unadjusted treatment comparisons. We found that approximately 10% of NICE HTA submissions used RWD to directly inform treatment effects estimation. Over one-third of the submissions relied on naïve and/or unadjusted treatment comparisons, which may have introduced biases. To ensure that RWD provide sound evidence for HTA, submissions should follow published guidelines, including the NICE real-world evidence (RWE) framework.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"123-131"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782325/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625480","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":"Costs of Care in Relation to Alzheimer's Disease Severity in Sweden: A National Registry-Based Cohort Study.","authors":"Sandar Aye, Oskar Frisell, Henrik Zetterberg, Tobias Borgh Skillbäck, Silke Kern, Maria Eriksdotter, Emil Aho, Xin Xia, Bengt Winblad, Anders Wimo, Linus Jönsson","doi":"10.1007/s40273-024-01443-2","DOIUrl":"10.1007/s40273-024-01443-2","url":null,"abstract":"<p><strong>Background: </strong>The advancement of diagnostic and therapeutic interventions in early Alzheimer's disease (AD) has demanded the economic evaluation of such interventions. Resource utilization and cost estimates in early AD and, more specifically, the amyloid-positive population are still lacking. We aimed to provide cost estimates in AD in relation to disease severity and compare these with the control population. We also aimed to provide cost estimates for a subset of the AD population with both clinical diagnosis and amyloid-positive confirmation.</p><p><strong>Materials and methods: </strong>This was a retrospective longitudinal analysis of resource utilization using data from national registries. A cohort from the national Swedish registry for cognitive/dementia disorders (SveDem) includes all clinically diagnosed AD between 2013 and 2020. The study population included 31,951 people with AD and 63,902 age- and sex-matched controls (1:2). The population was followed until death, the end of December 2020, or 2 years from the last clinic visit. Direct medical and social costs were estimated from other national registries. Direct medical costs include costs for medications and inpatient and outpatient clinical visits. Direct social costs include costs for institutionalization, home care, short-term care, support for daytime activities, and housing support. Mean annual costs and 95% confidence intervals were obtained by bootstrapping, presented in 2021 Swedish Krona (SEK) (1 SEK = 0.117 USD, 1 SEK = 0.0985 EUR in 2021), and disaggregated by AD severity, cost component, sex, age group, and care setting.</p><p><strong>Results: </strong>Mean annual costs for individuals with clinically diagnosed AD were SEK 99,906, SEK 290,972, SEK 479,524, and SEK 795,617 in mild cognitive impairment (MCI), mild, moderate, and severe AD. The mean annual costs for the population with both clinical diagnosis and amyloid-positive AD confirmation (N = 5610) were SEK 57,625, SEK 179,153, SEK 333,095, and SEK 668,073 in MCI, mild, moderate, and severe AD, respectively. The mean annual costs were higher in institutionalized than non-institutionalized patients, females than males, and older than younger age groups. Inpatient and drug costs were similar in all AD severity stages, but outpatient costs decreased with AD severity. Costs for institutionalization, home care, support for daytime activities, and short-term care increased with AD severity, whereas the cost of housing support decreased with AD severity.</p><p><strong>Conclusions: </strong>This is the first study estimating annual costs in people with AD from MCI to severe AD, including those for the amyloid-positive population. The study provides cost estimates by AD severity, cost components, care settings, sex, and age groups, allowing health economic modelers to apply the costs based on different model structures and populations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"153-169"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782292/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142562905","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":"Value-Based Healthcare in Practice: IDEATE, a Collaboration to Design and Test an Outcomes-Based Agreement for a Medicine in Wales.","authors":"Jessica R Burton, Kate Halsby, Graciela Sáinz de la Fuente, Jonathan Pearson-Stuttard, Rebecca Sloan, Thomas Porter, Gareth John, Andrew Warburton, Jennifer Selby, Gail Povey, Ruhe Chowdhury, Catherine Bale, Mark Davies, Emma Clifton-Brown, Hamish Laing","doi":"10.1007/s40273-024-01445-0","DOIUrl":"10.1007/s40273-024-01445-0","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To develop a sustainable, scalable methodology for the design of outcome-based agreements (OBAs) that works on the ground and dynamically overcomes historical challenges.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Project IDEATE co-created solutions to known (and emergent) challenges via iterative workshops and real-world data analysis to develop and refine a hypothetical model for an OBA in a trusted research environment. A cross-disciplinary collaboration between National Health Service (NHS) Wales, industry and academia was developed. Data were collected from Welsh national datasets and used to construct a novel linked dataset. OBA scenarios, with different contract parameters, were analysed to assess impact on the proportion of contract payment due and the volatility of payments.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;An approved, in market, locally advanced and metastatic breast cancer treatment was selected as the test case. The total number of patients in the treatment cohort (2017-2020) was n = 99, and 286 in the control cohort (2014-2016). The final outcome variables selected were: (1) 1-year survival,( 2) intolerance to treatment (deferral), and (3) the total days disrupted by care. The primary scenario included all three outcomes measured at the population level and used a linear payment model. Volatility analyses demonstrated contract parameters can dramatically alter the contract output with greatest risk from a single, binary outcome contract design.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;The design of an OBA is a complex process that requires a multi-disciplinary approach. By assessing solutions to data, outcomes and contracting challenges, IDEATE provides a strong foundation for future success of OBAs in the UK. Outcome-based agreements (OBAs) are a way to pay for medicines if they help patient health in a specific way over time. These agreements can make it faster for people to get new medicines, but they also have challenges, like needing a lot of time and effort to manage them. A team from the NHS Wales, life sciences, and Swansea University created Project IDEATE to find a better way to design OBAs and solve some of these problems. Welsh datasets were used to create a new breast cancer dataset to test different OBAs and see how payments would change. A breast cancer treatment was used for the project. The project had 99 patients who got the medicine (2017-2020) and 286 patients who had breast cancer but did not get the medicine (2014-2016). Three health outcomes were measured: (1) living for one year after treatment, (2) patients needing to stop the medicine, and (3) days spent in care. The main OBA option we tested used all three health outcomes; the more the outcomes improved, the more the payments could go up until they hit the highest amount agreed. The analysis showed that the way an OBA is designed can make a big difference in how stable or risky it is, especially if one of the health outcomes has only two opti","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"191-207"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625499","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":"Comparison of EQ-5D-Y-3L Utility Scores Using Nine Country-Specific Value Sets in Chinese Adolescents.","authors":"Ya'nan Wu, Yanjiao Xu, Zhao Shi, Junchao Feng, Zhihao Yang, Zhuxin Mao, Lei Dou, Shunping Li","doi":"10.1007/s40273-024-01451-2","DOIUrl":"10.1007/s40273-024-01451-2","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to assess and compare the measurement properties of EQ-5D-Y-3L utilities derived from available countries' value sets (Chinese, Japanese, Slovenian, German, Spanish, Hungarian, Netherlandish, Belgian, and Indonesian), among Chinese adolescents.</p><p><strong>Methods: </strong>From July to September 2021, a large-scale cross-sectional survey was administered across 16 cities in Shandong, China, with the objective of assessing the health status of junior high school students aged 10-18 years. Supported by the educational authorities, quick response (QR) codes and questionnaire links were disseminated to schools. A total of 97,413 junior high school students completed the questionnaire. Agreement, convergent validity, and known-group validity were determined in the nine country-specific value sets.</p><p><strong>Results: </strong>The Indonesian value set demonstrated the highest mean health utility score (0.970), followed by the Japanese (0.961), Chinese (0.960), Netherlandish (0.948), Hungarian (0.942), German (0.938), Belgian (0.932), Slovenian (0.926), and Spanish (0.926) value sets, respectively. The utility scores derived from Asian value sets were higher than those from Europe. Good or excellent agreements (intraclass correlation coefficients > 0.7) were found between each paired value set. In Bland-Altman plots, the 95% limits of agreement for any two value sets were 0.046-0.348. A strong relationship (Spearman's correlation coefficients > 0.99) between any two value sets was found. The EQ-5D-Y-3L utility scores discriminated equally well for the nine value sets across three known groups. The effect size and the relative efficiency statistics showed the Chinese value sets were more sensitive in general. Referring to the Chinese value set, all the relative efficiency values in each value set were similar across three known groups, ranging from 0.9 to 1.0.</p><p><strong>Conclusions: </strong>A total of nine country-specific EQ-5D-Y-3L value sets showed an overall high level of agreement, strong correlation, and good known-group validity. However, the utility scores derived from nine EQ-5D-Y-3L value sets were different and the country-specific value sets were not interchangeable.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"209-221"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625477","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":"Estimating the Incremental Cost Per QALY Produced by the Spanish NHS: A Fixed-Effect Econometric Approach.","authors":"Laura Vallejo-Torres","doi":"10.1007/s40273-024-01441-4","DOIUrl":"10.1007/s40273-024-01441-4","url":null,"abstract":"<p><strong>Background: </strong>Knowing the health opportunity costs of funding decisions is crucial to assess whether the health gains associated with new interventions are larger than the health losses imposed by the displacement of resources. Empirical estimates based on the effect of health spending on health outcomes have been proposed in several countries, including Spain, as a proxy to capture these opportunity costs. However, there is a need to regularly update existing health opportunity cost estimates and to explore the role of omitted variable bias in these estimations.</p><p><strong>Objective: </strong>The aim of this paper is to provide an updated and refined estimate of the causal impact of health spending on health in Spain that can be translated into an estimate of the incremental cost per quality-adjusted life-year produced by the Spanish national health system.</p><p><strong>Methods: </strong>We applied fixed-effect models using data for 17 Spanish regions from 2002 until 2022 to estimate the impact of public health spending on health outcomes and explored the extent of omitted variable bias. Changes in these estimates over time were assessed and alternative specifications were tested.</p><p><strong>Results: </strong>Based on fixed-effect models with control variables, the estimated spending elasticity was 0.061, which translated into an incremental cost per quality-adjusted life-year of approximately €34,000. The bias-corrected elasticity was 0.075, with a corresponding incremental cost per quality-adjusted life-year of €27,000. We found that the estimated impact of spending on health decreases when recent years of data are added, and that the extent of omitted variable bias appears to increase, particularly when adding the COVID-19 pandemic period.</p><p><strong>Conclusions: </strong>This study provides an updated estimation of the incremental cost per quality-adjusted life-year produced by the Spanish national health system. The estimates provided can be easily updatable as new data become accessible, and the methods applied might be transferable to other settings with similar available data.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"109-122"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724785/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505503","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":"Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability.","authors":"Marcelien H E Callenbach, Rick A Vreman, Christine Leopold, Aukje K Mantel-Teeuwisse, Wim G Goettsch","doi":"10.1007/s40273-024-01433-4","DOIUrl":"10.1007/s40273-024-01433-4","url":null,"abstract":"<p><strong>Objective: </strong>To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions.</p><p><strong>Methods: </strong>A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population. To demonstrate the potential of the framework, three case studies were used: onasemnogene abeparvovec (Zolgensma^®), brexucabtagene autoleucel (Tecartus^®) and etranacogene dezaparvovec (Hemgenix^®). A hypothetical case study was used to illustrate the output of the calculation tool.</p><p><strong>Results: </strong>Part 1 of the framework presents steps for matching a suitable reimbursement and payment model with the disease and treatment characteristics. The reimbursement and payment models are further specified in Part 2. Part 3 guides end users through the setup of a calculation tool with which the financial impact can be calculated of two payment models: a price discount model and an outcome-based spread payment model with a discount. Part 4 concerns the output of the calculation tool, showing how different payment models lead to different financial consequences under three assumptions of longer term effectiveness.</p><p><strong>Conclusions: </strong>The presented framework provides decision makers with insight into the financial consequences of their chosen payment model under different assumptions. This can aid reimbursement negotiations by clarifying the optimal choice given a therapy's characteristics.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"53-66"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724790/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142378071","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}