PharmacoEconomics最新文献

{"title":"Developing a Comprehensive Framework for Cost-Effectiveness Evaluation in Metastatic Castration-Sensitive Prostate Cancer: Insights from a Systematic Review.","authors":"Christopher G Fawsitt, Elaine Gallagher, Alka Singh, Hannah Baker, Edward Kayongo, Howard Thom, Noman Paracha","doi":"10.1007/s40273-025-01532-w","DOIUrl":"https://doi.org/10.1007/s40273-025-01532-w","url":null,"abstract":"<p><strong>Background and objectives: </strong>Metastatic castration-sensitive prostate cancer (mCSPC) imposes a significant economic burden and necessitates more cost-effective treatment strategies. The variability among the components of published economic evaluation models leads to methodological inconsistencies, underscoring the need for an optimal framework to minimise unwarranted structural variation. This paper reviews existing economic evaluations, establishes a comprehensive framework and aims to support future economic evaluations and decision-making in mCSPC.</p><p><strong>Methods: </strong>A systematic literature review (SLR) was conducted to identify relevant economic evaluations in mCSPC. Health technology assessments (HTAs) by the National Institute for Health and Care Excellence, and Canada's Drug Agency were reviewed to gather insights on critiques and limitations. On the basis of these findings, a comprehensive cost-effectiveness modelling framework was established. Furthermore, two additional SLRs were conducted to identify cost and resource utilisation inputs, as well as health state utility scores derived from published studies and HTA assessments.</p><p><strong>Results: </strong>Markov models and partitioned survival models (PSMs) were commonly reported in literature and published HTA evaluations. Despite the strong precedence of PSMs, we propose an optimal framework for mCSPC utilising a semi-Markov structure. This approach offers increased flexibility, allowing transition rates from progressed states to depend on time since progression occurred. We also present key sources of cost and utility data identified in the SLR.</p><p><strong>Discussion: </strong>This work aligns with methodologies recommended by the Innovative Medicine Initiative (IMI) PIONEER external group and published studies. The optimal framework, including healthcare resource utilisation and utility data, consolidates existing modelling precedents in mCSPC and will assist the cost-effectiveness assessment of treatments for this condition.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144874459","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":"Lifetime Healthcare and Long-Term Care Costs of Heart Failure: Estimates Using Administrative Data from Hospitalized Patients in the Netherlands.","authors":"Hamraz Mokri, Pieter van Baal, Maureen Rutten-van Mölken","doi":"10.1007/s40273-025-01533-9","DOIUrl":"https://doi.org/10.1007/s40273-025-01533-9","url":null,"abstract":"<p><strong>Background and objective: </strong>Heart failure (HF) is a complex clinical syndrome associated with high mortality and extensive healthcare use. Using longitudinal data, we aimed to estimate the lifetime healthcare and long-term care (LTC) use and costs of Dutch patients with HF.</p><p><strong>Methods: </strong>We used linked administrative data on mortality, LTC, and healthcare use covering the entire Dutch population for the period 2013-2024. Newly diagnosed patients with HF were defined as patients who were not hospitalized for HF 2 years before their index hospitalization for HF in 2015. Using regression modeling, we estimated hospitalized patients' life expectancy and lifetime healthcare costs as a function of age, sex, comorbidity, and income (all costs were adjusted to 2021 values).</p><p><strong>Results: </strong>We identified 21,011 unique hospitalized patients with HF (mean age 80 years), of whom 86% died during follow-up. Estimated lifetime total healthcare and LTC costs varied between €35,000 and €170,000, depending on patient characteristics. Lifetime LTC costs varied between €9000 and €50,000. While comorbidity affects life expectancy substantially, it did not have a strong impact on lifetime costs. Income level affects costs more than comorbidities, and lower-income groups incur higher lifetime LTC costs.</p><p><strong>Conclusions: </strong>Despite people with lower incomes having shorter lifespans than those with higher incomes, their lifetime LTC costs are higher. However, people with higher incomes have higher hospital costs, partly owing to their longer life expectancy.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144874461","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":"Bayesian Cost-Effectiveness Analysis Using Individual-Level Data is Sensitive to the Choice of Uniform Priors on the Standard Deviations for Costs in Log-Normal Models.","authors":"Xiaoxiao Ling, Andrea Gabrio, Gianluca Baio","doi":"10.1007/s40273-025-01511-1","DOIUrl":"https://doi.org/10.1007/s40273-025-01511-1","url":null,"abstract":"<p><strong>Background: </strong>Bayesian cost-effectiveness analysis (CEA) requires the specification of prior distributions for all parameters to be empirically estimated via Bayes' rule. When costs are modelled via Log-Normal distributions, Uniform prior distributions are commonly applied on the logarithm-scale standard deviations for costs due to the ease of implementation. However, the consequences of placing wide Uniform priors on standard deviations of log costs for the interpretation of original-scale CEA results remain unclear. The purpose of our study is to explore the impact of using Uniform priors for the standard deviations of cost data on CEA conclusions when costs are assumed to be log-normally distributed.</p><p><strong>Methods: </strong>The analysis has been performed using individual-level cost-utility data from a randomised controlled trial. Costs are initially jointly modelled with quality-adjusted life years (QALYs) using Log-Normal and Beta distributions, respectively. Uniform prior distributions with different upper bounds are applied to log-scale standard deviations in the cost Log-Normal model. We compare the performance of Uniform priors under the Log-Normal distribution with other distributional assumptions for costs. A simulation study has then been conducted to explore the impact of these models and prior choices on cost estimates in CEAs.</p><p><strong>Results: </strong>Results show that the choice of Uniform priors on standard deviations of log costs in a Log-Normal model can substantially induce large fluctuations in cost estimates, and thus potentially affect the final estimates of the intervention being cost-effective compared with other distributional assumptions. This is potentially driven by the occurrence of zero values in cost data.</p><p><strong>Conclusion: </strong>Bayesian CEAs may be sensitive to the choice of upper bounds of the Uniform priors for the standard deviations of log costs in Log-Normal models, particularly when data contain zero values. Our results suggest that caution should be taken when Uniform distributions with large upper bounds are used.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144822235","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":"Surrogate Outcomes Used as Proxies in Rare Long-Term Conditions: Evidence Assessment Group Perspective.","authors":"Eugenie Evelynne Johnson, Giovany Orozco-Leal, Aalya Al-Assaf, Hangjian Wu, Opeyemi Agbeleye, Sedighe Hosseini-Jebeli, Tumi Sotire, Emma Dobson, Sheila A Wallace, Fiona Pearson, Stephen Rice","doi":"10.1007/s40273-025-01525-9","DOIUrl":"https://doi.org/10.1007/s40273-025-01525-9","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144804518","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":"Evaluating the Role and Policy Implications of Using External Evidence in Survival Extrapolations: A Case Study of Axicabtagene Ciloleucel Therapy for Second-Line DLBCL.","authors":"Sam Harper, Daniela Afonso, Karina Watts, Brett Doble, Oskar Eklund, Sachin Vadgama, Julia Thornton Snider, Stephen Palmer, Matthew Taylor","doi":"10.1007/s40273-025-01529-5","DOIUrl":"10.1007/s40273-025-01529-5","url":null,"abstract":"<p><strong>Background and objective: </strong>Health technology assessment (HTA) of haemato-oncology therapies typically requires extrapolation of long-term survival beyond a trial's follow-up. Health technology assessment agencies must balance caution around uncertainty in early follow-up trial data whilst aiming to provide timely access. This study qualitatively and quantitatively assessed how eight HTA agencies considered maturing data and external evidence.</p><p><strong>Methods: </strong>The eight HTA appraisals were based on ZUMA-7, a phase III trial for axicabtagene ciloleucel (axi-cel) for second-line diffuse large B-cell lymphoma. ZUMA-7 survival data were submitted with either a 25-month ('Interim') or 47-month ('Primary') follow-up. To inform axi-cel Interim survival extrapolations, external evidence was available from a prior mature single-arm trial for third-line or later diffuse large B-cell lymphoma (ZUMA-1). A qualitative assessment of eight different submissions to HTA agencies was undertaken to determine key discussion points. The value and cost of waiting for evidence to mature between Interim and Primary analyses were quantified using value of information methods to evaluate the impact of waiting for further evidence collection on population health.</p><p><strong>Results: </strong>Agencies used varied approaches to account for uncertainty in survival extrapolations in both Interim and Primary analyses. No agency considered external evidence fully during Interim submissions; one used it partially to inform clinical plausibility; four did not consider it. Health technology assessment agencies that did not consider the relevance of ZUMA-1 were more inclined to wait for more mature evidence to mitigate uncertainty. When ZUMA-1 aided in determining a plausible range for Interim extrapolations, the less valuable more mature evidence became, with the cost of waiting for Primary analysis results exceeding the value conferred.</p><p><strong>Conclusions: </strong>There was limited consideration of external evidence during the included HTA submissions. In the future, it is recommended that external evidence should be considered to a greater degree by both manufacturers and HTA agencies when extrapolating survival to ensure appropriate and timely HTA decisions that minimise the undue burden on healthcare systems.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.6,"publicationDate":"2025-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144799867","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":"Validation of the Cancer-Specific Preference-Based Measure EORTC QLU-C10D against the Generic Instruments EQ-5D-5L and SF-6Dv2 in a Prospectively Collected Sample of Patients with Cancer in Austria and France.","authors":"Micha J Pilz, Simone Seyringer, Virginie Nerich, Madeleine T King, Richard Norman, Eva M Gamper","doi":"10.1007/s40273-025-01501-3","DOIUrl":"10.1007/s40273-025-01501-3","url":null,"abstract":"<p><strong>Background: </strong>The Quality of Life Utility - Core 10 Dimensions (QLU-C10D) is a disease-specific preference-based measure (PBM) designed to obtain health state utility values from patients with cancer. Previously, satisfactory psychometric properties were established from retrospective trial analyses using clinical anchors. This study aimed to validate the QLU-C10D against two generic PBMs in a prospective sample of Austrian and French patients with cancer using patient-reported anchors.</p><p><strong>Methods: </strong>Patients completed the European Organisation of Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 (QLQ-C30), EQ-5D-5L and Short Form 36 (SF-36) at study baseline (any time during anti-cancer treatment) plus a follow-up assessment 3-6 months later. Sociodemographic and clinical characteristics were assessed. QLU-C10D and SF-6Dv2 utilities were calculated from QLQ-C30 and SF-36 data, respectively. German, French and UK value sets were applied for all three PBMs. Floor and ceiling effects were assessed. Known-group validity (independent t-test) and responsiveness (paired t-tests) were assessed respectively by ability to detect health status differences and changes over time according to patient-rated overall quality of life/health perception assessed by the QLQ-C30 Global Health Status scale, the EQ-5D-5L VAS and the SF-36 General Health scale.</p><p><strong>Results: </strong>A total of 465 patients were included in the analysis. QLU-C10D index scores (intra-class correlation) and domains (Pearson) were correlated with EQ-5D-5L and Short-Form Six Dimensions (SF-6Dv2) conceptual counterparts. Correlation coefficients for the index scores of QLU-C10D and the generic PBMs ranged from 0.63 to 0.81. The QLU-C10D detected statistically significant differences between groups at baseline in 100% of tests performed (n = 27). For changes over time, QLU-C10D detected expected effects in 68% of cases (n = 29). In comparison with the generic PBMs, QLU-C10D detected differences and changes with a higher statistical efficiency in 76% of cases (77 of 102).</p><p><strong>Conclusions: </strong>The QLU-C10D is a fit-for-purpose ready-to-use PBM to estimate health state utilities of patients with cancer. This study adds to evidence that QLU-C10D has appropriate psychometric properties and appears to have higher statistical efficiency than generic PBMs in cancer.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"937-953"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255563/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031413","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":"Under-reporting of Validation Efforts for Health Economic Models Persists Despite the Availability of Validation Tools: A Systematic Review.","authors":"Katharina Abraham, Isaac Corro Ramos, C Louwrens Braal, Talitha Feenstra, Anne Kleijburg, George A K van Voorn, Carin Uyl-de Groot","doi":"10.1007/s40273-025-01491-2","DOIUrl":"10.1007/s40273-025-01491-2","url":null,"abstract":"<p><strong>Objective: </strong>In this study we aimed to identify possible changes over time in validation efforts and the way in which they are reported for model-based health economic (HE) evaluations, given the introduction of several new validation tools and methods in the past decade.</p><p><strong>Methods: </strong>A systematic review was conducted using PubMed and Embase on published HE models for early breast cancer (EBC) for the period 2016 to 2024. AdViSHE-consisting of four validation categories that cover the main HE model aspects-was utilized to systematically evaluate the reported evaluation efforts. The percentage of studies reporting validation per category was compared with the review by de Boer et al. that covers the years 2008 to 2015.</p><p><strong>Results: </strong>Of the 2199 records, 78 studies fulfilled the eligibility criteria. Reported validation efforts did not significantly improve compared with the previous period, except for the validation of input data by experts. Reporting on the validation of the conceptual model remained low with around 10% of the studies providing validation. Validation of the computerized model and validation against outcomes using alternative input data were the most underreported validation categories with < 4% of the studies. The validation of model outcomes, specifically cross validity and the comparison with empirical data, remained the most reported categories in this review also, with 52% and 36%, respectively. When validation efforts were reported, this was done in a non-systematic manner, and the tests and results were rarely detailed.</p><p><strong>Conclusion: </strong>Overall reporting of validation efforts for model-based HE evaluations in the past decade did not significantly change compared with the previous decade.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"849-858"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255658/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144008428","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 Evolving Landscape of Discrete Choice Experiments in Health Economics: A Systematic Review.","authors":"Sven Petrus Henricus Nouwens, Stella Maria Marceta, Michael Bui, Daisy Maria Alberta Hendrika van Dijk, Catharina Gerarda Maria Groothuis-Oudshoorn, Jorien Veldwijk, Janine Astrid van Til, Esther Wilhelmina de Bekker-Grob","doi":"10.1007/s40273-025-01495-y","DOIUrl":"10.1007/s40273-025-01495-y","url":null,"abstract":"<p><strong>Introduction: </strong>Stakeholder preference evaluations are increasingly emphasized in healthcare policy and health technology assessment. Discrete choice experiments (DCEs) are the most common method for quantifying preferences among patients, the public, and healthcare professionals. While prior reviews (1990-2017) have examined DCE trends, no comprehensive synthesis exists for studies published since 2018. This updated review (2018-2023) provides critical insights into evolving methodologies and global trends in health-related DCEs.</p><p><strong>Methods: </strong>A systematic search (2018-2023) of Medline, Embase, and Web of Science identified relevant studies. Studies were screened for inclusion and data were extracted, including details on DCE design and analysis. To enable trend comparisons, the search strategy and extraction items aligned with previous reviews.</p><p><strong>Results: </strong>Of 2663 identified papers, 1279 met the inclusion criteria, reflecting a significant rise in published DCEs over time. DCEs were conducted globally, with a remarkable increase in publications from Asia and Africa compared with previous reviews. Experimental designs and econometric models have advanced, continuing prior trends. Notably, most recent DCEs were administered online.</p><p><strong>Discussion: </strong>The rapid growth of DCE applications underscores their importance in health research. While the methodology is advancing rapidly, it is crucial that researchers provide full transparency in reporting their methods, particularly in detailing experimental designs and validity tests, which are too often overlooked. Key recommendations include improving reporting of experimental designs, applying validity tests, following good practices for presenting benefit-risk attributes, and adopting open science practices. Ensuring methodological rigor will maximize the impact and reproducibility of DCE research in health economics.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"879-936"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255651/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111597","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 EQ-5D in the Middle East and North Africa Region: A Systematic Literature Review.","authors":"Abeer Al Rabayah, Sibylle Puntscher, Fatima Al Sayah, Razan Sawalha, Elly Stolk, Judit Simon, Michael Drummond, Uwe Siebert","doi":"10.1007/s40273-025-01483-2","DOIUrl":"10.1007/s40273-025-01483-2","url":null,"abstract":"<p><strong>Introduction: </strong>The EQ-5D is the most commonly used preference-based measure of health-related quality of life. There is limited evidence about the use of the EQ-5D in the Middle East and North Africa (MENA) region. This study aimed to systematically identify, review, summarize, and synthesize the published literature on using the EQ-5D in this region.</p><p><strong>Methods: </strong>A systematic literature review was conducted, according to the PRISMA 2020 guidelines, using PubMed, Cochrane, PsycINFO, and CINAHL and covering the period up to 30 August 2024. Studies using any version of the EQ-5D in adults or youth in the MENA region were included. Pilot studies, guidelines, study protocols, and reviews were excluded. Key study characteristics and outcomes assessed included study design, clinical area, population, type of EQ-5D data reported, reference value set used, and mode of administration. Title/abstract screening was conducted independently by two reviewers to assess eligibility for inclusion. Two researchers completed full-text screening and extracted data using a standardized form. Disagreements were referred to a third reviewer if not resolved by discussion. Results were summarized in systematic evidence tables.</p><p><strong>Results: </strong>After removing duplicates, 18,034 references were considered for title/abstract screening. In total, 184 studies were included with a total sample size of 128,164 subjects. Of the included single-country studies, 42% were reported in Iran, 20% in Saudi Arabia, and 11% in Jordan. Patient populations were investigated in 86% of the studies, 23% of which targeted endocrine diseases. Study design was observational in 57% and experimental in 14% of the studies. Only 10% of the included studies applied the EQ-5D in an economic evaluation. The EQ-5D-3L version was used in 40% of the studies. However, the trend is towards a greater use of the 5L version in more recent years. Twenty percent of the studies reported EQ-5D results using the index score, frequencies of severity levels per dimension, and visual analog scale scores. EQ-5D modes of administration and funding sources were not reported in 16% and 20% of the studies, respectively.</p><p><strong>Conclusion: </strong>There is an increased use of the EQ-5D in the MENA region, especially since 2020. In the region, the use of the EQ-5D is more prevalent in clinical studies than in economic evaluation studies. The reporting heterogeneity indicates the need for guidance in reporting EQ-5D study results in this region.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"859-877"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255660/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143992196","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":"Societal Versus Healthcare Perspectives on the Cost Effectiveness of Ocrelizumab for Treatment of Primary Progressive Multiple Sclerosis in Aotearoa New Zealand.","authors":"Richard J Milne, Carsten Schousboe, Julie A Campbell, John Mottershead","doi":"10.1007/s40273-025-01486-z","DOIUrl":"10.1007/s40273-025-01486-z","url":null,"abstract":"<p><strong>Objectives: </strong>Multiple sclerosis (MS) is a progressive, degenerative, autoimmune neuronal disease. This study compares the impact of a societal versus a healthcare perspective on the cost effectiveness of treatment of primary progressive MS (PPMS) with ocrelizumab (OCR) versus best supportive care (BSC) in New Zealand.</p><p><strong>Methods: </strong>The analysis utilises a lifetime cohort Markov model based on ten Expanded Disability Status Scale (EDSS) states, plus death. It has two structurally identical arms, with forward transition probabilities in the treatment arm obtained by multiplying forward transition probabilities (converted to rates, and back to probabilities) in the control arm by the 12-week disability progression hazard ratio in the clinical trial ORATORIO. Direct and indirect costs of MS were estimated from a 2017 Australian survey and converted to 2024 NZ dollars using purchasing power parity and the NZ consumer price index. Future costs and benefits were discounted at 3.5% per annum. The model is calibrated to NZ mortality for PPMS, and therapy ends when wheelchair dependence (EDSS7) is reached.</p><p><strong>Results: </strong>For a modelled cohort 40 years of age starting at the ORATORIO distribution of EDSS, at 50% of the list price (viz. 50% rebate on $NZ37,384 per patient per annum), the incremental cost-effectiveness ratio (ICER) of OCR versus BSC is $NZ114,427 per QALY ($US64,651) from a societal perspective or $NZ146,711 ($US82,892) from a healthcare perspective. From a societal perspective, at Treasury's willingness to pay (WTP) criterion of $NZ120,200, an acquisition cost up to 56% of list price ($NZ20,935; 44% rebate) is justifiable. From a healthcare payer perspective, at Treasury's implied WTP of $NZ43,313, an acquisition cost up to 10% of list price ($NZ3738; 90% rebate) is justifiable. Based on this metric, a 5.6-fold higher investment in OCR can be justified from a societal perspective compared with a healthcare perspective. Alternatively, an acquisition cost up to 37.9% of list price (viz. 62.1% rebate or $NZ14,169) could be justified if the criterion of one GDP per capita ($NZ83,011) is used as a societal funding threshold. These results are sensitive to the cost of BSC from a societal perspective but not from a healthcare perspective. From both perspectives the cost effectiveness is sensitive to the acquisition cost and efficacy of OCR, potential waning of efficacy, the age and EDSS state when therapy begins, the cost and timing of entry of a biosimilar pharmaceutical and the discount rate.</p><p><strong>Conclusions: </strong>Treatment of PPMS with OCR is more cost effective from a societal than a healthcare perspective, therefore prioritisation of public funding of novel pharmaceuticals for MS and other resource intensive chronic health conditions will depend critically upon the study perspective.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"969-985"},"PeriodicalIF":4.4,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12255576/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143975564","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}