PharmacoEconomics Open最新文献

{"title":"Cost-Effectiveness Modelling of Multiple System Atrophy for Early Health Technology Assessment.","authors":"Tobias Sydendal Grand, Shijie Ren, Praveen Thokala, Stefano Zanigni, Daniel Oudin Åström, Aroussi Bidani, Günter Höglinger, Werner Poewe, Florian Krismer, Stephane Regnier","doi":"10.1007/s41669-025-00609-8","DOIUrl":"10.1007/s41669-025-00609-8","url":null,"abstract":"<p><strong>Background and objectives: </strong>Multiple system atrophy is a neurodegenerative and rapidly progressing disease. Although symptoms often overlap, diagnostic criteria define two motor phenotypes: cerebellar and Parkinsonian. Little is known about available health-economic parameters for multiple system atrophy, and it remains unclear if the current available data are sufficient to construct an early health technology assessment. This assessment considered (i) if data gaps can be minimised or cost-effectiveness modelling can be facilitated by conducting literature searches for disease analogues, (ii) if an early health technology assessment is feasible with the currently available data, and (iii) the potential for developing user-friendly model interfaces in rare diseases.</p><p><strong>Methods: </strong>Literature searches were conducted for economic evaluations and health-economic parameters (including costs, utilities, natural history of disease) for multiple system atrophy. The searches for disease analogues focussed on economic evaluations and showed a widespread use of functional or disability scales to inform model structures; we therefore used the unified multiple system atrophy rating scale part IV (global disability scale) to inform the model structure. Natural history of disease data were used to inform utilities and transition probabilities. Analyses of a hypothetical intervention were conducted from the perspective of the United Kingdom National Health Service with a cost-effectiveness threshold of £30,000 per quality-adjusted life year. The cost-effectiveness analysis was presented using a user-friendly interface in R Shiny, which provides stakeholders with an opportunity to explore results.</p><p><strong>Results: </strong>Cost-effectiveness analyses were not identified for multiple system atrophy; however, two cost-of-illness, 20 quality-of-life, and nine natural history of disease studies were identified. Health-economic parameters were estimated from registry data, such as utilities and transition probabilities. A cost-effectiveness analysis was constructed for a hypothetical intervention despite evidence shortcomings, for example, aggregated cost data. An R Shiny app with a user-friendly interface was developed for stakeholders to change inputs and evaluate results.</p><p><strong>Discussion: </strong>Literature searches for disease analogues proved useful for early modelling in multiple system atrophy, for example, to inform model structures, but surrogate data could only be used when sufficient granularity was available (e.g., micro costing). These findings suggest that early conceptual modelling can help identify data gaps and guide future evidence generation, such as selecting appropriate endpoints for natural history of disease studies. Cost-effectiveness results can be highly uncertain for rare diseases at these early stages and should be considered as part of an iterative modelling framework.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"179-196"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796084/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145422160","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness Analysis of Etrasimod Compared With Biologic Therapies for the Treatment of Patients with Moderately-to-Severely Active Ulcerative Colitis in Spain.","authors":"Iago Rodríguez-Lago, Fernando Muñoz Núñez, Alfredo J Lucendo, Alfonso De Lossada Juste, Ana Cabez, Alberto de la Cuadra-Grande, Itziar Oyagüez, Emilio Monte-Boquet","doi":"10.1007/s41669-025-00619-6","DOIUrl":"10.1007/s41669-025-00619-6","url":null,"abstract":"<p><strong>Background: </strong>Etrasimod (2 mg orally, once daily) is a novel advanced therapy (AT) for patients aged 16 years and older, with moderately-to-severely active ulcerative colitis (UC). This cost-effectiveness analysis compared etrasimod with biologics for the treatment of moderately-to-severely active UC within the Spanish healthcare system.</p><p><strong>Methods: </strong>A mixed model was used to estimate direct health-related costs (€, 2025) and outcomes (quality-adjusted life years [QALYs]) during the patient's lifetime (annual discount rate: 3%). The model simulated the course of UC, including induction and maintenance treatments, modeled by a decision tree and a Markov model. Model inputs were derived from scientific literature. All data were validated by a multidisciplinary panel of national experts. Etrasimod was compared against commonly used treatments in Spain in AT-naïve (vs adalimumab, golimumab, infliximab, intravenous vedolizumab [vedolizumab-IV], subcutaneous vedolizumab [vedolizumab-SC]) and AT-experienced patients (vs adalimumab, golimumab, infliximab, ustekinumab, vedolizumab-IV, vedolizumab-SC) by estimating incremental cost-effectiveness ratios.</p><p><strong>Results: </strong>Etrasimod yielded 19.505 QALYs and €600,289 per AT-naïve patient lifetime, being associated with more QALYs and cost savings than adalimumab (+0.042; -€35,084), golimumab (+0.107; -€39,424), infliximab (+0.039; -€49,514), vedolizumab-IV (+0.170; -€16,153) and vedolizumab-SC (+0.166; -€55,850). In AT-experienced patients, etrasimod produced 18.407 QALYs and €589,423/patient lifetime. In this population, etrasimod was also associated with more QALYs and cost-savings versus adalimumab (+0.013; -€20,850), ustekinumab (+0.018; -€39,354), vedolizumab-IV (+0.166; -€572) and vedolizumab-SC (+0.171; -€26,890). Etrasimod was dominant versus all biologic therapies. Deterministic and probabilistic sensitivity analyses varying all model inputs confirmed the robustness of the results (etrasimod dominancy).</p><p><strong>Conclusions: </strong>In AT-naïve and AT-experienced patients, etrasimod is a dominant therapy (cost savings, greater effectiveness) compared with commonly used ATs.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"121-142"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796078/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145530880","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness of Chimeric Antigen Receptor (CAR) T-Cell Therapy for Blood Cancers: An Updated Systematic Review.","authors":"Nishma Patel, Suzanne Farid, Manuel Gomes","doi":"10.1007/s41669-025-00614-x","DOIUrl":"10.1007/s41669-025-00614-x","url":null,"abstract":"<p><strong>Background: </strong>Chimeric antigen receptor (CAR) T-cell therapy is an area of rapid development, showing the promise of curing blood cancers. While substantial health gains may justify high costs, it is currently unclear the extent to which the overall cost effectiveness of these therapies is driven by i) context-specific factors, such willingness-to-pay thresholds and study perspective, or ii) important subgroups such as line of treatment and therapy product.</p><p><strong>Objective: </strong>This paper aims to critically review published evidence on the cost effectiveness of CAR T-cell therapies and assess the key factors that drive their cost effectiveness.</p><p><strong>Methods: </strong>We conducted a systematic review using PubMed, Scopus and Ovid (Embase) databases to identify full economic evaluations of CAR T-cell therapies published up to January 2024. One reviewer screened and extracted data from the studies and the second reviewer assessed a sample of the full-text studies against the inclusion/exclusion criteria. Studies were critically appraised using the CHEERS checklist. Cost data are presented in 2022 US dollars.</p><p><strong>Results: </strong>The review identified 45 full cost-effectiveness studies of CAR T-cell therapies. These studies considered a total of 92 treatment comparisons, which included tisagenlecleucel (n = 37), axicabtagene ciloleucel (n = 28), brexucabtagene autoleucel (n = 7), lisocabtagene maraleucel (n = 8), idecabtagene vicleucel (n = 6), ciltacabtagene autoleucel (n = 4) and relmacabtagene autoleucel (n = 2). Incremental cost ranged from - US$74,980 to US$714,178 and incremental quality-adjusted life year (QALY) gains ranged from - 0.02 to 10.77. The resulting cost-per-QALY-gained ratios ranged from - US$37,490,000 to US$7,972,845, and the range of willingness-to-pay (WTP) thresholds between US$36,184 to US$317,825. The price of CAR T-cell therapy represented 75% (mean US$391,060) of the total cost of CAR T-cell therapy but was not the sole factor influencing cost effectiveness. Hospitalisation made up 6% of the total cost (mean US$34,152), while adverse events accounted for 9% (mean US$47,350). Regression analysis indicated cost effectiveness did not change according to important clinical or contextual factors.</p><p><strong>Conclusions: </strong>The findings demonstrate that the cost effectiveness of CAR T-cell therapies is determined by a combination of factors: the relative difference between the cost of the CAR T-cell therapy and comparator, the magnitude of the QALY gains and the WTP thresholds. Their cost- effectiveness does not differ according to therapy product, line of treatment, or country.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"35-51"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796026/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145757129","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness Analysis of Abrocitinib Compared with Dupilumab for Adolescents With Severe Atopic Dermatitis in Spain.","authors":"Rosa María Romero Jiménez, Pedro Herranz Pinto, Minia Campos Domínguez, Alba Bellmunt, Neus Vidal-Vilar, María Del Pilar Fortes Soto, Alfonso De Lossada Juste","doi":"10.1007/s41669-025-00616-9","DOIUrl":"10.1007/s41669-025-00616-9","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"209-213"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796019/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145489466","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Venetoclax in Combination with Obinutuzumab in Previously Untreated Fit Patients with Chronic Lymphocytic Leukemia: A Canadian Cost-Utility Analysis.","authors":"Gijs van de Wetering, Carolyn Owen, Versha Banerji, Ariane Gosselin, Shivani Kamdar, Nancy Paul Roc, Lytske Bakker, Gargi Ratnaparkhi, Stephane Barakat, Colin Vicente, Beenish S Manzoor","doi":"10.1007/s41669-025-00610-1","DOIUrl":"10.1007/s41669-025-00610-1","url":null,"abstract":"<p><strong>Background/objective: </strong>Chronic lymphocytic leukemia (CLL) is the most common lymphoproliferative disorder diagnosed in Canada and is associated with a significant economic burden. This study evaluates the cost effectiveness of fixed-treatment duration venetoclax plus obinutuzumab (VEN + O) for previously untreated, fit CLL patients in Canada, for whom this therapy is not universally reimbursed provincially.</p><p><strong>Methods: </strong>A three-state partitioned survival model was developed using data from the CLL13 trial and a Bayesian network meta-analysis. Health states included progression-free, progressed disease, and death. A lifetime horizon (40-year) was used and a 1.5% discount rate was applied to costs and effects. Costs included drug acquisition, administration, monitoring, adverse events, subsequent treatment, and terminal care. Utility values were derived from previous NICE technology appraisals in CLL. Extensive sensitivity and scenario analyses were conducted.</p><p><strong>Results: </strong>VEN + O was associated with lower total costs and higher quality-adjusted life years and thus dominant compared with ibrutinib, zanubrutinib, and fludarabine plus cyclophosphamide plus rituximab. Compared with acalabrutinib and venetoclax plus ibrutinib, the incremental cost-utility ratios (ICURs) were in the south-west quadrant and substantially above a CA$50,000/QALY willingness-to-pay threshold, meaning VEN + O was cost effective versus these comparators. VEN + O was cost effective versus bendamustine plus rituximab. Extensive sensitivity and scenario analyses confirmed the robustness of these results.</p><p><strong>Conclusions: </strong>This analysis demonstrates that VEN + O, a 12-month fixed duration treatment, is a cost-effective option for previously untreated fit CLL patients in Canada. VEN + O offers potential health benefits and cost savings when compared with relevant comparator treatments.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"153-165"},"PeriodicalIF":2.1,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12796052/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145471616","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acknowledgement to Reviewers.","authors":"","doi":"10.1007/s41669-025-00630-x","DOIUrl":"https://doi.org/10.1007/s41669-025-00630-x","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145714991","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost Effectiveness of Recombinant Zoster Vaccine and Live-Attenuated Vaccine Against Herpes Zoster for Adults Aged 50 and Over in China.","authors":"Lulu Liu, Qilin Zhang, Yamin Shu, Lei Wang, Zhenqingyun Shuai, Mingming Chu, Zhe Zhang, Rong Zhang","doi":"10.1007/s41669-025-00604-z","DOIUrl":"10.1007/s41669-025-00604-z","url":null,"abstract":"<p><strong>Objective: </strong>Two vaccines against herpes zoster (HZ) are currently authorized for use in China: the adjuvanted recombinant zoster vaccine (RZV) and live-attenuated Zoster Vaccine Live (ZVL). The significant disparities in prices and efficacy between the two vaccines necessitate an evaluation of their relative value in order to make an informed choice. This study aimed to evaluate the comparative cost effectiveness of RZV, ZVL, and no vaccination for older adults at different ages from the societal perspective.</p><p><strong>Methods: </strong>The Markov decision model was parameterized using vaccine efficacy data, the latest incidence rates, costs, and health-related quality-of-life data. Participants consisted of a cohort comprising adults aged ≥ 50 years in China. The lifetime health impacts, cost, life-year (LY), quality-adjusted life-year (QALY) gained and the incremental cost-effectiveness ratio (ICER), net monetary benefits (NMB), net health benefits (NHB), and expected value of perfect information (EVPI) were calculated. Sensitivity analyses, scenario analyses, and subgroup analyses were performed.</p><p><strong>Results: </strong>Compared with no vaccination, RZV and ZVL could avoid 2,009,292 versus 510,192 HZ cases, and 334,637 versus 90,996 post-herpetic neuralgia (PHN) cases in the overall cohort, respectively. RZV and ZVL were more costly by US$1624 million versus US$896 million compared with no vaccination, with an additional 58,180 versus 23,390 QALYs. At threefold gross domestic product per capita, RZV had cost-effectiveness probabilities of 47.37% for ages 50-59 years, 83.24% for ages 60-69 years, 28.28% for ages 70-79 years, and 51.93% for ages ≥ 80 years, while ZVL had 7.07%, 12.79%, 69.55%, 2.82% probability, and no vaccination had 45.56%, 3.97%, 2.17%, 45.25% probability of being cost effective for the corresponding age cohorts.</p><p><strong>Conclusions: </strong>The strategy utilizing RZV demonstrated superior efficacy in reducing the burden of HZ compared with ZVL. RZV was most cost effective in the 50-59, 60-69 and ≥ 80 years age groups, and ZVL was most cost effective in the 70-79 years group.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"887-902"},"PeriodicalIF":2.1,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12559535/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145023822","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An Introduction to Costing and the Types of Costs Used within Health Economic Studies.","authors":"Hugo C Turner, Juan Carlos Rivillas-Garcia, Shankar Prinja, Trinh Manh Hung, Saudamini Vishwanath Dabak, Brian A Asare, Mark Jit, Yot Teerawattananon","doi":"10.1007/s41669-025-00602-1","DOIUrl":"10.1007/s41669-025-00602-1","url":null,"abstract":"<p><p>The number of published health economic analyses, especially economic evaluations, has rapidly expanded globally since the 1990s, and costs are an essential component of such studies. Cost is a general term that refers to the value of the resources/inputs used to produce a good or service. However, within health economics, there are several different types of costs (such as financial, economic, unit, average, etc.). The terminology and application of these cost types often differ, leading to inconsistencies in the health economics literature. These inconsistencies create challenges in comparing studies and hinder the use of health economic analyses to effectively inform policy decisions. This paper aims to provide an up-to-date overview of the cost types, key cost terms, and definitions of different cost measures used within health economics, while highlighting key inconsistencies in the literature. We also discuss common adjustments made to cost data, such as accounting for inflation, discounting, and currency conversions, as well as the influence of economies of scale and scope on cost estimates. We highlight that the different definitions/categories for the different types of costs are not mutually exclusive and that the type of cost that should be used will depend on the purpose of the study, highlighting recommendations of what to do in practice where relevant. The content was tailored to be relevant across both high-income and LMIC contexts.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"849-868"},"PeriodicalIF":2.1,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12559562/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145329649","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimating Health State Utilities for IDH-Mutant Diffuse Glioma.","authors":"Ellen M Howard, Marc Massetti, Gin Nie Chua, Millie Gaydon, Sara Savar, Andrew Lloyd","doi":"10.1007/s41669-025-00603-0","DOIUrl":"10.1007/s41669-025-00603-0","url":null,"abstract":"<p><strong>Background: </strong>Isocitrate dehydrogenase-mutant (mIDH) gliomas are malignant central nervous system tumours. After initial resection, patients with mIDH gliomas with favourable prognosis may live without receiving oncologic treatment for years, but ultimately patients will experience recurrence and require radio- and/or chemotherapy (RT/CT). Cost-utility analyses (CUA) can explore the value of treatments that delay recurrence and initiation of RT/CT. This study aimed to estimate health state utilities for mIDH glioma over the full disease course.</p><p><strong>Method: </strong>Vignettes were developed to describe states from post-initial resection with no immediate need for RT/CT through to RT/CT and palliative care after recurrence. These were developed on the basis of literature review, patient-reported outcomes data from a clinical trial and qualitative interviews with clinicians and patients. The health state vignettes were valued by members of the UK public using the EQ-5D-5L and time trade-off methods. Subsequently, the vignette-derived utilities were re-weighted with the utility data from a clinical trial to estimate health state utilities for CUA.</p><p><strong>Results: </strong>Health state utilities were similar following the initial resection for targeted therapy or active observation (0.74) but lower in later stages of disease. Mean utilities for receiving first- and second-line RT/CT ranged from 0.34 to 0.55. Mean utilities ranged from 0.21 to 0.42 for recovery after tumour resection following recurrence and from -0.05 to 0.14 for palliative care.</p><p><strong>Conclusion: </strong>This study estimates health state utilities in glioma from after the initial resection through to palliative care and suggests that new treatments capable of delaying time to disease recurrence and initiation of RT/CT could produce a net gain in quality of life for individuals with mIDH glioma.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"973-984"},"PeriodicalIF":2.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12559505/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145008317","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identifying Potential Gaps in Care for Patients with Higher-Risk Myelodysplastic Syndromes in a Large US Health System.","authors":"Kristin J Moore, Nicole M Engel-Nitz, Peter McMahon, Jason Beal, Teraneh Z Jhaveri, Mellissa Williamson, Kate Andrade, Christina Steiger, Cosmina Hogea","doi":"10.1007/s41669-025-00600-3","DOIUrl":"10.1007/s41669-025-00600-3","url":null,"abstract":"<p><strong>Background: </strong>Higher-risk myelodysplastic syndromes (HR-MDS) may be difficult to diagnose because patients present with nonspecific signs and symptoms. This can prolong diagnosis, even though disease progression can occur quickly in HR-MDS.</p><p><strong>Objectives: </strong>This study identified places along the care journey where there are gaps in care in the identification, testing, diagnosis, and treatment of insured US patients with HR-MDS.</p><p><strong>Methods: </strong>This retrospective study utilized nationally representative US administrative claims and linked socioeconomic and social determinants of health (SDoH) data to characterize newly diagnosed patients with HR-MDS between 1 January 2017 and 30 April 2022.</p><p><strong>Results: </strong>The study included 1710 patients, of which a large proportion experienced some level of unmet need for modifiable SDoH characteristics. The median time between first sign or symptom and HR-MDS diagnosis was nearly the full look-back period of 12 months. Fewer than half of the patients received guideline-recommended treatment. Few patients had clinical trial participation.</p><p><strong>Conclusions: </strong>Opportunities to improve care include designing programs that support SDoH needs of patients (such as transportation access or social isolation), shortening the time between initial signs or symptoms and diagnosis and improving access to clinical trial participation.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"985-996"},"PeriodicalIF":2.1,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12559494/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144963957","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}