ClinicoEconomics and Outcomes Research最新文献

{"title":"Economic-Clinical Burdens of Tuberculosis Treatment in Vulnerable Patients in a Provincial General Referral Hospital in Lubumbashi, DR Congo.","authors":"Michael Mika Mukanya, Edouard Mbaya Munianji, Chadrack Kabeya Diyoka, Laetitia Mwanvua Ngongo, Noémie Kisimba Kapala, Criss Koba Mjumbe","doi":"10.2147/CEOR.S551874","DOIUrl":"https://doi.org/10.2147/CEOR.S551874","url":null,"abstract":"<p><strong>Background: </strong>According to an analysis of the catastrophic costs incurred by people with tuberculosis, 56% of households in the Democratic Republic of Congo bear the economic burden of tuberculosis. The objective of this study was to determine the clinical and economic burdens of tuberculosis treatment in vulnerable patients.</p><p><strong>Methods: </strong>A descriptive, cross-sectional study was conducted at Janson Sendwe Provincial General Referral Hospital between September and December 2023, a four-month period. Data collection was based on a previously developed survey form with several parameters.</p><p><strong>Results: </strong>In this study, 59 tuberculosis cases with vulnerability factors were recorded. Male sex was the most prevalent with 62.71%, the average patient age was 33.37 ± 9.92 (15-68) years, TB/HIV coinfection was the most prominent with 94.92%, the average total cost was 56.74 ± 20.81 USD, direct costs were 10.61 ± 0.00 and indirect costs were 47.75 ± 19.23 USD. The average direct costs were 1.56 ± 0.19 USD.</p><p><strong>Conclusion: </strong>Catastrophic costs for tuberculosis patients can be addressed by improving the delivery and financing of tuberculosis services and strengthening social protection for tuberculosis patients.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"18 ","pages":"551874"},"PeriodicalIF":2.2,"publicationDate":"2026-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12997766/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147487763","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":"Economic Evaluation of Ready-to-Dilute Thiotepa (Tepylute^®): Institutional Cost Offsets and Time Savings Compared to Lyophilized Thiotepa in a United States Hospital Pharmacy.","authors":"Orlaith Ryan, Maurice Clancy, Alanna MacDonald, Laura Pastor, Megan Bourque","doi":"10.2147/CEOR.S559251","DOIUrl":"https://doi.org/10.2147/CEOR.S559251","url":null,"abstract":"<p><strong>Purpose: </strong>Thiotepa is an intravenous alkylating agent used with other chemotherapy drugs to treat several forms of cancers. Preparation of thiotepa requires reconstitution of powder in sterile water, followed by dilution of the solution with sodium chloride 0.9% solution for injection. The reconstitution of lyophilized powdered drug formulations may pose challenges as it can increase the risk of preparation errors, leading to wastage or potential clinical consequences, and extend preparation time compared to ready-to-dilute formulations; one study found that 15% of thiotepa doses were made out-of-tolerance. Tepylute^® is a ready-to-dilute formulation of thiotepa and has been approved by the United States Food and Drug Administration for the treatment of breast and ovarian adenocarcinoma. The objective of this study was to explore the economic impact to a hospital pharmacy of using ready-to-dilute Tepylute^® versus a lyophilized powdered formulation of thiotepa.</p><p><strong>Patients and methods: </strong>A cost offset model was created in Microsoft Excel™, the analysis compared a current scenario (thiotepa: 100% market share) with a future scenario (Tepylute^®: 100% market share) for a hospital pharmacy treating 96 cancer patients annually. Costs (2024 USD) included drug acquisition, compounding time, drug wastage, and clinical consequences due to reconstitution errors. Incremental costs, cost offsets, and pharmacy time saved were evaluated.</p><p><strong>Results: </strong>For a hypothetical hospital pharmacy, use of Tepylute^® versus a lyophilized powdered formulation of thiotepa resulted in savings of $339,777 over 1-year ($3539 per patient), primarily from reduced drug acquisition costs, as well as a reduction in drug wastage, clinical consequences due to reconstitution errors, and compounding time. Results were consistent across sensitivity and scenario analyses.</p><p><strong>Conclusion: </strong>Modeled results demonstrate that ready-to-dilute Tepylute^® may save time, reduce acquisition and preparation costs, and lower clinical risks from preparation errors. This study is among the first to quantify potential savings from switching to a ready-to-dilute formulation.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"18 ","pages":"559251"},"PeriodicalIF":2.2,"publicationDate":"2026-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12997768/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147487714","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":"Creating A Multi-Criteria Decision Analysis Tool to Enhance Value-Based Purchasing of Generic and Biosimilar Medications in Saudi Arabia: A Pilot Study.","authors":"Yazed Alruthia, Horiyah Alhajri, Omar A Almohammed, Bander Balkhi, Mai F Alsaqa'aby, Mohammed A Almuqbil, Hana A Al-Abdulkarim, Abdulaali R Almutairi","doi":"10.2147/CEOR.S568722","DOIUrl":"10.2147/CEOR.S568722","url":null,"abstract":"<p><strong>Background: </strong>Saudi Arabia is reforming its healthcare system to promote the use of generic and biosimilar medications to improve spending efficiency. However, concerns regarding therapeutic equivalence persist. This study aims to establish a stakeholder-driven, value-based procurement framework for off-patent pharmaceuticals (OPPs).</p><p><strong>Methods: </strong>The Saudi chapter of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) convened stakeholders to define procurement criteria. Participants reviewed 29 potential criteria during a workshop, followed by a multi-criteria decision analysis (MCDA) simple scoring exercise and conjoint analysis to determine the relative weight of essential factors.</p><p><strong>Results: </strong>Forty-nine stakeholders participated, primarily pharmacists (57%) and regulators (27%). Sixteen criteria were identified as important: published effectiveness data, drug delivery systems, bioequivalence, real-world data generation, drug stability, direct costs, supply track record, labeled indications, drug safety profile, quality assurance of production, interchangeability, contribution to national healthcare priorities, availability in reference countries, manufacturing site quality certification, pharmaceutical equivalence, and pharmacovigilance. Effectiveness data received the highest weight (11.56%), while pharmacovigilance received the lowest (3.23%).</p><p><strong>Conclusion: </strong>This pilot study establishes the first consensus-based MCDA criteria for Saudi Arabia, prioritizing effectiveness and drug stability over costs. It emphasizes scientific validity over price, serving as a foundation for a national value-based procurement system, though further research is needed to test the framework in healthcare tenders.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"18 ","pages":"568722"},"PeriodicalIF":2.2,"publicationDate":"2026-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12790905/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145960436","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":"Adherence to Acthar Gel (RCI) and Outcomes in Patients with Membranous Nephropathy: A Claims Database Analysis.","authors":"Kyle Hayes, Mohammed Fahim, Feng Sheng Hu, Mary P Panaccio, George J Wan","doi":"10.2147/CEOR.S530426","DOIUrl":"https://doi.org/10.2147/CEOR.S530426","url":null,"abstract":"<p><strong>Purpose: </strong>To describe real-world patient characteristics and outcomes after initiation of Acthar Gel (repository corticotropin injection, RCI) in the management of nephrotic syndrome (NS). This real-world study characterized patients with membranous nephropathy (MN) who initiated RCI therapy and evaluated the impact of higher versus lower adherence to RCI on NS-related outcomes, healthcare resource utilization, and medical costs.</p><p><strong>Patients and methods: </strong>We retrospectively analyzed data from the Integrated Dataverse from Symphony Health database. Adult (≥18 years) patients were included if they had ≥1 inpatient or ≥2 outpatient claims for an NS and MN diagnosis (January 1, 2016-December 31, 2022); ≥1 prescription claim for RCI; and continuous eligibility ≥12 months before and ≥12 months following and including the index date. Patients with ≥2 RCI claims and a calculated proportion of days covered (PDC) were included. Those with a PDC above the sample mean were classified as \"above-average\" adherence and those below the mean as \"below-average\" adherence in that period.</p><p><strong>Results: </strong>The analyzed population comprised 28 patients with below-average and 34 with above-average RCI adherence. The above-average adherence cohort showed a noticeable reduction compared with the below-average adherence cohort in corticosteroid use, extended corticosteroid use, opioid use, NS-related renal biopsy procedures, all-cause and NS-related inpatient hospitalizations, and NS-related office visits. From baseline to follow-up, inpatient hospitalization rates were similar in the below-average adherence cohort for all-cause (50.0% vs 53.6%) and NS-related (35.7% vs 28.6%) admissions but markedly lower in the above-average cohort for all-cause (50.0% vs 23.5%) and NS-related (41.2% vs 8.8%) hospitalizations. Despite similar baseline medical costs, all-cause ($131,306 vs $50,048) and NS-related costs ($111,743 vs $29,868) were noticeably higher at follow-up in the below-average adherence cohort than the above-average adherence cohort.</p><p><strong>Conclusion: </strong>This real-world study shows that greater adherence to RCI during treatment improves overall outcomes studied.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"18 ","pages":"530426"},"PeriodicalIF":2.2,"publicationDate":"2026-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12983396/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147469637","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":"Impacts and Economic Burden of Pompe Disease on Patients and Families in Thailand: A Mixed Method Study.","authors":"Sitaporn Youngkong, Montarat Thavorncharoensap, Usa Chaikledkaew, Chaisiri Luangsinsiri, Thipwimol Tim-Aroon, Chulaluck Kuptanon, Achara Sathienkijkanchai, Kitiwan Rojnueangnit, Khunton Wichajarn, Boonchai Boonyawat, Kanya Suphapeetiporn, Duangrurdee Wattanasirichaigoon","doi":"10.2147/CEOR.S542203","DOIUrl":"https://doi.org/10.2147/CEOR.S542203","url":null,"abstract":"<p><strong>Purpose: </strong>This study aimed to explore the impacts and economic burden experienced by patients and families affected by Pompe disease in Thailand.</p><p><strong>Patients and methods: </strong>A mixed-methods analysis was employed. To estimate the direct medical cost, databases and/or medical records of the seven rare disease (RD) centers in Thailand from 2010 to 2021 were reviewed. Structured interviews were also conducted to examine direct non-medical and indirect costs. The economic burden was presented in 2024 US dollars (USD) value (1 USD = 35.29 THB). In-depth interviews were conducted with four patients and two caregivers to explore the impacts of this disease.</p><p><strong>Results: </strong>Frequently reported symptoms included fatigue, muscle weakness, and difficulty breathing during sleep. These symptoms had significant and profound impacts on patients' functioning, family and social roles, self-esteem, emotion, and financial status. Frequent visits to the RD centers, along with the referral process, were reported as a substantial burden to the patients and families. The mean annual direct medical cost per patient, excluding the cost of enzyme replacement therapy, ranged between 2,505 and 14,042 USD from the provider's perspective and 1,484 USD from the patient's perspective. Direct non-medical costs were highly significant, with the annual cost of informal care of 1,878-1,992 USD per patient. Around 43% of the patients reported requiring informal care. On average, each patient required 2.6 ± 3.5 hours of informal care per day.</p><p><strong>Conclusion: </strong>Our findings revealed substantial impacts of Pompe disease on individuals' physical, social, emotional, and functional capacities as a result of its symptoms. Coordinated care, where patients can be treated by clinicians at local hospitals who operate in close liaison with specialists from the RD centers, is warranted. Psychosocial, welfare, and transportation supports are clearly justified to alleviate the burden and improve the quality of life of the patients.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"18 ","pages":"542203"},"PeriodicalIF":2.2,"publicationDate":"2026-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12983394/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147468671","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":"Consequences of Canada's Drug Agency Reimbursement Recommendations for New Medicines and Pan-Canadian Pharmaceutical Alliance Price Negotiations on Patient Access.","authors":"Nigel S B Rawson","doi":"10.2147/CEOR.S567932","DOIUrl":"10.2147/CEOR.S567932","url":null,"abstract":"<p><strong>Introduction: </strong>Prescription drugs are excluded from Canada's federal legislation covering health care. Each provincial government has developed its own drug plan. To get new prescription medicines listed in these plans, developers must pass regulatory review, health technology assessment and price negotiation, and convince individual government plans to list their drugs. The objective of this research is to assess how many reimbursement recommendations issued by Canada's Drug Agency (CDA) have clinical and/or price conditions and what the consequences are.</p><p><strong>Methods: </strong>Data were obtained on drugs with CDA recommendations issued between January 2020 and December 2024, together with dates of price negotiations between the pan-Canadian Pharmaceutical Alliance (pCPA) and manufacturers by the end of July 2025 and listings in government plans relating to the same drugs by early November 2025.</p><p><strong>Results: </strong>Of 344 CDA recommendations, only three (0.9%) were unconditional reimbursement, 291 (84.6%) reimbursement with clinical criteria and/or a price condition, and 50 (14.5%) no reimbursement. Median time for CDA reviews was 221 days (interquartile range (IQR): 199-282 days). Where recommended to achieve cost-effectiveness of $50,000/quality-adjusted life-year, median reduction was 74.5% (IQR: 50.0%-90.0%). Median time for the pCPA to decide whether to negotiate was 128 days (IQR: 73-191 days) and median negotiation time was 131 days (IQR: 82-219 days). The median time between submission to CDA and pCPA outcome was 518 days (IQR: 394-633 days). Government drug plan listing rates for drugs successfully negotiated with the pCPA ranged from 58.6% to 91.6%. Five patients had prior-authorization requests to a private insurer for costly drugs denied because the drugs had conditional CDA recommendations.</p><p><strong>Conclusion: </strong>CDA and pCPA processes take considerable time and listing decisions by government drug plans add extra time before potential access by patients. Nearly all CDA reimbursement recommendations, which are intended for government drug plans (not private payers), are conditional.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"17 ","pages":"975-989"},"PeriodicalIF":2.2,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12743471/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145851081","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":"Substantial Increase in the Costs of Antineoplastic Agents in the USA from 2010 to 2021.","authors":"Abdullah U Althemery","doi":"10.2147/CEOR.S548758","DOIUrl":"10.2147/CEOR.S548758","url":null,"abstract":"<p><strong>Purpose: </strong>This study provides one of the few national comparisons of antineoplastic expenditures from 2010 to 2021, offering new insight into how spending patterns have evolved over the past decade. In addition, it examines how out-of-pocket expenses relate to patient quality of life, an essential domain to evaluate healthcare interventions.</p><p><strong>Patients and methods: </strong>This study provides an updated estimate of the costs of antineoplastic agents using 2021 data and compares them with estimates from 2010. Medical Expenditures Panel Survey (MEPS) files were analyzed for national estimates. Antineoplastic treatment was defined in the MEPS using Multum Lexicon variables from the Cerner Multum. All reported prescriptions and refills were included in the expense and usage estimates. The 2010 costs were adjusted to 2021 figures using the consumer price index for out-of-pocket expenses and gross domestic products for third-party payers. SAS Studio 3.81 (Enterprise Edition) was employed for analysis.</p><p><strong>Results: </strong>Between 2010 and 2021, cancer diagnoses in the United States rose by 20.46%, while patients getting antineoplastic therapy increased by 7.6%. During this time, the cost of these medicines increased by thrice, from $9.78 billion to $35.12 billion. Prescription numbers were steady, with an average of four per patient per year. Males, older patients, and the insured were more likely to use the service. Breast cancer was the most common, with prostate and skin cancers increasing. The average prescription expenditure for cancer patients increased significantly compared to non-cancer individuals. Finally, there was no significant relationship observed between patients' quality of life and their out-of-pocket expenses.</p><p><strong>Conclusion: </strong>The substantial increase in cancer treatment expenses had no positive impact on patients' physical or mental well-being. This cost-quality gap necessitates a study on spending efficiency and patient well-being.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"17 ","pages":"965-974"},"PeriodicalIF":2.2,"publicationDate":"2025-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12722186/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145828860","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":"Economic Evaluation of Extended-Release Amphetamine (Dyanavel XR) Among Individuals with Attention-Deficit/Hyperactivity Disorder From a United States Societal Perspective.","authors":"Ishveen Chopra, Jennifer Horng, Melanie S Krause, Elli P Sellinger, Jim Potenziano","doi":"10.2147/CEOR.S575004","DOIUrl":"10.2147/CEOR.S575004","url":null,"abstract":"<p><strong>Introduction: </strong>In the treatment of attention-deficit/hyperactivity disorder (ADHD), supplemental use of immediate-release (IR) stimulants can lead to fluctuating plasma levels that impede symptom control and are more prone to diversion. The negative implications of IR stimulant supplementation are broad, leading to extra costs and harm to individuals and society. To quantify the economic impact of IR supplementation from a United States societal perspective, this study evaluated the incremental cost difference between Dyanavel XR and other extended-release (ER) stimulants over a one-year base-case horizon and summarized value using a benefit-cost ratio.</p><p><strong>Methods: </strong>As a hypothesis-generating approach, a decision-tree model compared these interventions across direct medical, non-medical, and indirect costs. Analysis was conducted for the general ADHD population and stratified by age group to account for differences in medication use, adherence, and costs. Suboptimal response combined IR supplementation and patient-reported end-of-dose crash, with all inputs drawn from the published literature.</p><p><strong>Results: </strong>The average per-person cost for Dyanavel XR was $43,219 versus $51,071 for other ER stimulants, resulting in savings of $7,852 per person over one year. Nationally, Dyanavel XR resulted in $44.6 billion in aggregate savings. The benefit-cost ratio of Dyanavel XR was 12.59, indicating that benefits outweighed the treatment cost; 16.89 in young adults and 0.89 in children. In one-way deterministic sensitivity analysis, Dyanavel XR remained cost-saving, highlighting the robustness of the economic benefit.</p><p><strong>Conclusion: </strong>From a United States societal perspective, Dyanavel XR demonstrates an economic advantage over other ER stimulants, with value most pronounced in young adults and lower in children. Dyanavel XR's potential to reduce IR supplementation and end-of-dose crash, thereby mitigating downstream costs, makes it a compelling ADHD treatment option. The model's operational definition of suboptimal response is intended as a pragmatic framework for future research to test its predictive validity for clinical outcomes and quality-of-life measures.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"17 ","pages":"945-964"},"PeriodicalIF":2.2,"publicationDate":"2025-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12718508/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145811472","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-Utility Analysis of Renal Replacement Therapy Modalities in the Management of Severe Acute Kidney Injury in US Critically Ill Patients.","authors":"Rui Martins, Jay Koyner, Ron Wald, Kai Harenski, Jorge Echeverri","doi":"10.2147/CEOR.S546850","DOIUrl":"10.2147/CEOR.S546850","url":null,"abstract":"<p><strong>Background: </strong>Acute kidney injury (AKI) is common among patients admitted to the intensive care unit (ICU), with 5-15% receiving renal replacement therapy (RRT). Continuous renal replacement therapy (CRRT) and intermittent hemodialysis (IHD) are well-established treatments for severe AKI, but renal recovery is variable and often incomplete, leading to long-term morbidity and mortality. The clinical and cost-effectiveness of either therapy are under active debate. This analysis aims to strengthen the evidence on the cost-utility of CRRT compared with IHD to manage severe AKI in ICU using a US third-party costing perspective.</p><p><strong>Methods: </strong>The analysis used a 90-day decision tree simulating hospital admission and a semi-Markov process with annual cycles and half-cycle correction to capture lifetime costs and outcomes, discounted at 3% annually. Survivors at 90 days either progressed to ESKD on dialysis (ESKD-D), with some receiving transplants, or became dialysis-independent. In the case of transplant failure, patients returned to ESKD-D. Tunnel states addressed Markov memoryless properties. A US-representative analysis of real-world data applying propensity score matching to control for selection bias informed the probability of lifetime dialysis dependence. Costs and utilities were sourced from peer-reviewed publications or national data. Uncertainty was investigated using deterministic and probabilistic sensitivity analyses.</p><p><strong>Results: </strong>In the base case, lifetime costs and quality-adjusted life-years (QALYs) were $273,314 and 5.681 for CRRT compared to $268,449 and 5.457 for IHD. CRRT had an 89.6% probability of being cost-effective ($23,860/QALY gained), being associated with 0.269 additional life-years. Long-term CKD management costs, accounting for 50% of CRRT's excess costs, significantly influenced results and were examined in scenario analyses.</p><p><strong>Conclusion: </strong>CRRT is likely a cost-effective option for managing severe AKI in the ICU compared with IHD. This study builds on existing economic evaluations by incorporating large comparative studies and exploring clinical uncertainty. The model highlights the need to clarify RRT's role in CKD progression and enhance post-AKI care to improve patient outcomes.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"17 ","pages":"931-944"},"PeriodicalIF":2.2,"publicationDate":"2025-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12717216/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145806045","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":"Analysis of Cost-Effectiveness of Biologic Therapies in Axial Spondyloarthritis Based on Real-World Data.","authors":"Jamal Al-Saleh, Ahmed Abdelmoniem Negm, Ahlam Almarzooqi, Nasir Elamin Elhag Elsidig, Noura Zamani","doi":"10.2147/CEOR.S556012","DOIUrl":"10.2147/CEOR.S556012","url":null,"abstract":"<p><strong>Purpose: </strong>To assess the cost-effectiveness of biologics in patients with axial spondyloarthritis (axSpA) in a real-world setting.</p><p><strong>Patients and methods: </strong>This is a non-interventional, registry-based, prospective cohort study included 203 consecutive patients with axSpA who attended the Rheumatology Department of a public hospital in the Emirate of Dubai between July 2018 and September 2020. Demographic and clinical data were collected and disease activity and treatment response were assessed. Patients were grouped according to the treatment received, distinguishing between biologics and non-biologics, and classified them based on their disease activity at baseline and 52 and 104 weeks. The direct and indirect costs associated with their management were collected. The incremental cost-effectiveness ratio (ICER) was calculated and the impact of patient-related factors on its value across subgroups was examined. A state-transition model was used to simulate disease progression across four health states over a lifetime (62 years), and Monte Carlo simulation was applied to address uncertainty.</p><p><strong>Results: </strong>The total cost of managing the patients was AED 27,532,189, resulting in a gain of 293.7 quality-adjusted life years (QALYs) over a 2-year follow-up period. Biological therapies were associated with higher direct costs, accounting for 83.3% of the total costs of biologics. The overall ICER was AED 253,616 per QALY, influenced by higher medication acquisition costs. Patients with < 5 years of disease duration, female patients, those with radiographic-axSpA, co-existing fibromyalgia, and those receiving a combination of biologic treatments and conventional disease-modifying antirheumatic drugs showed a higher ICER than the median ICER of all biologic therapies.</p><p><strong>Conclusion: </strong>Biologic therapies are cost-effective for patients with axSpA, especially those not achieving clinical targets. Patients' selection, and targeted cost-containment strategies, such as biosimilars and medication price reductions, can enhance clinical benefits and reduce societal costs.</p>","PeriodicalId":47313,"journal":{"name":"ClinicoEconomics and Outcomes Research","volume":"17 ","pages":"915-930"},"PeriodicalIF":2.2,"publicationDate":"2025-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12714858/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145806106","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}