Journal Of Medical Economics[JOURNAL]
Sun
200 papers
The economic and health consequences of shared clinical decision-making for hepatitis B birth dose vaccination.
Yang YT
J Med Econ · 2026 Dec · PMID 42397262 · Publisher ↗
AIMS: In December 2025, the Advisory Committee on Immunization Practices recommended individual-based decision-making-termed shared clinical decision-making (SCDM) on CDC schedules-for hepatitis B (HepB) birth-dose vacci...
AIMS: In December 2025, the Advisory Committee on Immunization Practices recommended individual-based decision-making-termed shared clinical decision-making (SCDM) on CDC schedules-for hepatitis B (HepB) birth-dose vaccination in infants of mothers documented as HBsAg-negative at delivery. We evaluated projected economic, health, and distributional consequences for the 2026 US birth cohort. MATERIALS AND METHODS: A hybrid decision tree-Markov cohort model took the societal perspective over a lifetime horizon for 3.6 million 2026 US births, under three coverage-decline scenarios (10-, 20-, and 30-percentage-point). Costs (2026 US dollars) and outcomes were discounted 3% annually. Outcomes included costs, infections, deaths, quality-adjusted life years (QALYs), and equity impacts by insurance, race/ethnicity, hospital type, and geography. Probabilistic sensitivity analysis used 10,000 iterations; reporting followed CHEERS 2022. RESULTS: Under the base-case 20-percentage-point decline, SCDM was projected to produce approximately 44 additional acute infections, 8 additional chronic HBV cases, 2 additional HBV-related deaths (incomplete-linkage scenario), and 56 discounted QALYs lost per cohort. Assuming incremental counseling time for all policy-sensitive births, SCDM generated approximately $301 million in net societal cost-driven principally by provider counseling opportunity cost, not disease treatment-and was dominated by universal vaccination. With counseling time assigned zero cost, SCDM remained less effective but less costly, implying approximately $0.7 million per QALY to retain universal vaccination. Modeled burdens concentrated among Medicaid/CHIP and safety-net populations. LIMITATIONS: This early assessment used scenario-based coverage-decline, counseling-time, mortality, and completion assumptions as post-policy data were unavailable; results are projections, not observations. CONCLUSIONS: Moving from universal birth-dose vaccination to SCDM was projected to reduce timely vaccination and increase preventable infections across all scenarios. The societal-cost conclusion hinged on whether SCDM imposed counseling-time burden at scale, whereas the unfavorable health-effect direction was robust. Postimplementation evidence on coverage, counseling, completion, and linkage to care is needed before treating SCDM as low-cost or low-risk.
TIME-trend and forecasting of the economic impact of inflammatory bowel disease in Catalonia: a population-based analysis.
Garcia-Sagué B, Brunet-Mas E, Vela E … +12 more , Coward S, Calvet X, Pontes C, Melcarne L, Frisancho LE, Llovet LP, Pedregal-Pascual P, Puy A, Ramirez-Lazaro MJ, Villoria A, Lario S, Kaplan GG
J Med Econ · 2026 Dec · PMID 42394418 · Publisher ↗
INTRODUCTION: Few studies have assessed the distribution and temporal trends of healthcare expenditures related to inflammatory bowel disease (IBD). The aims of the study were to analyze the expenditure trends for IBD pa...
INTRODUCTION: Few studies have assessed the distribution and temporal trends of healthcare expenditures related to inflammatory bowel disease (IBD). The aims of the study were to analyze the expenditure trends for IBD patients in Catalonia from 2011 to 2024, to identify key cost drivers, and to forecast future costs through 2036. METHODS: All patients with a diagnosis of IBD included in the Catalan Health Surveillance System (CHSS) were eligible. CHSS compiles prospective data from public healthcare coverage of 8 million people in 2024. Healthcare utilization was analyzed, and expenditures were calculated using standard costs defined by the Catalan Department of Health. All expenditures were adjusted to 2024 euros using the Consumer Price Index to enable comparisons across years. Costs were estimated for IBD overall, Crohn's disease (CD) and ulcerative colitis (UC). An autoregressive integrated moving average model was used to forecast total costs up to 2036. RESULTS: IBD-related healthcare expenditure tripled from €67.4 M in 2011 to €201.6 M in 2024, while per-patient costs rose from €3,981 to €4,753. Biologic therapies were the main cost driver, especially in CD. Mean per-patient biologic costs decreased by 16.6% in CD but increased by 44.5% in UC. CD patients consistently incurred higher per capita costs. Forecasts indicate continued growth in total expenditure, reaching €319.0 M by 2036. CONCLUSION: Overall, IBD-related healthcare expenditures in Catalonia markedly increased from 2011 to 2024, driven mainly by the increase in IBD prevalence. Per-patient cost moderately increased. Per-patient cost containment was observed in pharmaceutical costs, probably due to a strict policy favoring the use of biosimilars. These findings may be of help for designing future healthcare policies.
The financial impact of surgical interventions for benign prostatic hyperplasia within a single NHS trust.
Sarna S, Ranasinghe S, Boaz RJ … +1 more , Rochester M
J Med Econ · 2026 Dec · PMID 42387670 · Publisher ↗
AIMS: To evaluate the financial impact of surgical modalities for benign prostatic hyperplasia (BPH) from the perspective of an NHS Trust. METHODS: A retrospective analysis of patients undergoing Aquablation, Holmium Las...
AIMS: To evaluate the financial impact of surgical modalities for benign prostatic hyperplasia (BPH) from the perspective of an NHS Trust. METHODS: A retrospective analysis of patients undergoing Aquablation, Holmium Laser Enucleation of the Prostate (HoLEP) and Transurethral Resection of the Prostate (TURP) was conducted at a single NHS teaching hospital between January 2023 and June 2025. Financial data was extracted from the patient-level information and costing system, including clinical income based on Healthcare Resource Group tariffs, direct and indirect costs, and overheads. Primary outcomes were median episode clinical income, total costs, and net surplus/deficit. RESULTS: 436 procedures were analysed (140 Aquablation, 160 HoLEP, 136 TURP). Mean operative time was shortest for Aquablation (61.8 ± 27.0 min), while HoLEP had the shortest length of stay (0.34 ± 1.18 days). Aquablation was the only procedure to yield a median net surplus (+£1,859, IQR +785 to +2,736), whereas both HoLEP (-£157, IQR -679 to +189) and TURP (-£852, IQR -1,716 to +84) resulted in median deficits (). Aquablation's financial advantage was driven by its robotic-assisted classification, attracting a higher median clinical income (£7,659) than HoLEP (£3,038) and TURP (£2,997) (). Surplus-generating episodes were more frequent for Aquablation (83.6%) than HoLEP (28.1%) or TURP (22.1%) (). Major individual losses were driven by complications and prolonged admissions across all cohorts. LIMITATIONS: This single-centre retrospective study focused on short-term hospital-level costs. It did not account for initial capital expenditure, long-term clinical outcomes, or Quality-Adjusted Life-Years. CONCLUSIONS: Under the current NHS Payment Scheme, Aquablation is the only financially sustainable surgical modality for the provider among those analysed. Its higher robotic-assisted tariff compensates for greater consumable costs. National tariff reviews may be warranted to ensure reimbursement reflects the true resource intensity of all surgical interventions. Establishing broader NHS cost-effectiveness will require long-term evaluation of clinical outcomes and retreatment rates.
Clinical events and healthcare costs of dapagliflozin in adults with cardiovascular-kidney-metabolic syndrome in Spain: a cost-offset analysis.
Alcázar Arroyo R, Comín-Colet J, Espigares MJ … +9 more , Gutiérrez R, Navarro González JF, Vallès Callol JA, Capel Sanchez M, Rubio Ponce de León S, Castro Gasch M, Pomares E, Gómez G, Caudron C
J Med Econ · 2026 Dec · PMID 42343656 · Publisher ↗
AIMS: Adults with cardiovascular-kidney-metabolic (CKM) syndrome, characterized by interactions among metabolic, chronic kidney disease (CKD) and cardiovascular disease, face a high symptomatic burden, mortality risk and...
AIMS: Adults with cardiovascular-kidney-metabolic (CKM) syndrome, characterized by interactions among metabolic, chronic kidney disease (CKD) and cardiovascular disease, face a high symptomatic burden, mortality risk and costs. Dapagliflozin has demonstrated significant benefits in adults with type 2 diabetes (T2D), heart failure (HF) and CKD. This modelling study aimed to estimate the clinical events and associated healthcare costs of dapagliflozin in adults with CKM syndrome from the Spanish National Health System (NHS) perspective over 3 years. METHODS AND RESULTS: A cost-offset model compared clinical events and their associated costs of 100,000 adults with CKM syndrome treated with dapagliflozin added to standard of care (SoC) and SoC alone. Clinical event rates were derived from dapagliflozin trials (DECLARE-TIMI 58, DAPA-HF, DAPA-CKD and DELIVER), and costs from national databases and literature. Robustness was assessed through scenario and subgroup analyses. The carbon dioxide (CO) footprint impact associated with hospitalization for heart failure (HF), urgent HF visits and end-stage renal disease was assessed in an exploratory analysis. Over 3 years, dapagliflozin added to SoC prevented 10,151 clinical events (2,822 cardiovascular; 5,519 renal; 1,810 deaths) and saved €159.11 million per 100,000 adults compared to SoC, mainly by preventing end-stage renal disease (69%; -€110.32 million). Considering drug acquisition cost, the net cost saving was €52.47 million per 100,000 adults, representing a €1.49 return for every euro invested in dapagliflozin. Scenario analyses were consistent with the base case. Subgroup analyses showed greater benefits in adults with multiple comorbidities, especially those with all three conditions. The CO footprint reduction was 2,176 tons of CO per 100,000 adults. CONCLUSION: Dapagliflozin in adults with CKM syndrome reduced the incidence of clinical events, generating cost savings and improving the sustainability of the Spanish NHS.
Cost-effectiveness of multitarget stool DNA testing for colorectal cancer screening in Chinese primary healthcare settings: a modelling study.
Su H, Li J, Zhang X … +12 more , Xiu Z, Ge T, Tang N, Du X, Jiang R, Li S, Zhang J, Wei T, Xu L, Zhu S, Si L, Tian M
J Med Econ · 2026 Dec · PMID 42339709 · Publisher ↗
AIM: To evaluate the cost-effectiveness of a one-time mt-sDNA-based colorectal cancer screening program compared with no screening, using real-world data from a government-led population-based screening program in Harbin...
AIM: To evaluate the cost-effectiveness of a one-time mt-sDNA-based colorectal cancer screening program compared with no screening, using real-world data from a government-led population-based screening program in Harbin Municipality of China. METHODS: This modelling study was based on a real-world mt-sDNA CRC screening program in Chinese primary healthcare settings. The program targeted residents aged 45-64 years without a prior history of CRC. A decision analytic model was developed from the healthcare system perspective to compare mt-sDNA screening with a hypothetical no-screening scenario. Costs included mt-sDNA testing, diagnostic colonoscopy after positive results, adenoma resection, and colorectal cancer treatment by disease stage. Effectiveness was measured in quality-adjusted life-years (QALYs). The primary outcome was the incremental cost-effectiveness ratio (ICER), assessed against a willingness-to-pay threshold of CNY 166,744 per QALY gained. Costs and QALYs were discounted at 5% annually over a lifetime horizon. One-way deterministic sensitivity analysis and probabilistic sensitivity analysis with 10,000 simulations were conducted. RESULTS: Among 749,517 participants, 79,169 had positive mt-sDNA results and 17,364 completed diagnostic colonoscopy, corresponding to a colonoscopy adherence rate of 21.93%. In the base-case analysis, mt-sDNA screening incurred CNY 264 higher lifetime costs and yielded an additional 0.006 QALYs per person versus no screening. The mt-sDNA screening was cost-effective with an ICER of CNY 43,627 per QALY gained. The cost-effectiveness results were particularly sensitive to colonoscopy adherence (ranging from CNY 94,907 to 10,037 per QALY as adherence varied from 10% to 100%) and to the unit cost of the mt-sDNA test (ranging from CNY 22,113 to 65,140 per QALY with ± 50% variation in cost). All ICERs remained below the cost-effectiveness threshold. CONCLUSIONS: The mt-sDNA screening is cost-effective compared to no screening. These findings provide economic evidence to support the large-scale adoption of mt-sDNA-based screening in China.
From options to decisions: an innovative model for treatment sequencing in relapsing-remitting multiple sclerosis.
Oreja-Guevara C, Landete L, Rodriguez Sagrado MÁ … +8 more , Moya I, Alcala Revilla B, DelosSantos Real H, Bernat Pinto ML, Harty G, Fenwick E, Sigworth E, Brieva Ruiz L
J Med Econ · 2026 Dec · PMID 42339705 · Publisher ↗
AIMS: With the expanding range of disease-modifying therapies (DMTs) for relapsing-remitting multiple sclerosis (RRMS), clinicians face increasing complexity in defining optimal treatment sequences and timing of therapy...
AIMS: With the expanding range of disease-modifying therapies (DMTs) for relapsing-remitting multiple sclerosis (RRMS), clinicians face increasing complexity in defining optimal treatment sequences and timing of therapy switches. In this study, we adapted an earlier computer-assisted model to optimize therapeutic decisions and identify preferred treatment pathways, based on expert opinion and observations from clinical practice in Spain. MATERIALS AND METHODS: The original model was updated to integrate magnetic resonance imaging (MRI) activity data and define reaching an Expanded Disability Status Scale (EDSS) score of 3 - indicating moderate disability without ambulation impairment - as a criterion for switching treatment. Matrices were designed to model options when switching DMTs, triggered by either a lack of effectiveness or safety concerns. Change in DMT was based on a set of composite criteria (encompassing contributions from relapses, disability worsening, MRI activity, costs, and quality of life) according to the disease activity level. A maximum of three DMTs could be administered within the 8-year time horizon evaluated. RESULTS: The revised model identified high efficacy treatment, in particular cladribine tablets as the preferred initial DMT for patients with RRMS with mild or moderate disease activity. Of these patients with mild and moderate disease activity, most were switched to ofatumumab (79.4%) and ocrelizumab (75.9%), respectively, when disease progression occurred. Patients with high disease activity mostly received natalizumab if they were John Cunningham virus (JCV)-negative, or ocrelizumab if they were JCV-positive. LIMITATIONS: The model is informed by a Spain-based healthcare expert panel and has been evaluated using a simulated cohort of 10,000 patients with RRMS. CONCLUSION: This computational model helps to inform clinicians towards making optimal treatment decisions for RRMS, and identified high-efficacy DMTs as the preferred model-based option for all levels of disease activity, supporting early and effective control of disease activity in real-world clinical practice.
Budget impact of implementing AI-enabled chest X-ray based incidental pulmonary nodule detection for early lung cancer diagnosis: models from Colombia, Costa Rica, Mexico, Thailand and Vietnam.
Sloof AC, Sheveleva S, Pawar S … +7 more , Reddy B, Dirodi B, Garcia Peña BP, Mata Coto R, Cortés S, Chinnacom D, Pham HTV
J Med Econ · 2026 Dec · PMID 42339680 · Publisher ↗
OBJECTIVE: To assess the budget impact of incidental pulmonary nodule (IPN) detection using an artificial intelligence-software for chest X-ray (CXR) interpretation - qXR - for early lung cancer (LC) detection in Vietnam...
OBJECTIVE: To assess the budget impact of incidental pulmonary nodule (IPN) detection using an artificial intelligence-software for chest X-ray (CXR) interpretation - qXR - for early lung cancer (LC) detection in Vietnam, Colombia, Thailand, Costa Rica, and Mexico. METHODS: Country-specific hybrid decision-tree and budget impact models were developed from the public payer perspective over a 5-year time horizon. Each compared current symptomatic LC detection with a scenario adding AI-enabled Chest X-Ray for IPN detection and referral to low-dose computed tomography. Costs included diagnostics, set-up, treatment, healthcare resource use, and end-of-life care, expressed in 2024 USD. One-way sensitivity and scenario analyses tested parameter and structural uncertainty. RESULTS: Over the 5-year time horizon, the implementation of AI-enabled CXR for IPN detection is estimated to avert 24,763 premature deaths across the five countries, improving survival and lowering long-term expenditures. Following the introduction of qXR, more costs were incurred due to additional patients being diagnosed and treated. This resulted in fewer late-stage patients and cost savings. The breakeven point was reached in year 3 for Vietnam, Thailand, Colombia, and Mexico and in year 4 for Costa Rica. Cost savings were primarily driven by the stage shift towards earlier detection, where treatment costs are lower and survival outcomes are better. CONCLUSIONS: Integrating AI-enabled CXR for IPN detection into national hospital workflows may enable earlier LC detection and achieve cost neutrality within 5 years in low- and middle-income countries. These findings support the economic feasibility and scalability of AI-assisted imaging as part of national lung cancer control strategies.
Economic evaluation of adjuvant chemotherapy for non-metastatic sarcoma using the real-world data from the French nationwide DEEPSARC study.
Perrier L, Morelle M, Bonvalot S … +27 more , Orbach D, Llacer C, Ducassou A, Drézen E, Chemin-Airiau C, Crochet H, Ducimetiere F, Gouin F, Happe A, Huchet N, Le Loarer F, Lecesne A, Mathoulin-Pelissier S, Méeus P, Oger E, Thévenet V, Toulmonde M, Vaz G, Karanian M, Ngo C, De Pinieux G, Gomez-Mascard A, Bompas E, Macagno N, Firmin N, Penel N, Blay JY
J Med Econ · 2026 Dec · PMID 42334926 · Publisher ↗
OBJECTIVE: The efficacy of adjuvant chemotherapy (AC) in improving survival for patients with sarcoma is debated. We performed a cost-effectiveness analysis (CEA) comparing AC vs. no AC for non-metastatic sarcoma based o...
OBJECTIVE: The efficacy of adjuvant chemotherapy (AC) in improving survival for patients with sarcoma is debated. We performed a cost-effectiveness analysis (CEA) comparing AC vs. no AC for non-metastatic sarcoma based on the nationwide retrospective study DEEPSARC. METHODS: The CEA was carried out over a 1-, 3-, and five-year horizon, using data from the French NETSARC+ database linked to the French national health data system (SNDS).There was no age limit and no specific histological type selection in the reference case analysis. Costs (expressed in 2021 EUR) were provided by the SNDS from the French national health insurance perspective. Incremental cost effectiveness ratios (ICER) were expressed in cost per life-year gained (LYG). Propensity score analysis with 1:1 matching was undertaken. Sensitivity and subgroup analyses were performed. RESULTS: Of the 33,548 patients from the French NETSARC+ database, 24,539 were linked to the SNDS. A total of 14,808 patients diagnosed between 2012 and 2017 were included in the reference case analysis with 2,784 patients after propensity score matching. Mean costs (SD) [95% CI] differences per patient between AC and no AC were €12,826 (36,758) [10,883-14,719], €15,706 (49,849) [13,330-18,383], and €16,841 (56,060) [13,590-19,657] at 1, 3, and 5 years, respectively. Mean overall survival differences per patient (in years) were 0.0066 (0.1685) [-0.0021 to 0.0157], -0.0728 (0.9336) [-0.1237 to -0.0229], and -0.1204 (1.3595) [-0.1926 to -0.0475] at 1, 3, and 5 years, respectively. ICER was €1,934,511 [-11,767,351-15,829,959] per LYG at 1 year. AC lagged behind at 3 and 5 years. CONCLUSIONS: In the reference case analysis, AC outperformed its use in terms of outcomes at 1 year, but a high level of willingness to pay would be required for AC to be cost-effective. At 3 and 5 years, AC was deemed to be not cost-effective for patients with non-metastatic sarcoma.
Natalizumab is associated with improved relapse outcomes and lower healthcare utilization versus ocrelizumab in treatment-naïve people with multiple sclerosis.
Kaplan J, Beenstock D, Belkin M … +7 more , Crayton H, Eckstein C, Fredericks R, Bian B, Belviso N, Ni D, Rutledge D
J Med Econ · 2026 Dec · PMID 42323726 · Publisher ↗
OBJECTIVES: Natalizumab (NTZ) and ocrelizumab (OCR) are well-established therapies and commonly considered first-line for newly diagnosed people with multiple sclerosis (pwMS) in the United States. However, there is a pa...
OBJECTIVES: Natalizumab (NTZ) and ocrelizumab (OCR) are well-established therapies and commonly considered first-line for newly diagnosed people with multiple sclerosis (pwMS) in the United States. However, there is a paucity of data comparing healthcare resource utilization (HCRU) and relapse outcomes between NTZ and OCR in this population. METHODS: This retrospective longitudinal analysis using the Komodo Health Claims Database included newly diagnosed treatment-naïve pwMS aged 18-64 years with ≥1 prescription claims for NTZ or OCR between 1 January 2017 and 31 March 2022. Propensity score matching (1:2) was used to balance baseline characteristics. Outcomes included HCRU and costs, as well as relapses. Relapse events were identified using a claims‑based algorithm. RESULTS: The analysis included 1,261 NTZ- and 2,522 OCR-treated pwMS. First-line NTZ treatment was associated with significantly lower annualized rates of all-cause inpatient admissions (rate ratio = 0.61, 95% CI = 0.44-0.85; = 0.003) and costs as well as emergency department visits (rate ratio = 0.80, 95% CI = 0.68-0.96; = 0.015) than first-line OCR treatment. Annualized relapse rates (ARR) were significantly lower with first-line NTZ versus OCR treatment (0.20 vs. 0.28; rate ratio = 0.70, 95% CI = 0.59-0.82; < 0.001). There was also a significantly higher probability of pwMS being relapse-free at 3 years with first-line NTZ versus OCR (69.9% vs. 63.9%; = 0.002). LIMITATIONS: This study has limitations inherent to claims data, including limited clinical detail and reliance on a claims-based relapse algorithm. CONCLUSIONS: This real-world, claims-based analysis demonstrates that first-line NTZ treatment was associated with significantly lower inpatient and emergency department HCRU, inpatient healthcare costs, and ARR than first-line OCR treatment. Given the paucity of comparative data among these therapies in newly diagnosed MS patients, these results provide valuable real-world evidence to help guide treatment decisions in this group.
Evaluation of the budget impact of single-inhaler triple therapy (fluticasone furoate/umeclidinium/vilanterol) in patients with moderate to severe asthma in the Saudi healthcare system.
Alabdulkarim HA, Abushal M, Alasiri A … +5 more , Ahmed Alenzi K, Alabdulaali S, Tahoon H, Soliman H, Hassan MEK
J Med Econ · 2026 Dec · PMID 42318691 · Publisher ↗
AIM: This study investigated the financial impact of introducing the once-daily single-inhaler triple therapy (SITT) fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) in Saudi Arabia for adult patients with uncont...
AIM: This study investigated the financial impact of introducing the once-daily single-inhaler triple therapy (SITT) fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) in Saudi Arabia for adult patients with uncontrolled asthma. METHODS: The expected 5-year budget impact of introducing FF/UMEC/VI for asthma was modeled using an epidemiology-based approach that included both acquisition and efficacy-related costs. Year-on-year costs were estimated for 2026 through 2030. Changes in healthcare resource utilization after FF/UMEC/VI introduction were estimated from the expected change in asthma control reported in the Phase IIIa CAPTAIN trial. The perspective of the model was the Saudi national healthcare payer; only direct medical costs were included. The robustness of the model was tested using sensitivity and scenario analyses. RESULTS: It was estimated that in 2026, 12,108 patients in Saudi Arabia would receive FF/UMEC/VI, which will likely increase over subsequent years (2027: 29,615 patients; 2028: 31,191 patients; 2029: 32,938 patients; 2030: 34,727 patients), as validated by local expert opinion. The introduction of FF/UMEC/VI was estimated to save a total of United States Dollars (USD) 10,654,389 over 5 years, or USD 16.32 per patient, with savings increasing each year after introduction. Drug acquisition costs contributed to the greatest impact on the budget, and the model was most sensitive to changes in the market uptake of FF/UMEC/VI. Across all sensitivity and scenario analyses, FF/UMEC/VI was consistently cost saving. LIMITATIONS: Various model inputs could not be derived from published sources, so multiple assumptions were used. Future market share was estimated from current competitor market share data; as FF/UMEC/VI is the first SITT for asthma to be introduced into Saudi Arabia, there is no historical data on which to base these estimates. CONCLUSION: Introducing FF/UMEC/VI in Saudi Arabia has the potential to deliver meaningful clinical and economic benefits at both the individual patient and healthcare system level.
Real-world economic comparison of preemptive kidney transplant vs maintenance dialysis in patients with end-stage kidney disease.
Schold JD, Potukuchi P, Voss J … +6 more , Preblick R, Sienko D, Yu T, Noshad S, Boame N, Devlin A
J Med Econ · 2026 Dec · PMID 42318627 · Publisher ↗
BACKGROUND: A direct comparison of healthcare resource utilization (HCRU) and associated costs in patients with end-stage kidney disease (ESKD) who received preemptive kidney transplants (PKT; defined as the receipt of a...
BACKGROUND: A direct comparison of healthcare resource utilization (HCRU) and associated costs in patients with end-stage kidney disease (ESKD) who received preemptive kidney transplants (PKT; defined as the receipt of a kidney transplant in a patient without a prior history of dialysis) vs maintenance dialysis (MD) in US clinical practice is lacking. METHODS: This retrospective, observational study evaluated data from Optum's deidentified Clinformatics Data Mart Database (January 2012-July 2024) on patients with ESKD who received PKT or MD. HCRU and costs (standardized to 2024 US dollars) at 24-month follow-up were compared by weighted generalized linear modeling. RESULTS: In total, 14,135 ESKD patients (1153 who received PKT and 12,982 undergoing MD) were analyzed. Patients in the PKT cohort were more likely to be hospitalized vs the MD cohort (odds ratio [OR] = 10.68; 95% CI = 6.00-19.02), with more hospitalizations per person (event rate ratio [ERR] = 1.21; 95% CI = 1.05-1.39). Patients receiving PKT were less likely to visit the emergency department vs those undergoing MD (OR = 0.49; 95% CI = 0.33-0.72), with fewer emergency department visits per person (ERR = 0.31; 95% CI = 0.24-0.42). Almost all patients had ≥1 outpatient visit; however, the number of outpatient visits per person was lower in the PKT cohort vs the MD cohort (ERR= 0.30, 95% Cl = 0.27-0.33). PKT costs initially exceeded MD, but MD costs surpassed PKT from approximately month 5 onward. Estimated total all-cause per person costs were $303,725 (for PKT) and $637,134 (for MD), representing a saving of $333,409 (95% CI = $263,461-$403,356) at 24 months for PKT. CONCLUSIONS: Despite substantially higher initial costs, patients receiving PKT had lower HCRU and costs at 24 months, demonstrating that PKT was less costly compared with MD during the study follow-up.
Behind the counter: a time-driven activity-based costing analysis of community pharmacy dispensing services in Taiwan.
Chen PA, Huang YM, Wang YH … +2 more , Chan HY, Ho YF
J Med Econ · 2026 Dec · PMID 42312482 · Publisher ↗
BACKGROUND: Sustainable financing of community pharmacy services requires reimbursement models that accurately reflect resource use. However, empirical cost data for dispensing services remain limited in many health syst...
BACKGROUND: Sustainable financing of community pharmacy services requires reimbursement models that accurately reflect resource use. However, empirical cost data for dispensing services remain limited in many health systems, including Taiwan's single-payer National Health Insurance (NHI) program. This study estimated the cost of dispensing services in community pharmacies and assessed the extent to which current reimbursement reflects underlying resource utilization. METHODS: A cost analysis was conducted from the healthcare provider perspective using a time-driven activity-based costing approach. Eight community pharmacies in Taiwan were included. Dispensing workflows were systematically mapped into six sequential steps, and time-and-motion data were collected across six simulated standardized dispensing scenarios designed to capture variation in medication number and type. Resource use included direct, indirect, and personnel-related cost components. Indirect costs were allocated using both space-based and revenue-based approaches. Personnel costs were calculated using capacity cost rates based on pharmacist salaries and practical working capacity. All costs were expressed in New Taiwan Dollars (NT$), and sensitivity analyses assessed robustness under alternative allocation assumptions. RESULTS: Mean dispensing time ranged from 195 to 303 s per prescription and increased with prescription complexity. Total dispensing costs ranged from NT$30.82 to NT$61.21 per prescription. Each additional medication increased costs by NT$13-17, while controlled substances added NT$18-23 due to additional regulatory procedures. Personnel-related costs were the primary cost driver, and cognitive verification activities accounted for the largest share of workload. Compared with current NHI dispensing reimbursement of approximately NT$49-68 per prescription, reimbursement may inadequately reflect the resources required for more complex prescriptions. Cost patterns were consistent across allocation methods. CONCLUSIONS: Dispensing services are resource-intensive and vary with prescription complexity, yet current flat-fee reimbursement under Taiwan's NHI does not adequately reflect this variation. Complexity-adjusted reimbursement mechanisms, such as per-medication or controlled-substance modifiers, may better align payment with actual resource utilization and support sustainable community pharmacy services.
Rett syndrome and real-world treatment patterns of trofinetide in the United States.
Rashid N, Yakkala VK, Syed SS … +1 more , Rajagopalan K
J Med Econ · 2026 Dec · PMID 42287113 · Publisher ↗
BACKGROUND: Trofinetide (TROF) remains the first and only FDA-approved pharmacologic treatment for Rett syndrome (RTT). There is limited real-world evidence on TROF use, restarts, dosing patterns and predictors of non-pe...
BACKGROUND: Trofinetide (TROF) remains the first and only FDA-approved pharmacologic treatment for Rett syndrome (RTT). There is limited real-world evidence on TROF use, restarts, dosing patterns and predictors of non-persistence. This study evaluated long-term treatment patterns and baseline predictors of non-persistence among RTT individuals initiating TROF. METHODS: A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed. RTT individuals initiating 1 TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent. Median (IQR) time on treatment was analyzed, and Kaplan-Meier assessed time to non-persistence. Restarts among non-persistent were examined. Dosing patterns such as mean dose in milligrams (mg) and percentage target daily dose (%TDD) at each RX were evaluated. Predictors of non-persistence were assessed using logistic regression. RESULTS: Among 1,175 TROF initiators, 54.9% were persistent and 45.1% were non-persistent; 7.6% of non-persistent individuals restarted. Mean ± SD age was 14.7 ± 10.8 vs. 16.5 ± 11.6 years and median ± IQR time on TROF was 14.3 ± 4.6 vs. 3.0 ± 3.8 months, respectively. Over 75% remained on treatment beyond 3 months, and more than half continued through study follow-up. Mean twice daily (BID) TROF dose was lower among persistent (7422.0 mg) vs. non-persistent (7850.8 mg) at RX1 and increased to 7953.7 mg vs 8423.4 mg, respectively at RX2. Thereafter, the persistent individuals maintained stable dosing, while the non-persistent showed greater fluctuation over time. Similar patterns were observed for %TDD. Older age and history of infectious disorders were significantly associated with non-persistence. CONCLUSIONS: In this real-world analysis, more than half of RTT individuals remained persistent on TROF for ≥14 months. Stable dosing patterns were observed among persistent individuals, while greater dose variability was observed among non-persistent individuals. Thus, these findings may provide guidance to clinicians about long-term TROF use in real-world practice.
Potential implications of increased utilization of oral nirmatrelvir/ritonavir in outpatient care for COVID-19: modeled findings from the Spanish National Health System perspective.
Benner J, Lee J, Cianciolo L … +4 more , Mugwagwa T, de Lossada A, Peral C, Sussman M
J Med Econ · 2026 Dec · PMID 42287108 · Publisher ↗
INTRODUCTION: This analysis evaluates the clinical and societal impact of increasing utilization of oral nirmatrelvir/ritonavir (NMV/r) for outpatient treatment of mild-to-moderate COVID-19 among high-risk adults in Spai...
INTRODUCTION: This analysis evaluates the clinical and societal impact of increasing utilization of oral nirmatrelvir/ritonavir (NMV/r) for outpatient treatment of mild-to-moderate COVID-19 among high-risk adults in Spain. Despite strong evidence from clinical trials and real-world studies demonstrating NMV/r's effectiveness in reducing hospitalization and death, uptake in Spain has remained suboptimal. METHODS: Using a decision tree adapted from a previously published model, we estimated outcomes from the perspective of the Spanish National Health System over a 30-day time horizon. Model inputs included Spanish epidemiological data, healthcare resource utilization rates, and productivity loss parameters. Five NMV/r uptake scenarios were compared: current utilization (30.9%), three hypothetical increased utilization rates (45%, 60%, 75%), and a 0% utilization scenario. RESULTS: Results indicate that increasing NMV/r uptake substantially reduces hospitalizations, ICU admissions, and mortality, while improving productivity outcomes. Compared with the base case, hospitalizations and deaths declined by 15-47% and productivity loss hours by 15-45% across higher utilization scenarios; conversely, the 0% scenario resulted in a 33% increase in hospitalizations and a 32% rise in productivity loss. Sensitivity analyses confirmed the robustness of findings across key parameters. These results underscore the clinical and societal benefits of broader NMV/r adoption and provide timely, policy-relevant evidence to inform strategies that enhance treatment uptake. CONCLUSIONS: Greater utilization of NMV/r could alleviate strain on the Spanish health system and prevent avoidable hospitalizations and deaths, reinforcing the importance of interventions that promote timely access for high-risk patients.
Cost-consequence analysis of diagnostic strategies for suspected community-acquired pneumonia in France and Spain.
Borisenko O, Stas JI, Maslov A … +2 more , Moola S, Das L
J Med Econ · 2026 Dec · PMID 42281418 · Publisher ↗
AIMS: The study evaluated clinical and health economic consequences of two diagnostic strategies, standard of care (SOC) and SOC plus MeMed BV (MMBV; a host-response test designed to discriminate between bacterial and vi...
AIMS: The study evaluated clinical and health economic consequences of two diagnostic strategies, standard of care (SOC) and SOC plus MeMed BV (MMBV; a host-response test designed to discriminate between bacterial and viral infections), for suspected community-acquired pneumonia (CAP) in adults from a French and Spanish healthcare perspective. METHODS: A cost-consequence analysis using a decision tree for adults presenting to the emergency department with suspected CAP. The time horizon was less than one year, modeling a hypothetical cohort of 1,000 patients with low-to-moderate disease severity. Clinical outputs were derived from published studies; unit costs were sourced from national or regional tariffs and cost studies. Deterministic one-way sensitivity analyses accounted for uncertainty in model parameters. RESULTS: In the base case analysis, SOC + MMBV was cost-saving versus SOC alone in both countries. The total cost per 1,000 patients decreased from €523,557 to €304,064 in France and from €467,356 to €267,786 in Spain, resulting in savings of approximately €220 and €200 per patient, respectively. The cost of MMBV was offset by reductions in other diagnostic tests and hospital admissions. Per 1,000 patients, SOC + MMBV correctly identified 181 more viral infections than SOC alone, resulting in 181 fewer unnecessary antibiotic prescriptions and almost 600 fewer additional diagnostic tests performed. LIMITATIONS: Key limitations include use of a simplified patient pathway, assuming correctly diagnosed patients are not admitted due to clinical deterioration, and a short-term time horizon focused on immediate clinical and economic outcomes. Conclusion Adding MMBV to SOC for adults with suspected CAP in EDs may be cost-saving for public payers in France and Spain. CONCLUSION: Adding MMBV to SOC for adults with suspected CAP in EDs can be cost-saving for public payers in France and Spain.
Impact of high body mass index on health state utilities: a time trade-off study in the general population of Japan.
Tanaka T, Kagitani H, Kojima T … +5 more , Cai Z, Aranishi T, Matsuyama F, Murata T, Yoshinaga Y
J Med Econ · 2026 Dec · PMID 42273840 · Publisher ↗
AIMS: Given the increasing burden of patients with obesity disease, the impact of weight loss on health state utility needs to be reflected in cost-effectiveness analyses of new treatments. Equally important is that, hea...
AIMS: Given the increasing burden of patients with obesity disease, the impact of weight loss on health state utility needs to be reflected in cost-effectiveness analyses of new treatments. Equally important is that, health state utilities that reflect the preferences of the general population are of value for cost-effectiveness analyses. The aim of this time trade-off (TTO) study in the general population of Japan is to estimate the health state utilities of patients with obesity disease. METHODS: Vignettes were generated for health states categorized by BMI (kg/m), as follows: ≥25 to <30, ≥30 to <35, ≥25 to <35, or ≥35. The vignettes were validated by a medical expert and patients with obesity disease. A pilot survey examined the general population's comprehension of the vignettes before the main survey. Face-to-face interviews were conducted with the general population in both the pilot and main surveys by trained interviewers. Respondents were asked to imagine themselves in each vignette. Health state utilities were measured for each vignette using the TTO method. RESULTS: The main survey and pilot survey included 240 adult respondents from the general population of Japan in total (female 50%; mean age, 44.5 years; and mean BMI, 22.4 kg/m). The mean (95% confidence interval) health state utility tended to decrease as BMI category increased, as follows: 0.827 (0.804-0.849) for ≥25 to <30 kg/m, 0.604 (0.563-0.645) for ≥30 to <35 kg/m, 0.771 (0.741-0.800) for ≥25 to <35 kg/m, and 0.317 (0.255-0.380) for ≥ 35 kg/m. CONCLUSIONS: The results of this TTO study in the general population of Japan demonstrate that an increase in BMI from 25 kg/m upward is associated with a decrease of health state utilities. These findings can be utilized to perform cost-effectiveness analyses of anti-obesity medications in Japan.
Impact of Crohn's perianal fistulas on healthcare resource utilization: a US chart review study.
Deepak P, Wong SY, Parian AM … +6 more , Sun M, Nazarey P, Cazzetta S, Terreri B, Turner B, Goodwin B
J Med Econ · 2026 Dec · PMID 42267525 · Publisher ↗
AIM: Perianal fistulas are a common complication of Crohn's disease. Despite the current treatment options, fistula recurrence/treatment failure rates are high. The aim of this study was to assess healthcare resource uti...
AIM: Perianal fistulas are a common complication of Crohn's disease. Despite the current treatment options, fistula recurrence/treatment failure rates are high. The aim of this study was to assess healthcare resource utilization (HCRU) among patients with new, persistent, and recurrent Crohn's perianal fistulas (CPF) in the USA. METHOD: A retrospective, multi-site medical chart review was conducted in patients with CPF. Data on HCRU were collected for ≥2 years after the index date (date of most-recent episode of CPF symptoms between January 2016 and December 2018). RESULTS: In total, 227 patients were included among whom there were 236 fistulas: new ( 94, 39.8%), persistent ( 108, 45.8%), and recurrent ( = 34, 14.4%). Mean (standard deviation [SD]) age was 47.7 (13.7) years, 81.5% were White, 50.7% were male. After index (mean follow-up 33.8 months), 67.0% of patients received medical treatments, 59.9% had surgical interventions, and 42.7% received both. The mean (SD) number of CPF-related and all-cause outpatient visits was 8.75 (8.20) and 18.27 (16.96), the mean (SD) number of CPF-related and all-cause emergency room visits was 0.06 (0.27) and 0.65 (1.55), and the mean (SD) number of CPF-related and all-cause inpatient visits was 0.30 (0.79) and 0.83 (1.81), respectively. Patients with new fistulas had more CPF-related and all-cause outpatient visits and fewer all-cause inpatient visits than patients with persistent/recurrent fistulas ( < 0.05). LIMITATIONS: The use of biologics for underlying Crohn's disease before index was not captured. Furthermore, this study was subject to missing data and limited generalizability (most sites located in Midwest USA). CONCLUSION: There is a substantial HCRU burden among patients with CPF, likely owing to the requirement for multiple and repeated interventions, indicating an unmet need for effective management strategies that promote long-term fistula healing to ultimately support reduced patient burden and HCRU.
Cost-effectiveness and budget impact of ezetimibe-atorvastatin single-pill combination versus free combination in Chinese adults with hypercholesterolemia.
Xing Q, Cheng W, Wang W … +3 more , Ren W, Wang Y, Wang H
J Med Econ · 2026 Dec · PMID 42262268 · Publisher ↗
BACKGROUND: Hypercholesterolemia imposes a growing cardiovascular burden in China, yet low-density lipoprotein cholesterol (LDL-C) target attainment remains low. The single-pill combination (SPC) of ezetimibe and atorvas...
BACKGROUND: Hypercholesterolemia imposes a growing cardiovascular burden in China, yet low-density lipoprotein cholesterol (LDL-C) target attainment remains low. The single-pill combination (SPC) of ezetimibe and atorvastatin (E/A SPC) was approved in China in 2023 and added to the National Reimbursement Drug List (NRDL) in 2024. Compared with free combination therapy (E/A FCT), E/A SPC may improve adherence and LDL-C reduction, but its economic value has not been assessed in the Chinese context. OBJECTIVE: To evaluate the cost-effectiveness of E/A SPC versus E/A FCT in Chinese adults with hypercholesterolemia inadequately controlled on statin monotherapy, and to estimate the 5-year budget impact of E/A SPC adoption. METHODS: An 11-state Markov model was developed from the Chinese healthcare system perspective, projecting 20-year costs and health outcomes in patients aged 69 years with uncontrolled LDL-C (>70 mg/dL) after statin therapy. Clinical efficacy inputs were derived from a large real-world German head-to-head study ( = 311,242). Subgroup analyses were conducted by age, sex, and cardiovascular risk. A 5-year budget impact analysis estimated the financial consequences of increasing E/A SPC uptake. Costs and outcomes were discounted at 5% annually. RESULTS: E/A SPC generated 0.06 additional quality-adjusted life years (QALYs) at an incremental cost of USD 250.12, yielding an incremental cost effectiveness ratio (ICER) of USD 4,070/QALY, well below the willingness-to-pay threshold of USD 14,423. The probability of cost-effectiveness was 91%. Subgroup analyses consistently favored E/A SPC, with the most favorable ICERs observed in very high-risk patients and middle-aged patients. Over 5 years, E/A SPC was associated with incremental drug spending of USD 372.5 million, partly offset by USD 292.0 million in avoided cardiovascular event costs. CONCLUSION: E/A SPC is a cost-effective strategy for Chinese adults with hypercholesterolemia inadequately controlled on statin monotherapy, supporting its NRDL inclusion. The study findings should be further validated using larger-scale real-world data from China.
Modeling the cost-effectiveness of the COVID-19 mRNA-1273 vaccine in the United States.
Fust K, Beck E, Kohli M … +4 more , Cartier S, Van de Velde N, Weinstein M, Joshi K
J Med Econ · 2026 Dec · PMID 42258466 · Publisher ↗
OBJECTIVE: The main objective was to estimate the potential public health impact and cost-effectiveness of an annual dose of mRNA-1273 in the United States (US) for a one-year time horizon compared with no vaccination in...
OBJECTIVE: The main objective was to estimate the potential public health impact and cost-effectiveness of an annual dose of mRNA-1273 in the United States (US) for a one-year time horizon compared with no vaccination in the mRNA-1273 licensed population (6 months-64 years with underlying medical conditions and all ≥65 years). mRNA-1273 was also compared to BNT162b2 in high-risk adults ages 18-64 years and all ≥65 years. METHODS: Analyses were conducted using a previously developed static decision-analytic model (1-year horizon) from the societal cost perspective. Vaccine effectiveness (VE) against infection and hospitalization for mRNA-1273 versus no vaccination was based on a 2024-2025 real world effectiveness study. VE estimates for mRNA-1273 versus BNT162b2 were based on systematic literature reviews and meta-analyses. Cost-effectiveness was assessed in terms of incremental cost per quality-adjusted life-year (QALY) gained and the benefit cost ratio (BCR) in the licensed target population as well as age-specific subgroups. Sensitivity and scenario analyses were performed. RESULTS: Between September and August, a single dose of mRNA-1273 was estimated to yield an incremental cost per QALY gained of $23,265 compared to no vaccine. For every 1 USD of mRNA-1273 vaccine related costs, there is a return of 1.91-7.90 dollars in societal perspective cost savings and monetized health benefit gained. In the subgroup of high-risk individuals 6 months-4 years, mRNA-1273 was associated with lower costs and improved health outcomes, resulting in mRNA-1273 dominating no vaccine. Study results are sensitive to COVID-19 incidence, percentage hospitalized, post-discharge mortality, and VE assumptions. Compared to BNT162b2, given improved clinical outcomes, combined with a lower vaccine unit cost, mRNA-1273 was estimated to dominate BNT162b2. CONCLUSIONS: mRNA-1273, the only licensed vaccine for those <5 years of age at high risk of severe COVID-19 related outcomes, could substantially reduce the clinical and economic burden of COVID-19 among US high-risk populations and older adults. These benefits were observed both in comparison to no vaccination and the BNT162b2 vaccine.
Tracking economic change over a decade: an analysis of Austria's industry-sponsored clinical trial landscape and implications for policy.
Walter E, Eichhober G, Voit M … +15 more , Alfanz A, Atarac-Simic E, Bauer B, Celedin A, Gansch K, Hauer K, Holzhauser C, Lilla C, Mraz B, Janitsch V, Schneider-Schwarz S, Schweighofer D, Weingartmann G, Kaehler S, Baltic D
J Med Econ · 2026 Dec · PMID 42258360 · Publisher ↗
AIMS: Industry-sponsored clinical trials (ISCTs) play a central role in pharmaceutical innovation and economic value creation. Building on a previous assessment, the aim of this study is to update the quantification of t...
AIMS: Industry-sponsored clinical trials (ISCTs) play a central role in pharmaceutical innovation and economic value creation. Building on a previous assessment, the aim of this study is to update the quantification of the industry-funded medical treatments and macroeconomic impact of ISCTs in Austria for 2020-2024 and to compare their development over time with the earlier observation (2012-2017), with particular attention to structural changes associated with the transition to the Clinical Trials Regulation (CTR). MATERIALS AND METHODS: The analysis followed a multi-step approach consistent with the previous assessment. ISCT activity in Austria between 2020 and 2024 was quantified, and characteristics from a representative ISCT dataset used to estimate trial duration, therapeutic distribution, and patient enrolment. The monetary value of protocol-defined medical treatments was derived using national reimbursement tariffs. Macroeconomic effects on domestic value-added and employment were estimated using an extended Leontief input-output model. RESULTS: ISCTs generated an annual medical treatment value of €122.28 million (price level 2025), corresponding to €611.38 million over the five-year period, thereby relieving the Austrian healthcare system by 0.21% of annual current health expenditures. At 2025 price levels, the annual medical treatment value declined from approximately €135 million in the earlier observation period. On average, 2,314 patients were treated each year in 463 ISCTs totaling to 11,570 patients in 782 studies across the entire period (2020-2024). The total annual value-added amounted to €174.05 million, with a value-added multiplier of 1.97. ISCTs secured 2,276 full-time equivalent jobs, corresponding to an employment multiplier of 1.74; both multipliers exceeded those reported in the previous assessment and are higher than those observed in Austria's tourism sector. CONCLUSIONS: ISCTs generate substantial medical and economic value in Austria. Ensuring that Austria remains in the future an attractive location for clinical research also requires sustained investment in research infrastructure and digital systems.
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