Journal Of Medical Economics[JOURNAL]
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200 papers
Cost-effectiveness of vasopressin in the treatment of septic shock: insights from a European societal perspective.
Walter E, Ghinelli F, Goyer I … +2 more , Leone M, Pirracchio R
J Med Econ · 2026 Dec · PMID 41637478 · Publisher ↗
BACKGROUND: Septic shock is a life-threatening condition associated with high morbidity, mortality, and healthcare costs. Vasopressin (VA) is recommended as a second-line vasopressor in septic shock, but its cost-effecti...
BACKGROUND: Septic shock is a life-threatening condition associated with high morbidity, mortality, and healthcare costs. Vasopressin (VA) is recommended as a second-line vasopressor in septic shock, but its cost-effectiveness-especially regarding the timing of administration-remains unclear in European settings. METHODS: A hybrid decision-analytic model combining a short-term decision tree and a long-term Markov model was developed to evaluate the cost-effectiveness of VA in adult patients with septic shock. The analysis was conducted from both a healthcare payer and societal perspective. Clinical efficacy inputs were derived from high-quality meta-analyses and systematic reviews. The model incorporated health-states such as end-stage renal-disease (ESRD) with need for renal replacement therapy (RRT), atrial fibrillation (AF), and mortality over a lifetime horizon. Two comparisons were analyzed: VA versus No VA, and early (within 3-12 h of shock onset) versus late VA administration. Outcomes included incremental cost-effectiveness ratio (ICER), life-years (LYs), quality-adjusted life-years (QALYs), and direct and indirect cost estimates. RESULTS: Adding VA was a dominant strategy, improving clinical outcomes while reducing lifetime costs by 10,570 €per patient and yielding 0.09 additional QALYs. VA therapy reduced RRT dependence by 2.5% and increased AF-free survival by 6.2%. Early VA administration was even more cost-effective, providing 0.55 additional QALYs, 0.77 extra LYs, and 4,746 €in additional savings compared to late administration. CONCLUSION: Second-line VA is a cost-effective intervention for septic shock, notably when initiated early. These findings support guideline recommendations for early vasopressor use and emphasize the clinical and economic value of timely VA therapy.
Economic burden of non-transfusion-dependent thalassemia in the United States.
Langer AL, Rane A, Gilroy KS … +4 more , Zhao J, Lombard L, Lew CR, Sheth S
J Med Econ · 2026 Dec · PMID 41632448 · Publisher ↗
OBJECTIVES: To assess all-cause healthcare resource utilization (HCRU) and costs among patients with α- or β-non-transfusion-dependent thalassemia (NTDT) vs. matched controls in the United States. METHODS: Adults with ≥1...
OBJECTIVES: To assess all-cause healthcare resource utilization (HCRU) and costs among patients with α- or β-non-transfusion-dependent thalassemia (NTDT) vs. matched controls in the United States. METHODS: Adults with ≥1 inpatient setting or ≥2 outpatient settings claims for α- or β‑thalassemia between January 1, 2013 and June 30, 2021 were identified from the Merative MarketScan Commercial/Medicare database. Patients with <8 transfusions or ≥6 weeks between any two adjacent transfusions in a 1-year period post-index date (date of first observed α- or β-thalassemia diagnosis code) were considered to have NTDT. Patients were required to have mean hemoglobin (Hgb) levels <10 g/dL during follow-up as an additional measure to ensure exclusion of patients with thalassemia trait. Each patient was matched with five controls based on age, sex, length of follow-up, availability of lab data, and payer type. All-cause HCRU and costs were assessed over ≥12 months post-index. Data were also analyzed for the non-transfusion-dependent α- and β-thalassemia subgroups. RESULTS: A total of 149 patients with NTDT and Hgb levels <10 g/dL were matched with 745 controls. The mean follow-up period was approximately 3 years. All-cause inpatient admissions (48.3% vs. 16.5%; < 0.001) and emergency room visits (61.1% vs. 39.1%; < 0.001) during follow-up were higher with NTDT vs. controls, and total costs (total medical + outpatient pharmacy) were $29,107 per patient per year (PPPY) in patients with NTDT vs. $9,042 PPPY in controls ( < 0.001). Similar trends were seen in the subgroups of patients with non-transfusion-dependent α- and β-thalassemia vs. matched controls. CONCLUSIONS: Patients with NTDT in the United States, including those with α- and β-thalassemia, have significantly higher all-cause HCRU and costs vs. matched controls. There is a need for effective treatment options to reduce the healthcare burden of NTDT and improve patient outcomes.
Assessing public economic gains from expanding HPV vaccination in Portugal: a governmental perspective analysis.
Kotsopoulos N, Connolly MP, Pavelyev A … +3 more , Rodrigues B, Silva J, Sabale U
J Med Econ · 2026 Dec · PMID 41608993 · Publisher ↗
OBJECTIVE: To assess the public economic impact of expanding human papillomavirus (HPV) vaccination coverage rates in Portugal. METHODS: A cost-benefit analysis from a macroeconomic (societal) and public economic (fiscal...
OBJECTIVE: To assess the public economic impact of expanding human papillomavirus (HPV) vaccination coverage rates in Portugal. METHODS: A cost-benefit analysis from a macroeconomic (societal) and public economic (fiscal) was conducted to compare the current vaccination strategy to no vaccination and to alternative scenarios assuming expansions of HPV vaccination to adult males up to 26 years of age and to unvaccinated women 18-26 years of age. The long-term incidence of HPV-related diseases and attributable mortality, under different scenarios were estimated for 100 years using a dynamic transmission model (DTM). Subsequently, economic gains from reductions in HPV-related morbidity and mortality, including avoided productivity losses and healthcare costs-savings, were estimated and compared to vaccination costs. Costs and benefits were discounted at 4%. RESULTS: The public health benefits of the current HPV vaccination strategy under the National Immunization Plan (NIP) are expected to reduce the cumulative 100-year societal and fiscal burden of disease by 57.8% and 59.9% compared to no vaccine, respectively. Extending HPV vaccination to adult males up to 26 years of age and to unvaccinated women 18-26 years of age is estimated to further reduce societal and fiscal burden by €124 million and €109 million, respectively. Compared to no vaccination, the current NIP results in societal and fiscal benefit-cost ratios of 4.4 and 3.8, respectively. Expanding the current HPV vaccination program would result in benefit cost ratios (BCRs) of 1.9 and 1.7 from the societal and fiscal perspectives, respectively. The BCR remained stable and >1 despite changes in projected healthcare spending over the model duration. CONCLUSIONS: Expanding HPV vaccination to adult populations up to 26 years of age likely offers additional economic gains that exceed expenditures when compared to the current vaccination strategy, translating public health benefits of vaccination strategies into macroeconomic and fiscal outcomes for Portugal.
Cost implications of introducing the BIOFIRE FILMARRAY meningitis/encephalitis panel vs. real-time PCR in adult and pediatric populations in the UK.
Veljanoska E, Szende A, McGill F … +2 more , Gomes S, Malik S
J Med Econ · 2026 Dec · PMID 41608961 · Publisher ↗
AIMS/BACKGROUND: Meningitis is a life-threatening infection of the protective membranes surrounding the brain and spinal cord, requiring early and accurate diagnosis to inform clinical management. Standard diagnostic met...
AIMS/BACKGROUND: Meningitis is a life-threatening infection of the protective membranes surrounding the brain and spinal cord, requiring early and accurate diagnosis to inform clinical management. Standard diagnostic methods, a mix of cerebrospinal fluid (CSF) culture and polymerase chain reaction (PCR) testing, can be constrained by delayed processing or reduced sensitivity following antibiotic administration. The BIOFIRE FILMARRAY Meningitis/Encephalitis (ME) Panel is a rapid multiplex PCR test that detects 14 specific pathogens from a CSF sample in approximately 1 h, thereby avoiding multiple targeted assays, accelerating diagnostic turnaround, and reducing the cumulative diagnostic burden. METHODS: A cost-consequence model was developed from the United Kingdom (UK) National Health Service (NHS) perspective for adults and children with suspected or confirmed meningitis. The model includes costs (diagnostic, treatment, hospital stay) and consequences (time to targeted treatment, antimicrobial use, length of stay) over a per-episode time horizon, from initial presentation through inpatient discharge. RESULTS: Results indicate that the panel reduced total costs compared to real-time PCR by 8.4% (£1,151 per adult) and 15.7% (£1,266 per child) in suspected cases; and by 7.8% (£1,071) and 14.0% (£1,131), respectively, in confirmed cases. CONCLUSIONS: The results indicate that the BIOFIRE ME panel may improve clinical outcomes while reducing NHS resource use, aligning with national goals to optimize antimicrobial stewardship and hospital efficiency.
Estimating the long-term health outcomes of treatment with lecanemab in early Alzheimer's disease: a modelling study.
Burn O, Molloy K, Kanekiyo M … +5 more , Parker C, Rothwell S, Pan JJ, Trueman D, Ritchie C
J Med Econ · 2026 Dec · PMID 41603883 · Publisher ↗
AIMS: To assess the long-term effects of lecanemab plus standard of care (SoC) compared with SoC alone in a cohort of patients with early Alzheimer's disease (AD; mild cognitive impairment [MCI] due to AD, or mild AD dem...
AIMS: To assess the long-term effects of lecanemab plus standard of care (SoC) compared with SoC alone in a cohort of patients with early Alzheimer's disease (AD; mild cognitive impairment [MCI] due to AD, or mild AD dementia) using different modeling approaches and data from Clarity AD (NCT0388745538). METHODS: A Markov model was employed using health states based on disease severity, long-term institutionalization, and death, with disease severity defined using the Clinical Dementia Rating - Sum of Boxes (CDR-SB) classification for MCI due to AD, and Mild, Moderate, and Severe AD. State transitions during the first 18 months of treatment were estimated using either patient count data (Approach 1) or multistate survival analysis (Approach 2). Transition probabilities beyond 18 months for the lifetime of the cohort were informed by longitudinal natural history data for the SoC arm with a hazard ratio for time-to-worsening health state applied to estimate outcomes in the lecanemab arm. RESULTS: Over a lifetime horizon, the model predicted a delayed time to Mild, Moderate, and Severe AD for patients treated with lecanemab compared to SoC by 1.31, 1.85, and 2.04 years, respectively when using Approach 1. Patients treated with lecanemab experienced a survival benefit of 1.36 years, comprised of an additional 1.85 years in early AD and 0.49 years less in moderate and severe AD, compared to patients treated with SoC alone. The model also predicted that compared to SoC, lecanemab increased the time in community care and reduced time spent in institutional care. Results were similar when using Approach 2. LIMITATIONS: Long-term disease progression was informed by constant annual transition probabilities derived from the published literature. CONCLUSIONS: Patients treated with lecanemab experience delayed progression to Moderate and Severe AD, resulting in additional life-years (LYs) and reduced time in institutional care.
Population-level assessment of healthcare cost-effectiveness from the payer's perspective in the Czech Republic: methodology and threshold setting using administrative data.
Fojtíková M, Fiala J
J Med Econ · 2025 Dec · PMID 41572836 · Publisher ↗
OBJECTIVE: The aim of this study was to propose a methodology for assessing the cost-effectiveness of healthcare services at the population level and to establish a corresponding cost-effectiveness threshold from the pay...
OBJECTIVE: The aim of this study was to propose a methodology for assessing the cost-effectiveness of healthcare services at the population level and to establish a corresponding cost-effectiveness threshold from the payer's perspective, taking into account economic sustainability and the data available to the payer. MATERIALS AND METHODS: Analysis was based on data from a Czech health insurance fund covering the period 2014-2024. A binary health status metric, Health Services Derived Disability (HSDD), was developed and calibrated against the Healthy Life Years (HLY) indicator. Subsequently, two cost-effectiveness threshold values were derived from real-world data on healthcare expenditures, mortality, and HSDD: CET (Cost-Effectiveness Threshold per Life Year) and CET (Cost-Effectiveness Threshold per Disability-Free Life Year). Cost-effectiveness can be evaluated using both thresholds within a net monetary benefit framework. RESULTS: CET was derived from expenditures in the 0-69 age group, while the calculation of CET excluded individuals who died in a given year to avoid distortion from high end-of-life costs. For the year 2024, CET was estimated at 465,500 CZK (20,056 USD; 35,918 USD adjusted for PPP), and CET at 96,600 CZK (4,162 USD; 7,454 USD adjusted for PPP). In nominal terms, the year-on-year increase in CET was approximately 7.9%, and in CET approximately 6.5%. LIMITATIONS: The methodology does not account for the subjective dimension of quality of life and may underestimate certain types of disability. HSDD is a binary indicator that does not reflect severity levels. The transferability of the methodology to other healthcare systems depends on the equity of access to health care and the availability of administrative data. CONCLUSION: The proposed methodology enables continuous assessment of the cost-effectiveness of healthcare services at the population level using administrative data. It can serve as a supporting tool for payers in the evaluation, and optimization of healthcare programs and interventions.
Incorporating underreporting of epidemiological burden in COVID-19 models: a targeted literature review.
Chopra I, Yang J, Yehoshua A … +2 more , Mendoza CF, Di Fusco M
J Med Econ · 2026 Dec · PMID 41569003 · Publisher ↗
BACKGROUND: Underreporting of infections, hospitalizations, and deaths can pose challenges to accurately estimating the true burden of COVID-19. Consequently, health burden assessments and economic evaluations may undere...
BACKGROUND: Underreporting of infections, hospitalizations, and deaths can pose challenges to accurately estimating the true burden of COVID-19. Consequently, health burden assessments and economic evaluations may underestimate the public health impact of interventions such as vaccination. METHODS: This targeted literature review summarized economic evaluations of COVID-19 that reported having adjusted for underreporting of epidemiological burden. Searches were performed in PubMed through 08/31/2025 with no geographic restrictions. Key study characteristics extracted: country, time period, population, parameters adjusted for underreporting, and the adjustment multipliers used. A high-level quality assessment of evidence was conducted, building on Drummond checklist and CHEERS. Given the qualitative nature of the question and the expected heterogeneity in study designs, the results were summarized qualitatively. RESULTS: A total of 20 studies met the inclusion criteria. Of these, 14 (70%) reported numerical adjustment factors, and the remaining 30% did not report a numerical factor. The studies covered diverse geographic regions and time frames, with adjustments applied to parameters such as infections, hospitalizations, and mortality. The study quality was moderate to high. The multipliers used ranged widely across studies: 1 to 5 for mortality, 1 to 5 for hospitalizations, and 1 to 10 for infections, where a value higher than 1.0 reflects an adjustment factor for underreporting. The methodologies used to estimate underreporting varied, including comparisons to excess mortality data, Monte Carlo simulations, and validation against external datasets. LIMITATIONS: Most studies used pandemic time horizons. CONCLUSIONS: This review identified 14 modelling studies reporting numerical adjustment factors. The studies used diverse approaches and adjustment factors, reflecting variability in data availability and estimation methods. Recognizing and standardizing these adjustments is crucial for improving the accuracy and comparability of health economic analyses that inform policy decisions. Further research could refine underreporting estimates and assess their impact on economic model outcomes.
Calculating cost per event avoided using a composite number needed to treat.
Toliver J, Wang J, Bhavsar J … +1 more , Sullivan SD
J Med Econ · 2026 Dec · PMID 41562647 · Publisher ↗
OBJECTIVE: Number needed to treat (NNT) and cost per event avoided (CPEA) are measures used to represent the clinical and economic value of chronic treatments and are commonly calculated based on primary endpoints from t...
OBJECTIVE: Number needed to treat (NNT) and cost per event avoided (CPEA) are measures used to represent the clinical and economic value of chronic treatments and are commonly calculated based on primary endpoints from trials. This approach, although widely used, does not reflect the complete value of a treatment, as it does not consider outcomes beyond the primary endpoints. This research aims to overcome this limitation by developing multiple composite NNTs to derive the CPEA to estimate a total value of semaglutide and dulaglutide in people with established cardiovascular disease. METHODS: Two cardiovascular outcomes trials were selected, SELECT (NCT03574597, semaglutide 2.4 mg) and REWIND (NCT01394952, dulaglutide 1.5 mg). NNT was calculated as NNT = 1/ARR where ARR = control event rate - experimental event rate. NNT was estimated for 3-point major adverse cardiovascular events (MACE-3; primary endpoint of the trials), 5-point MACE (MACE-5), and cardiovascular-kidney-metabolic events (CKM). CPEA of MACE-3, MACE-5, and CKM was calculated as NNT*duration of mean follow-up (semaglutide) or duration of median follow-up (dulaglutide)*estimated net price. RESULTS: The NNT decreased as the number of outcomes in the composite endpoint increased, where NNT, NNT, and NNT were 67, 49, and 8 for semaglutide, and 72, 64, and 23 for dulaglutide, respectively. Similarly, the CPEA decreased as the number of outcomes in the composite endpoint increased, where the CPEA for MACE-3, MACE-5 and CKM calculations were $1,662,001, $1,232,417, and $190,387 for semaglutide and $1,884,013, $1,670,366, and $607,886 for dulaglutide. CONCLUSIONS: This research illustrates the limitations of using a NNT focused only on the primary endpoint, as it does not capture the total benefit of the treatment. When considering the value of treatments through NNT or CPEA analyses, a composite endpoint capturing broader benefit should be utilized.
Correction.
J Med Econ · 2026 Dec · PMID 41562633 · Publisher ↗
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Cost-utility and budget impact analysis of dupilumab and oral Janus kinase (JAK) inhibitors for treating moderate-to-severe atopic dermatitis in Taiwan.
Yang RA, Liu VT, Wu DB … +4 more , Shen TK, Tan HH, Wang YH, Lee JT
J Med Econ · 2026 Dec · PMID 41556641 · Publisher ↗
BACKGOROUND: Atopic dermatitis (AD), a chronic inflammatory skin disease, affects 1.28% of Taiwan's population, with 26.69% having moderate-to-severe cases, causing significant healthcare costs and quality-of-life impair...
BACKGOROUND: Atopic dermatitis (AD), a chronic inflammatory skin disease, affects 1.28% of Taiwan's population, with 26.69% having moderate-to-severe cases, causing significant healthcare costs and quality-of-life impairments. Conventional treatments often fail these patients, necessitating novel therapies like dupilumab and Janus kinase (JAK) inhibitors (abrocitinib, baricitinib, upadacitinib). This study evaluates the cost-utility and budget impact of these therapies versus best supportive care (BSC) for adults with moderate-to-severe AD from Taiwan's NHI perspective. METHODS: A hybrid decision tree-Markov model assessed short- and long-term (Years 1-20) outcomes using trial efficacy, NHI costs (2022 NT$), and EQ-5D utilities. Interventions included topical corticosteroids/calcineurin inhibitors. Outcomes were quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs), discounted at 3%, with a NT$2,000,000/QALY threshold. Sensitivity analyses tested robustness. A five-year (2026-2030) budget impact analysis (BIA) included epidemiology and market uptake. RESULTS: Upadacitinib 30 mg was most cost-effective (11.0782 QALYs, ICER NT$1,250,903/QALY vs BSC), followed by Upadacitinib 15 mg (NT$1,376,435/QALY). Dupilumab was dominated; Baricitinib's ICERs exceeded NT$2,000,000/QALY. Sensitivity analyses confirmed robustness, with utility gains, medication costs, and discontinuation rates as key drivers. The BIA estimated a NT$117 billion incremental impact, driven by drug costs, with per-patient increments falling from NT$339,769 to NT$264,728. CONCLUSION: Upadacitinib 30 mg was the most cost-effective option for moderate-to-severe atopic dermatitis in Taiwan, but its high budget impact underscores the need for strategic pricing and reimbursement policies to ensure long-term affordability and access.
Cost-effectiveness analysis of a 20-valent pneumococcal conjugate vaccine for pneumococcal disease prevention in the Slovak pediatric population.
Wannaadisai W, Nassar TP, Šebo P … +2 more , Hroncova D, Kuchta L
J Med Econ · 2026 Dec · PMID 41553236 · Publisher ↗
BACKGROUND: Slovakia has introduced pneumococcal conjugate vaccines (PCVs) in the national immunization program (NIP) since 2011. Recently licensed for use in the pediatric population in Europe, the 20-valent pneumococca...
BACKGROUND: Slovakia has introduced pneumococcal conjugate vaccines (PCVs) in the national immunization program (NIP) since 2011. Recently licensed for use in the pediatric population in Europe, the 20-valent pneumococcal conjugate vaccine (PCV20) is poised to become a new potential standard of care (SoC) in the pediatric national immunization program (NIP). Therefore, this study aims to assess the cost-effectiveness and the clinical and economic outcomes of the PCV20 compared to PCV13 and PCV15 in Slovakian infants. METHODS: A decision-analytic Markov model was employed to estimate the cost-effectiveness of implementing PCV20 versus two lower-valent PCVs in Slovakian infants aged over 10 years. Health outcomes comprised cases of pneumococcal disease, life years, and quality-adjusted life years (QALY). Economic outcomes included vaccination- and disease-associated costs. Outcomes were evaluated for the entire population to capture full vaccine benefits (indirect effects). Epidemiological inputs were sourced from local surveillance reports or published literature. Direct and indirect vaccine effects (VE) were estimated from vaccine impact, effectiveness, and clinical studies of PCV7 and PCV13. Economic inputs were informed by local studies and 2024 Slovak national reimbursement cost. Comprehensive deterministic sensitivity analysis and scenario analyses were examined. The study assumed a willingness-to-pay threshold (WTP) of €40,377/QALY. RESULTS: Over 10 years, PCV20 3 + 1 was predicted to prevent over 810 invasive pneumococcal disease (IPD) cases, 885,000 pneumonia cases, 6,600 otitis media (OM) cases, and 16,600 disease-associated deaths compared with PCV13 2 + 1. The disease prevention subsequently resulted in €326.7 million direct medical costs savings, 96,430,859 additional QALYs gain and an ICER of €1,349/QALY. Comparable trends were observed versus PCV15 2 + 1, with PCV20 preventing more pneumococcal disease cases, providing greater additional QALYs, and being cost-effective under the prespecified threshold. Results were consistent across base case and scenario analyses. CONCLUSIONS: Implementing PCV20 into the Slovakian pediatric NIP is projected to yield the most significant clinical and economic benefits compared to the two lower-valent PCVs, due to its more extensive protection against a broader range of pneumococcal serotypes. Despite an additional cost of dose and visit, PCV20 was estimated to be cost-effective at the Slovak WTP threshold versus other SoC options over a 10-year time horizon.
Prolonged progression-free survival with zanubrutinib in relapsed/refractory CLL: an indirect treatment comparison versus other BTK inhibitors using multilevel network meta-regression.
Shadman M, Bouwmeester W, Mohseninejad L … +5 more , Xu S, Jevdjevic M, Yang K, Williams R, Jansen JP
J Med Econ · 2026 Dec · PMID 41553230 · Publisher ↗
BACKGROUND: Bruton tyrosine kinase inhibitors (BTKis) are therapeutic agents for relapsed/refractory chronic lymphocytic leukemia (R/R CLL). Previous indirect treatment comparisons are limited in simultaneously comparing...
BACKGROUND: Bruton tyrosine kinase inhibitors (BTKis) are therapeutic agents for relapsed/refractory chronic lymphocytic leukemia (R/R CLL). Previous indirect treatment comparisons are limited in simultaneously comparing multiple interventions and adjusting for population differences. This study aimed to use a more rigorous approach called multilevel network meta-regression (ML-NMR) to estimate the relative treatment effects of zanubrutinib compared to acalabrutinib and ibrutinib in two target populations: a general R/R CLL population similar to the phase 3 ALPINE trial's intention-to-treat (ITT) population, and a high-risk population with del(17p) and/or del(11q), similar to the ITT population of the phase 3 ELEVATE-RR trial. METHODS: The ML-NMR was conducted using data from three phase 3 randomized controlled trials: ALPINE ( = 652), ELEVATE-RR ( = 533), and ASCEND ( = 310). Progression-free survival (PFS) and overall survival (OS) were the outcomes of interest. The ML-NMR integrated individual patient data from ALPINE with aggregate data from the other trials, incorporating important effect modifiers to estimate relative treatment effects for the target populations. RESULTS: In the general R/R CLL population, zanubrutinib showed an improved PFS compared to ibrutinib (HR = 0.67, 95% Credible Interval [CrI] = 0.52-0.87) and acalabrutinib (HR = 0.57, 95% CrI = 0.34-0.95). In the high-risk population, zanubrutinib maintained its PFS advantage over ibrutinib and acalabrutinib. OS was similar across BTKis in both populations, with wide CrIs that included an estimate of no difference between treatments. CONCLUSION: This ML-NMR suggests that zanubrutinib offers improved PFS compared to ibrutinib and acalabrutinib in both general and high-risk R/R CLL populations. OS results were uncertain due to limited follow-up.
Cost-effectiveness of female-only and gender-neutral HPV vaccination strategies in Japan.
Kim M, Shim E
J Med Econ · 2026 Dec · PMID 41524327 · Publisher ↗
AIMS: This study evaluated the cost-effectiveness of female-only vaccination (FOV) and gender-neutral vaccination strategies (GNV) using 4-valent (4vHPV) and 9-valent HPV (9vHPV) vaccines among adolescents aged 12-16 yea...
AIMS: This study evaluated the cost-effectiveness of female-only vaccination (FOV) and gender-neutral vaccination strategies (GNV) using 4-valent (4vHPV) and 9-valent HPV (9vHPV) vaccines among adolescents aged 12-16 years in Japan. It provides the first comprehensive assessment of all feasible vaccine-population combinations using a unified dynamic transmission model. MATERIALS AND METHODS: An age- and sex-structured dynamic transmission model simulated HPV transmission and disease progression over 100 years, incorporating cervical, vulvar, vaginal, anal, head and neck, and penile cancers. Six vaccination strategies were evaluated: no further vaccination, FOV with 4vHPV, FOV with 9vHPV, GNV with 9vHPV for females and 4vHPV for males, GNV with 4vHPV for both sexes, and GNV with 9vHPV for both sexes. Analyses adopted a healthcare payer perspective, with costs in 2025 Japanese yen, discounted at 3%. Incremental cost-effectiveness ratios (ICERs) were calculated using a willingness-to-pay threshold of ¥5 million per quality-adjusted life year (QALY). RESULTS: FOV with 4vHPV yielded an ICER of ¥171,725/QALY versus no vaccination, and FOV with 9vHPV yielded ¥1,366,226/QALY versus FOV with 4vHPV - both below the willingness-to-pay threshold. Furthermore, GNV with 9vHPV for both sexes provided the greatest health gains and remained below the threshold (¥3,594,296/QALY) versus FOV with 9vHPV. GNV with 4vHPV and mixed 9vHPV/4vHPV schedules were dominated and excluded. LIMITATIONS: The healthcare payer perspective excludes indirect costs. The model hypothetically includes 9vHPV for boys despite its unavailability for males in Japan. CONCLUSIONS: Both female-only 4vHPV and 9vHPV strategies are cost-effective under Japan's economic standards. Furthermore, GNV with 9vHPV provides greater health benefits and cost-effectiveness.
Cost-effectiveness of pasireotide long-acting release in acromegaly: a systematic literature review and methodology assessment.
Gilis-Januszewska A, Bronikowska M, Binowski G … +4 more , Jachimowicz M, Gueron B, Schmidt F, Kossi SD
J Med Econ · 2026 Dec · PMID 41518621 · Publisher ↗
BACKGROUND: Acromegaly is a rare and progressive condition caused by excessive secretion of growth hormone and insulin-like growth factor type 1. Pasireotide long-acting release (LAR) is indicated as a second-line therap...
BACKGROUND: Acromegaly is a rare and progressive condition caused by excessive secretion of growth hormone and insulin-like growth factor type 1. Pasireotide long-acting release (LAR) is indicated as a second-line therapy for adults with acromegaly who are unsuitable for or unresponsive to surgery and inadequately controlled with first-generation somatostatin receptor ligands (FGSRLs). Although its efficacy and safety have already been established, its cost-effectiveness remains unclear. The primary objective of this study was to systematically assess the cost-effectiveness of pasireotide LAR compared to other second-line medical options, particularly pegvisomant. METHODS: A systematic literature review was conducted in May 2024 in Medline, Medline In Process, Web of Science, and the Centre for Reviews and Dissemination (CRD), York. Studies were eligible if they were full or partial economic analyses of pasireotide LAR as a second-line pharmacological treatment for adult patients with acromegaly. Studies not available in English and publications prior to 2009 were excluded. Included articles were assessed for transparency using the appropriate checklists. Data extraction focused on costs, incremental cost-effectiveness ratios and quality-adjusted life years. Formal data synthesis of outcomes was not undertaken due to the small number of studies and notable heterogeneity between them. Methodological assessment involving the evaluation of model inputs, data sources, and their alignment with the evidence on the course of disease and clinical practice was performed to examine the main factors contributing to discrepancies in cost-effectiveness outcomes and assess the credibility of the results. RESULTS: Of 160 records identified, six publications met the inclusion criteria. Their findings demonstrated variability. The critical assessment highlighted considerable variability in methodological rigor among the included studies. CONCLUSIONS: Evidence on pasireotide LAR's cost-effectiveness versus pegvisomant regimens remains inconclusive. Although two studies indicated that pegvisomant is more cost-effective, the study with the highest methodological credibility found that pasireotide LAR is a cost-effective alternative.
Cost per responder analysis of iptacopan versus eculizumab and ravulizumab in treatment of paroxysmal nocturnal hemoglobinuria: implications for decision-making.
Than KS, Shafrin J, Muthukrishnan S … +3 more , Paulose J, Bilano V, Kuypers N
J Med Econ · 2026 Dec · PMID 41518611 · Publisher ↗
OBJECTIVE: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and debilitating hematological disease with significant economic burden. Despite the availability of multiple therapies, there is a lack of consensus on opti...
OBJECTIVE: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and debilitating hematological disease with significant economic burden. Despite the availability of multiple therapies, there is a lack of consensus on optimal treatment strategies among physicians and payers in the United States. This study aimed to evaluate the economic value of iptacopan, a novel oral treatment option, compared to terminal complement inhibitors (specifically, complement component C5 inhibitor or C5i)-including eculizumab and ravulizumab-among patients with PNH who are either (i) C5i-experienced or (ii) complement-inhibitor-naïve. METHODS: A cost per responder analysis was conducted based on treatment efficacy from clinical trials comparing iptacopan with C5i treatments. Treatment response was defined as the proportion of patients achieving red blood cell transfusion independence. Treatment costs were estimated as pharmaceutical wholesale acquisition cost and treatment administration costs, accounting for discontinuation. Outcomes evaluated included the number needed to treat to achieve a response and the cost per responder over the treatment duration of 24 weeks. RESULTS: Over 24 weeks, the number needed to treat to achieve an additional response was lower for iptacopan than all C5i comparators (C5i-experienced: 1.05 with iptacopan vs. 3.86 with C5is; complement-inhibitor-naïve: 1.02 with iptacopan vs. 1.69 with C5is). Cost per responder was lower for iptacopan than C5i comparators for both C5i-experienced ($264,337 for iptacopan vs. $975,298 for ravulizumab, $1,060,511 for eculizumab, and $744,561-$955,194 for eculizumab biosimilar with 10%-30% discount from eculizumab cost) and complement-inhibitor-naïve patients ($256,754, vs. $428,139 for ravulizumab, $465,546 for eculizumab, and $326,849-$419,314 for eculizumab biosimilar). CONCLUSION: Among both C5i-experienced and complement-inhibitor-naïve patients, treatment with iptacopan resulted in higher response rates and lower cost per responder compared to C5is.
Clinical benefits and cost saving of achieving composite treatment targets for type 2 diabetes - A modeling study.
Si S, Wang R, Li J … +3 more , Ding Y, Liu Y, Osumili B
J Med Econ · 2026 Dec · PMID 41504525 · Publisher ↗
OBJECTIVES: This study assessed the long-term clinical benefits and cost savings associated with achieving composite treatment targets (CTT) of stringent glycemic control, weight reduction and no hypoglycemia in predomin...
OBJECTIVES: This study assessed the long-term clinical benefits and cost savings associated with achieving composite treatment targets (CTT) of stringent glycemic control, weight reduction and no hypoglycemia in predominantly Chinese patients with Type 2 Diabetes (T2D) inadequately controlled with metformin and/or sulfonylurea. METHODS: The study was conducted using an implementation of the UK Prospective Diabetes Study Outcomes Model Version 2 (UKPDS OM2) in Microsoft Excel, with additional modules for treatment switching, weight change, and hypoglycemia. Thirty-year healthcare costs were projected to capture macro- and microvascular complications, hypoglycemia and diabetic treatment. Baseline and efficacy inputs were extracted from the SURPASS-AP-Combo trial (NCT04093752), a predominantly Chinese cohort. Cost inputs were derived from literature review. Patients were categorized as "Achieved" or "Failed" based on whether they met the CTT (i.e. HbA1c ≤6.5%, ≥10% weight reduction, and no hypoglycemia event [blood glucose < 3.0 mmol/L or severe hypoglycemia]) at the end of SURPASS-AP-Combo trial, regardless of treatment received. For the Achieved group, sustained CTT was assumed for 3, 5, or 10 years before natural disease progression per UKPDS OM2 progression trajectories. The Failed group followed UKPDS OM2 progression trajectories throughout. Treatment intensification to basal-bolus insulin was triggered when HbA1c levels reached predefined CTT-based thresholds. Scenario analyses applied less stringent CTT. RESULTS: Sustained achievement of CTT for 3, 5 and 10 years yielded 0.31, 0.40 and 0.56 quality-adjusted life years (QALYs) and cost savings of ¥22,336, ¥32,692, ¥53,234 per patient, respectively. These savings were attributable to reduced complications, hypoglycemia and delayed treatment intensification. Slightly smaller savings were observed applying less stringent CTT. CONCLUSIONS: In this modeling study, a sustained achievement of CTT led to improved clinical benefits and significant direct medical cost savings. The longer the achievement period and the more stringent CTT, the greater the clinical benefits and cost savings.
A value assessment of patient-level outcomes and productivity loss for intravenous and subcutaneous lecanemab for patients with early Alzheimer's disease.
Ressa R, Ettinger J, Chowdhury E … +4 more , Graham L, Ndirangu K, Mancebo JZ, Bodnar C
J Med Econ · 2025 Dec · PMID 41488982 · Publisher ↗
AIMS: Intravenous (IV) lecanemab is approved for the treatment of patients with early Alzheimer's disease (AD); a subcutaneous (SC) option may offer additional benefits. We assessed the overall value of SC treatments, an...
AIMS: Intravenous (IV) lecanemab is approved for the treatment of patients with early Alzheimer's disease (AD); a subcutaneous (SC) option may offer additional benefits. We assessed the overall value of SC treatments, and direct/indirect outcomes associated with IV and SC lecanemab. METHODS AND MATERIALS: For the narrative review, PubMed was searched (February 2025) for studies comparing patient preferences for IV/SC treatment administration published between 2015-2025. Study eligibility was determined using patient, intervention, comparator, outcomes, and study criteria. For the decision-analytic model, a Markov model was developed with four lecanemab treatment scenarios. Scenarios one to three included IV initiation (10 mg/kg biweekly) to month 18, followed by either IV initiation continued (10 mg/kg biweekly), SC maintenance (250 mg weekly) or IV maintenance (10 mg/kg every 4 weeks). Scenario four included SC initiation (500 mg weekly) for an 18-month period, followed by SC maintenance (250 mg weekly). Outcomes were administration time/frequency; patient, caregiver, and healthcare professional time; and caregiver productivity loss. RESULTS: Forty-three publications reported patient treatment preferences. Most (88.4%) reported that patients preferred SC over IV. Key reasons for this were time savings ( = 13/43 studies; 30.2%), convenience ( = 11/43; 25.6%), treatment frequency ( = 12/43; 27.9%). Two studies ( = 2/43; 4.7%) reported an IV preference over SC; for three studies ( = 3/43; 7.0%), treatment preference was driven by administration frequency. Decision-analytic modeling of lecanemab treatment scenarios revealed that IV initiation to IV maintenance had the lowest number of administrations, whereas SC initiation to SC maintenance had the lowest number of treatment hours and caregiver productivity losses. LIMITATIONS: Caution must be taken when generalizing these results for all AD patients. CONCLUSIONS: SC treatments show value as a therapeutic option. IV and SC lecanemab availability may offer benefits to patients, caregivers, and society, and improve shared decision making.
Cost-effectiveness of sotorasib versus adagrasib in previously treated G12C-mutated advanced NSCLC: a US healthcare payer perspective.
Karim N, Waterhouse D, Jones S … +1 more , Stollenwerk B
J Med Econ · 2025 Dec · PMID 41488979 · Publisher ↗
AIM: To evaluate the cost-effectiveness of sotorasib versus adagrasib in the second- and later lines of treatment for G12C non-small cell lung cancer (NSCLC) from a US private payer perspective. METHODS: A standard thre...
AIM: To evaluate the cost-effectiveness of sotorasib versus adagrasib in the second- and later lines of treatment for G12C non-small cell lung cancer (NSCLC) from a US private payer perspective. METHODS: A standard three-state partitioned survival model was used with a 20 year (life-time) horizon. Equivalent progression-free survival (PFS) and overall survival (OS) were assumed in the base case based on published matching-adjusted indirect comparisons (MAICs) of Phase 2 and 3 data, and time-to-death utilities applied. Treatment-related adverse events (TRAEs) common to both treatments were included. Direct costs and health benefits were discounted at 1.5% annually. Model robustness was explored through probabilistic sensitivity analysis (PSA) and inputs varied in scenario analyses. RESULTS: In the base case, sotorasib was more cost-effective than adagrasib, driven by lower acquisition cost and TRAE frequency. Total discounted costs were $18,004 higher for adagrasib than sotorasib ($246,557 vs $228,553), comprising: drug acquisition ($9,478), TRAE management ($4,103) and comedications (antiemetics and antidiarrheal agents; $4,424). Sotorasib was dominant at equivalent efficacy (1.20 quality-adjusted life-years [QALYs]). Net monetary benefit was $18,031 at a willingness-to-pay threshold (WTP) of $150,000/QALY. In the PSA, there was a higher probability of sotorasib being more cost-effective than adagrasib at all WTP thresholds (62% at a WTP of $150,000/QALY). Sotorasib was consistently more cost-effective than adagrasib in scenario analyses exploring relative efficacy, discount rate, time horizon, and utilities, with ICERs well below the $150,000/QALY WTP threshold. LIMITATIONS: MAIC-based comparative effectiveness was used in the absence of head-to-head trial data; conclusions informed by MAIC should be interpreted with caution; long-term projections are limited without mature OS data; published data sources may be based on different populations. CONCLUSION: Sotorasib was more cost-effective than adagrasib in the second- and subsequent-line treatment of G12C NSCLC, based on current efficacy and safety data.
Surgical aortic valve replacement with a novel bovine pericardial tissue valve: a real world comparison of short-term costs and outcomes in US hospitals.
Brown CR, Szeto WY, Dratch A … +4 more , Murphy SME, Shanbhag A, Reifenberger M, Umaña JP
J Med Econ · 2025 Dec · PMID 41487071 · Publisher ↗
AIMS: Use of bioprosthetic tissue valves in surgical aortic valve replacement (SAVR) has increased in recent years. The effect of novel bovine pericardial tissue valves on short-term costs and utilization has not been es...
AIMS: Use of bioprosthetic tissue valves in surgical aortic valve replacement (SAVR) has increased in recent years. The effect of novel bovine pericardial tissue valves on short-term costs and utilization has not been established. We assessed INSPIRIS RESILIA aortic tissue valves (*Edwards Lifesciences, Irvine CA) during SAVR hospitalization and 90 days post-discharge compared to other tissue valves for all ages, and to mechanical valves for patients of shared decision-making age (50-65). MATERIALS AND METHODS: Retrospective observational study using US hospital data. Adults admitted for first-time SAVR 2018-2021 with identifiable tissue or mechanical valves were analyzed. Outcomes were adjusted for demographics, comorbidities, procedure and hospital characteristics using generalized linear modeling. RESULTS: 20,125 patients were analyzed; majority were male, with elective procedures in academic hospitals. Compared to other tissue valves, length of stay and ICU stay were shorter with INSPIRIS ( < 0.001). In-hospital mortality was not statistically different. Readmissions at 30 days were 7.1% with INSPIRIS, 9.0% other tissue valves; 10.4% vs. 13.0% at 90 days (both < 0.001). Reduced cost of readmission (-$1,013 at 90 days) partially offset the difference of $2,429 in initial hospitalization cost. INSPIRIS valves compared to mechanical valves (ages 50-65) showed similar patterns in utilization. In-hospital mortality was lower with INSPIRIS than mechanical valves (2.0% vs. 3.2%, = 0.009), but did not differ when limited to isolated SAVR only. Initial hospitalization cost was higher for INSPIRIS ($7,079, < 0.001), with lower 90-day readmissions cost (-$899, < 0.05). LIMITATIONS: Non-randomized analysis of real-world data. CONCLUSIONS: Patients undergoing SAVR with INSPIRIS valves had shorter length of stay and fewer readmissions compared to other tissue valves and to mechanical valves. Reduced cost of readmissions partially offset higher cost of initial SAVR admission. Further research is warranted to explore this association between valve used and utilization outcomes.
Costs per responder for patients with relapsed or refractory multiple myeloma treated with Talquetamab compared with usual care.
Lee HC, Yang C, Liu YH … +7 more , Mu F, Wang J, Goble J, Patel N, Zhang X, Grajales-Cruz AF, Jiao T
J Med Econ · 2026 Dec · PMID 41483183 · Publisher ↗
AIMS: To evaluate costs per responder for patients with triple-class exposed (TCE) relapsed or refractory multiple myeloma (RRMM) receiving talquetamab (Tal) on weekly (QW) and biweekly (Q2W) dosing schedules, compared w...
AIMS: To evaluate costs per responder for patients with triple-class exposed (TCE) relapsed or refractory multiple myeloma (RRMM) receiving talquetamab (Tal) on weekly (QW) and biweekly (Q2W) dosing schedules, compared with usual care from a United States commercial payer's perspective. METHODS: A cost per responder model was developed over a 6-month time horizon, incorporating pre-progression and post-progression costs. For Tal QW and Tal Q2W, pre-progression costs included costs of drug acquisition, inpatient step-up doses (hospitalization, pre-medication, and tocilizumab), outpatient visits, and monitoring. Pre-progression costs for usual care were estimated based on a weighted average of the 10 most used regimens in a real-world LocoMMotion/MoMMent study, including costs of acquisition, administration, co-medication, and monitoring. Post-progression costs included subsequent treatment for a subset of patients and terminal care costs prior to death. All costs were reported in 2025 United States Dollars. Clinical data of overall response rate (ORR), progression-free survival, and overall survival were obtained from an indirect treatment comparison using MonumenTAL-1 (September 2024 data cut) and LocoMMotion/MoMMent (October 2022 and August 2023 data cuts) as data sources. Deterministic sensitivity analyses and scenario analyses were conducted to assess the robustness of model results. RESULTS: Over the 6-month period, the total cost of care was $179,556 for usual care, $295,993 for Tal QW, and $315,135 for Tal Q2W. Despite higher costs, Tal demonstrated superior ORR, resulting in lower cost per responder: $575,962 for usual care, $405,470 for Tal QW, and $443,165 for Tal Q2W, representing a 23-30% reduction in cost per responder with Tal. Sensitivity and scenario analyses showed consistent findings. CONCLUSION: Although Tal QW and Q2W are associated with higher total per-patient costs compared with usual care, they offer improved clinical effectiveness, resulting in lower cost per responder. These findings suggest greater economic value for Tal in the treatment of TCE RRMM.
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