Journal of Medical Economics最新文献

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 (N = 652), ELEVATE-RR (N = 533), and ASCEND (N = 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.

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.

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.

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_LY (Cost-Effectiveness Threshold per Life Year) and CET_DFLY (Cost-Effectiveness Threshold per Disability-Free Life Year). Cost-effectiveness can be evaluated using both thresholds within a net monetary benefit framework.

Results: CET_LY was derived from expenditures in the 0-69 age group, while the calculation of CET_DFLY excluded individuals who died in a given year to avoid distortion from high end-of-life costs. For the year 2024, CET_LY was estimated at 465,500 CZK (20,056 USD; 35,918 USD adjusted for PPP), and CET_DFLY at 96,600 CZK (4,162 USD; 7,454 USD adjusted for PPP). In nominal terms, the year-on-year increase in CET_LY was approximately 7.9%, and in CET_DFLY 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.

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 (n = 13/43 studies; 30.2%), convenience (n = 11/43; 25.6%), treatment frequency (n = 12/43; 27.9%). Two studies (n = 2/43; 4.7%) reported an IV preference over SC; for three studies (n = 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.

Aim: To evaluate the cost-effectiveness of sotorasib versus adagrasib in the second- and later lines of treatment for KRAS 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 KRAS G12C NSCLC, based on current efficacy and safety data.

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 (p < 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 p < 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%, p = 0.009), but did not differ when limited to isolated SAVR only. Initial hospitalization cost was higher for INSPIRIS ($7,079, p < 0.001), with lower 90-day readmissions cost (-$899, p < 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.

Aims: Systemic corticosteroids (SCS) are used to manage asthma exacerbations. Among the broad population of patients with asthma, SCS-related risk of adverse events (AEs), health care resource utilization (HCRU), and costs remain unclear.

Materials and methods: This retrospective cohort study used the Optum Research Database claims to identify adults with asthma from 1/1/2017 to 6/30/2022. The index date was the earliest SCS claim for SCS users; non-SCS users were randomly selected and adjusted proportionally to SCS users by index year. SCS use was measured during the first 12 months of follow-up. Inverse probability of treatment weighting balanced the two cohorts for selected baseline demographic and clinical characteristics. SCS users were further stratified into low, medium, and high dose sub-cohorts. SCS-related AEs were assessed up to 48 months, while HCRU and costs were assessed during the first 12 months of follow-up. A generalized linear model (GLM) analyzed follow-up costs by SCS exposure.

Results: The 130,739 patients included 55,363 non-SCS users (42.3%), while 75,376 were SCS users stratified into 60,319 low-, 12,235 medium-, and 2,822 high-dose users. The mean age was 49.6 years; 61.8% were female and 68.9% were non-Hispanic White. SCS users had a significantly greater risk of new-onset acute and chronic SCS-related AEs, increasing incrementally with dose exposure (all p < .001) across numerous physiological systems. Follow-up HCRU and costs also rose incrementally with dose exposure (all p < .001). Compared with non-users, SCS-related costs were 1.43, 1.97, and 3.21 times higher among low-, medium-, and high-dose users, respectively. The adjusted GLM predicted a 9.9% cost increase per 100 mg of prednisone equivalents.

Limitations: Retrospective administrative claims studies cannot randomize patients and may not capture all patient events.

Conclusions: Among a broad population of adults with asthma, even low doses of SCS were associated with significantly increased risk of new-onset AEs, HCRU, and costs.

Introduction: Despite the inclusion of pneumococcal conjugate vaccines (PCV) in the pediatric national immunization program (NIP) since 2017, Romania continues to face a substantial clinical, economic, and societal burden of pneumococcal disease. Higher-valent vaccines, such as 20-valent PCV (PCV20), offer broader serotype coverage versus the current standard of care (13-valent PCV; PCV13) with the potential to reduce disease burden. To test this, we conducted a cost-effectiveness analysis of switching from PCV13 or a potential future comparator (15-valent PCV; PCV15), both under a 2 + 1 schedule, to PCV20 under a 3 + 1 schedule in the Romanian pediatric NIP.

Methods: A population-based, multi-cohort Markov model with a target population of children aged <2 years was utilized to estimate the cost and health impact of PCV20 versus lower-valent comparators over 10 years. The model adopted a Romanian societal perspective, encompassing both direct and indirect costs, with an annual cycle. Sensitivity and scenario analyses were conducted to assess the robustness of the model and its assumptions.

Results: In the base-case analysis, PCV20 demonstrated dominance versus PCV13 and PCV15 (i.e. was more effective and less costly), with total predicted cost-savings of 79,123,267 and 206,623,098 Romanian Leu, respectively, plus reduction in pneumococcal disease cases by 246,245 and 223,914, respectively. The majority of sensitivity and scenario analyses of both pairwise comparisons were aligned with the base case.

Conclusion: The results of this analysis indicate that PCV20 implementation into the Romanian pediatric NIP would greatly reduce pneumococcal disease burden and would be a cost-effective approach versus PCV13 or PCV15 from a societal perspective over 10 years.