Journal of Medical Economics最新文献

Aim: The objective of this study was to evaluate the value of three secondary stroke prevention strategies in Australia: the Cardioform and Amplatzer Patent Foramen Ovale (PFO) closure devices, and medical management.

Materials and methods: An eight-state Markov model was employed to simulate a cohort of 1,000 patients with a history of PFO-associated stroke over a five-year time horizon. Treatment strategies included Cardioform, Amplatzer, and medical therapy alone. Effectiveness data were derived from the REDUCE and RESPECT trials, a matching-adjusted indirect comparison (MAIC), and prior cost-effectiveness studies. Costs, presented from an Australian healthcare perspective and expressed in 2023 AUD, were used to calculate quality-adjusted life-years (QALYs), strokes prevented, the incremental cost-effectiveness ratio (ICER), and net monetary benefit (NMB).

Results: Compared to Amplatzer, treatment with Cardioform yielded cost savings to the Australian health care systems (NMB of AUD 3.7 million) and improved patient outcomes (yielded 26.48 additional QALYs and prevented 28 more recurrent strokes). Relative to medical therapy alone, Cardioform resulted in improved patient outcomes and was cost-effective, with an ICER of $11,784/QALY. Cardioform provides an NMB of AUD 14.3 million and yielded 374.5 additional QALYs beside preventing 67 more strokes compared to medical therapy alone.

Conclusion: Cardioform appears more cost-effective in the prevention of secondary PFO-associated strokes, supporting its adoption in clinical practice.

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.

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.

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: COVID-19 disease burden in United States (US) adults ≥65 years and persons with underlying medical conditions remains high. This modeling study estimated the cost-effectiveness of the next-generation COVID-19 mRNA-1283 vaccine in persons aged 12-64 at high risk of severe COVID-19 outcomes and all adults ≥65 years.

Methods: mRNA-1283 was compared with no annual vaccination and originally licensed mRNA vaccines mRNA-1273 and BNT162b2. Analyses were conducted using a static decision-analytic model (1-year horizon). Vaccine effectiveness (VE) against infection and hospitalization for mRNA-1283 versus no vaccination was based on relative VE (rVE) from the Phase 3 pivotal randomized controlled trial comparing mRNA-1283 against mRNA-1273, and mRNA-1273 real-world data. rVE estimates for mRNA-1283 versus BNT162b2 were based on an indirect treatment comparison. The societal incremental cost per quality-adjusted life-year (QALY) gained and the benefit-cost ratio (BCR) were calculated.

Results: During the 2025/2026 season, a single dose of mRNA-1283 was estimated to yield an incremental cost per QALY gained of $16,247 compared with no vaccine. The BCR for the base case strategy ranged from $2.16-9.74 returned for every $1 spent for mRNA-1283. mRNA-1283 was estimated to dominate originally-licensed mRNA-COVID-19 vaccines in analyses of the target population. Results were sensitive to COVID-19 incidence, hospitalization rates, post-discharge mortality rates, and VE.

Limitations: The real-world effectiveness and safety of mRNA-1283 have not yet been established, and relative VE estimates should be validated with real-world data. Full year 2025/2026 COVID-19 incidence and vaccine uptake in the US is uncertain.

Conclusions: Study results suggest mRNA-1283 may represent a highly cost-effective strategy (considering a $100,000-150,000 per QALY willingness-to-pay threshold) to reduce COVID-19 burden. Based on rVE assumptions made, mRNA-1283 was estimated to dominate originally-licensed mRNA vaccines in this recommended population. mRNA-1283 may provide a valuable option to optimize US COVID-19 immunization programs and protect those most vulnerable.

Background: Ciltacabtagene autoleucel (cilta-cel) has demonstrated remarkable efficacy in relapsed or refractory multiple myeloma (RRMM). Outpatient (OP) administration of cilta-cel is increasingly used to improve access and reduce healthcare resource utilization (HCRU) compared to inpatient (IP) administration. We compared all-cause HCRU and clinical outcomes of IP versus OP administration of cilta-cel in patients with RRMM after ≥4 prior lines of therapy (LOT) in clinical practice.

Methods: We identified adults receiving cilta-cel between 02/28/2022 and 06/30/2024 after ≥4 prior LOT using Komodo Research Database claims data and classified patients into cohorts by setting of administration (IP/OP). All-cause HCRU, treatment-free interval (TFI), overall survival (OS), and clinical events (e.g. cytokine release syndrome [CRS], immune effector cell-associated neurotoxicity syndrome [ICANS]) were assessed. Outcomes were compared using multivariate regression and reported as incidence rate ratios (IRR) with 95% confidence intervals (CI).

Results: Among 242 patients, 148 (61.2%) received cilta-cel in IP and 94 (38.8%) in OP. Baseline characteristics were comparable between cohorts. Of patients in the OP cohort, 31.9% did not require an IP admission within 3 months post-infusion. During this period, the OP cohort had significantly fewer IP days per-patient-per-month (2.4 vs. 6.6; IRR [95% CI]: 0.37 [0.28; 0.48], p < 0.001) and more OP days (8.5 vs 5.4; IRR [95% CI]: 1.43 [1.26; 1.63], p < 0.001) than the IP cohort. From the fourth month post-infusion, no significant differences in HCRU were observed. Rates of CRS and ICANS, and long-term outcomes including 6 and 12 month TFI and OS were similar.

Conclusion: OP administration of cilta-cel yielded similar effectiveness and safety outcomes relative to IP administration, while significantly reducing IP resource use. These findings support the feasibility of OP administration of cilta-cel in treating RRMM patients and its potential to reduce the burden on the healthcare system.

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.