Journal of Medical Economics最新文献

Aims: In the phase 3 KEYNOTE-689 trial (NCT03765918) among patients with resectable locally advanced head and neck squamous cell carcinoma (LA HNSCC), perioperative pembrolizumab (pembrolizumab before surgery, then continued with standard-of-care [SOC] radiotherapy +/- cisplatin after surgery followed by pembrolizumab alone) significantly prolonged event-free survival vs. SOC alone, both in the intention-to-treat population and PD-L1 combined positive score (CPS) ≥1 subgroup. Perioperative pembrolizumab + SOC received Food and Drug Administration approval in June 2025 for resectable LA HNSCC expressing PD-L1 (CPS ≥ 1). The present study evaluated the cost-effectiveness of perioperative pembrolizumab + SOC versus SOC in this indication, from a US healthcare payer perspective.

Materials and methods: A Markov cohort model with four states (event-free, local recurrence, incurable recurrence/progression, death) was developed to estimate lifetime costs, life years (LYs), and quality-adjusted LYs (QALYs) with 3% annual discounting. Transition probabilities were fitted to patient-level time-to-event data from KEYNOTE-689 through parametric multistate modelling. Costs of initial and subsequent treatment, adverse events, disease management, and terminal care were estimated in 2025$ based on trial results, drug labels, public databases, and literature. Utilities were derived through analyses of EQ-5D-5L data collected in KEYNOTE-689. Deterministic and probabilistic sensitivity analyses were performed.

Results: Compared to SOC, perioperative pembrolizumab + SOC increased total costs by $82,311 and provided gains of 1.47 QALYs and 1.77 LYs. Incremental cost-effectiveness ratios of perioperative pembrolizumab + SOC vs. SOC were $55,863/QALY and $46,406/LY. Higher initial treatment costs of perioperative pembrolizumab (incurred mainly in Year 1) were partially offset by lower recurrence-related costs. At the typical $150,000/QALY threshold, perioperative pembrolizumab + SOC was cost-effective in 96% of probabilistic simulations.

Limitations: Survival extrapolations beyond the available trial period are subject to uncertainty.

Conclusions: Perioperative pembrolizumab + SOC was found to be cost-effective versus SOC for the treatment of resectable LA HNSCC with CPS ≥ 1.

Background: In the US, breast cancer is the most common female cancer. Personalized treatment strategies, informed by genomic assays and germline genetic testing (GGT), can optimize therapeutic decisions and improve patient outcomes along the hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer treatment pathway.

Purpose: To evaluate the cost-effectiveness of combining molecular tools, including the 21-gene assay, GGT, and comprehensive genomic profiling (CGP) when compared to no testing, to inform the treatment of patients with HR+/HER2- breast cancer from the US societal perspective.

Methods: A health economic model was developed which stratified patients with node-negative (N0) and node-positive (1-3 positive nodes) (N1) early-stage breast cancer according to the 21-gene assay and GGT. Long-term outcomes were simulated using a Markov model. CGP was used to stratify patients who progressed to advanced disease for actionable genomic alterations. Clinical inputs and costs were sourced from published literature. A clinician survey was used for inputs to reflect current US clinical practice.

Results: Over a lifetime horizon, the full testing strategy was dominant (more effective and cost-saving) in N0 patients and cost-effective in N1 (ICER = $54,734/QALY) patients. The model estimated quality-adjusted life year (QALY) gains of 0.322 and cost savings of $7,168 for N0 patients and 0.130 QALYs gain and additional costs of $7,109 for N1 patients. Sensitivity analyses supported the robustness of the analysis.

Conclusion: Compared to no testing, the full testing strategy of genetic and genomic testing was more effective at a lower cost or was cost-effective, supporting the goal to increase the survival and the quality-of-life of women with breast cancer along the cancer care continuum.

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.

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%; p < 0.001) and emergency room visits (61.1% vs. 39.1%; p < 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 (p < 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.

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.

Aim: Emicizumab has changed prophylactic treatment for HA, but the need for FVIII replacement still exists for the treatment of breakthrough bleeds, perioperative management, and participation in high-risk activities/sports. The primary objectives of this study were to describe the characteristics of individuals with congenital hemophilia A (HA) without inhibitors receiving emicizumab prophylaxis and to compare clinical outcomes, healthcare resource utilization (HCRU), and costs among those prescribed octocog alfa [Advate, standard half-life factor VIII (FVIII)] versus extended half-life (EHL) FVIII agents.

Methods: Patients with congenital HA without inhibitors and emicizumab use were identified retrospectively from Inovalon claims data and stratified by concomitant octocog alfa vs. EHL FVIII use. Inverse probability treatment weighting (IPTW) and multivariable regression were used to balance baseline covariates and compare billable annualized rate of bleeding (ABR_b), healthcare utilization, and costs between subgroups.

Results: Among the 1,282 patients receiving emicizumab prophylaxis, 501 received concomitant FVIII prescription for octocog alfa (n = 274) or EHL (n = 227) during follow-up. Following IPTW adjustment, total and medical costs were comparable between groups; however, pharmacy costs associated with concomitant octocog alfa therapy were significantly lower compared with EHL products ($35,381 vs. $61,739; p < 0.001). Importantly, ABR_b (0.35 vs. 0.28; p = 0.34) and bleed-related healthcare utilization remained similar across cohorts.

Limitations: Findings are limited by the retrospective design and reliance on claims data, which may omit home-treated bleeds, and misrepresent FVIII utilization due to billing complexities and assumptions about dispensed product use.

Conclusions: While the choice of concomitant FVIII does not substantially influence ABR_b or bleed-related HCRU, significantly lower pharmacy costs with octocog alfa compared with EHL agents highlight its potential cost saving and support its consideration as a preferred on-demand treatment option in patients with HA without inhibitors on emicizumab prophylaxis.

Aim: This retrospective observational cohort study aimed to assess healthcare utilization (HCRU) and costs before and after initiation of treatment with maribavir using a United States (US) administrative claims database to further the understanding of the impact of maribavir in the real world setting.

Methods: Inovalon Databases were used to identify post-transplant patients with refractory (with or without resistant) CMV treated with maribavir between November 1, 2021 and March 31, 2024 (index = earliest treatment date). HCRU and costs were assessed during variable length periods both prior to and following initiation of maribavir. McNemar's tests were used to evaluate the statistical significance of differences for categorical variables and paired t-tests were used for continuous variables.

Results: There were 230 patients eligible for analysis. Compared with the pre-maribavir phase, after the initiation of maribavir there was a significant decrease in the utilization of all-cause acute care services, including inpatient hospital admissions (54.3% v. 30.9%; p < 0.001) and emergency room visits (47.0% v. 36.5%; p = 0.014). There was a significant decrease in the number of outpatient office visits per patient per month (PPPM) (2.33 v. 1.47; p < 0.001) as well as a decrease in the number of all outpatient pharmacy prescriptions PPPM (9.64 v. 7.69; p < 0.001). Total medical costs measured PPPM prior to maribavir initiation were $9,986 compared with $5,480 (p < 0.001) after initiation of maribavir.

Conclusions: In real world practice, initiation of maribavir for the treatment of refractory or drug-resistant post-transplant CMV was associated with decreased acute care service utilization, reduced outpatient visits, and lower healthcare costs, resulting in meaningful benefits for both patients and healthcare systems. These reductions in healthcare utilization highlight maribavir's role in optimizing treatment strategies and improving the efficiency of CMV management.

Background: COVID-19 annual vaccination uptake in Spain remains suboptimal. This study aimed to estimate the clinical and economic impact of the 2023/2024 COVID-19 vaccination campaign in individuals aged ≥60 years (scenario A: coverage of 33.14% for ages 60-69, 53.15% for 70-79, and 65.32% for ≥80), and to compare it with a hypothetical scenario (scenario B) where coverage reaches the 75% target set by the Spanish Ministry of Health.

Methods: A combined Markov-decision tree model adapted to the Spanish context simulated the weekly progression of the target population through six health states over one year. Infected individuals entered a decision tree reflecting different care pathways (outpatient, hospital ward, ICU with/without invasive mechanical ventilation [IMV], or death), each associated with specific health outcomes and direct costs (€2024). Clinical and economic outcomes were compared between scenarios A and B. Sensitivity analyses explored incremental increases in coverage and age-specific impacts. The analysis was conducted from the National Healthcare System (NHS) perspective.

Results: Under scenario A, 378,970 symptomatic infections occurred, leading to 27,611 hospitalizations, 742 ICU admissions (47.3% requiring IMV), and 3,611 deaths. A total of 2,750 quality-adjusted life years (QALYs) were lost, and COVID-19-related care costs reached €240.4 million (85.7% from inpatient care). Scenario B, with 75% coverage, averted -19,409 symptomatic infections, 1,094 hospitalizations, 41 ICU admissions, and 129 deaths, 138 lost QALYs and total cost savings of about €10.5 million. Sensitivity analysis showed how the model is sensitive to sequential increases (10% by 10%) in vaccination rates and highlighted the importance of achieving high vaccination rates, especially in older age groups.

Conclusions: This analysis reveals the significant impact that increasing annual COVID-19 vaccination coverage among the Spanish population over 60 could have in preventing new infections, reducing severe disease consequences, and generating considerable cost savings for the NHS.

Detecting and treating cancer at an early stage is critical for improving patient survival, quality of life, and health system efficiency. Early diagnosis offers substantial clinical benefits, reduces the need for aggressive treatments associated with advanced disease, and lowers healthcare costs. Despite these benefits, disparities in early-stage detection persist across tumor types due to challenges in screening, public awareness, and the aggressive nature of certain cancers. While early-stage diagnosis generally offers a better prognosis than late-stage detection, disease recurrence remains a significant reality and concern. Many patients experience a relapse of cancer despite initial curative treatment, which adversely affects their survival, quality of life, and financial stability. While effective new treatments for early-stage cancers have emerged, including immunotherapy and targeted therapies, barriers to reimbursement and access persist. One challenge is the absence of mature overall survival data at the time of regulatory and reimbursement approvals for most tumor types, which can result in delayed decision-making, reduced patient access, and worse outcomes. This policy paper combines insights from clinicians, health economists, outcomes researchers, and policy experts to address gaps in early-stage cancer care and provide recommendations to enhance diagnosis rates, reduce the burden of recurrence, and optimize access to innovative treatments. Central to these recommendations is the integration of early cancer care into national cancer control plans, including robust data collection and monitoring, as well as improvements in health literacy. A key factor is the use of early clinical endpoints that measure key outcomes in addition to overall survival, providing timely insights into treatment effectiveness that can guide early regulatory and reimbursement decisions prior to reaching overall survival maturity. This paper is a call to action for relevant stakeholders to take a coordinated approach that optimizes outcomes for cancer patients by promoting early detection and treatment.