ClinicoEconomics and Outcomes Research最新文献

Background: Reverse transcriptase-polymerase chain reaction (RT-PCR) testing has been central to Indonesia's COVID-19 response. However, limited empirical evidence exists on the actual cost of RT-PCR testing in public laboratories, raising concerns that regulated tariffs may not accurately reflect resource use, particularly in low- and middle-income settings.

Purpose: This study aimed to estimate the unit cost of COVID-19 RT-PCR testing and identify key cost drivers to inform evidence-based tariff setting and support the financial sustainability of public laboratories in Indonesia.

Methods: A micro-costing analysis using an Activity-Based Costing (ABC) approach was conducted across eight government-owned laboratories in Southeast Sulawesi, Indonesia. Costs were categorized into direct and indirect components, and the unit cost was calculated by dividing total operational costs by the average number of tests performed per month. One-way sensitivity analyses were performed to assess the influence of major cost components.

Results: The adjusted mean unit cost of RT-PCR testing was USD 21.02, based on an average testing volume of 5409 samples per month and total adjusted monthly operating expenditures of USD 100,973.89. Direct costs accounted for 92.58% of total costs, driven primarily by consumables (75.12%) and human resources (13.32%). Extraction kits and RT-PCR reagents were the most influential cost drivers in sensitivity analyses. The estimated unit cost exceeded the government-mandated RT-PCR tariff outside Java and Bali (approximately USD 17-18.5).

Conclusion: This study provides empirical evidence on the actual cost of COVID-19 RT-PCR testing in Indonesian public laboratories. The findings highlight a mismatch between regulated tariffs and real service delivery costs, underscoring the need for tariff adjustments that balance affordability for patients with financial sustainability for laboratories. Transparent cost information is essential to support evidence-based pricing policies and strengthen preparedness for future public health emergencies.

Importance: Most pancreatic cancer (PC) cases are diagnosed at a late stage, leading to poor prognosis. New-onset diabetes (NOD) increases the risk of PC by 6- to 8-fold within the first 3 years of diagnosis. This study aims to assess the clinical utility and economic benefits of early detection of PC in patients with NOD using a cell-free DNA (cfDNA) epigenomic blood test (Avantect; ClearNote Health, CA).

Design: A Markov model compares two primary strategies: no testing and testing higher-risk NOD patients using the cfDNA epigenomic test. Using the enriching new-onset diabetes for pancreatic cancer (END-PAC) criteria ≥3, approximately 20% of the NOD patients were at higher risk for PC. The cfDNA epigenomic test has a sensitivity of 68% and a specificity of 97%.

Results: The cfDNA test is robustly cost-effective in NOD high-risk patients, with an Incremental Cost-Effectiveness Ratio (ICER) of $56,564 at a Willingness-to-Pay (WTP) threshold of $100,000; its cost-effectiveness is superior to the standard of care, ie, no testing. In a modeled cohort of 10,000 NOD patients, the no-testing strategy would result in 1% of PC cases, with only 7.1% being eligible for potentially curative surgery. By contrast, cfDNA testing identified 71 cases of PC, with 32.4% of these cases being eligible for surgical resection. Early-stage detection through this approach more than quadruples surgical eligibility and significantly enhances the chances of a cure.

Conclusions and relevance: These findings underscore the transformative clinical and economic value of early PC detection in high-risk patients with NOD. Implementing cfDNA testing during the first three years following diabetes onset has the potential to shift the diagnosis paradigm, leading to early interventions and meaningful improvements in expected survival, while remaining cost-effective.

Purpose: To examine willingness to pay (WTP) for acne treatment and its associated factors, to assess the relationship between WTP and dermatology-specific quality of life as measured by the Dermatology Life Quality Index (DLQI), and to develop and internally validate a nomogram for predicting high WTP, with the aim of supporting individualized acne management and informed healthcare resource allocation.

Methods: Patients with acne completed an electronic questionnaire assessing their WTP for a hypothetical one-time curative treatment, along with demographic, clinical, and DLQI data. High WTP was defined as >4000 CNY. Multivariable logistic regression identified factors associated with high WTP, and restricted cubic spline analysis explored the relationship between WTP and DLQI scores. The dataset was randomly divided into training (70%) and testing (30%) cohorts. LASSO regression was used to identify key predictors of WTP. The variables retained by LASSO were subsequently entered into a multivariate logistic regression prediction model, and the independent predictors were incorporated into a nomogram to estimate the probability of high WTP for each patient.

Results: A total of 456 patients were included. Multivariate logistic regression analysis revealed that female sex, aged ≥25 years, monthly income ≥5000 CNY, moderate or severe acne, prior treatment history (medications, chemical peels, or combination therapies), recurrent acne, and annual acne-related expenditure >1000 CNY were significantly associated with high WTP. DLQI showed a linear relationship with WTP (P_overall = 0.04, P _nonlinear = 0.77). Eight independent predictors identified from multivariable analyses were incorporated into the predictive nomogram, which showed good calibration and discrimination (AUC_train = 0.80; AUC_test = 0.77). Decision-curve analysis indicated clinical utility across threshold probabilities between 0.19 and 0.94.

Conclusion: Patients' WTP for acne treatment reflects disease burden and quality-of-life impairment. A nomogram developed and validated in this study provides an effective tool for estimating WTP in acne patients and may support clinical decision-making and health-economic assessment.

Purpose: Real-world data on healthcare costs associated with SGLT2 inhibitor use during its early adoption remains limited, particularly across heart failure (HF) phenotypes. This study assessed trends in healthcare resource utilization (HCRU) and costs among HF patients, stratified by SGLT2 inhibitor use and left ventricular ejection fraction (LVEF).

Patients and methods: Using Finnish specialty care registry data, adults with a first HF diagnosis between January 1, 2016, and June 30, 2022 were identified and categorized by LVEF as reduced (HFrEF), mildly reduced (HFmrEF), and preserved (HFpEF) LVEF. Annual SGLT2 inhibitor uptake (2016-2022) was assessed. Patients were stratified by SGLT2 inhibitor use within 365 days of HF diagnosis to assess HCRU and costs trends (2016-2021).

Results: Among 119,314 patients, 7,626 (6.4%) initiated SGLT2 inhibitors within a year. Among those with LVEF data (n=16,312/119,314 [13.7%]), HFpEF predominated (58%). SGLT2 inhibitor use in HFrEF increased from 14.4% (2020) to 50.1% (2022). Patients receiving SGLT2 inhibitors were younger (70.8 vs 78.6 years), more often male (65.0% vs 47.2%), and had higher prevalence of type 2 diabetes (72.4% vs 28.5%) compared with those without SGLT2 inhibitor use. From 2016 to 2021, inpatient admissions declined modestly across all groups, with consistently shorter stays among patients with SGLT2 inhibitors compared with those not using them (mean: 20.0 vs 26.3 days [2016]; 17.2 vs 21.7 days [2021]). Outpatient visits and drug dispensations were higher in the SGLT2 inhibitor group. Total annual HCRU costs declined over time, remaining lower for SGLT2 inhibitor users (€30,742 vs €34,235 in 2021), driven by reduced inpatient admission costs.

Conclusion: SGLT2 inhibitor uptake increased notably following regulatory approvals. Patients who received SGLT2 inhibitors had lower healthcare costs driven by reduced hospitalization costs, suggesting economic benefits of early SGLT2 inhibitor initiation in HF management and supporting further research evaluation of long-term cost-effectiveness.

Introduction: Elranatamab, a B-cell maturation antigen-CD3 bispecific antibody, was approved in Japan in March 2024 for the treatment of relapsed or refractory multiple myeloma (MM). However, real-world (RW) data on its use remain limited. This retrospective study evaluated patient characteristics and elranatamab treatment patterns in patients with MM in Japan using the Medical Data Vision (MDV) database.

Methods: This EVEREST study used de-identified claims data from the MDV database for adult patients with MM who initiated treatment with elranatamab between March 26, 2024, and March 31, 2025. Follow-up was divided, based on expected dosing schedules, into a step-up dosing (SUD) period, a weekly (QW) maintenance period (MP1), an every 2 weeks (Q2W) maintenance period (MP2), and an overall follow-up period. Treatment patterns were reported descriptively. Estimated annual vial usage was extrapolated.

Results: Patients (N=253) with a median age of 74 years (range 41-93 years) and median treatment duration of 60.0 days (interquartile range [IQR], 26.0-136.0) were included. The mean (median [IQR]) time between elranatamab administrations across dosing periods were SUD 4.0 days (4.0 [3-4]), MP1 9.8 days (7.0 [7-9]), and MP2 13.0 days (14.0 [7-14]), indicating less frequent dosing than recommended in MP1, and comparable dosing in MP2. Nearly all administrations were in inpatient settings during SUD, and most were in outpatient settings during maintenance periods. Despite variable dosing during the maintenance periods, annual elranatamab usage was lower than the approved label recommendations for Japan (34.4 vs 39.0 vials per year).

Conclusion: Early RW data on elranatamab administration and vial usage in patients with MM in Japan suggest that dosing is generally aligned with label expectations, with variable dosing during QW and Q2W maintenance for some patients. Projected vial usage in this RW setting was lower than the expected usage per label.

Background: Chemotherapy-induced alopecia is a common side effect with psychological impacts that affect quality of life. Up to 14% of patients may decline chemotherapy due to concerns over hair loss. While existing scalp cooling therapies can reduce alopecia, constraints including space, staffing, and extended chair time limit their use in health services. Hence, a novel scalp cooling cap ("Product X") was developed to address this gap. Product X is cordless and portable and does not require patients to remain in treatment chairs post-chemotherapy. This early-stage cost-effectiveness analysis addresses the potential economic value of adopting Product X versus current practice (no scalp cooling).

Methods: We developed a decision tree and a lifetime Markov model to estimate change to total costs and health benefits for female patients with early breast cancer, from a health system perspective in a Singapore tertiary cancer care setting. The model incorporated costs related to scalp cooling equipment and administration, chemotherapy, and treatment for cancer recurrence. Assumptions regarding Product X's efficacy and its potential impact on chemotherapy compliance were tested in nine scenario analyses (efficacy: 50%, 75%, 100%; compliance improvement: 0, 1%, 5%). Probabilistic scenario analysis was conducted using Monte Carlo simulation with 1000 iterations from appropriate parameter distributions.

Results: In the base case (1% improvement in compliance and 100% efficacy), Product X yielded an incremental cost of S$265 per patient (95% UI: S$251-S$281) and incremental quality-adjusted life years (QALY) of 0.0717 (95% UI: 0.0705-0.0729). At a willingness-to-pay of S$45,000 per QALY, incremental net monetary benefits (INMB) was S$2961 (95% UI: S$2906-S$3015), with >99.9% probability of cost-effectiveness. Across all scenarios, INMB ranged from S$1158 to S$3330.

Conclusion: Scenario and probabilistic analyses suggest that Product X is a cost-effective solution for chemotherapy-induced alopecia, supporting its adoption from a health system perspective.

Purpose: Eosinophilic esophagitis (EoE) is a chronic immune-mediated disease requiring long-term management. Loss to follow-up (LTFU) is a significant issue, leading to increased complications and higher healthcare costs. One key factor contributing to LTFU is the lack of structured transition models for patients moving from pediatric to adult care. This study provides the first model-based economic evaluation of LTFU in EoE within the Italian National Healthcare Service (NHS) and aims to quantify the economic impact of LTFU as well as evaluate the potential cost reduction associated with implementing a structured transition model.

Patients and methods: A health economic model was developed to assess the financial burden of LTFU in EoE from the perspective of the Italian NHS. The model incorporated epidemiological, clinical, and economic inputs, estimating, within a time-horizon of one year, the economic burden of LTFU and the cost differential between patients with continuous care and those experiencing a care gap (≥2 years). The analysis included costs related to emergency department (ED) visits, hospitalizations, pharmacological treatments, and outpatient services. All costs were reported in 2024 euros. Additionally, a simulation was conducted to evaluate the potential economic benefits of a structured transition model.

Results: The total economic burden of LTFU in EoE was estimated at € 84.9 million, with an average cost per patient of € 15,468, nearly double the cost of patients receiving continuous care (€ 7,744). The primary cost drivers were hospitalizations (69%) and pharmacological treatments (30%). The introduction of a transition model reducing LTFU by 30% could result in a € 25.4 million cost reduction, primarily through decreased hospital admissions and optimized treatment strategies.

Conclusion: LTFU in EoE is associated with a significant economic burden. Implementing a structured transition model could improve patient retention, enhance adherence to treatment, and generate important cost savings.

Purpose: This study aimed to assess neoadjuvant (neo), post-neo, and adjuvant (adj) treatment (tx) patterns, recurrence rates, and the impact of recurrence timing on the cumulative cost burden among HER2+ early breast cancer (eBC) patients.

Methods: Merative™ MarketScan^® Databases were used to identify adults newly diagnosed with eBC between 1/1/2017-9/30/2022 with ≥1 HER2 targeted treatment following BC date. Surgery within a year of the BC date delineated neo and post-neo/adj periods before and after the surgery date. Recurrence was reported during the post-surgery period and was defined as evidence of additional chemotherapy treatment, metastasis, or end-of-life care. Generalized linear model (GLM) (gamma distribution and log link) was used to assess the impact of disease recurrence on cumulative 3-year total all-cause costs during the post-surgery period.

Results: A total of 3745 patients with HER2+ eBC were included in the study (mean age 53.7 yrs): 57.4% (n=2151) with adj tx only, 40.2% (n=1504) with neo and post-neo tx, 1.9% (n=70) with surgery only, and 0.5% (n=20) neo tx only. During follow-up (median duration post-surgery: 2 years), the rate of first recurrence was highest for surgery only (70.0%) and similar for adj only (16.0%) and neo and post-neo tx (14.3%) cohorts. GLM showed that the cumulative cost burden following surgery was higher among patients who experienced the first recurrence in <12 months vs no recurrence ($348,834 vs $265,279). Patients with chemo only as adj tx had a higher cumulative cost burden (Risk Ratio [RR] 1.28; p <0.001) than those with HER2 targeted treatment; and patients with neo tx had a lower cost burden (RR 0.85, p <0.001) compared with those with no neo tx.

Conclusion: Delays in recurrence were associated with lower cumulative cost burden. Study findings highlight that the appropriate use of more effective HER2 targeted treatments that delay the time of first recurrence in neoadjuvant and adjuvant settings may improve patient outcomes and reduce the long-term healthcare burden associated with BC.

Purpose: US Medicare beneficiaries in fee-for-service (FFS) and Medicare Advantage (MA) plans may differ in sociodemographics, treatment patterns, and healthcare costs, but real-world evidence is limited. Our objective was to gain a holistic understanding of Medicare beneficiaries using long-acting injectable antipsychotic medications (LAI-AM) as a case study.

Patients and methods: This observational study describes two distinct cohorts of new LAI-AM users enrolled in FFS or MA plans from July-2017 through December-2020. Data were extracted from the Centers for Medicare & Medicaid Services' Chronic Conditions Data Warehouse (for FFS) and the Carelon Research Healthcare Integrated Research Database^® (for MA). Setting the first LAI-AM claim as index date, we identified adult beneficiaries enrolled for ≥12-months before (baseline) and after (follow-up) the index date. We described LAI-AM users' sociodemographic and clinical characteristics, medication adherence (proportion of days covered [PDC]), and healthcare costs.

Results: We identified 3180 FFS- and 525 MA-enrolled LAI-AM initiators. Compared to the MA cohort, the FFS cohort was younger (mean 49 vs 53 years), had more males (56% vs 45%), and had more living in the South and West. Many had a depressive mood disorder (70%) and/or substance use disorder (40%). The mean PDC was 0.52 (SD 0.34) for FFS and 0.46 (SD 0.34) for MA. Only 33% (FFS)/27% (MA) had a PDC≥0.8 (adherent). In both cohorts, total medical costs decreased and total pharmacy costs increased from baseline to follow-up.

Conclusion: Differences in sociodemographic characteristics between FFS and MA plan LAI-AM initiators did not correspond with different patterns or costs of treatment.

Objective: To conduct an economic analysis of the use of combined ESR (Erythrocyte Sedimentation Rate) and CRP (C-Reactive Protein) testing strategies compared to CRP testing alone in U.S hospitals.

Methods: A decision tree model was developed to evaluate the cost-effectiveness and cost-benefit of combined ESR and CRP testing compared to CRP alone. The model estimated the laboratory costs, number of misdiagnoses, and follow-up costs associated with misdiagnoses. Model inputs were sourced from published literature and clinical guidelines. Two combined testing strategies were evaluated: 1) result is positive only if both ESR and CRP results are positive (ESR + CRP) and 2) result is positive if either the ESR or CRP result is positive (ESR/CRP). Strategies were evaluated for five individual and three grouped conditions.

Results: Results demonstrated that the ESR + CRP testing strategy is a cost-effective strategy for reducing misdiagnoses and is expected to result in a net cost reduction to the healthcare system when accounting for the reduction in follow-up costs associated with misdiagnoses. In contrast, the ESR/CRP strategy led to increased misdiagnoses when compared to CRP alone.

Conclusion: Results indicate that adopting the ESR + CRP strategy would reduce misdiagnoses and overall costs to healthcare systems.