Journal of Medical Economics最新文献

Aim: Although liver biopsy is considered the most reliable diagnostic tool for metabolic dysfunction-associated steatohepatitis (MASH), it is invasive and can be costly. Clinicians are increasingly relying on routine biomarkers and other noninvasive tests (NITs) for diagnosis. We examined real-world diagnostic pathways for patients newly diagnosed with MASH with a primary focus on NITs.

Materials and methods: This retrospective, observational study analyzed healthcare claims data (Merative MarketScan Commercial and Medicare Databases) from patients in the United States newly diagnosed with MASH from October 1, 2016, to March 31, 2023. Patients ≥18 years old with ≥12 months of continuous enrollment with medical and pharmacy benefits prior to diagnosis were included. Diagnostic pathways leading up to MASH diagnosis, including NITs (blood-based and imaging-based tests) and liver biopsies were assessed. Prevalence of comorbid conditions, MASH-associated medication use, and the diagnosing physician specialty were also examined.

Results: A total of 18,396 patients were included in the analysis. Routine laboratory tests (alanine aminotransferase [ALT], albumin, aspartate aminotransferase [AST], cholesterol, complete blood count, and hemoglobin A1c) were performed among ≥70% of patients prior to MASH diagnosis, including 89% of patients with a liver enzyme test (ALT and/or AST). More than 75% of patients had necessary laboratory tests to calculate AST to platelet ratio index (APRI) and fibrosis-4 index (FIB-4) scores. The most common imaging performed was ultrasound (62%); liver biopsy was only performed in 10% of patients. There was a high prevalence of cardio metabolic risk factors such as hyperlipidemia (66%), hypertension (62%), obesity (58%), type 2 diabetes (40%), and cardiovascular disease (21%). Nearly half of the patients (49%) were diagnosed by a primary care physician.

Limitations and conclusions: This study highlights real-world diagnostic pathways among patients newly diagnosed with MASH, supporting previous findings that liver biopsies are infrequently used in favor of noninvasive methods.

Objective: This study aimed to investigate the willingness to pay (WTP) of the Japanese population for the transmission prevention function of SARS-CoV-2 antiviral treatments and identify the attributes associated with higher WTP.

Methods: A web-based survey (registration number: UMIN000054955) was conducted from May 17 to June 1, 2024, targeting a general population using a survey company panel. We aimed to obtain around 3,000 valid responses. Respondents were randomly divided into two groups: one assuming a COVID-19 infection (infection-assumed group) and the other without this assumption (non-infection-assumed group). WTP was assessed using an open-ended question format, asking how much they would be willing to pay out-of-pocket for a hypothetical antiviral drug that reduces the risk of transmitting COVID-19 to others by half. The survey also collected demographic information, COVID-19 related attributes, empathy levels using the Multidimensional Empathy Scale (MES), and health literacy using the Communicative and Critical Health Literacy scale. The mean WTP for COVID-19 treatment was calculated for all respondents and for the infection-assumed and non-infection-assumed groups. Subgroup analyses examined the effects of respondent attributes on WTP. A linear regression model with stepwise selection identified factors associated with WTP.

Results: Responses were obtained from 3,657 individuals, with 3,131 valid responses analyzed. The mean WTP among all respondents was JPY 3,205 (USD 20.85) (standard error: JPY 84 [USD 0.55]). The infection-assumed group showed a 21% higher WTP than the non-infection-assumed group (p < 0.001). Subgroup analyses indicated that WTP varied based on attributes such as co-residing children, occupation, empathy levels, and health literacy. Higher WTP was significantly associated with being aged 65 years and older, higher household income, absence of co-residing children, being a company employee, executive, or public servant, fear of COVID-19 infection, higher other-oriented emotional reactivity (a factor of MES), and higher health literacy.

Conclusion: We presented the WTP of the Japanese population for the transmission prevention function of COVID-19 treatments as an actual monetary value. Factors such as empathy, health literacy, and some attributes were significantly associated with WTP. These findings might help inform policymakers in developing health policies based on the universal health insurance system in Japan.

Aims: The cardioprotective effects of semaglutide 2.4 mg reported in the SELECT cardiovascular (CV) outcomes trial (ClinicalTrials.gov NCT03574597) provide clinical benefit for subjects with overweight or obesity and established CV disease without type 2 diabetes (T2D). We assessed cost-effectiveness of semaglutide 2.4 mg in this population against the American College of Cardiology/American Heart Association value framework.

Materials and methods: A cohort-level Markov-state cost-effectiveness model using trial-derived data with outcomes from a healthcare sector perspective measured over a lifetime horizon was developed. Treatment costs were based on US list prices; scenario analyses used literature-reported estimated rebates. Healthcare costs and benefits were discounted at 3.0%. A simulated cohort of 100,000 subjects was aligned to the SELECT trial population baseline characteristics and time-on-treatment. Subjects received either semaglutide 2.4 mg or placebo in addition to standard of care (SoC). Modelled outcomes included clinical events (CV events, progression to T2D, chronic kidney disease [CKD]) and health economic measures, including direct costs and quality-adjusted life years (QALYs).

Results: Mean semaglutide 2.4 mg treatment duration was 2.79 years. Per 100,000 subjects, treatment avoided 2,791 non-fatal myocardial infarctions, 3,000 coronary revascularizations, 487 non-fatal strokes, and 115 CV deaths over the modeled lifetime horizon. Average per-subject lifetime treatment costs were $47,353; savings arose from avoided T2D ($14,431), CKD ($2,074), and CV events ($1,512). Semaglutide 2.4 mg was associated with increased lifetime costs ($29,767), additional QALYs gained (0.218) and an incremental cost-effectiveness ratio of $136,271/QALY at list price; a scenario using an empirically estimated 48% rebate predicted $32,219/QALY.

Limitations: The generalizability of observations from SELECT to a broader US population is unknown. Our model does not capture all outcomes nor costs that may be affected by weight loss. Modeling assumptions may present limitations.

Conclusions: Semaglutide 2.4 mg use as in SELECT is cost-effective at list price, using a $150,000/QALY willingness-to-pay threshold.

Background: Pulsed field ablation (PFA) has emerged as an effective technology in the treatment of paroxysmal atrial fibrillation (AF).

Objective: To evaluate the cost-effectiveness of PFA vs. thermal ablation from a US healthcare payer perspective using data from a randomized trial.

Methods: A hybrid decision tree and Markov model was developed comparing patients receiving PFA to thermal ablation (either radiofrequency or cryoballoon ablation) from a US healthcare payer perspective at 5-, 10-, 20-, and 40-year time horizons. Direct medical costs (in 2024 US Dollars), quality-adjusted life years (QALYs), and the net monetary benefit were evaluated at a willingness-to-pay (WTP) threshold of $100,000/QALY. Univariate and probabilistic sensitivity analyses were performed to test model uncertainty. The budget impact for a standard US healthcare payer with 1 million beneficiaries was also assessed.

Results: Over a 40-year time horizon, PFA resulted in an additional 0.044 QALYs at a lower cost of $2,871 compared to thermal ablation. PFA was cost-effective in 54.9% of simulations. Anticoagulation and ablation procedure costs had the largest impact on model uncertainty. The expected cost savings per member per month for a US healthcare payer adopting PFA were $0.00015, $0.0059, and $0.02343 in years 1, 4, and 6, respectively.

Conclusions: PFA was at least as cost-effective as conventional thermal ablation modalities for treatment of paroxysmal AF and potentially reduces US healthcare payer costs. Providers and payers should consider designating PFA among the preferred first-line therapies for eligible patients.

Aims: Catheter-based radiofrequency renal denervation (RF RDN) is an interventional treatment for uncontrolled hypertension. This analysis explored the therapy's lifetime cost-effectiveness in a Canadian healthcare setting.

Materials and methods: A decision-analytic Markov model was used to project health events, costs, and quality-adjusted life years over a lifetime horizon. Seven primary health states were modeled, including hypertension alone, stroke, myocardial infarction (MI), other symptomatic coronary artery disease, heart failure (HF), end-stage renal disease (ESRD), and death. Multivariate risk equations and a meta-regression of hypertension trials informed transition probabilities. Contemporary clinical evidence from the SPYRAL HTN-ON MED trial informed the base case treatment effect (-4.9 mmHg change in office systolic blood pressure (oSBP) observed vs. sham control). Costs were sourced from published literature. A 1.5% discount rate was applied to costs and effects, and the resulting incremental cost-effectiveness ratio (ICER) was evaluated against a willingness-to-pay threshold of $50,000 per QALY gained. Extensive scenario and sensitivity analyses were performed.

Results: Over 10 years, RF RDN resulted in relative risk reduction in clinical events (0.80 for stroke, 0.88 for MI, and 0.72 for HF). Under the base case assumptions, RF RDN was found to add 0.51 (15.81 vs. 15.30) QALYs at an incremental cost of $6,031 ($73,971 vs. $67,040) over a lifetime, resulting in an ICER of $11,809 per QALY gained. Cost-effectiveness findings were found robust in sensitivity analyses, with the 95% confidence interval for the ICER based on 10,000 simulations ranging from $4,489 to $22,587 per QALY gained.

Limitations and conclusion: Model projections suggest RF RDN, under assumed maintained treatment effect, is a cost-effective treatment strategy for uncontrolled hypertension in the Canadian healthcare system based on meaningful reductions in clinical events.

Introduction: This study analyzed the health and economic impact of the 21-valent pneumococcal conjugate vaccine (V116) and the 20-valent pneumococcal conjugate vaccine (PCV20), as well as their relative cost-effectiveness, in Japanese adults aged 65 years using a delta pricing approach.

Methods: A Markov model was employed to simulate the movement of the Japanese population among four health states: healthy, pneumococcal disease (consisting of invasive pneumococcal disease [IPD] with or without meningitis and non-bacteremic pneumococcal pneumonia [NBPP]), post-meningitis sequelae, and death. The model was populated with publicly available demographic and epidemiologic data, stratified by risk level. Pneumococcal serotype distribution and vaccine effectiveness, as well as direct and indirect treatment costs and health-related utilities, were derived from published sources. The model used a lifetime horizon and 2% discounting of costs and life-years. Costs were adjusted to 2023 values in Japanese yen (¥). Outcomes were cases and deaths, life-years and quality-adjusted life-years (QALYs), vaccination and treatment costs, and incremental cost-effectiveness ratios. The range over which V116 was cost-saving and cost-effective was determined.

Results: Compared to PCV20, V116 averted an additional 28 cases of IPD, 918 cases of NBPP, 5 deaths from IPD, and 51 deaths from NBPP over the lifetime of a single age 65 cohort. Life-years and QALYs gained were 1,019 and 642, respectively, relative to PCV20; V116 saved ¥733 million in direct medical costs and ¥557 million in indirect costs, compared to PCV20. V116 was found to be cost-saving at price premiums up to ¥1,322 (payer perspective) or ¥2,327 (societal perspective) and remained below a willingness-to-pay threshold of ¥5 million/QALY for premiums up to ¥7,113 (payer perspective) or ¥8,117 (societal perspective).

Conclusions: V116 is projected to provide more population health benefits in Japan than PCV20, and to be cost-effective at a variety of price premiums.

Objectives: Health-related quality of life (HRQoL) studies in patients with advanced non-small-cell lung cancer (NSCLC) according to KRAS mutational status are limited. This study aimed to report real-world evidence on HRQoL outcomes based on KRAS mutational status in patients with advanced NSCLC tumors receiving second-line or later (2L+) treatment in France and Germany.

Methods: In this real-world, non-interventional, cross-sectional, multicenter, patient-reported outcome (PRO) study conducted in France (15 contributing sites) and Germany (8 contributing sites), physicians enrolled adult patients with locally advanced and unresectable or metastatic NSCLC with known KRAS mutation status (KRAS G12C, KRAS non-G12C, or KRAS wildtype [WT]), who received a 2L + treatment. Study outcomes included sociodemographic characteristics; HRQoL evaluations based on EORTC Global Health Status QoL scores (QLQ-C30) and EQ-5D-5L scores. Data were analyzed descriptively.

Results: Of 156 enrolled patients, data from 149 patients were included in the final analysis (France, n = 103; Germany, n = 46). Median (quartile [Q]1, Q3) age was 67.0 (61.0, 71.0) years; 56.4% of patients were male. In total, 38.9% (n = 58), 26.2% (n = 39), and 34.9% (n = 52) of patients had tumors with KRAS G12C mutation, KRAS non-G12C mutation and WT KRAS, respectively. Mean (±SD) QLQ-C30 Global Health Status QoL scores were 56.99 (20.30) for the overall population, and 56.03 (22.55), 58.97 (18.67) and 56.57 (19.05) for KRAS G12C, non-G12C, and WT subpopulations. In the overall population, moderate-to-extreme problems were reported in all EQ-5D-5L dimensions (range: overall population, 15.5%-39.6%; KRAS G12C, 15.6%-46.6%; non-G12C, 7.8%-23.1%; WT, 21.1%-44.2%).

Conclusion: HRQoL was broadly similar across KRAS G12C, non-G12C, and WT subpopulations.

Background: Research is needed to understand the impact of mental health disorders (MHD) on healthcare resource utilization (HCRU) and costs among people with human immunodeficiency virus (PWH).

Objectives: Examine the HCRU and cost burden among treatment-naïve PWH with and without MHD initiating single tablet antiretroviral regimens (STRs) and multi-tablet regimens (MTRs).

Methods: A retrospective database analysis of the US Medicaid population from Anlitiks' All Payor Claims database between 1 January 2016 and 30 June 2023 was conducted. Treatment-naïve MTR-initiators vs STR-initiators (the index was the first prescription fill claim date) with ≥ 12-months pre- and post-index continuous enrollment and no pre-index HIV-2 diagnosis among PWH/MHD and PWH/no-MHD during 1 January 2017-30 June 2022 were selected. Demographics, clinical characteristics, HCRU and costs between MTR-initiators vs STR-initiators among PWH/MHD and PWH/no-MHD were described using Chi-square tests and Wilcoxon rank-sum or t-tests for categorical and continuous variables, as appropriate. HCRU and costs were examined using multivariable logistic and gamma-log link regression models, controlling for potential confounders.

Results: MTR-initiators (PWH/MHD: n = 7,874, PWH/no-MHD: n = 3,612) vs. STR-initiators (PWH/MHD: 46,024, PWH/no-MHD: 23,452) were significantly younger (PWH/MHD: 43.6 vs. 47.2 years; PWH/no-MHD: 39.2 vs. 43.3 years) and more likely to be female (PWH/MHD: 46.4% vs. 35.7%; PWH/no-MHD: 42.3% vs 29.7%) in both groups (all p-values < 0.05). MTR-initiators vs. STR-initiators had significantly higher rates of inpatient (IP) hospitalizations (PWH/MHD: 28.9% vs. 27.1%; PWH/no-MHD:13.9% vs. 11.9%) and emergency department (ED) visits (PWH/MHD: 53.3% vs. 49.2%; PWH/no-MHD: 35.2% vs. 31.8%) among both those with and without MHD (all p-values < 0.05). MTR-initiators vs. STR-initiators also had significantly higher adjusted all-cause medical costs (PWH/MHD: $60,228 vs $40,634; PWH/no-MHD: $33,623 vs. $17,996) (all p-values < 0.05).

Conclusions: Among PWH/MHD and PWH/no-MHD, MTR-initiators experienced significantly higher HCRU, and 1.5 times greater costs compared to STR-initiators. In both MTR and STR-initiator groups, the PWH/MHD cohort consistently demonstrated a greater HCRU and cost burden than the PWH/no-MHD.

Aim: The objective of this study was to assess the burden of hospital-acquired venous thromboembolism (VTE) on healthcare systems and patients across ten countries.

Methods: A multi-methodological approach was taken to estimate the burden of hospital-acquired VTE across five key clinical specialties and ten countries (Australia, Brazil, China, France, Mexico, South Korea, Spain, Taiwan, Thailand, and the United Kingdom). Surveys with healthcare professionals (surgeons, hematologists, and hospital management) were conducted to identify clinical specialties of interest. A systematic literature review and interviews were conducted to identify data for incidences and costs. A health-economic model was developed, using a decision tree and Markov model to estimate 1-year costs. Costs are presented in 2022 USD.

Results: Orthopedics, oncology, long-term ICU, cardiology, and obstetrics and gynecology were identified as the clinical specialties of interest. The total cost burden of hospital-acquired VTE was estimated to be $41,280 million, which equals $503 per patient at risk. Expressed as a share of 2022 GDP, an average spending per country of 0.05% to 0.18% was observed. The VTE-associated mortality was substantial, accounting for 150,081 deaths in a 74.2 million population, translating into an average mortality rate of 2.02 (0.64-3.05) per 1,000 patients at risk.

Limitations: There were limited data available concerning VTE incidences in some countries and clinical specialties. Where data were available, there was heterogeneity of incidence definitions across the identified studies. Generalizations, imputations, and the country-agnostic structure of the model might have contributed to biases.

Conclusions: The burden of hospital-acquired VTE is substantial both from an economic and from a patient perspective in all countries evaluated.

Aims: Direct-acting oral anticoagulants (DOACs) have emerged as the preferred treatment for nonvalvular atrial fibrillation (NVAF). However, evidence concerning the economic outcomes of DOAC switching remains limited. This study aimed to assess the economic outcomes of DOAC switching in the US and Germany, two countries with a high AF prevalence and DOAC utilization.

Methods: A decision model was developed to assess the incidence and cost of stroke/systemic embolism (SE) and major bleeding (MB) associated with switching from apixaban to rivaroxaban in patients with NVAF. The model compared two scenarios: continuers (patients continuing apixaban) and switchers (patients switching from apixaban to rivaroxaban). Model inputs on clinical event rates were sourced from a published real-world study, cost inputs were from a standard costing database and published literature. The analysis was conducted over a 1-year time horizon from US Medicare fee-for-service and German public healthcare payer perspectives.

Results: Over one year, 47,036 patients among a hypothetical plan size of 1,000,000 US Medicare fee-for-service members and 1,019,079 patients among the German adult population size of 70,107,122 were estimated to be treated for NVAF with apixaban. Switching all patients from apixaban to rivaroxaban resulted in 1,498 and 32,447 additional clinical events (stroke/SE and MB) and deaths in the US and Germany, respectively, compared to continuing with apixaban. This led to a total incremental cost of $17.3 million and €153 million from Medicare fee-for-service and German public healthcare perspectives, respectively.

Limitations: The incidence and hazard ratios of clinical events informing this analysis were based on a US commercial and Medicare Advantage population and may not be generalizable to other populations.

Conclusions: Switching from apixaban to rivaroxaban was associated with increased clinical events, deaths, and higher medical care costs, potentially representing a less favorable strategy economically compared to continuing apixaban among patients with NVAF.