Journal of Medical Economics最新文献

Objective: The US Food and Drug Administration (FDA) recently approved the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), resmetirom, without clarifying the most effective strategy for diagnosing or monitoring response to therapy. Current standards-of-care (SoC) for Veterans Health Administration (VHA) and Medicare patients largely rely on vibration-controlled transient elastography (VCTE) and/or liver biopsy. Multiparametric MRI corrected T1 (cT1) is a cost-effective, noninvasive liver disease assessment (NILDA) tool in MASH. We evaluated the budgetary impact of pathways using cT1 versus SoC (liver biopsy or VCTE) to assign suspected MASH patients to resmetirom and for those assigned treatment, monitor response.

Methods: A model of the VHA and Medicare populations was used to derive a budget impact comparing cT1, VCTE and liver biopsy. Clinical and cost data were taken from publicly available sources and used to estimate the number of patients prescribed resmetirom, the identification of those benefiting from therapy, the budgetary impact of each diagnosis and monitoring strategy and the cost of medication prescribed.

Results: For the VHA, cT1 resulted in the lowest per-patient costs ($7,022 (cT1) vs $7,268 (liver biopsy) vs $28,509 (VCTE)), with results remaining consistent across scenario analyses. In the Medicare population, cT1 also resulted in the lowest per-patient costs ($11,866 (cT1) vs $15,488 (biopsy) vs $27,539 (VCTE)) and these results also remained consistent across scenario analyses.

Conclusion: The use of cT1 to assign and monitor resmetirom treatment improves treatment allocation and reduces health system cost vs VCTE and liver biopsy.

Aims: Nirmatrelvir/ritonavir (NMV/r) is an orally administered antiviral indicated for the outpatient treatment of adult patients with mild-to-moderate COVID-19 at high risk for disease progression to severe illness. We estimated the cost-effectiveness of NMV/r versus best supportive care for 54 patient cohorts, specified according to age, vaccination status and comorbidity burden.

Materials and methods: A previously published and validated cost-effectiveness model was utilized and adapted to the Swedish setting. The model used a short-term decision-tree (1 year) followed by a lifetime 2-state Markov model. The short-term decision-tree captured costs and outcomes associated with the primary infection. Post-acute COVID-19 syndrome was only considered in terms of quality-of-life decrements for one year. Baseline hospitalization and mortality risks were taken from a Swedish, nationwide, uniquely granular, Omicron-era, real-world study. NMV/r effectiveness were taken from an Omicron-era US real-world study. Remaining inputs were informed by previous COVID-19 studies and publicly available Swedish sources.

Results: The incremental cost-effectiveness ratios (ICERs) showed a large variation ranging from almost nine million SEK for some of the youngest cohorts to being dominant (i.e. cost-saving with higher gains in quality-of-life vs standard of care) for twelve elderly cohorts. In general, higher age in combination with non-recent (>180 days) or no vaccination led to lower ICERs. Specifically, NMV/r was cost-effective for all but one patient cohorts at least 70 years old, and for most patient cohorts 60-69 years old.

Limitations: As the COVID-19 landscape changes, symptom burden and baseline risks constantly change. Thus, the cost-effectiveness of NMV/r will change with time. However, the future risks could be related to the risks in the current study, and thus remain useful for decision makers.

Conclusions: This study shows that NMV/r is a cost-effective or even cost-saving treatment option for many patient cohorts, including most elderly and not-recently vaccinated patients with at least some comorbidity burden.

Objective: Chronic kidney disease (CKD) is the leading cause of kidney failure, end-stage kidney disease (ESKD), and cardiovascular (CV) events in patients with type 2 diabetes (T2D). The FIDELIO-DKD trial demonstrated that finerenone lowered the risk of renal and CV events in patients with CKD and T2D, regardless of cardiovascular disease history. This study evaluated the cost-effectiveness of finerenone added to background treatment (finerenone + BT) versus background treatment (BT) alone in patients with CKD and T2D from the perspective of the National Health Service in England and Wales.

Methods: A lifetime Markov model assessed the indicated usage of finerenone for the treatment of stage 3 or 4 CKD with albuminuria associated with T2D in adults, as per the relevant marketing authorization. The model structure considered kidney disease progression and CV risk, with health states encompassing patients' kidney disease stage and CV event profiles, using patient-level data from the FIDELIO-DKD trial. Model outcomes were life years, quality-adjusted life years (QALYs), per-patient costs, incremental costs, and incremental cost-effectiveness ratio (ICER). Sensitivity and scenario analysis were performed, including an analysis exploring the impact of real-world data which suggests more frequent sodium-glucose co-transporter-2 (SGLT2) inhibitor use in the United Kingdom since FIDELIO-DKD.

Results: Patients receiving finerenone experienced kidney and CV benefits, including reduced rates of nonfatal CV events and CV deaths, translating to improvements in survival and quality-adjusted life years (QALYs) of 6.11 and 5.97 per patient for finerenone + BT versus BT, respectively. Total discounted per-patient costs were £48,940 for finerenone + BT and £47,716 for BT alone, resulting in an incremental cost-effectiveness ratio of £8,808 per QALY gained for finerenone + BT versus BT.

Conclusion: Sensitivity and scenario analyses, including more frequent SGLT2 inhibitor use consistent with real-world data, indicate a robust ICER that remains within the bounds of what is typically considered cost-effective.

Aim: Urinary incontinence (UI) is a common condition for adult women impacting over 60% of women with 9.8% experiencing daily symptoms and 32.4% experiencing monthly symptoms. It is associated with significant negative impacts on patients' quality of life, well-being, and social functioning, resulting in substantial healthcare costs to payers. The goal of this study was to analyze 24-month budget impact of treatment of urinary incontinence (UI) in adult women enrolled in a 1-million-member US commercial health plan by comparing clinical practice that includes the use of the Leva Pelvic Health System (CP with Leva) to current clinical practice without Leva (CCP).

Methods: A budget-impact model compared 24-month costs associated with first-line pelvic floor muscle training (PFMT) in women seeking UI treatment in two cohorts: 85% receiving first-line CCP treatment/15% receiving the CP with Leva, compared to all patients treated with CCP. Medical spending per treated patient and per-member-per-month were calculated by summing 24-month UI treatment costs comparing CCP to CP with Leva. The treatment pathway was developed based on published guidelines and literature to obtain estimates of success and complications. Commercial payer costs were estimated by applying a 1.50× multiplier to published Medicare costs based on Congressional Budget Office data for Hospital and Physician Services.

Results: In a 1-million-beneficiary US commercial health plan with 334,191 adult women, 31,438 (9.4%) adult women were treated for UI over a 24-month period. Total estimated 24-month cost per treated patient was $11,267 in the CCP and $10,447 in the CP with Leva groups, respectively. Estimated total health plan 24-month savings was $25,782,112, or $1.07 per-member-per-month.

Limitations: The model may not capture all events in the care pathway for female UI patients seeking medical treatment, as there are significant variations in practice patterns; the rate of Leva adoption as a first-line therapy is based on estimates. The costs and savings calculated in this model may not be generalizable to every commercial health plan, given that actual costs routinely rely on specifically negotiated reimbursement rates.

Conclusions: The model demonstrates that access to first-line Leva therapy can reduce two-year UI treatment costs compared to CCP.

Background: Pimavanserin (PIM) is the only FDA approved atypical antipsychotic treatment (AAP) for hallucinations and delusions associated with Parkinson's disease psychosis (PDP) among patients with or without coexisting dementia; however, Other-AAPs (i.e. quetiapine (QUE), risperidone, olanzapine, aripiprazole) are commonly prescribed off-label. Healthcare resource utilization (HCRU) patterns among patients with PDP and coexisting dementia (PDP+D) who newly initiate PIM versus (vs.) Other-AAPs (i.e. other AAP-mix) or QUE in real-world settings is limited.

Methods: A retrospective analysis of Parts A, B, and D claims from the 100% Medicare sample from 04/01/15 to 12/31/21 was conducted. AAP-naïve patients with PDP+D who initiated ≥12-month continuous monotherapy with PIM vs. Other-AAPs or vs. QUE during 04/01/16-12/31/20 were propensity score matched 1:1 on thirty-one variables (age, sex, race, region and 27 Elixhauser comorbidity characteristics). Adjusted log binomial regressions compared all-cause HCRU [(e.g. inpatient hospitalizations and by hospitalization-type [short-term stays (ST-stays), long-term stays (LT-stays), skilled nursing facility stays (SNF-stays)], and emergency room (ER) visits] risk between cohorts.

Results: Of the 5,932 patients with PDP+D, matched cohorts (n = 1,294 in each) on continuous- monotherapy of PIM vs. Other-AAPs or QUE had similar demographics and comorbidities. Adjusted regression results showed those who initiated PIM vs. Other-AAPs had significantly lower relative risk (RR) of ≥1 all-cause inpatient hospitalizations (RR = 0.88, 95% CI: 0.80-0.97), ST-stays (RR = 0.86, 95% CI: 0.77-0.95), SNF-stays (RR = 0.79, 95% CI: 0.68-0.92), and ER visits (RR = 0.89, 95% CI: 0.84-0.94). PIM vs. QUE also experienced significantly lower RR for ≥1 all-cause IP hospitalizations (RR = 0.88, 95% CI: 0.80-0.96), ST-stays (RR = 0.85, 95% CI: 0.77-0.95), SNF-stays (RR = 0.81, 95% CI: 0.70-0.94), and ER visits (RR = 0.88, 95% CI: 0.83-0.94).

Conclusions: Patients initiating PIM-monotherapy for PDP+D experienced 12% lower all-cause inpatient hospitalizations vs. Other-AAPs or QUE. These results are consistent with prior real-world research in PDP with or without dementia.

Background: Streptococcus pneumoniae represents a significant global public health threat, causing approximately 45 million lower respiratory tract infections and 350,000 deaths annually among children under 5 years of age. Conjugate pneumococcal vaccines (PCVs), such as PCV15 and PCV20, have been developed to mitigate this burden by providing protection against serotypes responsible for the disease. The present analysis aims to evaluate the cost-utility of PCV20 compared to PCV15 as a vaccination strategy for preventing pneumococcal diseases in children in Italy.

Methods and materials: A cost-utility analysis (CUA) was conducted using a static Markov model adapted to the Italian context to simulate the economic and clinical effects of vaccination over a 10-year time horizon. The study adopted the perspective of the Italian National Health Service (NHS), considering only direct healthcare costs. Deterministic and probabilistic sensitivity analyses were performed to explore parameter uncertainty.

Results: The model showed that PCV20 is a dominant strategy compared to PCV15, generating cost savings of €6.45 million and a gain of 101,708 QALYs (quality-adjusted life years). These benefits are attributed to PCV20's broader serotype coverage, which significantly reduces the incidence of invasive and non-invasive pneumococcal diseases. Vaccination with PCV20 offers substantial clinical and economic advantages over PCV15.

Conclusions: The introduction of PCV20 as a vaccination strategy for children in Italy represents a cost-effective and clinically advantageous option. Its implementation can reduce the burden of pneumococcal disease and associated healthcare costs, improving public health outcomes and the economic efficiency of the healthcare system.

Introduction: Obstructive sleep apnea (OSA) is characterized by repeated episodes of airway obstruction during sleep, resulting in intermittent hypoxia, disrupted sleep, and reduced quality of life. Obesity management medications may provide new options for patients with OSA and obesity. Health state utilities will be needed as inputs in economic modeling conducted to inform decision-making about these medications for treatment of OSA. The purpose of this study was to estimate utilities associated with OSA severity based on preferences of individuals with OSA and obesity.

Methods: Participants with OSA and obesity (BMI ≥ 30) in the UK and US valued health state vignettes in time trade-off interviews. Vignettes described four levels of OSA severity defined by apnea-hypopnea index: remission (<5), mild (5-14.9), moderate (15-29.9), and severe (≥30). Participants were instructed to imagine having the option to use non-pharmacologic treatment for OSA when valuing the health states. Utilities were first estimated for OSA with these treatment options. Then, for health states where participants imagined using positive airway pressure or another device, they were asked to value the vignettes a second time without this treatment to estimate utilities associated with symptoms of OSA at each severity level.

Results: A total of 208 participants (UK = 105; US = 103) completed interviews (50.0% male; mean age = 53.6y). Mean (SD) utilities ranged from 0.93 (0.08) for mild OSA to 0.85 (0.15) for severe OSA when considering health states with the option to use non-pharmacologic treatment. When participants imagined living with the symptoms of OSA without the non-pharmacologic treatment option, mean utilities ranged from 0.89 (0.13) for mild OSA to 0.68 (0.27) for severe OSA.

Discussion: Results provide insight into preferences associated with OSA among people with OSA and obesity. The health state utilities estimated in this study may be useful in cost-effectiveness models evaluating treatments for OSA in individuals with obesity.

Aim: Food allergy (FA) imposes a large burden on patients/caregivers, but there is limited recent information on the societal economic burden of FA. We aimed to quantify this burden in the United States (US) using a health economic population cost exercise.

Methods: A prevalence-based model was adopted to estimate the US population with FA and the annual societal/patient costs of FA (in 2024 US$). The Markov model consisted of 3 mutually exclusive health states: "avoidance (with sensitivity)," "full tolerance," and death. Inputs included published data on epidemiology, clinical outcomes, and estimated costs. The model focuses on a comprehensive societal scenario. Scenario analyses utilized a conservative costing approach, determined if the estimated burden exhibited heterogeneity across underserved populations of race/ethnicity and household income, and assessed the impact of allergen desensitization on estimated costs.

Results: The model estimated 16,678,832 people with ≥1 physician-diagnosed FA, an annual total societal cost of $370.8 billion, and an annual cost per patient of $22,234, which was higher for children and adolescents than adults. Societal cost was primarily attributed to indirect costs (92.7% of total cost). In the scenario analysis, conservative total societal cost was $39.6 billion; Hispanic, African American, and other races/multiracial patients had ∼3% increased cost relative to White patients; direct medical cost for the low-income group was 39.1% higher than for the high-income group; and total cost was reduced by ∼4% for each additional 10% of the population that entered the model as desensitized.

Conclusions: Using a population cost exercise that incorporated a range of epidemiologically plausible explanations, we estimated the annual total societal cost of FA in the US may approach $370.8 billion, mostly from indirect costs borne by patients/caregivers during daily living. This assessment may assist population-level healthcare decision-makers in investing in measures that reduce economic burden, valuing interventions, and considering approaches to reduce economic/health disparities for underserved populations.

Objective: Long-acting reversible contraception (LARC) is the most effective form of reversible contraception. Leading organizations agree that LARC should be offered immediately postpartum. The etonogestrel implant is the most effective LARC but carries higher upfront acquisition costs. This study evaluated whether the acquisition costs for the etonogestrel implant are offset via pregnancy prevention and pregnancy-related costs in the year after childbirth.

Methods: A Markov model with 1-year time horizon simulated pregnancy outcomes among 1,000 women 18-49 years initiating 1 of 8 hormonal contraceptives for postpartum contraception: progestin-only oral contraception (OC), combined OC, progestin-only injectable, etonogestrel/ethinyl estradiol vaginal ring, norelgestromin/ethinyl estradiol transdermal patch, 3- or ≥5-year levonorgestrel IUD, and the etonogestrel implant. Time to postpartum initiation for each method and IUD expulsion rates were incorporated. Contraceptive acquisition costs, failure rates, discontinuation rates, pregnancy outcomes and costs, and healthcare resource use were examined from Commercial and Medicaid payor perspectives. Sensitivity analyses were conducted.

Results: The etonogestrel implant followed by IUDs were associated with the fewest short-interval pregnancies (8 [etonogestrel implant], 49 [3-year IUD], and 47 [≥5-year IUD], respectively) and the lowest Commercial (Medicaid) per-woman costs ($1,650 ($1,535) [etonogestrel implant]; $2,364 ($1,979) [3-year IUD]; and $2,519 ($2,145) [≥5-year IUD], respectively). In comparison, short-acting reversible contraception (SARC) resulted in 59-167 pregnancies, and Commercial (Medicaid) per-woman costs ranged from $2,244 ($1,749) to $5,102 ($4,106). Acquisition costs and discontinuation rates were the key drivers impacting overall costs.

Conclusions: The immediate postpartum provision of the etonogestrel implant produced the greatest cost savings due to fewer short-interval pregnancies and related expenditures. These results highlight the etonogestrel implant as a cost-effective option for clinicians seeking to reduce short-interval pregnancy and health system costs.