Journal of Medical Economics最新文献

Objective: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and debilitating hematological disease with significant economic burden. Despite the availability of multiple therapies, there is a lack of consensus on optimal treatment strategies among physicians and payers in the United States. This study aimed to evaluate the economic value of iptacopan, a novel oral treatment option, compared to terminal complement inhibitors (specifically, complement component C5 inhibitor or C5i)-including eculizumab and ravulizumab-among patients with PNH who are either (i) C5i-experienced or (ii) complement-inhibitor-naïve.

Methods: A cost per responder analysis was conducted based on treatment efficacy from clinical trials comparing iptacopan with C5i treatments. Treatment response was defined as the proportion of patients achieving red blood cell transfusion independence. Treatment costs were estimated as pharmaceutical wholesale acquisition cost and treatment administration costs, accounting for discontinuation. Outcomes evaluated included the number needed to treat to achieve a response and the cost per responder over the treatment duration of 24 weeks.

Results: Over 24 weeks, the number needed to treat to achieve an additional response was lower for iptacopan than all C5i comparators (C5i-experienced: 1.05 with iptacopan vs. 3.86 with C5is; complement-inhibitor-naïve: 1.02 with iptacopan vs. 1.69 with C5is). Cost per responder was lower for iptacopan than C5i comparators for both C5i-experienced ($264,337 for iptacopan vs. $975,298 for ravulizumab, $1,060,511 for eculizumab, and $744,561-$955,194 for eculizumab biosimilar with 10%-30% discount from eculizumab cost) and complement-inhibitor-naïve patients ($256,754, vs. $428,139 for ravulizumab, $465,546 for eculizumab, and $326,849-$419,314 for eculizumab biosimilar).

Conclusion: Among both C5i-experienced and complement-inhibitor-naïve patients, treatment with iptacopan resulted in higher response rates and lower cost per responder compared to C5is.

Objective: Number needed to treat (NNT) and cost per event avoided (CPEA) are measures used to represent the clinical and economic value of chronic treatments and are commonly calculated based on primary endpoints from trials. This approach, although widely used, does not reflect the complete value of a treatment, as it does not consider outcomes beyond the primary endpoints. This research aims to overcome this limitation by developing multiple composite NNTs to derive the CPEA to estimate a total value of semaglutide and dulaglutide in people with established cardiovascular disease.

Methods: Two cardiovascular outcomes trials were selected, SELECT (NCT03574597, semaglutide 2.4 mg) and REWIND (NCT01394952, dulaglutide 1.5 mg). NNT was calculated as NNT = 1/ARR where ARR = control event rate - experimental event rate. NNT was estimated for 3-point major adverse cardiovascular events (MACE-3; primary endpoint of the trials), 5-point MACE (MACE-5), and cardiovascular-kidney-metabolic events (CKM). CPEA of MACE-3, MACE-5, and CKM was calculated as NNT*duration of mean follow-up (semaglutide) or duration of median follow-up (dulaglutide)*estimated net price.

Results: The NNT decreased as the number of outcomes in the composite endpoint increased, where NNT_MACE-3, NNT_MACE-5, and NNT_CKM were 67, 49, and 8 for semaglutide, and 72, 64, and 23 for dulaglutide, respectively. Similarly, the CPEA decreased as the number of outcomes in the composite endpoint increased, where the CPEA for MACE-3, MACE-5 and CKM calculations were $1,662,001, $1,232,417, and $190,387 for semaglutide and $1,884,013, $1,670,366, and $607,886 for dulaglutide.

Conclusions: This research illustrates the limitations of using a NNT focused only on the primary endpoint, as it does not capture the total benefit of the treatment. When considering the value of treatments through NNT or CPEA analyses, a composite endpoint capturing broader benefit should be utilized.

Background and aim: Bruton tyrosine kinase inhibitors (BTKis) are the standard of care for patients with relapsed/refractory (R/R) chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Ibrutinib and acalabrutinib are two commonly used BTKis, though hypertension (HTN) and other cardiovascular medical events of interest (CV MEOIs) can impact treatment tolerability and long-term use. This study compared cardiovascular safety and health care resource use (HCRU) of acalabrutinib versus ibrutinib monotherapy for R/R CLL/SLL in real-world community practice.

Methods: This retrospective comparative study included 270 RR CLL/SLL patients (90 acalabrutinib; 180 ibrutinib) treated from January 2017 through December 2023 across the ONCare Alliance Network, comprising 30 US community hematology practices. Endpoints included new or worsening of existing HTN, development of CV MEOIs, and treatment discontinuation due to adverse events. HCRU metrics consisted of emergency department visits, hospital admissions, length of stay, specialist consultations, and related medical procedures. Propensity score weighted multivariable logistic regression was used for the comparative analysis on the tolerability endpoints.

Results: At a median follow-up of 33 months, patients treated with acalabrutinib had significantly fewer HTN events (OR = 0.27, 95% CI = 0.074-0.98; p = .046) and adverse events leading to treatment discontinuation (OR = 0.39, 95% CI = 0.20-0.73; p = .002) compared to those on ibrutinib. Hospital admission rates for MEOIs were lower in the acalabrutinib group (0.20 vs. 0.24 per patient), with a shorter median length of stay (3.0 vs. 6.0 days). Additionally, acalabrutinib patients had fewer specialist consultations (0.11 vs. 0.22 per patient) and associated medical procedures (0.11 vs. 0.18 per patient).

Conclusions: In R/R CLL/SLL, acalabrutinib demonstrated a more favorable cardiovascular safety profile than ibrutinib, with fewer HTN events and CV MEOIs. This translated into reduced hospital admissions and lower HCRU, supporting acalabrutinib as a potentially better tolerated long-term treatment option in community practice.

Aims: This study evaluated the cost-effectiveness of female-only vaccination (FOV) and gender-neutral vaccination strategies (GNV) using 4-valent (4vHPV) and 9-valent HPV (9vHPV) vaccines among adolescents aged 12-16 years in Japan. It provides the first comprehensive assessment of all feasible vaccine-population combinations using a unified dynamic transmission model.

Materials and methods: An age- and sex-structured dynamic transmission model simulated HPV transmission and disease progression over 100 years, incorporating cervical, vulvar, vaginal, anal, head and neck, and penile cancers. Six vaccination strategies were evaluated: no further vaccination, FOV with 4vHPV, FOV with 9vHPV, GNV with 9vHPV for females and 4vHPV for males, GNV with 4vHPV for both sexes, and GNV with 9vHPV for both sexes. Analyses adopted a healthcare payer perspective, with costs in 2025 Japanese yen, discounted at 3%. Incremental cost-effectiveness ratios (ICERs) were calculated using a willingness-to-pay threshold of ¥5 million per quality-adjusted life year (QALY).

Results: FOV with 4vHPV yielded an ICER of ¥171,725/QALY versus no vaccination, and FOV with 9vHPV yielded ¥1,366,226/QALY versus FOV with 4vHPV - both below the willingness-to-pay threshold. Furthermore, GNV with 9vHPV for both sexes provided the greatest health gains and remained below the threshold (¥3,594,296/QALY) versus FOV with 9vHPV. GNV with 4vHPV and mixed 9vHPV/4vHPV schedules were dominated and excluded.

Limitations: The healthcare payer perspective excludes indirect costs. The model hypothetically includes 9vHPV for boys despite its unavailability for males in Japan.

Conclusions: Both female-only 4vHPV and 9vHPV strategies are cost-effective under Japan's economic standards. Furthermore, GNV with 9vHPV provides greater health benefits and cost-effectiveness.

Background: Slovakia has introduced pneumococcal conjugate vaccines (PCVs) in the national immunization program (NIP) since 2011. Recently licensed for use in the pediatric population in Europe, the 20-valent pneumococcal conjugate vaccine (PCV20) is poised to become a new potential standard of care (SoC) in the pediatric national immunization program (NIP). Therefore, this study aims to assess the cost-effectiveness and the clinical and economic outcomes of the PCV20 compared to PCV13 and PCV15 in Slovakian infants.

Methods: A decision-analytic Markov model was employed to estimate the cost-effectiveness of implementing PCV20 versus two lower-valent PCVs in Slovakian infants aged over 10 years. Health outcomes comprised cases of pneumococcal disease, life years, and quality-adjusted life years (QALY). Economic outcomes included vaccination- and disease-associated costs. Outcomes were evaluated for the entire population to capture full vaccine benefits (indirect effects). Epidemiological inputs were sourced from local surveillance reports or published literature. Direct and indirect vaccine effects (VE) were estimated from vaccine impact, effectiveness, and clinical studies of PCV7 and PCV13. Economic inputs were informed by local studies and 2024 Slovak national reimbursement cost. Comprehensive deterministic sensitivity analysis and scenario analyses were examined. The study assumed a willingness-to-pay threshold (WTP) of €40,377/QALY.

Results: Over 10 years, PCV20 3 + 1 was predicted to prevent over 810 invasive pneumococcal disease (IPD) cases, 885,000 pneumonia cases, 6,600 otitis media (OM) cases, and 16,600 disease-associated deaths compared with PCV13 2 + 1. The disease prevention subsequently resulted in €326.7 million direct medical costs savings, 96,430,859 additional QALYs gain and an ICER of €1,349/QALY. Comparable trends were observed versus PCV15 2 + 1, with PCV20 preventing more pneumococcal disease cases, providing greater additional QALYs, and being cost-effective under the prespecified threshold. Results were consistent across base case and scenario analyses.

Conclusions: Implementing PCV20 into the Slovakian pediatric NIP is projected to yield the most significant clinical and economic benefits compared to the two lower-valent PCVs, due to its more extensive protection against a broader range of pneumococcal serotypes. Despite an additional cost of dose and visit, PCV20 was estimated to be cost-effective at the Slovak WTP threshold versus other SoC options over a 10-year time horizon.

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.

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.

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.