Journal of Medical Economics最新文献

Introduction: Tirzepatide is a treatment for type 2 diabetes associated with improvements in glycemic control and weight loss, and a low risk of hypoglycemia when not used in combination with insulin or insulin secretagogues. A cost to target analysis of tirzepatide 5, 10 and 15 mg versus subcutaneous semaglutide 1 mg in the UK setting was performed, calculating the annual pharmacy cost per patient treated to six composite endpoints combining glycemic control (glycated hemoglobin [HbA1c] ≤ 6.5% [48 mmol/mol] and <7.0% [53 mmol/mol]) with weight loss (≥5%, ≥10%, ≥15%) and avoidance of hypoglycemia. The costing analysis was based on the tirzepatide costs appraised by NICE as part of TA924.

Methods: The proportions of patients achieving composite treatment targets with tirzepatide and semaglutide (both in combination with metformin) were taken from a post-hoc analysis of the SURPASS-2 clinical trial (N = 1,845). Costs per patient treated to target were calculated by dividing the annual treatment costs associated with each intervention by the proportion of patients achieving the treatment target with each intervention.

Results: Tirzepatide 5, 10 and 15 mg were associated with a lower pharmacy cost per patient achieving treatment target than semaglutide 1 mg for the majority of endpoints evaluated. Differences became greater at more strict treatment targets. For example, the cost per patient achieving HbA1c ≤6.5%, weight loss ≥15%, and no hypoglycemia was GBP 5,650, GBP 8,665 and GBP 9,462 lower with tirzepatide 5, 10 and 15 mg, respectively, compared with semaglutide 1 mg over a 1-year time horizon. The only endpoint where semaglutide 1 mg was associated with a lower cost per patient achieving target was HbA1c <7.0%, weight loss ≥5%, and no hypoglycemia.

Conclusions: Compared to semaglutide, tirzepatide was associated with a lower annual pharmacy cost per patient with diabetes achieving treatment target in the UK for the majority of endpoints, with greater differences at more strict treatment targets.

Aims: A systematic review of the economic burden of advanced non-small-cell lung cancer (NSCLC).

Methods: Articles from 2011 onwards reporting the economic burden of locally advanced (stage IIIB/C)/metastatic (stage IV) NSCLC were identified through systematic and supplementary searches. Outcomes included hospitalizations, emergency department (ED) and outpatient visits, and direct and indirect costs, amongst others.

Results: Across 50 publications (43 studies), patients with advanced NSCLC had high rates of healthcare resource utilization (HCRU), with most reporting hospitalization (ranging from 13.0% to 98.2% of patients), ED visits (2.5% to 83.1%), outpatient visits (74.6% to 100.0%), and diagnostic or monitoring tests (45.9% to 92.0%). HCRU (hospitalizations, ED visits and pharmacy visits) appeared to be lower with immunotherapy as compared to chemotherapy. Brain/central nervous system (CNS) metastases were the major clinical factor influencing HCRU. Mean direct costs ranged from US$5,647 (Brazil) to US$158,908 (US) over 12-24 months, and were generally higher in the US, Korea, Germany, and the UK (vs. Brazil, France, and Italy). The main direct cost drivers were drug-related costs (9.5-76.0% of total), overall outpatient costs (39-70.6%), and inpatient costs (5.0-58.1%). Costs were higher for chemotherapy than for immunotherapy. In China, indirect medical costs were US$1,413 per case. In general, mean total healthcare costs were higher for metastatic disease. Disease severity/diagnosis, presence of brain/CNS metastases, targeted therapy and chemotherapy (vs. immunotherapy) and the presence of comorbidities were the main factors influencing higher costs.

Limitations and conclusions: Patients with advanced NSCLC had high rates of HCRU, and costs were substantial, though varying greatly across countries. HCRU and costs were higher in patients with brain/CNS metastases. Since this was a qualitative review, no formal quantitative synthesis was attempted. Costs reported in different currencies and heterogeneity across studies limited comparability. Finally, a single reviewer extracted data.

Aim: This study evaluates the incremental cost‑effectiveness and budget impact of polyacrylate Superabsorbent (SAP) dressings compared with foam dressings for the management of moderate‑to‑highly exuding leg ulcers in the Spanish healthcare setting.

Methods: We developed a weekly-cycle microsimulation state-transition model over 24 weeks from the Spanish National Health System perspective. Clinical effectiveness inputs for SAP dressings were derived from IPD from two observational clinical studies (n = 84, 2-week follow-up). Foam-dressing effectiveness was informed by a systematic review and meta-analysis of three eligible studies reporting 14-day wound-area reduction. The modelled cohort represented patients with moderate-to-highly exuding leg ulcers managed with compression therapy as standard of care; baseline severity was parameterised from the IPD (mean ulcer duration 15.1 months; baseline wound size 5,765 mm²).

Results: This early-stage model predicts an absolute incremental improvement in healing rate of 2.41 percentage points, an absolute incremental gain of 0.135 quality‑adjusted life weeks (QALWs) over a 24‑week period, and an absolute per‑patient direct cost reduction of €729 when SAP dressings are compared with foam dressings. When these incremental results are extrapolated to a hypothetical scenario of 100% adoption, the Spanish National Health System could achieve annual cost savings of €55.557 million. These estimates should be interpreted cautiously given the early‑stage, non‑comparative nature of the underlying evidence.

Limitations: Treatment‑effect estimates were derived from non‑randomized studies, which may introduce unmeasured confounding. As a result, the magnitude of the incremental effects is uncertain. To evaluate robustness, we conducted deterministic and probabilistic sensitivity analyses, both of which showed that the direction of the base‑case findings remained consistent across all plausible parameter ranges.

Conclusions: Preliminary model predictions suggest that polyacrylate wound dressings may provide additional health benefits and potentially reduce costs compared with foam dressings within the Spanish Health Care System.

Background: Biosimilar infliximab (B-IFX) offers a lower-cost alternative to originator infliximab (O-IFX) for treating Crohn's disease (CD), but real-world data on comparative effectiveness and economic impact in China remain limited. This study assessed clinical outcomes and economic consequences of B-IFX versus O-IFX from a healthcare payer perspective.

Methods: This retrospective cohort study evaluated 473 adult CD patients initiating O-IFX (n = 345) or B-IFX (n = 128) at a tertiary care center for Chron's disease in China between October 2021 and February 2024. The clinical endpoints over 12 months included treatment failure, clinical remission, endoscopic response, and adverse events. A budget impact analysis evaluated direct healthcare costs (Chinese Yuan, CNY) and projected medical insurance fund expenditures under a reference scenario (continued O-IFX use) and a base-case scenario with increased B-IFX uptake for 2025-2027. One-way sensitivity analyses assessed model robustness.

Results: Rates of treatment failure, clinical remission, endoscopic response, and adverse events were comparable between O-IFX and B-IFX. Relative to the reference scenario, increased B-IFX adoption was associated with lower annual direct medical costs and reduced insurance fund expenditures, yielding estimated savings of 0.60, 1.09, and 1.75 million CNY in 2025-2027, respectively (cumulative savings: 3.44 million CNY). Sensitivity analyses identified the unit price of O-IFX as the main cost driver; B-IFX remained cost saving across all parameter ranges, with cumulative 3-year savings ranging from 2.49 to 4.39 million CNY.

Conclusions: B-IFX demonstrated comparable real-world effectiveness and safety to O-IFX while generating substantial cost savings for China's medical insurance system. These findings support broader biosimilar adoption as a value-based strategy to enhance the affordability and sustainability of biologic therapy for CD.

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.

Aim: Conventional therapies in Japan for relapsed or refractory large B-cell lymphoma (r/r LBCL) require intravenous infusion (IV), while epcoritamab is administered subcutaneously. Despite the expected quality of life (QOL) improvement from reduced drug administration burden, limited data exists on QOL for common r/r LBCL treatment modalities. This study aimed to estimate the impact of drug administration on QOL (i.e. process utility) across treatments for r/r LBCL using responses from the Japanese general population.

Methods: An in-person questionnaire survey using the vignette-based time trade-off method was conducted. Participants from the Japanese general population, aged 20 years or older who were residing in Japan at the time of screening, valued vignettes representing therapy routes and schedules of epcoritamab monotherapy, salvage chemotherapy (R-ICE and Pola-BR as representatives) or no treatment. These vignettes were developed based on previous studies, clinical trial data, and expert opinions. Descriptive statistics were calculated for each vignette's utility values and the differences in utility between vignettes.

Results: The survey included 308 participants (mean age: 45.5 years; female: 50%). Utility for epcoritamab monotherapy was 0.459 for Cycle 1, 0.585 for Cycle 2-3, 0.642 for Cycle 4-9 and 0.678 for Cycle 10+ and beyond. Utilities for R-ICE were 0.151 for Cycle 1 and 0.380 for Cycle 2+; for Pola-BR were 0.216 for Cycle 1 and 0.490 for Cycle 2+. Relative to Pola-BR and R-ICE, epcoritamab was associated with higher utility values at all corresponding cycles, with differences of 0.243 and 0.307 in Cycle 1 and 0.094 and 0.205 in Cycles 2-3, respectively.

Conclusions: This study provides early economic insights, based on general population preferences, suggesting that epcoritamab's subcutaneous injection and outpatient administration beyond Cycle 2 may be associated with improved QOL compared to other therapies, regardless of efficacy and safety.

Aims: To assess the cost-effectiveness of hydrolyzed collagen as an adjunct to standard care for reducing postoperative complications in high-risk spinal surgery patients.

Methods: A decision tree model was developed from the U.S. healthcare sector perspective to compare hydrolyzed collagen with standard care alone over a one-year time horizon. The model followed a cohort of high-comorbidity patients undergoing complex spinal procedures, capturing postoperative infections, seromas, hematomas, readmissions, and revision surgeries. Clinical inputs were derived from published evidence, and costs were reported in 2025 U.S. dollars. Outcomes included direct medical costs, QALYs, and net monetary benefit (NMB) at a willingness-to-pay threshold of $100,000/QALY. Univariate, probabilistic, and threshold sensitivity analyses assessed parameter uncertainty.

Results: Hydrolyzed collagen dominated no treatment, generating $3,853 in cost savings and a 0.007 QALY gain per patient over one year, yielding an NMB of $4,542. Avoided readmissions and revision procedures were the primary contributors to cost savings. Baseline complication rates, relative risk reduction, and complication persistence rates were the most influential parameters. In probabilistic analysis, hydrolyzed collagen was cost-effective in more than 99% of simulations.

Limitations: The analysis applies simplifying assumptions, a one-year time horizon, and heterogeneous clinical inputs, factors that may limit the generalizability of the findings.

Conclusions: Compared to standard care alone, hydrolyzed collagen demonstrated a dominant strategy associated with cost savings and improved health outcomes for high-risk spinal surgery patients. These findings support its consideration as a value-enhancing component of the spine surgery care pathway, particularly for patients with elevated complication risk.

Background: Combination vaccines offer simultaneous protection against different diseases or strains of the same pathogen. They could improve vaccine coverage and health outcomes while reducing healthcare visits and injection costs. This systematic literature review (CRD420251135227) aimed to characterise methodologies employed in full economic evaluations of combination vaccines targeting multiple diseases.

Methods: Searches were performed in August 2025 in MEDLINE, Embase, Web of Science, CEA Registry, International HTA Database, and the NHS Economic Evaluation Database. Studied included cost-utility (CUA), cost-effectiveness (CEA) or cost-benefit analyses (CBA) evaluating all the diseases covered by combination vaccines. Given the qualitative research question, findings were narratively synthesised. Quality assessment used CHEERS and the WHO framework for Immunisation Programmes.

Results: Seven studies (3 CUAs; 4 CEAs) were included. They targeted five different combination vaccines across four countries and ∼20 years (1999-2019). All employed static models and compared to single vaccines (n = 3) or no vaccination (n = 5). Two studies adopted a societal perspective, three a healthcare perspective, and two adopted both. Four targeted paediatrics, three targeted adults. The efficacy of combination vaccines was assumed to be equivalent to single vaccines, and value was modelled as the incremental reduction in burden and costs of the additional disease covered, or due to higher coverage and fewer visits. There was variation in assessment of severe and long-term outcomes, adverse events, and productivity loss. Cross-disease data integration (e.g. co-infection, interference) was not considered. All studies generally supported cost-effectiveness of the assessed combination vaccines, but these results largely reflected the choice of comparator and modelling assumptions. Methodological and reporting quality varied widely.

Conclusions: Economic evaluations of combination vaccines targeting multiple diseases remain scarce, outdated and simplistic, highlighting opportunities to explore dynamic transmission models, broader societal perspectives, comparisons to single vaccines and extensive sensitivity analyses.

Aims: Early detection of hepatocellular carcinoma (HCC) in patients with compensated cirrhosis (CC) is critical for improving prognosis. The GAAD algorithm (gender [biological sex], age, alpha-fetoprotein [AFP], protein induced by vitamin K absence-II [PIVKA-II]) demonstrated good performance for the detection of early-stage HCC. This study aimed to assess the cost-effectiveness of the GAAD algorithm for HCC surveillance in patients with CC in Italy, from the Italian Health Service perspective.

Methods: A probabilistic micro-simulation Markov model was adapted to the Italian context to estimate lifetime clinical outcomes and costs of CC patients undergoing bi-annual surveillance with ultrasound (US), US+AFP, GAAD, and US+GAAD. Clinical inputs and utility values were derived from Italian real-world data and published literature. Direct healthcare costs were collected from Italian sources. Costs and outcomes were discounted at an annual 3% rate. Sensitivity analyses were conducted to evaluate the uncertainties in input parameters.

Results: In a simulated cohort of 100,000 CC patients, QALYs and costs per patient were 6.53 and €35,524 for US, 6.56 and €35,825 for US+AFP, 6.57 and €35,423 for GAAD, and 6.58 and €35,939 for US+GAAD. Compared to US and US+AFP, GAAD was dominant, while US+GAAD was cost-effective (ICUR of €9,482 and €10,951 per QALY gained, respectively). At a willingness-to-pay threshold of €30,000, GAAD was the most cost-effective strategy. Sensitivity analyses confirmed the robustness of results.

Limitations: Assumptions were required to estimate the diagnostic performance of US+GAAD, given the absence of prospective validation data. Some clinical parameters were derived from non-Italian sources, which may limit generalizability.

Conclusion: GAAD, alone or combined with US, is a cost-effective strategy for HCC surveillance in CC patients in Italy, improving the detection of early-stage disease. Better performance data for US+GAAD is needed to confirm results.