PharmacoEconomics最新文献

Background: Chronic kidney disease (CKD) is a major global cause of morbidity and mortality. Recent nephroprotective therapies have improved CKD management, yet their cost effectiveness across settings remains uncertain. This review systematically identified and compared cost-effectiveness studies of novel CKD treatments for both broad CKD populations and disease-specific subgroups.

Methods: A systematic search was conducted in PubMed and the Cochrane Library using terms related to "chronic kidney disease," "cost-effectiveness," "cost-utility," "health technology assessment," "SGLT2 inhibitor," and commercial and generic names of nephroprotective drugs approved since 2013. Eligible studies were full-length articles in English published between January 2015 and September 2025. Costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios were extracted. All monetary values were standardized to 2025 US dollars.

Results: The search yielded 172 records, of which 26 met inclusion criteria. A supplementary search identified ten additional studies, resulting in 36 evaluations. Most studies assessed sodium-glucose cotransporter 2 inhibitors or finerenone. Across evaluations, these therapies consistently improved outcomes, with QALY gains reported in all studies (0.012-1.44 QALYs gained). Most concluded that the interventions were cost effective compared with standard of care, and 13 reported cost-saving results. Only three studies reported an incremental cost-efffectiveness ratio above $100,000 per QALY threshold. Cost effectiveness was observed in both general CKD and CKD with diabetes mellitus, although estimates varied by country, time horizon, and analytic perspective.

Conclusions: Current evidence indicates that novel nephroprotective therapies for CKD are generally cost effective, and in some settings cost saving. These findings support their value in both general CKD and diabetic populations and highlight the importance of early treatment adoption to delay disease progression and reduce long-term healthcare costs.

Background and objective: Despite evidence on the cost effectiveness of catheter ablation (CA) as a rhythm control strategy in patients with atrial fibrillation, CAs form a substantial share of medical resource consumption, raising questions about optimal timing and maximum CAs per patient. This study addresses these questions using a newly developed open source model integrating observational and clinical trial data. The objective was to estimate the cost effectiveness of rhythm control strategies including anti-arrhythmic drugs (AADs) and/or CA in different sequences from a societal perspective in the Netherlands.

Methods: Time to atrial fibrillation symptom recurrence after a CA was estimated using parametric survival functions estimated on health insurance data (n = 24,286). Relative efficacy of CAs versus AADs was derived from meta-analyses, accounting for previous treatment exposure. Six treatment lines were modeled, incorporating AADs and CAs as rhythm control strategies. Medical and societal costs were included and the model had a lifetime time horizon. Model results were generated in 2024 Euros using Dutch input data with a cost-per-quality-adjusted life-year threshold of €20,000. For the probabilistic sensitivity analyses, we simultaneously varied all parameters across 1000 model runs with 5000 patients each.

Results: Treatment sequences including at least one CA were cost effective compared with only AADs. Catheter ablation costs are counterbalanced by reduced medical resource consumption in the years following CA. 51.6% of patients with first-line CA remain symptom free over a lifetime versus 6.9% with AADs. The most cost-effective strategy starts with CA, manages atrial fibrillation recurrences with AADs, and uses a maximum of three repeat ablations.

Conclusions: Our model suggests that rhythm control with at least one CA is cost effective compared with only AADs in patients with atrial fibrillation requiring rhythm control. Within shared decision making, first-line CA, followed by AADs to manage atrial fibrillation recurrences, with a maximum of three repeat ablations, represents the most cost-effective strategy for patients with symptomatic atrial fibrillation requiring rhythm control.

The majority of decision analytic models submitted to health technology assessment (HTA) agencies are developed using Microsoft Excel. The approaches commonly used to construct these models have not been substantially updated in decades, and studies have found that spreadsheet models are often slower and more difficult to validate than models built using R. However, Excel and Google Sheets were recently upgraded to add support for dynamic array functions. This allows for many of the techniques used in R modeling to be applied to spreadsheet models. This paper provides a tutorial on how these new functions can be leveraged to build efficient Markov cohort models using modern spreadsheet software. A novel approach is presented for conducting Monte Carlo simulation using a single formula in one cell, without the need for Visual Basic for Applications (VBA) macros. A number of template formulas are also provided that can be used to assist in common modeling tasks, including constructing a Markov trace and calculating the table of probabilities needed to plot cost-effectiveness acceptability curves (CEACs). These template formulas may be directly copied and pasted into any spreadsheet model, with no add-ons, plug-ins, or additional packages required. These advancements have the potential to modernize how spreadsheet models are developed, simplifying their construction, improving their calculation speed, and reducing the time needed for validation. They will also aid in teaching more efficient approaches for decision analytic modeling to a new generation of students.

Objectives: Non-adherence to cardiovascular disease (CVD) treatments leads to suboptimal health outcomes and increased healthcare and societal costs. We assessed the long-term effects of adherence to CVD and diabetes medications using population data and microsimulation modelling.

Methods: We developed a CVD microsimulation model using individual participant data from the SIDIAP database (2012-2021) for 152,117 adults who received new prescriptions for antihypertensive, lipid-lowering, oral glucose-lowering or antiplatelet treatments in Catalonia between January 2012 and December 2013. Model inputs included demographic and clinical characteristics, medication adherence and cardiovascular events. Costs (€, 2025) and treatment effects were sourced from the literature, and utilities were estimated using national population-based surveys. Model validity was assessed by comparing simulated and observed cumulative incidences over 8 years. The model simulated life-years (LYs), quality-adjusted life-years (QALYs) and healthcare and societal costs under three scenarios: non-adherent, observed adherence and full adherence. We estimated the maximum per-patient cost at which adherence-enhancing interventions would remain cost-effective.

Results: Simulated cumulative incidences of cardiovascular events and all-cause death closely matched observed data. Improved adherence increased survival by 0.19-0.58 years and QALYs by 0.25-0.70, while increasing lifetime healthcare costs by €2,431-€8,093 per patient. The additional cost per QALY ranged from €8,946 to €12,614 per QALY, indicating that improving adherence is likely to be a cost-effective if achieved at additional cost of up to €4,041 to €10,098 per patient.

Conclusions: Long-term extrapolation of real-world data using microsimulation modelling shows that optimising adherence to CVD and diabetes medications can enhance health outcomes cost-effectively.

Introduction: The addition of a glycopeptide antimicrobial to cephalosporin therapy has been reportedly adopted in practice to prevent surgical site infections (SSIs). We conducted an economic evaluation from the healthcare sector perspective to assess the cost-effectiveness of adding vancomycin to cefazolin prophylaxis in patients undergoing arthroplasty.

Methods: Data were collected over 180 days in a randomised controlled trial conducted at 11 hospitals in Australia, involving 2044 patients assigned to vancomycin and 2069 to placebo, both in addition to cefazolin. Health utilities were measured using EQ-5D-3L at baseline and 30 days, 90 days, and 180 days post-surgery. Healthcare costs (Australian dollars [AU$]; 2022 values) were estimated using Medicare claim data and hospital administrative records. Bootstrap was used to estimate means and 95% confidence intervals (CIs) of healthcare costs and quality-adjusted life years (QALYs). The net monetary benefit framework was used to construct a cost-effectiveness acceptability curve.

Results: Over 180 days, 96 SSIs occurred in the vancomycin group, and 79 in the placebo group. Mean QALYs were 0.392 (95% CI 0.389-0.395) in the vancomycin group and 0.394 (95% CI 0.391-0.397) in the placebo group. Mean total healthcare costs were AU$5184 (95% CI 4926-5480) in the vancomycin group and AU$5018 (95% CI 4772-5293) in the placebo group. Using willingness-to-pay and willingness-to-accept thresholds from AU$0 to AU$3,000,000, the probability of vancomycin being not cost-effective ranged from 0.79 to 0.87.

Conclusions: Adding vancomycin to cephazolin prophylaxis in arthroplasty is most likely not cost-effective. Omitting vancomycin could lead to substantial annual savings for the healthcare sector without compromising health outcomes.

Objectives: Bloodstream infections (BSI) are a leading cause of mortality worldwide. Rapid detection of the causative pathogen can help optimise therapy, reduce mortality, curb antimicrobial resistance, and lower healthcare costs. This study evaluates the cost effectiveness of adding molecular rapid diagnostic tests (mRDTs) to microbiology standard-of-care (SoC) methods. mRDTs evaluated include the Cobas^® Eplex blood culture identification (BCID) panels, BioFire BCID panel, BioFire BCID2 panel, Accelerate PhenoTest^™ blood culture (BC) kit and Diasorin Verigene^® BCID panels.

Methods: A decision-tree model was built to quantify the incremental costs and outcomes associated with adding mRDTs to the SoC. The inputs were derived from the published literature. The analysis considered a population aged 65 years and 45% female, admitted to a United States (US) hospital with a suspected BSI. Model outcomes included costs, 30-day mortality, quality-adjusted life years (QALYs) and adverse events (Clostridioides difficile infection and acute kidney injury [AKI]). A United Kingdom (UK) setting in place of the US setting was also considered in the scenario analysis.

Results: A strategy involving the Cobas Eplex BCID panels as an adjunct test to the SoC dominated SoC alone without Cobas Eplex BCID panels, saving US$164 per patient and averting 24 deaths per 10,000 patients. Earlier optimisation of ineffective empiric therapy generated half of the lives saved, with the majority of the remainder from reductions in AKI. This strategy was also dominant compared with other mRDTs. In a UK setting, Cobas Eplex BCID panels remained cost effective, saving £51 compared with SoC. Results were robust to scenarios varying key model inputs including time to pathogen identification with SoC.

Conclusions: The model demonstrated improved patient survival and reduced average total costs with mRDT. The Cobas Eplex BCID panels, which have the largest pathogen coverage, reduced both mortality and overall costs compared with other mRDTs.

Background: Two immunising products are emerging to prevent the burden of respiratory syncytial virus (RSV) in infants: long-lasting monoclonal antibodies (mAbs) and maternal vaccines given during pregnancy (MV). This study assesses the potential cost-effectiveness of programs involving each product to help inform policy decisions related to their implementation in the Australian context.

Methods: We developed an individual-based dynamic transmission model of RSV infection, linked to a clinical pathways model and cost-effectiveness model. We modelled key scenarios exploring varying eligibility and coverage of immunisation products for at-risk and not-at-risk populations, in addition to sensitivity analyses of immunisation characteristics, program costs and the impact of potential under-ascertainment of RSV burden. We estimated the cost-effectiveness of each program from a health system perspective, with results presented as incremental cost-effectiveness ratios in terms of cost per quality-adjusted life year gained (QALY).

Results: We found a combined program in which administration of MV during pregnancy is supplemented with a birth-dose of mAbs for newborns born without protection from MV is likely to be cost-saving, compared with the status quo of no MV or mAbs delivered. This program averted on average 41% of infant hospitalisations per year and reduced QALY losses by 33%.

Conclusions: Programs combining infant immunisation products are likely to significantly reduce the burden of RSV disease in Australia, and be cost-saving. However, their estimated impact and cost-effectiveness is strongly dependent on key assumptions (i) the consistency and completeness of ascertainment of disease burden over time; (ii) the cost of a hospitalisation and immunising dose; (iii) the efficacy and durability of protection of the modelled products; and (iv) the timing and coverage of the immunisation delivery.

Purpose: Conventional value-of-information (VOI) analysis bases decisions on a composite cost-effectiveness measure that aggregates clinical and cost outcomes. We sought to highlight potentially informative tradeoffs by estimating and reporting the "disaggregated" components, allowing stakeholders-such as clinicians, patients, trial funders, policymakers, and trial networks-to identify what drives the value of additional research.

Methods: We used a microsimulation to project the value of a hypothetical trial comparing mortality associated with different tuberculosis screening strategies among hospitalized patients with human immunodeficiency virus (HIV) in South Africa. Apart from expressing VOI as conventional, "aggregated" net monetary benefit (NMB), we assessed "disaggregated" outcomes that contribute to NMB calculations: tuberculosis cases detected, deaths, life-years, and healthcare costs associated with trial-informed decisions. We varied key parameters in probabilistic sensitivity analysis. We considered two willingness-to-pay thresholds: US $3000/year of life saved (YLS) (~50% of South Africa's per-capita gross domestic product [GDP]); and $780/YLS, the willingness-to-pay value where decision uncertainty is highest, per the baseline analysis.

Results: At willingness to pay = $3000/YLS, implementing the post-trial optimal strategy would produce favorable incremental NMB ($654.3 million) and better clinical outcomes (57,000 additional tuberculosis cases detected, 16,000 fewer deaths, and 294,000 more life-years), but it would incur an additional $37.4 million in healthcare costs over 5 years. At willingness to pay = $780/YLS, implementing the post-trial optimal strategy would yield positive incremental NMB of $2.5 million while lowering healthcare costs by $11 million, but it would produce worse clinical outcomes: 18,000 fewer tuberculosis cases detected, 6,000 additional deaths, and 80,000 fewer life-years.

Conclusions: Though a trial may yield positive NMB in conventional VOI analysis owing to expected cost savings, some clinical outcomes might be unfavorable. To better inform priorities, VOI studies should include disaggregated outcomes alongside aggregated NMB.

Background: In 2025, invasive meningococcal disease (IMD) vaccination in United States (US) adolescents consisted of two standard of care (SoC) schedules: Q-Q-B-B (routine MenACWY [Q] at ages 11 and 16 years; MenB [B] under shared clinical decision-making [SCDM] at age 16 years and 6 months later) or Q-P-B (routine MenACWY at ages 11 and 16 years; MenABCWY [P] at age 16 years and MenB under SCDM 6 months later). We assessed public health impact (PHI) and cost-effectiveness (CE) of intervention strategies on the basis of potential implementation options and revisions to the adolescent meningococcal vaccine schedule proposed in June 2024 by the Centers for Disease Control and Prevention's Advisory Committee on Immunization Practices.

Methods: A model utilizing epidemiological and economic inputs compared projected outcomes for 27 intervention strategies (distinguished by vaccine type [Q, P, and/or B], recommendation type [routine, risk-based], and dosing interval for MenB-containing vaccines [0,6-month , 0,24-month]) versus no vaccination or SoC over 15 years (2025-2039). Discounted outcomes (societal perspective) included IMD cases, deaths, and quality-adjusted life-year losses averted; total direct and indirect costs; and incremental CE ratios.

Results: Retaining routine MenACWY at age 11-12 years and including routine or risk-based MenB-containing vaccines at age 16 years improved PHI versus SoC, particularly with 0,6-month dosing interval for MenB-containing vaccines. Five strategies incorporating MenABCWY improved PHI and were cost-saving versus SoC: Q-Q-B-B.

Conclusions: Adolescent meningococcal vaccine schedule revisions retaining routine MenACWY and incorporating risk-based or routine MenABCWY may increase PHI in a cost-effective manner, minimizing the humanistic and economic burden of IMD.