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.

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.

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.

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.

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

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.

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.

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.

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

This study utilized the latest data from the 2021 Global Burden of Disease Study to analyze the incidence, prevalence, and years lived with disability due to vertebral fractures from 1990 to 2021, providing information for effective management and prevention strategies.

This study describes the trends in incidence, prevalence, and years lived with disability (YLDs) due to vertebral fractures caused by falls. It employs methods such as the Age-Period-Cohort (APC) model, joinpoint regression analysis, and decomposition analysis for further investigation, and calculates the ASIR, ASPR, and ASYR. Finally, it predicts the incidence trend for the next 15 years using the Autoregressive Integrated Moving Average (ARIMA) model.

In 2021, the number of new cases of vertebral fractures due to falls globally reached 4.7 million, with a total prevalence of 3.67 million cases, and years lived with disability (YLDs) amounted to 370,000. Compared to 1990, the estimated annual percentage change (EAPC) was -0.37 (-0.41, -0.32), -0.35 (-0.39, -0.31), and -0.37 (-0.41, -0.33) respectively, indicating a declining trend. There are significant differences in the disease burden among different countries and regions. The APC model, Joinpoint model, and ARIMA forecasting model indicate a global declining trend in the disease burden of vertebral fractures.

Although the burden of vertebral fractures is on a downward trend, it continues to increase in low and middle SDI regions, as well as among the elderly population. Therefore, targeted preventive measures are still necessary to address the health outcomes related to vertebral fractures.

Cardiometabolic diseases (CMDs), including cardiovascular disease (CVD) and type 2 diabetes (T2DM), are major contributors to morbidity, mortality, and rising healthcare costs. Effective disease prevention programs rely on robust mathematical models to generate long-term evidence regarding the effectiveness, cost-effectiveness, and policy implications of interventions in the population. Population-level interventions, such as dietary policies, are recognised as essential prevention strategies, yet there is limited syntheis of policy models assessing their impact. This study systematically reviews existing CMD policy models to provide: (i) a comprehensive overview of current models, and (ii) a critical appraisal of their application, particularly in the context of primordial prevention programmes.

A systematic search was conducted across MEDLINE (Ovid), EMBASE (Ovid), CINAHL, Google Scholar, and Open Grey. The search focused on publications from 1st January 2000, to 31st May 2024, using Medical Subject Headings (MeSH) for "cardiovascular," "diabetes," "decision model," and "policy model." Full-text articles were independently appraised independently by three reviewers using the Phillips et al. checklist, and the review process adhered to PRISMA guidelines.

Thirty-two articles met the inclusion criteria and were critically appraised. Policy models were assessed across three domains: structure, data, and consistency. Most models (79%) demonstrated well-defined structures, aligning inputs and objectives with the stated perspective and initial justifications. However, fewer than 60% of studies clearly reported the quality of their data sources and provided clear information in terms of consistency. The reviewed studies employed diverse methodologies, including parameter incorporation, simulation modelling, and outcome analysis.

The review highlights substantial heterogeneity in the quality, structure, and data use of policy models evaluating dietary interventions for CMD prevention. To advance CMD policy modeling, this study provides recommendations for improving conceptualisation, methodological rigor, and applicability to prevention programmes.

Registered protocol at PROSPERO: CRD42022354399.

Host-response-based transcriptional signatures (HrTS) have been developed to identify "incipient tuberculosis (TB)". No study has reported the cost-effectiveness of HrTS for post-arrival migrant screening programs in low-incidence countries. The aim of this study was to assess the potential health impact and cost-effectiveness of HrTS for post-arrival TB infection screening among new migrants in the United States.

We used a discrete-event simulation model to compare four strategies: (1) no screening for TB infection or incipient TB; (2) 'IGRA-only', screen all with interferon-gamma release assay (IGRA), provide TB preventive treatment for IGRA-positives; (3) 'IGRA-HrTS', screen all with IGRA followed by HrTS for IGRA-positives, provide incipient TB treatment for individuals testing positive with both tests; and (4) 'HrTS-only', screen all with HrTS, provide incipient TB treatment for HrTS-positives. We assessed outcomes over the lifetime of migrants entering the United Stataes (U.S.) in 2019, assuming HrTS met WHO Target Product Profile (TPP) optimal criteria. We conducted sensitivity analyses to evaluate the robustness of results. Our findings show that at a willingness-to-pay threshold of $150,000 per quality-adjusted life-year (QALY) gained, the IGRA-only strategy was the optimal strategy under both healthcare sector and societal perspectives, with an incremental cost-effectiveness ratio (ICER) of $104,138 and $143,103 per QALY gained, respectively. At a willingness-to-pay of $100,000 per QALY gained the IGRA-HrTS strategy appeared optimal. When the cohort was stratified by TB incidence in the country-of-origin, the IGRA-only strategy was optimal for country-of-origin incidence [Formula: see text]100 per 100,000, and the no-screening strategy was optimal for country-of-origin incidence <10 per 100,000. The IGRA-HrTS strategy was potentially cost-effective with country-of-origin incidence of 10-100 per 100,000, though this result had substantial uncertainty. Results were sensitive to time trends in TB progression risk after U.S. entry.

An HrTS test meeting WHO TPP optimal criteria would be potentially cost-effective for post-arrival screening among a subset of U.S. migrants, but this result was sensitive to multiple factors.

Probabilistic sensitivity analysis (PSA) is a method to account for uncertainty in cost-effectiveness analysis. The degree of correlation between input parameters is not well reported and is often overlooked in PSA. This means PSA results could be mis-estimating uncertainty. This study aimed to develop a simple model to explore the impact of input correlation on the incremental cost-effectiveness ratio (ICER) and the reported likelihood of cost-effectiveness.

A Markov model was developed with 3 different approaches to correlation: no correlation, partial correlation, and perfect correlation. A hypothetical case study was used to explore the impact of each correlation option on the intervention's likelihood of cost-effectiveness. Scenario analyses were also used to investigate whether the findings were consistent across different scenarios.

The ICER was comparable across the correlation options. In all scenarios, the no-correlation option had the most certain decision outcomes, and the perfect-correlation option had the least certain likelihood. The proximity of the ICER to the willingness-to-pay threshold influenced the impact of correlation on the PSA results.

This study suggests that the approach toward modeling parameter correlation in PSA has a substantial impact on the level of certainty in model outputs. By ignoring this, the level of certainty of cost-effectiveness could be over or underestimated. Therefore, researchers and decision makers should be careful to consider the potential impact of inter-parameter correlation.

People with low socioeconomic status (SES) or serious psychological distress (SPD) in the U.S. face ongoing and future disparities in tobacco smoking. We sought to estimate how smoking disparities contribute to disparities in life expectancy and aggregate life-years in these marginalized subpopulations.

We used the Simulation of Tobacco and Nicotine Outcomes and Policy (STOP) microsimulation model to project life expectancy as a function of subpopulation (low SES, higher SES, SPD, or non-SPD) and cigarette smoking status. Low SES was defined as having at least one of the following: income below poverty, less than high school education, or Medicaid insurance. Higher SES individuals belonged to none of these categories. SPD was defined as Kessler-6 score ≥ 13; non-SPD was a Kessler-6 score < 13. To project individual life expectancy losses from smoking, we simulated 40-year-olds stratified by gender, subpopulation (by SES or by SPD, with no change), and smoking status (current/never, with no change). To project time to reach 5% cigarette smoking prevalence (U.S.) - reflecting one tobacco "endgame" threshold - in each subpopulation, we simulated the entire subpopulations of people with low SES, higher SES, SPD, and non-SPD, incorporating corresponding distributions of gender, age, and smoking status and accounting for changes in smoking behaviors and secular smoking trends. We then estimated total life-years accumulated under status quo and alternate scenarios in which smoking dynamics in the marginalized subpopulations matched those of their less marginalized counterparts.

The model showed that, for individuals with low SES or SPD, smoking is associated with substantial loss of life expectancy (9.8-11.5y). Marginalized subpopulations would reach 5% smoking prevalence 20y (low SES) and 17y (SPD) sooner if smoking trends mirrored their less marginalized counterparts; these differences result in 5.3 million (low SES) and 966,000 (SPD) excess life-years lost over 40y.

Differences in cigarette smoking portend substantial ongoing and future disparities in life expectancy and time to reach 5% smoking prevalence. Reducing tobacco-related disparities in the U.S. will require an explicitly equity-focused vision, and the tobacco endgame will only be truly achieved when it includes all groups.

Tuberculosis services in many settings rely heavily on international donor funding. In 2025, the United States Agency for International Development (USAID) was dismantled, and other countries also announced cuts to overseas development assistance. We quantified potential epidemiological impacts attributable to these reductions in international donor funding.

We calibrated a deterministic tuberculosis model to epidemiological indicators in low- and middle-income countries. We projected three future scenarios assuming: a) levels of funding in 2024 continue through 2035, b) termination of USAID funding from 2025, and c) additional reductions in funding through The Global Fund in line with current donor announcements from 2025. We assumed a reduction in tuberculosis treatment initiation rates proportional to budget reductions for each scenario, estimating cumulative excess incident episodes of symptomatic tuberculosis and tuberculosis deaths.

We modelled 79 countries, representing 91% of global tuberculosis incidence and 90% of global tuberculosis mortality in 2023. Our modelling suggested that the termination of USAID funding may lead to 420 500 excess tuberculosis deaths by 2035. Further reductions in funding in line with current announcements by the United States, France, the United Kingdom, and Germany may lead to an additional 699 200, 63 100, 50 500, and 30 500 TB deaths, respectively. Impacts would be greatest in low-income countries.

We estimate substantial potential impacts on tuberculosis morbidity and mortality due to reductions in international donor funding. Expanded support from domestic and international donors is essential to address immediate gaps in prevention, diagnosis, and treatment.

This work was unfunded.

In the context of oropharyngeal cancer poised to impose a significant disease burden, this study conducted an economic evaluation of HPV vaccination in Chinese male adolescents for the prevention of HPV-positive oropharyngeal cancer (OPC-HPV+), by constructing a multi-state Markov model from the societal perspective.

The model estimated the cost, effectiveness, and health utility of the bivalent, quadrivalent, and nonavalent HPV vaccines in preventing OPC-HPV+. Incremental cost-utility ratio (ICUR) was used to evaluate the economic viability of the vaccination strategies. One-way sensitivity analysis and probabilistic sensitivity analysis were employed to assess the model's stability.

At a vaccine coverage rate of 70%, the incremental cost-effectiveness ratios of the bivalent, quadrivalent, and nonavalent vaccines were all lower than the per capita GDP compared to no vaccination, indicating that the vaccination strategies are highly cost-effective. The nonavalent vaccine has the highest incremental cost-effectiveness ratio, at 64,913.42 yuan ($9,211.86)/QALY. This strategy also has the highest cost, at 112.34 billion yuan, but it provides the best protection outcomes, preventing 2,545,988 cases of persistent HPV infection, 31,186 cases of OPC-HPV+, and 15,138 deaths, saving a total of 2,641,783 QALYs. Sensitivity analysis indicates that the discount rate, vaccine efficacy, HPV infection rate in the general population, and the probability of spontaneous clearance are the main factors affecting the pairwise comparison results of the strategies, which may lead to instability in the cost-effectiveness of the nonavalent vaccine.

HPV vaccination for male adolescents to prevent oropharyngeal cancer is cost-effective compared to no vaccination. China could expand the coverage of the appropriate-priced HPV vaccine to male adolescents in order to reduce the incidence of oropharyngeal cancer, improve male health quality, and protect public health.

Lecanemab slows cognitive decline among people with early Alzheimer's disease (early AD) but appears to increase the risk of intracranial hemorrhages (ICHs), including anticoagulant-related ICHs.

To examine the benefits and harms of co-prescribing lecanemab and anticoagulants in people with atrial fibrillation (AF) experiencing early AD.

Microsimulation model to compare four treatment strategies. Using inputs from the literature, we modeled increased ICH risk with lecanemab (2.02-fold), apixaban (1.84-fold), and lecanemab/apixaban interaction (2.67-fold). We assigned quality-of-life estimates and increased mortality risk with cognitive decline, stroke, and ICH.

Clinical trials, observational cohorts.

People 65-90 years with AF and early AD.

18-month.

Apixaban ( APIX ), apixaban and lecanemab ( APIX/LEC ), lecanemab ( LEC ), neither.

ICH, ischemic stroke, cognitive decline, quality-adjusted life months (QALMs), and survival, age-stratified.

For 100,000 simulated persons aged 65-74 years, APIX , APIX/LEC , and LEC would result in a similar clinical benefit (13.2 QALMs). Compared to APIX , APIX/LEC would result in more ICH events (1,990 vs. 400), all-cause deaths (5,820 vs. 5,140), but slower cognitive decline (mean CDR-SB change, 1.11 vs. 1.53). For persons ≥75 years, APIX alone would always be preferred.

Results are sensitive to lecanemab-anticoagulant interaction on ICH, baseline ICH risk, and lecanemab's effect on cognition.

Significant parameter uncertainty; treatment burden and costs were not modeled.

Model-based results support anticoagulants alone as the preferred strategy for people ≥75 years with early AD and AF. There was greater equipoise across treatment strategies for persons 65-74 years, for whom improved estimates of the ICH risk and lecanemab-anticoagulant interaction are critical to identifying the preferred strategy.

National Institute on Aging/National Institutes of Health (K76AG074919, P30AG062421, U01AG076478, and R01AG069575).

Deroofing and local excision are common clinic-based surgical options for hidradenitis suppurativa. Evidence suggests deroofing may have lower rates of adverse events (AEs), defined as disease recurrence or postsurgical complications.

This cost-utility analysis evaluates the economic and health-related impacts of clinic-based deroofing vs excision for hidradenitis suppurativa, comparing direct medical costs and quality-adjusted life-years (QALYs).

A Markov model was developed based on a literature review of clinical outcomes, EQ-5D utilities, and resource utilization. Patients began in a preprocedural state and transitioned monthly among 3 health states: responders (no AEs), nonresponders (≥1 AE), and death. The model assessed cost-effectiveness over a 2-year horizon from the U.S. healthcare system perspective.

Deroofing provided an additional 0.19 QALYs at a cost of USD$311.39 per patient relative to excision, yielding a favorable incremental cost-effectiveness ratio of USD$1677.10/QALY, below the USD$50,000/QALY threshold.

Methodological constraints from limited published data were addressed through multiple sensitivity analyses. Cost-effectiveness was sensitive to AE rates, secondary costs, and utility values.

When clinically appropriate, deroofing is more cost-effective than excision for clinic-based procedural management of HS, offering improved quality of life at a modest incremental cost.

To assess the cost-effectiveness sand the budgetary impact of the combination of botulinum toxin (BT) + conventional treatment (CT) (hormonal treatments + analgesics) compared with CT alone in patients suffering from severe dysmenorrhoea, using a Markov model.

A Markov model was developed to estimate, from the perspective of French Health Insurance (HI), the cost effectiveness and the budgetary impact of BT+CT compared with CT alone. The main health states in the model were based on Visual Analogue Scale (VAS) scores and expert opinion. All model parameters were derived from a cohort of patients treated for 12 months at the Centre de Recherche de la Santé et de la Femme (CRSF) for severe dysmenorrhoea in 2021. A Cost-Utility Analysis (CUA) was carried out to assess the quality of life of patients, crucial in this context, in which the direct healthcare costs were considered in and Budget Impact Analysis (BIA). The main decision-making criteria were the Incremental Cost-Utility Ratio (ICUR) for the CUA and the net impact for the BIA. Deterministic and probabilistic univariate sensitivity analyses were performed to assess the robustness of our results.

Over the 1-year time horizon (main analysis), the costs and quality-adjusted life year (QALY) of BT+CT versus CT alone were equal to €1895.65 vs €3055.20 and 2.03 QALYs vs 1.23 QALYs, respectively. Consequently, the ICUR equalled -€1651.5/QALY, which shows that, although the initial costs of BT are higher than those of CT, the reduced follow-up costs associated with the long-term efficacy of BT make it the most effective and economically dominant option at 1, 5 and 10 years. Sensitivity analyses show that 100 % of Monte Carlo iterations are below the willingness-to-pay threshold of €30,0001/QALY, making BT+CT an efficient strategy that could be adopted and reimbursed.

In the absence of a reference treatment for the management of severe dysmenorrhoea, BT+CT offering an improvement in quality of life, as well as a reduction in follow-up costs. It is therefore the most cost-effective strategy over 10 years.

The ABATE Infection trial investigated the effects of universal bacterial decolonization with chlorhexidine for patients in non-intensive care unit settings to reduce hospital-onset bacteremia and fungemia (HOB) events. Among patients with medical devices (central venous catheters, midline catheters, and lumbar drains), universal decolonization (UD) resulted in a significant and meaningful reduction in bacteremia compared with the standard of care (SOC), but cost-effectiveness is unclear.

To examine the cost-effectiveness of universal and targeted bathing strategies compared with SOC in general medical and surgical units.

A decision analytic model was constructed from June 1, 2021, to May 31, 2024, to simulate the frequency of HOB and costs under 3 strategies: SOC, UD, and targeted decolonization (TD). The model included a simulated cohort representative of the cluster-randomized ABATE Infection trial, which involved more than 500 000 participants across the US.

In TD, decolonization was administered for patients with medical devices only. Upstream costs of bathing and downstream costs of HOB, under payer and hospital perspectives were included. Parameters were informed by the ABATE Infection Trial and additional literature. Willingness-to-pay per HOB prevented was adopted as $25 000 for payers and $10 000 for hospitals. Sensitivity analyses were tailored to populations with different characteristics.

The simulated cohort, based on the population from the ABATE trial, included 529 000 adult admissions with a mean (SD) age of 63 (18) years, 54% female, and 13% with a central venous catheter, midline catheter, or lumbar drain. In the base case, the SOC was least effective and most costly. Targeted decolonization was least costly and UD resulted in the fewest HOB events. Targeted decolonization was the cost-effective strategy from payer and hospital perspectives. Compared with TD, UD had an incremental cost-effectiveness ratio of $119 700 per HOB averted from the payer perspective, and $126 600 per HOB averted from the hospital perspective. Depending on willingness-to-pay, UD may be preferred in scenarios with a higher proportion of patients with medical devices, greater reductions in HOB from decolonizing in those with devices, and lower adherence under TD.

In this decision analytic model studying universal and targeted bathing, TD was cost-effective under a broad range of scenarios for both hospital system and payer decision-makers. Universal decolonization was cost-effective in some scenarios, such as in specific units where many patients have medical devices or if it were difficult to implement a targeted approach.

Hunter syndrome is among the costliest life-long genetic conditions associated with a substantial burden-of-illness and a significant impact on the health systems, families, and society. We estimated the cost-effectiveness of long-term enzyme replacement therapy with idursulfase versus the standard of care from a societal perspective using a streamlined modeling strategy in R.

A de novo 4-state partitioned survival model was developed to compare lifetime cost and outcomes of 2 care models operationalized in R. The disease progression was based on independent survival modeling of relevant Kaplan-Meier data. The healthcare and out-of-pocket costs were drawn from the local setting. The quality of life was measured using the EQ5D5L and the time trade-off valuation of health-state vignettes that match the states in the model. Probabilistic and deterministic sensitivity analyses were conducted to test the uncertainty around the model results.

The lifetime incremental quality-adjusted life years were 4.1 years (95% CI, 2.37-5.68). Incremental costs were estimated to be $9.5 million (95% CI, 9.0 million-10.0 million), which primarily consists of drug costs (99%). The incremental costs per quality-adjusted life year were estimated to be approximately $2.4 million (95% CI, 1.7 million-3.8 million). Sensitivity analyses showed that the key drivers of incremental cost-effectiveness ratio were quality of life in the preprogression state and differential discounting approach, besides the acquisition cost of enzyme replacement therapy of idursulfase.

The incremental cost-effectiveness ratios were beyond any conventionally used cost-effectiveness threshold in all cases. At the current price, there is a significant discrepancy between the therapy's funding decision and the cost-effectiveness assessment as a basis for guiding healthcare prioritization in Malaysia.

Health technology assessment is used extensively by the Pharmaceutical Benefits Advisory Committee (PBAC) to inform medicine funding recommendations in Australia. The PBAC often does not recommend medicines due to uncertainties in economic modelling that result in delaying access to medicines for patients. The systematic identification of which uncertainties can be reduced with alternative evidence or the collection of additional data can help inform recommendations. This study aims to characterise different types of uncertainty in economic models and empirically assess their association with the PBAC recommendations.

A framework was developed to characterise four types of uncertainties: methodological, structural, generalisability and parameter uncertainty. The first two types were further subcategorised into parameterisable and unparameterisable uncertainty. Data on uncertainty and other factors were extracted from PBAC's Public Summary Documents of first submissions for 193 medicine (vaccine)-indication pairs including economic modelling between 2014 and 2021. Logistic regression was used to estimate the average marginal effect of each type of uncertainty on the probability of a positive recommendation.

The PBAC more often raised issues regarding parameter uncertainty (95%) and parameterisable structural uncertainty (83%) than generalisability uncertainty (48%) and unparameterisable methodological uncertainty (56%). The logistic regression results suggested that the PBAC was more likely to recommend a medicine without unparameterisable methodological, generalisability, and parameterisable structural uncertainty by 15.0%, 10.2 %, and 17.6%, respectively. Parameterisable methodological, unparameterisable structural and parameter uncertainty were not significantly associated with the PBAC recommendations.

This study identified the uncertainties that had significant associations with PBAC recommendations based on the first submission. This may help improve model quality and reduce resubmissions in the future, thus improving patients' access to medicines.

Low-dose aspirin and rivaroxaban are the cornerstone treatment for cardiovascular prevention in patients with peripheral artery disease (PAD) and/or stable coronary artery disease (SCAD). The combination of rivaroxaban with aspirin imposes a synergistic effect on the inhibition of factor-induced platelet aggregation. The present work aimed at comparing the cost-utility and cost-effectiveness of rivaroxaban (2.5 mg twice daily) plus aspirin (100 mg once daily), rivaroxaban alone (5 mg twice daily) with aspirin alone in patients with peripheral artery disease (PAD) or coronary artery disease (CAD) and related subgroups.

This pharmacoeconomic study was performed based on the insurance organization and utilized a state-transition decision Markov model. From the COMPASS trial, Clinical efficacy and Clinical events were collected. Health outcomes and cost were assessed over a 20-year time horizon (lifetime). The direct costs of medical services were included in the analysis. The results were stated based on Incremental Cost-Utility (ICUR) and Incremental Cost Effectiveness Ratio (ICER). Uncertainty was assessed utilizing deterministic and probabilistic sensitivity analyses. Discount rates of .058 and .03 were included for cost and effectiveness data, respectively. The budget impact based on the Markov model was estimated as the financial burden resulting from the insurance coverage of rivaroxaban.

In the total of CAD and PAD patients, treatment with rivaroxaban plus aspirin and rivaroxaban alone were more expensive than the aspirin alone, but also more effective, resulting in ICUR being $4594/QALY and $13601/QALY respectively, and for ICER being $3348/LYG and $9901/LYG. In PAD patients rivaroxaban plus aspirin had higher effectiveness than aspirin alone that ICUR and ICER being $11929/QALY and $9896/LYG respectively. In CAD patients, treatment with rivaroxaban plus aspirin was expensive and less effective than aspirin alone. The estimated annual budget impact was $28,253,135 for the rivaroxaban plus aspirin and $292,593,909 for the rivaroxaban alone in the total of CAD and PAD patients.

This study showed that rivaroxaban plus aspirin is a cost-effective alternative in PAD and total of CAD and PAD patients. In CAD patients, rivaroxaban plus aspirin and rivaroxaban alone were not cost-effective.

Different types of mathematical models can be used to forecast the development of diseases as well as associated costs and analyse the cost-effectiveness of interventions. The set of models available to assess these parameters, reach from simple independent equations to highly complex agent-based simulations. For many diseases, it is simple to distinguish between infectious diseases and chronic-degenerative diseases. For infectious diseases, dynamic models are most appropriate because they allow for feedback from the number of infected to the number of new infections, while for the latter Markov models are more appropriate since this feedback is not required. However, for some diseases, the aforementioned distinction is not as clear. Cervical cancer, for instance, is caused by a sexually transmitted virus, and therefore falls under the definition of an infectious disease. However, once infected, the condition can progress to a chronic disease. Consequently, cervical cancer could be considered an infectious or a chronic-degenerative disease, depending on the stage of infection. In this paper, we will analyse the applicability of different mathematical models for epidemiological and economic processes focusing on cervical cancer. For this purpose, we will present the basic structure of different models. We will then conduct a literature analysis of the mathematical models used to predict the spread of cervical cancer. Based on these findings we will draw conclusions about which models can be used for which purpose and which disease. We conclude that each type of model has its advantages and disadvantages, but the choice of model type often seems arbitrary. In the case of cervical cancer, homogenous Markov models seem appropriate if a cohort of newly infected is followed for a shorter period, for instance, to assess the impact of screening programs. For long-term consequences, such as the impact of a vaccination program, a feedback loop from former infections to the future likelihood of infections is required. This can be done using system dynamics or inhomogeneous Markov models. Discrete event or agent-based simulations can be used in the case of cervical cancer when small cohorts or specific characteristics of individuals are required. However, these models require more effort than Markov or System Dynamics models.

To provide an introduction to the uses of generative artificial intelligence (AI) and foundation models, including large language models, in the field of health technology assessment (HTA).

We reviewed applications of generative AI in 3 areas: systematic literature reviews, real-world evidence, and health economic modeling.

(1) Literature reviews: generative AI has the potential to assist in automating aspects of systematic literature reviews by proposing search terms, screening abstracts, extracting data, and generating code for meta-analyses; (2) real-world evidence: generative AI can facilitate automating processes and analyze large collections of real-world data, including unstructured clinical notes and imaging; (3) health economic modeling: generative AI can aid in the development of health economic models, from conceptualization to validation. Limitations in the use of foundation models and large language models include challenges surrounding their scientific rigor and reliability, the potential for bias, implications for equity, as well as nontrivial concerns regarding adherence to regulatory and ethical standards, particularly in terms of data privacy and security. Additionally, we survey the current policy landscape and provide suggestions for HTA agencies on responsibly integrating generative AI into their workflows, emphasizing the importance of human oversight and the fast-evolving nature of these tools.

Although generative AI technology holds promise with respect to HTA applications, it is still undergoing rapid developments and improvements. Continued careful evaluation of their applications to HTA is required. Both developers and users of research incorporating these tools, should familiarize themselves with their current capabilities and limitations.

Genomic screening to identify individuals with Lynch Syndrome (LS) and those with a high polygenic risk score (PRS) promises to personalize colorectal cancer (CRC) screening. Understanding its clinical and economic impact is needed to inform screening guidelines and reimbursement policies.

We developed a Markov model to simulate individuals over a lifetime. We compared LS+PRS genomic screening with standard of care (SOC) for a cohort of US adults at age 30. The Markov model included health states of no CRC, CRC stages (A-D), and death. We estimated incidence, mortality, and discounted economic outcomes of the population under different interventions.

Screening 1000 individuals for LS+PRS resulted in 1.36 fewer CRC cases and 0.65 fewer deaths compared with SOC. The incremental cost-effectiveness ratio was $124,415 per quality-adjusted life year; screening had a 69% probability of being cost-effective using a willingness-to-pay threshold of $150,000/quality-adjusted life year . Setting the PRS threshold at the 90th percentile of the LS+PRS screening program to define individuals at high risk was most likely to be cost-effective compared with 95th, 85th, and 80th percentiles.

Population-level LS+PRS screening is marginally cost-effective, and a threshold of 90th percentile is more likely to be cost-effective than other thresholds.

Probabilistic analysis, also referred to as probabilistic sensitivity analysis (PSA), is used extensively in cost-effectiveness evaluations of health technologies. We present methodological guidance for implementing probabilistic analysis and interpreting its results for policy and decision-making.

We review the methodological issues related to common practices in probabilistic analysis, explore aspects that are currently not widely addressed in the health economics literature, and provide an overview of recent methodological developments.

We use examples to highlight the advantages and disadvantages of common tools used for presenting probabilistic analysis results, including the cost-effectiveness acceptability curve (CEAC), cost-effectiveness acceptability frontier (CEAF), and value of information (VOI) analysis. We raise and address issues related to using Monte Carlo standard error to determine the number of iterations required, the implications of large uncertainty, and the credibility and meaningfulness of small differences in quality-adjusted life-years (QALYs). We then discuss evolving methods in probabilistic analysis, cautious uses of probabilistic analysis, and factors impacting parameter uncertainty.

A deeper understanding of probabilistic analysis methods enables health economists and decision-makers to more effectively address and interpret parameter uncertainty in health economic evaluations, which is essential for making informed policy decisions.

This systematic literature review addresses model-based cost-effectiveness studies for therapy response monitoring with positron emission tomography (PET) generally combined with low-dose computed tomography (CT) for various cancer types. Given the known heterogeneity in therapy response events, studies should consider patient-level modelling rather than cohort-based modelling because of its flexibility in handling these events and the time to events. This review aims to identify the modelling methods used and includes a systematic assessment of the assumptions made in the current literature.

This study was conducted and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 statement. Information sources included electronic bibliographic databases, reference lists of review articles and contact with experts in the fields of nuclear medicine, health technology assessment and health economics. Eligibility criteria included peer-reviewed scientific publications and published grey literature. Literature searches, screening and critical appraisal were conducted by two reviewers independently. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) were used to assess the methodological quality. The Bias in Economic Evaluation (ECOBIAS) checklist was used to determine the risk of bias in the included publications.

The search results included 2959 publications. The number of publications included for data extraction and synthesis was ten, representing eight unique studies. These studies addressed patients with lymphoma, advanced head and neck cancers, brain tumours, non-small cell lung cancer and cervical cancer. All studies addressed response to chemotherapy. No study evaluated response to immunotherapy. Most studies positioned PET/CT as an add-on modality and one study positioned PET/CT as a replacement for conventional imaging (X-ray and contrast-enhanced CT). Three studies reported decision-tree structures, four studies reported cohort-level state-transition models and one study reported a partitioned survival model. No patient-level models were reported. The simulation horizons adopted ranged from 1 year to lifetime. Most studies reported a probabilistic analysis, whereas two studies reported a deterministic analysis only. Two studies conducted a value of information analysis. Multiple studies did not adequately discuss model-specific aspects of bias. Most importantly and regularly observed were a high risk of structural assumptions bias, limited simulation horizon bias and wrong model bias.

Model-based cost-effectiveness analysis for therapy response monitoring with PET/CT was based on cohorts of patients instead of individual patients in the current literature. Therefore, the heterogeneity in therapy response events was commonly not addressed appropriately. Further research should include more advanced and patient-level modelling approaches to accurately represent the complex context of clinical practice and, therefore, to be meaningful to support decision making.

This review is registered in PROSPERO, the international prospective register of systematic reviews funded by the National Institute for Health Research, with CRD42023402581.

Antimicrobial resistance (AMR) is a growing threat to the efficacy of antimicrobials in humans and animals, including those used to control bovine respiratory disease (BRD) in high-risk calves entering western Canadian feedlots. Successful mitigation strategies require an improved understanding of the epidemiology of AMR. Specifically, the relative contributions of antimicrobial use (AMU) and contagious transmission to AMR emergence in animal populations are unknown.

A stochastic, continuous-time agent-based model (ABM) was developed to explore the dynamics of population-level AMR in Mannheimia haemolytica in pens of high-risk cattle on a typical western Canadian feedlot. The model was directly informed and parameterized with proprietary data from partner veterinary practices and AMU/AMR surveillance data where possible. Hypotheses about how AMR emerges in the feedlot environment were represented by model configurations in which detectable AMR was impacted by (1) only selection arising from AMU; (2) only transmission between animals in the same pen; and (3) both AMU-linked selection and transmission. Automated calibration experiments were used to estimate unknown parameters of interest for select antimicrobial classes. Calibrated parameter values were used in a series of Monte Carlo experiments to generate simulated outputs at both the pen and feedlot levels. Key model outputs included the prevalence of AMR by class at multiple time points across the feeding period. This study compared the relative performances of these model configurations with respect to reproducing empirical AMR data.

Across all antimicrobial classes of interest, model configurations which included the potential for contagious acquisition of AMR offered stronger fits to the empirical data. Notably, sensitivity analyses demonstrated that model outputs were more robust to changes in the assumptions underscoring AMU than to those affecting the likelihood of transmission.

This study establishes a feedlot simulation tool that can be used to explore questions related to antimicrobial stewardship in the context of BRD management. The ABM stands out for its unique hierarchical depiction of AMR in a commercial feedlot and its grounding in robust epidemiological data. Future experiments will allow for both AMU-linked selection and transmission of AMR and can accommodate parameter modifications as required.

The ORIENT-15 double-blind randomized controlled trial demonstrated that the addition of sintilimab to chemotherapy for locally advanced or metastatic oesophageal squamous cell carcinoma (OSCC) resulted in better clinical outcomes. In this analysis, we sought to evaluate the cost-effectiveness of sintilimab as a first-line treatment for locally advanced or metastatic OSCC from a healthcare system perspective in China.

A partitioned survival model was constructed to perform a cost-effectiveness analysis comparing chemotherapy alone with sintilimab for locally advanced or metastatic OSCC patients. Clinical data were obtained from the ORIENT-15 trial and extrapolated to 10 years. Health state utilities and costs were sourced from the literature and from public healthcare institutions. The primary outcomes included the incremental cost-effectiveness ratio (ICER) and quality-adjusted life-years (QALYs). Two different sensitivity analyses, one-way and probabilistic, were performed to assess model uncertainty.

Sintilimab-based chemotherapy was more costly ($31699.21 vs. $20687.42) and more effective (0.74 vs. 0.53) than placebo-based chemotherapy, resulting in an ICER of $51908.19 /QALY, which is greater than the willingness-to-pay (WTP) threshold of China ($38223/QALY). Sensitivity analysis demonstrated that the PFS and cost of sintilimab were the major influencing factors affecting the results.

In patients with locally advanced or metastatic OSCC, sintilimab chemotherapy could improve survival time and health benefits compared with traditional chemotherapy, but the present analysis suggests that sintilimab is not a cost-effective treatment option in China.

Health technology assessment (HTA) guidelines are intended to support the successful implementation of HTA by enhancing consistency and transparency in concepts, methods, processes, and use, thereby enhancing the legitimacy of the decision-making process. This report lays out good practices and practical recommendations for developing or updating HTA guidelines to ensure successful implementation.

The task force was established in 2022 and comprised experts and academics from various geographical regions, each with substantial experience in developing HTA guidelines for national health policy making. Literature reviews and key informant interviews were conducted to inform these good practices. Stakeholder consultations, open peer reviews, and expert opinions validated the recommendations. A series of teleconferences among task force members was held to iteratively refine the report.

The recommendations cover six key aspects throughout the guideline development cycle: (1) setting objectives, scope, and principles of the guideline, (2) building a team for a quality guideline, (3) defining a stakeholder engagement plan, (4) developing content and utilizing available resources, (5) putting in place appropriate institutional arrangements, and (6) monitoring and evaluating guideline success.

This report presents a set of resources and context-appropriate practices for developing or updating HTA guidelines. Across all contexts, the recommendations emphasize transparency, building trust among stakeholders, and fostering a culture of ongoing learning and improvement. The report recommends timing development and revision of guidelines according to the HTA landscape and pace of HTA institutionalization. Because HTA is increasingly used to inform different kinds of decision making in a variety of country contexts, it will be important to continue to monitor lessons learned to ensure the recommendations remain relevant and effective.

Chronic kidney disease (CKD)-associated pruritus is a condition that strongly impacts CKD patients and is associated with increased morbidity/mortality, and worse health-related quality of life (HRQoL). Difelikefalin is currently the only drug approved in Europe specifically for treating moderate to severe CKD-associated pruritus in patients undergoing hemodialysis. The KALM-1 and KALM-2 trials showed better efficacy of difelikefalin vs placebo and best supportive care. The aim of this study was to investigate the cost-effectiveness of difelikefalin according to the Italian National Health Service (NHS) perspective.

A cohort model represented by four health states (No, Mild, Moderate, and Severe pruritus) was adapted to the Italian setting. The model used data from the KALM-1 and -2 trials for efficacy, integrated with other publications for HRQoL estimations. To assess the cost of disease management, a recent Italian publication on CKD-associated pruritus was used and a price of €27 per difelikefalin vial was assumed. The base case analysis over a 15-year time horizon, and an additional 10-year scenario analysis, were established. Additionally, both deterministic univariate analysis and probabilistic multivariate sensitivity analyses were developed. Discount rates of 3% were applied. An acceptability threshold of 40,000 €/quality-adjusted life-year (QALY) was considered.

The results show that difelikefalin plus best supportive care is cost-effective vs best supportive care alone, with an incremental cost-effectiveness ratio, in the base case, of €35,823/QALY. Both the scenario and sensitivity analyses confirmed the strength of the results.

Difelikefalin was found to be a cost-effective treatment for the Italian NHS. These results support its reimbursement and its inclusion in routine clinical practice.

(1) To demonstrate the use of quality-adjusted life-years (QALYs) as an outcome measure for comparing performance between simulation models and identifying the most accurate model for economic evaluation and health technology assessment. QALYs relate directly to decision making and combine mortality and diverse clinical events into a single measure using evidence-based weights that reflect population preferences. (2) To explore the usefulness of Q2, the proportional reduction in error, as a model performance metric and compare it with other metrics: mean squared error (MSE), mean absolute error, bias (mean residual), and R2.

We simulated all EXSCEL trial participants (N = 14,729) using the UK Prospective Diabetes Study Outcomes Model software versions 1 (UKPDS-OM1) and 2 (UKPDS-OM2). The EXSCEL trial compared once-weekly exenatide with placebo (median 3.2-y follow-up). Default UKPDS-OM2 utilities were used to estimate undiscounted QALYs over the trial period based on the observed events and survival. These were compared with the QALYs predicted by UKPDS-OM1/2 for the same period.

UKPDS-OM2 predicted patients' QALYs more accurately than UKPDS-OM1 did (MSE: 0.210 v. 0.253; Q2: 0.822 v. 0.786). UKPDS-OM2 underestimated QALYs by an average of 0.127 versus 0.150 for UKPDS-OM1. UKPDS-OM2 predictions were more accurate for mortality, myocardial infarction, and stroke, whereas UKPDS-OM1 better predicted blindness and heart disease. Q2 facilitated comparisons between subgroups and (unlike R2) was lower for biased predictors.

Q2 for QALYs was useful for comparing global prediction accuracy (across all clinical events) of diabetes models. It could be used for model registries, choosing between simulation models for economic evaluation and evaluating the impact of recalibration. Similar methods could be used in other disease areas.

Diabetes simulation models are currently validated by examining their ability to predict the incidence of individual events (e.g., myocardial infarction, stroke, amputation) or composite events (e.g., first major adverse cardiovascular event).We introduce Q2, the proportional reduction in error, as a measure that may be useful for evaluating and comparing the prediction accuracy of econometric or simulation models.We propose using the Q2 or mean squared error for QALYs as global measures of model prediction accuracy when comparing diabetes models' performance for health technology assessment; these can be used to select the most accurate simulation model for economic evaluation and to evaluate the impact of model recalibration in diabetes or other conditions.

Cost-utility analyses (CUAs) increasingly use models to predict long-term outcomes and translate trial data to real-world settings. Model structure uncertainty affects these predictions. This study compares pharmacometric against traditional pharmacoeconomic model evaluations for CUAs of sunitinib in gastrointestinal stromal tumors (GIST).

A two-arm trial comparing sunitinib 37.5 mg daily with no treatment was simulated using a pharmacometric-based pharmacoeconomic model framework. Overall, four existing models [time-to-event (TTE) and Markov models] were re-estimated to the survival data and linked to logistic regression models describing the toxicity data [neutropenia, thrombocytopenia, hypertension, fatigue, and hand-foot syndrome (HFS)] to create traditional pharmacoeconomic model frameworks. All five frameworks were used to simulate clinical outcomes and sunitinib treatment costs, including a therapeutic drug monitoring (TDM) scenario.

The pharmacometric model framework predicted that sunitinib treatment costs an additional 142,756 euros per quality adjusted life year (QALY) compared with no treatment, with deviations - 21.2% (discrete Markov), - 15.1% (continuous Markov), + 7.2% (TTE Weibull), and + 39.6% (TTE exponential) from the traditional model frameworks. The pharmacometric framework captured the change in toxicity over treatment cycles (e.g., increased HFS incidence until cycle 4 with a decrease thereafter), a pattern not observed in the pharmacoeconomic frameworks (e.g., stable HFS incidence over all treatment cycles). Furthermore, the pharmacoeconomic frameworks excessively forecasted the percentage of patients encountering subtherapeutic concentrations of sunitinib over the course of time (pharmacoeconomic: 24.6% at cycle 2 to 98.7% at cycle 16, versus pharmacometric: 13.7% at cycle 2 to 34.1% at cycle 16).

Model structure significantly influences CUA predictions. The pharmacometric-based model framework more closely represented real-world toxicity trends and drug exposure changes. The relevance of these findings depends on the specific question a CUA seeks to address.

A new induction therapy strategy of a single 3 mg/kg dose of rabbit antithymocyte globulin (r-ATG) showed a lower incidence of acute rejection.

The objective of this study was to use real-world data to determine the incremental cost-effectiveness ratio (ICER) of r-ATG induction for the prevention of acute rejection (AR) in the first year following kidney transplantation and for kidney graft survival over 1, 4, and 10 years of post-transplantation from the perspective of the national public healthcare system. A Markov state transition model was developed utilizing real-world data extracted from medical invoices from a single center. The study population consisted of adults at low immunological risk undergoing their initial transplantation and received kidneys from either living or deceased donors. The intervention of r-ATG induction was compared to no induction. The clinical outcomes considered for this analysis were acute rejection, cytomegalovirus infection/disease, death, graft loss, and retransplantation.

The cost-effectiveness analysis in the first year revealed that the r-ATG group was more cost-effective, with an ICER of US$ 399.96 per avoided AR episode, an effectiveness gain of 0.01 year in graft survival and a total incremental cost of US$ 147.50. The 4- and 10-year analyses revealed an effectiveness gain of 0.06 and 0.16 years in graft survival in the r-ATG induction group, and a total incremental cost of US$ -321.68 and US$ -2,440.62, respectively.

The single 3 mg/kg dose of r-ATG is cost-effective in preventing acute rejection episodes and dominant in the long term of transplantation, conferring survival gain.

International development agendas increasingly push for access to healthcare for all through universal healthcare coverage. Health economic evaluations and health technology assessment (HTA) could provide evidence to support this but do not routinely incorporate consideration of equitable access.

We undertook an international scoping review of health economic evaluation and HTA guidelines to examine how well issues of healthcare access and equity are represented, evidence recommendations, and gaps in current guidance to support evidence generation in this area. Guidelines were sourced from guideline repositories and websites of international agencies and organizations providing best practice methods guidance. Articles providing methods guidance for the conduct of HTA, or health economic evaluation, were included, except where they were not available in English and a suitable translation could not be obtained.

The search yielded forty-seven national, four international, and nine independent guidelines, along with eighty-six articles providing specific methods guidance. The inclusion of equity and access considerations in current guidance is extremely limited. Where they do feature, detail on specific methods for providing evidence on these issues is sparse.

Economic evaluation could be a valuable tool to provide evidence for the best healthcare strategies that not only maximize health but also ensure equitable access to care for all. Such evidence would be invaluable in supporting progress towards universal healthcare coverage. Clear guidance is required to ensure evaluations provide evidence on the best strategies to support equitable access to healthcare, but such guidance rarely exists in current best practice and guidance documents.

The delivery of healthcare in conflict-affected regions places tremendous strains to health systems, and the economic value of surgical care in conflict settings remains poorly understood. Our aims were to evaluate the cost-effectiveness, societal economic benefits, and return on investment (ROI) for surgical care in a conflict-affected region in Sudan. We conducted a retrospective study of surgical care from January to December 2022 at the Mother of Mercy-Gidel Hospital (MMH) in the Nuba Mountains of Sudan, a semi-autonomous region characterized by chronic and cyclical conflict. We collected data on all patients undergoing surgical procedures (n = 3016), including age, condition, and procedure. We used the MMH budget and financial statements to measure direct medical and non-medical expenditures (costs) for care. We estimated the proportion of expenditures for surgical care through a survey of surgical vs non-surgical beds. The benefits of care were calculated as averted disability-adjusted life-years (DALYa) based on predicted outcomes for the most common 81% of procedures, and then extrapolated to the overall cohort. We calculated the average cost-effectiveness ratio (CER) of care. The societal economic benefits of surgical care were modeled using a human capital approach, and we performed a ROI analysis. Uncertainty was estimated using sensitivity analysis. We found that the CER for all surgical care was $72.54/DALYa. This CER is far less than the gross domestic product per capita in the comparator economy of South Sudan ($585), qualifying it as very cost-effective by World Health Organization standards. The total societal economic impact of surgical care was $9,124,686, yielding a greater than 14:1 ROI ratio. Sensitivity analysis confirmed confidence in all output models. Surgical care in this conflict-affected region of Sudan is very cost-effective, provides substantial societal economic benefits, and a high return on investment.

SHAPE (Simple Hysterectomy And PElvic node assessment) was an international phase III trial demonstrating that simple hysterectomy was non-inferior to radical hysterectomy for pelvic recurrence risk, but superior for quality of life and sexual health. The objective was to conduct a cost-effectiveness analysis comparing simple vs. radical hysterectomy for low-risk early-stage cervical cancer.

Markov model compared the costs and benefits of simple vs. radical hysterectomy for early cervical cancer over a 5-year time horizon. Quality-adjusted life years (QALYs) were estimated from health utilities derived from EQ-5D-3L surveys. Sensitivity analyses accounted for uncertainty around key parameters. Monte Carlo simulation estimated complication numbers according to surgical procedure.

Simple hysterectomy was more effective and less costly than radical hysterectomy. Average overall costs were $11,022 and $12,533, and average gains were 3.56 and 3.54 QALYs for simple and radical hysterectomy, respectively. Baseline health utility scores were 0.81 and 0.83 for simple and radical hysterectomy, respectively. By year 3, these scores improved for simple hysterectomy (0.82) but not for radical hysterectomy (0.82). Assuming 800 early cervical cancer patients annually in Canada, the model estimated 3 vs. 82 patients with urinary retention, and 49 vs. 86 patients with urinary incontinence persisting 4 weeks after simple vs. radical hysterectomy, respectively. Results were most sensitive to variability in health utilities after surgery, but stable through wide ranges of costs and recurrence estimates.

Simple hysterectomy is less costly and more effective in terms of quality-adjusted life expectancy compared to radical hysterectomy for early cervical cancer.

ClinicalTrials.gov Identifier: NCT01658930.

Healthcare decision making, including regulatory and reimbursement decisions, is based on uncertain assessments of clinical and economic value. This arises from the evidence supporting those assessments being uncertain, incomplete, or even absent. Qualitative, structured expert elicitation (SEE) is a valuable tool for extracting expert knowledge about an uncertain quantity and formulating that knowledge as a probability distribution. This creates a useful input to decision modeling and support, particularly in areas with limited evidence, such as advanced therapy products, precision medicine, rare diagnoses, and other areas with high uncertainty. Structured SEE protocols are used to improve the transparency, accuracy, and consistency of quantitative judgments from experts, limiting the effect of heuristics and biases. This task force report introduces 5 commonly used protocols for SEE (Sheffield elicitation framework; modified Delphi method; Cooke's classical method; investigate, discuss, estimate, aggregate protocol; and the Medical Research Council reference protocol). It describes the common elements of SEE, discusses how these protocols differ in their implementation of those elements and illustrates the use of the protocols. The report then reviews the relevant constraints on implementing SEE within the context of healthcare decision making and considers the strengths and weaknesses of these protocols in light of those considerations. Because this is an introductory report on an emerging topic, specific recommendations on practice are not made. However, there are broad recommendations based on the suitability of the different protocols in various decision contexts. The report concludes with recommendations for further research to better guide future practice.

The opioid epidemic has severely impacted the US over the last 15 years. Buprenorphine is a partial opioid agonist indicated for the treatment of opioid use disorder (OUD) and is recognized as an effective treatment when taken as prescribed. However, adherence rates have been low in real-world settings. Blister-packaging has been shown to promote medication adherence across a variety of disease states, although it has never been studied in OUD.

An economic analysis was conducted to assess the impact of increased adherence of blister-packaged buprenorphine on health care resource utilization (HCRU) and health care costs for 10,000 patients initiating therapy for OUD. The model analyzed a commercially insured population within the US over a one-year time horizon. Medication adherence was defined in the model as proportion of days covered (PDC) of at least 80%. Literature-based references were used to inform both the impact of blister-packaging on the number of patients who became adherent as well as the impact of medication adherence on HCRU and health care costs. Model input uncertainty was assessed in one-way sensitivity analyses.

With the implementation of blister-packaging buprenorphine, adherence rates increased from 37.1% of patients in the pre-intervention period to 45.3%, resulting in an additional 818 patients becoming adherent post-intervention. The increase in adherence led to a reduction of medical costs of $12,138,757 (-$1,214 per-patient (PP)). Specifically, inpatient costs decreased by $7,127,073 (-$713 PP) while outpatient costs decreased by $5,013,319 (-$501 PP). Pharmacy costs increased by $3,432,705 ($343 PP). Despite the increase in pharmacy costs, total health care costs saw a reduction of $8,559,684 (-$856 PP).

Blister-packaging buprenorphine for treatment of OUD has potential to improve medication adherence and health outcomes while reducing HCRU and health care costs. Future studies are necessary to assess the real-world application and impact of blister-packaging buprenorphine for OUD across various patient populations and health care settings.

Host-response-based transcriptional signatures (HrTS) have been developed to identify "incipient tuberculosis (TB)". No study has reported the cost-effectiveness of HrTS for post-arrival migrant screening programs in low-incidence countries.

To assess the potential health impact and cost-effectiveness of HrTS for post-arrival TB infection screening among new migrants in the United States.

We used a discrete-event simulation model to compare four strategies: (1) no screening for TB infection or incipient TB; (2) 'IGRA-only', screen all with interferon gamma release assay (IGRA), provide TB preventive treatment for IGRA-positives; (3) 'IGRA-HrTS', screen all with IGRA followed by HrTS for IGRA-positives, provide incipient TB treatment for individuals testing positive with both tests; and (4) 'HrTS-only', screen all with HrTS, provide incipient TB treatment for HrTS-positives. We assessed outcomes over the lifetime of migrants entering the U.S. in 2019, assuming HrTS met the WHO Target Product Profile (TPP) optimal criteria. We conducted sensitivity analyses to evaluate the robustness of results.

The IGRA-only strategy dominated the HrTS-based strategies under both healthcare sector and societal perspectives, with an incremental cost-effectiveness ratio of $78,943 and $89,431 per quality-adjusted life-years (QALY) gained, respectively. This conclusion was robust to varying costs ($15-300) and characteristics of HrTS, and the willingness-to-pay threshold ($30,000-150,000/ QALY gained), but sensitive to the rate of decline in TB progression risk after U.S. entry.

Our findings suggest that HrTS meeting the WHO TPP is unlikely to be a cost-effective component of post-arrival screening for migrants entering the U.S.

The Mount Hood Diabetes Challenge Network aimed to examine the impact of model structural uncertainty on the estimated cost-effectiveness of interventions for type 2 diabetes.

Ten independent modeling groups completed a blinded simulation exercise to estimate the cost-effectiveness of 3 interventions in 2 type 2 diabetes populations. Modeling groups were provided with a common baseline population, cost and utility values associated with different model health states, and instructions regarding time horizon and discounting. We collated the results to identify variation in predictions of net monetary benefit (NMB) and the drivers of those differences.

Overall, modeling groups agreed which interventions had a positive NMB (ie, were cost-effective), Although estimates of NMB varied substantially-by up to £23 696 for 1 intervention. Variation was mainly driven through differences in risk equations for complications of diabetes and their implementation between models. The number of modeled health states was also a significant predictor of NMB.

This exercise demonstrates that structural uncertainty between different health economic models affects cost-effectiveness estimates. Although it is reassuring that a decision maker would likely reach similar conclusions on which interventions were cost-effective using most models, the range in numerical estimates generated across different models would nevertheless be important for price-setting negotiations with intervention developers. Minimizing the impact of structural uncertainty on healthcare decision making therefore remains an important priority. Model registries, which record and compare the impact of structural assumptions, offer one potential avenue to improve confidence in the robustness of health economic modeling.

The strategies to control scabies in highly endemic populations include individual case/household management and mass drug administration (MDA). We used a decision-analytic model to compare ivermectin-based MDA and individual case/household management (referred to as "usual care") for control of scabies in Ethiopia at different prevalence thresholds for commencing MDA.

A decision-analytic model was based on a repeated population survey conducted in Northern Ethiopia in 2018-2020, which aimed to evaluate the secondary impact of single-dose ivermectin MDA for the control of onchocerciasis on scabies prevalence. The model estimates the number of scabies cases and costs of two treatment strategies (MDA and usual care) based on their effectiveness, population size, scabies prevalence, compliance with MDA, medication cost, and other parameters.

In the base-case analysis with a population of 100,000 and scabies prevalence of 15%, the MDA strategy was both more effective and less costly than usual care. The probability of MDA being cost-effective at the current cost-effectiveness threshold (equivalent to the cost of usual care) was 85%. One-way sensitivity analyses showed that the MDA strategy remained dominant (less costly and more effective) in 22 out of 26 scenarios. MDA was not cost-effective at scabies prevalence <10%, MDA effectiveness <85% and population size <5,000. An increase in the cost of ivermectin from 0 (donated) to 0.54 US$/dose resulted in a decrease in the probability of MDA being cost-effective from 85% to 17%. At 0.25 US$/dose, the MDA strategy was no longer cost-effective.

The model provides robust estimates of the costs and outcomes of MDA and usual care and can be used by decision-makers for planning and implementing scabies control programmes. Results of our analysis suggest that single-dose ivermectin MDA is cost-effective in scabies control and can be initiated at a scabies prevalence >10%.

Colorectal cancers (CRCs) arise from adenomas, which can produce fecal occult blood and can be detected endoscopically, or sessile serrated lesions (SSLs), which rarely bleed and may be more challenging to detect. Models informing CRC screening policy should reflect both pathways, accounting for uncertainty.

Novel decision-analytic model of the adenoma and serrated pathways for CRC (ANSER) to compare current and emerging screening strategies, accounting for differential test sensitivities for adenomas and SSLs, and uncertainty. Strategies included colonoscopy every 10 years, stool-DNA/FIT (sDNA-FIT) every 1-3 years, or fecal immunochemical testing (FIT) every year from age 45 to 75 years. Outcomes included CRC cases and deaths, cost-effectiveness (cost/quality-adjusted life-year [QALY] gained), and burden-benefit (colonoscopies/life-year gained), with 95% uncertainty intervals (UIs).

ANSER predicted 62.5 (95% UI = 58.8-66.3) lifetime CRC cases and 24.1 (95% UI = 22.5-25.7) CRC deaths/1000 45-year-olds without screening, and 78%-87% CRC mortality reductions with screening. The tests' outcome distributions overlapped for QALYs gained but separated for required colonoscopies and costs. All strategies cost less than $100 000/QALY gained vs no screening. Colonoscopy was the most effective and cost-effective, costing $9300/life-year gained (95% UI = $500-$21 900) vs FIT. sDNA-FIT cost more than $500 000/QALY gained vs FIT. As more CRCs arose from SSLs, colonoscopy remained preferred based on clinical benefit and cost-effectiveness, but cost-effectiveness improved for a next-generation sDNA-FIT.

When the serrated pathway is considered, modeling suggests that colonoscopy is cost-effective vs FIT. In contrast, modeling suggests that sDNA-FIT is not cost-effective vs FIT despite its greater sensitivity for SSLs, even if a substantial minority of CRCs arise from SSLs.

Individuals who were formerly incarcerated have high tuberculosis incidence, but are generally not considered among the risk groups eligible for tuberculosis prevention. We investigated the potential health impact and cost-effectiveness of Mycobacterium tuberculosis infection screening and tuberculosis preventive treatment (TPT) for individuals who were formerly incarcerated in Brazil.