ClinicoEconomics and Outcomes Research最新文献

Background: Acute kidney injury (AKI) is common among patients admitted to the intensive care unit (ICU), with 5-15% receiving renal replacement therapy (RRT). Continuous renal replacement therapy (CRRT) and intermittent hemodialysis (IHD) are well-established treatments for severe AKI, but renal recovery is variable and often incomplete, leading to long-term morbidity and mortality. The clinical and cost-effectiveness of either therapy are under active debate. This analysis aims to strengthen the evidence on the cost-utility of CRRT compared with IHD to manage severe AKI in ICU using a US third-party costing perspective.

Methods: The analysis used a 90-day decision tree simulating hospital admission and a semi-Markov process with annual cycles and half-cycle correction to capture lifetime costs and outcomes, discounted at 3% annually. Survivors at 90 days either progressed to ESKD on dialysis (ESKD-D), with some receiving transplants, or became dialysis-independent. In the case of transplant failure, patients returned to ESKD-D. Tunnel states addressed Markov memoryless properties. A US-representative analysis of real-world data applying propensity score matching to control for selection bias informed the probability of lifetime dialysis dependence. Costs and utilities were sourced from peer-reviewed publications or national data. Uncertainty was investigated using deterministic and probabilistic sensitivity analyses.

Results: In the base case, lifetime costs and quality-adjusted life-years (QALYs) were $273,314 and 5.681 for CRRT compared to $268,449 and 5.457 for IHD. CRRT had an 89.6% probability of being cost-effective ($23,860/QALY gained), being associated with 0.269 additional life-years. Long-term CKD management costs, accounting for 50% of CRRT's excess costs, significantly influenced results and were examined in scenario analyses.

Conclusion: CRRT is likely a cost-effective option for managing severe AKI in the ICU compared with IHD. This study builds on existing economic evaluations by incorporating large comparative studies and exploring clinical uncertainty. The model highlights the need to clarify RRT's role in CKD progression and enhance post-AKI care to improve patient outcomes.

Purpose: To assess the cost-effectiveness of biologics in patients with axial spondyloarthritis (axSpA) in a real-world setting.

Patients and methods: This is a non-interventional, registry-based, prospective cohort study included 203 consecutive patients with axSpA who attended the Rheumatology Department of a public hospital in the Emirate of Dubai between July 2018 and September 2020. Demographic and clinical data were collected and disease activity and treatment response were assessed. Patients were grouped according to the treatment received, distinguishing between biologics and non-biologics, and classified them based on their disease activity at baseline and 52 and 104 weeks. The direct and indirect costs associated with their management were collected. The incremental cost-effectiveness ratio (ICER) was calculated and the impact of patient-related factors on its value across subgroups was examined. A state-transition model was used to simulate disease progression across four health states over a lifetime (62 years), and Monte Carlo simulation was applied to address uncertainty.

Results: The total cost of managing the patients was AED 27,532,189, resulting in a gain of 293.7 quality-adjusted life years (QALYs) over a 2-year follow-up period. Biological therapies were associated with higher direct costs, accounting for 83.3% of the total costs of biologics. The overall ICER was AED 253,616 per QALY, influenced by higher medication acquisition costs. Patients with < 5 years of disease duration, female patients, those with radiographic-axSpA, co-existing fibromyalgia, and those receiving a combination of biologic treatments and conventional disease-modifying antirheumatic drugs showed a higher ICER than the median ICER of all biologic therapies.

Conclusion: Biologic therapies are cost-effective for patients with axSpA, especially those not achieving clinical targets. Patients' selection, and targeted cost-containment strategies, such as biosimilars and medication price reductions, can enhance clinical benefits and reduce societal costs.

Background and objectives: Global demand for health economics/pharmacoeconomics (HE/PE) has significantly increased, leading to a greater need for competent and skilled healthcare professionals in this field. However, the current literature indicates a lack of research regarding HE/PE among healthcare professionals, including those in the Kingdom of Saudi Arabia (KSA), particularly among registered nurses. Hence, this study assessed registered nurses' levels of awareness and knowledge of HE/PE and their sociodemographic and HE/PE-related predictors.

Methods: A descriptive-correlational, cross-sectional design was used. The study was conducted on a convenience sample of 324 registered nurses working at the three hospitals within King Saud University Medical City, Riyadh, KSA. Data were collected from May 2025 to June 2025, and analyzed using SPSS v.30. Binary logistic regression analyses were conducted for HE/PE awareness, and multiple linear regression analyses were performed for knowledge about HE/PE. The significance level was established at p <0.05.

Results: Registered nurses demonstrated an overall low level of knowledge (2.39/5), while showing a good level awareness about HE/PE across four awareness items (52.8% to 84.6%) but suboptimal for the remaining two items (38.6% and 38.9%). Several sociodemographic and HE/PE-related factors were identified as significant predictors of the registered nurses' awareness and knowledge regarding HE/PE. Notably, four out of the six awareness items: "have heard about HE/PE" (p =0.040), "read articles related to HE/PE regularly" (p <0.001), "participate in HE/PE decisions at work" (p <0.042), and "implement HE/PE techniques to make decisions" (p <0.001) were revealed as significant predictors of registered nurses' knowledge about HE/PE.

Conclusion: The findings indicate that the knowledge level regarding HE/PE among registered nurses was low; nevertheless, their awareness was acceptable. It is crucial to develop and implement targeted educational strategies and interventions aimed at enhancing both awareness and knowledge about HE/PE among registered nurses in the KSA, while meticulously considering pertinent sociodemographic and HE/PE-related factors.

The global burden of end-stage kidney disease (ESKD) is rising, compelling patients to increasingly confront the choice of initiating kidney replacement therapy (KRT). Cost-utility analysis (CUA) has the advantage of incorporating patient-reported outcomes regarding KRT. The aim of this study is to summarize the CUA of hemodialysis (HD), peritoneal dialysis (PD), and kidney transplantation in patients with ESKD. A systematic search was conducted to identify relevant articles in three primary databases (PubMed, SCOPUS, and ProQuest). CUA studies of dialysis for ESKD patients from 2000 to 2023 in all countries were included. Non-full-text, non-English language, review articles, systematic reviews, and studies that did not compare distinct dialysis methods were excluded. All the information was summarized narratively. Out of the 130 studies identified, 13 met the inclusion criteria and were included in this review. Most studies demonstrated good reporting quality, with CHEERS checklist scores ranging from 75% to 96%. Kidney transplantation was consistently found to be the most cost-effective KRT, offering the highest quality-adjusted life years (QALYs) and the lowest cost per QALY in the long term, despite higher initial expenses. PD showed better cost-effectiveness than HD in several studies. HD generally incurred the highest costs with lower QALY gains. Cost-effectiveness trends varied depending on regional context, healthcare perspective, and patient comorbidities. This study found that kidney transplantation is consistently the most cost-effective kidney replacement therapy, with lower cost per QALY and improved quality-adjusted life years (QALY) in most settings. In particular, transplantation showed favorable long-term outcomes despite higher initial costs, whereas peritoneal dialysis emerged as a more cost-effective alternative to hemodialysis, especially in resource-limited settings. These findings offer practical implications for treatment prioritization and policy-making in both high- and low-resource countries.

Purpose: Malnutrition in cancer patients may stem from both tumor progression and treatment regimens. Nutritional interventions have shown potential for enhancing treatment efficacy and overall quality of life, but their cost-effectiveness requires exploration. Accordingly, this study systematically investigated research on economic evaluations of nutritional interventions implemented as either preventive or therapeutic strategies in cancer patients.

Methods: PubMed and Scopus databases were searched from inception to September 9, 2025. Following study selection and data extraction, the reporting quality of included studies was evaluated using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist, while risk of bias was assessed using the ECOBIAS checklist.

Results: Eight studies, published between 1989 and 2022, primarily focused on gastrointestinal cancers and were conducted in high-income countries. Most of these investigations (6 out of 8) performed cost-utility analyses. Four indicated that combining oral supplements or parenteral nutrition with nutritional counseling was more cost-effective, although these studies differed in terms of time horizons, types of interventions evaluated, and types of comparators used. Most of the studies adhered to the CHEERS standards, but no reported on characterizing heterogeneity, and none discussed approaches to stakeholder involvement in study design. Based on the ECOBIAS framework, the three most frequently encountered risks of bias were limited sensitivity analysis bias, bias related to quality-of-life weights, and limited scope bias.

Conclusion: The findings offer physicians valuable guidance for optimizing treatments and potentially support policy decision-making.

Purpose: Uganda's digital health transformation anchored on District Health Information Software 2 (DHIS2) and the FAIR (Findable, Accessible, Interoperable, Reusable) Data Principles has reshaped health data governance. Nevertheless, systemic constraints in privacy, infrastructure, and human resources threaten sustainability and equity.

Objective: To compare DHIS2 and FAIR-based approaches on (i) privacy protection, (ii) interoperability and data usability, and (iii) regulatory/institutional readiness for privacy-preserving health data analytics in Uganda.

Methods: Systematic review of 84 peer-reviewed and grey-literature sources (2010-2025) following PRISMA 2020; extracted indicators on reuse, interoperability, privacy, and institutional readiness.

Results: 36% of included studies were Uganda-specific; 50% were published in 2020-2024. DHIS2 reached near-national coverage, ~12,000 trained users, and integration across >20 programmes. Persistent gaps include limited rural internet (≈12% of facilities with stable connectivity), high staff turnover (~35%), and low analytics literacy (~25% with intermediate skills). FAIR efforts (since ~2019) remain early: ~10% of institutions with formal policies; low dataset reuse (~22%), machine-readable metadata (~18%), and documented digital consent (<10%). Privacy infrastructure is weak: <30% of facilities with formal privacy frameworks/secure platforms and <10% with Data Protection Officers.

Conclusion: DHIS2 improved reporting and availability, while FAIR initiatives began enabling governed, interoperable reuse. To achieve ethical analytics at scale, priorities are legal enforcement, secure rural ICT, standardized machine-readable metadata/consent, and workforce development.

Purpose: Three prenatal screening protocols are currently used in Thailand to prevent severe thalassemia in newborns; however, economic evaluation data to guide the selection of the most cost-effective strategy remain limited. This study aimed to conduct the cost-effectiveness of these screening strategies to support policy and clinical decision-making.

Patients and methods: A decision-tree model was employed to evaluate the costs and outcomes associated with three prenatal screening algorithms: 1) a protocol starting with mean corpuscular volume (MCV), mean corpuscular hemoglobin (MCH), and the dichlorophenol indophenol precipitation (DCIP) test for pregnancies; 2) a protocol starting with MCV/MCH and hemoglobin (Hb) typing for pregnancies; and 3) a protocol starting with MCV/MCH and DCIP testing for couples based on a societal perspective. Results were presented as incremental cost-effectiveness ratios (ICERs) to compare the three prenatal screening strategies. Both one-way and probabilistic sensitivity analyses were performed to account for uncertainties in the parameters used.

Results: From a societal perspective, Strategies 2 and 3 were found to be cost-effective compared to Strategy 1. Screening 10,000 couples under Strategies 2 and 3 could prevent 60 and 49 additional cases of severe thalassemia, respectively, compared to Strategy 1. The ICERs for Strategies 2 and 3 were approximately 141,863 Baht (4,023 United States Dollar, USD) and 97,996 Baht (2,779 USD) per severe thalassemia case prevented, respectively. The most sensitive parameter for comparing Strategy 2 with Strategy 1 was the specificity of prenatal diagnosis for α-thalassemia.

Conclusion: Strategy 3 is the most cost-effective approach in Thailand. However, Strategy 2 may be implemented in hospitals with the capacity to perform Hb typing. These results offer significant value to policymakers by presenting strong evidence that could inform adjustments to the reimbursement framework in Thailand's Universal Health Coverage benefit package, ultimately enhancing thalassemia management and prenatal care.

Purpose: The study investigated the potential association between commercial blenderized tube feeding formulas (CBTF) or plant-based standard tube feeding formulas (STD-TF), and healthcare resource utilization (HCRU) and costs, in children and adults receiving enteral nutrition (EN) in a post-acute care setting.

Patients and methods: United States insurance claims were retrospectively analyzed to evaluate HCRU and costs for visits to emergency department (ED), inpatient, outpatient, urgent care, and other services at 12 weeks post hospital discharge. Between-cohort comparisons were performed for proportions of patients with claims for each place of service, mean number of visits, and unadjusted and adjusted costs.

Results: Significantly fewer children receiving CBTF vs plant-based STD-TF required inpatient services (16% vs 35%), urgent care (3% vs 10%), ED (15% vs 28%), and other facilities (10% vs 22%) (p < 0.001 for each comparison), with a lower mean number of visits to any healthcare service reported (28 vs 96; p < 0.001). In adults, significantly fewer patients in the CBTF cohort vs the plant-based STD-TF cohort required inpatient (23% vs 38%; p = 0.003) and other services (13% vs 27%; p < 0.001), with a corresponding lower mean number of visits to any healthcare service (24 vs 79; p < 0.001). Total unadjusted costs were significantly lower in both children and adults receiving CBTF compared with plant-based STD-TF (children: $140,962 vs $1,060,345; adults: $166,591 vs $820,905; each p < 0.001). Cost savings associated with CBTF were maintained when costs were adjusted for age, sex, and Charlson's comorbidity index. Children and adults receiving CBTF had significantly lower total adjusted costs compared with those receiving plant-based STD-TF (children: $222,735 vs $965,451; adults: $258,460 vs $919,060 each p ≤ 0.001).

Conclusion: The use of CBTF was associated with lower HCRU and costs both in children and adults receiving EN in post-acute care settings. To our knowledge, this is the first large real-world study comparing these formulas in post-acute care across both populations. Although the retrospective design and lack of adjustment for all potential confounders preclude conclusions about causation, these findings may guide formula selection based on potential economic impact.

Introduction: The World Health Organization (WHO) has advocated for multiple micronutrient supplementation (MMS) for pregnant women since 2016 to mitigate the risk of various complications. As a country considering investment in MMS, Indonesia requires an assessment of the cost-effectiveness analysis and priority setting in the transition from IFA to MMS, which is essential in the decision-making process.

Methods: An open-access online modelling tool was used to estimate the cost-effectiveness of MMS (ICERs per DALY averted) at national and sub-national levels (38 provinces) in Indonesia. The results were then prioritized via a cost-effectiveness league table, with a deterministic sensitivity analysis testing the robustness of the results.

Results: Implementing MMS under 44% and 100% coverage scenarios has the potential to avert 54,897 and 124,766 DALYs in Indonesia, respectively, and produced an equivalent incremental cost-effectiveness ratio (ICER) value of USD 10 per DALY averted, which is regarded as highly cost-effective. Since costs and benefits scale linearly with coverage, ICER per DALY averted stays unchanged. The MMS program is recommended to be prioritized in 18 provinces having favorable ICERs, which are Southwest Papua, Highlands Papua, Bali, West Java, South Kalimantan, North Maluku, West Papua, Aceh, North Sumatra, Central Java, Central Kalimantan, West Sulawesi, Maluku, Yogyakarta, East Java, North Kalimantan, South Sulawesi, and South Papua. Related to the sensitivity analysis, the cost of MMS and IFA are the most significant variables influencing the ICER value.

Conclusion: The findings show that the implementation of MMS, in comparison to IFA, produces a highly cost-effective outcome in 44% and 100% scenarios. The MMS implementation strategy may initiate with a 44% coverage scenario across 18 prioritized provinces, subsequently expanding to achieve 100% coverage for all pregnant women in Indonesia.

Purpose: The EuroQoL-five dimensions-three levels (EQ-5D-3L) utility values vary across regions, hence become non-interchangeable. This study details a methodology to adjust EQ-5D-3L utility values from the published United States (US) scores for use in the United Kingdom (UK) when individual-level patient data (IPD) are inaccessible.

Methods: Linear and polynomial regression models were fitted to map the US-derived EQ-5D-3L health state utility weights (HSUWs) into UK values. Model fit was evaluated via visual inspection and coefficient of determination (R ²). The best-fit model was used to develop a utility-adjustment algorithm. Data for external validation of the algorithm were collected through a targeted literature review, which identified studies reporting mean EQ-5D-3L utilities from both the US and UK. The coefficients of the best-fit model were validated against the published mean US and UK utilities, and the algorithm was validated by comparing published and predicted mean UK utilities.

Results: The linear model confirmed linearity between US and UK HSUWs, and was the best-fit over the polynomial model, both visually and with R ² (0.9574 vs 0.9576). Eleven publications (N = 93,625 participants) reporting the mean US and UK EQ-5D-3L utilities based on the valuation of EQ-5D-3L responses were included for validation. The linearity between the published mean US and UK EQ-5D-3L utilities was deemed valid (R ² = 0.9955) with similar slopes (1.45 vs 1.39) and intercepts (-0.43 vs -0.38) between the linear model and the published utility-based line. The algorithm (Utility^UK = [-0.3813 + 1.3904 × Utility^US]) predicted the mean UK utilities, which marginally differed from the reported mean UK utilities, confirming its validity.

Conclusion: A robust method was developed to adjust US-derived EQ-5D-3L utilities and reflect UK preferences. This method helps predict and transpose utilities from the literature across countries as a supporting tool when IPD are unavailable, which is necessary when adapting cost-effectiveness models to different settings.