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Abstract Background The morbidity and mortality of adult diseases caused by S. pneumoniae increase with age and presence of underlying chronic diseases. Currently, two vaccine technologies against S. pneumoniae are used: the 23-valent pneumococcal polysaccharide vaccine (PPV23) and the pneumococcal conjugate vaccines, one of which is the 20-valent pneumococcal conjugate vaccine (PCV20) that has recently been approved for adults. Objective This study was conducted to investigate the cost-effectiveness of implementing PCV20 in a reimbursement scheme for Norwegian adults aged 18–99 years at risk of pneumococcal diseases and those aged 65 years and older at low risk compared to PPV23. Methods An established Markov model was adapted to a Norwegian setting to estimate the economic and clinical consequences of vaccinating the Norwegian population in specific age and risk groups against pneumococcal diseases. Inputs for the model were found in Norwegian or Danish real-world evidence or retrieved from available studies. The costs and clinical outcomes were assessed using a health sector perspective and a lifetime time horizon. Results The results showed that PCV20 was associated with better health outcomes including fewer disease cases, fewer disease-attributable fatalities, a higher gain of life years and quality-adjusted life years compared to PPV23. In addition, PCV20 had a lower total cost compared to PPV23. Therefore, PCV20 was the dominant vaccination strategy. The base case result was investigated in multiple sensitivity analyses, which showed that the results were robust to changes in input parameters and methodological assumptions, as PCV20 remained the dominant vaccination strategy in almost all scenarios. Conclusion Results showed that vaccinating the Norwegian adults with PCV20 was cost-effective compared to PPV23. Changes in the hospital cost of pneumonia, the price of PCV 20, the effectiveness of PCV20 against pneumonia, and the pneumonia disease incidence had the highest impact on the ICER, i.e., were the main drivers of the results.

Abstract Aims The DELIVER study demonstrates a significant improvement in cardiovascular death or hospitalization for heart failure among heart failure with mildly reduced ejection fraction (HFmrEF) or heart failure with preserved ejection fraction (HFpEF).Cost‐utility of the adjunct use of dapagliflozin to standard therapy among patients with HFpEF or HFmrEF remains unclear. Methods and results A five‐state Markov mode was constructed to project health and clinical outcomes of the adjunct use of dapagliflozin to standard therapy among 65‐year‐old patients with HFpEF or HFmrEF. A cost‐utility analysis was performed based on the DELIVER study and national statistical database. The cost and utility was inflated to 2022 by the usual discount rate of 5%. The primary outcomes were total cost and quality‐adjusted life‐years (QALYs) per patients as well as the incremental cost‐effectiveness ratio. Sensitivity analyses were also applied. Over a 15 year lifetime horizon, the average cost per patient was $7245.77 and $5407.55 in the dapagliflozin group and the standard group, along with an incremental cost of $1838.22. The average QALYs per patient was 6.00 QALYs and 5.84 QALYs in the dapagliflozin group and the standard group, along with an incremental QALYs of 0.15 QALYs, resulting in the incremental cost‐effectiveness ratio of $11 865.33/QALY, which was below the willingness‐to‐pay (WTP) of $12 652.5/QALY. The univariate sensitivity analysis indicated the cardiovascular death in both group was the most sensitive variable. Probability sensitivity analysis revealed that when the WTP thresholds were $12 652.5/QALY and $37 957.5/QALY, the probabilities of being cost‐effective with dapagliflozin as an add‐on were 54.6% and 71.6%, respectively. Conclusions From a public healthcare system perspective, the adjunct use of dapagliflozin to standard therapy among patients with HFpEF or HFmrEF generated advantages in cost‐effectiveness in China at a WTP of $12 652.5/QALY, which promoted the rational use of dapagliflozin for heart failure.

Abstract Background and Aims Coronavirus has burdened considerable expenditures on the different health systems. Vaccination programs, the critical solution against pandemic diseases, are known as safe and effective interventions to prevent and control epidemics. We aimed to perform a systematic review to provide economic evidence of the value of different types of vaccines available to combat the Covid‐19 to all health policymakers worldwide. Methods Electronic searches conducted on Medline/PubMed, Cochrane Library, Web of Science, Scopus, Embase, and other economic evaluation databases. Related and published articles searched up to March 2022 by using keywords such as “Vaccination,” “Covid‐19,” “Cost‐benefit,” “Cost‐utility,” “Cost‐effectiveness,” “Economic Assessment,” and “Economic evaluation.” Followed by choosing the most suitable articles according to inclusion and exclusion criteria, data captured and the results extracted. The quality assessment of the articles performed by the checklist of CHEERS 2022. Finally, 13 articles included in the review. Results All messenger RNA vaccines were dominant with approximately 70% coverage against no vaccination in the primary vaccination program except in one study that looked at booster effects. From a payer's perspective, a dollar invested in a vaccine would be less profitable than from a societal perspective. Therefore, primary mass vaccination can be considered a cost‐effective intervention in primary vaccination to save more lives and produce more positive externalities. However, the cost‐benefit ratio for all vaccines increases when statistical lifetime value and global economic and educational disadvantages are considered. Conclusion The COVID‐19 primary vaccination programs in regional outbreaks, from a long‐term perspective, will demonstrate substantial cost‐effectiveness. It is suggested that due to the positive externalities of vaccination, primary mass vaccination, with the help of COVAX‐19TM, could be considered a reliable way to combat viral epidemics compared to the loss of individual lives and economic and educational disturbances around the world.

Objective: To perform an economic evaluation on a multicomponent intervention programme for patients with fibromyalgia syndrome compared with usual clinical practice in primary care. Design: A cost-utility analysis was conducted alongside a pragmatic randomised controlled trial (ClinicalTrials.gov: https://clinicaltrials.gov/ct2/show/record/NCT04049006) from a societal perspective, a human capital approach, and a 1-year time horizon. Patients: Patients diagnosed with fibromyalgia syndrome from the public health system in south Catalonia, Spain. Methods: Crude and adjusted incremental cost- utility ratios were estimated to compare the treatment strategies based on cost estimations (direct medical costs and productivity losses) and quality-adjusted life years. One-way and 2-way deterministic sensitivity analyses were performed. Results: The final analysed sample comprised 297 individuals, 161 in the intervention group and 136 in the control group. A crude incremental cost-utility ratio of € 1,780.75 and an adjusted ratio of € 851.67 were obtained, indicating that the programme significantly improved patients’ quality of life with a cost-increasing outcome that fell below the cost-effectiveness threshold. The sensitivity analysis confirmed these findings when varying large cost components, and showed dominance when increasing session attendance. Conclusion: The proposed multicomponent intervention programme was cost-effective compared with usual care for fibromyalgia, which supports its addition to standard practice in the regional primary care service.

ABSTRACTBackground The evidence base of tisagenlecleucel is uncertain.Objective To evaluate the cost-effectiveness of tisagenlecleucel. To conduct expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses.Study Design A three-state partitioned survival model. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 5 years; general population mortality with a standardised mortality ratio was then applied. EVPI and EVPPI were scaled up to population according to the incidence of the decision.Setting Irish healthcare payer.Participants Patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL).Interventions Tisagenlecleucel versus Salvage Chemotherapy (with or without haematopoietic stem cell transplant).Main Outcome Measure Incremental cost-effectiveness ratio (ICER). Population EVPI and EVPPI.Results At list prices, the ICER was €119,509 per quality-adjusted life year (QALY) (incremental costs €218,092; incremental QALYs 1.82). Probability of cost-effectiveness, at a €45,000 per QALY threshold, was 0%. Population EVPI was €0.00. Population EVPI, at the price of tisagenlecleucel that reduced the ICER to €45,000 per QALY, was €3,989,438. Here, survival analysis had the highest population EVPPI (€1,128,053).Conclusion Tisagenlecleucel is not cost-effective, versus salvage chemotherapy (with or without haematopoietic stem cell transplant), for R/R DLBCL in Ireland. At list prices, further research to decrease decision uncertainty may not be of value.

Objectives: As the initial crisis of the COVID-19 pandemic recedes, healthcare decision makers are likely to want to make rational evidence-guided choices between the many interventions now available. We sought to update a systematic review to provide an up-to-date summary of the cost-effectiveness evidence regarding tests for SARS-CoV-2 and treatments for COVID-19. Methods: Key databases, including MEDLINE, EconLit and Embase, were searched on 3 July 2023, 2 years on from the first iteration of this review in July 2021. We also examined health technology assessment (HTA) reports and the citations of included studies and reviews. Peer-reviewed studies reporting full health economic evaluations of tests or treatments in English were included. Studies were quality assessed using an established checklist, and those with very serious limitations were excluded. Data from included studies were extracted into predefined tables. Results: The database search identified 8,287 unique records, of which 54 full texts were reviewed, 28 proceeded for quality assessment, and 15 were included. Three further studies were included through HTA sources and citation checking. Of the 18 studies ultimately included, 17 evaluated treatments including corticosteroids, antivirals and immunotherapies. In most studies, the comparator was standard care. Two studies in lower-income settings evaluated the cost effectiveness of rapid antigen tests and critical care provision. There were 17 modelling analyses and 1 trial-based evaluation. Conclusion: A large number of economic evaluations of interventions for COVID-19 have been published since July 2021. Their findings can help decision makers to prioritise between competing interventions, such as the repurposed antivirals and immunotherapies now available to treat COVID-19. However, some evidence gaps remain present, including head-to-head analyses, disease-specific utility values, and consideration of different disease variants. [ABSTRACT FROM AUTHOR]

Background: Palbociclib and Ribociclib are cyclin-dependent kinase 4/6 oral molecular inhibitors that have the potential to improve overall survival (OS), progression-free survival (PFS), and quality of life in patients with metastatic breast cancer (MBC). The objective of this study was to analyze the cost-utility of Palbociclib and Ribociclib in comparison with Letrozole monotherapy as the first-line treatment for hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2–) MBC patients in Iran. Methods: A Cost-Utility Analysis (CUA) was conducted using a partitioned survival model (PSM) from the perspective of the Iranian healthcare system. The comparative strategies considered were Palbociclib + Letrozole, Ribociclib + Letrozole, and Letrozole monotherapy. The model was structured with a 1-month cycle length and a 15-year time horizon. Clinical safety, efficacy, and survival data in terms of PFS and OS for Palbociclib + Letrozole and Ribociclib + Letrozole were obtained from the latest updates of the PALOMA-1, 2, and MONALEESA-2 studies, respectively. Direct medical costs, including drug costs, visits, hospitalization, CT scans, bone x-rays, monitoring and laboratory testing, as well as medication side effects, were considered. Uncertainty evaluations were performed through deterministic sensitivity analysis and probabilistic sensitivity analysis. Excel 2016 and TreeAge 2020 were used for all stages of the evaluation. Results: The base case results indicated that, despite its lower effectiveness, Letrozole monotherapy was the most cost-effective strategy, while Palbociclib + Letrozole and Ribociclib + Letrozole were not cost-effective. The incremental cost-effectiveness ratios (ICERs) for Palbociclib + Letrozole and Ribociclib + Letrozole compared to Letrozole monotherapy were estimated at $137,302 and $120,478 per quality-adjusted life-year (QALY), respectively, which exceeded the target threshold of $4565. Deterministic sensitivity analysis demonstrated that the CUA results were not sensitive to changes in the values of uncertain variables. Probabilistic sensitivity analysis also indicated that Palbociclib + Letrozole and Ribociclib + Letrozole had no chance of being cost-effective based on changes in various parameters and simulations. Conclusions: Palbociclib and Ribociclib showed significant efficacy in combination with Letrozole, as evidenced by improvements in PFS. However, in the first-line treatment of MBC in Iran, these strategies were not cost-effective compared to Letrozole monotherapy. [ABSTRACT FROM AUTHOR]

Simple Summary: There are various chemotherapy regimens used to treat patients diagnosed with stage III colorectal cancer, one of which is an oral chemotherapy drug called "capecitabine". Our study examined the cost-effectiveness of eight chemotherapy regimens using a Markov model. The analysis was performed from a societal perspective with a lifetime time horizon. The results demonstrated that the most cost-effective strategy for treating patients is to begin with a chemotherapy regimen that includes capecitabine and oxaliplatin and then add irinotecan if the disease progresses. However, the budget impact of this strategy was estimated to be approximately USD 25.1 million, which is about three times higher than the regimen that involves only 5-fluorouracil/leucovorin and oxaliplatin. Policymakers should consider the relatively high budgetary burden of the regimen. This study conducted a cost–utility analysis and a budget impact analysis (BIA) of outpatient oral chemotherapy versus inpatient intravenous chemotherapy for stage III colorectal cancer (CRC) in Thailand. A Markov model was constructed to estimate the lifetime cost and health outcomes based on a societal perspective. Eight chemotherapy strategies were compared. Clinical and cost data on adjuvant chemotherapy were collected from the medical records of 1747 patients at Siriraj Hospital, Thailand. The cost-effectiveness results were interpreted against a Thai willingness-to-pay threshold of USD 5003/quality-adjusted life year (QALY) gained. A 5-year BIA was performed. Of the eight strategies, CAPOX then FOLFIRI yielded the highest life-year and QALY gains. Its total lifetime cost was also the highest. An incremental cost-effectiveness ratio of CAPOX then FOLFIRI compared to 5FU/LV then FOLFOX, a commonly used regimen USD was 4258 per QALY gained.The BIA showed that when generic drug prices were applied, 5-FU/LV then FOLFOX had the smallest budgetary impact (USD 9.1 million). CAPOX then FOLFIRI required an approximately three times higher budgetary level (USD 25.1 million). CAPOX then FOLFIRI is the best option. It is cost-effective compared with 5-FU/LV then FOLFOX. However, policymakers should consider the relatively high budgetary burden of the CAPOX then FOLFIRI regimen. [ABSTRACT FROM AUTHOR]

Background: Multiple sclerosis is an inflammatory demyelination process in the central nervous system (CNS) causing neurological disability and poor quality of life. Currently, Thai Food and Drug Administration (FDA)-approved disease-modifying therapy is costly, and most patients with multiple sclerosis are ineligible for treatment in Thailand as previous studies have challenged its cost-effectiveness. Off-label use of rituximab is inexpensive and highly effective in treating multiple sclerosis, but evidence of its cost-effectiveness in Thailand is yet to be collected. Methods: This study aimed to evaluate the cost-utility and budget impact of rituximab for multiple sclerosis treatment compared with best supportive care, the standard practice in Thailand to treat the disease. A Markov model with a one-month cycle length and lifetime horizon was applied to compare the costs and outcomes of rituximab and best supportive care based on a societal perspective. Accordingly, incremental cost-effectiveness ratios were estimated. Probabilistic and one-way sensitivity analyses were conducted to investigate parameter uncertainty. In addition, the Markov model was used to assess the 5-year budget impact from the government perspective. Results: A rituximab biosimilar demonstrated higher effectiveness and lower associated costs, compared to best supportive care, with the highest probability of being cost-effective (96%). The probability of relapse was the most sensitive parameter according to the one-way sensitivity analysis. The calculated budget impact of treating patients with multiple sclerosis in Thailand was 26,360,000 Thai baht (THB) or 844,255 United States dollars (USD) in the first fiscal year, and approximately 20,810,000–23,080,000 THB (666,608–739,388 USD) in the next four fiscal years. Conclusion: In Thailand, a rituximab biosimilar would reduce the overall costs of multiple sclerosis treatment and should, therefore, be included in the National List of Essential Medicines. [ABSTRACT FROM AUTHOR]

Decision-analytic models (DAMs) with varying structures and assumptions have been applied in economic evaluations (EEs) to assist decision making for heart failure with reduced ejection fraction (HFrEF) therapeutics. This systematic review aimed to summarize and critically appraise the EEs of guideline-directed medical therapies (GDMTs) for HFrEF. A systematic search of English articles and gray literature, published from January 2010, was performed on databases including MEDLINE, Embase, Scopus, NHSEED, health technology assessment, Cochrane Library, etc. The included studies were EEs with DAMs that compared the costs and outcomes of angiotensin-converting enzyme inhibitors, angiotensin-receptor blockers, angiotensin-receptor neprilysin inhibitors, beta-blockers, mineralocorticoid-receptor agonists, and sodium-glucose cotransporter-2 inhibitors. The study quality was evaluated using the Bias in Economic Evaluation (ECOBIAS) 2015 checklist and Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklists. A total of 59 EEs were included. Markov model, with a lifetime horizon and a monthly cycle length, was most commonly used in evaluating GDMTs for HFrEF. Most EEs conducted in the high-income countries demonstrated that novel GDMTs for HFrEF were cost-effective compared with the standard of care, with the standardized median incremental cost-effectiveness ratio (ICER) of $21 361/quality-adjusted life-year. The key factors influencing ICERs and study conclusions included model structures, input parameters, clinical heterogeneity, and country-specific willingness-to-pay threshold. Novel GDMTs were cost-effective compared with the standard of care. Given the heterogeneity of the DAMs and ICERs, alongside variations in willingness-to-pay thresholds across countries, there is a need to conduct country-specific EEs, particularly in low- and middle-income countries, using model structures that are coherent with the local decision context. • Consistent with previous review, Markov model was most commonly used in evaluating the cost-effectiveness of guideline-directed medical therapies for heart failure with reduced ejection population. • This study revealed that novel guideline-directed medical therapies for heart failure with reduced ejection (including sacubitril-valsartan, dapagliflozin, empagliflozin, and eplerenone) were cost-effective, particularly in high-income countries. • Given the heterogeneity of decision-analytic models and incremental cost-effectiveness ratios, alongside variations in willingness-to-pay thresholds across countries, there is a need to conduct country-specific economic evaluations, particularly in low- and middle-income countries, using model structures that are coherent with the local decision context. [ABSTRACT FROM AUTHOR]

Primary spontaneous pneumothorax (PSP) has several commonly used management strategies: observation, aspiration, and chest tube placement. Economic modelling of pooled data comparing techniques has not been performed. Based on studies from the past 20 years, which approach to management of PSP delivers the highest utility? A systematic review of PSP management strategies (observation, aspiration, or chest tube placement) included in the Medline and EMBASE databases from January 1, 2000, through April 10, 2020, was conducted. Text screening, bias assessment, and data extraction were performed by two authors (G. E. and C. A. P.). Inclusion and exclusion criteria were defined a priori. The primary outcome was PSP resolution after the initial intervention. Secondary outcomes were PSP recurrence, length of stay, rate of surgical management, and complications. The meta-analysis compared treatment arms; dichotomous outcomes were reported as relative risk (RRs) and continuous outcomes were reported as mean differences. A cost-utility analysis within the Canadian health care system context with deterministic and probabilistic sensitivity analyses was performed. Five thousand one hundred seventy-nine articles were identified; after screening, 22 articles were included. Most trials showed a high risk of bias, but randomized trials showed a lower risk. Compared with chest tube placement, observation (mean difference, 5.17; 95% CI, 3.75-6.59; P <.01; I 2 = 62%) and aspiration (mean difference, 2.72; 95% CI, 2.39-3.04; P <.01; I 2 = 0%) showed a shorter length of stay. Compared with observation, chest tube placement (RR, 0.81; 95% CI, 0.71-0.91; P <.01; I 2 = 62%) and aspiration (RR, 0.73; 95% CI, 0.61-0.88; P <.01; I 2 = 67%) showed higher resolution without additional intervention. Two-year recurrence rates did not differ between management strategies. Observation showed the best utility (0.82) and lowest cost; observation was the optimal strategy in 98.2% of Monte Carlo simulations. Observation is the dominant choice compared with aspiration and chest tube placement for PSP. It should be considered as the first-line therapy in appropriately selected patients. [Display omitted] [ABSTRACT FROM AUTHOR]

Background: Lungsco01 is the first study assessing the real benefits and the medico-economic impact of video-thoracoscopy versus open thoracotomy for non-small cell lung cancer in the French context. Methods: Two hundred and fifty nine adult patients from 10 French centres were randomised in this prospective multicentre randomised controlled trial, between July 29, 2016, and November 24, 2020. Survival from surgical intervention to day 30 and later was compared with the log-rank test. Total quality-adjusted-life-years (QALYs) were calculated using the EQ-5D-3L®. For medico-economic analyses at 30 days and at 3 months after surgery, resources consumed were valorised (€ 2018) from a hospital perspective. First, since mortality was infrequent and not different between the two arms, cost-minimisation analyses were performed considering only the cost differential. Second, based on complete cases on QALYs, cost-utility analyses were performed taking into account cost and QALY differential. Acceptability curves and the 95% confidence intervals for the incremental ratios were then obtained using the non-parametric bootstrap method (10,000 replications). Sensitivity analyses were performed using multiple imputations with the chained equation method. Results: The average cumulative costs of thoracotomy were lower than those of video-thoracoscopy at 30 days (€9,730 (SD = 3,597) vs. €11,290 (SD = 4,729)) and at 3 months (€9,863 (SD = 3,508) vs. €11,912 (SD = 5,159)). In the cost-utility analyses, the incremental cost-utility ratio was €19,162 per additional QALY gained at 30 days (€36,733 at 3 months). The acceptability curve revealed a 64% probability of efficiency at 30 days for video-thoracoscopy, at a widely-accepted willingness-to-pay threshold of €25,000 (34% at 3 months). Ratios increased after multiple imputations, implying a higher cost for video-thoracoscopy for an additional QALY gain (ratios: €26,015 at 30 days, €42,779 at 3 months). Conclusions: Given our results, the economic efficiency of video-thoracoscopy at 30 days remains fragile at a willingness-to-pay threshold of €25,000/QALY. The economic efficiency is not established beyond that time horizon. The acceptability curves given will allow decision-makers to judge the probability of efficiency of this technology at other willingness-to-pay thresholds. Trial registration: NCT02502318. [ABSTRACT FROM AUTHOR]

Osteoarthritis (OA) is the degeneration of cartilage in joints that results in bones rubbing against each other; it causes uncomfortable symptoms such as pain, swelling, and stiffness and can lead to disability. It usually occurs in the elderly and causes a large medical burden. The aim of this study is to evaluate the cost-effectiveness between the standard treatment for osteoarthritis and standard treatment with added crystalline glucosamine sulfate at various stages. Markov analysis modeling was applied to evaluate the effect of both adding glucosamine compared to standard treatment from a societal perspective during whole patients' lifetimes. Data input was collected from reviews in previous studies. The outcome was measured in quality-adjusted life years (QALYs), and the Incremental Cost-Effectiveness Ratio (ICER) from a societal perspective was applied with 3% and discounted for all costs and outcomes. One-way analysis via the Tornado diagram was performed to investigate the change in factors in the model. In general, adding glucosamine into the standard treatment proved to be more cost-effective compared to the standard treatment. Particularly, the early-stage addition of glucosamine in the treatment was cost-effective compared to the post-stage addition of glucosamine. The addition of supplementing crystalline glucosamine sulfate to the whole regimen at any stage was cost-effective at the willingness-to-pay (WTP) threshold. [ABSTRACT FROM AUTHOR]

Background: Today, heart failure is one of the leading causes of death and disability in most developed and developing countries. By 2030, more than 23.3 million people are projected to die of cardiovascular diseases each year, and the prevalence of heart failure is expected to increase by 25%. One of the preventive interventions is pharmacological interventions which can be used to reduce the complications of cardiovascular diseases such as heart failure. One of the most important pharmacological interventions in patients with heart failure is the use of antihypertensive drugs such as candesartan, enalapril, and valsartan. This study aimed to compare the cost-utility of candesartan, enalapril, and valsartan in patients with heart failure using the Markov model in Iran in 2020. Methods: In the present study, a four-state Markov model was designed to compare the cost-utility of candesartan, enalapril, and valsartan for a hypothetical cohort of 10,000 heart failure patients older than 24 years. The payers' perspective was used to calculate the costs. The Markov states included outpatients with heart failure, patients with heart failure admitted to general hospital wards, patients with heart failure admitted to the intensive care units (ICUs), and death. The effectiveness measure in this study was the quality-adjusted life years (QALYs). The one-way and probabilistic sensitivity analyses were used to determine the robustness of the results. The TreeAge Pro 2011 software was used for data analysis. Results: The results showed that the average expected costs and QALYs were 119645.45 USD and 16.15 for valsartan, 113,019.68 USD and 15.16 for enalapril, and 113,093.37 USD and 15.06 for candesartan, respectively. Candesartan was recognized as the dominated option. Because the calculated incremental cost-effectiveness ratio (ICER) value (6,692.69 USD) was less than the threshold value (7,256 USD), valsartan was cost-effective compared to enalapril. The results of the cost-effectiveness acceptability curve showed that at the threshold of 7,256 USD, valsartan had a 60% chance of being cost-effective compared to enalapril. The results of one-way and probabilistic sensitivity analyses confirmed the robustness of the results. Moreover, the results showed that ICU (1,112 USD) had the highest cost among cost items. Conclusion: According to the results, it is recommended that health policymakers consider the use of valsartan by cardiologists when designing clinical guidelines for the treatment of patients with heart failure. [ABSTRACT FROM AUTHOR]

Background: The aim of this study is to perform a cost‐utility analysis of separated continuous renal replacement therapy (CRRT) compared with intermittent hemodialysis (IHD) in critically ill patients with acute kidney injury (AKI). Methods: Cost and clinical data were gathered from adult patients with AKI who received separated CRRT or IHD at a tertiary hospital in Thailand. We applied a Markov model in this study. Our primary outcome was the incremental cost‐effectiveness ratio (ICER). We performed sensitivity analysis to assess the influence of parameter uncertainty. Results: We enrolled 199 critically ill patients with AKI. Of these patients, 129 underwent separated CRRT, and the rest underwent IHD. The mortality rate and dialysis dependence status were not significantly different between the groups. The total costs of separated CRRT were lower than IHD ($73 042.20 vs. $89 244.37). We estimated that separated CRRT increased quality‐adjusted life years (QALYs) by 0.21 compared with IHD. The ICER of −74 035.16 USD/QALY gained in the case‐based analysis indicated that separated CRRT is superior to IHD due to the lower cost and more cumulative QALYs. After performing sensitivity analysis by varying parameter ranges, separated CRRT remained a cost‐saving approach. Conclusions: Separated CRRT is a cost‐saving modality compared with IHD in critically ill patients with AKI. This approach can be applied in resource‐limited settings. [ABSTRACT FROM AUTHOR]

Purpose: The purpose of this study was to determine the cost-effectiveness of antibiotic-laden bone cement (ALBC) in primary total knee arthroplasty (TKA) from the perspective of a single-payer healthcare system. Methods: A cost-utility analysis (CUA) was performed over a 2-year time horizon comparing primary TKA with either ALBC or regular bone cement (RBC) from the perspective of the single-payer Canadian healthcare system. All costs were in 2020 Canadian dollars. Health utilities were in the form of quality-adjusted life years (QALYs). Model inputs for cost, utilities and probabilities were derived from the literature as well as regional and national databases. One-way deterministic sensitivity analysis was performed. Results: Primary TKA with ALBC was found to be more cost-effective compared to primary TKA with RBC with an incremental cost-effectiveness ratio (ICER) of -3,637.79 CAD/QALY. The use of routine ALBC remained cost-effective even with cost increases of up to 50% per bag of ALBC. TKA with ALBC was no longer cost-effective if the rate of PJI following this practice increased 52%, or the rate of PJI following the use of RBC decreased 27%. Conclusions: The routine use of ALBC in TKA is a cost-effective practice in the single-payer Canadian healthcare system. This remains to be the case even with a 50% increase in the cost of ALBC. Policy makers and hospital administrators of single-payer healthcare systems can leverage this model to inform their local funding policies. Future prospective reviews and randomized controlled trials from the perspective of various healthcare models can further shed light on this issue. Level of evidence: III. [ABSTRACT FROM AUTHOR]

Background: The combinations of BRAF + MEK inhibitors—encorafenib (ENC) + binimetinib (BIN), cobimetinib (COB) + vemurafenib (VEM), and dabrafenib (DAB) + trametinib (TRA)—are recommended for the treatment of BRAF-mutated advanced melanoma. Objective: To assess the cost-effectiveness and cost-utility of ENC + BIN versus COB + VEM versus DAB + TRA from a US payer perspective. Methods: A Markov model was constructed to simulate a hypothetical cohort over a time horizon of 10 years. The overall survival (OS) and progression-free survival (PFS) curves were independently digitized from a randomized controlled trial for ENC + BIN and fitted using R software. Published and indirectly estimated hazard ratios were used to fit OS and PFS curves for COB + VEM and DAB + TRA. Costs, life-year gains, and quality-adjusted life years (QALYs) associated with the 3 treatment combinations were estimated. A base case analysis and probabilistic sensitivity analysis (PSA) were conducted to estimate the incremental cost-utility ratio (ICUR). A discount rate of 3.5% was applied on cost and outcomes. Results: The ENC + BIN versus COB + VEM comparison was associated with an ICUR of $656 233 per QALY gained. The ENC + BIN versus DAB + TRA comparison was associated with an ICUR of $3 135 269 per QALY gained. The DAB + TRA combination dominated COB + VEM. The base case analysis estimates were confirmed by the PSA estimates. ENC + BIN was the most cost-effective combination at a high willingness-to-pay (WTP) threshold of $573 000 per QALY and $1.5 million/QALY when compared to COB + VEM and DAB + TRA, respectively. Conclusion and Relevance: Given current prices and acceptable WTP thresholds, our study suggests that DAB + TRA is the optimum treatment. In this study, ENC + BIN was cost-effective only at a very high WTP per QALY threshold. [ABSTRACT FROM AUTHOR]

Prioritizing health technologies requires comparisons of improvements in longevity or quality of life (QoL), or both. For this purpose, value sets are constructed that contain weights assigned to health states based on societal preferences. I show that how this is typically done may distort the results by giving unjustifiably larger impact to individuals who prioritize improvements in QoL over longevity. The problem results from equating the utility differences between being dead and full health across people, ignoring the fact that interpersonal utility comparisons are forbidden (or at least problematic) in economics. I propose another approach: the numerical value of maximal health gain (either in longevity or QoL) is assumed to be equal across individuals, to remove the impact of the range of utilities differing between people. I test this approach using EQ-5D-5L and EQ-5D-3L utilities elicited in Poland for two modeling techniques: a simple econometric model and a Bayesian one that accounts for censoring. The proposed approach increases the utilities of the worst health state: from - 0.41 to - 0.25 in EQ-5D-5L and from about - 0.54 to - 0.36 in EQ-5D-3L. In the Bayesian approach, the impact is greater: from - 0.45 to - 0.11 for EQ-5D-5L and from - 0.54 to - 0.22 for EQ-5D-3L. I discuss some normative arguments why the proposed approach may be more justifiable for aggregating individual preferences for health outcomes. [ABSTRACT FROM AUTHOR]

Abstract Introduction We sought to evaluate the cost-effectiveness of newborn screening (NBS) versus no NBS for 5q spinal muscular atrophy (SMA) in England. Methods A cost-utility analysis using a combination of decision tree and Markov model structures was developed to estimate the lifetime health effects and costs of NBS for SMA, compared with no NBS, from the perspective of the National Health Service (NHS) in England. A decision tree was designed to capture NBS outcomes, and Markov modeling was used to project long-term health outcomes and costs for each patient group following diagnosis. Model inputs were based on existing literature, local data, and expert opinion. Sensitivity and scenario analyses were conducted to assess the robustness of the model and the validity of the results. Results The introduction of NBS for SMA in England is estimated to identify approximately 56 (96% of cases) infants with SMA per year. Base-case results indicate that NBS is dominant (less costly and more effective) than a scenario without NBS, with a yearly cohort of newborns accruing incremental savings of £62,191,531 and an estimated gain in quality-adjusted life-years of 529 years over their lifetime. Deterministic and probabilistic sensitivity analyses demonstrated the robustness of the base-case results. Conclusions NBS improves health outcomes for patients with SMA and is less costly compared with no screening; therefore, it is a cost-effective use of resources from the perspective of the NHS in England.

Abstract Background New biologic disease-modifying antirheumatic drugs (bDMARDs), targeted synthetic DMARDs (tsDMARDs) and biosimilar DMARDs (bsDMARDs) all showed greater clinical benefits in the treatment of patients with rheumatoid arthritis (RA) with high disease activity, but imposed higher costs than standard treatment. This study evaluated the cost-effectiveness of 11 alternative treatment strategies for RA patients with high disease activity whose treatment with three conventional synthetic DMARDs (csDMARDs) failed. Methods A Markov model was constructed using a societal perspective to estimate relevant costs and health outcomes in terms of quality-adjusted life years (QALYs) for a lifetime horizon (100 years), given a 3% annual discount. Alternative treatment strategies including five bDMARDs, two tsDMARDs, and four bsDMARDs in combination with methotrexate (MTX) were compared with the standard of care (SoC), i.e., cyclosporine and azathioprine. Direct and non-medical care costs were estimated by identifying the resources used, then multiplied by the standard costing menu in the year 2022. Utility and transitional probabilities were collected in three advanced tertiary hospitals. A network meta-analysis was used to estimate the efficacy of each treatment. Lifetime cost, QALYs and an incremental cost-effectiveness ratio were calculated and compared to the cost-effectiveness threshold of 160,000 THB per QALY gained (US $4,634, where 1 USD = 34.53 THB in 2022). Probabilistic and one-way sensitivity analyses were performed to estimate parameter uncertainties. Results The bDMARDs, tsDMARDs or bsDMARDs combined with MTX provided 0.09 to 0.33 QALYs gained with additional costs of 550,986 to 2,096,744 THB (US $15,957 to $60,722) compared to the SoC. The ICER ranged from 2.3 to 8.1 million THB per QALY (US $65,935 to $234,996) compared to the SoC. None of these combinations was cost-effective in the Thai context. The results were sensitive to the mortality hazard ratio of patients with high disease activity. Conclusions Combinations of MTX with either bDMARDs, tsDMARDs or bsDMARDs were not economically attractive compared to the standard practice. However, they reduced disease activity and improved patient quality of life. The price negotiation process for these treatments must be conducted to ensure their financial value and affordability before they are included in the pharmaceutical reimbursement list.

Abstract Background Venous access devices commonly used in clinical practice for long-term chemotherapy of breast cancer include central venous catheters (CVCs), peripherally inserted central venous catheters (PICCs), and implantable venous access ports (IVAPs). CVCs and PICCs are less costly to place but have a higher complication rate than IVAPs. However, there is a lack of cost-utility comparisons among the three devices. The aim of this study was to assess the cost-effectiveness of three catheters for long-term chemotherapy in breast cancer patients. Methods This study used propensity score matching (PSM) to establish a retrospective cohort. Decision tree models were used to compare the cost-effectiveness of three different intravenous lines in breast cancer chemotherapy patients. Cost parameters were derived from data extracted from the outpatient and inpatient charging systems, and total costs included costs of placement, maintenance, extraction, and handling of complications; utility parameters were derived from previous cross-sectional survey results of the research group; and complication rates were derived from breast cancer catheterization patient information as well as follow-up information. Quality-adjusted life years (QALYs) were measured for efficacy outcomes. Incremental cost-effectiveness ratios (ICERs) were used to compare the three strategies. To assess uncertainty in model parameters, sensitivity analyses (univariate sensitivity analysis and probabilistic sensitivity analysis) were performed. Results A total of 10,718 patients (3780 after propensity score matching) were included. IVAPs had the smallest cost-utility ratio, and PICCs had the largest cost-utility ratio when left in place for more than 12 months. The incremental cost-utility ratio of PICC to CVC was $2375.08/QALY, IVAP to PICC was $522.01/QALY, and IVAP to CVC was $612.98/QALY. Incremental cost-effectiveness ratios showed that IVAPs were more effective than CVCs and PICCs. Model regression analysis showed that the IVAP was recommended as the best regimen regardless of the catheter indwelling time (6 months, 12 months or more than 12 months). The reliability and stability of the model were verified by single-factor sensitivity analysis and Monte Carlo simulation (probabilistic sensitivity analysis). Conclusion This study provides economic evidence for the selection of vascular access in breast cancer chemotherapy patients. In the case of limited resources in China, establishing a decision tree model comparing the cost-effectiveness of three vascular access devices for breast cancer chemotherapy patients determined that the IVAP was the most cost-effective regimen.

Abstract Background Radiofrequency ablation (RFA) is the standard of care in the management of supraventricular tachycardia (SVT). Its cost‐effectiveness in an emerging Asian country has not been studied. Objectives A cost‐utility analysis of RFA versus optimal medical therapy (OMT) among Filipinos with SVT was conducted using the public healthcare provider's perspective. Methods A simulation cohort using a lifetime Markov model was constructed using patient interviews, a review of literature, and expert consensus. Three basic health states were defined: stable, SVT recurrence, and death. The incremental cost per quality‐adjusted life year (ICER) was determined for both arms. Utilities for the entry states were derived from patient interviews using the EQ5D‐5L tool; utilities for other health states were taken from publications. Costs were assessed from the healthcare payer perspective. A sensitivity analysis was done. Results Base case analysis showed that RFA versus OMT is both highly cost‐effective at 5 years and over a lifetime. RFA at 5 years costs about PhP276,913.58 (USD5,446) versus OMT of PhP151,550.95 (USD2,981) per patient. Discounted lifetime costs were PhP280,770.32 (USD5,522) for RFA, versus PhP259,549.74 (USD5,105) for OMT. There was improved quality of life with RFA (8.1 vs. 5.7 QALYs per patient). The 5‐year and lifetime incremental cost‐effectiveness ratios were PhP148,741.40 (USD2,926) and Php15,000 (USD295), respectively. Sensitivity analysis showed 56.7% of simulations for RFA fell below a GDP‐benchmarked willingness‐to‐pay (WTP) threshold. Conclusion Despite the initial higher cost, RFA versus OMT for SVT is highly cost‐effective from the Philippine public health payer's perspective.

Objectives: A cost-utility analysis was conducted to evaluate pharmacogenomic (PGx)-guided treatment compared to the standard-of-care intervention among patients diagnosed with colorectal cancer (CRC) in Italy. Methods: Data derived from a prospective, open-label, block randomized clinical trial, as a part of the largest PGx study worldwide (355 patients in both arms) were used. Mortality was used as the primary health outcome to estimate life years (LYs) gained in treatment arms within a survival analysis context. PGx-guided treatment was based on established drug-gene interactions between capecitabine, 5-fluorouracil and irinotecan with DPYD and/or UGT1A1 genomic variants. Utility values for the calculation of Quality Adjusted Life Year (QALY) was based on Visual Analog Scale (VAS) score. Missing data were imputed via the Multiple Imputation method and linear interpolation, when possible, while censored cost data were corrected via the Replace-From-The-Right algorithm. The Incremental Cost-Effectiveness Ratio (ICER) was calculated for QALYs. Raw data were bootstrapped 5000 times in order to produce 95% Confidence Intervals based on non-parametric percentile method and to construct a cost-effectiveness acceptability curve. Cost differences for study groups were investigated via a generalized linear regression model analysis. Total therapy cost per patient reflected all resources expended in the management of any adverse events, including medications, diagnostics tests, devices, surgeries, the utilization of intensive care units, and wards. Results: The total cost of the study arm was estimated at €380 (∼ US$416; 95%CI: 195–596) compared to €565 (∼ US$655; 95%CI: 340–724) of control arm while the mean survival in study arm was estimated at 1.58 (+0.25) LYs vs 1.50 (+0.26) (Log Rank test, X2 = 4.219, df=1, p-value=0.04). No statistically significant difference was found in QALYs. ICER was estimated at €13418 (∼ US$14695) per QALY, while the acceptability curve indicated that when the willingness-to-pay was under €5000 (∼ US$5476), the probability of PGx being cost-effective overcame 70%. The most frequent adverse drug event in both groups was neutropenia of severity grade 3 and 4, accounting for 82.6% of total events in the study arm and 65.0% in the control arm. Apart from study arm, smoking status, Body-Mass-Index and Cumulative Actionability were also significant predictors of total cost. Subgroup analysis conducted in actionable patients (7.9% of total patients) indicated that PGx-guided treatment was a dominant option over its comparator with a probability greater than 92%. In addition, a critical literature review was conducted, and these findings are in line with those reported in other European countries. Conclusion: PGx-guided treatment strategy may represent a cost-saving option compared to the existing conventional therapeutic approach for colorectal cancer patient management in the National Health Service of Italy.

BackgroundMost Chlamydia trachomatis (CT) infections are asymptomatic. The infection can persist and lead to severe sequelae. Therefore, screening for CT can primarily prevent serious sequelae.AimTo systematically evaluate CT screening from the perspective of health economics, summarize previous findings from different target populations, and make practical recommendations for developing local CT screening strategies.MethodsPubMed, Web of Science, Embase, Cochran Library, and National Health Service Economic Evaluation Database (Ovid) were searched from January 1, 2000, to March 4, 2023. Studies reporting the cost-effectiveness, cost-benefit, or cost-utility of CT screening were eligible to be included. A narrative synthesis was used to analyze and report the results following the PRISMA guidelines. The Consensus on Health Economic Criteria (CHEC) list was used to assess the methodological quality of included studies.ResultsOur review finally comprised 39 studies addressing four populations: general sexually active people (n = 25), pregnant women (n = 4), women attending STD and abortion clinics (n = 4), and other high-risk individuals (n = 6). The total number of participants was ~7,991,198. The majority of studies assessed the cost-effectiveness or cost-utility of the screening method. The results showed that the following screening strategies may be cost-effective or cost-saving under certain conditions: performing CT screening in young people aged 15–24 in the general population, military recruits, and high school students; incorporating CT screening into routine antenatal care for pregnant women aged 15–30; opportunistic CT screening for women attending STD and abortion clinics; home-obtained sampling for CT screening using urine specimens or vaginal swab; performing CT screening for 14–30-year-old people who enter correctional institutions (i.e., jail, detention) as soon as possible; providing CT screening for female sex workers (FSWs) based on local incidence and prevalence; adding routine CT screening to HIV treatment using rectal samples from men who have sex with men (MSM).ConclusionWe found that CT screening in general sexually active people aged 15–24, military recruits, high school students, pregnant women aged 15–30, women attending STD and abortion clinics, people entering jail, detention, FSWs, and MSM has health economic value. Due to the different prevalence of CT, diversities of economic conditions, and varying screening costs among different populations and different countries, regions, or settings, no uniform and standard screening strategies are currently available. Therefore, each country should consider its local condition and the results of health economic evaluations of CT screening programs in that country to develop appropriate CT screening strategies.

Aim: Clinical trials and real-world data for Type 2 diabetes have shown that real-time continuous glucose monitoring (rt-CGM) lowers glycated hemoglobin (A1c) and reduces hypoglycemia relative to self-monitoring of blood glucose (SMBG). This analysis examined the long-term health and economic outcomes associated with using rt-CGM versus SMBG in people with insulin-treated Type 2 diabetes in Canada. Materials & methods: Clinical data were sourced from a real-world study, inwhich rt-CGM reduced A1C by 0.56% versus continued SMBG. The analysis was performed using the IQVIA Core Diabetes Model, from a Canadian payer perspective over a lifetime horizon for a cohort aged 65 years with an A1C of 8.3% at baseline. Future costs and clinical outcomes were discounted at 1.5% annually. Results: Projected total mean lifetime costs were CAD 207,466 for rt-CGM versus CAD 189,863 for SMBG (difference: CAD 17,602) and projected mean quality-adjusted life expectancy was 9.97 quality-adjusted life years (QALYs) for rt-CGM versus 9.02 QALYs for SMBG (difference: 0.95 QALYs), resulting in an incremental cost-utility ratio (ICUR) of CAD 18,523 per QALY gained for rt-CGM versus SMBG. Findings were sensitive to changes in the A1C treatment effect, annual cost and quality of life benefit associated with using rt-CGM, SMBG frequency, and baseline age, but ICURs remained below CAD 50,000 per QALY in all analyses. Conclusion: For people in Canada with insulin-treated Type 2 diabetes and poor glycemic control, use of rt-CGM is likely to be cost-effective relative to SMBG.

Objective: Long-acting injections (LAIs) of paliperidone palmitate have been shown to improve medication adherence and relieve psychotic symptoms. However, the specific cost-utility analysis of these LAIs in schizophrenia in China remains unclear. Methods: A multi-state Markov model was constructed to simulate the economic outcomes of patients with schizophrenia in China who received paliperidone palmitate 1-month formulation (PP1M), paliperidone palmitate 3-month formulation (PP3M), and paliperidone extended-release (ER). A cost-utility analysis was conducted, mostly derived from published literature and clinical databases. All costs and utilities were discounted at a rate of 5% per annum. The primary outcome measure was the incremental cost-effectiveness ratios (ICERs). A series of sensitivity analyses were also applied. Results: After 20 years, compared to ER, using PP1M resulted in an increased discounted cost from $36,252.59 to $43,207.28. This increased cost was associated with a gain in quality-adjusted life years (QALYs) from 8.60 to 9.45. As a result, the ICER for PP1M was estimated to be $8,247.46/QALY, which was lower than the willingness-to-pay (WTP) threshold of $12,756.55/QALY. When using PP3M instead of ER, the incremental cost was $768.81 and the incremental utility was 0.88 QALYs, projecting an ICER of $873.13/QALY, which was also lower than the WTP threshold of $12,756.55/QALY. The univariate sensitivity analysis showed that the costs of PP1M, PP3M, and ER had the greatest impact on ICERs. The probability sensitivity analysis (PSA) revealed that when the WTP thresholds were $12,756.55/QALY, the probability of PP1M and PP3M being cost-effective was 59.2% and 66.0%, respectively. Conclusion: From the Chinese healthcare system perspective, PP3M and PP1M are both more cost-effective compared to ER, and PP3M has notable cost-utility advantages over PP1M. [ABSTRACT FROM AUTHOR]

Treatment-resistant depression (TRD) accounts for a large fraction of the burden of depression. The interventions currently used are mostly pharmacological and short-term psychotherapies, but their effectiveness is limited. The Tavistock Adult Depression Study found evidence for the effectiveness of long-term psychoanalytic psychotherapy (LTPP) plus treatment as usual (TAU), versus TAU alone, for TRD. Even after a 2-year follow-up, moderate effect sizes were sustained. This study assessed the cost-effectiveness of this LTPP + TAU. We conducted a within-trial economic evaluation using a Bayesian framework. Quality-adjusted life years (QALYs) were 0.16 higher in the LTPP + TAU group compared with TAU. The direct cost of LTPP was £5500, with no substantial compensating savings elsewhere. Overall, average health and social care costs in the LTPP + TAU group were £5000 more than in the TAU group, employment rates were unchanged, and effects on other non-healthcare costs were uncertain. Accordingly, the incremental cost-effectiveness ratio was ≈£33,000/QALY; the probability that LTPP + TAU was cost-effective at a willingness to pay of £20,000/QALY was 18 %. The sample size of this study was relatively small, and the fraction of missing service-use data was approximately 50 % at all time points. The study was conducted at a single site, potentially reducing generalizability. Although LTPP + TAU was found to be clinically effective for treating TRD, it was not found to be cost-effective compared with TAU. However, given the sustained effects over the follow-up period it is likely that the time horizon of this study was too short to capture all benefits of LTPP augmentation. • Quality-adjusted life years were 0.16 higher in the intervention group. • The direct costs of the intervention were approximately £5500. • These intervention costs were not offset by savings elsewhere. • The intervention was not cost-effective, but underestimation of benefits is likely. [ABSTRACT FROM AUTHOR]

Background: The morbidity and mortality of adult diseases caused by S. pneumoniae increase with age and presence of underlying chronic diseases. Currently, two vaccine technologies against S. pneumoniae are used: the 23-valent pneumococcal polysaccharide vaccine (PPV23) and the pneumococcal conjugate vaccines, one of which is the 20-valent pneumococcal conjugate vaccine (PCV20) that has recently been approved for adults. Objective: This study was conducted to investigate the cost-effectiveness of implementing PCV20 in a reimbursement scheme for Norwegian adults aged 18–99 years at risk of pneumococcal diseases and those aged 65 years and older at low risk compared to PPV23. Methods: An established Markov model was adapted to a Norwegian setting to estimate the economic and clinical consequences of vaccinating the Norwegian population in specific age and risk groups against pneumococcal diseases. Inputs for the model were found in Norwegian or Danish real-world evidence or retrieved from available studies. The costs and clinical outcomes were assessed using a health sector perspective and a lifetime time horizon. Results: The results showed that PCV20 was associated with better health outcomes including fewer disease cases, fewer disease-attributable fatalities, a higher gain of life years and quality-adjusted life years compared to PPV23. In addition, PCV20 had a lower total cost compared to PPV23. Therefore, PCV20 was the dominant vaccination strategy. The base case result was investigated in multiple sensitivity analyses, which showed that the results were robust to changes in input parameters and methodological assumptions, as PCV20 remained the dominant vaccination strategy in almost all scenarios. Conclusion: Results showed that vaccinating the Norwegian adults with PCV20 was cost-effective compared to PPV23. Changes in the hospital cost of pneumonia, the price of PCV 20, the effectiveness of PCV20 against pneumonia, and the pneumonia disease incidence had the highest impact on the ICER, i.e., were the main drivers of the results. [ABSTRACT FROM AUTHOR]

Introduction: We sought to evaluate the cost-effectiveness of newborn screening (NBS) versus no NBS for 5q spinal muscular atrophy (SMA) in England. Methods: A cost-utility analysis using a combination of decision tree and Markov model structures was developed to estimate the lifetime health effects and costs of NBS for SMA, compared with no NBS, from the perspective of the National Health Service (NHS) in England. A decision tree was designed to capture NBS outcomes, and Markov modeling was used to project long-term health outcomes and costs for each patient group following diagnosis. Model inputs were based on existing literature, local data, and expert opinion. Sensitivity and scenario analyses were conducted to assess the robustness of the model and the validity of the results. Results: The introduction of NBS for SMA in England is estimated to identify approximately 56 (96% of cases) infants with SMA per year. Base-case results indicate that NBS is dominant (less costly and more effective) than a scenario without NBS, with a yearly cohort of newborns accruing incremental savings of £62,191,531 and an estimated gain in quality-adjusted life-years of 529 years over their lifetime. Deterministic and probabilistic sensitivity analyses demonstrated the robustness of the base-case results. Conclusions: NBS improves health outcomes for patients with SMA and is less costly compared with no screening; therefore, it is a cost-effective use of resources from the perspective of the NHS in England. [ABSTRACT FROM AUTHOR]

Aims: The DELIVER study demonstrates a significant improvement in cardiovascular death or hospitalization for heart failure among heart failure with mildly reduced ejection fraction (HFmrEF) or heart failure with preserved ejection fraction (HFpEF).Cost‐utility of the adjunct use of dapagliflozin to standard therapy among patients with HFpEF or HFmrEF remains unclear. Methods and results: A five‐state Markov mode was constructed to project health and clinical outcomes of the adjunct use of dapagliflozin to standard therapy among 65‐year‐old patients with HFpEF or HFmrEF. A cost‐utility analysis was performed based on the DELIVER study and national statistical database. The cost and utility was inflated to 2022 by the usual discount rate of 5%. The primary outcomes were total cost and quality‐adjusted life‐years (QALYs) per patients as well as the incremental cost‐effectiveness ratio. Sensitivity analyses were also applied. Over a 15 year lifetime horizon, the average cost per patient was $7245.77 and $5407.55 in the dapagliflozin group and the standard group, along with an incremental cost of $1838.22. The average QALYs per patient was 6.00 QALYs and 5.84 QALYs in the dapagliflozin group and the standard group, along with an incremental QALYs of 0.15 QALYs, resulting in the incremental cost‐effectiveness ratio of $11 865.33/QALY, which was below the willingness‐to‐pay (WTP) of $12 652.5/QALY. The univariate sensitivity analysis indicated the cardiovascular death in both group was the most sensitive variable. Probability sensitivity analysis revealed that when the WTP thresholds were $12 652.5/QALY and $37 957.5/QALY, the probabilities of being cost‐effective with dapagliflozin as an add‐on were 54.6% and 71.6%, respectively. Conclusions: From a public healthcare system perspective, the adjunct use of dapagliflozin to standard therapy among patients with HFpEF or HFmrEF generated advantages in cost‐effectiveness in China at a WTP of $12 652.5/QALY, which promoted the rational use of dapagliflozin for heart failure. [ABSTRACT FROM AUTHOR]

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Background: Oseltamivir is usually not often prescribed (or reimbursed) for non-high-risk patients consulting for influenza-like-illness (ILI) in primary care in Europe. We aimed to evaluate the cost-effectiveness of adding oseltamivir to usual primary care in adults/adolescents (13 years +) and children with ILI during seasonal influenza epidemics, using data collected in an open-label, multi-season, randomised controlled trial of oseltamivir in 15 European countries. Methods: Direct and indirect cost estimates were based on patient reported resource use and official country-specific unit costs. Health-Related Quality of Life was assessed by EQ-5D questionnaires. Costs and quality adjusted life-years (QALY) were bootstrapped (N = 10,000) to estimate incremental cost-effectiveness ratios (ICER), from both the healthcare payers' and the societal perspectives, with uncertainty expressed through probabilistic sensitivity analysis and expected value for perfect information (EVPI) analysis. Additionally, scenario (self-reported spending), comorbidities subgroup and country-specific analyses were performed. Results: The healthcare payers' expected ICERs of oseltamivir were €22,459 per QALY gained in adults/adolescents and €13,001 in children. From the societal perspective, oseltamivir was cost-saving in adults/adolescents, but the ICER is €8,344 in children. Large uncertainties were observed in subgroups with comorbidities, especially for children. The expected ICERs and extent of decision uncertainty varied between countries (EVPI ranged €1–€35 per patient). Conclusion: Adding oseltamivir to primary usual care in Europe is likely to be cost-effective for treating adults/adolescents and children with ILI from the healthcare payers' perspective (if willingness-to-pay per QALY gained > €22,459) and cost-saving in adults/adolescents from a societal perspective. [ABSTRACT FROM AUTHOR]

Background: Health state utilities (HSU) are a health-related quality-of-life (HRQoL) input for cost-utility analyses used for resource allocation decisions, including medication reimbursement. New Zealand (NZ) guidelines recommend the EQ-5D instruments; however, the EQ-5D-5L may not sufficiently capture psychosocial health. We evaluated HRQoL among people with multiple sclerosis (MS) in NZ using the EQ-5D-5L and assessed the instrument's discriminatory sensitivity for a NZ MS cohort. Methods: Participants were recruited from the NZ MS Prevalence Study. Participants self-completed a 45-min online survey that included the EQ-5D-5L/EQ-VAS. Disability severity was classified using the Expanded Disability Status Scale (EDSS) to categorise participant disability as mild (EDSS: 0–3.5), moderate (EDSS: 4.0–6.0) and severe (EDSS: 6.5–9.5). Anxiety/depression were also measured using the Hospital Anxiety and Depression Score (HADS). In the absence of an EQ-5D-5L NZ tariff, HSUs were derived using an Australian tariff. We evaluated associations between HSUs and participant characteristics with linear regression models. Results: 254 participants entered the study. Mean age was 55.2 years, 79.5% were female. Mean (SD) EQ-5D-5L HSU was 0.58 (0.33). Mean (SD) HSUs for disability categories were: mild 0.80 ± 0.17, moderate 0.57 ± 0.21 and severe 0.14 ± 0.32. Twelve percent reported HSU = 1.0 (i.e., no problems in any domain). Participants who had never used a disease-modifying therapy reported a lower mean HSU. Multivariable modelling found that the HADS anxiety score was not associated with EQ-5D-5L. Conclusions: HRQoL for people with MS in NZ was lower than comparable countries, including Australia. We suggest a comparison with other generic tools that may have improved sensitivity to mental health. [ABSTRACT FROM AUTHOR]

This study aimed to determine the cost-utility of ocrelizumab versus rituximab in patients with RRMS, from the perspective of the Colombian healthcare system. Cost-utility study based on a Markov model, with a 50-year horizon and payer perspective. The currency was the US dollar for the year 2019, with a cost-effectiveness threshold of $5180 defined for Colombian health system. The model used annual cycles according to the health status determined by the disability scale. Direct costs were considered, and the incremental cost-effectiveness ratio per 1 quality-adjusted life-year (QALY) gained was used as the outcome measure. A discount rate of 5% was applied to costs and outcomes. Multiple one-way deterministic sensitivity analyses and 10 000 Monte Carlo simulation were conducted. For the treatment of patients with RRMS, ocrelizumab versus rituximab had an incremental cost-effectiveness ratio of $73 652 for each QALY gained. After 50 years, 1 subject treated with ocrelizumab earns 4.8 QALYs >1 subject treated with rituximab, but at a higher cost of $521 759 versus $168 752, respectively. Ocrelizumab becomes a cost-effective therapy if its price is discounted > 86% or if there is a high willingness to pay. Ocrelizumab was not a cost-effective drug as compared with rituximab in treating patients with RRMS in Colombia. • Ocrelizumab and rituximab are 2 anti-CD20 antibodies with similar effectiveness according to a network meta-analysis. Nevertheless, they have not been evaluated in a head-to-head study. • This is the first study evaluating the cost-effectiveness of ocrelizumab and rituximab. In different scenarios, ocrelizumab has proven not to be a cost-effective therapy; either its price needs be reduced significantly or there needs to be an increased willingness to pay for it to become cost-effective. • Rituximab could be a cost-saving therapy. Public policy decision makers should consider this information to facilitate the use of rituximab. [ABSTRACT FROM AUTHOR]

Clinical outcomes in chronic lymphocytic leukemia (CLL) have improved with targeted therapy, including Bruton tyrosine kinase inhibitors such as acalabrutinib. A semi-Markov model with three health states (progression-free, progressed disease, and death) estimated cost per quality-adjusted life-year (QALY) gained for acalabrutinib ± obinutuzumab vs chlorambucil + obinutuzumab in treatment-naive CLL (based on ELEVATE-TN). The model used direct costs and resource utilization from the US Medicare perspective and utility values sourced from literature. Sensitivity analyses tested the robustness of the model. Over a 30-year lifetime horizon, the model base case analysis suggested that acalabrutinib monotherapy had an incremental cost of $206,329 and 2.52 QALYs gained versus chlorambucil + obinutuzumab, resulting in an incremental cost-effectiveness ratio (ICER) of $81,960/QALY. Acalabrutinib + obinutuzumab had an incremental cost of $423,747 and 2.79 QALYs gained (ICER: $152,153/QALY). Probabilistic sensitivity analysis showed the probability of acalabrutinib monotherapy being cost-effective as 59% to 73% at a $100,000-to-150,000/QALY willingness-to-pay threshold; the probability of acalabrutinib + obinutuzumab being cost-effective ranged from 34% to 51%. Although the analysis is limited by uncertainty in postprogression survival outcomes, acalabrutinib monotherapy is likely cost-effective vs chlorambucil + obinutuzumab in treatment-naive CLL in the US Medicare setting. [ABSTRACT FROM AUTHOR]

Abstract Introduction Deep learning (DL) for screening diabetic retinopathy (DR) has the potential to address limited healthcare resources by enabling expanded access to healthcare. However, there is still limited health economic evaluation, particularly in low- and middle-income countries, on this subject to aid decision-making for DL adoption. Methods In the context of a middle-income country (MIC), using Thailand as a model, we constructed a decision tree-Markov hybrid model to estimate lifetime costs and outcomes of Thailand’s national DR screening program via DL and trained human graders (HG). We calculated the incremental cost-effectiveness ratio (ICER) between the two strategies. Sensitivity analyses were performed to probe the influence of modeling parameters. Results From a societal perspective, screening with DL was associated with a reduction in costs of ~ US$ 2.70, similar quality-adjusted life-years (QALY) of + 0.0043, and an incremental net monetary benefit of ~ US$ 24.10 in the base case. In sensitivity analysis, DL remained cost-effective even with a price increase from US$ 1.00 to US$ 4.00 per patient at a Thai willingness-to-pay threshold of ~ US$ 4.997 per QALY gained. When further incorporating recent findings suggesting improved compliance to treatment referral with DL, our analysis models effectiveness benefits of ~ US$ 20 to US$ 50 depending on compliance. Conclusion DR screening using DL in an MIC using Thailand as a model may result in societal cost-savings and similar health outcomes compared with HG. This study may provide an economic rationale to expand DL-based DR screening in MICs as an alternative solution for limited availability of skilled human resources for primary screening, particularly in MICs with similar prevalence of diabetes and low compliance to referrals for treatment.

Abstract Background Lumbar interbody fusion techniques treat degenerative lumbar diseases effectively. Minimally invasive lateral lumbar interbody fusion (LLIF) decreases soft tissue disruption and accelerates recovery better than standard open posterior lumbar interbody fusion (PLIF). However, the material cost of LLIF is high, especially in Thailand. The cost-effectiveness of LLIF and PLIF in developing countries is unclear. This study compared the cost-utility and clinical outcomes of LLIF and PLIF in Thailand. Methods Data from patients with lumbar spondylosis who underwent single-level LLIF and PLIF between 2014 and 2020 were retrospectively reviewed. Preoperative and 1-year follow-up EuroQol-5D-5L and healthcare costs were collected. A cost-utility analysis with a lifetime time horizon was performed using a societal perspective. Outcomes are reported as the incremental cost-effectiveness ratio (ICER) and quality-adjusted life-year (QALY) gained. A Thai willingness-to-pay threshold of 5003 US dollars (USD) per QALY gained was used. Results The 136 enrolled patients had a mean age of 62.26 ± 11.66 years. Fifty-nine patients underwent LLIF, while 77 underwent PLIF. The PLIF group experienced greater estimated blood loss (458.96 vs 167.03 ml; P

Abstract Purpose To evaluate the cost-effectiveness of Platelet Rich Plasma (PRP), Plasma Rich in Growth Factors (PRGF), Hyaluronic Acid (HA) and ozone as effective treatment approaches in knee osteoarthritis management from Iran Health care perspective. Methods A decision tree model was conducted to assess the cost-effectiveness of four common intra-articular treatment approaches in patients with mild and moderate knee osteoarthritis. The data on clinical effectiveness was obtained from a randomized controlled trial (RCT) conducted in Iran and used to estimate utility values. The direct medical costs were estimated according to tariffs for public medical centers and hospitals, approved by the Iran Ministry of Health and Medical Education in 2021. The incremental cost-effectiveness ratio (ICER) and the net monetary benefit (NMB) were used to evaluate the cost-utility analysis. Deterministic and probabilistic sensitivity analyses are performed to investigate the robustness of the results and account for the different sources of uncertainty. Results In this study, HA intra-articular injection-related costs ($581.67/patient) were defined as the highest cost, followed by PRGF ($328.10/patient), PRP (318.58/patient), and Ozone (103.20/patient). According to the utility value, PRP and PRGF (0.68) have the same and the most utility among Intra-articular injections in knee osteoarthritis management. However, the PRP injection method was identified as the most cost-effective intervention due to its high NMB and ICER estimates. Based on the Monte Carlo Simulation, PR intervention, compared to other ones, was introduced as the dominant strategy regarding knee OA management, with a WTP of $10,000 for 100% of cases. Conclusion The study result demonstrated that intra-articular injection of PRP, compare to other injections, is a cost-effective treatment option for patients with mild and moderate knee osteoarthritis. In addition, intra-articular injection of PRP was identified as the best injection, with the highest level of net monetary benefit, for knee OA management.

Taru Hallinen,1 Santtu Kivelä,1 Erkki Soini,1 Veli-Pekka Harjola,2 Mari Pesonen3 1ESiOR Oy, Kuopio, Finland; 2Emergency Medicine, University of Helsinki, Department of Emergency Medicine and Services, Helsinki University Hospital, Helsinki, Finland; 3Boehringer Ingelheim Ky, Helsinki, FinlandCorrespondence: Taru Hallinen, ESiOR Oy, Tulliportinkatu 2 LT 4, Kuopio, FI-70100, Finland, Tel +358 50 568 1894, Email taru.hallinen@esior.fiPurpose: Sodium-glucose cotransporter-2 (SGLT2) inhibitor empagliflozin has recently been shown to improve the outcomes of heart failure (HF) patients regardless of patient’s left ventricular ejection fraction by reducing the combined risk of cardiovascular death or hospitalization for worsening HF. The aim of this study was to assess the cost-effectiveness of adding empagliflozin to the standard care (SC) in comparison to SC only in the treatment of HF in Finland.Patients and Methods: The assessment was performed in the cost-utility framework using two Markov cohort state-transition models, one for HF with reduced ejection fraction (HFrEF) and one for HF with preserved ejection fraction (HFpEF). The models have been primarily developed based on the EMPEROR-Reduced and EMPEROR-Preserved trials which informed the modelled patient characteristics, efficacy of treatments in terms of associated risks for heart failure hospitalizations, cardiovascular (CV) and non-CV death, treatment related adverse events (AE), and state- and event-specific health-related quality of life weights (EQ-5D). Direct health care costs were estimated from Finnish published references. Cost-effectiveness was assessed from health care payer perspective based on incremental cost-effectiveness ratio (ICER; cost per quality adjusted life-year [QALY] gained) and probability of cost-effectiveness (at willingness-to-pay [WTP] of 35,000 euros/QALY). The ICER was reported as the weighted (HFrEF, 43.5%; HFpEF, 56.5%) average result of the two models.Results: Empagliflozin + SC treatment increased the average quality-adjusted life-expectancy, and treatment costs of HF patients by 0.15 QALYs and 1,594 euros, respectively, when compared to SC. An additional QALY with empagliflozin was thus gained at a cost of 10,621 euros. The probability of empagliflozin + SC being cost-effective compared to placebo + SC was 77.6% and 83.5% with WTP of 35,000 and 100,000 euros/QALY, respectively.Conclusion: Empagliflozin is a cost-effective treatment for patients with HF in the Finnish health care setting.Keywords: sodium-glucose cotransporter 2 inhibitor, cost-utility analysis, heart failure with reduced ejection fraction, heart failure with preserved ejection fraction

Abstract Background Understanding what an economic evaluation is, how to interpret it, and what it means for making choices in a health delivery context is necessary to contribute to decisions about healthcare resource allocation. The aim of this paper to demystify the working parts of a health economic evaluation, and explain to clinicians and clinical researchers how to read and interpret cost-effectiveness research. Main body This primer distils key content and constructs of economic evaluation studies, and explains health economic evaluation in plain language. We use the PICOT (participant, intervention, comparison, outcome, timeframe) clinical trial framework familiar to clinicians, clinical decision-makers, and clinical researchers, who may be unfamiliar with economics, as an aide to reading and interpreting cost-effectiveness research. We provide examples, primarily of physiotherapy interventions for osteoarthritis. Conclusions Economic evaluation studies are essential to improve decisions about allocating resources, whether those resources be your time, the capacity of your service, or the available funding across the entire healthcare system. The PICOT framework can be used to understand and interpret cost-effectiveness research.

Treatment for acute myeloid leukemia (AML) typically involves intensive chemotherapy (IC); however, there is an unmet need for approximately 50% of AML patients who are deemed unfit or ineligible for IC. The purpose of this study was to evaluate, from a Canadian perspective, the economic impact of venetoclax in combination with azacitidine (Ven+Aza) for the treatment of patients with newly diagnosed AML who are 75 years or older or who have comorbidities that preclude using IC. A lifetime partitioned survival model was developed to assess the cost-effectiveness of Ven+Aza compared with Aza. Health states included event-free survival, progressive/relapsed disease, and death. Efficacy parameters were based on the VIALE-A trial. Analyses were conducted from Ministry of Health (MoH) and societal perspectives. Over a lifetime horizon, Ven+Aza was associated with a gain of 1.65 quality-adjusted life years (QALYs) compared with Aza. From an MoH perspective, Ven+Aza and Aza were associated with total costs of $204,305 and $82,333, respectively, resulting in an incremental cost–utility ratio of $73,841/QALY. Results were similar from a societal perspective. This economic evaluation demonstrates that, in comparison with Aza, Ven+Aza is a cost-effective strategy for the treatment of patients with newly diagnosed AML who are deemed unfit for IC.

Amyotrophic lateral sclerosis is a progressive neuromuscular disease. Mechanical ventilation delays death and use at home can reduce costs and increase quality of life. The aim of the study is comparison of home mechanical ventilation with mechanical ventilation in a healthcare facility for adult patients with amyotrophic lateral sclerosis from the societal perspective in the Czech Republic. The Markov model from a previous Czech study was used and updated with data revelant to the societal perspective, which was obtained from the literature and opinions of experts from companies dealing with home mechanical ventilation. The cost-utility analysis was carried out over a time horizont of 10 years and results were presented in incremental cost-utility ratio. Accuracy and validity were verified by probabilistic sensitivity analysis and scenario analysis. The cumulative costs of home mechanical ventilation are CZK 6,116,394 and the cumulative utilities of home mechanical ventilation are CZK 8,195,848. The cumulative utilities of home mechanical ventilation are 3.55 QALY and the cumulative utilities of mechanical ventilation are 3.78 QALY. The incremental cost-utility ratio is CZK 9,094,042. Probabilistic sensitivity analysis and scenario analysis do not show significant changes. Home mechanical ventilation may be considered cost-effective for patients with amyotrophic lateral sclerosis from the societal perspective.

Background: New biologic disease-modifying antirheumatic drugs (bDMARDs), targeted synthetic DMARDs (tsDMARDs) and biosimilar DMARDs (bsDMARDs) all showed greater clinical benefits in the treatment of patients with rheumatoid arthritis (RA) with high disease activity, but imposed higher costs than standard treatment. This study evaluated the cost-effectiveness of 11 alternative treatment strategies for RA patients with high disease activity whose treatment with three conventional synthetic DMARDs (csDMARDs) failed. Methods: A Markov model was constructed using a societal perspective to estimate relevant costs and health outcomes in terms of quality-adjusted life years (QALYs) for a lifetime horizon (100 years), given a 3% annual discount. Alternative treatment strategies including five bDMARDs, two tsDMARDs, and four bsDMARDs in combination with methotrexate (MTX) were compared with the standard of care (SoC), i.e., cyclosporine and azathioprine. Direct and non-medical care costs were estimated by identifying the resources used, then multiplied by the standard costing menu in the year 2022. Utility and transitional probabilities were collected in three advanced tertiary hospitals. A network meta-analysis was used to estimate the efficacy of each treatment. Lifetime cost, QALYs and an incremental cost-effectiveness ratio were calculated and compared to the cost-effectiveness threshold of 160,000 THB per QALY gained (US $4,634, where 1 USD = 34.53 THB in 2022). Probabilistic and one-way sensitivity analyses were performed to estimate parameter uncertainties. Results: The bDMARDs, tsDMARDs or bsDMARDs combined with MTX provided 0.09 to 0.33 QALYs gained with additional costs of 550,986 to 2,096,744 THB (US $15,957 to $60,722) compared to the SoC. The ICER ranged from 2.3 to 8.1 million THB per QALY (US $65,935 to $234,996) compared to the SoC. None of these combinations was cost-effective in the Thai context. The results were sensitive to the mortality hazard ratio of patients with high disease activity. Conclusions: Combinations of MTX with either bDMARDs, tsDMARDs or bsDMARDs were not economically attractive compared to the standard practice. However, they reduced disease activity and improved patient quality of life. The price negotiation process for these treatments must be conducted to ensure their financial value and affordability before they are included in the pharmaceutical reimbursement list. [ABSTRACT FROM AUTHOR]

Background: Venous access devices commonly used in clinical practice for long-term chemotherapy of breast cancer include central venous catheters (CVCs), peripherally inserted central venous catheters (PICCs), and implantable venous access ports (IVAPs). CVCs and PICCs are less costly to place but have a higher complication rate than IVAPs. However, there is a lack of cost-utility comparisons among the three devices. The aim of this study was to assess the cost-effectiveness of three catheters for long-term chemotherapy in breast cancer patients. Methods: This study used propensity score matching (PSM) to establish a retrospective cohort. Decision tree models were used to compare the cost-effectiveness of three different intravenous lines in breast cancer chemotherapy patients. Cost parameters were derived from data extracted from the outpatient and inpatient charging systems, and total costs included costs of placement, maintenance, extraction, and handling of complications; utility parameters were derived from previous cross-sectional survey results of the research group; and complication rates were derived from breast cancer catheterization patient information as well as follow-up information. Quality-adjusted life years (QALYs) were measured for efficacy outcomes. Incremental cost-effectiveness ratios (ICERs) were used to compare the three strategies. To assess uncertainty in model parameters, sensitivity analyses (univariate sensitivity analysis and probabilistic sensitivity analysis) were performed. Results: A total of 10,718 patients (3780 after propensity score matching) were included. IVAPs had the smallest cost-utility ratio, and PICCs had the largest cost-utility ratio when left in place for more than 12 months. The incremental cost-utility ratio of PICC to CVC was $2375.08/QALY, IVAP to PICC was $522.01/QALY, and IVAP to CVC was $612.98/QALY. Incremental cost-effectiveness ratios showed that IVAPs were more effective than CVCs and PICCs. Model regression analysis showed that the IVAP was recommended as the best regimen regardless of the catheter indwelling time (6 months, 12 months or more than 12 months). The reliability and stability of the model were verified by single-factor sensitivity analysis and Monte Carlo simulation (probabilistic sensitivity analysis). Conclusion: This study provides economic evidence for the selection of vascular access in breast cancer chemotherapy patients. In the case of limited resources in China, establishing a decision tree model comparing the cost-effectiveness of three vascular access devices for breast cancer chemotherapy patients determined that the IVAP was the most cost-effective regimen. [ABSTRACT FROM AUTHOR]

Introduction: Secondary prevention of stroke is a leading challenge globally and only a few strategies have been tested to be effective in supporting stroke survivors. The system-integrated and technology-enabledmodel of care (SINEMA) intervention, a primary care-based and technology-enabled model of care, has been proven effective in strengthening the secondary prevention of stroke in rural China. The aimof this protocol is to outline themethods for the cost-effectiveness evaluation of the SINEMA intervention to better understand its potential economic benefits. Methods: The economic evaluation will be a nested study based on the SINEMA trial; a cluster-randomized controlled trial implemented in 50 villages in rural China. The effectiveness of the intervention will be estimated using quality-adjusted life years for the cost-utility analysis and reduction in systolic blood pressure for the cost-effectiveness analysis. Health resource and service use and program costs will be identified, measured, and valued at the individual level based on medication use, hospital visits, and inpatients' records. The economic evaluation will be conducted from the perspective of the healthcare system. Conclusion: The economic evaluation will be used to establish the value of the SINEMA intervention in the Chinese rural setting, which has great potential to be adapted and implemented in other resource-limited settings. [ABSTRACT FROM AUTHOR]

The objective of this study was to compare the concurrent and construct validity, as well as the sensitivity of 5 multiattribute utility instruments (MAUIs), including the Assessment of Quality of Life-6D (AQoL-6D), EQ-5D-Y, Health Utilities Index (HUI)-2 and HUI-3, and the Child Health Utility 9D, 1 generic pediatric quality of life instrument, with 3 routinely collected outcome measures in Australian mental health services (Strengths and Difficulties Questionnaire, Clinical Global Assessment Scale [CGAS] and the Health of the Nation Outcome Scale for Children and Adolescents) in children and adolescents diagnosed of internalizing (eg, anxiety/depression), externalizing (eg, attention deficit hyperactivity disorder/conduct disorders), and trauma/stress related mental disorders. A cross-sectional survey of measures, including demographic and basic treatment information, in children/adolescents recruited via 5 child and youth mental health services in Queensland and Victoria, Australia. Measures were either proxy or self-report completed, the CGAS and the Health of the Nation Outcome Scale for Children and Adolescents were clinician completed. The sample included 426 participants and had a mean age of 13.7 years (range 7-18 years). Utilities (as calculated from MAUIs) were generally lower in older adolescents and those with internalizing disorders. All MAUIs and self-reported clinical measures significantly correlated with each other (absolute correlation range 0.40-0.90), with the AQoL-6D showing generally higher levels of correlations. Correlations between the MAUIs and clinician/proxy-reported measures were weak, regardless of diagnosis (absolute correlation range 0.09-0.47). Generally, EQ-5D-Y, HUI-2, and AQoL-6D were more sensitive than Child Health Utility 9D and HUI-3 when distinguishing between different severities according to clinician-assessed CGAS (effect size range 0.17-0.84). The study showed that the commonly used MAUIs had good concurrent and construct validity compared with routinely used self-complete measures but poor validity when compared with clinician/proxy-completed measures. These findings generally held across different diagnoses. • Multiattribute utility instruments (MAUIs), such as the EQ-5D-Y, Child Health Utility 9D, Health Utilities Index-2 and -3, and the Assessment of Quality of Life-6D—used to assess quality-adjusted life-years—have not been psychometrically evaluated in children/adolescents with mental disorders. • This study evaluated commonly used MAUIs in children/adolescents with mental disorders and compared them with frequently used non-MAUI outcome measures—both self- and clinician/parent reported. • The MAUIs were found to have good concurrent and construct validity when compared with self-reported outcome measures but poor validity when compared with clinician or parent/guardian-reported measures. • This research raises the question of whether quality-adjusted life-year gains should be exclusively estimated using self-reported measures. [ABSTRACT FROM AUTHOR]