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Abstract Background Hybrid emergency room (ER) systems, consisting of an angiography-computed tomography (CT) machine in a trauma resuscitation room, are reported to be effective for reducing death from exsanguination in trauma patients. We aimed to investigate the cost-effectiveness of a hybrid ER system in severe trauma patients without severe traumatic brain injury (TBI). Methods We conducted a cost-utility analysis comparing the hybrid ER system to the conventional ER system from the perspective of the third-party healthcare payer in Japan. A short-term decision tree and a long-term Markov model using a lifetime time horizon were constructed to estimate quality-adjusted life years (QALYs) and associated lifetime healthcare costs. Short-term mortality and healthcare costs were derived from medical records and claims data in a tertiary care hospital with a hybrid ER. Long-term mortality and utilities were extrapolated from the literature. The willingness-to-pay threshold was set at $47,619 per QALY gained and the discount rate was 2%. Deterministic and probabilistic sensitivity analyses were conducted. Results The hybrid ER system was associated with a gain of 1.03 QALYs and an increment of $33,591 lifetime costs compared to the conventional ER system, resulting in an ICER of $32,522 per QALY gained. The ICER was lower than the willingness-to-pay threshold if the odds ratio of 28-day mortality was < 0.66. Probabilistic sensitivity analysis indicated that the hybrid ER system was cost-effective with a 79.3% probability. Conclusion The present study suggested that the hybrid ER system is a likely cost-effective strategy for treating severe trauma patients without severe TBI.

Abstract Background Expansion of designated cycling networks increases cycling for transport that, in turn, increases physical activity, contributing to improvement in public health. This paper aims to determine whether cycle-network construction in a large city is cost-effective when compared to the status-quo. We developed a cycle-network investment model (CIM) for Oslo and explored its impact on overall health and wellbeing resulting from the increased physical activity. Methods First, we applied a regression technique on cycling data from 123 major European cities to model the effect of additional cycle-networks on the share of cyclists. Second, we used a Markov model to capture health benefits from increased cycling for people starting to ride cycle at the age of 30 over the next 25 years. All health gains were measured in quality-adjusted life years (QALYs). Costs were estimated in US dollars. Other data to populate the model were derived from a comprehensive literature search of epidemiological and economic evaluation studies. Uncertainty was assessed using deterministic and probabilistic sensitivity analyses. Results Our regression analysis reveals that a 100 km new cycle network construction in Oslo city would increase cycling share by 3%. Under the base-case assumptions, where the benefits of the cycle-network investment relating to increased physical activity are sustained over 25 years, the predicted average increases in costs and QALYs per person are $416 and 0.019, respectively. Thus, the incremental costs are $22,350 per QALY gained. This is considered highly cost-effective in a Norwegian setting. Conclusions The results support the use of CIM as part of a public health program to improve physical activity and consequently avert morbidity and mortality. CIM is affordable and has a long-term effect on physical activity that in turn has a positive impact on health improvement.

Summary: Background: A growing literature has documented the high global morbidity, mortality and mental health burden associated with the current Covid-19 pandemic. In this paper, we aimed to quantify the total utility and quality of life loss resulting from Covid-19-related government restrictions imposed at the national, regional and global levels. Methods: We conducted quality of life online surveys in France, India, Italy, UK and the United States of America between June 21st and September 13th 2021, and used regression models to estimate the average quality of life loss due to light and severe restrictions in these countries. We then combined estimated disutility weights from the pooled sample with the latest data on Covid-19 restrictions exposure in each country to estimate the total disutility generated by restrictions at the national, regional and global level. We also embedded a discrete choice experiment (DCE) into the online survey to estimate average willingness to pay to avoid specific restrictions. Findings: A total of 947 surveys were completed. Thirty-five percent of respondents were female, and 69.5% were between 18 and 39 years old. The weighted average utility weight was 0.71 (95% CIs 0.69–0.74) for light restrictions, and 0.65 (0.63–0.68) for severe restrictions. At the global scale, this implies a total loss of 3259 million QALYs (95% 3021, 3496) as of September 6th, 2021, with the highest burden in lower and upper middle-income countries. Utility losses appear to be particularly large for closures of schools and daycares as well as restaurants and bars, and seem relatively small for wearing masks and travel restrictions. Interpretation: The results presented here suggest that the QALY losses due to restrictions are substantial. Future mitigation strategies should try to balance potential reductions in disease transmission achievable through specific measures against their respective impact on quality of life. Additional research is needed to determine differences in restriction-specific disutilities across countries, and to determine optimal policy responses to similar future disease threats. Funding: No funding was received for this project.

Introduction: Multiple sclerosis (MS) is an inflammatory disease in which the myelin sheaths of the nerve cells in the brain and spinal cord, which are responsible for communication, are destroyed and cause physical signs and symptoms. According to studies, anti-CD20 monoclonal antibodies have significant results in the treatment of this disease. Thus, the aim of the present study was to determine the cost-effectiveness of rituximab against natalizumab in the patients with RRMS in southern Iran in 2020.Methods: This is an economic evaluation including cost-effectiveness analysis in which the Markov model with a lifetime horizon was used. The study sample consisted of 120 patients randomly selected from among those referred to the MS Association and the Special Diseases Unit of Shiraz University of Medical Sciences. In this study, the costs were collected from a societal perspective, and the outcomes were obtained in the form of Quality Adjusted Life Years (QALY) and the mean relapse rate. The TreeAge pro 2020 and Excel 2016 software were used for data analysis.Results: The comparative study of rituximab and natalizumab showed that the patients receiving rituximab had lower costs ($ 58,307.93 vs. $ 354,174.85) and more QALYs (7.77 vs. 7.65). In addition, the incidence of relapse by rituximab was lower compared to natalizumab (1.15 vs. 2.57). The probabilistic one-way sensitivity analysis showed the robustness of the results. The scatter plots also showed that rituximab was more cost-effective for the patients in 100% of the simulations for the threshold of < $ 37,641.Discussion and Conclusion: According to the results of this study, rituximab had higher cost-effectiveness than natalizumab. Therefore, it could be a priority for RRMS patients compared to natalizumab because it reduced treatment costs and increased effectiveness. [ABSTRACT FROM AUTHOR]

To evaluate the cost-effectiveness of mepolizumab added to standard of care (SOC) compared with SOC alone among patients with severe uncontrolled eosinophilic asthma in the Singapore setting. A Markov model with three health states (asthma on mepolizumab and SOC, asthma on SOC alone, and death) was developed from a healthcare system perspective over a lifetime horizon. During each 4-week cycle, patients in the non-death health states could experience asthma exacerbations requiring oral corticosteroid burst, emergency department visit, or hospitalization. Asthma-related mortality following an exacerbation or all-cause mortality could also occur at each cycle. The model was populated using local costs while utilities were derived from international literature. Transition probabilities were obtained from a mixture of Singapore-specific and internationally published data. The base-case analysis comparing mepolizumab plus SOC with SOC alone resulted in an incremental cost-effectiveness ratio (ICER) of SGD335 486 (USD238 195) per quality-adjusted life-year (QALY) gained. Sensitivity analysis demonstrated that the ICER was most sensitive to the price of mepolizumab, followed by the proportion of exacerbations which required hospital intensive care. Despite restricting mepolizumab use to patients with a higher baseline exacerbation rate (3 in the past year) in a scenario analysis, the ICER remained high at SGD238 876 (USD 169 602) per QALY gained. At its current price, mepolizumab is not considered a cost-effective use of healthcare resources in Singapore. Substantial price reductions for mepolizumab are required to improve its cost-effectiveness to an acceptable range. These results will be useful to inform national funding decisions. [ABSTRACT FROM AUTHOR]

Background: End-of-life care is a driver of increasing healthcare costs; however, palliative care interventions may significantly reduce these costs. Economic evaluations that measure the incremental cost per quality adjusted life years (QALY) are warranted to inform cost-effectiveness of the intervention relative to a comparator and permit evaluation of investment against other therapeutic interventions. Evidence from the literature up to 2011 indicates a scarcity of cost-utility studies in palliative care research. Aim: This literature review evaluates economic studies published between 2011 and 2019 to determine whether the methods of economic evaluations have evolved since 2011. Design and Data Sources: A literature search was completed using CENTRAL, OVID MEDLINE, EMBASE and other sources for publications between 2011 and 2019. Study characteristics, methodology and key findings of publications that met the inclusion criteria were reviewed. Quality of studies were assessed using indicators developed by authors of the previous literature review. Results: 46 papers were included for qualitative synthesis. Among them only 6 studies conducted formal cost-effectiveness evaluations-of these 5 measured QALYs and 1 employed probabilistic analyses. In addition, with the exception of 1 costing analysis, all other economic evaluations undertook a healthcare payer perspective. Quality of evidence were comparable to the previous literature review published in 2011. Conclusion: Despite the small increase in the number of cost-utility studies, the methods of palliative care economic evaluations have not evolved significantly since 2011. More probabilistic cost-utility analyses of palliative care interventions from a societal perspective are necessary to truly evaluate the value for money. [ABSTRACT FROM AUTHOR]

Medicare's Hospital Value-Based Purchasing Program (HVBP) is the first national pay-for-performance program to combine measures of quality of care with a measure of episode spending. We estimated the implicit tradeoffs between mortality reduction and spending reduction. To earn points in HVBP, a hospital can either lower mortality or reduce spending, creating a tradeoff between the 2 measures. We analyzed the quality performance and earned points of 2814 hospitals using publicly available data. We then quantified the tradeoffs between spending and mortality in terms of quality-adjusted life-years (QALYs). If incentives in the program were balanced, then the tradeoff between spending and QALYs should be comparable with those of high-value health interventions, roughly $50,000 to $200,000 per QALY. Instead, the tradeoff in HVBP was about $1.2 million per QALY. HVBP overvalues improvements in quality of care relative to spending reductions. We propose 2 possible policy adjustments that could improve incentives for hospitals to deliver high-value care. [ABSTRACT FROM AUTHOR]

Purpose: This study aimed to determine whether continual electronic patient-reported outcome (ePRO) measurements at home can capture the fluctuations in health-related quality of life (HRQOL) scores between visits. Methods: We performed a randomized controlled trial to compare the scores obtained by standard practice (paper-based measurements in the hospital) to scores by continuous measurement of ePRO at home. Metastatic cancer patients were randomly assigned to either the paper-based (n = 50) or the ePRO group (n = 52). EQ-5D-5L and EORTC QLQ C-30 scores were obtained on 3 different chemotherapy days in the paper-based group. Meanwhile, scores were obtained on the chemotherapy day and on days 3, 7, 10, and 14 in the ePRO group during 2 cycles. The first hypothesis of our study was that both scores at the same time points would be equivalent despite different measurement frequency, place, or mode of measurement. The second hypothesis was that PRO score–adjusted time would be different between the groups. For equivalence, the endpoint was the mean EQ-5D-5L index value on the chemotherapy day before the outpatient treatment. Only if equivalence was shown, quality-adjusted life-days (QALDs) were considered using all the data. Results: The adjusted mean difference in the EQ-5D-5L index was determined to be −0.013 (95% confidence interval [CI]: −0.049 to 0.022); the 95% CI did not exceed the equivalence margin. Similarly, the mean difference in global health status (2.28 [95% CI: −2.55 to 7.11]) also showed equivalence. However, the QALD by EQ-5D-5L was significantly lower in the ePRO group by 1.36 per 30 d (95% CI: −2.22 to −0.51; P = 0.0021). Conclusions: Continual measurements of the HRQOL at home by ePRO may yield more detailed profiles of the HRQOL. [ABSTRACT FROM AUTHOR]

Approximately, 4% of Stage IV colorectal cancers (CRC) are microsatellite instability-high (MSI-H)/deficient mismatch repair (dMMR) tumors. Patients with metastatic MSI-H/dMMR CRC receiving conventional therapies experience lower response rates and tend to have worse overall survival compared with patients with microsatellite stable (MSS)/proficient mismatch repair (pMMR) CRC. Pembrolizumab received FDA approval in 2020 for first-line treatment of Stage IV MSI-H/dMMR CRC based on significantly longer progression-free survival versus standard of care (SoC, 5-fluorouracil-based therapy with or without bevacizumab or cetuximab). This study evaluated the cost-effectiveness of pembrolizumab vs. SoC as per KEYNOTE-177 and other first-line treatments for MSI-H/dMMR CRC from a US healthcare system perspective. A three-health-state partitioned-survival model was built using progression-free and overall survival data from KEYNOTE-177 and a network meta-analysis. Utilities were derived from KEYNOTE-177 EQ-5D-3L data. Drug acquisition, administration, AE, surgery, monitoring, subsequent treatment, and terminal care costs were included. Sensitivity and scenario analyses were performed, including utilizing a state-transition model structure and adopting a societal perspective. Over a lifetime time horizon, pembrolizumab and SoC were associated with total QALYs of 4.85 and 3.23, and total costs of $381,735 and $370,465, respectively, resulting in an ICER of $6,984 per QALY. QALY gains were mainly driven by extended survival with pembrolizumab. Pembrolizumab incurred higher drug acquisition costs relative to SoC but was cost-saving in terms of drug administration, AE, monitoring, subsequent treatment, and terminal care. Pembrolizumab dominated FOLFOX + panitumumab, FOLFOXIRI, and FOLFOXIRI + bevacizumab, and presented ICERs of $35,220 and $276 against XELOX and XELOX + bevacizumab. Results were robust to sensitivity and scenario analyses. Pembrolizumab is highly cost-effective for the first-line treatment of unresectable or metastatic MSI-H/dMMR CRC in the US at a willingness-to-pay threshold of $100,000/QALY. Pembrolizumab is a highly cost-effective option for the first-line treatment of patients with unresectable or metastatic MSI-H/dMMR colorectal cancer in the United States at a willingness-to-pay threshold of $100,000. Compared with the current standard of care for these patients, pembrolizumab: Increases survival due to delaying and preventing progression; Increases QALYs due to longer survival, improvement in HRQoL in the progression-free health state, and fewer Grade 3+ adverse events; Reduces costs associated with administering treatment, managing adverse events, monitoring post-progression disease, providing subsequent treatment, and providing terminal care; and Reduces indirect health care costs when taking a societal perspective due to productivity gains from delaying and preventing progression and death, less frequent treatment administration and less frequent Grade 3+ adverse events. [ABSTRACT FROM AUTHOR]

This research aimed to review the theoretical and methodological aspects of the quality-adjusted life year (QALY) which give rise to potential for bias against certain patient populations, including those with problems with walking or an inability to walk (ambulatory disabilities), when health technology assessment decisions rely on QALY gain to show cost-effectiveness. Societal preferences for treating ambulatory versus non-ambulatory patients were also investigated. We reviewed published literature to identify information on theoretical underpinnings of the QALY, measurement of utilities for QALY assessment, and empirical evidence of societal preferences for the treatment of ambulatory and non-ambulatory patients. Health states which represent mobility impairment and the inability to walk receive low valuation from general public preferences. Non-ambulatory patients, for example those with advanced neuromuscular disease, have lower utilities determined by standardized preference-based measurement (PBM) tools. Any treatment that increases survival but could not restore ambulation would result in lower lifetime QALY gains for non-ambulatory versus ambulatory patients. Treatments could therefore potentially be deemed less cost-effective, or not cost-effective at all for this patient population. Empirical research indicates a societal preference for equal treatment of patients regardless of ambulatory status. The main limitation of our review was the non-systematic approach to evidence search and review, however, given the broad scope of content required to meet the aims of the review, we believe that the targeted approach was appropriate. The evidence presented in this article highlights the need for alternatives to strict QALY-based approaches to prevent avoidable health inequities when determining cost-effectiveness of healthcare interventions for non-ambulatory populations against fixed cost-effectiveness thresholds. An alternative metric, the Equal Value of Life Years Gained (evLYG), has been proposed as a supplementary measure for use alongside the QALY for its potential to alleviate bias against disabled patient populations during the assessment of healthcare treatments. [ABSTRACT FROM AUTHOR]

We assess the health-related costs of the first two waves of COVID-19 pandemic in Iceland, Norway and Sweden, based on the number of quality-adjusted life-years (QALYs) lost from excess deaths, reduced health during a fixed period of the illness, and the costs of inpatient hospital days. The cost per capita is estimated at 664 euros in Sweden (in total 1.4 percent of GDP), 84 euros in Norway (0.12 percent of GDP), and 115 euros in Iceland (0.2 percent of GDP). As a demonstration of use for policy evaluation, we do a benefit-cost analysis of the closure of upper secondary schools in Sweden during the first wave. [ABSTRACT FROM AUTHOR]

Background: Due to the lack of a constant Willingness to Pay per one additional Quality Adjusted Life Years gained based on the preferences of Iran's general public, the cost-effectiveness of health system interventions is unclear and making it challenging to apply economic evaluation to health resources priority setting.Methods: We have measured this cost-effectiveness threshold with the participation of 2854 individuals from five provinces, each representing an income quintile, using a modified Time Trade-Off-based Chained-Approach. In this online-based empirical survey, to extract the health utility value, participants were randomly assigned to one of two green (21121) and yellow (22222) health scenarios designed based on the earlier validated EQ-5D-3L questionnaire.Results: Across the two health state versions, mean values for one QALY gain (rounded) ranged from $6740-$7400 and $6480-$7120, respectively, for aggregate and trimmed models, which are equivalent to 1.35-1.18 times of the GDP per capita. Log-linear Multivariate OLS regression analysis confirmed that respondents were more likely to pay if their income, disutility, and education level were higher than their counterparts.Conclusions: In the health system of Iran, any intervention that is with the incremental cost-effectiveness ratio, equal to and less than 7402.12 USD, will be considered cost-effective. [ABSTRACT FROM AUTHOR]

Background: Electroconvulsive therapy (ECT) has long been used for treating individuals with treatment-resistant depression (TRD). Esketamine has recently emerged as a new treatment for TRD due to its rapid antidepressant effects. To further inform the decision regarding choice of treatment, this paper aims to evaluate whether ECT or esketamine is the more cost-effective option. Methods: The cost-effectiveness was derived as cost per quality-adjusted life-year (QALY) using a Markov model from a societal and life-time perspective. The incremental cost-effectiveness ratio (ICER) was calculated. Health states included different depression and remission states and death. Data to populate the model was derived from randomised controlled trials and other research. Various sensitivity analyses were carried out to test the robustness of the model. Results: The base case scenario shows that ECT is cost-effective compared to esketamine and yields more QALYs at a lower cost. The sensitivity analysis shows that ECT is cost-effective in all scenarios and ECT dominates esketamine in 12 scenarios. Conclusions: This study found that, from a cost-effectiveness point of view, ECT should be the first-hand option for individuals with TRD, when other first line treatments have failed. Considering the lack of economic evaluation of ECT and esketamine, this study is of great value to decision makers. [ABSTRACT FROM AUTHOR]

Objectives To evaluate the 1-year cost-effectiveness between three different initial treatment strategies in autoantibody-negative RA patients, according to 2010 criteria. Methods For this analysis we selected all RA patients within the intermediate probability stratum of the treatment in the Rotterdam Early Arthritis Cohort (tREACH) trial. The tREACH had a treat-to-target approach, aiming for low DAS <2.4, and treatment adjustments could occur every 3 months. Initial treatment strategies consisted of MTX 25 mg/week (initial MTX, iMTX), iHCQ 400 mg/day or an oral glucocorticoids tapering scheme without DMARDs (iGCs). Data on quality-adjusted life-years, measured with the European Quality of Life 5-Dimensions 3 Levels (EQ-5D-3L), healthcare and productivity costs were used. Results Average quality-adjusted life-years (s. d.), for iMTX, iHCQ and iGCs were respectively 0.71 (0.14), 0.73 (0.14) and 0.71 (0.15). The average total costs (s. d.) for iMTX, iHCQ and iGCs were, respectively, €10 832 (14.763), €11 208 (12.801) and €10 502 (11.973). Healthcare costs were mainly determined by biological costs, which were significantly lower in the iHCQ group compared with iGCs (P  < 0.05). However, costs due to presenteeism were the highest in the iHCQ group (55%) followed by iMTX (27%) and iGCs (18%). The incremental cost-effectiveness ratios did not differ between treatment strategies. At a willingness-to-pay level of €50 000, the Dutch threshold for reimbursement of medical care, iHCQ had the highest probability (38.7%) of being cost-effective, followed by iGCs (31.1%) and iMTX (30.2%). Conclusion iHCQ had the lowest healthcare and highest productivity costs, resulting in a non-significant incremental cost-effectiveness ratio. However, iHCQ had the highest chance of being cost-effective at the Dutch willingness-to-pay threshold for healthcare reimbursement. Therefore, we believe that iHCQ is a good alternative to iMTX in autoantibody-negative RA patients, but validation is needed. Clinical trial registration number ISRCTN26791028 [ABSTRACT FROM AUTHOR]

To assess the cost-effectiveness and cost utility of sitagliptin/metformin for the treatment of type 2 diabetes mellitus compared to those of glibenclamide/metformin in a semiprivate hospital and to compare the cost-effectiveness and cost utility of sitagliptin/metformin in a semiprivate hospital to those in the public health system (PHS) of Ecuador in 2019. A cost-effectiveness study considering the probability of cardiovascular death as the outcome and quality-adjusted life-year as a measure of utility, estimating direct medical costs in US dollars by a model case from the perspective of the third payer. The results will be presented as an incremental cost-effectiveness ratio. One-way and 2-way sensitivity analyses with tornado diagrams were performed. Direct medical costs were lower at the hospital than from the PHS in Ecuador. Considering the drugs metformin/sitagliptin, the total cost was $35.69 less in the hospital ($880.38) than from the comparator ($916.07). The highest percentage of direct medical costs corresponded to drugs (between 63.94% and 84.65%). An ICER of –$19 131.61 was obtained at the Hospital Un Canto a la Vida and –$1621.85 at PHS. In addition, the cost per quality-adjusted life-year earned was $611.11. Sensitivity analysis showed that the probability of drug use and the relative risk of cardiovascular death associated with such prescription were parameters that most affected the model. The combination therapy metformin/sitagliptin compared to metformin/glibenclamide was shown not to be cost-effective in the Hospital Un Canto a la Vida, and highly cost-effective in the PHS. • Some controversy persists regarding the drug of choice to be administered in conjunction with metformin when control goals are not achieved in patients with diabetes. Evidence of good quality did not show significant differences among 9 families of antidiabetic drugs; thus, it is recommended to rely on the specific characteristics of the patients to prescribe a second antidiabetic. Cost-effectiveness and cost-utility studies of dipeptidyl peptidase 4 inhibitors versus sulfonylureas showed that the former had better results. • The therapeutic combination of metformin/sitagliptin compared to metformin/glibenclamide was shown to be not cost-effective, because it exceeded the World Health Organization suggested ceiling of 3 times a country's gross domestic product per capita, in a second-level semiprivate hospital. Instead, in PHS the metformin/sitagliptin alternative was highly cost-effective. In the 2-way sensitivity analysis, the variable that modified the incremental cost-effectiveness ratio in both scenarios was the probability of using the metformin/glibenclamide combination. • The frequency of prescription of the different combinations of antidiabetics is a parameter to be considered so that new therapeutic options for diabetes mellitus can be cost-effective in clinical practice, especially considering that the drug cost is the most important parameter in direct medical costs. [ABSTRACT FROM AUTHOR]

Physical inactivity is the leading cause of non‐communicable diseases, and further research on the cost‐effectiveness of interventions that target inactivity is warranted. Socioeconomic status is vital in this process. We aim to evaluate the cost‐effectiveness of a cycle‐network expansion plan in Oslo compared to the status quo by income quintiles. We applied a Markov model using a public payer perspective. Health outcomes were measured by quality‐adjusted life years (QALYs) gained from the prevention of coronary heart disease, stroke, type 2 diabetes, and cancer. We measured equity impact by the concentration index and social welfare using the achievement index. We conducted sensitivity analyses. The intervention was generally more costly and more effective than the status quo. Incremental cost per QALY falls with income quintile, ranging from $10,098 in the richest quintile to $23,053 per QALY gained in the poorest quintile. The base‐case intervention increased health inequality. However, a scenario targeting low‐income quintiles reduced inequality and increased social welfare. In conclusion, the cycle‐network expansion is likely to be cost‐effective, but with equity concerns. If decision makers care about health inequalities, the disadvantaged groups could be targeted to produce more equitable and socially desirable outcomes instead of a uniform intervention across income quintiles. [ABSTRACT FROM AUTHOR]

Purpose: To examine how mobility and mobility impairment affect quality of life; to develop a descriptive system (i.e., questions and answers) for a novel mobility-related quality of life outcome measure. Materials and methods: Data were collected through semi-structured interviews. Participants were recruited predominantly from NHS posture and mobility services. Qualitative framework analysis was used to analyse data. In the first stage of analysis the key dimensions of mobility-related quality of life were defined, and in the second stage a novel descriptive system was developed from the identified dimensions. Results: Forty-six interviews were conducted with 37 participants (aged 20-94 years). Participants had a wide range of conditions and disabilities which impaired their mobility, including cerebral palsy, multiple sclerosis, and arthritis. Eleven dimensions of mobility-related quality of life were identified: accessibility, safety, relationships, social inclusion, participation, personal care, pain and discomfort, independence, energy, self-esteem, and mental-wellbeing. A new outcome measure, known as MobQoL, was developed. Conclusions: Mobility and mobility impairment can have significant impacts on quality of life. MobQoL is the first outcome measure designed specifically to measure the impact of mobility on quality of life, and therefore has utility in research and practice to measure patient outcomes related to rehabilitation. [ABSTRACT FROM AUTHOR]

Objectives: The importance of economic evaluations of mental healthcare interventions is increasingly recognized. Despite the multitude of available quality of life instruments, concerns have been raised regarding the content validity of these instruments, and hence suitability for use in mental health. The aim of this paper, therefore, was to assess the content validity and the suitability of existing quality of life instruments for use in economic evaluations in mental health problems.Methods: In order to identify available quality of life instruments used in people with mental health problems, a systematic review was performed using the Embase, Medline and PsycINFO databases (time period January 2012 to January 2018). Two reviewers independently assessed study eligibility and executed data extraction. The evaluation framework of Connell and colleagues was used to assess whether the identified quality of life instruments cover the dimensions valued highly by people with mental health problems. Two reviewers independently mapped the content of each identified instrument onto the evaluation framework and indicated the extent to which the instrument covered each of the dimensions of the evaluation framework.Results: Searches of databases yielded a total of 5727 references. Following duplicate removal and double-independent screening, 949 studies were included in the qualitative synthesis. A total of 44 unique quality of life instruments were identified, of which 12 were adapted versions of original instruments. The best coverage of the dimensions of the evaluation framework of Connell and colleagues was by the WHOQOL-100, S-QoL, SQLS, EDQoL, QLI and the IMHQOL, but none fully covered all dimensions of the evaluation framework.Conclusions: The results of this study highlight the multitude of available quality of life instruments used in people with mental health problems and indicate that none of the available quality of life instruments fully cover the dimensions previously found to be important in people with mental health problems. Future research should explore the possibilities of refining or expanding existing instruments as well as the development and testing of new quality of life instruments to ensure that all relevant quality of life dimensions for people with mental health problems are covered in evaluations. [ABSTRACT FROM AUTHOR]

Aims: There is limited published evidence for the cost-effectiveness of treatments for unresectable or metastatic endometrial cancer (mEC). The objective of this analysis was to assess the cost-effectiveness of pembrolizumab versus chemotherapy for previously treated unresectable or mEC, in women whose tumors have deficient mismatch repair (dMMR) or high microsatellite instability (MSI-H). The analysis was carried out from a US healthcare payer perspective. Materials and methods: A lifetime partitioned survival model comprising three health states (progression-free, progressed disease and death) was constructed. Chemotherapy was represented by singleagent paclitaxel or doxorubicin. Overall survival, progression-free survival and time on treatment data for pembrolizumab were obtained from a Phase II clinical study that included women with previously treated dMMR/MSI-H unresectable or mEC (KEYNOTE-158, NCT02628067). Survival data for chemotherapy were obtained from a published Phase III study for previously treated advanced endometrial cancer. Costs included were drug acquisition and administration, health-state, end-of-life, and adverse event management. Costs were presented in 2019 US$. Outcomes were calculated as quality-adjusted life-years (QALYs), using EQ-5D data from KEYNOTE-158. Model results were tested extensively in deterministic and probabilistic sensitivity analyses. Results: Results demonstrated that pembrolizumab is a highly cost-effective treatment option when compared with chemotherapy, with estimated deterministic and probabilistic incremental cost-effectiveness ratios (ICERs) of $58,165 and $57,668 per QALY gained, respectively. Pembrolizumab was associated with a large QALY and life-year gain per person versus chemotherapy over the model time horizon (deterministic 4.68 life year gain, 3.80 QALYs), with the majority of QALYs accrued in the progression-free health state. Limitations: The key limitation of the analysis was the lack of comparative effectiveness data for pembrolizumab versus chemotherapy. Conclusions: Pembrolizumab is a highly cost-effective treatment option when compared with chemotherapy for women with previously treated dMMR/MSI-H unresectable or mEC. Results were robust to the changes in parameters and assumptions explored. [ABSTRACT FROM AUTHOR]

Background: In August 2018, the US FDA granted accelerated approval for nivolumab in small cell lung cancer (SCLC) that has progressed after platinum-based chemotherapy and at least one other line of therapy. The objective of this study was to evaluate the cost-effectiveness of nivolumab vs. usual care as third-line (3 L) therapy for patients with recurrent SCLC (rSCLC) from the health payer perspective. Given the potential for a meaningful fraction of treated patients to achieve long-term response to nivolumab, we also assessed the impact of using mixture cure modeling (MCM) vs. parametric survival modeling on survival estimates and cost-effectiveness from the US Medicare payer perspective. Methods: We created a partitioned survival decision model to assess the cost-effectiveness of 3 L nivolumab vs. usual care in rSCLC, based on observed US treatment patterns. Using this approach, we assessed the impact of extrapolating long-term survival from the CheckMate 032 trial, using both MCM and standard parametric curve fits. Nivolumab survival, resource use, and Grade 3/4 adverse event rates were derived from CheckMate 032. Usual care survival, resource use, and costs were derived from an analysis of patients receiving 3 L treatment for rSCLC in the SEER-Medicare registry. We applied 2020 Wholesale Acquisition Cost for drugs and 2020 CMS reimbursement for procedures. Utilities were derived from the literature. We estimated life years (LY), quality-adjusted life years (QALYs), and costs over a lifetime horizon. Results: MCM and parametric survival model extrapolations resulted in 0.43 versus 0.38 more LYs, 0.34 versus 0.30 more QALYs, and $69,308 versus $61,336 more expenditure for nivolumab vs. usual care, respectively. The costs per QALY gained using mixture cure versus parametric survival modeling were $204,386 and $207,431, respectively. Conclusions: Mixture cure modeling was equivalent compared to parametric modeling in estimating the cost-effectiveness of nivolumab-based therapy due to the small fraction of patients achieving a long-term response with nivolumab (12.9%). [ABSTRACT FROM AUTHOR]

Abstract Background Matching survival of a donor kidney with that of the recipient (longevity matching), is used in some kidney allocation systems to maximize graft-life years. It is not part of the allocation algorithm for Australia. Given the growing evidence of survival benefit due to longevity matching based allocation algorithms, development of a similar kidney allocation system for Australia is currently underway. The aim of this research is to estimate the impact that changes to costs and health outcomes arising from ‘longevity matching’ on the Australian healthcare system. Methods A decision analytic model to estimate cost-effectiveness was developed using a Markov process. Four plausible competing allocation options were compared to the current kidney allocation practice. Models were simulated in one-year cycles for a 20-year time horizon, with transitions through distinct health states relevant to the kidney recipient. Willingness to pay was considered as AUD 28000. Results Base case analysis indicated that allocating the worst 20% of Kidney Donor Risk Index (KDRI) donor kidneys to the worst 20% of estimated post-transplant survival (EPTS) recipients (option 2) and allocating the oldest 25% of donor kidneys to the oldest 25% of recipients are both cost saving and more effective compared to the current Australian allocation practice. Option 2, returned the lowest costs, greatest health benefits and largest gain to net monetary benefits (NMB). Allocating the best 20% of KDRI donor kidneys to the best 20% of EPTS recipients had the lowest expected incremental NMB. Conclusion Of the four longevity-based kidney allocation practices considered, transplanting the lowest quality kidneys to the worst kidney recipients (option 2), was estimated to return the best value for money for the Australian health system.

Abstract Background Depressive disorders are associated with a high burden of disease. However, due to the burden posed by the disease on not only the sufferers, but also on their relatives, there is an ongoing debate about which costs to include and, hence, which perspective should be applied. Therefore, the aim of this paper was to examine whether the change between healthcare payer and societal perspective leads to different conclusions of cost-utility analyses in the case of depression. Methods A systematic literature search was conducted to identify economic evaluations of interventions in depression, launched on Medline and the Cost-Effectiveness Registry of the Tufts University using a ten-year time horizon (2008–2018). In a two-stepped screening process, cost-utility studies were selected by means of specified inclusion and exclusion criteria. Subsequently, relevant findings was extracted and, if not fully stated, calculated by the authors of this work. Results Overall, 53 articles with 92 complete economic evaluations, reporting costs from healthcare payer/provider and societal perspective, were identified. More precisely, 22 estimations (24%) changed their results regarding the cost-effectiveness quadrant when the societal perspective was included. Furthermore, 5% of the ICURs resulted in cost-effectiveness regarding the chosen threshold (2% of them became dominant) when societal costs were included. However, another four estimations (4%) showed the opposite result: these interventions were no longer cost-effective after the inclusion of societal costs. Conclusions Summarising the disparities in results and applied methods, the results show that societal costs might alter the conclusions in cost-utility analyses. Hence, the relevance of the perspectives chosen should be taken into account when carrying out an economic evaluation. This systematic review demonstrates that the results of economic evaluations can be affected by different methods available for estimating non-healthcare costs.

Abstract Background While hospitals remain the most common place of death in many western countries, specialised palliative care (SPC) at home is an alternative to improve the quality of life for patients with incurable cancer. We evaluated the cost-effectiveness of a systematic fast-track transition process from oncological treatment to SPC enriched with a psychological intervention at home for patients with incurable cancer and their caregivers. Methods A full economic evaluation with a time horizon of six months was performed from a societal perspective within a randomised controlled trial, the DOMUS trial ( Clinicaltrials.gov : NCT01885637). The primary outcome of the health economic analysis was a incremental cost-effectiveness ratio (ICER), which is obtained by comparing costs required per gain in Quality-Adjusted Life Years (QALY). The costs included primary and secondary healthcare costs, cost of intervention and informal care from caregivers. Public transfers were analysed in seperate analysis. QALYs were measured using EORTC QLQ-C30 for patients and SF-36 for caregivers. Bootstrap simulations were performed to obtain the ICER estimate. Results In total, 321 patients (162 in intervention group, 159 in control group) and 235 caregivers (126 in intervention group, 109 in control group) completed the study. The intervention resulted in significantly higher QALYs for patients when compared to usual care (p-value = 0.026), while being more expensive as well. In the 6 months observation period, the average incremental cost of intervention compared to usual care was €2015 per patient (p value

Chakrapani Balijepalli,1 Lakshmi Gullapalli,1 Eric Druyts,1 Kevin Yan,1 Kamal Desai,1 Stephane Barakat,2 Jason Locklin2 1Pharmalytics Group, Vancouver, British Columbia, Canada; 2Alexion Pharmaceuticals, Vaughan, Ontario, CanadaCorrespondence: Chakrapani Balijepalli Email chak.balijepalli@pharmalyticsgroup.comAbstract: Orphan drugs have high acquisition costs and when standard health technology assessment (HTA) approaches are used to assess their cost-effectiveness, they often appear not cost-effective. The Canadian Patented Medicine Review Board (PMPRB), through new regulations, will apply HTA assessment results from the Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d’excellence en santé et en services sociaux (INESSS) when setting the maximum price that can be charged for Category I patented medicines (treatments with an annual cost exceeding 150% of GDP per capita of Canada or with expected annual market size >$50M). Through these regulations, PMPRB has also established a willingness-to-pay threshold of CAD$200,000 or CAD$150,000 per quality adjusted life year (QALY) for medications with a prevalence of no more than 1 in 2000 across all approved indications. We reviewed the orphan drug submissions made to CADTH’s Common Drug Review (CDR) January 2015–May 2020 to understand how the methodology of assessing cost-effectiveness of orphan drugs has guided pricing in Canada. A total of 35 orphan drug submissions were assessed by CDR in this period, none of which met the willingness-to-pay threshold of CAD$50,000 per QALY. Only one drug met the CAD$200,000 per QALY for Therapeutic Criteria Level I, and two drugs met CAD$150,000 per QALY for other Therapeutic Criteria Levels proposed by PMPRB. Price reductions of 32– 99% were recommended for treatments that were approved in order to be listed for reimbursement. This review showed that the new PMPRB regulations could be creating challenges for manufacturers of rare disease treatments to meet Canadian pricing regulations. These regulations may jeopardize the launch of new medicines and limit opportunities to add to the development of real-world evidence of orphan drugs, which can be used in reimbursement approaches such as pay-for-performance.Keywords: orphan drugs, rare diseases, CADTH, QALY, HTA

Abstract Background Health systems are under pressure to deliver more effective care without expansion of resources. This is particularly pertinent to diseases like chronic kidney disease (CKD) that are exacting substantial financial burden to many health systems. The aim of this study is to systematically review the Cost Utility Analysis (CUA) evidence generated across interventions for CKD patients undergoing kidney transplant (KT). Methods A systemic review of CUA on the interventions for CKD patients undergoing KT was carried out using a search of the MEDLINE, CINAHL, EMBASE, PsycINFO and NHS-EED. The CHEERS checklist was used as a set of good practice criteria in determining the reporting quality of the economic evaluation. Quality of the data used to inform model parameters was determined using the modified hierarchies of data sources. Results A total of 330 articles identified, 16 met the inclusion criteria. Almost all (n = 15) the studies were from high income countries. Out of the 24 characteristics assessed in the CHEERS checklist, more than 80% of the selected studies reported 14 of the characteristics. Reporting of the CUA were characterized by lack of transparency of model assumptions, narrow economic perspective and incomplete assessment of the effect of uncertainty in the model parameters on the results. The data used for the economic model were satisfactory quality. The authors of 13 studies reported the intervention as cost saving and improving quality of life, whereas three studies were cost increasing and improving quality of life. In addition to the baseline analysis, sensitivity analysis was performed in all the evaluations except one. Transplanting certain high-risk donor kidneys (high risk of HIV and Hepatitis-C infected kidneys, HLA mismatched kidneys, high Kidney Donor Profile Index) and a payment to living donors, were found to be cost-effective. Conclusions The quality of economic evaluations reviewed in this paper were assessed to be satisfactory. Implementation of these strategies will significantly impact current systems of KT and require a systematic implementation plan and coordinated efforts from relevant stakeholders.

Objective To evaluate the cost-effectiveness of the current strategy combining universal vaccination with hepatitis B immunoglobulin (HBIG) treatment for infants of hepatitis B surface antigen (HBsAg) positive mothers compared with universal vaccination with hepatitis B vaccine only. Methods A decision tree model with a Markov process was constructed and used to simulate the lifetime of the birth cohort in Zhejiang Province during 2016. The current strategy was compared against universal vaccination with respect to costs and health effects. Costs were assessed from the health care system perspective. Health effects were measured by the number of hepatitis B virus (HBV) related diseases and deaths avoided and quality-adjusted life-years (QALYs) gained. The incremental cost‑effectiveness ratio (ICER) is calculated and compared to standard willingness-to-pay thresholds. A one-way sensitivity analysis and a probabilistic sensitivity analysis (PSA) were performed to assess parameter uncertainties. Results Over the cohort’s lifetime, 182 acute symptomatic infections, 2215 chronic infections, 872 cases of cirrhosis, 595 cases of hepatocellular carcinoma (HCC) and 1,350 HBV-related deaths among the cohort of 624,000 infants would be further avoided by the current strategy compared to universal vaccination. Universal vaccination was dominated by the current strategy that produced not only higher total QALYs, but also had lower costs. The results remained robust over a wide range of assumptions. Conclusions The current strategy was cost saving compared to universal vaccination, and continuing the current strategy is recommended to further decrease the burden of hepatitis B.

Thomas G Poder,1– 3 Nathalie Carrier3 1School of Public Health, Department of Management, Evaluation and Health Policy, University of Montreal, Montreal, QC, Canada; 2Centre de recherche de l’Institut universitaire en santé mentale de Montréal, CIUSSS de l’Est-de-l’Île-de-Montréal, Montreal, QC, Canada; 3Centre de recherche du CHUS, CIUSSS de l’Estrie-CHUS, Sherbrooke, QC, CanadaCorrespondence: Thomas G PoderSchool of Public Health, Department of Management, Evaluation and Health Policy, University of Montreal, Montreal, QC, CanadaEmail thomas.poder@umontreal.caBackground: Cost utility analysis is important for measuring the impact of chronic disease and helps clinicians and policymakers in patient management and policy decisions, but generic preference-based measures are not always considered in clinical studies.Objective: To evaluate if health-related quality-of-life (HRQoL)-specific questionnaires used in chronic low back pain (CLBP) can predict EQ-5D-5L utility scores.Methods: The data come from an online survey on low back pain conducted between October 2018 and January 2019. Health utility scores for EuroQol Five Dimensions Five Levels (EQ-5D-5L) were calculated with the recommended model of Xie et al. The EQ-5D-5L health states ranged from − 0.148 for the worst (55555) to 0.949 for the best (11111). Univariate and multivariate linear regression were performed to predict EQ-5D-5L with Oswestry Disability Index (ODI), Roland-Morris Disability Questionnaire (RMDQ) and clinical variables.Results: Analyses were performed in 408 subjects who completed the questionnaires EQ-5D-5L, ODI or RMDQ. Median (range) of EQ-5D-5L was 0.622 (− 0.072 to 0.905). There was high correlation between EQ-5D-5L and ODI (r=− 0.78, p< 0.001), while it was moderate with RMDQ (r=− 0.62, p< 0.001). The multivariate model to predict EQ-5D-5L with ODI explained 67.6% of variability, and the correlation between actual and predicted EQ-5D-5L was 0.82. Principal predictors were ODI, duration of LBP, invalidity, health satisfaction (0– 10 cm), life satisfaction (0– 10 cm), and intensity of pain today (0– 10 cm).Conclusion: Data from this study demonstrated that individual correlation between ODI and EQ-5D-5L was high, but moderate with RMDQ. Correlations between actual and predicted EQ-5D-5L from multivariate models were higher and very high. Considering these results, the multivariate model can be used in similar studies for patient with CLBP to estimate the utility scores from the ODI when the EQ-5D-5L was not measured.Keywords: low back pain, EQ-5D-5L, utility score, QALY, health-related quality of life

Abstract Background The oral health of Indigenous children in remote communities is much worse than other population groups in Australia. Providing and maintaining an oral health service is challenging due to the remoteness of communities, the associated high cost, and the low retention of clinical staff. An annual preventive intervention delivered by fly-in clinicians may be a more cost-effective way to manage this problem. In this analysis we estimate the cost-effectiveness of an annual professional intervention for the prevention of dental caries in children of a remote Indigenous community in Far North Queensland. Methods A cost-effectiveness analysis was conducted based on an annual preventive intervention protocol. This included treating all dental decay in those with disease, applying fissure sealants, a disinfectant swab, fluoride varnish and providing oral hygiene instructions and dietary advice to all participating school children. This study included an intervention group and a natural comparison group and both groups were followed-up for 2 years after the initial preventive intervention. A Markov model was built to assess the cost-effectiveness of the intervention compared with the usual care. Costs of treatment from the Queensland Department of Health were used and effectiveness was measured as quality-adjusted life years (QALYs) with the CHU-9D. One-way and probabilistic sensitivity analyses were conducted to identify key drivers and quantify uncertainty. Results The preventive intervention was found to be highly cost-effective. The incremental cost per QALY gained was AU$3747. Probability of new caries and seeking treatment were identified as the main drivers of the model. In probabilistic sensitivity analysis intervention was cost effective in 100% of simulations. Conclusion An annual preventive intervention for remote Indigenous communities in Australia is a highly cost-effective strategy to prevent dental caries and improve the quality of life of children.

Abstract Background This analysis evaluates the cost-effectiveness of insulin degludec (degludec) versus biosimilar insulin glargine U100 (glargine U100) in patients with type 1 (T1DM) and type 2 diabetes mellitus (T2DM) in Bulgaria. Methods A simple, short-term model was used to compare the treatment costs and outcomes associated with hypoglycaemic events with degludec versus glargine U100 in patients with T1DM and T2DM from the perspective of the Bulgarian National Health Insurance Fund. Cost-effectiveness was analysed over a 1-year time horizon using data from clinical trials. The incremental cost-effectiveness ratio (ICER) was the main outcome measure. Results In Bulgaria, degludec was highly cost-effective versus glargine U100 in people with T1DM and T2DM. The ICERs were estimated to be 4493.68 BGN/quality-adjusted life year (QALY) in T1DM, 399.11 BGN/QALY in T2DM on basal oral therapy (T2DMBOT) and 7365.22 BGN/QALY in T2DM on basal bolus therapy (T2DMB/B), which are below the cost-effectiveness threshold of 39,619 BGN in Bulgaria. Degludec was associated with higher insulin costs in all three patient groups; however, savings from a reduction in hypoglycaemic events with degludec versus glargine U100 partially offset these costs. Sensitivity analysis demonstrated that the results were robust and largely insensitive to variations in input parameters. At a willingness-to-pay threshold of 39,619 BGN/QALY, the probability of degludec being cost-effective versus glargine U100 was 60.0% in T1DM, 99.4% in T2DMBOT and 91.3% in T2DMB/B. Conclusion Degludec is a cost-effective alternative to biosimilar glargine U100 for patients with T1DM and T2DM in Bulgaria. Degludec could be of particular benefit to those patients suffering recurrent hypoglycaemia and those who require additional flexibility in the dosing of insulin.

Background: It is unknown whether lockdown due to severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) may affect both clinical outcome in kidney transplant recipients and health care economics in Denmark. Methods: We compared kidney transplant data at a tertiary university hospital before and during the lockdown period from March 13, 2020 until March 31, 2021, as well as kidney transplant data from Scandiatransplant for entire Denmark. Outcome variables included fall of plasma creatinine during the first postoperative day, and graft function three months posttransplant. We calculated the quality-adjusted life-years (QALYs) and costs which were caused by the lockdown recommendations. Findings: The portion of living donation kidney transplantation was largely reduced during the lockdown period compared to before the lockdown: AB0-incompatible living donation declined from 14% to 7% (P < 0.01), and AB0-compatible living donation declined from 34% to 20% (p < 0.01).In entire Denmark during the lockdown period 78 living donor kidney transplants out of 268 kidney transplants (29%) were performed, whereas there were 878 living donor kidney transplants out of 2218 kidney transplants (39%) before the lockdown (P = 0.01).The observed reduction of living donor kidney transplants and consecutive reduction of graft survival will cause a loss of 5.04 QALYs.The additional costs in kidney transplant recipients who received a kidney transplant during the lockdown period will be 277,298 EUR. Interpretation: SARS-CoV-2 lockdown period largely reduced living donation kidney transplants which will lead to reduced QALY as well as higher costs in kidney transplant recipients.

Objectives: Quality-adjusted life years (QALYs) have been challenged as a measure of benefit for people with disabilities, particularly for those in low-utility health states or with irreversible disability. This study examined the impact of a QALY-based assessment on the price for a hypothetical treatment for Duchenne muscular dystrophy (DMD), a progressive, genetic neuromuscular disease., Methods: A previously published, 5-state model, which analyzed treatments for early ambulatory (EA) DMD patients, was replicated, validated, and adapted to include early nonambulatory (ENA) DMD patients. The model was used to assess a QALY-based threshold price (maximum cost-effective price) for a hypothetical treatment for 13-year-old ENA and 5-year-old EA patients (initial health states with lower and higher utility, respectively). All inputs were replicated including willingness-to-pay thresholds of $50 000 to $200 000/QALY., Results: In contrast to EA patients, ENA patients had a 98% modeled decline in QALY-based threshold price at a willingness-to-pay of $150 000/QALY or higher, despite equal treatment benefit (delayed progression/death). At $100 000/QALY or lower, net nontreatment costs exceeded health benefits, implying any treatment for ENA patients would not be considered cost-effective, even at $0 price, including an indefinite pause in disease progression., Conclusions: For certain severe, disabling conditions, traditional approaches are likely to conclude that treatments are not cost-effective at any price once a patient progresses to a disabled health state with low utility value. These findings elucidate theoretical/ethical concerns regarding potential discriminatory properties of traditional QALY assessments for people with disabilities, particularly those who have lost ambulation or have other physical limitations., Competing Interests: Author Disclosures Author disclosure forms can be accessed below in the Supplemental Material section., (Copyright © 2024. Published by Elsevier Inc.)

Purpose: The aim of this study is to investigate the cost-effectiveness of revision total knee arthroplasty compared to primary total knee arthroplasty in terms of cost-per-quality-adjusted life year (QALY)., Methods: Data were retrieved for all primary and revision total knee replacement (TKA) procedures performed at a tertiary Swiss hospital between 2006 and 2019. A Markov model was created to evaluate revision risk and we calculated lifetime QALY gain and lifetime procedure costs through individual EuroQol 5 dimension (EQ-5D) scores, hospital costs, national life expectancy tables and standard discounting processes. Cost-per-QALY gain was calculated for primary and revision procedures., Results: EQ-5D data were available for 1343 primary and 103 revision procedures. Significant QALY gains were seen following surgery in all cases. Similar, but significantly more QALYs were gained following primary TKA (PTKA) (5.67 ± 3.98) than following revision TKA (RTKA) (4.67 ± 4.20). Cost-per-QALY was €4686 for PTKA and €10,364 for RTKA. The highest average cost-per-QALY was seen in two-stage RTKA (€12,292), followed by one-stage RTKA (€8982)., Conclusion: RTKA results in a similar QALY gain as PTKA. The costs of achieving health gain are two to three times higher in RTKA, but both procedures are highly cost-effective., Level of Evidence: Economic level II., (© 2024 European Society of Sports Traumatology, Knee Surgery and Arthroscopy.)

Background: Gastrointestinal (GI) cancer burden in Asia is increasing, and Vietnam is no exception. Assessing the affordability of achieving a quality-adjusted life year (QALY) in gastrointestinal cancer patients Vietnam, as well as identifying predictors of willingness to pay (WTP) per QALY, is crucial to decision-making around medical intervention prioritization and performing medical technology assessments for these cancers., Objectives: Our study aimed to estimate WTP/QALY gained and associated factors among patients diagnosed with GI cancer at a tertiary hospital in Hue, Vietnam., Methods: A cross-sectional descriptive study, using contingent valuation methodology was conducted among 231 patients at tertiary hospital in 2022. A double limited dichotomous choice and the EQ-5D-5L were utilised to estimate WTP and QALY, respectively. Quantile regression was applied to determine predictors of WTP/QALY., Results: The mean and median maximum WTP/QALY gained among GI patients was $15,165.6 (42,239.6) and $4,365.6 (IQR: 1,586.5-14,552.0), respectively, which was equal to 3.68 times the 2022 gross domestic product (GDP) per capita in Vietnam.  Additionally, cancer severity was found to have a significant impact  on WTP per QALY gained, with a higher amount identified among patients with earlier stages of GI cancer. Furthermore, living in an urban dwelling and patients' treatment modalities were significantly associated with WTP/QALY., Conclusion: Evidence from our study can be used to inform how decision-makers in Vietnam to determine the cost-effectiveness of GI cancer interventions.

Since late 2020, the Canadian Agency of Drugs and Technologies in Health (CADTH) has been using a threshold of $50,000 (CAD) per quality-adjusted life-year (QALY) for both oncology and non-oncology drugs. When used for oncology products, this threshold is hypothesized to have a higher impact on the time to access these drugs in Canada. We studied the impact of price reductions on time to engagement and negotiation with the pan-Canadian Pharmaceutical Alliance for oncology drugs reviewed by CADTH between January 2020 and December 2022. Overall, 103 assessments reported data on price reductions recommended by CADTH to meet the cost-effectiveness threshold for reimbursement. Of these assessments, 57% (59/103) recommendations included a price reduction of greater than 70% off the list price. Eight percent (8/103) were not cost-effective even at a 100% price reduction. Of the 47 assessments that had a clear benefit, in 21 (45%) CADTH recommended a price reduction of at least 70%. The median time to price negotiation (not including time to engagement) for assessments that received at least 70% vs >70% price reduction was 2.6 vs 4.8 months. This study showed that there is a divergence between drug sponsor's incremental cost-effectiveness ratio (ICER) and CADTH revised ICER leading to a price reduction to meet the $50,000/QALY threshold. For the submissions with clear clinical benefit the median length of engagement (2.5 vs 3.3 months) and median length of negotiation (3.1 vs 3.6 months) were slightly shorter compared with the submissions where uncertainties were noted in the clinical benefit according to CADTH. This study shows that using a $50,000 per QALY threshold for oncology products potentially impacts timely access to life saving medications.

Background: Alzheimer's disease (AD) and other dementias currently represent the fifth most common cause of death in the world, according to the World Health Organization, with a projected future increase as the proportion of the elderly in the population is growing. Air pollution has emerged as a plausible risk factor for AD, but studies estimating dementia cases attributable to exposure to fine particulate matter (PM2.5) air pollution and resulting monetary estimates are lacking. Methods: We used data on average population-weighted exposure to ambient PM2.5 for the entire population of Sweden above 30 years of age. To estimate the annual number of dementia cases attributable to air pollution in the Swedish population above 60 years of age, we used the latest concentration response functions (CRF) between PM2.5 exposure and dementia incidence, based on ten longitudinal cohort studies, for the population above 60 years of age. To estimate the monetary burden of attributable cases, we calculated total costs related to dementia, including direct and indirect lifetime costs and intangible costs by including quality-adjusted life years (QALYs) lost. Two different monetary valuations of QALYs in Sweden were used to estimate the monetary value of reduced quality-of-life from two different payer perspectives. Results: The annual number of dementia cases attributable to PM2.5 exposure was estimated to be 820, which represents 5% of the annual dementia cases in Sweden. Direct and indirect lifetime average cost per dementia case was estimated to correspond € 213,000. A reduction of PM2.5 by 1 μg/m3 was estimated to yield 101 fewer cases of dementia incidences annually, resulting in an estimated monetary benefit ranging up to 0.01% of the Swedish GDP in 2019. Conclusion: This study estimated that 5% of annual dementia cases could be attributed to PM2.5 exposure, and that the resulting monetary burden is substantial. These findings suggest the need to consider airborne toxic pollutants associated with dementia incidence in public health policy decisions. [ABSTRACT FROM AUTHOR]

Objective: Efficacy of pharmacological treatments for acromegaly has been assessed in many clinical or real-world studies but no study was interested in economics evaluation of these treatments in France. Therefore, the objective of this study was to estimate the cost-utility of second-line pharmacological treatments in acromegaly patients. Methods: A Markov model was developed to follow a cohort of 1,000 patients for a lifetime horizon. First-generation somatostatin analogues (FGSA), pegvisomant, pasireotide and pegvisomant combined with FGSA (off label) were compared. Efficacy was defined as the normalization of insulin-like growth factor-1 (IGF-1) concentration and was obtained from pivotal trials and adjusted by a network meta-analysis. Costs data were obtained from French databases and literature. Utilities from the literature were used to estimate quality-adjusted life year (QALY). Results: The incremental cost-utility ratios (ICUR) of treatments compared to FGSA were estimated to be 562,463 € per QALY gained for pasireotide, 171,332 € per QALY gained for pegvisomant, and 186,242 € per QALY gained for pegvisomant + FGSA. Pasireotide seems to be the least cost-efficient treatment. Sensitivity analyses showed the robustness of the results. Conclusion: FGSA, pegvisomant and pegvisomant + FGSA were on the cost-effective frontier, therefore, depending on the willingness-to-pay for an additional QALY, they are the most cost-effective treatments. This medico-economic analysis highlighted the consistency of the efficiency results with the efficacy results assessed in the pivotal trials. However, most recent treatment guidelines recommend an individualized treatment strategy based on the patient and disease profile. [ABSTRACT FROM AUTHOR]

Objectives: Health state utilities (HSUs) are an input metric for estimating quality-adjusted life-years (QALY) in cost–utility analyses. Currently, there is a paucity of data on association of knee symptoms with HSUs for middle-aged populations. We aimed to describe the association of knee symptoms and change in knee symptoms with SF-6D HSUs and described the distribution of HSUs against knee symptoms' severity. Methods: Participants (36–49-years) were selected from the third follow-up (completed 2019) of Australian Childhood Determinants of Adult Health study. SF-6D HSUs were generated from the participant-reported SF-12. Association between participant-reported WOMAC knee symptoms' severity, change in knee symptoms over 6–9 years, and HSUs were evaluated using linear regression models. Results: For the cross-sectional analysis, 1,567 participants were included; mean age 43.5 years, female 54%, BMI ± SD 27.18 ± 5.31 kg/m2. Mean ± SD HSUs for normal, moderate, and severe WOMAC scores were 0.820 ± 0.120, 0.800 ± 0.120, and 0.740 ± 0.130, respectively. A significant association was observed between worsening knee symptoms and HSUs in univariable and multivariable analyses after adjustment (age and sex). HSU decrement for normal-to-severe total-WOMAC and WOMAC-pain was − 0.080 (95% CI − 0.100 to − 0.060, p < 0.01) and − 0.067 (− 0.085 to − 0.048, p < 0.01), exceeding the mean minimal clinically important difference (0.04). Increase in knee pain over 6–9 years was associated with a significant reduction in HSU. Conclusion: In a middle-aged population-based sample, there was an independent negative association between worse knee symptoms and SF-6D HSUs. Our findings may be used by decision-makers to define more realistic and conservative baseline and ongoing HSU values when assessing QALY changes associated with osteoarthritis interventions. [ABSTRACT FROM AUTHOR]

Background: The concept of cost-effectiveness is necessary for optimal utilization of limited health care resources. However, few studies have assessed the cost-effectiveness of palliative care using quality-adjusted life years (QALYs), considered common outcomes in health economics. Objective: We aimed to perform a cost-effectiveness analysis of palliative care for terminal cancer patients by using QALYs. Design: A retrospective cohort study was performed. Setting/Patients: We included 401 patients with stage IV cancer, who were hospitalized and died at a Japanese general hospital during the period April 2014 to March 2019. Methods: Using the hospital database, we compared the total admission costs and QALYs based on pain levels of patients admitted to the palliative care (PC) department with those of patients admitted to other usual care (UC) departments. Patients in each group were matched through propensity scores to reduce bias. Bootstrapping estimated the 95% confidence intervals (95% CIs) and the probability that PC was more cost-effective than UC. Results: After matching, 128 patients in each group were selected. Converting 1 U.S. dollar (USD) to 100 Japanese yen, PC reduced mean total admission costs by 1732 USD (95% CI: 1584–1879) and improved mean health benefits by 0.0028 QALYs (95% CI: 0.0025–0.0032) compared with UC. Based on the Japanese cost-effectiveness threshold, there was an 82% probability that PC was more cost-effective than UC. Conclusions: Our results indicated that admission of terminal cancer patients to the PC department was associated with improvement in cost-effectiveness. This finding could support the introduction of palliative care for terminal cancer patients. Our study was approved at St. Luke's International University (receipt number 18-R061 and at the Graduate School of Pharmaceutical Sciences, The Univesity of Tokyo (receipt number 31–29). [ABSTRACT FROM AUTHOR]

Economic evaluation combines costs and benefits to support decision-making when assessing new interventions using preference-based measures to measure and value benefits in health or health-related quality of life. These health-focused instruments have limited ability to capture wider impacts on informal carers or outcomes in other sectors such as social care. Sector-specific instruments can be used but this is problematic when the impact of an intervention straddles different sectors. An alternative approach is to develop a generic preference-based measure that is sufficiently broad to capture important cross-sector outcomes. We consider the options for the selection of domains for a cross-sector generic measure including how to identify domains, who should provide information on the domains and how this should be framed. Beyond domain identification, considerations of criteria and stakeholder needs are also identified. This paper sets out the case for an approach that relies on the voice of patients, social care users and informal carers as the main source of domains and describes how the approach was operationalised in the 'Extending the QALY' project which developed the new measure, the EQ-HWB (EQ health and wellbeing instrument). We conclude by discussing the strengths and limitations of this approach. The new measure should be sufficiently generic to be used to consistently evaluate health and social care interventions, yet also sensitive enough to pick up important changes in quality of life in patients, social care users and carers. [ABSTRACT FROM AUTHOR]

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ObjectiveEfficacy of pharmacological treatments for acromegaly has been assessed in many clinical or real-world studies but no study was interested in economics evaluation of these treatments in France. Therefore, the objective of this study was to estimate the cost-utility of second-line pharmacological treatments in acromegaly patients.MethodsA Markov model was developed to follow a cohort of 1,000 patients for a lifetime horizon. First-generation somatostatin analogues (FGSA), pegvisomant, pasireotide and pegvisomant combined with FGSA (off label) were compared. Efficacy was defined as the normalization of insulin-like growth factor-1 (IGF-1) concentration and was obtained from pivotal trials and adjusted by a network meta-analysis. Costs data were obtained from French databases and literature. Utilities from the literature were used to estimate quality-adjusted life year (QALY).ResultsThe incremental cost-utility ratios (ICUR) of treatments compared to FGSA were estimated to be 562,463 € per QALY gained for pasireotide, 171,332 € per QALY gained for pegvisomant, and 186,242 € per QALY gained for pegvisomant + FGSA. Pasireotide seems to be the least cost-efficient treatment. Sensitivity analyses showed the robustness of the results.ConclusionFGSA, pegvisomant and pegvisomant + FGSA were on the cost-effective frontier, therefore, depending on the willingness-to-pay for an additional QALY, they are the most cost-effective treatments. This medico-economic analysis highlighted the consistency of the efficiency results with the efficacy results assessed in the pivotal trials. However, most recent treatment guidelines recommend an individualized treatment strategy based on the patient and disease profile.

Background: In health economic evaluation, utility associated with a health state is outcome-oriented and usually measured using the QALY methodology. Even though there is consistent evidence of utility not only being derived from outcomes but also from procedures, process utility has not been fully integrated in QALY calculations. The aim of this paper is twofold: first, to provide evidence of process utility associated with an alternative treatment to angioplasty, and second, to estimate a monetary value of such process utility using the willingness to pay (WTP) approach. Methods: A total of 1514 people were polled on their WTP to avoid angioplasty to have a drug-eluting stent (DES) implanted. WTP is estimated with a contingent valuation (CV) survey. Individuals are also asked if they would be WTP for a non-invasive procedure with similar results being achieved. WTP responses were analyzed using a double bounded (DB) logit model. Results: Most of the participants showed positive preferences for avoiding angioplasty, with an estimated mean WTP of €5692.87. Using QALY gains for avoiding angioplasty, varying from 0.0035 to 0.08 QALYs, our WTP estimate imply monetary values per QALY that range from €71,160.87 to €1,626,534.28. Discussion: A WTP of €5692.87 to avoid angioplasty imply a monetary value per QALY that greatly exceed the cost per QALY thresholds established in different countries to consider health programs as beneficial to society. Our results reflect how different methodologies for HTA may lead to different conclusions. From the ICER perspective, the cost that would make the treatment with pills option cost-effective, using a threshold of €40,000/QALY, would be €224. However, a cost-benefit approach could support health programs even with a higher cost. Conclusion: WTP methodology captures outcome and process factors related to angioplasty as our WTP estimations are non-significantly different for the costs of angioplasty. WTP approach must be considered as a genuine alternative to QALY approaches to value process utility. [ABSTRACT FROM AUTHOR]

Cirrhosis is a burden on the individual and on public health. The World Health Organization's metric of public health burden is the disability-adjusted life-year (DALY), the sum of years of life lost due to premature death and years of life lived with disability. The more DALYs attributable to a disease, the greater its burden on public health. Cirrhosis was responsible for 26.8% fewer DALYs in 2019 than in 1990, which is positive, but the reduction in DALYs across the spectrum of diseases in and outside the liver was 34.4%. Hepatitis C (26% of DALYs), alcohol (24%), and hepatitis B (23%) contribute almost equally to the global burden of cirrhosis. The contribution from non-alcoholic fatty liver disease (8%) is small but increasing. There is substantial global variation in the burden and causes of cirrhosis. We find that the poorest countries carry the greatest burden of cirrhosis, and that this burden is primarily caused by cirrhosis from hepatitis B infection. Interventions targeting hepatitis B infection are known, but not fully implemented. In more affluent countries, alcohol and hepatitis C are the dominant causes of cirrhosis, but non-alcoholic fatty liver will likely become a dominant cause of cirrhosis in parallel with the increasing prevalence of obesity. We also argue that the World Health Organization underestimates the public health burden associated with cirrhosis because it assigns zero disability to compensated cirrhosis and considers decompensated cirrhosis as only mildly disabling. [ABSTRACT FROM AUTHOR]