Cost-Effectiveness of Laparoscopic Sleeve Gastrectomy for Chinese Patients.

BACKGROUND: Laparoscopic sleeve gastrectomy (LSG) is the most popular bariatric surgery procedure in China. However, its cost-effectiveness in Chinese patients is currently unknown. OBJECTIVES: This study aims to assess the cost-effectiveness of LSG vs no surgery in Chinese patients with severe and complex obesity, taking into account both healthcare expenses and the potential improvement in health-related quality of life (HRQoL). METHODS: A retrospective cohort study was conducted, encompassing 135 Chinese patients who underwent LSG between January 3, 2022 and December 29, 2022, at a major bariatric center. The study evaluated the cost-effectiveness from a healthcare service perspective, employing the incremental cost-effectiveness ratio (ICER) for quality-adjusted life years (QALYs) gained. The analyses compared LSG with the alternative of not undergoing surgery over a 1-year period, using actual data, and extended to a lifetime horizon by projecting costs and utilities at an annual discount rate of 3.0%. Subgroup analyses were undertaken to explore cost-effectiveness variations across different sex, age and BMI categories, and diabetes status, employing a one-way analysis of variance (ANOVA). To ensure the reliability of the findings, one-way and probabilistic sensitivity analyses were executed. RESULTS: The results indicated that 1-year post-LSG, patients achieved an average total weight loss (TWL) of (32.7 ± 7.3)% and an excess weight loss (EWL) of (97.8 ± 23.1)%. The ICER for LSG compared to no surgery over a lifetime was $4,327/QALY, significantly below the willingness-to-pay (WTP) threshold for Chinese patients with severe and complex obesity. From a lifetime perspective, LSG proved to be cost-effective for all sex and age groups, across all BMI categories, and for both patients with and without diabetes. Notably, it was more cost-effective for younger patients, patients with higher BMI, and patients with diabetes. CONCLUSIONS: LSG is a highly cost-effective intervention for managing obesity in Chinese patients, delivering substantial benefits in terms of HRQoL improvement at a low cost. Its cost-effectiveness is particularly pronounced among younger individuals, those with higher BMI, and patients with diabetes.

Direct oral anticoagulants versus low-molecular-weight heparin in patients with cancer-associated venous thrombosis: a cost-effectiveness analysis.

Background: Direct oral anticoagulants (DOACs) have demonstrated clinical benefits and better patient adherence over low-molecular-weight heparin (LMWH) in treating patients with cancer-associated venous thrombosis (CAT). We aimed to compare the cost-effectiveness of DOACs against LMWH in patients with CAT from the perspective of the Hong Kong healthcare system. Methods: A Markov state-transition model was performed to estimate the incremental cost-effectiveness ratio (ICER) per quality-adjusted life years (QALYs) for DOACs and LMWH in a hypothetical cohort of 10,000 patients with CAT over a 5-year lifetime horizon. The model was primarily based on the health states of no event, recurrent venous thromboembolism, bleeding, and death. Transition probabilities, relative risks, and utilities were derived from the literature. Resource cost data were obtained from the Hong Kong Hospital Authority. Deterministic and probabilistic sensitivity analyses tested the robustness of the results. Results: Relative to LMWH, DOACs were associated with increased QALYs (1.52 versus 1.50) at a lower medical cost of USD 2,232 versus 8,224 in five years. The cost of LMWH was the main contributor to the outcome. Out of 10,000 simulated cases, DOACs were dominant in 15.8% and cost-effective in 42.1%, at the willingness-to-pay threshold of USD 148,392 per additional QALY. Conclusions: DOACs were associated with greater QALY improvements and lower overall costs compared to LMWH. Accounting for uncertainty, DOACs were between cost-effective and dominant in 57.9% of cases. DOACs are a cost-effective alternative to LMWH in the management of CAT in Hong Kong.

Cost-utility analysis of second-line axicabtagene ciloleucel versus standard of care in Japan based on the ZUMA-7 trial.

Aim: To perform a cost-effectiveness analysis comparing axicabtagene ciloleucel (axi-cel) with standard of care (SoC; salvage chemoimmunotherapy, followed by high-dose therapy with autologous stem cell rescue for responders) for second-line (2L) treatment of adults with relapsed or refractory large B-cell lymphoma (r/r LBCL) in the pivotal ZUMA-7 trial data from a Japanese payer perspective. Materials & methods: A three-state partitioned survival model was utilized using population and clinical inputs from the ZUMA-7 trial data over a lifetime horizon. Results: Axi-cel was associated with greater incremental quality-adjusted life-years (2.06) and higher incremental total costs ($48,685.59/¥6.9 million) leading to an incremental cost-effectiveness ratio of $23,590.34/¥3.3 million per quality-adjusted life-years compared with SoC. Conclusion: Axi-cel is a cost-effective treatment alternative to SoC for 2L treatment of adults with r/r LBCL.

[Box: see text].

Sleeping Sound Autism Spectrum Disorder (ASD): Cost-Effectiveness of a Brief Behavioural Sleep Intervention in Primary School-Aged Autistic Children.

Disordered sleep is common in autistic children. This study aimed to evaluate the cost-effectiveness of a brief behavioural sleep intervention, the 'Sleeping Sound intervention', in primary school-aged autistic children in Australia. A cost-effectiveness analysis was undertaken alongside a randomised controlled trial over a 6-month follow-up period from both a societal and healthcare sector perspective. Resources used by participants were collected from a resource-use questionnaire and administrative data; intervention costs were determined from study records. Mean costs and quality-adjusted life-years (QALYs) were compared between the intervention and treatment as usual (TAU) groups. Uncertainty analysis using bootstrapping and sensitivity analyses were conducted. The sample included 245 children, with 123 participants randomised to the intervention group and 122 to TAU. The mean total costs were higher for the Sleeping Sound intervention with a mean difference of A$745 (95% CI 248; 1242; p = 0.003) from a healthcare sector perspective and A$1310 (95% CI 584; 2035, p < 0.001) from a societal perspective. However, the intervention also resulted in greater QALYs compared with TAU, with a mean difference of 0.038 (95% CI 0.004; 0.072; p = 0.028). The incremental cost-effectiveness ratio was A$24,419/QALY (95% CI 23,135; 25,703) from a healthcare sector perspective and A$41,922/QALY (95% CI 39,915; 43,928) from a societal perspective; with a probability of being cost-effective of 93.8% and 74.7%, respectively. Findings remained robust in the sensitivity analyses. The Sleeping Sound intervention offers a cost-effective approach in improving sleep in primary school-aged autistic children.Trial registration The trial was registered with the International Trial Registry (ISRCTN14077107).

Decision-Making for Preventive Interventions in Asymptomatic Patients.

The decision to treat an incidental finding in an asymptomatic patient results from careful risk-benefit consideration and is often challenging. One of the main aspects is after how many years the group who underwent the intervention and faced the immediate treatment complications will gain a treatment benefit over the conservatively managed group, which maintains a lower but ongoing risk. We identify a common error in decision-making. We illustrate how a risk-based approach using the classical break-even point at the Kaplan-Meier curves can be misleading and advocate for using an outcome-based approach, counting the cumulative number of lost quality-adjusted life years instead. In clinical practice, we often add together the yearly risk of the natural course up to the time point where the number equals the risk of the intervention and assume that the patient will benefit from an intervention beyond this point in time. It corresponds to the crossing of the Kaplan-Meier curves. However, because treatment-related poor outcome occurs at the time of the intervention, while the poor outcome in the conservative group occurs over a given time period, the true benefit of retaining more quality-adjusted life years in the interventional group emerges at a much later time. To avoid overtreatment of patients with asymptomatic diseases, decision-making should be outcome-based with counting the cumulative loss of quality-adjusted life years, rather than risk-based, comparing the interventional risk with the ongoing yearly risk of the natural course.

Cefiderocol for treating severe aerobic Gram-negative bacterial infections: technology evaluation to inform a novel subscription-style payment model.

Background: To limit the use of antimicrobials without disincentivising the development of novel antimicrobials, there is interest in establishing innovative models that fund antimicrobials based on an evaluation of their value as opposed to the volumes used. The aim of this project was to evaluate the population-level health benefit of cefiderocol in the NHS in England, for the treatment of severe aerobic Gram-negative bacterial infections when used within its licensed indications. The results were used to inform the National Institute for Health and Care Excellence guidance in support of commercial discussions regarding contract value between the manufacturer and NHS England. Methods: The health benefit of cefiderocol was first derived for a series of high-value clinical scenarios. These represented uses that were expected to have a significant impact on patients' mortality risks and health-related quality of life. The clinical effectiveness of cefiderocol relative to its comparators was estimated by synthesising evidence on susceptibility of the pathogens of interest to the antimicrobials in a network meta-analysis. Patient-level costs and health outcomes of cefiderocol under various usage scenarios compared with alternative management strategies were quantified using decision modelling. Results were reported as incremental net health effects expressed in quality-adjusted life-years, which were scaled to 20-year population values using infection number forecasts based on data from Public Health England. The outcomes estimated for the high-value clinical scenarios were extrapolated to other expected uses for cefiderocol. Results: Among Enterobacterales isolates with the metallo-beta-lactamase resistance mechanism, the base-case network meta-analysis found that cefiderocol was associated with a lower susceptibility relative to colistin (odds ratio 0.32, 95% credible intervals 0.04 to 2.47), but the result was not statistically significant. The other treatments were also associated with lower susceptibility than colistin, but the results were not statistically significant. In the metallo-beta-lactamase Pseudomonas aeruginosa base-case network meta-analysis, cefiderocol was associated with a lower susceptibility relative to colistin (odds ratio 0.44, 95% credible intervals 0.03 to 3.94), but the result was not statistically significant. The other treatments were associated with no susceptibility. In the base case, patient-level benefit of cefiderocol was between 0.02 and 0.15 quality-adjusted life-years, depending on the site of infection, the pathogen and the usage scenario. There was a high degree of uncertainty surrounding the benefits of cefiderocol across all subgroups. There was substantial uncertainty in the number of infections that are suitable for treatment with cefiderocol, so population-level results are presented for a range of scenarios for the current infection numbers, the expected increases in infections over time and rates of emergence of resistance. The population-level benefits varied substantially across the base-case scenarios, from 896 to 3559 quality-adjusted life-years over 20 years. Conclusion: This work has provided quantitative estimates of the value of cefiderocol within its areas of expected usage within the NHS. Limitations: Given existing evidence, the estimates of the value of cefiderocol are highly uncertain. Future work: Future evaluations of antimicrobials would benefit from improvements to NHS data linkages; research to support appropriate synthesis of susceptibility studies; and application of routine data and decision modelling to assess enablement value. Study registration: No registration of this study was undertaken. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment Policy Research Programme (NIHR award ref: NIHR135591), conducted through the Policy Research Unit in Economic Methods of Evaluation in Health and Social Care Interventions, PR-PRU-1217-20401, and is published in full in Health Technology Assessment; Vol. 28, No. 28. See the NIHR Funding and Awards website for further award information.

This project tested new methods for estimating the value to the NHS of an antimicrobial, cefiderocol, so its manufacturer could be paid fairly even if very little drug is used in order to reduce the risk of bacteria becoming resistant to the product. Clinicians said that the greatest benefit of cefiderocol is when used for complicated urinary tract infections and pneumonia acquired within hospitals caused by two types of bacteria (called Enterobacterales and Pseudomonas aeruginosa), with a resistance mechanism called metallo-beta-lactamase. Because there were no relevant clinical trial data, we estimated how effective cefiderocol and alternative treatments were by doing a systematic literature review of studies that grew bacteria from infections in the laboratory and tested the drugs on them. We linked this to data estimating the long-term health and survival of patients. Some evidence was obtained by asking clinicians detailed questions about what they thought the effects would be based on their experience and the available evidence. We included the side effects of the alternative treatments, some of which can cause kidney damage. We estimated how many infections there would be in the UK, whether they would increase over time and how resistance to treatments may change over time. Clinicians told us that they would also use cefiderocol to treat intra-abdominal and bloodstream infections, and some infections caused by another bacteria called Stenotrophomonas. We estimated how many of these infections there would be, and assumed the same health benefits as for other types of infections. The total value to the NHS was calculated using these estimates. We also considered whether we had missed any additional elements of value. We estimated that the value to the NHS was £18­71 million over 20 years. This reflects the maximum the NHS could pay for use of cefiderocol if the health lost as a result of making these payments rather than funding other NHS services is not to exceed the health benefits of using this antimicrobial. However, these estimates are uncertain due to limitations with the evidence used to produce them and assumptions that had to be made.

Cost-utility analysis of Social Stories™ for children with autism spectrum disorder in mainstream primary schools: results from a randomised controlled trial.

BACKGROUND: One in 57 children are diagnosed with autism in the UK, and the estimated cost for supporting these children in education is substantial. Social Stories™ is a promising and widely used intervention for supporting children with autism in schools and families. It is believed that Social Stories™ can provide meaningful social information to children that can improve social understanding and may reduce anxiety. However, no economic evaluation of Social Stories has been conducted. AIMS: To assess the cost-effectiveness of Social Stories through Autism Spectrum Social Stories in Schools Trial 2, a multi-site, pragmatic, cluster-randomised controlled trial. METHOD: Children with autism who were aged 4-11 years were recruited and randomised (N = 249). Costs measured from the societal perspective and quality-adjusted life-years (QALYs) measured by the EQ-5D-Y-3L proxy were collected at baseline and at 6-month follow-up for primary analysis. The incremental cost-effectiveness ratio was calculated, and the uncertainty around incremental cost-effectiveness ratios was captured by non-parametric bootstrapping. Sensitivity analyses were performed to evaluate the robustness of the primary findings. RESULTS: Social Stories is likely to result in a small cost savings (-£191 per child, 95% CI -767.7 to 337.7) and maintain similar QALY improvements compared with usual care. The probability of Social Stories being a preferred option is 75% if society is willing to pay £20 000 per QALY gained. The sensitivity analysis results aligned with the main study outcomes. CONCLUSIONS: Compared with usual care, Social Stories did not lead to an increase in costs and maintained similar QALY improvements for primary-aged children with autism.

Clinical and cost-effectiveness of pessary self-management versus clinic-based care for pelvic organ prolapse in women: the TOPSY RCT with process evaluation.

Background: Pelvic organ prolapse is common, causes unpleasant symptoms and negatively affects women's quality of life. In the UK, most women with pelvic organ prolapse attend clinics for pessary care. Objectives: To determine the clinical effectiveness and cost-effectiveness of vaginal pessary self-management on prolapse-specific quality of life for women with prolapse compared with clinic-based care; and to assess intervention acceptability and contextual influences on effectiveness, adherence and fidelity. Design: A multicentre, parallel-group, superiority randomised controlled trial with a mixed-methods process evaluation. Participants: Women attending UK NHS outpatient pessary services, aged ≥ 18 years, using a pessary of any type/material (except shelf, Gellhorn or Cube) for at least 2 weeks. Exclusions: women with limited manual dexterity, with cognitive deficit (prohibiting consent or self-management), pregnant or non-English-speaking. Intervention: The self-management intervention involved a 30-minute teaching appointment, an information leaflet, a 2-week follow-up telephone call and a local clinic telephone helpline number. Clinic-based care involved routine appointments determined by centres' usual practice. Allocation: Remote web-based application; minimisation was by age, pessary user type and centre. Blinding: Participants, those delivering the intervention and researchers were not blinded to group allocation. Outcomes: The patient-reported primary outcome (measured using the Pelvic Floor Impact Questionnaire-7) was prolapse-specific quality of life, and the cost-effectiveness outcome was incremental cost per quality-adjusted life-year (a specifically developed health Resource Use Questionnaire was used) at 18 months post randomisation. Secondary outcome measures included self-efficacy and complications. Process evaluation data were collected by interview, audio-recording and checklist. Analysis was by intention to treat. Results: Three hundred and forty women were randomised (self-management, n = 169; clinic-based care, n = 171). At 18 months post randomisation, 291 questionnaires with valid primary outcome data were available (self-management, n = 139; clinic-based care, n = 152). Baseline economic analysis was based on 264 participants (self-management, n = 125; clinic-based care, n = 139) with valid quality of life and resource use data. Self-management was an acceptable intervention. There was no group difference in prolapse-specific quality of life at 18 months (adjusted mean difference -0.03, 95% confidence interval -9.32 to 9.25). There was fidelity to intervention delivery. Self-management was cost-effective at a willingness-to-pay threshold of £20,000 per quality-adjusted life-year gained, with an estimated incremental net benefit of £564.32 and an 80.81% probability of cost-effectiveness. At 18 months, more pessary complications were reported in the clinic-based care group (adjusted mean difference 3.83, 95% confidence interval 0.81 to 6.86). There was no group difference in general self-efficacy, but self-managing women were more confident in pessary self-management activities. In both groups, contextual factors impacted on adherence and effectiveness. There were no reported serious unexpected serious adverse reactions. There were 32 serious adverse events (self-management, n = 17; clinic-based care, n = 14), all unrelated to the intervention. Skew in the baseline data for the Pelvic Floor Impact Questionnaire-7, the influence of the global COVID-19 pandemic, the potential effects of crossover and the lack of ethnic diversity in the recruited sample were possible limitations. Conclusions: Self-management was acceptable and cost-effective, led to fewer complications and did not improve or worsen quality of life for women with prolapse compared with clinic-based care. Future research is needed to develop a quality-of-life measure that is sensitive to the changes women desire from treatment. Study registration: This study is registered as ISRCTN62510577. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 16/82/01) and is published in full in Health Technology Assessment; Vol. 28, No. 23. See the NIHR Funding and Awards website for further award information.

Pelvic organ prolapse is a common and distressing condition experienced by large numbers of women. Prolapse is when the organs that are usually in the pelvis drop down into the vagina. Women experience a feeling of something coming down into the vagina, along with bowel, bladder and sexual problems. One possible treatment is a vaginal pessary. The pessary is a device that is inserted into the vagina and holds the pelvic organs back in their usual place. Women who use a vaginal pessary usually come back to clinic every 6 months to have their pessary removed and replaced; this is called clinic-based care. However, it is possible for a woman to look after the pessary herself; this is called self-management. This study compared self-management with clinic-based care. Three hundred and forty women with prolapse took part; 171 received clinic-based care and 169 undertook self-management. Each woman had an equal chance of being in either group. Women in the self-management group received a 30-minute teaching appointment, an information leaflet, a 2-week follow-up telephone call and a telephone number for their local centre. Women in the clinic-based care group returned to clinic as advised by the treating healthcare professional. Self-management was found to be acceptable. Women self-managed their pessary in ways that suited their lifestyle. After 18 months, there was no difference between the groups in women's quality of life. Women in the self-management group experienced fewer pessary complications than women who received clinic-based care. Self-management costs less to deliver than clinic-based care. In summary, self-management did not improve women's quality of life more than clinic-based care, but it did lead to women experiencing fewer complications and cost less to deliver in the NHS. The findings support self-management as a treatment pathway for women using a pessary for prolapse.

Fair Funding Decisions: Consistency of the Time Horizons Used in the Calculation of Quality-Adjusted Life Years for Therapies for Very Rare Diseases by the National Institute for Health and Care Excellence in England.

The National Institute for Health and Care Excellence (NICE) in England uses quality-adjusted life years (QALYs) to assess the cost-effectiveness of treatments. A QALY is a measure that combines the size of the clinical benefit of a treatment with the time the patient benefits from it, i.e., the time horizon. We wanted to know how consistently QALY gains are calculated at NICE. Therefore, we have analysed information on the time horizons used for the QALY calculations of the concluded evaluations conducted under the Highly Specialised Technologies programme for treatments of very rare diseases at NICE. For treatments with final guidance published by December 2023 (n = 29), a time horizon of median 97.5 years (range: 35 to 125 years) was used to calculate the QALY gains. For most QALY calculations, the accepted time horizon was longer than either the expected treatment duration or the estimated life expectancy. In contrast, for the only technology with a final negative funding decision, i.e., afamelanotide for treating the lifelong chronic disease erythropoietic protoporphyria, a time horizon that was shorter than the expected treatment duration was used. The fairness and consistency of the evaluation process of treatments for very rare diseases at NICE should be reviewed.

Cost-effectiveness of the children and young People's health partnership (CYPHP) model of integrated care versus enhanced usual care: analysis of a pragmatic cluster randomised controlled trial in South London.

Background: Integration of paediatric health services across primary and secondary care holds great promise for the management of chronic conditions, yet limited evidence exists on its cost-effectiveness. This paper reports the results of the economic evaluation of the Children and Young People's Health Partnership (CYPHP) aimed at integrating care for children with common chronic conditions (asthma, eczema, and constipation). Methods: Cost-effectiveness, cost-utility and cost-benefit analyses were conducted alongside a pragmatic cluster randomised controlled trial involving 97,970 children in 70 general practices in South London, including 1,731 participants with asthma, eczema and or constipation with self-reported health-related quality of life measures. Analyses considered the National Health Service (NHS)/Personal Social Service (PSS) and societal perspectives, and time horizons of 6 and 12-months. Costs included intervention delivery, health service use (primary and secondary care), referrals to social services, and time lost from work and school. Health outcomes were measured through the Paediatric Quality of Life Inventory, the Child Health Utility 9-Dimensions, and monetarised benefit combining Quality-Adjusted Life Years (QALYs) for children and parental mental well-being. Results present incremental cost-effectiveness ratios (ICERs), compared to a willingness to pay threshold (WTP) of £20,000-30,000/QALY, and net monetary benefit (NMB), with deterministic sensitivity analyses. Findings: At 6 months, from the NHS/PSS perspective, CYPHP is not cost-effective (ICER = £721,000/QALY), and this result holds at 12 months (ICER = £45,586/QALY). However, under the societal perspective CYPHP falls within WTP thresholds (ICER = £22,966/QALY), with a probability of being cost-effective between 0.4 and 0.6 at £20,000/QALY and £30,000/QALY, respectively. The cost-benefit analysis yields a positive NMB of CYPHP at 12 months £109 under the societal perspective, with similar probabilistic results. Interpretation: CYPHP was not cost-effective at 6 months or under the NHS/PSS perspective. Trends towards cost-effectiveness are observed once a longer time horizon and a more inclusive perspective on effects is considered. Further research beyond 12 months is needed as the model becomes firmly embedded into the paediatric healthcare delivery system. Funding: This research was funded by Guy's and St Thomas' Charity, Lambeth and Southwark Clinical Commissioning Groups. The funders had no role in the writing of the manuscript, decision to submit it for publication, or any other process involved in the research.

Development of algorithms for estimating the Child Health Utility 9D from Caregiver Priorities and Child Health Index of Life with Disability.

PURPOSE: The primary aim was to determine Child Health Utility 9D (CHU9D) utilities from the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) for non-ambulatory children with cerebral palsy (CP). METHODS: One hundred and eight surveys completed by Australian parents/caregivers of children with CP were analysed. Spearman's coefficients were used to investigate the correlations between the two instruments. Ordinary least square, robust MM-estimator, and generalised linear models (GLM) with four combinations of families and links were developed to estimate CHU9D utilities from either the CPCHILD total score or CPCHILD domains scores. Internal validation was performed using 5-fold cross-validation and random sampling validation. The best performing algorithms were identified based on mean absolute error (MAE), concordance correlation coefficient (CCC), and the difference between predicted and observed means of CHU9D. RESULTS: Moderate correlations (ρ 0.4-0.6) were observed between domains of the CHU9D and CPCHILD instruments. The best performing algorithm when considering the CPCHILD total score was a generalised linear regression (GLM) Gamma family and logit link (MAE = 0.156, CCC = 0.508). Additionally, the GLM Gamma family logit link using CPCHILD comfort and emotion, quality of life, and health domain scores also performed well (MAE = 0.152, CCC = 0.552). CONCLUSION: This study established algorithms for estimating CHU9D utilities from CPCHILD scores for non-ambulatory children with CP. The determined algorithms can be valuable for estimating quality-adjusted life years for cost-utility analysis when only the CPCHILD instrument is available. However, further studies with larger sample sizes and external validation are recommended to validate these findings.

Psychometric Performance of the EQ Health and Wellbeing Short in a United Kingdom Population Sample.

OBJECTIVES: This study assessed the psychometric performance and construct validity of the EQ Health and Wellbeing Short (EQ-HWB-S), using a validated measure, the EQ-5D-5L, as a comparator. METHODS: The experimental version of the EQ-HWB-S was compared with the EQ-5D-5L to assess the psychometric performance of the measures. Data were drawn from the valuation stages of the Extending the Quality-Adjusted Life-Year project (UK general population, n = 429) and the EQ-5D-5L UK valuation pilot study (UK general population, n = 248). Construct validity was assessed based on convergent validity, using Spearman correlations and Pearson correlations. Known-group validity was assessed by estimating effect sizes to assess the ability of the EQ-HWB-S and EQ-5D-5L to discriminate between known groups based on "healthy" status, presence of a long-term condition, health and life satisfaction, age, and employment status. The degree of agreement in utility values across instruments was also evaluated using Bland-Altman plots. RESULTS: Strong associations (rs ≥ 0.5, P < .001) were found between conceptually overlapping dimensions and the utility scores of the EQ-HWB-S and EQ-5D-5L. The instruments performed comparably in discriminating between known groups including healthy versus unhealthy groups (based on the visual analog scale ≥ 80), long-term condition (vs no long-term condition), and above versus below average health and life satisfaction and employed (vs unemployed and long-term sick). CONCLUSIONS: The EQ-HWB-S performs favorably with utility values successfully discriminating between groups in which differences are expected. Convergence between the EQ-HWB-S and EQ-5D-5L is evident, especially between conceptually overlapping dimensions.

Economic evaluation of Be a Mom, a web-based intervention to prevent postpartum depression in high-risk women alongside a randomized controlled trial.

BACKGROUND: Postpartum depression (PPD) poses significant challenges, affecting both mothers and children, with substantial societal and economic implications. Internet-based cognitive behavioral therapy interventions (iCBT) offer promise in addressing PPD, but their economic impact remains unexplored. This study aimed to evaluate the cost-utility of Be a Mom, a self-guided iCBT intervention, compared with a waiting-list control among postpartum women at high risk of PPD. METHODS: This economic evaluation was conducted alongside a 14-month randomized controlled trial adopting a societal perspective. Participants were randomized to Be a Mom (n = 542) or a waitlisted control group (n = 511). Self-report data on healthcare utilization, productivity losses, and quality-adjusted life years (QALYs) were collected at baseline, post-intervention, and 4 and 12 months post-intervention. Incremental cost-effectiveness ratios (ICERs) were calculated, and cost-effectiveness acceptability curves were generated using nonparametric bootstrapping. Sensitivity analyses were conducted to assess result robustness. RESULTS: Over 14 months, Be a Mom generated a QALY gain of 0.0184 (0.0022, 0.0346), and cost savings of EUR 34.06 (-176.16, 108.04) compared to the control group. At a willingness to pay of EUR 20,000, Be a Mom had a 97.6 % probability of cost-effectiveness. LIMITATIONS: Results have limitations due to self-selected sample, potential recall bias in self-reporting, missing data, limited follow-up, and the use of a waiting-list control group. CONCLUSIONS: This study addresses a critical gap by providing evidence on the cost-utility of an iCBT intervention tailored for PPD prevention. Further research is essential to identify scalable and cost-effective interventions for reducing the burden of PPD.

A comparison of quality-adjusted life years in older adults after heart transplantation versus long-term mechanical support: Findings from the SUSTAIN-IT study.

BACKGROUND: The quality-adjusted life year (QALY) measures disease burden and treatment, combining overall survival and health-related quality of life (HRQOL). We estimated QALYs in 3 groups of older patients (60-80 years) with heart failure (HF) who underwent heart transplantation (HT, with pre-transplant mechanical circulatory support [HT MCS] or HT without pre-transplant MCS [HT Non-MCS]) or long-term MCS (destination therapy). We also identified factors associated with gains in QALYs through 24 months follow-up. METHODS: Of 393 eligible patients enrolled (10/1/15-12/31/18) at 13 U.S. sites, 161 underwent HT (n = 68 HT MCS, n = 93 HT Non-MCS) and 144 underwent long-term MCS. Survival and HRQOL data were collected through 24 months. QALY health utilities were based on patient self-report of EQ-5D-3L dimensions. Mean-restricted QALYs were compared among groups using generalized linear models. RESULTS: For the entire cohort, mean age in years closest to surgery was 67 (standard deviation, SD: 4.7), 78% were male, and 83% were White. By 18 months post-surgery, sustained significant differences in adjusted average ± SD QALYs emerged across groups, with the HT Non-MCS group having the highest average QALYs (24-month window: HT Non-MCS = 22.58 ± 1.1, HT MCS = 19.53 ± 1.33, Long-term MCS = 19.49 ± 1.3, p = 0.003). At 24 months post-operatively, a lower gain in QALYs was associated with HT MCS, long-term MCS, a lower pre-operative LVEF, NYHA class III or IV before surgery, and an ischemic or other etiology of HF. CONCLUSIONS: Determination of QALYs may provide important information for policy makers and clinicians to consider regarding benefits of HT and long-term MCS as treatment options for older patients with HF.

Monetary valuation of one year in full capability and health based on demographics, health status, income and well-being.

BACKGROUND: Budget constraints in health-care systems have led to the popularity of Cost Effectiveness Thresholds (CET) to achieve efficient allocation of resources. The capability approach has been hailed for its potentially richer evaluative capabilities compared to the QALY in terms of thresholds. Extensive research, however, is still limited. RESEARCH DESIGN AND METHODS: This study estimated the monetary value of a year in full capability (YFC) and compared it to monetary value of a QALY for the Hungarian population. Data was collected from a large, cross sectional, representative online survey on the adult Hungarian population. Applying the wellbeing valuation method, health, capability, and income were then regressed against wellbeing to estimate 'shadow prices' for one QALY and YFC controlling for gender, age, employment, education, marital and social support. To examine 'core' regression coefficients, a robustness check was conducted. RESULTS: Health (VAS) and capability (ICECAP-A) had a positive and significant effect on Subjective Well-Being. The monetary values of one QALY and one YFC were 39 459 EUR and 58 148 EUR respectively. CONCLUSIONS: These tools provide a systematic approach to determining 'compensating income' for certain illnesses, disabilities and levels of pain. The capability approach shown to be broader than the QALY.

Can Generalized Cost-effectiveness Analysis Leverage Meaningful Use of Novel Value Elements in Pharmacoeconomics to Inform Medicare Drug Price Negotiation?

OBJECTIVES: Decision makers considering using cost-effectiveness analysis (CEA) to inform health-technology assessment must contend with documented and controversial shortfalls of CEA, including its assumption of disease severity independence and static pricing. ISPOR has recently introduced novel value elements besides direct healthcare cost and effectiveness for the patient, and these should be captured in CEA. Although novel value elements advance our understanding of "what" should be measured (value of hope, severity of disease, health equity, etc), there is limited direction on "how" to measure them in conventional CEA. Furthermore, with Medicare empowered to set drug prices under the Inflation Reduction Act, it is not clear what role CEA might have on where prices are set, given objections to the quality-adjusted life year in conventional approaches. METHODS: We critically reviewed the evidence for expanding conventional CEA methods to a more generalized approach of generalized CEA (GCEA). RESULTS: GCEA accounts for methods that address objections to the quality-adjusted life year and incorporate novel value elements. Although GCEA offers advantages, it also requires further research to develop "off-the-shelf" resources to help inform, for example, maximum fair price in the context of Medicare drug price negotiation. CONCLUSIONS: Should a shift toward GCEA reveal that the societal value of novel medicines exceeds their market-based costs, which will raise the key question of what market failure Medicare negotiation is meant to solve, if any, and therefore what the appropriate role of such negotiation might be to maximize the value society might garner from the development of novel medicines.

Cost-effectiveness analysis of single-dose or 2-dose of bivalent, quadrivalent, or nonavalent HPV vaccine in a low/middle-income country setting.

OBJECTIVE: To compare the health impact and economic benefits among individuals who did not receive the human papillomavirus (HPV) vaccine to those who received a single dose, or 2 doses. The comparison was stratified by 4 types of vaccine in conjunction with primary HPV screening in a low/middle-income country setting. METHODS: A Markov model was employed to simulate HPV infection and cervical cancer in a cohort of 100,000 12-year-old girls free of HPV. The study scrutinized 9 strategies: 1 dose and 2 doses of 2vHPV (Cervarix®), 2vHPV (Cecolin®), 4vHPV (Gardasil®), 9vHPV vaccine (Gardasil9®), and no vaccination. The primary outcome measure was the quality-adjusted life year (QALY) of each strategy. Incremental cost-effectiveness ratios were estimated over a lifetime horizon, accompanied by sensitivity analyses conducted. RESULTS: All vaccination programs yielded 41,298-71,057 QALYs gained accompanied by cost savings of 14,914,186-19,821,655 USD compared to no vaccination. Administering 2 doses of 9vHPV vaccine emerged as the most cost-effective strategy, boasting 406 USD/QALY, within a lower willingness to pay threshold. Sensitivity analysis demonstrated an 80% probability of the cost-effectiveness of the 2 doses of 9vHPV vaccine regimen. Furthermore, uncertainty around the costs of vaccination and vaccine efficacy exerted the most substantial influence on the cost-effectiveness findings. CONCLUSION: Oping for 2 doses of 9vHPV vaccine in conjunction with a primary HPV screening represents the most cost-effective option for implementing a school-based HPV vaccination program targeting 12-year-old girls in Thailand. Such findings provide valuable insights for policymakers in the realm of cervical cancer prevention.

Using a Dynamic Model to Estimate the Cost-Effectiveness of HPV Vaccination in Iran.

This study aimed to determine the cost-effectiveness of vaccination against HPV. An age-sex structured dynamic disease transmission model was created to estimate the spread of HPV and the HPV-related incidence of cervical cancer (CC) in Iran. Sixteen age groups of men and women were incorporated to reflect the differences in sexual preferences, vaccination uptake, and disease-related outcomes. Three scenarios were evaluated by using an Incremental Cost-Effectiveness Ratio (ICER) with gained quality-adjusted life years (QALYs). ICER values below one gross domestic product (GDP) per capita are evaluated as highly cost-effective. Vaccination reduces the number of infections and CC-related mortality. Over time, the vaccinated group ages and older age groups experience protection. An initial investment is required and savings in treatment spending reduce the impact over time. Vaccinating girls only was found to be cost-effective, with an ICER close to once the GDP per capita. Vaccinating both sexes was shown to be less cost-effective compared to girls only, and vaccinating boys only was not found to be cost-effective, with an ICER between once and three times, and greater than three times the GDP per capita, respectively. The estimates are conservative since societal cost-saving and the impact of other HPV-related illnesses were not considered and would likely reduce the ICERs.

Quality of life and quality-adjusted life years after stroke in Sierra Leone.

BACKGROUND: Stroke is a leading cause of mortality and negatively affects health-related quality of life (HRQoL). HRQoL after stroke is understudied in Africa and there are no reports of quality-adjusted life years after stroke (QALYs) in African countries. We determined the impact of stroke on HRQoL after stroke in Sierra Leone. We calculated QALYs at 1 year post-stroke and determined sociodemographic and clinical variables associated with HRQoL and QALYs in this population. METHODS: A prospective stroke register was established at the two-principal adult tertiary government hospitals in Freetown, Sierra Leone. Participants were followed up at 7, 90 days, and 1 year post-stroke to capture all-cause mortality and EQ-5D-3L data. QALYs were calculated at the patient level using EQ-5D-3L utility values and survival data from the register, following the area under the curve method. Utilities were based on the UK and Zimbabwe (as a sensitivity analysis) EQ-5D value sets, as there is no Sierra Leonean or West African value set. Explanatory models were developed based on previous literature to assess variables associated with HRQoL and QALYs at 1 year after stroke. To address missing values, Multiple Imputation by Chained Equations (MICE), with linear and logistic regression models for continuous and binary variables, respectively, were used. RESULTS: EQ-5D-3L data were available for 373/460 (81.1%), 360/367 (98.1%), and 299/308 (97.1%) participants at 7, 90 days, and 1 year after stroke. For stroke survivors, median EQ-5D-3L utility increased from 0.20 (95% CI: -0.16 to 0.59) at 7 days post-stroke to 0.76 (0.47 to 1.0) at 90 days and remained stable at 1 year 0.76 (0.49 to 1.0). Mean QALYs at 1 year after stroke were 0.28 (SD: 0.35) and closely associated with stroke severity. Older age, lower educational attainment, patients with subarachnoid hemorrhage and undetermined stroke types all had lower QALYs and lower HRQoL, while being the primary breadwinner was associated with higher HRQoL. Sensitivity analysis with the Zimbabwe value set did not significantly change regression results but did influence the absolute values with Zimbabwe utility values being higher, with fewer utility values less than 0. CONCLUSION: We generated QALYs after stroke for the first time in an African country. QALYs were significantly lower than studies from outside Africa, partially explained by the high mortality rate in our cohort. Further research is needed to develop appropriate value sets for West African countries and to examine QALYs lost due to stroke over longer time periods. DATA AVAILABILITY: The Stroke in Sierra Leone anonymized dataset is available on request to researchers, see data access section.

The role and impact of health economics in the optimization of patient care in osteoarthritis: insights from a practical example.

Osteoarthritis (OA) is a degenerative joint disease with a substantial global burden, causing chronic pain and reduced quality of life. Managing OA efficiently while maximizing healthcare resources is crucial. Health economics and health technology assessment (HTA) are central tools providing a framework to evaluate the clinical, economic, and ethical aspects of healthcare technologies and interventions. This article presents some insights into the role of health economics and the HTA process in OA management. It also illustrates an example of cost-effectiveness analysis in a specific healthcare context, on the basis of a recent clinical trial involving hyaluronic acid treatment for knee OA. While HTA offers valuable insights, it faces challenges like data availability and resource constraints. Integrating health economics into decision-making can enhance patient care and allocate resources effectively in OA and other healthcare domains.