Difference In Quality-adjusted Life-years Research Articles

P1251 A Care Pathway for Inflammatory Bowel Disease can Reduce Healthcare Costs and is Cost-Effective

Abstract Background Hospitals in the southwest of the Netherlands collaborate to improve inflammatory bowel disease (IBD) care. As part of this initiative, a standardised care pathway (CP) for the treatment of IBD with biologics and small new molecules has been developed. The CP addresses initiation, switching, and discontinuation of therapy, as well as diagnostic testing and treatment follow-up. The CP has been implemented in six of the eight hospitals to improve patient outcomes, standardise care and reduce healthcare costs. This natural experiment provides an opportunity to evaluate the cost-effectiveness of the CP using a quasi-experimental design. We aimed to estimate the causal effect of CP implementation on costs and health outcomes. Methods A cost-utility analysis with a societal perspective was conducted. Adult patients with an IBD diagnosis for at least 3 months and treated with biologics and small new molecules were eligible for inclusion. Costs were assessed for both standard care (December 2020 - December 2021) and the CP over a one-year period (March 2022 – March 2023), without discounting costs and effects and standardizing costs to 2022. Missing data were handled using single imputation nested within a bootstrap procedure. The analysis employed a difference-in-differences (DiD) approach to estimate causal effects of the CP implementation, controlling for pre-existing differences between hospitals and general time trends that would have occurred without the intervention. For the cost-effectiveness analysis, we utilized the cumulative incremental costs and mean incremental quality adjusted life years (QALYs) from the post-intervention DiD effects. The DiD-derived incremental costs and QALYs were used to calculate the incremental cost-effectiveness ratio (ICER) and construct cost-effectiveness acceptability curves using normal approximation. Results Approximately 25% of the eligible population was included. At baseline, patients in intervention (n=842) and control hospitals (n=331) had comparable characteristics in terms of age, sex, diagnosis and comorbidities. The DiD-analysis showed cost savings primarily driven by reduced hospital costs (-€1,107; 95%CI: -€2,130 to -€109) while maintaining quality of life (QALY difference: 0.01; 95%CI: -0.02 to 0.04). The ICER was located in the southeast corner of the cost-effectiveness plane, indicating that the treatment with the CP is a dominant strategy compared to standard care (Figure 1). Conclusion A CP for the treatment of IBD with biologics and small new molecules was developed to improve IBD care and reduce healthcare costs. Our results showed that standardising IBD care is effective for reducing healthcare costs, while maintaining quality of life.

P1277 Cost-effectiveness of BOOST online intervention for supported self-management of pain, fatigue and bowel incontinence in Inflammatory Bowel Disease

Abstract Background People with IBD often experience symptoms of abdominal pain, fatigue and bowel incontinence that affect substantially their daily lives1. IBD-BOOST is the first randomised controlled trial (RCT) assessing the effectiveness of an online self-management intervention, BOOST, in managing these symptoms in IBD2. We report the cost-effectiveness of BOOST intervention compared to care as usual for people with IBD. Methods A cost-utility analysis was conducted alongside the pragmatic IBD-BOOST RCT of 780 patients requesting help for pain, fatigue or incontinence symptoms (mean age 49 years, 67% women, 55% with Crohn’s disease)3. The BOOST intervention costs and costs from patient-reported health service use, out-of-pocket expenses and time off work were calculated for each participant over the 12 months of follow-up. Quality-adjusted life-years (QALYs) over the 12 months were evaluated using the EQ-5D-5L questionnaires administered to participants at baseline, 6- and 12-months. Missing data (13% at 6 months and 38% at 12 months across the trial arms) was imputed using multiple imputation. Differences in total costs and QALYs between study arms were estimated using mixed effects models adjusting for pre-specified baseline factors. Incremental cost effectiveness ratios (ICERs) and net monetary benefit from UK healthcare and societal perspectives are reported. Results At recruitment, study participants reported quality of life (EQ-5D utility) of 0.73 (SD 0.22) and overall IBD-related costs of £2321 (SD 3170) over the previous 3 months with cost of biologics accounting for 47% of these costs, followed by hospital outpatient services (22%) and work productivity loss costs (14%) (Figure 1). After missing data imputation and controlling for covariates, the BOOST intervention (£151 per participant) was estimated to have led to health care cost savings of £746 (SE 356), other costs savings of £89 (SE 236) and incremental QALYs of 0.017 (SE 0.007) over the one-year follow-up per participant resulting in cost savings per QALY gained of £34,599 from the health services perspective and £39,745 from the societal perspective. The probability that the BOOST intervention was cost effective when compared with usual care was above 98% for willingness-to-pay thresholds of £20,000 to £30,000 from both health services and societal perspectives (Figure 2). Conclusion Our findings indicate that the BOOST intervention for supported self-management of symptoms of pain, fatigue and bowel incontinence in people with IBD is cost-effective.

Cost-effectiveness of digoxin versus beta blockers in permanent atrial fibrillation: the Rate Control Therapy Evaluation in Permanent Atrial Fibrillation (RATE-AF) randomised trial

BackgroundAtrial fibrillation (AF) is a major and increasing burden on health services. This study aimed to evaluate the cost-effectiveness of digoxin versus beta-blockers for heart rate control in patients with...

A cost-effectiveness analysis of sotorasib as second-line treatment for patients with KRAS-G12C-mutated metastatic non-small cell lung cancer (mNSCLC) in Switzerland.

Because of the lack of effective targeted treatment options, docetaxel has long been the standard second-line therapy for patients with advanced non-small cell lung cancer, including the Kirsten rat sarcoma virus (KRAS) G12C mutation.The CodeBreak 200 trial demonstrated that sotorasib, a new drug targeting the G12C-mutated KRAS protein, modestly improved progression-free survival compared with docetaxel in patients whose cancer had progressed after receiving platinum chemotherapy and programmed cell death protein 1 (PD-1) / programmed death ligand 1 (PD-L1) inhibitors as first-line treatment. Consequently, sotorasib received temporary approval in Switzerland. Our analysis assessed the cost-effectiveness of sotorasib as a second-line treatment in Swiss patients with non-small cell lung cancer from the perspective of the Swiss statutory health insurance system. A partitioned survival model based on the CodeBreak 200 trial was constructed with a time horizon of 10 years and a discount rate of 3% for costs and quality-adjusted life years (QALYs). Parametric survival curves were fitted to the published Kaplan-Meier data, and survival was extrapolated. QALYs were obtained from the CodeBreak 100 trial and the literature. The costs of drugs, drug administration, diagnostics, disease management, and adverse events were considered. Because the price of sotorasib has not been established in Switzerland, two scenarios were analysed: the first used the published expected monthly United Kingdom (UK) price in Swiss francs (CHF 7870); the second used one-quarter of that price (CHF 1968), according to the lower dose used in the most recent trial, under the condition that one-quarter of the original sotorasib dose is equally effective. Treatment costs of adverse events were included. Log-normal functions best fitted the survival curves from CodeBreak 200. For sotorasib versus docetaxel, our estimation showed no difference in QALYs (1.28 QALYs for both treatments), as the reduced adverse events reported in CodeBreak 200 for sotorasib had a minimal impact on the QALYs in our calculation. This made an incremental cost-effectiveness ratio (ICER) calculation irrelevant. Total per-patient costs were CHF 138,894 for the full sotorasib dose, CHF 82,741 for the one-quarter dose, and CHF 80,383 for docetaxel. These results were robust in 99% of probabilistic simulations. Sotorasib did not demonstrate cost-effectiveness at the full dosage nor when reduced to a quarter of the dose. The primary factors motivating clinicians to prescribe sotorasib are its superior overall response rate compared with docetaxel and the reported improvement in patients' quality of life. These factors suggest that it would be reasonable to price it at approximately one-quarter of the assumed cost in the UK.

Longer-term survival, quality of life, and cost-effectiveness of conservative versus liberal oxygenation targets in critically ill children: a pre-specified analysis from Oxy-PICU, a multicentre, open, parallel-group, randomised controlled trial.

Peripheral oxygen saturation (SpO2) above 94% is typical in children in paediatric intensive critical care units (PICUs) who are receiving invasive ventilation and supplemental oxygen. In a previous report from the Oxy-PICU trial, we showed that lower (conservative) oxygenation targets (SpO2 88-92%) are beneficial, showing small but statistically significant differences in duration of organ support and large but non-significant cost reductions at 30 days. In this pre-specified analysis of the Oxy-PICU trial, we compare longer-term outcomes and cost-effectiveness of conservative versus liberal (SpO2 >94%) oxygenation targets in children with emergency PICU admission. Oxy-PICU was a pragmatic, multicentre, open-label, randomised controlled trial in England and Scotland. Eligible children were older than 38 weeks and younger than 16 years and had been admitted for emergency care in one of 15 participating PICUs, where they received invasive respiratory support for abnormal gas exchange. Participants were randomly assigned (1:1) to either a conservative oxygenation target (SpO2 88-92%) or liberal oxygenation target (SpO2 >94%). Survival status was assessed at 90 days and 1 year, and health-related quality of life (HRQoL), quality-adjusted life-years (QALYs), health-care costs, and incremental net monetary benefit were assessed at 1 year after the index hospital admission and randomisation. HRQoL was measured with age-appropriate Paediatric Quality of Life Generic Core Scales and mapped onto the Child Health Utility 9D index score. HRQoL and survival data were combined to construct QALYs. Costs at 1 year were derived from use of hospital, outpatient, and community health services. The trial was registered in the ISRCTN registry (ISRCTN92103439). 2040 children were enrolled between Sept 1, 2020 and May 15, 2022. 1868 (91·6%) children were included in the 90-day survival analysis; of these 930 (49·8%) had been assigned liberal oxygen and 938 (50·2%) conservative oxygen. 1867 (91·5%) children were included in the 1-year survival analysis; 930 (49·8%) had been assigned liberal oxygenation and 937 (50·2%) conservative oxygen. At 90 days, 35 (3·7%) patients in the conservative oxygenation group and 45 (4·8%) patients in the liberal oxygenation group had died (adjusted hazard ratio [aHR] 0·75 [95% CI 0·48 to 1·17]). By 1 year, 52 (5·5%) patients in the conservative oxygenation group and 66 (7·1%) patients in the liberal oxygenation group had died (aHR 0·77 [95%CI 0·53 to 1·10]). Overall, mean HRQoL, life-years, and QALYs at 1 year were similar in the two groups. The adjusted incremental effect on cost of conservative oxygenation versus liberal oxygenation was -£879 (95% CI -9036 to 7278), whereas the incremental difference in QALYs was estimated at 0·001 (-0·010 to 0·011), leading to an incremental net monetary benefit of £894 (-7290 to 9078) associated with conservative oxygenation relative to liberal oxygenation. These results did not vary by age (<12 months vs ≥12 months), comorbidity at baseline, age-adjusted heart rate, or haemoglobin level at admission and were robust to alternative assumptions. Compared with usual care (SpO2 >94%) for invasively ventilated children who are admitted as an emergency to a PICU, conservative oxygenation (SpO2 88-92%) was not associated with differences in longer-term survival, costs, or cost-effectiveness. Taken together with previous findings of Oxy-PICU that conservative oxygenation compared with liberal oxygenation leads to better patient-centred and parent-centred outcomes at 30 days, these findings support the use of conservative oxygenation targets for this population. UK National Institute for Health and Social Care Research Health Technology Assessment Programme.

Economic analysis of hyperbaric oxygen therapy for the treatment of ischaemic diabetic foot ulcers.

The aim was to determine the cost-effectiveness and cost-utility of additional hyperbaric oxygen therapy (HBOT) compared to standard care (SC) for ischaemic diabetic foot ulcers (DFUs) regarding limb salvage and health status. An economic analysis was conducted, comprising cost-effectiveness and cost-utility analyses, with a 12-month time horizon, using data from the DAMO₂CLES multicentre randomised clinical trial. Cost-effectiveness was defined as cost per limb saved and cost-utility as cost per quality-adjusted life year (QALY). The difference in cost effectiveness between HBOT+SC and SC alone was determined via an incremental cost-effectiveness ratio (ICER). One-hundred and twenty patients were included, with 60 allocated to HBOT+SC and 60 to SC. No significant cost difference was found in the intention-to-treat analysis: €3,791 (bias corrected and accelerated [BCA] 95% CI, €3,556 - €-11,138). Cost per limb saved showed an ICER of €37,912 (BCA 95% CI €-112,188 - €1,063,561) for HBOT+SC vs. SC. There was no significant difference in mean QALYs: 0.54 for HBOT+SC vs. 0.56 for SC alone (-0.02; BCA 95% CI -0.11-0.08). This resulted in a cost-utility of minus €227,035 (BCA 95% CI €-361,569,550 - €-52,588) per QALY. Subgroup analysis for Wagner stages III/IV showed an ICER of €19,005 (BCA 95%CI, -€18,487 - €264,334) while HBOT did not show any benefit for Wagner stage II. HBOT as an adjunct to SC showed no significant differences in costs and effectiveness for patients with DFUs regarding limb salvage and health status. However, for patients with Wagner stage III/IV ischaemic DFUs there was a trend towards better effectiveness and cost-effectiveness.

Clinical and economic implications of focal dissection treatment following percutaneous transluminal angioplasty of the superficial femoral artery: an exploratory analysis based on the TOBA II Study.

Aim: Percutaneous transluminal angioplasty (PTA) for peripheral artery disease (PAD) commonly leads to dissections which are associated with higher target lesion revascularization (TLR) rates. Clinical and economic consequences of dissection management in the femoropopliteal artery following PTA, and specifically the potential economic benefit of focal dissection repair using the novel Tack Endovascular System, remain unknown. Methods: A decision-analytic model was used to estimate 24-month clinical events, costs and quality-adjusted life year (QALY) gain for a Tack-supported versus status-quo PTA strategy. Patient and lesion characteristics and TLR rates were derived from the PTA cohort of the TOBA II clinical trial, an observational cohort, and literature. Cost-effectiveness was determined from a US payer and provider perspective separately for the non-severe (grade A or B), severe (grade C and higher) and the entire dissection cohort. Results: TLR rates were lower for the Tack-supported strategy compared with PTA (7.7 vs 27.4% in the non-severe, 13.9 vs 25.8% in the severe and 12.0 vs 26.3% in the entire dissection cohort). Cost and QALY differences were +$297/+0.0110 in the non-severe dissection cohort and -$1602/+0.0067 in the severe dissection cohort, resulting in an incremental cost-effectiveness ratio (ICER) of $25,622 in the non-severe cohort and dominance in the severe cohort and the entire cohort. Conclusion: Compared with a 'status-quo' approach, proactive focal stenting may lead to fewer reinterventions and improved quality of life. There appears to be a graded economic benefit of focal dissection treatment, being cost-effective in non-severe dissections and even cost saving in severe dissections.

Cost-utility analysis of provision of e-cigarette starter kits for smoking cessation in emergency departments: An economic evaluation of a randomized controlled trial.

To assess the cost-effectiveness of the Cessation of Smoking Trial in Emergency Department (COSTED) intervention compared with signposting to local stop smoking service (SSS) from the National Health Service (NHS) and personal social services (PSS) perspective. This was a two-group, multi-centre, pragmatic, individually randomized controlled trial set in six Emergency Departments (EDs) in urban and rural areas in the United Kingdom. Adult (≥ 18 years) daily smokers (at least one cigarette or equivalent per day) but not daily e-cigarette users, with carbon monoxide reading ≥8parts per million, attending the ED (n= 972) were included. The intervention consisted of provision of an e-cigarette starter kit plus brief smoking cessation advice and referral to a local SSS. Control was an information card on how to access local SSS. Intervention costs included costs of training and delivery. Control costs included costs of printing information cards. Costs of smoking cessation and health-care services were estimated based on quantities reported by participants and unit costs extracted from secondary sources. The effects were measured by quality-adjusted life years (QALYs) derived from EQ-5D-5L. Other outcomes were smoking cessation measures. The primary outcome was incremental cost-effectiveness ratio (ICER), which was calculated by dividing the difference in costs by the difference in QALYs between groups. The mean intervention costs were £48 [standard error (SE) = £0] per participant and the mean control costs were £0.2 (SE = £0) per participant. Using regression estimates, total costs were £31 [95% confidence interval (CI) = -£341 to £283] higher and 6-month QALYs were 0.004 (95% CI = -0.004 to 0.014) higher in the intervention group than in the control group. The ICER was calculated at £7750 (probability of cost-effective at range £20 000-30 000: 72.2-76.5%). The UK Cessation of Smoking Trial in Emergency Department (COSTED) intervention (provision of an e-cigarette starter kit plus brief smoking cessation advice) was cost-effective compared with signposting to local stop smoking services under the current recommendations of the maximum acceptable thresholds.

Assessing Health Gains: A Cost-Utility Analysis

Abstract Background With the increase in life expectancy and the consequent shift in the epidemiology of chronic diseases, the need to enhance and measure the population’s health status is even more imperative. The aim of this study is to assess health gains and their associated costs provided by an Italian clinic. Methods A retrospective study was conducted on 129 patients admitted for short-term planned hospitalization from June 2020 to August 2023. At both admission and discharge, these patients completed the EQ-5D-5L questionnaire. Quality-Adjusted Life Years (QALYs) were calculated from the difference in EQ-5D-5L scores between discharge and admission, assuming that health gains (discounted at a rate of 3.5%) accumulate up to two years after discharge. Cost-utility analysis was performed using QALYs and invoices issued by the clinic (cost of hospital stay: 250 Euro/day). Patients were stratified by gender, age (geriatric and non-geriatric), and length of stay (LoS). Descriptive statistics (median and interquartile range) and statistical analysis (Mann-Whitney test) were conducted using STATA software. Results The sample comprised 129 patients (55% female) with a median age of 81 [11] years and a median LoS of 16 [16] days. The median QALY gained was 0.33 [0.38], higher for males (0.35 [0.34]) than females (0.29 [0.45]). Patients younger than 65 years old experienced a greater gain in QALY (0.41 [0.42]) than geriatric patients (0.32 [0.38]) (p &amp;gt; 0.05). Patients with a longer LoS had a greater gain in QALY (0.35 [0.42]) than those with a shorter LoS (0.23 [0.29]) (p &amp;lt; 0.05). The median cost per QALY gained was 14,337 Euro, lower for males (13,803 Euro), non-geriatric patients (13,743 Euro), and those with a shorter LoS (10,670 Euro) (p &amp;gt; 0.05). Conclusions Despite disparities in QALY gains between groups, the median cost per QALY gained remained relatively constant. These findings underscore the importance of tailored interventions for effective resource allocation. Key messages • Differences in QALYs indicate the need for personalized healthcare strategies to optimize patient outcomes. • The constant cost per QALY underlines the importance of effective and efficient allocation of resources.

Cost-utility analysis of adapted problem adaptation therapy for depression in mild-to-moderate dementia caused by Alzheimer's disease: PATHFINDER randomised controlled trial.

Depression is common in people with dementia, and negatively affects quality of life. This paper aims to evaluate the cost-effectiveness of an intervention for depression in mild and moderate dementia caused by Alzheimer's disease over 12 months (PATHFINDER trial), from both the health and social care and societal perspectives. A total of 336 participants were randomised to receive the adapted PATH intervention in addition to treatment as usual (TAU) (n = 168) or TAU alone (n = 168). Health and social care resource use were collected with the Client Service Receipt Inventory and health-related quality-of-life data with the EQ-5D-5L instrument at baseline and 3-, 6- and 12-month follow-up points. Principal analysis comprised quality-adjusted life-years (QALYs) calculated from the participant responses to the EQ-5D-5L instrument. The mean cost of the adapted PATH intervention was estimated at £1141 per PATHFINDER participant. From a health and social care perspective, the mean difference in costs between the adapted PATH and control arm at 12 months was -£74 (95% CI -£1942 to £1793), and from the societal perspective was -£671 (95% CI -£9144 to £7801). The mean difference in QALYs was 0.027 (95% CI -0.004 to 0.059). At £20 000 per QALY gained threshold, there were 74 and 68% probabilities of adapted PATH being cost-effective from the health and social care and societal perspective, respectively. The addition of the adapted PATH intervention to TAU for people with dementia and depression generated cost savings alongside a higher quality of life compared with TAU alone; however, the improvements in costs and QALYs were not statistically significant.

A methodological guide for implementing and interpreting results of probabilistic analysis.

Probabilistic analysis, also referred to as probabilistic sensitivity analysis (PSA), is used extensively in cost-effectiveness evaluations of health technologies. We present methodological guidance for implementing probabilistic analysis and interpreting its results for policy and decision-making. We review the methodological issues related to common practices in probabilistic analysis, explore aspects that are currently not widely addressed in the health economics literature, and provide an overview of recent methodological developments. We use examples to highlight the advantages and disadvantages of common tools used for presenting probabilistic analysis results, including the cost-effectiveness acceptability curve (CEAC), cost-effectiveness acceptability frontier (CEAF), and value of information (VOI) analysis. We raise and address issues related to using Monte Carlo standard error to determine the number of iterations required, the implications of large uncertainty, and the credibility and meaningfulness of small differences in quality-adjusted life-years (QALYs). We then discuss evolving methods in probabilistic analysis, cautious uses of probabilistic analysis, and factors impacting parameter uncertainty. A deeper understanding of probabilistic analysis methods enables health economists and decision-makers to more effectively address and interpret parameter uncertainty in health economic evaluations, which is essential for making informed policy decisions.

Cost-Effectiveness of an Extended-Role General Practitioner Clinic for Persistent Physical Symptoms: Results From the Multiple Symptoms Study 3 Pragmatic Randomized Controlled Trial

ObjectivesThis study aimed to evaluate the cost-effectiveness of an extended-role general practitioner symptoms clinic (SC), added to usual care (UC) for patients with multiple persistent physical symptoms (sometimes known as medically unexplained symptoms). MethodsThis was a 52-week within-trial cost-utility analysis of a pragmatic multicenter randomized controlled trial comparing SC + UC (n = 178) with UC alone (n = 176), conducted from the primary perspective of the UK National Health Service and personal and social services (PSS). Base-case quality-adjusted life-years (QALYs) were measured using EQ-5D-5L. Missing data were imputed using multiple imputation. Cost-effectiveness results were presented as incremental cost-effectiveness ratios and incremental net monetary benefits. Uncertainty was explored using cost-effectiveness acceptability curves (using 1000 nonparametric bootstrapped samples) and sensitivity analysis (including societal costs, using SF-6D and ICECAP-A capability measure for adults outcomes to estimate QALYs and years of full capability, respectively, varying intervention costs, missing data mechanism assumptions). ResultsMultiple imputation analysis showed that compared with UC alone, SC + UC was more expensive (adjusted mean cost difference: 704; 95% CI £605-£807) and more effective (adjusted mean QALY difference: 0.0447; 95% CI 0.0067-0.0826), yielding an incremental cost-effectiveness ratio of £15 765/QALY, incremental net monetary benefit of £189.22 (95% CI −£573.62 to £948.28) and a 69% probability of the SC + UC intervention arm being cost-effective at a threshold of £20 000 per QALY. Results were robust to most sensitivity analyses but sensitive to missing data assumptions (2 of the 8 scenarios investigated), SF-6D, and ICECAP_A capability measure for adults quality-of-life outcomes. ConclusionsA symptoms clinic is likely to be a potentially cost-effective treatment for patients with persistent physical symptoms.

Cost-effectiveness and cost-utility of optimized mercaptopurine treatment versus placebo in ulcerative colitis patients: The randomized controlled OPTIC trial.

We assessed the cost-effectiveness and cost-utility of therapeutic drug monitoring (TDM)-guided mercaptopurine treatment compared with placebo in ulcerative colitis (UC) patients failing 5-aminosalicylates. Data were gathered alongside the randomized controlled OPTIC trial (EudraCT: 2015-005260-41). The evaluation was performed from a health care and societal perspective as cost-effectiveness and cost-utility analyses with a time horizon of one year. Volumes and costs of in-hospital care, out-of-hospital care, out-of-pocket expenses and productivity loss were assessed. The main outcomes were the extra costs per additional patient who achieved clinical remission and endoscopic improvement at 52weeks (responders) and extra costs per quality-adjusted life-year (QALY) gained. In total, 59 patients were randomized to the intervention (n=29) and control (n=30) group. Non-significant differences in costs were €63 (-€1267 to €1434; P=0.93) in favour of placebo from a health care perspective and -€742 (-€3683 to €2016; P=0.64) in favour of mercaptopurine from a societal perspective. The higher proportion of responders and a non-significant QALY difference of 0.0475 (-0.024-0.117) (P=0.184) favouring patients on mercaptopurine treatment resulted in €165 extra costs per additional responder and €1326 extra costs per QALY gained from a health care perspective. From a societal perspective, dominance over placebo was observed with cost savings of €1937 per additional responder and €15,621 per QALY gained. The probability of optimised mercaptopurine treatment being cost-effective was 0.80 at a willingness to pay per additional QALY of €20,000. TDM-based mercaptopurine treatment in UC patients failing 5-aminosalicylates is a cost-effective strategy from a societal perspective.

Osteochondroplasty with or without labral repair is more cost-effective than arthroscopic lavage with or without labral repair for treatment of young adults with femoroacetabular impingement: A cost-utility analysis based on data from a randomized controlled trial

ObjectivesThe objective of this study was to conduct a cost–utility analysis of osteochondroplasty with or without labral repair compared to arthroscopic lavage with or without labral repair for femoroacetabular impingement (FAI) from a Canadian public payer perspective. MethodsA Markov model was constructed to compare the lifetime quality-adjusted life years (QALYs) and costs of the two treatment strategies. The target population was surgical FAI patients aged 36 years. The primary data source was patient-level data from the Femoroacetabular Impingement Randomised Controlled Trial, which evaluated the efficacy of the surgical correction of FAI via arthroscopic osteochondroplasty with or without labral repair compared to arthroscopic lavage with or without labral repair in Canada. Long-term data were extrapolated using a generalized gamma model. The primary outcome was the incremental cost-effectiveness ratio, calculated by dividing the difference in costs by the difference in QALYs between osteochondroplasty and lavage, with or without labral repair. Probabilistic sensitivity analyses and one-way sensitivity analyses were used to characterize uncertainty of model parameters and assumptions. ResultsOver a lifetime horizon, osteochondroplasty, with or without labral repair, had a greater expected benefit (0.63 QALYs gained per patient) and lower costs ($955.89 saved per patient), as compared with lavage with or without labral repair. Probabilistic sensitivity analyses demonstrated that the probability of osteochondroplasty, with or without labral repair, being cost-effective was 90.5% at a commonly used willingness-to-pay threshold of $50,000/QALY in Canada. Across all one-way sensitivity analyses, osteochondroplasty with or without labral repair remained a cost-effective option. ConclusionOver a lifetime time horizon, osteochondroplasty, with or without labral repair, is a cost-effective treatment strategy for young adults with FAI. Future research involving real-word data is needed to further validate these findings. Level of evidenceIII.

The population-level effects of omitting chemotherapy guided by a 21-gene expression assay in node-positive breast cancer: a simulation modeling study

BackgroundA recent trial showed that postmenopausal women diagnosed with hormone receptor-positive, human epidermal growth factor receptor-2 (HER2)-negative, lymph node-positive (1–3 nodes) breast cancer with a 21-gene recurrence score of ≤ 25 could safely omit chemotherapy. However, there are limited data on population-level long-term outcomes associated with omitting chemotherapy among diverse women seen in real-world practice.MethodsWe adapted an established, validated simulation model to generate the joint distributions of population-level characteristics of women diagnosed with early-stage breast cancer in the U.S. Input parameters were derived from cancer registry, meta-analyses, and clinical trial data. The effects of omitting chemotherapy on 10-year distant recurrence-free survival, life-years, and quality adjusted life-years (QALYs) were modeled for premenopausal and postmenopausal women. QALYs were discounted at 3%. Results were evaluated for subgroups stratified by race and ethnicity. Sensitivity analyses included testing results across a range of inputs. The model was validated using the published RxPONDER trial data.ResultsIn premenopausal women, the 10-year distant recurrence-free survival rates were 85.3% with chemo-endocrine and 80.1% with endocrine therapy. The estimated life-years and QALYs gained with chemotherapy in premenopausal women were 2.1 and 0.6, respectively. There was no chemotherapy benefit in postmenopausal women. There was no variation in the absolute benefit of chemotherapy across racial or ethnic subgroups. However, there were differences in distant recurrence-free survival rates, life-years, and QALYs across groups. Sensitivity analysis showed similar results. The model closely replicated the RxPONDER trial.ConclusionsModeled population-level outcomes show a small chemotherapy benefit in premenopausal women, but no benefit among postmenopausal women. Simulation modeling provides a useful tool to extend trial data and evaluate population-level outcomes.

Cost-Utility Analysis of Antipsychotic Reduction and Discontinuation in Patients With Long-Term Schizophrenia and Psychosis in English Mental Health Trusts: The RADAR Study

ObjectivesThe current recommended treatment for patients with recurrent episodes of schizophrenia and related conditions is antipsychotic medication. However, many antipsychotic users remain functionally impaired and experience serious physical and mental side effects. This study aims to assess the cost-effectiveness of a gradual antipsychotic reduction and discontinuation strategy compared with maintenance treatment over 24 months from mental health services, health and social care, and societal perspectives. MethodsNineteen mental health trusts recruited patients to the Research into Antipsychotic Discontinuation and Reduction (RADAR) randomized controlled trial. Quality-adjusted life-years were calculated from patient-reported EQ-5D-5L, with years of full capability calculated from the patient-reported ICECAP-A. Mental health services use and medication was collected from medical records. Other resource use and productivity loss was collected using self-completed questionnaires. Costs were calculated from published sources. ResultsA total of 253 participants were randomized: 126 assigned to antipsychotic dose reduction and 127 to maintenance. There were no significant differences between arms in total costs for any perspectives. There were no significant difference in quality-adjusted life-years (−0.035; 95% CI: −0.123 to 0.052), whereas years of full capability were significantly lower in the reduction arm compared with the maintenance arm (baseline-adjusted difference: −0.103; 95% CI: −0.192 to −0.014). The reduction strategy was dominated by maintenance for all analyses and was not likely to be cost-effective. ConclusionsIt is unlikely that gradual antipsychotic reduction and discontinuation strategy is cost-effective compared with maintenance over 2-years for patients with schizophrenia and other recurrent psychotic disorders who are on long-term antipsychotics.

Cost-effectiveness analysis of soft bandage and immediate discharge versus rigid immobilization in children with distal radius torus fractures.

The aim of this trial was to assess the cost-effectiveness of a soft bandage and immediate discharge, compared with rigid immobilization, in children aged four to 15 years with a torus fracture of the distal radius. A within-trial economic evaluation was conducted from the UK NHS and personal social services (PSS) perspective, as well as a broader societal point of view. Health resources and quality of life (the youth version of the EuroQol five-dimension questionnaire (EQ-5D-Y)) data were collected, as part of the Forearm Recovery in Children Evaluation (FORCE) multicentre randomized controlled trial over a six-week period, using trial case report forms and patient-completed questionnaires. Costs and health gains (quality-adjusted life years (QALYs)) were estimated for the two trial treatment groups. Regression was used to estimate the probability of the new treatment being cost-effective at a range of 'willingness-to-pay' thresholds, which reflect a range of costs per QALY at which governments are typically prepared to reimburse for treatment. The offer of a soft bandage significantly reduced cost per patient (saving £12.55 (95% confidence interval (CI) -£5.30 to £19.80)) while QALYs were similar (QALY difference between groups: 0.0013 (95% CI -0.0004 to 0.003)). The high probability (95%) that offering a bandage is a cost-effective option was consistent when examining the data in a range of sensitivity analyses. In addition to the known clinical equivalence, this study found that the offer of a bandage reduced cost compared with rigid immobilization among children with a torus fracture of the distal radius. While the cost saving was small for each patient, the high frequency of these injuries indicates a significant saving across the healthcare system.

Short-term cost-utility analysis of biventricular cardiac resynchronization therapy in comparison with left bundle branch area pacing cardiac resynchronization therapy

Abstract Background Cardiac resynchronisation therapy (CRT) is a well-established form of treatment for patients with heart failure and cardiac dyssynchrony. Recently, left bundle branch area pacing (LBBAP) has emerged as a more physiological alternative for biventricular pacing (BiVP) cardiac resynchronization therapy (1,2). Purpose The purpose of this study was to conduct a cost-utility analysis for LBBAP CRT, compared to BiVP CRT in a cohort of patients with heart failure from the Spanish health system perspective. Methods A multicenter prospective quasi-experimental study in which 44 consecutive patients underwent CRT was conducted. According to the study protocol, the four centers would perform their usual clinical practice: two out of them would perform conventional BiVP-CRT while the other half LBBAP-CRT. Intention-to-treat analysis was carried-out. Health outcomes were quality-adjusted life years (QALYs), measured by the EuroQol-5D-5L questionnaire, which the patients were asked to complete at baseline and 12 weeks after the CRT device implantation. The total direct costs include device, leads, guidewires, introducer kits, sheats and operating theater time. The results were expressed as the incremental cost-utility ratio (ICUR). In order to analyze the uncertainty of ICUR results, we performed a non-parametric bootstrapping with 1000 replications. Results The LBBAP procedure was attempted for a total of 26 consecutive patients (1 patient underwent rescue BiVP), while BiVP was attempted for 18 patients (2 patients switched to LBBAP). Mean QALYs over 3 months were 0.1914 ± 0.009 in the LBBAP CRT group and 0.1904 ± 0.0112 in the BiVP CRT. Improvements from baseline were greater in the LBBA CRT group but not statistically significant. The total costs at the end of the intervention were 7546.6 €± 602.15€ in the LBBAP CRT group and 16064.56€ ± 723.7€ in the BiVP CRT group. When excluding from the analysis the cases in which ICD was implanted, the mean difference in QALYs is 0.002; 95% confidence interval [95%CI]: -0.03 to 0.036) and the mean saving is -3.510.38 €. The LBBAP CRT leads to cost savings for the health system and similar QALYs outcomes; 95%CI: -6.660 to -360.71. Based on 1000 bootstrapping replication, the cost savings of using LBBAP vs BiVP occur in 99% of the simulations. Conclusion LBBAP CRT is an efficient alternative to BiVP CRT for cardiac resynchronization in patients with heart failure with similar results in terms of QALYs but with significant average savings per patient.Cost-utility plane for 1000 bootstrappedBaseline characteristics of patients

Cost-effectiveness of pessary therapy versus surgery for symptomatic pelvic organ prolapse: an economic evaluation alongside a randomised non-inferiority controlled trial

ObjectiveTo evaluate the cost-effectiveness of pessary therapy as an initial treatment option compared with surgery for moderate to severe pelvic organ prolapse (POP) symptoms in secondary care from a healthcare...

Health utility values of breast cancer treatments and the impact of varying quality of life assumptions on cost-effectiveness.

In breast cancer research, utility assumptions are outdated and inconsistent which may affect the results of quality adjusted life year (QALY) calculations and thereby cost-effectiveness analyses (CEAs). Four hundred sixty four female patients with breast cancer treated at Erasmus MC, the Netherlands, completed EQ-5D-5L questionnaires from diagnosis throughout their treatment. Average utilities were calculated stratified by age and treatment. These utilities were applied in CEAs analysing 920 breast cancer screening policies differing in eligible ages and screening interval simulated by the MISCAN-Breast microsimulation model, using a willingness-to-pay threshold of €20,000. The CEAs included varying sets on normative, breast cancer treatment and screening and follow-up utilities. Efficiency frontiers were compared to assess the impact of the utility sets. The calculated average patient utilities were reduced at breast cancer diagnosis and 6 months after surgery and increased toward normative utilities 12 months after surgery. When using normative utility values of 1 in CEAs, QALYs were overestimated compared to using average gender and age-specific values. Only small differences in QALYs gained were seen when varying treatment utilities in CEAs. The CEAs varying screening and follow-up utilities showed only small changes in QALYs gained and the efficiency frontier. Throughout all variations in utility sets, the optimal strategy remained robust; biennial for ages 40-76 years and occasionally biennial 40-74 years. In sum, we recommend to use gender and age stratified normative utilities in CEAs, and patient-based breast cancer utilities stratified by age and treatment or disease stage. Furthermore, despite varying utilities, the optimal screening scenario seems very robust.