Cost Effectiveness of the Baveno VI Criteria Compared With Endoscopy for High-Risk Varices in Patients With Child-Pugh A Cirrhosis.

BACKGROUND & AIMS: Although upper gastrointestinal endoscopy (EGD) remains the gold standard for detecting varices in cirrhosis, the Baveno VI criteria proposed a combination of transient elastography and platelet count that could rule out high-risk varices, therefore sparing the need for an endoscopy, with significant potential cost savings. We performed a cost-effectiveness analysis of the Baveno VI criteria compared with EGD in the diagnosis of high-risk varices in cirrhosis. METHODS: We built an analytical decision model to estimate the cost and benefits of using the Baveno VI criteria compared with EGD in patients with Child-Pugh A cirrhosis. The analysis was performed from the UK National Health Service perspective, over 1, 5, and 20 years. A Markov model was populated with data from published evidence. Outcomes were measured in terms of quality-adjusted life years (QALYs) and avoided deaths. The analyses were repeated for Canada and Spain, using relevant cost inputs. RESULTS: The Baveno VI criteria were cost effective compared with endoscopy in all analyses. For 1000 patients, they produced 0.16 additional QALYs at an incremental cost of £326 ($443.41) over 5 years, resulting in an incremental cost of £2081 ($2830) per additional QALY gained. The incremental net monetary benefit of Baveno VI compared with EGD was £2808 ($3819) over 5 years per patient. Baveno VI criteria also were cost effective in Canada and Spain. Deterministic and probabilistic sensitivity analysis supported these findings. CONCLUSIONS: The findings demonstrate that the Baveno VI criteria are cost effective, suggesting that they should be considered for widespread implementation on the basis of safety, appropriateness, and economic grounds.

REMOTE-ILD study: Description of the protocol for a multicentre, 12-month randomised controlled trial to assess the clinical and cost-effectiveness of remote monitoring of spirometry and pulse oximetry in patients with interstitial lung disease.

INTRODUCTION: Remote monitoring of home physiological measurements has been proposed as a solution to support patients with chronic diseases as well as facilitating virtual consultations and pandemic preparedness for the future. Daily home spirometry and pulse oximetry have been demonstrated to be safe and acceptable to patients with interstitial lung disease (ILD) but there is currently limited evidence to support its integration into clinical practice. AIM: Our aim is to understand the clinical utility of frequent remote physiological measurements in ILD and the impact of integrating these into clinical practice from a patient, clinical and health economic perspective. METHODS AND ANALYSIS: 132 patients with fibrotic ILD will be recruited and randomised to receive either usual care with remote digital monitoring of home spirometry and pulse oximetry or usual care alone for 12 months. All participants will complete health-related quality of life and experience questionnaires.The primary outcome compares the availability of spirometry measurements within the 2 weeks preceding planned clinic appointments. Secondary outcomes will explore other aspects of clinical and cost-effectiveness of the remote monitoring programme. ETHICS AND DISSEMINATION: The study has been approved by the Camden and Kings Cross Research Ethics Committee (22/LO/0309). All participants will provide informed consent.This study is registered with www. CLINICALTRIALS: gov (NCT05662124).The results of the study will be submitted for presentation at regional and national conferences and submitted for peer-reviewed publication. Reports will be prepared for study participants with the support from our public involvement representatives through the charity Action for Pulmonary Fibrosis.

Cost-Effectiveness of Whole-Genome vs Whole-Exome Sequencing Among Children With Suspected Genetic Disorders.

Importance: The diagnosis of rare diseases and other genetic conditions can be daunting due to vague or poorly defined clinical features that are not recognized even by experienced clinicians. Next-generation sequencing technologies, such as whole-genome sequencing (WGS) and whole-exome sequencing (WES), have greatly enhanced the diagnosis of genetic diseases by expanding the ability to sequence a large part of the genome, rendering a cost-effectiveness comparison between them necessary. Objective: To assess the cost-effectiveness of WGS compared with WES and conventional testing in children with suspected genetic disorders. Design, Setting, and Participants: In this economic evaluation, a bayesian Markov model was implemented from January 1 to June 30, 2023. The model was developed using data from a cohort of 870 pediatric patients with suspected genetic disorders who were enrolled and underwent testing in the Ospedale Pediatrico Bambino Gesù, Rome, Italy, from January 1, 2015, to December 31, 2022. The robustness of the model was assessed through probabilistic sensitivity analysis and value of information analysis. Main Outcomes and Measures: Overall costs, number of definitive diagnoses, and incremental cost-effectiveness ratios per diagnosis were measured. The cost-effectiveness analyses involved 4 comparisons: first-tier WGS with standard of care; first-tier WGS with first-tier WES; first-tier WGS with second-tier WES; and first-tier WGS with second-tier WGS. Results: The ages of the 870 participants ranged from 0 to 18 years (539 [62%] girls). The results of the analysis suggested that adopting WGS as a first-tier strategy would be cost-effective compared with all other explored options. For all threshold levels above €29 800 (US $32 408) per diagnosis that were tested up to €50 000 (US $54 375) per diagnosis, first-line WGS vs second-line WES strategy (ie, 54.6%) had the highest probability of being cost-effective, followed by first-line vs second-line WGS (ie, 54.3%), first-line WGS vs the standard of care alternative (ie, 53.2%), and first-line WGS vs first-line WES (ie, 51.1%). Based on sensitivity analyses, these estimates remained robust to assumptions and parameter uncertainty. Conclusions and Relevance: The findings of this economic evaluation encourage the development of policy changes at various levels (ie, macro, meso, and micro) of international health systems to ensure an efficient adoption of WGS in clinical practice and its equitable access.

The COSI trial: a study protocol for a multi-centre, randomised controlled trial to explore the clinical and cost-effectiveness of the Circle of Security-Parenting Intervention in community perinatal mental health services in England.

BACKGROUND: Perinatal mental health difficulties affect up to 27% of birthing parents during pregnancy and the first postnatal year, and if untreated are associated with difficulties in bonding and long-term adverse outcomes to children. There are large evidence gaps related to psychological treatment, particularly in group therapy approaches and parent-infant interventions. One intervention showing preliminary efficacious findings and user acceptability is Circle of Security-Parenting (COS-P), which is a brief, weekly, group programme. However, these studies were underpowered and predominantly non-randomised, and there has never been a research trial in England or with birthing parents experiencing severe and complex perinatal mental health difficulties. The aim of the research is to conduct a randomised control trial to test whether COS-P will reduce perinatal mental health symptoms in birthing parents accessing NHS perinatal mental health services, compared to treatment as usual (TAU). Secondary objectives include exploring whether the intervention improves parenting sensitivity, emotion regulation skills, attachment security and infant development. Additionally, the project aims to examine whether the intervention is acceptable to parents and NHS staff, and whether it is cost-effective. METHODS: COSI is an individually randomised, single-blind parallel arm controlled trial with an embedded internal pilot aiming to recruit 369 participants in a 2:1 ratio (intervention: TAU). Participants will be recruited from ten NHS community perinatal mental health services in England and screened based on clinical levels of both mental health symptoms (average CORE-OM score ≥ 1.1) and postnatal bonding difficulties (total PBQ score ≥ 12). This trial has 90% power to detect a MCID of 5 points on the CORE-OM. Primary and secondary outcomes will be measured at baseline, 3, 7 and 12 months after baseline. Service use and quality of life measures will also be collected alongside a process evaluation of parents' and interveners' views and experiences. DISCUSSION: This will be the first large pragmatic trial to test whether COS-P is effective for birthing parents with severe and complex perinatal mental health difficulties in improving their mental health symptoms. If shown to be effective, the intervention could be delivered widely across the NHS and other similar services globally. TRIAL REGISTRATION: ISRCTN, ISRCTN18308962. Registered 18 February 2022.

Cost-utility analysis of robotic-assisted radical cystectomy for bladder cancer compared to open radical cystectomy in the United Kingdom.

BACKGROUND: Bladder cancer is the tenth most common cancer in the United Kingdom. Currently, open radical cystectomy (ORC) is the gold standard. Due to the risk of complications and a 2.3-8% mortality rate1, there is growing interest in the use of robot-assisted radical cystectomy (RARC). The aim of this study is to perform a cost-utility analysis, comparing RARC to ORC for bladder cancer patients from the perspective of the National Health Service England. METHODS: A three-stage decision tree: surgery, post-surgery transfusions and complications, in a 90-day time horizon, was produced to simulate possible pathways of patients. The incremental cost-effectiveness ratio (ICER) was calculated based on data derived from current literature. Multiple univariate sensitivity analysis was carried out to evaluate influences of varying costs of RARC and ORC on the ICER. RESULTS: The ICER for RARC compared to ORC resulted in £25,536/QALY. At the lower threshold of £20,000/QALY, RARC resulted in a negative NMB (£-4,843.32) and at the upper threshold of £30,000/QALY, a positive NMB (£624.61) compared to ORC. Threshold analysis showed that the intervention costs of £13,497 and £14,403 are met at the lower and upper threshold respectively. The univariate sensitivity analysis showed that the intervention costs of RARC or ORC, and the probabilities of complications, had the greatest impact on the ICER. CONCLUSION: As the resultant ICER did not fall below the £20,000/QALY threshold, our study did not provide a definitive recommendation for RARC for bladder cancer. Negative values for the NMB at the lower threshold indicated the intervention was not feasible from a cost perspective. At the upper threshold of £30,000/QALY, this situation was reversed. The intervention became cost-effective. Therefore, further research is needed to justify the intervention.

Cost-utility analysis of community occupational therapy in dementia (COTiD-UK) versus usual care: Results from VALID, a multi-site randomised controlled trial in the UK.

BACKGROUND: A community-based occupational therapy intervention for people with mild to moderate dementia and their family carers: the Community Occupational Therapy in Dementia-UK version (COTiD-UK); and Treatment as usual (TAU) were randomly assigned to 468 pairs (each comprising a person with dementia and a family carer) in the Valuing Active Life in Dementia (VALID) randomised controlled trial (RCT). OBJECTIVES: To compare the cost-utility of the COTiD-UK intervention compared to TAU, using data from the VALID RCT. METHODS: We performed a cost-utility analysis estimating mean costs and quality adjusted life years (QALYs) per person with dementia and carer for both treatments over a 26 weeks' time horizon based on resource use data and utility values collected in the trial. RESULTS: Taking the National Health Service and Personal Social Services perspective, including costs and benefits to the person with dementia only, measuring Health Related Quality of Life based on Dementia Quality of Life scale (DEMQOL), accounting for missing data and adjusting for baseline values, there was a significant difference in costs between COTiD-UK and TAU (mean incremental cost for COTiD-UK £784 (95% CI £233 to £1334)), but no significant difference in outcomes (mean QALYs gained 0.00664 (95% CI -0.00404, 0.01732)). The Incremental Net Monetary Benefit (INMB) for COTiD-UK versus TAU was negative at a maximum willingness to pay for a QALY of £20000 (mean -£651, 95% CI -£878 to -£424) or £30000 (mean -£585, 95% CI -£824 to -£345). Extensive sensitivity analyses confirmed the results. CONCLUSIONS: This community-based occupational therapy intervention has a very low probability of being cost-effective.

Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project.

The medicine development process is complex and requires time and effort to ensure safety, efficacy and quality. In paediatrics, this process is even more challenging, as it involves a subgroup of the population that already faces a considerable gap in the clinical evaluation of medicines and devices compared to the adult population. Moreover, access to therapies is heavily influenced by national health technology assessment (HTA) recommendations, which often form the basis for pricing and reimbursement decisions that affect the availability of effective treatments within the national health systems. Yet performing an HTA to assess the relative effectiveness and cost-effectiveness of a new children's treatment has several non-trivial implications, creating a critical issue for the paediatric population. In addition, the advent of innovative health technologies for children emphasises the need to empower the role of HTAs in paediatrics. This article aims at describing the most relevant elements of the drug development process in the paediatric field by focusing on the HTA. Particular attention will be paid to the factors that influence market access for new paediatric medicines and patients' access to treatment. The article will also highlight some central methodological challenges in conducting HTA in the paediatric field. Finally, the article will provide insight into how initiatives, such as conect4children, may subsequently reinforce HTA awareness in the paediatric community and strengthen collaborations through network mechanisms.

Projection of the health and economic impacts of Chronic kidney disease in the Chilean population.

BACKGROUND: Chronic Kidney Disease (CKD) is a leading public health problem, with substantial burden and economic implications for healthcare systems, mainly due to renal replacement treatment (RRT) for end-stage kidney disease (ESKD). The aim of this study is to develop a multistate predictive model to estimate the future burden of CKD in Chile, given the high and rising RRT rates, population ageing, and prevalence of comorbidities contributing to CKD. METHODS: A dynamic stock and flow model was developed to simulate CKD progression in the Chilean population aged 40 years and older, up to the year 2041, adopting the perspective of the Chilean public healthcare system. The model included six states replicating progression of CKD, which was assumed in 1-year cycles and was categorised as slow, medium or fast progression, based on the underlying conditions. We simulated two different treatment scenarios. Only direct costs of treatment were included, and a 3% per year discount rate was applied after the first year. We calibrated the model based on international evidence; the exploration of uncertainty (95% credibility intervals) was undertaken with probabilistic sensitivity analysis. RESULTS: By the year 2041, there is an expected increase in cases of CKD stages 3a to ESKD, ceteris paribus, from 442,265 (95% UI 441,808-442,722) in 2021 to 735,513 (734,455-736,570) individuals. Direct costs of CKD stages 3a to ESKD would rise from 322.4M GBP (321.7-323.1) in 2021 to 1,038.6M GBP (1,035.5-1,041.8) in 2041. A reduction in the progression rates of the disease by the inclusion of SGLT2 inhibitors and pre-dialysis treatment would decrease the number of individuals worsening to stages 5 and ESKD, thus reducing the total costs of CKD by 214.6M GBP in 2041 to 824.0M GBP (822.7-825.3). CONCLUSIONS: This model can be a useful tool for healthcare planning, with development of preventive or treatment plans to reduce and delay the progression of the disease and thus the anticipated increase in the healthcare costs of CKD.

The economic costs of child maltreatment in UK.

Child maltreatment is a major public health problem with significant consequences for individual victims and for society. In this paper, we quantify for the first time the economic costs of fatal and nonfatal child maltreatment in the UK in relation to several short-, medium-, and long-term outcomes ranging from physical and mental health problems to labor market outcomes and welfare use. We combine novel regression analysis of rich data from the National Child Development Study and the English Longitudinal Study of Aging with secondary evidence to produce an incidence-based estimate of the lifetime costs of child maltreatment from a societal perspective. The discounted average lifetime incidence cost of nonfatal child maltreatment by a primary caregiver is estimated at £89,390 (95% uncertainty interval £44,896 to £145,508); the largest contributors to this are costs from social care, short-term health, and long-term labor market outcomes. The discounted lifetime cost per death from child maltreatment is estimated at £940,758, comprising health care and lost productivity costs. Our estimates provide the first comprehensive benchmark to quantify the costs of child maltreatment in the UK and the benefits of interventions aimed at reducing or preventing it.

A model-based cost-utility analysis of multi-professional simulation training in obstetric emergencies.

OBJECTIVE: To determine the cost-utility of a multi-professional simulation training programme for obstetric emergencies-Practical Obstetric Multi-Professional Training (PROMPT)-with a particular focus on its impact on permanent obstetric brachial plexus injuries (OBPIs). DESIGN: A model-based cost-utility analysis. SETTING: Maternity units in England. POPULATION: Simulated cohorts of individuals affected by permanent OBPIs. METHODS: A decision tree model was developed to estimate the cost-utility of adopting annual, PROMPT training (scenario 1a) or standalone shoulder dystocia training (scenario 1b) in all maternity units in England compared to current practice, where only a proportion of English units use the training programme (scenario 2). The time horizon was 30 years and the analysis was conducted from an English National Health Service (NHS) and Personal Social Services perspective. A probabilistic sensitivity analysis was performed to account for uncertainties in the model parameters. MAIN OUTCOME MEASURES: Outcomes for the entire simulated period included the following: total costs for PROMPT or shoulder dystocia training (including costs of OBPIs), number of OBPIs averted, number of affected adult/parental/dyadic quality adjusted life years (QALYs) gained and the incremental cost per QALY gained. RESULTS: Nationwide PROMPT or shoulder dystocia training conferred significant savings (in excess of £1 billion ($1.5 billion)) compared to current practice, resulting in cost-savings of at least £1 million ($1.5 million) per any type of QALY gained. The probabilistic sensitivity analysis demonstrated similar findings. CONCLUSION: In this model, national implementation of multi-professional simulation training for obstetric emergencies (or standalone shoulder dystocia training) in England appeared to both be cost-saving when evaluating their impact on permanent OBPIs.

Evaluating the effectiveness of the smartphone app, Drink Less, compared with the NHS alcohol advice webpage, for the reduction of alcohol consumption among hazardous and harmful adult drinkers in the UK at 6-month follow-up: protocol for a randomised controlled trial.

BACKGROUND AND AIMS: Digital interventions are effective for reducing alcohol consumption but evidence is limited regarding smartphone apps. Drink Less is a theory- and evidence-informed app to help people reduce their alcohol consumption that has been refined in terms of its content and design for usability across the sociodemographic spectrum. We aim to evaluate the effectiveness and cost-effectiveness of recommending Drink Less at reducing alcohol consumption compared with usual digital care. DESIGN: Two-arm individually randomised controlled trial. SETTING: Online trial in the United Kingdom (UK). PARTICIPANTS: Hazardous or harmful drinkers (Alcohol Use Disorders Identification Test [AUDIT] score ≥8) aged 18+ who want to drink less alcohol (n = 5562). Participants will be recruited from July 2020 to May 2022 using multiple strategies with a focus on remote digital methods. INTERVENTION AND COMPARATOR: Participants will be randomised to receive either an email recommending that they use Drink Less (intervention) or view the National Health Service (NHS) webpage on alcohol advice (comparator). MEASUREMENTS: The primary outcome is change in self-reported weekly alcohol consumption, assessed using the extended AUDIT-Consumption, between baseline and 6-month follow-up. Secondary outcomes include change in self-reported weekly alcohol consumption assessed at 1- and 3-month follow-ups, and the proportion of hazardous drinkers; alcohol-related problems and injury; health-related quality of life; and use of health services assessed at 6-month follow-up. Effectiveness will be examined with adjusted regression models, adjusting for baseline alcohol consumption and using an intention-to-treat approach. A mixed-methods process evaluation will assess engagement, acceptability and mechanism of action. Economic evaluations will be conducted using both a short- and longer-term time horizon. COMMENTS: This study will establish the effectiveness and cost-effectiveness of the Drink Less app at reducing alcohol consumption among hazardous and harmful adult drinkers and will be the first randomised controlled trial of an alcohol reduction app for the general population in the United Kingdom. This study will inform the decision on whether it is worth investing resources in large-scale implementation.

Endovascular thrombectomy in patients with large core ischemic stroke: a cost-effectiveness analysis from the SELECT study.

BACKGROUND: It is unknown whether endovascular thrombectomy (EVT) is cost effective in large ischemic core infarcts. METHODS: In the prospective, multicenter, cohort study of imaging selection study (SELECT), large core was defined as computed tomography (CT) ASPECTS<6 or computed tomography perfusion (CTP) ischemic core volume (rCBF<30%) ≥50 cc. A Markov model estimated costs, quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio (ICER) of EVT compared with medical management (MM) over lifetime. The willingness to pay (WTP) per QALY was set at $50 000 and $100 000 and the net monetary benefits (NMB) were calculated. Probabilistic sensitivity analysis (PSA) and cost-effectiveness acceptability curves (CEAC) for EVT were assessed in SELECT and other pivotal trials. RESULTS: From 361 patients enrolled in SELECT, 105 had large core on CT or CTP (EVT 62, MM 43). 19 (31%) EVT vs 6 (14%) MM patients achieved modified Rankin Scale (mRS) score 0-2 (OR 3.27, 95% CI 1.11 to 9.62, P=0.03) with a shift towards better mRS (cOR 2.12, 95% CI 1.05 to 4.31, P=0.04). Over the projected lifetime of patients presenting with large core, EVT led to incremental costs of $33 094 and a gain of 1.34 QALYs per patient, resulting in ICER of $24 665 per QALY. EVT has a higher NMB compared with MM at lower (EVT -$42 747, MM -$76 740) and upper (EVT $155 041, MM $57 134) WTP thresholds. PSA confirmed the results and CEAC showed 77% and 92% acceptability of EVT at the WTP of $50 000 and $100 000, respectively. EVT was associated with an increment of $29 225 in societal costs. The pivotal EVT trials (HERMES, DAWN, DEFUSE 3) were dominant in a sensitivity analysis at the same inputs, with societal cost-savings of $37 901, $86 164 and $22 501 and a gain of 1.62, 2.36 and 2.21 QALYs, respectively. CONCLUSIONS: In a non-randomized prospective cohort study, EVT resulted in better outcomes in large core patients with higher QALYs, NMB and high cost-effectiveness acceptability rates at current WTP thresholds. Randomized trials are needed to confirm these results. CLINICAL TRIAL REGISTRATION: NCT02446587.

Cytoreductive surgery (CRS) with hyperthermic intraoperative peritoneal chemotherapy (HIPEC) versus standard of care (SoC) in people with peritoneal metastases from colorectal, ovarian or gastric origin: protocol for a systematic review and individual participant data (IPD) meta-analyses of effectiveness and cost-effectiveness.

INTRODUCTION: There is uncertainty about whether cytoreductive surgery (CRS)+hyperthermic intraoperative peritoneal chemotherapy (HIPEC) improves survival and/or quality of life compared with standard of care (SoC) in people with peritoneal metastases who can withstand major surgery. PRIMARY OBJECTIVES: To compare the relative benefits and harms of CRS+HIPEC versus SoC in people with peritoneal metastases from colorectal, ovarian or gastric cancers eligible to undergo CRS+HIPEC by a systematic review and individual participant data (IPD) meta-analysis. SECONDARY OBJECTIVES: To compare the cost-effectiveness of CRS+HIPEC versus SoC from a National Health Service (NHS) and personal social services perspective using a model-based cost-utility analysis. METHODS AND ANALYSIS: We will perform a systematic review of literature by updating the searches from MEDLINE, Embase, Cochrane library, Science Citation Index as well as trial registers. Two members of our team will independently screen the search results and identify randomised controlled trials comparing CRS+HIPEC versus SoC for inclusion based on full texts for articles shortlisted during screening. We will assess the risk of bias in the trials and obtain data related to baseline prognostic characteristics, details of intervention and control, and outcome data related to overall survival, disease progression, health-related quality of life, treatment related complications and resource utilisation data. Using IPD, we will perform a two-step IPD, that is, calculate the adjusted effect estimate from each included study and then perform a random-effects model meta-analysis. We will perform various subgroup analyses, meta-regression and sensitivity analyses. We will also perform a model-based cost-utility analysis to assess whether CRS+HIPEC is cost-effective in the NHS setting. ETHICS AND DISSEMINATION: This project was approved by the UCL Research Ethics Committee (Ethics number: 16023/001). We aim to present the findings at appropriate international meetings and publish the review, irrespective of the findings, in a peer-reviewed journal. PROSPERO REGISTRATION NUMBER: CRD42019130504.

Centralising acute stroke care within clinical practice in the Netherlands: lower bounds of the causal impact.

BACKGROUND: Authors in previous studies demonstrated that centralising acute stroke care is associated with an increased chance of timely Intra-Venous Thrombolysis (IVT) and lower costs compared to care at community hospitals. In this study we estimated the lower bound of the causal impact of centralising IVT on health and cost outcomes within clinical practice in the Northern Netherlands. METHODS: We used observational data from 267 and 780 patients in a centralised and decentralised system, respectively. The original dataset was linked to the hospital information systems. Literature on healthcare costs and Quality of Life (QoL) values up to 3 months post-stroke was searched to complete the input. We used Synthetic Control Methods (SCM) to counter selection bias. Differences in SCM outcomes included 95% Confidence Intervals (CI). To deal with unobserved heterogeneity we focused on recently developed methods to obtain the lower bounds of the causal impact. RESULTS: Using SCM to assess centralising acute stroke 3 months post-stroke revealed healthcare savings of $US 1735 (CI, 505 to 2966) while gaining 0.03 (CI, - 0.01 to 0.73) QoL per patient. The corresponding lower bounds of the causal impact are $US 1581 and 0.01. The dominant effect remained stable in the deterministic sensitivity analyses with $US 1360 (CI, 476 to 2244) as the most conservative estimate. CONCLUSIONS: In this study we showed that a centralised system for acute stroke care appeared both cost-saving and yielded better health outcomes. The results are highly relevant for policy makers, as this is the first study to address the issues of selection and unobserved heterogeneity in the evaluation of centralising acute stroke care, hence presenting causal estimates for budget decisions.

Cost-utility analysis of mechanical thrombectomy between 6 and 24 hours in acute ischemic stroke.

BACKGROUND: Recently, two randomized controlled trials demonstrated the benefit of mechanical thrombectomy performed between 6 and 24 h in acute ischemic stroke. The current economic evidence is supporting the intervention only within 6 h, but extended thrombectomy treatment times may result in better long-term outcomes for a larger cohort of patients. AIMS: We compared the cost-utility of mechanical thrombectomy in addition to medical treatment versus medical treatment alone performed beyond 6 h from stroke onset in the UK National Health Service (NHS). METHODS: A cost-utility analysis of mechanical thrombectomy compared to medical treatment was performed using a Markov model that estimates expected costs and quality-adjusted life years (QALYs) over a 20-year time horizon. We present the results of three models using the data from the DEFUSE 3 and DAWN trials and evidence from published sources. RESULTS: Over a 20-year period, the incremental cost per QALY of mechanical thrombectomy was $1564 (£1219) when performed after 12 h from onset, $5253 (£4096) after 16 h and $3712 (£2894) after 24 h. The probabilistic sensitivity analysis demonstrated that thrombectomy had a 99.9% probability of being cost-effective at the minimum willingness to pay for a QALY commonly used in the UK. CONCLUSIONS: The results of this study demonstrate that performing mechanical thrombectomy up to 24 h from acute ischemic stroke symptom onset is still cost-effective, suggesting that this intervention should be implemented by the NHS on the basis of improvement in quality of life as well as economic grounds.

Cost-effectiveness of a melanoma screening programme using whole disease modelling.

OBJECTIVE: To assess the potential impact of a melanoma screening programme, compared with usual care, on direct costs and life expectancy in the era of targeted drugs and cancer immunotherapy. METHODS: Using a Whole Disease Model approach, a Markov simulation model with a time horizon of 25 years was devised to analyse the cost-effectiveness of a one-time, general practitioner-based melanoma screening strategy in the population aged over 20, compared with no screening. The study considered the most up-to-date drug therapy and was conducted from the perspective of the Veneto regional healthcare system within the Italian National Health Service. Only direct costs were considered. Sensitivity analyses, both one-way and probabilistic, were performed to identify the parameters with the greatest impact on cost-effectiveness, and to assess the robustness of our model. RESULTS: Over a 25-year time horizon, the screening intervention dominated usual care. The probabilistic sensitivity analyses confirmed the robustness of these findings. The key drivers of the model were the proportion of melanomas detected by the screening procedure and the adherence of the target population to the screening programme. CONCLUSIONS: The screening programme proved to be a dominant option compared with usual care. These findings should prompt serious consideration of the design and implementation of a regional or national melanoma screening strategy within a National Health Service.

Cost-comparison analysis of FIB-4, ELF and fibroscan in community pathways for non-alcoholic fatty liver disease.

BACKGROUND: The identification of patients with advanced liver fibrosis secondary to non-alcoholic fatty liver disease (NAFLD) remains challenging. Using non-invasive liver fibrosis tests (NILT) in primary care may permit earlier detection of patients with clinically significant disease for specialist review, and reduce unnecessary referral of patients with mild disease. We constructed an analytical model to assess the clinical and cost differentials of such strategies. METHODS: A probabilistic decisional model simulated a cohort of 1000 NAFLD patients over 1 year from a healthcare payer perspective. Simulations compared standard care (SC) (scenario 1) to: Scenario 2: FIB-4 for all patients followed by Enhanced Liver Fibrosis (ELF) test for patients with indeterminate FIB-4 results; Scenario 3: FIB-4 followed by fibroscan for indeterminate FIB-4; Scenario 4: ELF alone; and Scenario 5: fibroscan alone. Model estimates were derived from the published literature. The primary outcome was cost per case of advanced fibrosis detected. RESULTS: Introduction of NILT increased detection of advanced fibrosis over 1 year by 114, 118, 129 and 137% compared to SC in scenarios 2, 3, 4 and 5 respectively with reduction in unnecessary referrals by 85, 78, 71 and 42% respectively. The cost per case of advanced fibrosis (METAVIR ≥F3) detected was £25,543, £8932, £9083, £9487 and £10,351 in scenarios 1, 2, 3, 4 and 5 respectively. Total budget spend was reduced by 25.2, 22.7, 15.1 and 4.0% in Scenarios 2, 3, 4 and 5 compared to £670 K at baseline. CONCLUSION: Our analyses suggest that the use of NILT in primary care can increases early detection of advanced liver fibrosis and reduce unnecessary referral of patients with mild disease and is cost efficient. Adopting a two-tier approach improves resource utilization.

Impact and experiences of delayed discharge: A mixed-studies systematic review.

BACKGROUND: The impact of delayed discharge on patients, health-care staff and hospital costs has been incompletely characterized. AIM: To systematically review experiences of delay from the perspectives of patients, health professionals and hospitals, and its impact on patients' outcomes and costs. METHODS: Four of the main biomedical databases were searched for the period 2000-2016 (February). Quantitative, qualitative and health economic studies conducted in OECD countries were included. RESULTS: Thirty-seven papers reporting data on 35 studies were identified: 10 quantitative, 8 qualitative and 19 exploring costs. Seven of ten quantitative studies were at moderate/low methodological quality; 6 qualitative studies were deemed reliable; and the 19 studies on costs were of moderate quality. Delayed discharge was associated with mortality, infections, depression, reductions in patients' mobility and their daily activities. The qualitative studies highlighted the pressure to reduce discharge delays on staff stress and interprofessional relationships, with implications for patient care and well-being. Extra bed-days could account for up to 30.7% of total costs and cause cancellations of elective operations, treatment delay and repercussions for subsequent services, especially for elderly patients. CONCLUSIONS: The poor quality of the majority of the research means that implications for practice should be cautiously made. However, the results suggest that the adverse effects of delayed discharge are both direct (through increased opportunities for patients to acquire avoidable ill health) and indirect, secondary to the pressures placed on staff. These findings provide impetus to take a more holistic perspective to addressing delayed discharge.

The Age of BLood Evaluation (ABLE) randomised controlled trial: description of the UK-funded arm of the international trial, the UK cost-utility analysis and secondary analyses exploring factors associated with health-related quality of life and health-care costs during the 12-month follow-up.

BACKGROUND: At present, red blood cells (RBCs) are stored for up to 42 days prior to transfusion. The relative effectiveness and safety of different RBC storage times prior to transfusion is uncertain. OBJECTIVE: To assess the clinical effectiveness and cost-effectiveness of transfusing fresher RBCs (stored for ≤ 7 days) compared with current standard-aged RBCs in critically ill patients requiring blood transfusions. DESIGN: The international Age of BLood Evaluation (ABLE) trial was a multicentre, randomised, blinded trial undertaken in Canada, the UK, the Netherlands and France. The UK trial was funded to contribute patients to the international trial and undertake a UK-specific health economic evaluation. SETTING: Twenty intensive care units (ICUs) in the UK, as part of 64 international centres. PARTICIPANTS: Critically ill patients aged ≥ 18 years (≥ 16 years in Scotland) expected to require mechanical ventilation for ≥ 48 hours and requiring a first RBC transfusion during the first 7 days in the ICU. INTERVENTIONS: All decisions to transfuse RBCs were made by clinicians. One patient group received exclusively fresh RBCs stored for ≤ 7 days whenever transfusion was required from randomisation until hospital discharge. The other group received standard-issue RBCs throughout their hospital stay. MAIN OUTCOME MEASURES: The primary outcome was 90-day mortality. Secondary outcomes included development of organ dysfunction, new thrombosis, infections and transfusion reactions. The primary economic evaluation was a cost-utility analysis. RESULTS: The international trial took place between March 2009 and October 2014 (UK recruitment took place between January 2012 and October 2014). In total, 1211 patients were assigned to receive fresh blood and 1219 patients to receive standard-aged blood. RBCs were stored for a mean of 6.1 days [standard deviation (SD) ± 4.9 days] in the group allocated to receive fresh blood and 22.0 days (SD ± 8.4 days) in the group allocated to receive standard-aged blood. Patients received a mean of 4.3 RBC units (SD ± 5.2 RBC units) and 4.3 RBC units (SD ± 5.5 RBC units) in the groups receiving fresh blood and standard-aged blood, respectively. At 90 days, 37.0% of patients in the group allocated to receive fresh blood and 35.3% of patients in the group allocated to receive standard-aged blood had died {absolute risk difference 1.7% [95% confidence interval (CI) -2.1% to 5.5%]}. There were no between-group differences in any secondary outcomes. The UK cohort comprised 359 patients randomised and followed up for 12 months for the cost-utility analysis. UK patients had similar characteristics and outcomes to the international cohort. Mean total costs per patient were £32,346 (95% CI £29,306 to £35,385) in the group allocated to receive fresh blood and £33,353 (95% CI £29,729 to £36,978) in the group allocated to receive standard-aged blood. Approximately 85% of the total costs were incurred during the index hospital admission. There were no significant cost differences between the two groups [mean incremental costs for those receiving fresh vs. standard-aged blood: -£231 (95% CI -£4876 to £4415)], nor were there significant differences in outcomes (mean difference in quality-adjusted life-years -0.010, 95% CI -0.078 to 0.057). LIMITATIONS: Adverse effects from the exclusive use of older RBCs compared with standard or fresh RBCs cannot be excluded. CONCLUSIONS: The use of RBCs aged ≤ 7 days confers no clinical or economic benefit in critically ill patients compared with standard-aged RBCs. FUTURE WORK: Future studies should address the safety of RBCs near the end of the current permitted storage age. TRIAL REGISTRATION: Current Controlled Trials ISRCTN44878718. FUNDING: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 21, No. 62. See the NIHR Journals Library website for further project information. The international ABLE trial was also supported by peer-reviewed grants from the Canadian Institutes of Health Research (177453), Fonds de Recherche du Québec - Santé (24460), the French Ministry of Health Programme Hospitalier de Recherche Clinique (12.07, 2011) and by funding from Établissement Français du Sang and Sanquin Blood Supply.

Does use of point-of-care testing improve cost-effectiveness of the NHS Health Check programme in the primary care setting? A cost-minimisation analysis.

OBJECTIVE: To determine if use of point of care testing (POCT) is less costly than laboratory testing to the National Health Service (NHS) in delivering the NHS Health Check (NHSHC) programme in the primary care setting. DESIGN: Observational study and theoretical mathematical model with microcosting approach. SETTING: We collected data on NHSHC delivered at nine general practices (seven using POCT; two not using POCT). PARTICIPANTS: We recruited nine general practices offering NHSHC and a pathology services laboratory in the same area. METHODS: We conducted mathematical modelling with permutations in the following fields: provider type (healthcare assistant or nurse), type of test performed (total cholesterol with either lab fasting glucose or HbA1c), cost of consumables and variable uptake rates, including rate of non-response to invite letter and rate of missed [did not attend (DNA)] appointments. We calculated total expected cost (TEC) per 100 invites, number of NHSHC conducted per 100 invites and costs for completed NHSHC for laboratory and POCT-based pathways. A univariate and probabilistic sensitivity analysis was conducted to account for uncertainty in the input parameters. MAIN OUTCOME MEASURES: We collected data on cost, volume and type of pathology services performed at seven general practices using POCT and a pathology services laboratory. We collected data on response to the NHSHC invitation letter and DNA rates from two general practices. RESULTS: TEC of using POCT to deliver a routine NHSHC is lower than the laboratory-led pathway with savings of £29 per 100 invited patients up the point of cardiovascular disease risk score presentation. Use of POCT can deliver NHSHC in one sitting, whereas the laboratory pathway offers patients several opportunities to DNA appointment. CONCLUSIONS: TEC of using POCT to deliver an NHSHC in the primary care setting is lower than the laboratory-led pathway. Using POCT minimises DNA rates associated with laboratory testing and enables completion of NHSHC in one sitting.