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This paper studies the effects of immigration on the allocation of occupational physical burden and work injury risks. Using data for England and Wales from the Labour Force Survey (2003–2013), we find that, on average, immigration leads to a reallocation of UK-born workers towards jobs characterised by lower physical burden and injury risk. The results also show important differences across skill groups. Immigration reduces the average physical burden of UK-born workers with medium levels of education, but has no significant effect on those with low levels. We also find that that immigration led to an improvement self-reported measures of native workers' health. These findings, together with the evidence that immigrants report lower injury rates than natives, suggest that the reallocation of tasks could reduce overall health care costs and the human and financial costs typically associated with workplace injuries. [ABSTRACT FROM AUTHOR]

Background: Hyperkalemia is an electrolyte abnormality with potentially life-threatening consequences. Published data have shown that potassium-binding polymer patiromer (Veltassa) is associated with reduced rates of severe edema and hospitalization for heart failure compared with sodium zirconium cyclosilicate (SZC, Lokelma) when treating hyperkalemia. The aim of this study was to evaluate the possible costs associated with these interventions in the Spanish and UK settings., Methods: A cost-analysis model was developed in Microsoft Excel to compare the costs associated with patiromer and SZC for the management of hyperkalemia. Clinical event rates were taken from a published real-world comparative study, with the base case capturing the statistically significant reduction in severe edema with patiromer vs SZC and a sensitivity analysis also including the non-statistically significant reduction in hospitalization for heart failure. Country-specific costs, expressed in 2022 Euros (EUR) and British pounds sterling (GBP), were evaluated from a healthcare payer perspective and included pharmacy costs and costs of clinical events., Results: Patiromer may be associated with cost savings of EUR 107 and GBP 630 per patient-year of treatment vs SZC in Spain and the UK, respectively. The majority of cost savings were due to the possible lower daily cost of patiromer compared with SZC. Including the difference in heart failure hospitalization rates in a sensitivity analysis led to greater cost savings with patiromer over SZC, increasing to EUR 460 and GBP 902 in Spain and the UK, respectively. Extrapolation of patient-level economic outcomes to a population level found that patiromer was associated with annual cost savings of EUR 30.6 million in Spain, and GBP 801.7 million in the UK vs SZC., Conclusions: Patiromer has the potential to be cost saving vs SZC for the treatment of hyperkalemia in Spain and the UK based on the results of a real-world evidence analysis.

Aims: Migraine is the most common disabling headache disorder and is characterized by recurrent throbbing head pain and symptoms of photophobia, phonophobia, nausea, and vomiting. Rimegepant 75 mg, an oral lyophilisate calcitonin gene-related peptide antagonist, is the first treatment approved for both the acute and preventative treatment of migraine, and the first acute therapy approved in over 20-years. The objective was to assess the cost-utility of rimegepant compared with best supportive care (BSC) in the UK, for the acute treatment of migraine in the adults with inadequate symptom relief after taking at least 2 triptans, or for whom triptans are contraindicated or not tolerated., Materials and Methods: A de novo model was developed to estimate incremental costs and quality-adjusted life years (QALYs), structured as a decision tree followed by Markov model. Patients received rimegepant or BSC for a migraine attack and were assessed for response (pain relief at 2-h). Responders and non-responders followed different pain trajectories over 48-h cycles. Non-responders discontinued treatment while responders continued treatment for subsequent attacks, with a proportion discontinuing over time. Data sources included a post-hoc pooled analysis of the phase 3 acute rimegepant trials (NCT03235479, NCT03237845, NCT03461757), and a long-term safety study (NCT03266588). The analysis was conducted from the perspective of the UK National Health Service and Personal Social Services over a 20-year time horizon., Results: Rimegepant resulted in an incremental cost-utility ratio (ICUR) of £10,309 per QALY gained vs BSC, which is cost-effectiveness at a willingness to pay threshold of £30,000/QALY. Rimegepant generated +0.44 incremental QALYs and higher incremental lifetime costs (£4,492). Improved QALYs for rimegepant were a result of less time spent with severe and moderate headache pain., Conclusion: This study highlights the economic value of rimegepant which was found to be cost-effective for the acute treatment of migraine in adults unsuitable for triptans.

Aim: To evaluate the real-world comparative effectiveness and the cost-effectiveness, from a UK National Health Service perspective, of natalizumab versus fingolimod in patients with rapidly evolving severe relapsing-remitting multiple sclerosis (RES-RRMS)., Methods: Real-world data from the MSBase Registry were obtained for patients with RES-RRMS who were previously either naive to disease-modifying therapies or had been treated with interferon-based therapies, glatiramer acetate, dimethyl fumarate, or teriflunomide (collectively known as BRACETD). Matched cohorts were selected by 3-way multinomial propensity score matching, and the annualized relapse rate (ARR) and 6-month-confirmed disability worsening (CDW6M) and improvement (CDI6M) were compared between treatment groups. Comparative effectiveness results were used in a cost-effectiveness model comparing natalizumab and fingolimod, using an established Markov structure over a lifetime horizon with health states based on the Expanded Disability Status Scale. Additional model data sources included the UK MS Survey 2015, published literature, and publicly available sources., Results: In the comparative effectiveness analysis, we found a significantly lower ARR for patients starting natalizumab compared with fingolimod (rate ratio [RR] = 0.65; 95% confidence interval [CI], 0.57-0.73) or BRACETD (RR = 0.46; 95% CI, 0.42-0.53). Similarly, CDI6M was higher for patients starting natalizumab compared with fingolimod (hazard ratio [HR] = 1.25; 95% CI, 1.01-1.55) and BRACETD (HR = 1.46; 95% CI, 1.16-1.85). In patients starting fingolimod, we found a lower ARR (RR = 0.72; 95% CI, 0.65-0.80) compared with starting BRACETD, but no difference in CDI6M (HR = 1.17; 95% CI, 0.91-1.50). Differences in CDW6M were not found between the treatment groups. In the base-case cost-effectiveness analysis, natalizumab dominated fingolimod (0.302 higher quality-adjusted life-years [QALYs] and £17,141 lower predicted lifetime costs). Similar cost-effectiveness results were observed across sensitivity analyses., Conclusions: This MSBase Registry analysis suggests that natalizumab improves clinical outcomes when compared with fingolimod, which translates to higher QALYs and lower costs in UK patients with RES-RRMS.

UK households have been exposed to economic recession followed by a government programme of austerity, putting many under severe financial strain. Using UK longitudinal household data, we find that the feeling of not being able to cope financially matters for individual mental health and general health status even when controlling for individual heterogeneity and potential reverse causation. We develop a theoretical model which brings some of the rigour of lifetime economic decisionmaking models to bear on our understanding of the causes of financial strain. Our estimation results for this model highlight that shocks to how we view our financial situation are more important for subjective financial well-being than not having enough income or being liquidity constrained. Recent welfare and pension reforms intended to reduce budget deficits may have exacerbated financial strain and thus increased public healthcare costs. In the case of disability benefits reform, we find that the uncertainty generated by an opaque process of reassessment caused financial strain to increase even when households were not materially worse off. [ABSTRACT FROM AUTHOR]

This study investigates how maternal working hours are related to various outcomes in children aged 11-15 using a sample of mothers and adolescents in the British Household Panel Survey. Research that examines the effects of maternal employment on children has been motivated by the rapid increase of female participation rates in the labour market and increased shares of children living in female-headed or single-mother households. The existing literature on this issue is very limited, mostly based on American data, and provides conflicting results. Fixed effects have been used in the present analysis to control for characteristics of children and mothers that do not vary over time. The results suggest that full-time maternal employment (as opposed to part-time) has little or no effect on the propensity of adolescents to smoke, their life satisfaction, self-esteem, or intention to leave school at 16. These results are stable and consistent across various specifications of the model and different socio-economic status. [ABSTRACT FROM AUTHOR]

We use a standard of living ( SoL) approach to estimate older people's disability costs, using data on 8000 individuals from the U. K. Family Resources Survey. We extend previous research in two ways. First, by allowing for a more flexible relationship between SoL and income, the structure of the estimated disability cost and equivalence scale is not dictated by a restrictive functional form assumption. Second, we allow for the latent nature of disability and SoL, addressing measurement error in the disability and SoL indicators in surveys. We find that disability costs are strongly related to severity of disability, and vary with income in absolute and proportionate terms. Older people above the median disability level require an extra £99 per week (2007 prices) on average to reach the SoL of an otherwise similar person at the median. Costs faced by older people in the highest decile of disability average £180. [ABSTRACT FROM AUTHOR]

We exploit migration patterns from the UK to Australia and the US to investigate whether a person's decision to smoke is determined by culture. For each country, we use retrospective data to describe individual smoking trajectories over the life-course. For the UK, we use these trajectories to measure culture by cohort and cohort-age, and more accurately relative to the extant literature. Our proxy predicts smoking participation of second-generation British immigrants but not that of non-British immigrants and natives. Researchers can apply our strategy to estimate culture effects on other outcomes when retrospective or longitudinal data are available. [ABSTRACT FROM AUTHOR]

Aim: In a randomized control trial mid-regional proadrenomedullin (MR-proADM)-guided decision-making has been proven to safely reduce hospital admissions based on an accurate assessment of disease severity in the Emergency Department (ED). This study aimed to assess the impact of a MR-proADM-Guided Triage (MR-GT) versus standard Hospital Triage (HT) on clinical and economic outcomes in ED patients with suspected infection in Spain, Italy, Germany, and the UK, using Patient-Level Data (PLD) from two observational studies., Methods: PLD was collected from patients enrolled at a Spanish hospital during two observational studies. Logistic regression was used to identify predictors of hospitalization. A patient-level simulation model was subsequently developed to evaluate the clinical and economic impact of MR-GT versus HT using results from the statistical analysis and country-specific cost data from the published literature. Probabilistic and deterministic sensitivity analyses were carried out., Results: Four hundred seventy-three patients were enrolled in this study. MR-proADM had the strongest association with hospital admission, followed by age and National Early Warning Score (NEWS). In the simulation model, MR-GT was associated with an overall reduction in hospitalization relative to HT, equal to 22.6 percentage points (40.9 vs. 63.5%). In addition, the use of MR-GT would reduce the total hospital cost per patient presenting to the ED with suspected infection by roughly 30%, with a mean cost saving per patient of €626, €1,484, €1,154, and £1,113 in Spain, Italy, Germany, and the UK, respectively. The robustness of these findings was confirmed by sensitivity analyses., Limitations: The statistical analyses were not performed on the same population simulated in the model. Clinical input parameters were assumed to be the same for all countries., Conclusions: MR-proADM showed to be the main predictor of hospitalization. An MR-proADM decision algorithm provides cost savings in Spain, Italy, Germany, and the UK.

Aim: Screening for non-valvular atrial fibrillation (NVAF) is key in identifying patients with undiagnosed disease who may be eligible for anticoagulation therapy. Understanding the economic value of screening is necessary to assess optimal strategies for payers and healthcare systems. We evaluated the cost effectiveness of opportunistic screening with handheld digital devices and pulse palpation, as well as targeted screening predictive algorithms for UK patients ≥75 years of age., Methods: A previously developed Markov cohort model was adapted to evaluate clinical and economic outcomes of opportunistic screening including pulse palpation, Zenicor (extended 14 days), KardiaMobile (extended), and two algorithms compared to no screening. Key model inputs including epidemiology estimates, screening effectiveness, and risks for medical events were derived from the STROKESTOP, ARISTOTLE studies, and published literature, and cost inputs were obtained from a UK national cost database. Health and cost outcomes, annually discounted at 3.5%, were reported for a cohort of 10,000 patients vs. no screening over a time horizon equivalent to a patient's lifetime, Analyses were performed from a UK National Health Services and personal social services perspective., Results: Zenicor, pulse palpation, and KardiaMobile were dominant (providing better health outcomes at lower costs) vs. no screening; both algorithms were cost-effective vs. no screening, with incremental cost-effectiveness ratios per quality-adjusted life-year (QALY) of £1,040 and £1,166. Zenicor, pulse palpation, and KardiaMobile remained dominant options vs. no screening in all scenarios explored. Deterministic sensitivity analyses indicated long-term stroke care costs, prevalence of undiagnosed NVAF in patients 75-79 years of age, and clinical efficacy of anticoagulant on stroke prevention were the main drivers of the cost-effectiveness results., Conclusions: Screening for NVAF at ≥75 years of age could result in fewer NVAF-related strokes. NVAF screening is cost-effective and may be cost-saving depending on the program chosen.

Abstract: Increased health care spending has placed pressure on public and private payers to prioritize spending. Cost-effectiveness (CE) analysis is the main tool used by payers to prioritize coverage of new therapies. We argue that reimbursement based on CE is subject to a form of the “Lucas critique”; the goals of CE policies may not materialize when firms affected by the policies respond optimally to them. For instance, because ‘costs’ in CE analysis reflect prices set optimally by firms rather than production costs, observed CE levels will depend on how firm pricing responds to CE policies. Observed CE is therefore endogenous. When CE is endogenously determined, policies aimed at lowering spending and improving overall CE may paradoxically raise spending and lead to the adoption of more resource-costly treatments. We empirically illustrate whether this may occur using data on public coverage decisions in the United Kingdom. [Copyright &y& Elsevier]

This paper estimates the financial cost of skin cancer in England. National Health Service (NHS) costs were calculated by combining published data on health service use by patients with skin cancer with published data on the unit cost of services. Indirect costs arising from individuals’ inability to function in their usual role as a result of skin cancer were estimated from incapacity benefit claims and numbers of registered deaths due to skin cancer. The total costs of skin cancer were around £240 million. Costs to the NHS represented 42% of the total. [ABSTRACT FROM AUTHOR]

Abstract: The hypothesis that enhanced nutrition is mainly responsible for massive IQ gains over time borrows plausibility from the height gains of the 20th century. However, evidence shows that the two trends are largely independent. A detailed analysis of IQ trends on the Raven''s Progressive Matrices tests in Britain dramatizes the poverty of the nutrition hypothesis. A multiple factor hypothesis that operates on three levels is offered as an alternative instrument of causal explanation. The Raven''s data show that over the 65 years from circa 1942 to the present, taking ages 5–15 together, British school children have gained 14 IQ points for a rate of 0.216 points per year. However, since 1979, gains have declined with age and between the ages of 12–13 and 14–15, small gains turn into small losses. This is confirmed by Piagetian data and poses the possibility that the cognitive demands of teen-age subculture have been stagnant over perhaps the last 30 years. [Copyright &y& Elsevier]

In England and Wales, the quantity and quality of renal services have improved significantly in the last decade. While acceptance rates for renal replacement therapy appear low by international standards, they are now commensurate with many other northern European countries. The major growth in renal services has been in hemodialysis, especially at satellite units. Health care is predominantly publicly funded through a tax-based National Health Service, and such funding has increased in the last 10 years. Improvements in health outcomes in England and Wales are expected to continue due to the recent implementation of standards, initiatives, and monitoring mechanisms for renal transplantation, vascular access, and patient transport. [ABSTRACT FROM AUTHOR]

Aim: To evaluate the public health impact of the UK COVID-19 booster vaccination program in autumn 2021, during a period of SARS-CoV-2 Delta variant predominance., Materials and Methods: A compartmental Susceptible-Exposed-Infectious-Recovered model was used to compare age-stratified health outcomes for adult booster vaccination versus no booster vaccination in the UK over a time horizon of September-December 2021, when boosters were introduced in the UK and the SARS-CoV-2 Delta variant was predominant. Model input data were sourced from targeted literature reviews and publicly available data. Outcomes were predicted COVID-19 cases, hospitalizations, post-acute sequelae of COVID-19 (PASC) cases, deaths, and productivity losses averted, and predicted healthcare resources saved. Scenario analyses varied booster coverage, virus infectivity and severity, and time horizon parameters., Results: Booster vaccination was estimated to have averted approximately 547,000 COVID-19 cases, 36,000 hospitalizations, 147,000 PASC cases, and 4,200 deaths in the UK between September and December 2021. It saved over 316,000 hospital bed-days and prevented the loss of approximately 16.5 million paid and unpaid patient work days. In a scenario of accelerated uptake, the booster rollout would have averted approximately 3,400 additional deaths and 25,500 additional hospitalizations versus the base case. A scenario analysis assuming four-fold greater virus infectivity and lower severity estimated that booster vaccination would have averted over 105,000 deaths and over 41,000 hospitalizations versus the base case. A scenario analysis assuming pediatric primary series vaccination prior to adult booster vaccination estimated that expanding vaccination to children aged ≥5 years would have averted approximately 51,000 additional hospitalizations and 5,400 additional deaths relative to adult booster vaccination only., Limitations: The model did not include the wider economic burden of COVID-19, hospital capacity constraints, booster implementation costs, or non-pharmaceutical interventions., Conclusions: Booster vaccination during Delta variant predominance reduced the health burden of SARS-CoV-2 in the UK, releasing substantial NHS capacity.

Aims: This study compared the psychometric properties of EQ-5D-5L and SF-6D to assess the interchangeability of both instruments in patients with a recent fracture presenting at a Fracture Liaison Service (FLS)., Materials and Methods: Data from a prospective observational study in a Dutch FLS clinic were used. Over 3 years, subjects were interviewed at several time points using EQ-5D-5L and SF-36. Floor and ceiling effects were evaluated. Agreement was evaluated by intra-class correlation coefficients and visualized in Bland-Altman plots. Spearman's rank correlation coefficients were applied to assess convergent validity. Mann-Whitney U test or Kruskal-Wallis H test as well as effect size (ES) were used to explore known-groups validity. Responsiveness was explored using standardized response mean (SRM) and ES. For each measurement property, hypotheses on direction and magnitude of effects were formulated., Results: A total of 499 patients were included. EQ-5D-5L had a considerable ceiling effect in comparison to SF-6D (21 vs. 1.2%). Moderate agreement between the (UK and Dutch) EQ-5D-5L and SF-6D was identified with intra-class correlation coefficients of 0.625 and 0.654, respectively. Bland-Altman plots revealed proportional bias as the differences in utilities between two instruments were highly dependent on the health states. High correlation between instruments was found (UK: rho = 0.758; Dutch: rho = 0.763). EQ-5D-5L and SF-6D utilities showed high correlation with physical component score but low correlation with mental component score of SF-36. Both instruments showed moderate discrimination (ES > 0.5) for subgroup by baseline fracture type, and moderate responsiveness (SRM > 0.5) in patients that sustained a subsequent fracture., Conclusion: Both EQ-5D-5L and SF-6D appeared to be valid utility instruments in patients with fractures attending the FLS. However, they cannot be used interchangeably given only moderate agreement was identified, and differences in utilities and ceiling effect were revealed. Comparable construct validity and responsiveness were indicated, and neither instrument was found to be clearly superior.

Aims: To assess the cost-effectiveness of carbetocin versus oxytocin for the prevention of postpartum hemorrhage (PPH) following vaginal birth from the perspective of the UK National Health Service (NHS)., Materials and Methods: A decision tree model was designed to analyze the cost per PPH event avoided associated with utilizing carbetocin versus oxytocin for prophylactic treatment of PPH in women following vaginal birth from a UK perspective. It modelled the potential for women to require an additional uterotonic after prophylaxis, and to still experience a PPH event and receive associated treatment. Inpatient recovery and follow-up periods post-PPH were also included in the model. Costs associated with drug acquisition and administration, PPH management (i.e. additional staffing and possible operating theater and high dependency unit utilization), inpatient hospitalization, and follow-up visits were all considered. Adverse event management costs were not included. Resource utilization varied depending on the severity of the PPH event (as defined by the amount of blood lost). PPH events avoided were estimated. In an exploratory analysis, quality adjusted life years (QALYs) were estimated as well., Results: In the deterministic base case, costs were £55 lower and PPH events were 0.0342 lower per woman with carbetocin use compared to oxytocin use. Across the cohort of 100 women the reduction in PPH events led to the largest cost savings (£4,233 saved) out of all cost categories, with total cost savings of £5,495. Carbetocin utilization amongst the entire cohort led to 3.42 avoided PPH events compared to oxytocin utilization, comprised of 3.03 fewer mild/moderate PPH events and 0.39 fewer severe PPH events. Carbetocin utilization led to 0.0001 additional QALYs per woman., Conclusion: Carbetocin utilization leads to lower prophylactic treatment costs and less PPH events versus oxytocin when utilized for the prevention of PPH following vaginal birth in the UK.

Aim: Atopic dermatitis (AD) is a chronic inflammatory skin disorder characterized by severe itching, erythema and scaling, causing pain, stigmatization and social isolation. Despite the growing availability of treatment options, unmet care needs remain. This research aimed to assess the cost-effectiveness of a novel JAK inhibitor (JAKi) compared to a monoclonal antibody and to identify key drivers of cost-effectiveness., Materials and Methods: A de novo economic model was developed to assess the cost-effectiveness of a novel JAKi compared to an established monoclonal antibody for the treatment of moderate-to-severe AD patients from a UK perspective. A targeted literature review was conducted to inform the development of the economic model with an advanced model structure. Various scenario and sensitivity analyses were performed to account for parameter- and structural uncertainty and to identify key drivers of cost-effectiveness., Results: The JAKi was not cost-effective compared to the monoclonal antibody (£219,733.88 per quality-adjusted life year (QALY) gained) at selected price levels when applying the UK willingness-to-pay threshold of £30,000 per QALY gained. Key drivers of cost-effectiveness were utility values, intervention efficacy and drug acquisition costs. A decrease in JAKi's dose costs, as well as a lower dose, lead to cost-effectiveness., Limitations: Assumptions regarding parameter inputs were necessary, therefore a considerable level of uncertainty regarding efficacy and cost data is to be accounted for in the interpretation of the results. In particular, the efficacy data were based on single clinical studies., Conclusions: This research revealed the cost-effectiveness of a JAKi compared to a monoclonal antibody for the treatment of moderate-to-severe AD to be highly sensitive to the costs and effectiveness inputs and identified further cost-effectiveness drivers. It demonstrated that the JAKi could be cost-effective compared to an established monoclonal antibody with a lower dose or a reduced price.

Aims: Accurately estimating mean survival after solid organ transplant (SOT) is crucial for efficient healthcare resource allocation decisions. However, registry-based post-transplant recipient survival estimates vary greatly and are incomplete. Often, the methods used in lifetime survival extrapolation may not fit complex transplant data and therefore alternative methods are required. We aimed to explore the flexible cubic spline methodology as a meaningful alternative for estimating lifetime survival following SOT., Methods: Survival analyses were conducted in kidney, liver, heart, and lung transplant recipients. Mean survival was estimated using flexible cubic splines on the hazard scale fitted with three knots, based on where hazards changed direction, clinical advice, and best-fit curve using Akaike and Bayesian information criterion. The tail was extrapolated when data were no longer available. Extrapolation tails were compared with general population mortality, using age-matched life table hazards, and the highest hazards were taken at all times., Results: We found that mean survival post-transplant was longest for kidney transplants (US: 22.79 years; UK: 26.58 years), followed by liver (US: 20.90 years; UK: 20.38 years), heart (US: 14.82 years; UK: 15.85 years), and lung (US: 9.28 years; UK: 9.21 years). A sensitivity analysis using two knots found differences in survival ranging from -1.30 to +4.83 years across SOTs examined., Limitations: This study does not represent individual patient survival, survival by age groups, multiple-organ transplants, or assess factors that may impact overall or organ survival., Conclusions: Our study estimates reflect real-world survival following SOTs and demonstrate the importance of including long-term hazards in survival estimations. These lifetime survival estimates can be used by decision-makers in situations where means are preferred over medians (e.g. population projections, budgetary estimates, and cost-effectiveness models) and can thus offer a meaningful alternative to the estimates used and accepted in current practice.

Aims: The MiniMed 670 G insulin pump system is the first commercially available hybrid closed-loop (HCL) insulin delivery system and clinical studies have shown that this device is associated with incremental benefits in glycemic control relative to continuous subcutaneous insulin infusion (CSII) with or without continuous glucose monitoring (CGM). The aim was to evaluate the long-term cost-effectiveness of the MiniMed 670 G system versus CSII alone in people with type 1 diabetes (T1D) in the UK., Materials and Methods: Cost-effectiveness analysis was performed using the IQVIA CORE Diabetes Model. Clinical input data were sourced from a clinical trial of the MiniMed 670 G system in 124 adults and adolescents with T1D. The analysis was performed over a lifetime time horizon and both future costs and clinical outcomes were discounted at 3.5% per annum . The analysis was performed from a healthcare payer perspective., Results: The use of the MiniMed 670 G system led to an improvement in quality-adjusted life expectancy of 1.73 quality-adjusted life years (QALYs), relative to CSII. Total lifetime direct costs were GBP 35,425 higher with the MiniMed 670 G system than with CSII resulting in an incremental cost-effectiveness ratio (ICER) of GBP 20,421 per QALY gained. Sensitivity analyses revealed that the ICER was sensitive to assumptions around glycemic control and assumptions relating to the quality-of-life benefit associated with a reduction in fear of hypoglycemia., Limitations: Long-term projections from short-term data are inherently associated with uncertainty but represent arguably the best available evidence in lieu of long-term clinical trials., Conclusions: In the UK, over patient lifetimes, the incremental clinical benefits associated with the use of MiniMed 670 G system means that it is likely to be cost-effective relative to the continued use of CSII in people with T1D, particularly for those with a fear of hypoglycemia or poor baseline glycemic control.

Background and Aims: A proportion of chronic heart failure (CHF) patients will experience regurgitation secondary to ventricular remodeling in CHF, known as functional mitral (MR) or tricuspid (TR) regurgitation. Its presence adversely impacts the prognosis and healthcare utilization in CHF patients. The advent of interventional devices for both atrioventricular valves modifies both aspects. We present an economic model structure suitable for comparing interventions used in MR and TR, and assess the cost-effectiveness of transcatheter mitral valve repair (TMVr) plus guideline directed medical therapy (GDMT) compared with GDMT alone in people with MR., Methods: An economic model with a lifetime time horizon was developed based on extrapolated survival data and using New York Heart Association classifications to describe disease severity in people with functional MR at high risk of surgical mortality or deemed inoperable. Cost and utility values (describing health-related quality-of-life) were assigned to patients dependent on their disease severity. The analysis was conducted from a UK National Health Service perspective. An incremental cost per additional quality-adjusted life year (QALY) was estimated, and sensitivity (one-way and probabilistic) and scenario analyses conducted., Results and Conclusions: Compared with GDMT, the use of TMVr results in an additional 1.07 QALYs and an increase in costs of £32,267 per patient over a lifetime time horizon. The estimated incremental cost per QALY gained is £30,057 and would therefore be on the threshold of cost-effectiveness at £30,000 per quality adjusted life year. Thus, from a UK reimbursement perspective, in patients with severe functional MR who are at high risk of surgical mortality or deemed inoperable with conventional surgery, TMVr plus medical therapy is likely to represent a cost-effective treatment option compared with GDMT alone. The choice of device (MitraClip or PASCAL) will need to be confirmed once further clinical data are reported.

Background: Limited treatment options are available in chemotherapy-refractory or -intolerant metastatic colorectal cancer (mCRC). The objective of the present analysis was to evaluate the cost-utility of SIR-Spheres Y-90 resin microspheres relative to best supportive care (BSC) in the treatment of chemotherapy refractory mCRC from the perspective of the UK national healthcare payer., Methods: A cost-utility model was developed in Microsoft Excel to simulate transitions from progression-free survival to post-progression survival and death in patients with mCRC. Unit costs were captured in 2019 pounds sterling (GBP) based on the literature, formulary listings, and National Health Service (NHS) England reference costs. Future costs and effects were discounted at 3.5% per annum . A series of one-way sensitivity analyses, and probabilistic sensitivity analysis (PSA) were conducted., Results: The base case analysis showed that SIR-Spheres Y-90 resin microspheres would result in an increase in discounted quality-adjusted life years gained from 0.69 quality-adjusted life years (QALYs) to 1.50 QALYs, with an associated increase in cost from GBP 15,268 to GBP 34,168 yielding an incremental cost-utility ratio of GBP 23,435 per QALY. PSA showed that there would be a 56% likelihood that SIR-Spheres Y-90 resin microspheres would be cost-effective relative to BSC at a willingness-to-pay threshold of GBP 30,000 per QALY gained., Conclusions: This cost-utility analysis showed that, relative to BSC, SIR-Spheres Y-90 resin microspheres would be a cost-effective treatment option for patients with mCRC in the UK setting from the national healthcare payer perspective.

Aims: Respiratory syncytial virus (RSV) is a common cause of respiratory infection in infants and severe infection can result in hospitalization. The passive immunization, palivizumab, is used as prophylaxis against RSV, however, use in the UK is restricted to populations at high risk of hospitalization. This study assesses the cost-effectiveness (CE) of palivizumab in premature infants with and without risk factors for hospitalization (congenital heart disease [CHD], bronchopulmonary dysplasia [BPD])., Methods: A decision tree model, based on earlier CE analyses, was updated using data derived from targeted literature reviews and advice gained from a Round Table meeting. All costs were updated to 2019 prices. One-way and probabilistic sensitivity analyses were performed to assess the degree of uncertainty surrounding the results., Results: Palivizumab is dominant (i.e. clinically superior and cost saving) when used in premature infants born ≤35 weeks gestational age (wGA) without CHD or BPD and aged <6 months at the start of the RSV season, infants aged <24 months with CHD and infants aged <24 months requiring treatment for BPD within the last 6 months., Limitations: One-way sensitivity analysis suggests that these results are highly sensitive to the efficacy of prophylaxis, number of doses, impact of long-term respiratory sequalae, rate of hospitalization and mortality due to RSV. A conservative approach has been taken toward long-term respiratory sequalae due to uncertainty around epidemiology and etiology and a lack of recent cost and utility data., Conclusions: Palivizumab prophylaxis is cost-effective in preventing severe RSV infection requiring hospital admission in a wider population than currently recommended in UK guidelines. Prophylaxis in premature infants born <29 wGA, 29-32 wGA and 33-35 wGA without CHD or BPD aged <6 months at the start of the RSV season is not funded under current guidance, however, prophylaxis has been demonstrated to be cost-effective in this analysis.

Aims: Familial chylomicronemia syndrome (FCS) is a rare genetic disorder with no currently approved therapies. Treatments are in development, and cost-utility analyses will be needed to examine their value. These models will require health state utilities representing FCS. Therefore, the purpose of this study was to estimate utilities for FCS and an associated episode of acute pancreatitis (AP). Methods: Because it is not feasible to gather a large enough sample of patients with this extremely rare condition to complete standardized preference-based measures, vignette-based methods were used to estimate utilities. In time trade-off interviews, general population participants in the UK and Canada valued health state vignettes drafted based on literature review, clinician input, and interviews with patients. Four health states described variations of FCS. A fifth health state, describing AP, was added to one of the other health states to evaluate its impact on utility. Results: A total of 308 participants provided utility data (208 UK; 100 Canada). Mean utilities for FCS health states ranged from 0.46 to 0.83, with higher triglycerides, more severe symptoms, and a history of AP associated with lower utility values. The disutility (i.e. utility decrease) of AP ranged from -0.17 to -0.25, with variations depending on the health state to which it was added. Utility means were similar in the UK and Canada. Conclusions: The vignette-based approach is useful for estimating utilities of a rare disease. The health state utilities derived in this study would be useful in models examining cost-effectiveness of treatments for FCS.

Aims: Model how moving from current disease-modifying drug (DMD) prescribing patterns for relapsing-remitting multiple sclerosis (RRMS) observed in the United Kingdom (UK) to prescribing patterns based on patient preferences would impact health outcomes over time. Materials and methods: A cohort-based Markov model was used to measure the effect of DMDs on long-term health outcomes for individuals with RRMS. Data from a discrete choice experiment were used to estimate the market shares of DMDs based on patient preferences (i.e. preference shares). These preference shares and real-world UK market shares were used to calculate the effect of prescribing behavior on relapses, disability progression, and quality-adjusted life-years (QALYs). The incremental benefit of patient-centered prescribing over current practices for the UK RRMS population was then estimated; scenario and sensitivity analyses were also conducted. Results: Compared to current prescribing practices, when UK patients with RRMS were treated following patient preferences, health outcomes were improved. This population was expected to experience 501,690 relapses and gain 1,003,263 discounted QALYs over 50 years under patient-centered prescribing practices compared to 538,417 relapses and 958,792 discounted QALYs under current practices (-6.8% and +4.6%, respectively). Additionally, less disability progression was observed when prescribed treatment was based on patient preferences. In a scenario analysis where only oral treatments were considered, the results were similar, although the magnitude of benefit was smaller. Number of relapses was most sensitive to how the annualized relapse rate was modeled; disability progression was most sensitive to mortality rate assumptions. Limitations: Treatment efficacy estimates applied to various models in this study were based on data derived from clinical trials, rather than real-world data; the impact of patient-centered prescribing on treatment adherence and/or switching was not modeled. Conclusions: The population of UK RRMS patients may experience overall health gains if patient preferences are better incorporated into prescribing practices.

Aims: Falls have devastating consequences in older people with a considerable cost burden. Glaucoma is a risk factor for falls, and patients with glaucoma who fall are at high risk of hospital admission. The aim was to quantify the cost burden of falls to NHS Trusts in people with glaucoma in the UK. Methods: Financial data were used to identify non-elective episodes and associated costs from 2012 to 2018, for all admissions where glaucoma was recorded as a secondary diagnosis and admissions for falls (all, with and without a glaucoma secondary diagnosis). A secondary diagnosis is only recorded by the admitting clinician if it is clinically relevant; therefore, a secondary diagnosis of glaucoma was used as a proxy for glaucoma as a contributory factor to falls. Limitations: Use of financial records means that data on other falls risk factors was unavailable and we cannot be certain that glaucoma was the only relevant factor in all falls. Although this methodology is imperfect, case capture was biased towards cases with clinically significant glaucoma, and financial data is robust. Potential coding errors mean that we may have excluded patients in whom glaucoma was a factor in their fall. Results: At Maidstone and Tunbridge Wells (MTW) NHS Trust, 11.7% (95% confidence intervals [CI] = 10.7-12.8) of admissions for falls were in patients with a secondary diagnosis of glaucoma. This extrapolates to an estimated annual 10,056 admissions at a cost of £28.6 million across the UK. This is an under-estimate of cost, as A&E attendance without admission and outpatient appointments are excluded. Conclusions: At MTW, glaucoma potentially plays a part in around one in eight falls resulting in hospital admission, at considerable personal and financial cost. It is suggested that further work should explore early diagnosis of glaucoma, treatment, and mitigation of falls risk.

Aim: To evaluate the relative cost-effectiveness of using rivaroxaban vs apixaban for the initial treatment plus extended prevention of venous thromboembolism (VTE) in the UK. Extended prevention was assessed using a 10-mg rivaroxaban dose, as the 20-mg dose has already been evaluated. Methods: A Markov model compared the health outcomes and costs of treating VTE patient cohorts with either rivaroxaban (15 mg twice daily for 3 weeks, followed by 20 mg once daily for 6 months, then extended prevention with 10 mg once daily) or apixaban (10 mg twice daily for 1 week, followed by 5 mg twice daily for 6 months, then extended prevention with 2.5 mg twice daily) over a lifetime horizon. The model included an initial acute treatment and prevention phase (0-6 months) and an extended prevention phase (6-18 months). Efficacy and safety data were derived from two network meta-analyses. Reference treatment comparators were derived from the EINSTEIN-Pooled study and EINSTEIN-CHOICE trial. Healthcare costs and utility data were derived from published literature. Results: The rivaroxaban regimen was associated with increased quality-adjusted life years (QALYs) and slightly lower total costs compared with apixaban over a lifetime horizon. Deterministic and probabilistic sensitivity analyses demonstrated that rivaroxaban remained a cost-effective alternative to apixaban over a wide range of parameters. Incremental cost-effectiveness ratio estimates were below the £20,000 per QALY threshold in 74.1% of 2,000 model simulations. Scenario analyses further supported that rivaroxaban is a cost-effective alternative to apixaban. Limitations: Clinical and safety inputs were derived from network meta-analysis, which are subject to inherent limitations whereby small differences between study designs may severely impact efficacy and safety outcomes. Furthermore, these inputs were based on data from clinical trials, which may not reflect real-world data. Conclusions: Rivaroxaban was associated with a slightly lower total cost and increased QALYs compared with apixaban for VTE management in the UK over a lifetime horizon.

Aims: To estimate the cost impact of non-medical switching from originator to biosimilar etanercept in stable patients with rheumatoid arthritis (RA) in the UK. Materials and methods: A cohort-based decision tree model was developed with a 1-year time horizon. The model population included patients with stable RA (patients who responded to originator etanercept treatment with no treatment changes in the previous 6 months). Patients could undergo a non-medical switch to a biosimilar and then switch treatment again, if medically required, after 3-6 months. Data on the proportion of patients switching therapies, baseline healthcare resource use, and impact of switching on resource use were sourced from a survey of 150 rheumatologists from EU5 markets (France, Germany, Italy, Spain, UK). The average impact of switching was evaluated as mean values for change in resource utilization due to switching. Also, low- and high-impact scenarios (lower and upper values of the 95% confidence intervals for change in resource utilization due to switching) were modelled as sensitivity analyses. Cost data came from published UK sources. Results: The model assumed that 5,000 patients were treated with originator etanercept, with 1,259 (25.2%) switching to a biosimilar. Of those, 875 (69.5%) and 384 (30.5%) switched to SB4 and GP2015, respectively. After 3 months, 26.3% of patients who switched treatments did so again: 8.3% back to originator, 3.8% to the other biosimilar, and 14.2% to another biologic. Although originator etanercept was more expensive than the biosimilars, switching was more costly than continuous originator treatment across all impact scenarios. Switching treatment chains had higher overall annual per-patient costs than continuous originator treatment. Switching was associated with increased healthcare resource use. Limitations: Results from this analysis are not transferable to other (non-RA) etanercept indications. Conclusion: Non-medical switching can result in increased payer costs because of increased healthcare resource use following switching.

Aims: To examine healthcare resource utilization associated with refractory myasthenia gravis (MG) in England. Materials and methods: This was a retrospective cohort study of linked data from the Clinical Practice Research Datalink and the Hospital Episode Statistics database collected between 1997 and 2016. Included patients were ≥18 years of age at the index MG diagnosis. Patients with refractory MG were identified using an algorithm based on treatments received. Healthcare resource utilization since the index date was compared between refractory and non-refractory cohorts. Results: The study included 1149 patients with MG, of whom 66 (5.7%) were refractory. Sex and age at diagnosis did not significantly differ between the refractory and non-refractory cohorts. Rates of healthcare resource utilization per person-year were significantly higher ( p  < .05) for patients with refractory compared to non-refractory MG for GP visits, visits to other healthcare professionals, outpatient visits and inpatient hospitalization. Patients in the refractory cohort spent more total days hospitalized since the index visit than patients in the non-refractory cohort (median, 33 vs. 16 days [ p  < .0001]). Limitations: The algorithm for identifying refractory patients did not include clinical criteria. Also, treatments administered in hospitals or by specialists were not available in the databases. Conclusions: Patients in England with refractory MG more often visit healthcare providers, are hospitalized and visit an emergency room than patients with non-refractory MG.

Background and Aims: Infection is a serious and expensive complication of Cardiac Implantable Electronic Device (CIED) procedures. A retrospective based cost analysis was performed to estimate Trust level savings of using the TYRX antibacterial envelope as a primary prevention measure against infection in a tertiary referral centre in South London, UK., Methods: A retrospective cohort of heart failure patients with reduced ejection fraction undergoing Implantable Cardioverter Defibrillator (ICD) or Cardiac Resynchronization Therapy (CRT) procedures were evaluated. Decision-analytic modelling was performed to determine economic savings of using the envelope during CIED procedure vs CIED procedure alone., Results: Over a 12 month follow-up period following CIED procedure, the observed infection rate was 3.14% (n = 5/159). The average cost of a CIED infection inpatient admission was £41,820 and, further to economic analysis, the additional costs attributable to infection was calculated at £62,213.94. A cost saving of £624 per patient by using TYRX during CIED procedure as a primary preventative measure against infection was estimated., Conclusions: TYRX would be a cost-saving treatment option amongst heart failure patients undergoing ICD and CRT device procedures based on analysis in the local geographical area of South London. If upscaled to the UK population, we estimate potential cost savings for the National Health Service (NHS).

Background: Validation of overall survival (OS) extrapolations of immune-checkpoint inhibitors (ICIs) during the National Institute for Health and Care Excellence (NICE) Single Technology Assessment (STA) process is limited due to data still maturing at the time of submission. Inaccurate extrapolation may lead to inappropriate decision-making. The availability of more mature trial data facilitates a retrospective analysis of the plausibility and validity of initial extrapolations. This study compares these extrapolations to subsequently available longer-term data., Methods: A systematic search of completed NICE appraisals of ICIs from March 2000 to December 2017 was performed. A targeted search was also undertaken to procure published OS data from the pivotal clinical trials for each identified STA made available post-submission to NICE. Initial Kaplan-Meier curves and associated extrapolations from NICE documentation were extracted to compare the accuracy of OS projections versus the most mature data., Results: The review identified 11 STAs, of which 10 provided OS data upon submission to NICE. The extrapolations undertaken considered parametric or piecewise survival models. Additional data cut-offs provided a mean of 18 months of OS beyond the end of the original data. Initial extrapolations typically under-estimated OS from the most mature data cut-off by 0.4-2.7%, depending on the choice of assessment method and use of the manufacturer- or ERG-preferred extrapolation., Conclusion: Long-term extrapolation of OS is required for NICE STAs based on initial immature OS data. The results of this study demonstrate that the initial OS extrapolations employed by manufacturers and ERGs generally predicted OS reasonably well when compared to more mature data (when available), although on average they appeared to underestimate OS. This review and validation shows that, while the choice of OS extrapolation is uncertain, the methods adopted are generally aligned with later-published follow-up data and appear appropriate for informing HTA decisions.

Aims: Percutaneous closure of a patent foramen ovale (PFO) is known to lower the risk of recurrent stroke in patients with a cryptogenic stroke. However, the economic implications of transcatheter PFO closure are less well known. From a UK payer perspective, a detailed economic appraisal of PFO closure was performed for prevention of recurrent ischemic stroke in patients with a PFO who had experienced a cryptogenic stroke., Materials and Methods: A Markov cohort model was constructed using a 5-year time-horizon with a patient mean age of 45.2 years, reflecting the characteristics reported in the REDUCE trial. Transition probabilities, clinical inputs, costs, and utility values were ascertained from published and national costing sources. Total costs, incremental costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios were calculated, utilizing a discount rate of 3.5%. A range of univariate and probabilistic sensitivity analyses were also performed., Results: When applying a willingness-to-pay (WTP) threshold of £20,000/QALY in accordance with NICE guidelines, PFO closure compared with antiplatelet therapy alone showed a beneficial cost/QALY of £18,584, attained at 4 years. Applying discount rates of 0% and 6% had a negligible effect on the base-case model findings. PFO closure demonstrated a 76.9% probability of being cost-effective at a WTP threshold of £20,000/QALY at a 5-year time-horizon., Limitations: This model focused specifically on UK stroke patients and typically enrolled young (mean age <65 years old) patients. Hence, caution should be taken when comparing data vs non-UK populations, and it remains unclear how older patients might have affected cost-effectiveness findings, as the risk of paradoxical embolism can persist as patients age., Conclusion: Percutaneous closure of a PFO is cost-effective compared with antiplatelet therapy alone, underlining the economic benefits potentially afforded by this treatment in selected patients.