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Short-course regimens are currently explored to improve multidrug-resistant tuberculosis effects, reduce costs, as well as enhance patient adherence. Currently, we are determining the most cost-effective shorter regimen out of seven short-course regimens (6–9 months) to treat drug-resistant tuberculosis (DR-TB) compared to the current standard of care (SoC) 9- to 11-month regimen.
Methods
Cost-effectiveness of various short-course DR-TB treatment regimens, namely BEAT, BPaL, BPaLM, BPaLC, mBPaL1, mBPaL2, and mBPaL3, was compared to the current SoC in India. Decision tree model was used from a health system perspective. The information on various costs – such as preinvestigations, regimens, adverse drug reactions (ADRs) management, inpatient treatment – and on effect – such as clinical outcomes and ADRs – was collected from different published sources. It estimated costs, quality-adjusted life years, and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were performed to validate outcomes against the willingness-to-pay threshold.
Results
When all the short-course regimens were compared with the current SoC regimen, the ICERs were ₹5,385, ₹2,014, ₹2,008, ₹2,435, ₹1,462, ₹1,159, and ₹1,895 for BEAT, BPaL, BPaLM, BPaLC, mBPaL1, mBPaL2, and mBPaL3, respectively. Among the short-course regimens, mBPaL2 is the dominant strategy, and mBPaL1 has extended dominance. For all Bedaquiline-containing regimens, the cost of the drug is a crucial factor in determining cost effectiveness. The cost-effectiveness acceptability curve showed that all shorter regimens were 100 percent cost-effective.
Conclusion
The implementation of Bedaquiline-based regimen to treat DR-TB has become more effective, shorter in duration, and less burdensome to the health system.
Predictive biomarkers can identify patients who are more likely to respond to immunotherapy, which can guide treatment decisions. The objective of this study was to assess the potential value of predictive biomarkers in advanced NSCLC patients to guide the development of cost-effective biomarkers in this field.
Methods
A decision analytical model was constructed to compare theoretical new strategies with biomarkers to the current standard of care. The analysis was performed for three different patient groups based on PD-L1 status. Differences in health outcomes (QALYs) and costs were assessed between the current practice and these biomarker strategies.
Results
Omitting immunotherapy in NSCLC patients with a PD-L1 score < 1 percent or between 1 and 49 percent, and a negative biomarker test, could potentially reduce healthcare costs significantly a small loss in QALYs. In these groups, a biomarker test is potentially cost-effective as the incremental cost-effectiveness ratio largely exceeds a willingness-to-accept threshold of €80,000 saved per QALY lost. For patients with a PD-L1 score > 50 percent, a considerable QALY gain can potentially be realized by adding chemotherapy to patients with a negative biomarker test. However, this comes at a significant increase in costs and appears not to be cost-effective.
Conclusions
In general, predictive biomarkers seem to have the potential to increase the cost-effectiveness of treatment with immunotherapy in patients with advanced NSCLC. Optimal positioning of a biomarker depends on the weighing between health impact and costs.
In the UK, around 1 in 4 adults over 65 years suffers from depression. Depression case finding followed by alerting patients and their general practioners (GPs) (screening + GP) is a promising strategy to facilitate depression management, but its cost-effectiveness remains unclear.
Aims
To investigate the cost-effectiveness of screening + GP compared with standard of care (SoC) in northern England.
Method
Conducted alongside the CASCADE study, 1020 adults aged 65+ years were recruited. Participants with baseline Geriatric Depression Scale (GDS) ≥5 were allocated to the intervention arm and those >5 to SoC. Resource use and EQ-5D-5L data were collected at baseline and 6 months. Incremental cost-effectiveness ratio was calculated. Non-parametric bootstrapping was performed to capture sampling uncertainty. The results are presented using cost-effectiveness acceptability curves. Sensitivity analyses were conducted to assess the robustness of primary findings. Subgroup analyses were undertaken to examine the cost-effectiveness among participants with more comparable baseline characteristics across treatment groups.
Results
Screening + GP incurred £37 more costs and 0.006 fewer quality-adjusted life years than SoC; the probability of the former being cost-effective was <5% at a £30 000 cost-effectiveness threshold. Sensitivity analyses confirmed the base-case findings. Subgroup analyses indicated that screening + GP was cost-effective when patients with baseline GDS 2–7, 3–6 and 4–5, respectively, were analysed.
Conclusions
Screening + GP was dominated by SoC in northern England. However, subgroup analyses suggested it could be cost-effective if patients with more balanced baseline characteristics were analysed. Economic evaluations alongside randomised controlled trials are warranted to validate these findings.
Mental health literacy can potentially be improved through a public mental health campaign. The aim of the campaign Every Mind Matters (EMM) was to support adults to help address common subclinical mental health problems and improve their mental well-being and literacy, by using its National Health Service-endorsed digital resources.
Aims
Although not an objective of the campaign itself, this study aims to (a) address the relationship of EMM through the use of general practitioners and mental health therapists and (b) explore the association of EMM with symptom management knowledge.
Method
Health Survey for England 2019 data were obtained on campaign awareness, uptake of campaign materials and the use of general practitioners and therapists. Logistic regression models were used to explore the impact of the campaign on whether services were used, and ordered logistic models explored the impact on the number of contacts. Campaign costs were viewed alongside symptom management outcomes.
Results
The analyses included 2023 individuals. Of those campaign aware, 16% had contact with a general practitioner for mental health reasons compared with 9% of those who were campaign unaware. Those who were campaign aware were also significantly more likely to have seen a mental health therapist. The campaign cost per unit improvement in symptom management knowledge was below £20.
Conclusions
Contact with general practitioners and therapists was associated with campaign awareness. If even a small proportion of symptom management knowledge improvement is due to the campaign, then it has the potential to be cost-effective. Further work is required to establish this.
Healthcare technologies are often appraised under considerable ambiguity over the size of incremental benefits and costs, and thus how decision-makers combine unclear information to make recommendations is of considerable public interest. This paper provides a conceptual foundation for such decision-making under ambiguity, formalizing and differentiating the decision problems of a representative policy-maker reviewing the results from an economic evaluation. A primary result is that presenting information to regulators in an incremental cost-effectiveness ratio or cost-effectiveness analysis (CEA) format instead of a net monetary benefit or cost–benefit analysis (CBA) framework may induce errors in decision-making when there exists ambiguity in incremental benefits and decision-makers use well-known decision rules to combine information. Ambiguity in incremental costs or the value of the cost-effectiveness threshold does not distort decision-making under these rules. In reasonable contexts, I show that the CEA framing may result in the approval of fewer technologies relative to CBA framing. I interpret these results as predictions on how the presentation of information from economic evaluations to regulators may frame and distort recommendations. All the results extend to non-healthcare contexts.
Due to the risk of Shiga-toxin producing Escherichia coli (STEC) transmission, current guidance advises excluding young children from childcare settings until microbiologically clear. Children can shed STEC for a prolonged period, and the cost-effectiveness of exclusion has not been evaluated. Our decision tree analysis, including probabilistic sensitivity analysis, estimated comparative health system costs and effects of exclusion until microbiological clearance versus return to childcare setting before this. Due to the risk of secondary cases, return before microbiological clearance resulted in the incremental loss of 0.019 QALYs, but savings of £156. Using the willingness-to-pay threshold of £20000 per QALY, the incremental net monetary benefit of exclusion until microbiological clearance was £215. Exclusion until microbiological clearance remained cost-effective if the total costs for managing the exclusion were below £576. Return before microbiological clearance may, therefore, become cost-effective in cases where the costs of managing exclusion until microbiological clearance are high and/or the risk of secondary cases is very low. Broadening the decision perspective, including the costs of exclusion to the families, may also impact the recommendation. Further research is needed to assess the risk of STEC transmission from children who have clinically recovered and the impact of STEC and exclusion on families of the affected children.
Early intervention in psychosis (EIP) services improve outcomes for young people, but approximately 30% disengage.
Aims
To test whether a new motivational engagement intervention would prolong engagement and whether it was cost-effective.
Method
We conducted a multicentre, single-blind, parallel-group, cluster randomised controlled trial involving 20 EIP teams at five UK National Health Service (NHS) sites. Teams were randomised using permuted blocks stratified by NHS trust. Participants were all young people (aged 14–35 years) presenting with a first episode of psychosis between May 2019 and July 2020 (N = 1027). We compared the novel Early Youth Engagement (EYE-2) intervention plus standardised EIP (sEIP) with sEIP alone. The primary outcome was time to disengagement over 12–26 months. Economic outcomes were mental health costs, societal costs and socio-occupational outcomes over 12 months. Assessors were masked to treatment allocation for primary disengagement and cost-effectiveness outcomes. Analysis followed intention-to-treat principles. The trial was registered at ISRCTN51629746.
Results
Disengagement was low at 15.9% overall in standardised stand-alone services. The adjusted hazard ratio for EYE-2 + sEIP (n = 652) versus sEIP alone (n = 375) was 1.07 (95% CI 0.76–1.49; P = 0.713). The health economic evaluation indicated lower mental healthcare costs linked to reductions in unplanned mental healthcare with no compromise of clinical outcomes, as well as some evidence for lower societal costs and more days in education, training, employment and stable accommodation in the EYE-2 group.
Conclusions
We found no evidence that EYE-2 increased time to disengagement, but there was some evidence for its cost-effectiveness. This is the largest study to date reporting positive engagement, health and cost outcomes in a total EIP population sample. Limitations included high loss to follow-up for secondary outcomes and low completion of societal and socio-occupational data. COVID-19 affected fidelity and implementation. Future engagement research should target engagement to those in greatest need, including in-patients and those with socio-occupational goals.
Selenium (Se) deficiency among populations in Ethiopia is consistent with low concentrations of Se in soil and crops that could be addressed partly by Se-enriched fertilisers. This study examines the disease burden of Se deficiency in Ethiopia and evaluates the cost-effectiveness of Se agronomic biofortification. A disability-adjusted life years (DALY) framework was used, considering goiter, anaemia, and cognitive dysfunction among children and women. The potential efficiency of Se agronomic biofortification was calculated from baseline crop composition and response to Se fertilisers based on an application of 10 g/ha Se fertiliser under optimistic and pessimistic scenarios. The calculated cost per DALY was compared against gross domestic product (GDP; below 1–3 times national GDP) to consider as a cost-effective intervention. The existing national food basket supplies a total of 28·2 µg of Se for adults and 11·3 µg of Se for children, where the risk of inadequate dietary Se reaches 99·1 %–100 %. Cereals account for 61 % of the dietary Se supply. Human Se deficiency contributes to 0·164 million DALYs among children and women. Hence, 52 %, 43 %, and 5 % of the DALYs lost are attributed to anaemia, goiter, and cognitive dysfunction, respectively. Application of Se fertilisers to soils could avert an estimated 21·2–67·1 %, 26·6–67·5 % and 19·9–66·1 % of DALY via maize, teff and wheat at a cost of US$129·6–226·0, US$149·6–209·1 and US$99·3–181·6, respectively. Soil Se fertilisation of cereals could therefore be a cost-effective strategy to help alleviate Se deficiency in Ethiopia, with precedents in Finland.
Depression is common in people with dementia, and negatively affects quality of life.
Aims
This paper aims to evaluate the cost-effectiveness of an intervention for depression in mild and moderate dementia caused by Alzheimer's disease over 12 months (PATHFINDER trial), from both the health and social care and societal perspectives.
Method
A total of 336 participants were randomised to receive the adapted PATH intervention in addition to treatment as usual (TAU) (n = 168) or TAU alone (n = 168). Health and social care resource use were collected with the Client Service Receipt Inventory and health-related quality-of-life data with the EQ-5D-5L instrument at baseline and 3-, 6- and 12-month follow-up points. Principal analysis comprised quality-adjusted life-years (QALYs) calculated from the participant responses to the EQ-5D-5L instrument.
Results
The mean cost of the adapted PATH intervention was estimated at £1141 per PATHFINDER participant. From a health and social care perspective, the mean difference in costs between the adapted PATH and control arm at 12 months was −£74 (95% CI −£1942 to £1793), and from the societal perspective was −£671 (95% CI −£9144 to £7801). The mean difference in QALYs was 0.027 (95% CI −0.004 to 0.059). At £20 000 per QALY gained threshold, there were 74 and 68% probabilities of adapted PATH being cost-effective from the health and social care and societal perspective, respectively.
Conclusions
The addition of the adapted PATH intervention to TAU for people with dementia and depression generated cost savings alongside a higher quality of life compared with TAU alone; however, the improvements in costs and QALYs were not statistically significant.
In Chapter 4, we identified commodification as the policy orientation most relevant to our analysis of the nexus between EU economic governance and labour politics and developed a corresponding novel analytical framework to assess new economic governance (NEG) prescriptions in the areas of employment relations and public services. Before engaging in this assessment however, we need to understand the prescriptions’ meaning, for which we must make an additional analytical move. The meaning of NEG policy prescriptions depends not only on their wording but also on their location in larger policy scripts and their uneven coercive power across countries, time, and policy areas. Hence, NEG prescriptions are embedded in larger semantic fields and taxonomies, in power struggles over the definition of appropriate solutions to social problems, and in the EU’s integrated but also uneven political economy. Chapter 5 thus first explains the semantic, communicative, and policy contexts in which we situate NEG prescriptions and then outlines the implications of this analytical move for our research design, including case selection, data collection, and comparative approach.
One in 57 children are diagnosed with autism in the UK, and the estimated cost for supporting these children in education is substantial. Social Stories™ is a promising and widely used intervention for supporting children with autism in schools and families. It is believed that Social Stories™ can provide meaningful social information to children that can improve social understanding and may reduce anxiety. However, no economic evaluation of Social Stories has been conducted.
Aims
To assess the cost-effectiveness of Social Stories through Autism Spectrum Social Stories in Schools Trial 2, a multi-site, pragmatic, cluster-randomised controlled trial.
Method
Children with autism who were aged 4–11 years were recruited and randomised (N = 249). Costs measured from the societal perspective and quality-adjusted life-years (QALYs) measured by the EQ-5D-Y-3L proxy were collected at baseline and at 6-month follow-up for primary analysis. The incremental cost-effectiveness ratio was calculated, and the uncertainty around incremental cost-effectiveness ratios was captured by non-parametric bootstrapping. Sensitivity analyses were performed to evaluate the robustness of the primary findings.
Results
Social Stories is likely to result in a small cost savings (–£191 per child, 95% CI −767.7 to 337.7) and maintain similar QALY improvements compared with usual care. The probability of Social Stories being a preferred option is 75% if society is willing to pay £20 000 per QALY gained. The sensitivity analysis results aligned with the main study outcomes.
Conclusions
Compared with usual care, Social Stories did not lead to an increase in costs and maintained similar QALY improvements for primary-aged children with autism.
Schizophreniform disorders tend to have an early onset. Early intervention in psychosis (EIP) services aim to provide early treatment, reduce long-term morbidity and improve social functioning. In 2016, changes to mental health policy in England mandated that the primarily youth-focused model should be extended to an ageless one, to prevent ageism; however, this was without strong research evidence.
Aims and method
An inner-city London EIP service compared sociodemographic and clinical factors between the under-35 years and over-35 years caseload cohorts utilising the EIP package following the implementation of the ageless policy.
Results
Both groups received similar care, despite the younger group having significantly more clinical morbidity and needs.
Clinical implications
Our results may indicate that service provisions are being driven by policy rather than clinical needs, potentially diverting resources from younger patients. These findings have important implications for future provision of EIP services and would benefit from further exploration.
Edited by
Allan Young, Institute of Psychiatry, King's College London,Marsal Sanches, Baylor College of Medicine, Texas,Jair C. Soares, McGovern Medical School, The University of Texas,Mario Juruena, King's College London
Patients with mood disorders experience substantial challenges in their lives, often over long-term periods and despite receiving treatment. Provision of clinical care for mood disorders involves direct monetary costs. Illness also leads to indirect socioeconomic costs due to reduced work capacity. The absence of these patients from wider economic activity within society is another indirect cost. Estimating the impact of mood disorders in monetary terms mainly relies on administrative records, patient surveys, and mathematical models. Although estimations may vary between studies depending on methodology, annual economic cost of major depressive disorder and bipolar disorder in the UK may exceed £8 billion and £7 billion, respectively, with the majority of this cost accounted for by lost production rather than provided healthcare. Other indirect costs are commonly ignored and require further research. Cost of illness studies may serve as the basis for economic evaluations (e.g., cost-effectiveness analyses) of interventions targeting mood disorders.
Patients with hematological malignancies are likely to develop hypogammaglobulinemia. Immunoglobulin (Ig) is commonly given to prevent infections, but its overall costs and cost-effectiveness are unknown.
Methods
A systematic review was conducted following the PRISMA guidelines to assess the evidence on the costs and cost-effectiveness of Ig, administered intravenously (IVIg) or subcutaneously (SCIg), in adults with hematological malignancies.
Results
Six studies met the inclusion criteria, and only two economic evaluations were identified; one cost-utility analysis (CUA) of IVIg versus no Ig, and another comparing IVIg with SCIg. The quality of the evidence was low. Compared to no treatment, Ig reduced hospitalization rates. One study reported no significant change in hospitalizations following a program to reduce IVIg use, and an observational study comparing IVIg with SCIg suggested that there were more hospitalizations with SCIg but lower overall costs per patient. The CUA comparing IVIg versus no Ig suggested that IVIg treatment was not cost-effective, and the other CUA comparing IVIg to SCIg found that home-based SCIg was more cost-effective than IVIg, but both studies had serious limitations.
Conclusions
Our review highlighted key gaps in the literature: the cost-effectiveness of Ig in patients with hematological malignancies is very uncertain. Despite increasing Ig use worldwide, there are limited data regarding the total direct and indirect costs of treatment, and the optimal use of Ig and downstream implications for healthcare resource use and costs remain unclear. Given the paucity of evidence on the costs and cost-effectiveness of Ig treatment in this population, further health economic research is warranted.
Older adults in aged care homes account for 30% of the population burden of hip fractures(1). Nutritional interventions to correct protein and calcium inadequacies reduce these and other debilitating fractures, perhaps partly by reducing falls and slowing deterioration in bone morphology. We aimed to determine whether a nutritional approach to fracture risk reduction in aged care homes is cost-effective. Costing was estimated based on results of a prospective two-year cluster-randomised controlled trial involving 3313 residents in 27 aged care homes (intervention using high dairy menus), 3911 residents in 29 aged care homes (controls consuming from normal menus) and cost of ambulance, hospital, rehabilitation, and residential care incurred after fracture. The incremental cost-effectiveness ratios per fracture averted within a 2-year time horizon were estimated from the Australian healthcare perspective applying a 5% discount rate on costs after the first year. Intervention resulted in a total of 3.5 servings of milk, yoghurt and/or cheese daily, achieving 1,142mg calcium and 69g protein versus usual daily intakes of 700mg calcium and 58g protein consumed by controls. This intervention reduced all fractures by 33% at a daily cost of AU$0.66 per resident. The base-case results showed that intervention was cost-saving per fracture averted, with robust results in a variety of sensitivity and scenario analyses. Scaling the benefits of intervention to the Australian community equated to a saving of AU$66,780,000 annually in Australia and remained cost saving up to a daily food expenditure of AU$1.07 per aged care resident. Averting hip and other non-vertebral fractures in older adults in aged care homes by restoring nutritional inadequacies of protein and calcium is cost saving and supports the wide-spread implementation of this type of nutritional intervention in similar settings.
Nasal septoplasty is one of the most performed procedures within ENT. Nasal obstruction secondary to a deviated nasal septum is the primary indication for functional septoplasty. Since the coronavirus disease 2019 pandemic, waiting lists have increased and are now long. This study assessed patients on the waiting list for septoplasty and/or inferior turbinate reduction surgery using the Nasal Obstruction Symptom Evaluation instrument.
Method
Patients on our waiting list for septoplasty and/or inferior turbinate reduction surgery were reviewed using a validated patient-reported outcome measure tool to assess symptom severity.
Results
Eighty-six out of a total of 88 patients (98 per cent) had Nasal Obstruction Symptom Evaluation scores of 30 or more. In addition, 78 (89 per cent) and 50 (57 per cent) patients were classified as having ‘severe’ or ‘extreme’ nasal obstruction, respectively. Two patients scored less than 30 and were classified as having non-significant nasal obstruction.
Conclusion
The Nasal Obstruction Symptom Evaluation instrument is a quick and easy way to validate septoplasty waiting lists. In this study, two patients were identified who no longer required surgery.
The personalised oncology paradigm remains challenging to deliver despite technological advances in genomics-based identification of actionable variants combined with the increasing focus of drug development on these specific targets. To ensure we continue to build concerted momentum to improve outcomes across all cancer types, financial, technological and operational barriers need to be addressed. For example, complete integration and certification of the ‘molecular tumour board’ into ‘standard of care’ ensures a unified clinical decision pathway that both counteracts fragmentation and is the cornerstone of evidence-based delivery inside and outside of a research setting. Generally, integrated delivery has been restricted to specific (common) cancer types either within major cancer centres or small regional networks. Here, we focus on solutions in real-world integration of genomics, pathology, surgery, oncological treatments, data from clinical source systems and analysis of whole-body imaging as digital data that can facilitate cost-effectiveness analysis, clinical trial recruitment, and outcome assessment. This urgent imperative for cancer also extends across the early diagnosis and adjuvant treatment interventions, individualised cancer vaccines, immune cell therapies, personalised synthetic lethal therapeutics and cancer screening and prevention. Oncology care systems worldwide require proactive step-changes in solutions that include inter-operative digital working that can solve patient centred challenges to ensure inclusive, quality, sustainable, fair and cost-effective adoption and efficient delivery. Here we highlight workforce, technical, clinical, regulatory and economic challenges that prevent the implementation of precision oncology at scale, and offer a systematic roadmap of integrated solutions for standard of care based on minimal essential digital tools. These include unified decision support tools, quality control, data flows within an ethical and legal data framework, training and certification, monitoring and feedback. Bridging the technical, operational, regulatory and economic gaps demands the joint actions from public and industry stakeholders across national and global boundaries.
To assess cost-effectiveness of late time-window endovascular treatment (EVT) in a clinical trial setting and a “real-world” setting.
Methods:
Data are from the randomized ESCAPE trial and a prospective cohort study (ESCAPE-LATE). Anterior circulation large vessel occlusion patients presenting > 6 hours from last-known-well were included, whereby collateral status was an inclusion criterion for ESCAPE but not ESCAPE-LATE. A Markov state transition model was built to estimate lifetime costs and quality-adjusted life-years (QALYs) for EVT in addition to best medical care vs. best medical care only in a clinical trial setting (comparing ESCAPE-EVT to ESCAPE control arm patients) and a “real-world” setting (comparing ESCAPE-LATE to ESCAPE control arm patients). We performed an unadjusted analysis, using 90-day modified Rankin Scale(mRS) scores as model input and analysis adjusted for baseline factors. Acceptability of EVT was calculated using upper/lower willingness-to-pay thresholds of 100,000 USD/50,000 USD/QALY.
Results:
Two-hundred and forty-nine patients were included (ESCAPE-LATE:n = 200, ESCAPE EVT-arm:n = 29, ESCAPE control-arm:n = 20). Late EVT in addition to best medical care was cost effective in the unadjusted analysis both in the clinical trial and real-world setting, with acceptability 96.6%–99.0%. After adjusting for differences in baseline variables between the groups, late EVT was marginally cost effective in the clinical trial setting (acceptability:49.9%–61.6%), but not the “real-world” setting (acceptability:32.9%–42.6%).
Conclusion:
EVT for LVO-patients presenting beyond 6 hours was cost effective in the clinical trial setting and “real-world” setting, although this was largely related to baseline patient differences favoring the “real-world” EVT group. After adjusting for these, EVT benefit was reduced in the trial setting, and absent in the real-world setting.