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Cost-effectiveness thresholds in policy and practice: do HTA guidelines align with estimates of health opportunity cost?
Get PDFHealth technology assessment (HTA) processes provide evidence to inform the supply of healthcare, often comparing results from economic evaluation to a policy threshold to judge cost-effectiveness. However, recommended policy thresholds may not always align with empirical estimates of the opportunity costs of health care expenditure, captured by marginal productivity of healthcare expenditure (‘k’). Such estimates are needed to inform the net health impact of funding decisions. We map policy thresholds in HTA guidelines against published estimates of k. We extract information from HTA guidelines identified in a previous literature review, including recommended perspective, relevant costs and outcomes, and justification for the threshold. Studies estimating k were obtained from a separate review. Of the 47 included HTA guidelines, 20 state an explicit policy threshold and 12 justify their choice. Estimates of k were available for 13 countries. Among the eight countries with explicit policy thresholds and k estimates, three matched. The recommended perspective influences whether k alone is sufficient or appropriate to inform cost-effectiveness judgements. It is important that guideline setters are aware of empirical estimates of k; and that economic evaluations consider k to reflect health opportunity costs even where the policy threshold is justified on other grounds
Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease:a systematic review and network meta-analysis
Get PDFBACKGROUND: Late-onset Pompe disease is a rare inherited genetic condition that causes progressive muscle dysfunction and damage. As the disease advances, the progressive weakening of respiratory muscles significantly increases the risk of respiratory failure, which is a major contributor to premature mortality. Enzyme replacement therapy is the primary treatment for Pompe disease. OBJECTIVE: To investigate the clinical impact of enzyme replacement therapies for the treatment and management of late-onset Pompe disease and establish the relative effectiveness of enzyme replacement therapy compared to best supportive care (in the absence of enzyme replacement therapy). METHODS: A systematic review and network meta-analysis of published evidence on the clinical effectiveness of enzyme replacement therapy and best supportive care was undertaken. Comprehensive bibliographic database searches were conducted up to May 2024 to identify randomised controlled trials or any other prospective enzyme replacement therapy studies in patients with Pompe disease. Network meta-analyses of randomised controlled trials were undertaken to estimate indirect treatment effects for forced vital capacity % predicted and the 6-minute walk test. Other studies were summarised using narrative synthesis. RESULTS: The review included 60 studies: 38 on enzyme replacement therapy and 22 on best supportive care. Enzyme replacement therapy studies comprised 3 randomised controlled trials, 3 randomised controlled trial extensions, 7 registry studies and 25 single-group prospective studies. Two randomised controlled trials had a high risk of bias. Best supportive care studies included 14 longitudinal and 8 cross-sectional studies. In the network meta-analyses, after approximately 1 year, enzyme replacement therapy-naive patients showed significant 6-minute walk test improvements versus placebo: ~25 m with alglucosidase alfa and ~54 m with avalglucosidase alfa. No significant differences were found for forced vital capacity % predicted or comparisons with cipaglucosidase alfa, although very few enzyme replacement therapy-naive patients taking cipaglucosidase alfa were available for inclusion in the analyses. Intra-enzyme replacement therapy comparisons showed a significant 6-minute walk test advantage for avalglucosidase alfa. However, a sensitivity analysis adjusting for skewed data revealed no significant differences. Long-term enzyme replacement therapy effectiveness was assessed in single-group studies, showing initial gains maintained for 1-3 years, followed by gradual 10- to 15-year declines in 6-minute walk test and forced vital capacity % predicted. However, small sample sizes and missing data introduce uncertainty. Long-term evidence on best supportive care is limited, with most of the evidence focused on characterising basic demographic information and support needs. A small number of studies reported declines in forced vital capacity % predicted. Formal comparisons with long-term enzyme replacement therapy studies were not possible, but declines appear to be similarly gradual. CONCLUSIONS: The network meta-analyses shows enzyme replacement therapy modestly improves 6-minute walk test and forced vital capacity % predicted after 1 year versus placebo in enzyme replacement therapy-naive patients. However, there is limited evidence to suggest meaningful differences in outcomes between alglucosidase alfa, avalglucosidase alfa and cipaglucosidase alfa with miglustat. Observational data suggest declines beyond 2-3 years, lasting up to 15 years. Long-term comparative effectiveness remains uncertain, as does enzyme replacement therapy's impact on disease progression and supportive care needs. FUNDING: This article presents independent research funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis programme as award number NIHR161219
Psychological interventions for women with polycystic ovary syndrome (PCOS): a systematic review and meta-analysis of randomised and non-randomised controlled trials
Get PDFPolycystic ovary syndrome (PCOS) is an endocrine condition affecting 8–13% of reproductive-aged women globally. Psychological features of PCOS are often overlooked despite their association with mental health complications. This systematic review synthesises existing evidence of psychological interventions for women with PCOS. Database searches returned 4982 articles, of which 20 papers were eligible; 12 studies were meta-analysed. Compared to control, psychological interventions had statistically beneficial effects on change from baseline values for depression, PCOS-specific quality of life, general health, and body image. Significant improvements were found in all PCOS Questionnaire (PCOSQ) domains except acne, yet the importance of these differences in clinical practice was indeterminable. Despite statistical effects, the quality of evidence was judged as low/very-low due to between study heterogeneity, risk of bias, and imprecision in effect estimates. Future studies should focus on rigorously designed, well-reported trials, in order to address the uncertainty around the effectiveness of psychological interventions. The protocol of this systematic review was prospectively registered on the International Prospective Register of Systematic Reviews (PROSPERO: CRD42023472417)
The association between gambling frequency and risk of harm: analysis using health survey data from England and Scotland
Get PDFBackground and aims
Health economic models can be used to assess the effectiveness and cost-effectiveness of public health policies for gambling. To develop such a model, we must understand how gambling behaviour is associated with risk of experiencing gambling-related harms. This study aimed to: (1) assess the strength of association between gambling frequency and the risk of gambling-related harm and to examine how these associations differ when lottery-only players are excluded; (2) apply the study's findings in a hypothetical policy model aimed at reducing gambling frequency.
Design
Observational study using six waves of cross-sectional data from the Health Survey for England and the Scottish Health Survey.
Setting
Survey conducted in England in 2015, 2016 and 2018 and Scotland in 2015, 2016 and 2017.
Participants
The sample included 16 648 adults (aged 18 and over) who reported gambling in the past year, generally representative of the populations of England and Scotland.
Measurements
Gambling frequency was measured using 6 categories which indicated frequency in the past 12 months: (a) 2 or more times a week; (b) once a week; (c) less than once a week, more than once a month; (d) once a month; (e) every 2–3 months; (f) once or twice a year. Risk of gambling-related harm was assessed using Problem Gambling Severity Index (PGSI) score (0–27) and its four categories: no-risk (0), low-risk (1–2), moderate-risk (3–7) and high-risk (≥8). Control variables included age, sex, deprivation, social grade, presence of mental disorder and frequency of drinking alcohol.
Findings
Using multinomial logistic regression and zero-inflated negative binomial models we found that gambling at least twice weekly was associated with a statistically significantly higher PGSI score than gambling once or twice a year (incidence rate ratio = 3.528, 95% confidence interval = 2.040–6.103, P value < 0.001). Reducing gambling to guideline levels for people gambling at least twice weekly moved 10% of the sub-sample from higher PGSI categories (low, medium and high risk) to the no-risk category and shifted the distribution of PGSI scores down.
Conclusions
There appears to be a statistically significant association between gambling frequency and risk of gambling-related harm. Data derived from this and similar analyses can be used to model gambling policies which impact gambling frequency
Does extracellular vesicle specificity truly exist?
Get PDFExtracellular vesicles (EVs) are nano-sized membranous particles secreted by nearly all cell types that facilitate intercellular communication through the transfer of bioactive cargo. Growing evidence suggests that EVs may exhibit targeting specificity toward particular cells, yet the mechanisms remain poorly understood. In this review, we critically examine the concept of EV target specificity by outlining three potential stages where it may arise: (1) EV binding to the recipient cell membrane, (2) internalization and cargo release and (3) the induction of functional responses within recipient cells. We further explore how EV-intrinsic properties, recipient cell characteristics, and the surrounding microenvironment collectively shape these interactions. A key challenge in the field is the frequent assumption that EV uptake equates to functional relevance. In reality, many internalized EVs are recycled or degraded in lysosomes without eliciting any measurable effect. We therefore propose a refined definition of EV specificity, emphasizing that functional outcomes should serve as the central criterion for establishing meaningful EV specificity. Rather than offering definitive answers, this review highlights unresolved questions and calls for deeper investigation into how EVs select their target cells. Understanding these fundamental principles of EV function is critical for advancing the clinical and therapeutic application of EVs
Statistical relations among the CORE-10, PHQ-9 and GAD-7 in an English urban community counselling and psychotherapy clinic
Get PDFBackground
Three widely used brief self-report symptom intensity measures used to assess clients seeking psychological therapy are the Patient Health Questionnaire-9 (PHQ-9), which focuses on depression, the Generalised Anxiety Disorder-7 (GAD-7), which focuses on anxiety, and the Clinical Outcomes in Routine Evaluation-10 (CORE-10), which focuses on general distress.
Aim
This study aimed to examine the statistical relations among the PHQ-9, GAD-7 and CORE-10, their level of inter-agreement and ability to predict clinical caseness.
Method
All three measures were administered contemporaneously to 1103 clients seeking psychological therapy at a community clinic in England. Data were anonymised and analysed following ethical guidelines. The data analysis plan was preregistered via the Open Science Framework.
Results
Correlations among the three measures ranged from r = 0.71 to 0.78, p < 0.001. The percent of clients meeting caseness criteria was highest for the CORE-10 (87.3%), followed by the GAD-7 (74.8%) and the PHQ-9 (71.0%). Agreement between pairs of measures ranged from fair to moderate (κ = 0.45 to 0.59), with the 3-way agreement also moderate (κ = 0.58). Structural Equation Modelling indicated these scales measured a common latent construct of psychological distress. The GAD-7 showed a modest ceiling effect, whereas the CORE-10 and PHQ-9 did not.
Conclusions
The high level of overlap among the three measures suggests they may not distinguish sharply among types of distress. Pending future repeated-measures research on the instruments' sensitivity to change, these findings suggest a single measure may suffice as a baseline measure of distress.
Implications for Policy and Practice
The high overlap among the CORE-10, PHQ-9 and GAD-7 suggests that services could simplify assessments by using a single measure of psychological distress, reducing client burden and administrative costs.
The CORE-10, with its broader scope and higher caseness prevalence, may be particularly suited for community and non-specialist settings, offering a comprehensive screening tool for diverse psychological difficulties.
Adjusting cut-off scores for measures such as the PHQ-9 and GAD-7 to align with the CORE-10 may improve consistency in identifying clinical caseness, facilitating standardised care pathways and outcome comparisons.
Policymakers and researchers might consider endorsing or developing integrative tools (e.g., PHQ-ADS) to encompass multiple domains of distress, leveraging the common underlying construct identified in this study.
This research aligns with the NHS Talking Therapies programme's goal of evidence-based assessments and may support recommendations for adopting measures like the CORE-10 to capture broader psychological impacts alongside anxiety and depression
Spatial Chirp-based Joint Optimization for Analog Beamforming in Reconfigurable Intelligent Surface Assisted Terahertz Ultra-wideband Systems
Get PDFThis paper proposes a spatial chirp-based joint optimization method for analog beamforming in RIS-assisted THz ultra-wideband communication systems. The THz band (0.1-10 THz) offers significantly wider bandwidth, enabling Tbps data rates, which are crucial for 6G communications. However, traditional phase shifters are optimized for a single frequency. In ultra-wideband systems, the phase response deviates as frequency varies, leading to beam squinting problem, which significantly degrades system performance. Existing solutions, such as true-time-delay architectures, impose excessive complexity, making them impractical for THz systems. In this work, we propose leveraging polynomial expansion to optimize the spatially dependent phase functions of RIS, Tx, and Rx. We formulate the optimization problem as maximizing system throughput, and develop a gradient ascent-based iterative algorithm to solve it. Simulation results demonstrate that our method outperforms conventional and state-of-the-art approaches in metrics including phase response, throughput, and BER, providing a cost-effective solution for optimizing analog beamforming
From interaction to competency: the mediating role of social engagement in the emergence of materials-use competency among pre-service EFL teachers
Get PDFDespite increasing attention to teacher professionalism, little is known about the micro-level classroom processes through which pre-service EFL teachers develop materials-use competency, especially in resource-constrained contexts where lecture materials dominate. Grounded in sociomaterialism, this study examines how social engagement during lecture-initiated interactions mediates the emergence of pre-service EFL teachers’ materials-use competency. The dataset comprises 1,770 student responses across 28 classes in a teacher education module in Southwest China, analysed through two-round grounded coding, multimodal conversation analysis, and thematic analysis of instructor and student interviews. Three patterns of interactional entanglement (low, medium, high) were identified. These patterns shaped the conditions for dialogic learning, and it was particularly high-quality social engagement that mediated whether pre-service teachers transformed lecture discourse into situated instructional resources, enabling interactive designs. Structural and interactional constraints, including multimodal asymmetries and superficial translingual interactions, limited the occurrence of high-quality social engagement, thereby constraining the emergence of materials-use competency. The study contributes to sociomaterial perspectives in teacher education by showing how interactional entanglement and social engagement jointly shape the emergence of materials-use competency. Findings highlight the need to foreground social engagement in fostering such competency in teacher preparation
An exploration of patient and family carers’ views of dietary intervention for psychosis care and management
Get PDFPsychosis greatly impacts affected individuals and their families. Current pharmacological treatments are not fully efficacious and are associated with a range of metabolic side-effects. As a result, patients and their families seek alternative interventions, including managing their diet. Psychosis treatment guidelines recommend dietetic support as part of psychosis treatment, however this has not become embedded within routine practice. The aim of this study was to determine the views of patients and their families on the value of dietary intervention in their psychosis care and management. Study participants (n = 14) were either accessing psychosis treatment services (n = 8) or were familial carers (n = 6). Data were collected via individual semi-structured interviews and were thematically analysed resulting in four themes: 1) ‘What constitutes a healthy diet?’, 2) ‘The connection between diet, the brain and symptoms’, 3) ‘Empowerment gained from finding my own self-management strategies’ and 4) ‘How we envision dietary management for psychosis’. Findings suggest participants would value having access to a diet intervention to aid their management of psychosis, delivered by knowledgeable health care practitioners within mental health services. Participants wanted the dietary intervention to be evidence-based, accessible and have outcomes which including improved dietary knowledge and skills. The principal recommendation following this research is to further develop a dietary intervention for psychosis care and management that is co-designed with health staff, people with psychosis and their familial carers, mental health service commissioners and academics responsible for educating health care practitioners' curricula
Patient and public perspectives on the availability of their health and advance care planning information to support care at the end of life: a mixed-methods questionnaire study
Get PDFObjective To explore patient and public views and experiences of health professional access to patient health records and advance care planning information to support care at the end of life.
Design A cross-sectional national online survey of patients and the public using a convergent-parallel approach.
Setting The survey was distributed across the UK by Compassion in Dying and promoted via newsletters and social media channels of the Professional Records Standards Body and NHS England’s digital workstream network. These partners were purposively selected for their active involvement in end-of-life care, including hospices, clinicians and related charities.
Participants A total of 1728 participants from 103 UK counties responded, including people with a terminal condition (n=33), with long-term condition (n=442), who provide or have provided care to a person with a long-term or terminal illness (n=229) and who identified as healthy and interested in planning for the future (n=1024).
Measures Both quantitative data (multiple-choice responses and numerical ratings) and qualitative data (open-ended comments) asking about experiences and views of access to their health and advance care planning information to support their care at the end of life.
Results Confidence that recorded care preferences would be accessed when needed was low for carers (median=2, IQR 1–4) and moderate for patients (median=3, IQR 1–4). Four themes derived from free-text responses included (1) experience of sharing health information; (2) preparation, communication and understanding; (3) concerns, unknowns and assurance seeking; and (4) preserving dignity and respect: understanding individual contexts.
Conclusions Respondents acknowledged the opportunity for digital systems to enable access to health and advance care planning information but expressed doubts that professionals would retrieve it when needed, citing past failures. Confidence in record accuracy could be strengthened by patient and carer access. Future research should examine whether such access improves alignment of care with patients’ wishes
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