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BACKGROUND: Sjögren’s disease (SjD) is a multifaceted, systemic autoimmune disease with substantial clinical heterogeneity. The objective of this study was to conduct a qualitative expert opinion exercise to explore how SjD is assessed, evaluated and managed in international clinical practice.
METHODS: A qualitative research design was used to elicit the expert opinion of 8 clinicians. Two researchers interviewed each clinician individually. The results of the interviews were synthesised using thematic analysis, and draft definitions of key terms were prepared. A group workshop was held to discuss/validate the interview results and refine the working definitions.
RESULTS: The clinicians had extensive experience in managing SjD from different countries. Seven topics emerged as major themes: disease classification, disease activity, relevant subpopulations, disease severity, disease progression, disease remission, and unmet needs. For 4 of these topics, there was no consensus – particularly regarding definitions that could be used in a clinical setting. The terms “systemic involvement” and “extra-glandular symptoms” have similar meanings but lack consistent application between clinicians. There was a lack of consensus on what “severity” refers to – whether it is severity of disease damage, the nature of disease manifestations, the potential for irreversible damage, or level of symptoms. There were also conflicting opinions regarding whether patient perspectives should be incorporated in key definitions.
CONCLUSION: There is currently a lack of standard definitions specifically for daily practice, which may contribute to high variability in clinical assessment and management. The definitions proposed in this study represent initial working concepts and are intended as a first step to promote further discussion among SjD experts to facilitate a more unified routine evaluation of patients.
BACKGROUND: In 2024, the UK National Institute of Health and Care Excellence (NICE) recommended TriageHF alerts as an option for remote monitoring of patients with heart failure (HF) and a compatible cardiac implantable electronic device (CIED). Data on the cost-effectiveness of this approach has not been published. This research evaluates the cost-effectiveness of TriageHF Plus in public hospital settings, using data from the TriageHF Plus multicentre study (758 participants, NCT04177199).
METHODS: An economic model was developed to capture the lifetime cost and benefits of TriageHF Plus versus usual care, based on a site of 300 eligible patients. Analysis on individual patient-level data informed model efficacy and resource use parameters. EuroQol five-dimensional questionnaire data and unit costs were obtained from published, peer-reviewed literature and national databases respectively. Costs and benefits were discounted at 3.5% per annum to adjust future costs and benefits to present value.
RESULTS: In a site size of 300, TriageHF Plus was predicted to prevent 384 (363–405) hospitalisations over 5 years. In total, TriageHF Plus saved £3989 (£1812–£5563) per person-lifetime versus usual care and was more effective and cost-saving in 99.4% of simulations. Results were robust to changes in key input parameters. Modelling showed that to avoid one hospitalisation, 1.7 people would need lifetime access to TriageHF Plus.
CONCLUSION: The TriageHF Plus pathway is cost-effective for the remote monitoring of HF in patients with CIEDs.
Diabetes affects over 500 million people worldwide and contributes substantially to the environmental impact of health care, including carbon emissions and plastic waste. As healthcare systems globally aim to reduce their environmental footprint, there is a need to embed environmental sustainability into decision making and foster innovation in health and life sciences.
This commentary outlines the environmental sustainability challenges and opportunities across the diabetes care pathway, highlighting innovations that reduce the demand for healthcare resources and associated environmental impact. We discuss the current and potential role of health technology assessment (HTA) agencies in promoting more sustainable health systems, by incorporating environmental considerations into the value assessment of technologies. Several approaches, such as integrated and parallel evaluation, are emerging to support this aim, whereas HTA agencies increasingly consider parameters of environmental life cycle assessment (eLCA), a comprehensive framework for evaluating the environmental sustainability of technology. Although a framework is evolving, early implementation by HTA bodies, for example, in the United Kingdom, Thailand, Canada, and Italy, highlights growing momentum. Moreover, sustainability policies at government and health system levels are developing globally, signaling opportunities to incorporate environmental sustainability in HTA (ESHTA).
Given the scale of health care’s environmental footprint, large disease areas offer critical opportunities for sustainable action. Diabetes, with its growing global prevalence, presents a particularly suitable domain for piloting the integration of environmental sustainability into HTA.
BACKGROUND: English Local authorities are responsible for delivering the Healthy Child Programme (HCP), to improve the health and wellbeing of children. Despite the importance of prevention, economic evidence to guide investment is limited, particularly for school-aged children. This scoping review aimed to map UK-based economic evidence relevant to the HCP for children aged 5–19 years, to inform policy and highlight gaps in the evidence.
METHODS: We searched sixteen databases in December 2023 to January 2024, in addition to citation and key websites searching. Eligible studies included economic evaluations and return on investment analyses of interventions that could be potentially delivered by local authorities under the HCP. Two reviewers screened titles and abstracts, and extracted data on study characteristics, methods, and outcomes. We assessed risk of bias using the Economic Evaluation Bias (ECOBIAS) checklist, synthesised the available evidence descriptively, and presented findings using summary tables and an evidence gap map.
RESULTS: We included 39 economic analyses. The most commonly evaluated interventions focused on mental health (12 studies), followed by physical activity (9 studies), healthy eating (4 studies, of which 2 were in combination with physical activity), risk behaviours (4 studies), bullying (3 studies), obesity (2 studies), hearing or vision screening (2 studies), social workers in schools (2 studies) and sexual health, ADHD prevention and improving oral health (each one study). Ten studies reported an incremental cost-effectiveness ratio (ICER), five studies reported either a benefit–cost ratio (BCR) or incremental net monetary benefit (INMB), whilst others described quality-adjusted life years (QALYs), costs or willingness to pay.
In general, robust evidence of the value of these interventions was partial because the interventions and methods of analysis were heterogenous. School nurse delivered services were particularly underrepresented despite their central role in the HCP delivery.
CONCLUSIONS: This review highlights the diverse range of interventions available to local authorities and the lack of robust economic evidence to support prioritisation of investment as part of the HCP 5–19 years. Further research should focus on developing more robust evidence of the value of public health interventions in this age group, using economic analysis to enable priority-setting, and addressing the evidence gaps identified.
INTRODUCTION: Safety performance indicators are important to assess and prevent work-related incidents, which are associated with high global morbidity and mortality. Safety can be measured using lagging indicators (past workplace incidents) or leading indicators (proactive measures to prevent incidents). Leading indicators in occupational safety and health (OSH) are already used in several industries, though it is unclear whether their use is supported by published evidence.
METHODS: We undertook a scoping review to identify studies evaluating the performance of OSH leading indicators. Literature searches (8 databases, August 2023) identified primary studies evaluating the impact of leading indicators on any safety outcomes in any industry. Researchers systematically identified and extracted relevant studies. No two studies assessed the same research question, preventing meaningful synthesis of the performance of leading indicators. Instead, we mapped the available studies to understand the quality and nature of this important evidence base.
RESULTS: The 48 studies comprise a substantial, though disparate, evidence base. Although most reported a positive impact of leading on lagging indicators, overall the evidence base was weak: (1) study designs were not appropriate for determining causality; (2) internal validity of studies was moderate to low; (3) studies were poorly generalizable. The biggest challenge was the inability to compare findings across studies.
CONCLUSIONS: Decisions on which leading indicators to use should be based on evidence that they improve safety. Future research should take measures to improve and standardize study design and conduct. Developing an OSH-specific evidence standards framework is likely to guide and assist this process. Practical applications: Identifying recommendations for practice was difficult due to the quality and heterogeneity of the evidence base. Future studies should improve approaches to minimizing bias and identify common tools to measure both leading and lagging indicators. To achieve this, we encourage practitioners and researchers to collaborate and share data.
BACKGROUND AND OBJECTIVE: Efanesoctocog alfa is a first-in-class high-sustained factor VIII therapy approved for prophylaxis, on-demand treatment, and peri-operative management of bleeding in hemophilia A. This analysis aimed to compare the cost effectiveness of efanesoctocog alfa prophylaxis with factor VIII extended half-life prophylaxis.
METHODS: A lifetime Markov model was developed from a US payer perspective, using clinical data from an indirect treatment comparison of phase III studies and inputs derived from the literature. A cohort of patients aged ≥ 12 years with severe hemophilia A without inhibitors, who received prophylaxis using any regimen or on-demand treatment, entered the model. Outcomes included joint and non-joint bleeds, quality-adjusted life-years, total direct costs, and the incremental cost-effectiveness ratio. Costs were expressed in US dollars and inflated to January 2023 prices. Discount rates of 3% were used. One-way probabilistic and scenario analyses were conducted. The willingness-to-pay threshold was assumed at $150,000 per quality-adjusted life-year.
RESULTS: Efanesoctocog alfa was more effective and less costly (also referred to as ‘dominant’) versus factor VIII extended half-life therapies, with a lower lifetime number of joint (undiscounted 34.00 vs 140.65) and non-joint (undiscounted 13.33 vs 55.99) bleeds, higher quality-adjusted life-years (24.00 vs 22.92), and lower total costs ($30,716,640 vs $32,953,485). A broad range of scenario analyses and probabilistic sensitivity analyses resulted in 100% of simulations being cost effective. Dosing level and drug costs had the largest impact on results in the deterministic sensitivity analyses.
CONCULSIONS: Our analysis suggests that efanesoctocog alfa was dominant versus prophylaxis with standard and elevated factor VIII extended half-life dosing regimens. Efanesoctocog alfa was associated with better joint health and, hence, contributed to fewer bleeds, lower costs, and higher quality-adjusted life-years.
BACKGROUND: Heartburn, chest pain, and regurgitation are characteristics of gastroesophageal reflux disease. The standard treatment in Norway is proton pump inhibitors (PPIs), but surgical alternatives such as Nissen fundoplication are pursued when medical management proves ineffective. RefluxStop is an implantable device presenting a new approach to restoration of the antireflux barrier. The aim of this study is to evaluate the cost-effectiveness of RefluxStop against Nissen fundoplication and PPI-based medical management (MM) in Norway.
METHODS: A Markov model was adapted from a published cost-effectiveness analysis developed for the UK NHS assessing the cost-effectiveness of RefluxStop in comparison with PPI-based MM and Nissen fundoplication. The perspective of the Norwegian healthcare payer, lifetime horizon, one-month cycle length, and a 4% annual discount rate for costs and health-benefits were used. Health states included in the model were initial MM, MM relapse, follow-on surgery, reoperations, MM with a higher dose, Barrett’s esophagus, esophageal cancer, and death. Adverse events related to MM and surgeries were included with benefits measured in quality-adjusted life-years (QALYs). Norwegian diagnostic-related group tariffs and literature were used as sources for unit costs. Clinical efficacy data for RefluxStop was based on an ongoing clinical study and comparator treatments were informed by published studies. Deterministic and probabilistic sensitivity analyses were used to address uncertainty of the parameter estimates.
RESULTS: Compared to PPI-based MM and Nissen fundoplication, the base case incremental cost-effectiveness ratios (ICERs) of RefluxStop were NOK 68,262 and NOK 79,543 per QALY gained, respectively. The results of the model were robust to variation in individual inputs with exception of monthly failure rate of RefluxStop relative to Nissen fundoplication. RefluxStop demonstrated a significantly high probability of cost-effectiveness at the cost-effectiveness threshold of NOK 275,000 per QALY gained, with probabilities of 100% and 92% against PPI-based MM and Nissen fundoplication, respectively.
CONCLUSION: The RefluxStop device is highly likely to be cost-effective compared to PPI-based MM and Nissen fundoplication in Norway. Further study is required to assess the real-world long-term safety and efficacy of this novel device.
