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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.
BACKGROUND: Respiratory syncytial virus (RSV) causes lower respiratory tract infections (LRTIs) in infants, leading to substantial morbidity and mortality. Nirsevimab, a long-lasting monoclonal antibody, has been demonstrated to reduce RSV-related outcomes in randomized controlled trials (RCTs) and real-world settings. The object was to review the existing real-world evidence (RWE) on the effectiveness of nirsevimab in preventing RSV-LRTIs.
METHODS: Searches of six databases in addition to trial registries, HTA/regulatory agency webpages and conference abstracts were conducted in November 2024. Observational studies evaluating nirsevimab prophylaxis in infants during their first RSV season or high-risk infants in their second season were included. For outcomes evaluated by more than one study, feasibility assessment was conducted and, where appropriate, studies were combined in meta-analyses.
RESULTS: Sixteen studies reporting effectiveness outcomes were included. The studies were conducted across four countries (Spain, Italy, US and France), and included 141,550 infants. Nirsevimab showed significant effectiveness for preventing RSV-LRTI hospitalization (reduction in risk from hazard ratios and odds ratios of 84.5%; 95% CI: 73.6%−90.9%; I2: 0 and 73.7%; 95% CI: 42.3%−88.0%; I2: 0%, respectively), intensive care unit (ICU) admission (85.9%; 95% CI: 13.2%−97.7%) and ventilatory support (87.1%; 95% CI: 70.2%−94.4%). Nirsevimab was also effective in preventing RSV-LRTI visits in primary care (75.8%; 95% CI: 40.4%−92.7%) and emergency departments (87.9%; 70.3%−95.1%).
CONCLUSIONS: Real-world evidence confirms the effectiveness of nirsevimab against RSV-LRTIs and underlines the public health impact of the intervention in preventing RSV-related health outcomes. There is no clear evidence that effectiveness differs for infants born in season (at birth immunization) compared with out of season groups, or that effectiveness varies in preterm infants compared to healthy term infants.
BACKGROUND/OBJECTIVE: Sickle cell disease (SCD) is a group of inherited health conditions affecting 7.74 million people worldwide. Regular automated red blood cell exchange (aRBCX) transfusions have been shown to improve control and management of SCD compared with manual RBCX (mRBCX). The aim of this study was to estimate the lifetime clinical and economic impact of aRBCX versus mRBCX in two United Kingdom-based populations with SCD (paediatrics initiated aged 5 years and adults initiated aged 38 years) that were clinically indicated for chronic disease-modifying transfusions (DMTs).
METHODS: An individual patient-level simulation model was developed to estimate lifetime quality-adjusted life years (QALYs) and healthcare costs. DMT administration programmes aligned with recommended treatment schedules. Monte Carlo methods determined baseline characteristics and clinical event occurrence. Pragmatic review findings and expert opinion informed model parameters and assumptions. Second-order probabilistic sensitivity analysis (PSA) was performed for 1000 individuals’ lifetimes over 500 iterations.
RESULTS: Per individual, aRBCX reduced acute clinical events by 19% in both populations versus mRBCX. The time spent receiving chelation therapy reduced by 63 and 32 months for paediatric-initiated and adult-initiated individuals, respectively. Total lifetime DMT costs were reduced by £71,217 and £30,740 for paediatric-initiated and adult-initiated individuals, respectively. Overall, aRBCX increased QALYs and reduced costs by 0.29 and £112,811 in paediatric-initiated individuals and 0.24 and £61,895 in adult-initiated individuals. aRBCX was cost-effective in 100% of PSA iterations for both populations.
CONCLUSION: aRBCX shows potential to improve health outcomes and reduce healthcare costs for individuals with SCD initiating a chronic DMT programme.
BACKGROUND: Primary immunodeficiency diseases (PIDDs) are rare genetic disorders impairing immunity. Studies evaluating diagnostic rates of PIDDs in historically marginalized US populations are limited.
OBJECTIVE: To conduct a scoping review that identifies the extent of race and ethnicity reporting in US-based observational studies of people with PIDDs, and the demographic composition of study populations compared with the broader US population.
METHODS: We conducted pragmatic searches of MEDLINE in April 2024 and ultimately included studies dating back 10 years. Results were screened and extracted against prespecified eligibility criteria by a single reviewer. Included data were compared with US census data using χ2 tests.
RESULTS: We identified 126 publications publishing observational PIDD studies that report patient characteristics, 62 of which (49%) reported race or ethnicity data. After grouping for data source and PIDD type to avoid overlapping studies, 25 publications were prioritized for extraction. Of these, seven were fully compliant with current Food and Drug Administration–recommended reporting guidelines. The populations of the extracted studies were not statistically representative of the broader US population, with overrepresentation of non-Hispanic White patients.
CONCLUSIONS: Primary immunodeficiency disease cohort and other studies inconsistently report demographic data on patient race and ethnicity according to current Food and Drug Administration recommendations. Efforts to improve understanding of the prevalence, characteristics, and diagnostic rates of PIDD in different US populations (as well as differences among study populations and overall US demographics) would likely be facilitated by a greater effort toward comprehensive demographic reporting.
INTRODUCTION: The life cycle of health technologies contribute to air pollution, ecotoxicity, and resource depletion, impacting the environment and human health. Increasing healthcare resource use globally increases emissions that accelerate climate change and negatively affect the health of current and future generations. Health Technology Assessment (HTA) should inform decision makers to prioritize the adoption of technologies demonstrating value in terms of health benefits, costs, and other relevant dimensions such as environmental sustainability. This paper reports on a multistakeholder approach to guiding an international working group for Environmental Sustainability in Health Technology Assessment (ESHTA) that has been formed by Health Technology Assessment international.
METHODS: A multistakeholder online workshop was held with 32 participants in May 2024 to define the critical issues to be considered. The resulting report underwent consultation among the ESHTA members and in a broader group of 90 additional worldwide stakeholder representatives.
RESULTS: The workshop participants recognized defining frameworks, mechanisms, and tools for embedding environmental sustainability into HTA as an opportunity to support sustainable development and quality improvement in healthcare. Achieving this requires (1) consensus on what environmental sustainability in healthcare means, (2) reconcilement with other healthcare and environmental policies, and (3) methods that are useful and applicable within HTA frameworks.
CONCLUSION: This novel collaboration aims to align the global HTA community on the role of environmental sustainability in HTA. The report provides a path for the way forward for incorporating environmental sustainability into HTA based on broad perspectives from global multistakeholders.
