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PURPOSE: Health-related quality of life (HRQoL) measures in the form of health utilities are valuable for economic evaluations of the effectiveness of food allergy interventions. However, traditional HRQoL instruments lack the sensitivity to generate health utilities that capture the impact that food allergies, such as peanut allergies, may have on children’s mental health and daily activities. This study used mapping and discrete choice experiment (DCE) methods to generate health utilities from the Food Allergy Quality of life Questionnaire–Parent Form (FAQLQ-PF), which were then applied to clinical trial data.
PATIENTS AND METHODS: Health utilities (HU) were generated using two methods: mapping and DCE. Parents of children with peanut allergies (N=159) completed the FAQLQ-PF and EQ-5D-Y-Proxy-1 questionnaires. Mapping algorithms were developed once the FAQLQ-PF responses were mapped onto the EQ-5D-3L utilities. A composite DCE with time trade-off and a vignette was conducted among parents without peanut allergic children (N=767). The utilities derived from the mapping and DCE methods were applied to clinical trial data (PEPITES and PEOPLE) for an epicutaneous peanut patch (DBV712).
RESULTS: The mapping algorithm showed an association of 0.199 between FAQLQ-PF and EQ-5D-3L utilities. The DCE disutilities were highest for severe food-related anxiety, emotional distress, and social limitations. Once applied to the clinical trial data, the HU derived from the mapping algorithm demonstrated statistically significant HRQoL improvements for the intervention group at 36 months. Using the DCE-derived utilities, statistically significant HRQoL improvements for the intervention group were demonstrated at both 24- and 36-months. The effect size analysis demonstrated that the DCE-derived utilities were more responsive than mapped utilities.
CONCLUSION: DCE-derived utilities demonstrated greater responsiveness to changes in HRQoL compared with mapped utilities, suggesting their potential use in economic evaluations and HTA submissions for peanut allergy interventions. The DCE-derived health utilities showed greater sensitivity to changes in HRQoL. These utilities can be used in health technology assessments to better capture the impact of peanut allergy treatments on children’s quality of life.
BACKGROUND: Lung cancer is a leading cause of cancer death, and smoking-related disease is a major cause of health inequality in England, driven by increased prevalence of smoking in deprived areas. Integrating smoking cessation support into the English Lung Cancer Screening (LCS) programme may optimize services and has been found cost-effective. However, limited evidence is available on its health equity impact.
AIM: The objective was to conduct an aggregate distributional cost-effectiveness analysis (DCEA) of providing smoking cessation as part of LCS compared with usual care (referral to stop smoking services) to estimate its impact on health inequalities and health-related social welfare in England.
METHODS: The DCEA used an aggregate approach, stratifying people who smoke, aged 55–74 who were attending LCS by Index of Multiple Deprivation (IMD) quintiles. Discounted incremental costs and quality-adjusted life years (QALYs) were derived from a Markov model, adapted from previous NICE guidelines. The base case analysis applied a Health Opportunity Cost (HOC) of £15,000 per QALY and an Atkinson inequality aversion parameter (IAP) of 6.5, assuming a equal distribution across groups for intervention uptake.
RESULTS: Under all base case assumptions, smoking cessation provided as part of targeted LCS had a positive net health benefit and health equity impact. The intervention dominated usual care, resulting in a total net health benefit of 142,035 QALYs. The distribution of benefit strongly favored the most deprived quintile (IMD1: 34,863 QALYs vs. IMD5: 23,612 QALYs), driven by smoking prevalence.
CONCLUSION: Reforming smoking cessation services as part of LCS would likely improve both population health and health equity. Uptake of smoking cessation is a key determinant of the impact on health inequalities, underscoring the importance of targeted implementation strategies for optimizing population health, while reducing health inequalities.
Undertaking health economic modelling to compare in vitro diagnostics (IVDs) with imaging tests in health technology assessment (HTA) is associated with several challenges. Ignoring these challenges can lead to inaccurate and misleading results. This research identified common challenges and developed practical recommendations for considering them. An iterative, multi-methods approach was taken, including a methods review of HTA submissions, journal articles and published reports; a workshop; and semi-structured interviews with experts in health economic modelling of diagnostics. Key challenges and potential solutions were identified in the methods review and expanded in the workshop. Interviews were conducted to validate and refine challenges and practical recommendations. Recommendations were scored (from 1 [strong disagreement] to 5 [strong agreement]) by experts before and after the interviews. Final recommendations had a median score ≥ 4 (post-interview). 85 challenges (from the methods review) were prioritised into 21 with 57 recommendations after the workshop. After the interviews, recommendations were categorised into those for overcoming general challenges or challenges arising from specific aspects of the value proposition. Overall, 19 challenges with 30 recommendations are presented. To our knowledge, this is the first study investigating challenges to health economic modelling when comparing IVDs with imaging tests in HTA. The recommendations cover several topics, including variability in the diagnostic pathway, uncertain data or lack of evidence, incidental findings, and capacity constraints. Our recommendations present non-binding, guiding principles for good practice. Researchers should judge the applicability of each challenge and consider those recommendations carefully.
OBJECTIVES: Gastroesophageal reflux disease (GERD) is estimated to affect up to one-quarter of the Italian population, generating immense treatment costs. An emergent surgical treatment involving implantation of a nonactive device, RefluxStop, is offered at a number of hospitals in Italy and several other European countries. Published clinical outcomes of this device from the Conformité Européenne (CE)-mark clinical trial and real-world settings are consistently encouraging and with a favorable safety profile. This study evaluated the cost-effectiveness of RefluxStop compared with the current medical and surgical standards of care in Italy.
METHODS: A Markov model recently developed for use in the United Kingdom was adapted to the Italian healthcare payer’s perspective, covering the GERD treatment field. The model incorporated a lifetime horizon, one-month cycle length, and a 3% annual discount rate. Standards of care to be compared to the RefluxStop procedure included proton pump inhibitor (PPI)-based medical management, laparoscopic Nissen fundoplication, and magnetic sphincter augmentation (MSA). The mutually exclusive health states of the model comprised PPI use, PPI relapse, follow-on surgery, reoperation, high-dose PPI, Barrett’s esophagus, esophageal cancer, and death. The model included adverse events (AEs) associated with PPIs and surgeries and quantified the benefits in terms of quality-adjusted life years (QALYs). Unit costs were derived from the Italian diagnosis-related group (DRG) tariffs and the literature. Additional clinical efficacy data on standard-of-care treatments were derived from published literature. Model uncertainty was evaluated using deterministic and probabilistic sensitivity analyses.
RESULTS: RefluxStop demonstrated a lifetime cost difference relative to PPIs, Nissen fundoplication, and MSA of €8,788, €5,312, and €376 per patient, respectively. The corresponding QALY gains per patient were 2.87, 0.79, and 2.20, respectively. Base-case analysis established corresponding incremental cost-effectiveness ratios (ICERs) of €3,067, €6,712, and €171 per QALY gained, against PPIs, Nissen fundoplication, and MSA, respectively. At a cost-effectiveness threshold of €50,000 per QALY gained, RefluxStop was predicted to be cost-effective against both PPIs and MSA with a probability of 100% and against Nissen fundoplication with a probability of 97%. Sensitivity analysis confirmed the robustness of the model.
CONCLUSION: The implementation of RefluxStop in the Italian national health service is highly likely to be cost-effective compared to standard medical and surgical options for GERD, providing an opportunity for healthcare optimization in Italy.
BACKGROUND: Ig replacement therapy (IgRT) is used in the treatment of patients with Primary Immunodeficiency Disorders (PIDDs). There are some references to differences between preparations in terms of characteristics and efficacy, though comparative evidence is limited.
OBJECTIVES: To identify and map the evidence-base of clinical outcomes and adverse events of IgRT studies used for the treatment of patients with PIDDs and determine any signals of difference in treatment effect between different Ig brands.
METHODS: A pragmatic literature search was conducted in February 2023 to identify studies assessing the efficacy of IgRT in PIDDs and an assessment of the feasibility of indirect treatment comparisons (ITC) was carried out. Annualized outcome data were presented in visualization plots and possible outlier results identified through naïve (unanchored, unadjusted) comparisons; results were considered outliers when no overlap in confidence interval was identified.
RESULTS: After single-reviewer screening, 103 studies were included; 70 prospective studies were prioritized for extraction. A feasibility assessment found that ITC was not possible. Few outlier results for any particular commercial IgRT brand were identified across the outcomes considered, and those that were identified may have been due to differences in study methods or intervention characteristics rather than differing efficacy.
CONCLUSIONS: Limited evidence on the comparative efficacy of different IgRT brands was identified. Reporting in studies of IgRT for PIDDs was found to vary widely, such that ITC was not possible. We recommend improving reporting to enable such comparisons in future, including suggestions on improving or standardizing reporting of patient and study characteristics, outcome definitions and follow-up duration and measures of variance.
BACKGROUND: Healthcare resource utilization (HCRU) costs in those on conventional systemics for mild-moderate psoriasis are poorly described.
OBJECTIVES: This study aimed to describe HRCU, disease severity and health-related quality of life in patients with mild-moderate psoriasis requiring systemic therapy.
METHODS: UK single-center retrospective longitudinal cohort study including adults with mild-moderate psoriasis (PASI < 10, no historical PASI ≥ 10, no prior biologics) on conventional systemic therapy with 3-year data capture from first PASI recording (2014–2019, pre-COVID). Patients discontinued due to reaching PASI ≥ 10, starting biologics or being lost to follow-up.
RESULTS: The median annual HCRU cost was £1923 (mean £3361), largely driven by visit costs. A total of 50.8% patients achieved a PASI ≤ 2 and 30.6% achieved PASI ≤ 2 and DLQI ≤ 5 during follow-up. The difference between the maximum and minimum PASI for a patient and follow-up time were statistically significant predictors of total costs (p < 0.05).
CONCLUSION: Despite high healthcare costs, nearly half of the patients did not achieve clear/nearly clear skin. These data, in the context of reducing costs for biosimilars, may provide a basis to challenge care pathways and access criteria for ‘high-cost’ treatments.
BACKGROUND: The aim of this study was to model the cost-effectiveness of continuous pressure monitoring (CPM) in a hypothetical cohort of patients with a fracture of the tibial diaphysis.
METHODS: Decision-tree model structure was utilised to capture 60-day costs and health related quality of life (HRQoL) following a fracture of the tibial diaphysis. Depending on the effectiveness of the diagnostic method, patients were allocated to one of six validated outcome grades for acute compartment syndrome (ACS). Mean costs, utilities, and hospital length of stay were applied to each of these grades. Parameter inputs and costs were obtained from UK-specific published literature where possible. Analysis was performed from the UK National Health Service (NHS) perspective to determine the estimated impact in a hypothetical cohort of 1,000 patients with a tibial diaphyseal fracture using CPM versus current standard diagnostic methods for ACS.
RESULTS: Base case results indicated that the utilisation of CPM was associated with significant cost savings of £452/patient when compared with the standard care of clinical assessment and/or single point pressure measurement (SPPM). An associated additional 2733 NHS bed days were saved. Incremental quality-adjusted life year (QALY) gain of 0.02 was found, resulting in a dominant incremental cost effectiveness ratio (ICER). When analyses were performed considering different diagnostic outcome grade distributions and for a varying time horizon, CPM was found to have both increased cost savings and cost-efficacy.
CONCLUSION: This early economic model suggests that CPM of patients with a tibial diaphyseal fracture could be a cost-effective diagnostic option, although more data is required in order to validate the model assumptions. However, given the established evidence demonstrating the superior diagnostic performance characteristics of CPM when compared to clinical symptoms and/or SPPM, this economic data adds further support to the use of CPM as an essential diagnostic adjunct in patients at risk of ACS.
