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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.
Digital symptom checkers (SCs) are increasingly used to support early symptom recognition and care-seeking, yet evidence on their cost-effectiveness remains limited. We conducted an economic evaluation of a digital SC for endometriosis, a prevalent but underdiagnosed condition, as a case study. We developed a Markov decision process model to compare the digital SC with the standard of care from a societal perspective. Over a 40-year horizon, the digital SC reduced diagnostic delay by 4.36 years, generated 0.049 quality-adjusted life years (QALYs) per person, saved $5196.22 in costs, and produced an incremental net monetary benefit (INMB) of $10,089.00 at a $100,000/QALY threshold. Probabilistic sensitivity analysis confirmed the robustness of these findings, with an INMB of $12,398.92 (95% CI: $11,893.11–$12,904.72). Scenario analyses showed that the SC remained cost-effective under a wide range of assumptions, with the greatest value realized when sensitivity and specificity were ≥0.7, compliance exceeded 45%, and a time horizon of at least 10 years. This study provides the first economic evaluation of a digital SC for endometriosis and illustrates when and how digital SCs can deliver value to patients and health systems.
PURPOSE: A number of measures have been proposed to evaluate meaningful within-person change in the Oxford Hip and Oxford Knee scores (OHS and OKS), however there is evidence of lower baseline scores being associated with higher change scores, that is, these instruments potentially demonstrate baseline dependency. The study aim was to identify and quantify the impact of baseline dependency for the OHS and OKS.
PATIENTS & METHODS: The data were collated from the National Health Service in England including the OHS, OKS, EQ-5D-3L and a global transition item (GTI). Change scores, including the minimally important change (MIC) were derived and categorised by the GTI and baseline scores for the OHS and OKS. Baseline dependency was evaluated using different baseline categories (OHS/OKS, EQ-5D quartiles and split-item method).
RESULTS: A total of 387,524 records were extracted. Although the overall MIC were in-line with previous research, the results showed these measures varied by pre-operative scores. Baseline dependency was present irrespective of the method employed to categorise change scores.
CONCLUSIONS: The MICs for both the OHS and OKS show distinct baseline dependencies. The use of a single MIC for either instrument is unlikely to capture the full range of meaningful change experienced by individual patients and therefore has implications for the interpretation of interventional outcomes with these instruments. A multifaceted approach involving multiple sources of patient-relevant measures is recommended to provide more robust measures for the evaluation of patient outcomes and healthcare services.
INTRODUCTION: Incorporating environmental sustainability into health technology assessment (HTA) is increasingly important for aligning with government targets, healthcare policies, and healthcare professional bodies endorsing the use of more sustainable health technologies, while maintaining clinical effectiveness and value for money. Although proposals exist to include environmental criteria in HTA, methodological development in this area remains limited. This study explored stakeholder perspectives on useful methods, frameworks and challenges for assessing environmental outcomes in HTA.
METHODS: Semi-structured interviews were conducted with eight stakeholders including HTA decision makers, industry representatives, sustainability professionals, and academics from different jurisdictions to ensure diverse views and cross-setting applicability. Data were analysed thematically to identify opportunities, barriers and facilitators for methodological development.
RESULTS: Stakeholders broadly supported integrating environmental outcomes into HTA, with carbon footprint highlighted as a key measurable and decision-relevant indicator. Environmental life cycle assessment (eLCA) was considered useful for quantifying emissions and identifying hotspots, but barriers included limited environmental data, lack of methodological consensus on scope and boundaries, and resource and expertise constraints. Broader environmental outcomes, such as impacts on resource use, biodiversity, and health, were also seen as important where evidence is available. Suggested approaches included carbon costing within economic evaluation, multi-criteria decision analysis (MCDA), expert consensus, and weighting of highly polluting technologies to address trade-offs. Stakeholders emphasised the need to understand societal preferences for environmental gains, including willingness to pay or trade-off health benefits.
CONCLUSIONS: This novel research provides the first qualitative insights into multidisciplinary stakeholder views on environmental sustainability in HTA, identifying both opportunities and challenges for methodological innovation and policy development.
BACKGROUND: The existing intravenous systemic anticancer therapy (SACT) pathway in pharmacies is operationally inefficient. Manual, paper-based workflows render the system prone to human error, and the need for time-consuming manual verification diverts pharmacy staff time. The introduction of an automated workflow solution for the intravenous SACT pathway could optimize treatment timeliness and improve oncological outcomes for patients, aligning with the National Health Service Long Term Plan for improved cancer care.
OBJECTIVE: This observational analysis aimed to assess the change in time, cost, and errors following the implementation of the Becton Dickinson (BD) Cato Pharmacy system in an aseptic unit producing intravenous SACT at Watford General Hospital.
METHODS: Data on compounding process times were collected manually by pharmacy staff before and after the implementation of the intravenous compounding software (BD Cato). The data were analyzed to estimate annual time savings, opportunity cost savings, and error reduction.
RESULTS: The intravenous compounding software produced a time saving of 18 (SD 9) minutes per drug, equating to 1034 hours saved per year (1034/2591, 39.9% reduction). If this time were repurposed to producing more intravenous SACT, Watford General Hospital could increase production by 66% (2298/3482) annually (2298 additional intravenous SACT). This represents an average cost saving of £11.29 (£1=US $1.273) per drug, equating to an annual opportunity cost saving of £39,246. The intravenous compounding software also decreased observed errors by 86% (43/50), a reduction of 43 errors over 2 months (approximately 258 fewer errors annually). Staff also preferred the intravenous compounding software to the manual system.
CONCLUSIONS: Implementing intravenous compounding software can save time, reduce costs, and lower errors in intravenous SACT preparation. This could improve timely treatment access for patients with cancer.
