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Respiratory diseases, including asthma and chronic obstructive pulmonary disease (COPD), are common in England with the worst respiratory outcomes observed in the most deprived areas. There is limited published research to establish whether the rate of oral corticosteroid (OCS) prescribing for asthma and COPD is linked to levels of deprivation. This study carried out a multivariable regression analysis of publicly available data and found that deprivation is associated with a statistically significant increase in the proportion of patients receiving an OCS prescription for asthma or COPD at a GP practice level (p < 0.001). The model estimated that the proportion of prescriptions is 1.88% (95% CI 1.83% to 1.92%) and 2.84% (95% CI 2.70% to 2.98%) for the least deprived GP practice and the most deprived GP practice, respectively. This study lays the groundwork for future research using individual patient level data to consider the impact of variation in OCS prescribing rates.
BACKGROUND: There is growing evidence to support the benefits of transcatheter aortic valve implantation (TAVI) over surgical aortic valve replacement (SAVR) in patients with symptomatic severe aortic stenosis (sSAS) who are at high- or intermediate-risk of surgical mortality. The PARTNER 3 trial showed clinical benefits with SAPIEN 3 TAVI compared with SAVR in patients at low risk of surgical mortality. Whether TAVI is also cost-effective compared with SAVR for low-risk patients in the Dutch healthcare system remains uncertain. This article presents an analysis using PARTNER 3 outcomes and costs data from the Netherlands to inform a cost-utility model and examine cost implications of TAVI over SAVR in a Dutch low-risk population.
METHODS: A two-stage cost-utility analysis was performed using a published and validated health economic model based on adverse events with both TAVI and SAVR interventions from a published randomized low risk trial dataset, and a Markov model that captured lifetime healthcare costs and patient outcomes post-intervention. The model was adapted using Netherlands-specific cost data to assess the cost-effectiveness of TAVI and SAVR. Uncertainty was addressed using deterministic and probabilistic sensitivity analyses.
RESULTS: TAVI generated 0.89 additional quality-adjusted life years (QALYs) at a €4742 increase in costs per patient compared with SAVR over a lifetime time horizon, representing an incremental cost-effectiveness ratio (ICER) of €5346 per QALY gained. Sensitivity analyses confirm robust results, with TAVI remaining cost-effective across several sensitivity analyses.
CONCLUSIONS: Based on the model results, compared with SAVR, TAVI with SAPIEN 3 appears cost-effective for the treatment of Dutch patients with sSAS who are at low risk of surgical mortality. Qualitative data suggest broader societal benefits are likely and these findings could be used to optimize appropriate intervention selection for this patient population.
The Consolidated Standards of Reporting Trials extension for Artificial Intelligence interventions (CONSORT-AI) was published in September 2020. Since its publication, several randomised controlled trials (RCTs) of AI interventions have been published but their completeness and transparency of reporting is unknown. This systematic review assesses the completeness of reporting of AI RCTs following publication of CONSORT-AI and provides a comprehensive summary of RCTs published in recent years. 65 RCTs were identified, mostly conducted in China (37%) and USA (18%). Median concordance with CONSORT-AI reporting was 90% (IQR 77-94%), although only 10 RCTs explicitly reported its use. Several items were consistently under-reported, including algorithm version, accessibility of the AI intervention or code, and references to a study protocol. Only 3 of 52 included journals explicitly endorsed or mandated CONSORT-AI. Despite a generally high concordance amongst recent AI RCTs, some AI-specific considerations remain systematically poorly reported. Further encouragement of CONSORT-AI adoption by journals and funders may enable more complete adoption of the full CONSORT-AI guidelines.
OBJECTIVES: This research aimed to develop best-practice recommendations for identifying the "standard of care" (SoC) and integrate it when it is the comparator in diagnostic economic models (SoC comparator).
METHODS: A multi-methods approach comprising 2 pragmatic literature reviews and 9 expert interviews was used. Experts rated their agreement with draft recommendations based on the authors' analysis of the reviews. These were refined iteratively to produce final recommendations.
RESULTS: Fourteen best-practice recommendations are provided. Care pathway mapping (using quantitative, qualitative, or mixed-methods approaches) should be used for identifying the SoC comparator. Guidelines analysis can be integrated with expert opinion to identify pathway variability and discrepancies from clinical practice. For integrating the SoC comparator into the model, recommendations around structure, input sourcing, data aggregation and reporting, input uncertainty, and model variability are presented. For example, modelers should consider that the reference standard is not synonymous with the SoC, and the SoC may not be the only comparator. The comparator limitations should be discussed with clinical experts, but elicitation of its diagnostic accuracy is not recommended. Probabilistic sensitivity analysis is recommended when evaluating the overall input uncertainty, and deterministic sensitivity analysis is useful when there is high model uncertainty or SoC variability. Consensus could not be reached for some topics (eg, the role of real-world data, model averaging, and alternative model structures), but the reported discussions provide points for consideration.
CONCLUSIONS: To our knowledge, this is the first guidance to support modelers when identifying and operationalizing the SoC comparator in diagnostic cost-effectiveness models.
Revascularization completeness after percutaneous coronary intervention (PCI) is associated with improved long-term outcomes. Mechanical circulatory support [intra-aortic balloon pump (IABP) or Impella] is used during high-risk PCI (HR-PCI) to enhance peri-procedural safety and achieve more complete revascularization. The relationship between revascularization completeness [post-PCI residual SYNTAX Score (rSS)] and left ventricular ejection fraction (LVEF) in HR-PCI has not been established. We investigated LVEF predictors at 90 days post-PCI with Impella or IABP support. Individual patient data (IPD) were analyzed from PROTECT II (NCT00562016) in the base case. IPD from PROTECT II and RESTORE-EF (NCT04648306) were naïvely pooled in the sensitivity analysis. Using complete cases only, linear regression was used to explore the predictors of LVEF at 90 days post-PCI. Models were refined using stepwise selection based on Akaike Information Criterion and included: treatment group (Impella, IABP), baseline characteristics [age, gender, race, New York Heart Association Functional Classification, LVEF, SYNTAX Score (SS)], and rSS. Impella treatment and higher baseline LVEF were significant predictors of LVEF improvement at 90 days post-PCI (p = 0.05), and a lower rSS contributed to the model (p = 0.082). In the sensitivity analysis, Impella treatment, higher baseline LVEF, and lower rSS were significant predictors of LVEF improvement at 90 days (p = 0.05), and SS pre-PCI contributed to the model (p = 0.070). Higher baseline LVEF, higher SS pre-PCI, lower rSS (i.e. completeness of revascularization), and Impella treatment were predictors of post-PCI LVEF improvement. The findings suggest potential mechanisms of Impella include improving the extent and quality of revascularization, and intraprocedural ventricular unloading.
OBJECTIVE: To conduct a systematic review of the effectiveness and safety of pharmacological treatments for adult-onset Still's disease (AOSD).
METHODS: Six databases, two trial registries and conference abstracts were searched from 2012 to February 2023 for studies of pharmacological interventions in people with AOSD. Outcomes were rates of remission and response, discontinuation of concurrent treatments, complications of AOSD and treatment-related adverse events. Risk of bias was assessed with the Cochrane RoB tool and the Joanna Briggs Institute tool for case series.
RESULTS: 44 studies evaluated treatments, including non-steroidal anti-inflammatory drugs (NSAIDs), corticosteroids, conventional synthetic disease-modifying anti-rheumatic drugs (csDMARDs) and biologic DMARDs (bDMARDs). For bDMARDS, tocilizumab, anakinra and canakinumab had the most available data. Although three randomised controlled trials did not show statistically significant benefits of bDMARDs, meta-analyses showed high rates of complete remission and corticosteroid discontinuation. Complete remission was 80% (95% confidence interval (CI): 59-92%, I2: 36%), 73% (95% CI: 58-84%, I2: 66%), and 77% (95% CI: 29-97%, I2: 82%) and corticosteroid discontinuation was 57% (95% CI: 29-81%, I2: 66%), 47% (95% CI: 18-78%, I2: 79%), and 34% (95% CI: 6-81%, I2: 59%), respectively, for tocilizumab, anakinra and canakinumab. Studies with a higher proportion of patients previously treated with bDMARDs showed a trend towards lower rates of corticosteroid discontinuation (p=0.05). The analyses had high clinical heterogeneity, largely because treatments were prescribed as different lines of therapy.
CONCLUSION: Evidence supports tocilizumab, anakinra and canakinumab therapy for AOSD. However, the magnitude of effect and comparative effectiveness of treatments is uncertain.
BACKGROUND: RSV is an important cause of lower respiratory tract disease (LRTD) in older adults and may cause substantial morbidity, mortality, and economic disruption. The Joint Committee on Vaccination and Immunisation (JCVI) advised introducing a one-off RSV vaccine into the national immunisation schedule for adults aged 75-80. The potential impact of mRNA-1345, an RSV vaccine, was explored for UK adults aged =65, aligned with current influenza vaccine recommendations.
METHODS: A decision-analytic model (Figure 1) was developed to estimate the clinical impact of mRNA-1345 compared with no vaccine over two-years from a UK perspective. Trial data (Wilson et al 2023) was used to populate mRNA-1345 efficacy in preventing RSV-LRTD, RSV-acute respiratory disease (ARD), and RSV-hospitalizations, projected over two-years. Table 1 shows the key inputs. Outcomes included RSV-case numbers, RSV-LRTD hospitalisations, numbers needed to vaccinate (NNV) to prevent these outcomes, incremental life years and quality-adjusted life-years (QALYs).
RESULTS: When targeting UK adults aged =65, compared to no vaccination, administering mRNA-1345 could result in a 33.8%, 38.6%, and 52.0% reduction in cases of RSV-ARD, RSV-LRTD, and deaths respectively. A reduction of 29,412 RSV-LRTD hospitalisations (52.0%) were estimated. Vaccination was estimated to save 95,556 QALYs and 121,885 life-years over two-years for a total population of 12,537,031. The NNV to prevent one RSV-LRTD case, one RSV hospitalisation and one death was 57, 426 and 755, respectively.
CONCLUSIONS: In UK adults aged =65, implementing mRNA-1345 could have a significant impact on reducing RSV-related morbidity and mortality, and as a result reduce healthcare resource use and improve population quality of life.
