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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.
BACKGROUND AND AIMS: Symptom control for atrial fibrillation can be achieved by catheter ablation or drug therapy. We assessed the cost effectiveness of a novel streamlined atrial fibrillation cryoballoon ablation protocol (AVATAR) compared with optimised antiarrhythmic drug (AAD) therapy and a conventional catheter ablation protocol, from a UK National Health Service (NHS) perspective.
METHODS: Data from the AVATAR study were assessed to determine the cost effectiveness of the three protocols in a two-step process. In the first stage, statistical analysis of clinical efficacy outcomes was conducted considering either a three-way comparison (AVATAR vs. conventional ablation vs. optimised AAD therapies) or a two-way comparison (pooled ablation protocol data vs. optimised AAD therapies). In the second stage, models assessed the cost effectiveness of the protocols. Costs and some of the clinical inputs in the models were derived from within-trial cost analysis and published literature. The remaining inputs were derived from clinical experts.
RESULTS: No significant differences between the ablation protocols were found for any of the clinical outcomes used in the model. Results of a within-trial cost analysis show that AVATAR is cost-saving (£1279 per patient) compared with the conventional ablation protocol. When compared with optimised AAD therapies, AVATAR (pooled conventional and AVATAR ablation protocols efficacy) was found to be more costly while offering improved clinical benefits. Over a lifetime time horizon, the incremental cost-effectiveness ratio of AVATAR was estimated as £21,046 per quality-adjusted life-year gained (95% credible interval £7086-£71,718).
CONCLUSIONS: The AVATAR streamlined protocol is likely to be a cost-effective option versus both conventional ablation and optimised AAD therapy in the UK NHS healthcare setting.
BACKGROUND: Gastro-oesophageal reflux disease (GORD) is a common condition associated with heartburn and regurgitation. Standard of care for GORD patients in the UK involves initial treatment with proton pump inhibitors (PPIs) and laparoscopic antireflux surgery in patients unwilling to continue or intolerant of long-term PPI treatment. Recently, RefluxStop™, a novel, implantable medical device, has proven to be an efficacious and cost-effective treatment for patients with GORD. The current analysis aimed to describe the budget impact of introducing RefluxStop™ within National Health Service (NHS) England and Wales.
OBJECTIVES: To estimate the more immediate, short-term clinical and economic effects of introducing RefluxStop™ as a therapeutic option for patients with GORD treated within NHS England and Wales.
METHODS: A model adherent to international best practice guidelines was developed to estimate the budget impact of introducing RefluxStop™ over a 5-year time horizon, from an NHS perspective. Two hypothetical scenarios were considered, one without RefluxStop™ (comprising PPI treatment, laparoscopic Nissen fundoplication, and magnetic sphincter augmentation using the LINX® system) and one with RefluxStop™ (adding RefluxStop™ to the aforementioned treatment options). Clinical benefits and costs associated with each intervention were included in the analysis.
RESULTS: Over 5 years, introducing RefluxStop™ allowed the avoidance of 347 surgical failures, 39 reoperations, and 239 endoscopic esophageal dilations. The financial impact of introducing RefluxStop™ was £3 029 702 in year 5, corresponding to a 1.68% increase in annual NHS spending on GORD treatment in England and Wales.
DISCUSSION: While the time horizon was too short to capture some of the adverse events of PPIs and complications of GORD, such as the development of Barrett's esophagus or esophageal cancer, the use of RefluxStop™ was associated with a substantial reduction in surgical complications, including surgical failures, reoperations, and endoscopic esophageal dilations. This favorable clinical profile resulted in cost offsets for the NHS and contributed to the marginal budget impact of RefluxStop™ estimated in the current analysis.
CONCLUSIONS: Introducing RefluxStop™ as a treatment option for patients with GORD in England and Wales may be associated with clinical benefits at the expense of a marginal budget impact on the NHS.
AIMS: Intraurethral catheter balloon inflation is a substantial contributor to significant catheter-relatedurethral injury. A novel safety valve has been designed to prevent these balloon-inflation injuries. The purpose of this evaluation was to assess the cost-effectiveness of urethral catheterisation with thesafety valve added to a Foley catheter versus the current standard of care (Foley catheter alone).
MATERIALS AND METHODS: The analysis was conducted from the UK public payer perspective on a hypothet-ical cohort of adults requiring transurethral catheterization. A decision tree was used to capture outcomes inthe first 30 days following transurethral catheterization, followed by a Markov model to estimate outcomesover a person's remaining lifetime. Clinical outcomes included catheter balloon injuries [CBIs], associatedshort-term complications, urethral stricture disease, life years and QALYs. Health-economic outcomes includedtotal costs, incremental cost-effectiveness ratio, net monetary benefit (NMB) and net health benefit.
RESULTS: Over a person's lifetime, the safety valve was predicted to reduce CBIs by 0.04 per personand CBI-related short-term complications by 0.03 per person, and nearly halve total costs. The safetyvalve was dominant, resulting in 0.02 QALYs gained and relative cost savings of £93.19 per person. Probabilistic sensitivity analysis indicated that the safety valve would be cost-saving in 97% of simula-tions run versus standard of care.
CONCLUSIONS: The addition of a novel safety valve aiming to prevent CBIs during transurethral cath-eterization to current standard of care was estimated to bring both clinical benefits and cost savings.
