BackgroundLittle is known about the prevalence of severe, uncontrolled eosinophilic asthma (SUEA) and associated costs.AimsWe sought to determine the prevalence of SUEA and compare asthma-related healthcare resource use (HCRU) and associated costs with overall means for a general asthma population.MethodsThis cohort study evaluated anonymised medical record data (December 1989 through June 2015) from the Clinical Practice Research Datalink and the Optimum Patient Care Research Database to study UK patients with active asthma (diagnostic code and one or more drug prescriptions in the baseline year), aged 5 years and older, without concomitant COPD, and with recorded eosinophil count. SUEA was defined as two or more asthma attacks during 1 baseline year preceding a high blood eosinophil count (≥0.3×109/L) for patients prescribed long-acting β2-agonist (LABA) and high-dosage inhaled corticosteroids (ICS) during baseline plus 1 follow-up year. We compared asthma-related HCRU and associated direct costs (2015 pounds sterling, £) during the follow-up year for SUEA versus the general asthma population.ResultsOf 363 558 patients with active asthma and recorded eosinophil count, 64% were women, mean (SD) age was 49 (21) years; 43% had high eosinophil counts, 7% had two or more attacks in the baseline year and 10% were prescribed high-dosage ICS/LABA for 2 study years. Overall, 2940 (0.81%; 95% CI 0.78% to 0.84%) patients had SUEA. Total mean per-patient HCRU and associated costs were four times greater for SUEA versus all patients (HCRU and cost ratios 3.9; 95% CI 3.7 to 4.1).ConclusionsLess than 1% of patients in a general asthma population had SUEA. These patients accounted for substantially greater asthma-related HCRU and costs than average patients with asthma.

Over the long term, adverse outcomes associated with SCS initiation were relatively frequent and costly, with a positive dosage-response relationship with SCS exposure.

Despite the availability of approved asthma treatments, this literature analysis confirms that SUA poses a substantial epidemiologic, clinical, humanistic, and economic burden. Published data are limited for certain aspects of SUA, highlighting a need for further research.

Benralizumab is an interleukin-5 receptor α-directed cytolytic monoclonal antibody that directly depletes eosinophils. Its relative efficacy versus other IL-5-targeted treatments for patients with severe, uncontrolled asthma is not yet fully characterised.We performed a matching-adjusted indirect comparison (MAIC) of benralizumab versus mepolizumab and reslizumab. Trials were selected through systematic review and evaluation of trial methods. Benralizumab patient-level data were weighted to match treatment-effect-modifying patient characteristics of comparator trials before indirect efficacy comparisons.After matching adjustment, benralizumab and mepolizumab reduced exacerbations versus placebo by 52% and 49%, respectively (rate ratio [RR] 0.94, 95% CI 0.78–1.13; n=1524) and reduced the rate of exacerbations requiring hospitalisation/emergency department visit by 52% and 52%, respectively (RR 1.00, 95% CI 0.57–1.75; n=1524). Benralizumab and mepolizumab similarly improved pre-bronchodilator forced expiratory volume in 1 s at 32 weeks (difference 0.03 L, 95% CI −0.06–0.12; n=1443). Benralizumab and reslizumab patient populations were too dissimilar to generate a sufficient effective sample size to produce a reliable estimate for MAIC.MAIC is a robust way to indirectly compare treatment efficacies from trials with heterogeneous patient populations. When baseline patient characteristics were matched across asthma trials, benralizumab and mepolizumab yielded similar efficacy.

Aim: Assess the comparative efficacy of anifrolumab 300 mg versus belimumab 10 mg/kg in adults with moderate-to-severe systemic lupus erythematosus (SLE) receiving standard therapy. Patients and methods: Population-adjusted simulated treatment comparisons (primary analyses) and matching-adjusted indirect comparisons (supporting analyses) were conducted using individual patient data from TULIP-1/TULIP-2 and summary-level data from BLISS-52/BLISS-76. Results: Compared with belimumab-treated patients, anifrolumab-treated patients were more than twice as likely to achieve a reduction of four or more points in SLE Disease Activity Index 2000 score (simulated treatment comparison odds ratio: 2.47; 95% CI: 1.16–5.25) and SLE Responder Index-4 response (odds ratio: 2.61; 95% CI: 1.22–5.58) at 52 weeks. Conclusion: Patients with moderate-to-severe SLE are more likely to achieve an improvement in disease activity with anifrolumab than with belimumab.

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Background The Global Initiative for Asthma recommends the use of as-needed low-dose inhaled corticosteroid (ICS)-formoterol as a preferred controller therapy for patients with mild asthma. These recommendations were based, in part, on evidence from the SYGMA 1 and 2 studies of as-needed budesonide-formoterol. This analysis aimed to compare the cost-effectiveness of as-needed budesonide-formoterol to low-dose maintenance ICS plus as-needed short-acting β2-agonist (SABA) in patients with mild asthma. Methods A Markov cohort model was designed that included three possible health states (non-exacerbation, severe exacerbation, and death) to compare as-needed budesonide-formoterol 200–6 μg to twice-daily budesonide 200 μg maintenance therapy (low-dose ICS) plus as-needed terbutaline 0.5 mg (SABA). The deterministic base-case analysis used severe exacerbation, adverse event (AE), and healthcare resource use data from SYGMA 2, and was conducted from a Canadian public payer perspective with a 50-year time horizon, and a discount rate of 1.5% per annum. Moderate exacerbation was modelled on data from SYGMA 1 in sensitivity analyses. Utility values were derived from SYGMA 2 quality of life data. All-cause- and asthma-related mortality rates and costs (reported in 2019 Canadian dollars) were based on published data, using Canada-specific values where available. One-way deterministic sensitivity, probabilistic sensitivity, and eight scenario analyses were conducted to examine the robustness of the results. Results As-needed budesonide-formoterol was the dominant treatment option in the base-case analysis, providing incremental cost savings of $9882 per patient and quality-adjusted life year (QALY) gains of 0.002 versus low-dose maintenance ICS plus as-needed SABA over a 50-year time horizon. Using a willingness-to-pay threshold of $50,000/QALY ($100,000/QALY), as-needed budesonide-formoterol had a 94% (95%) probability of being cost-effective compared with maintenance ICS plus as-needed SABA. Cost-saving was mostly driven by lower overall medication and AE-related costs. As-needed budesonide-formoterol remained the dominant treatment in sensitivity and scenario analyses. Conclusions As-needed budesonide-formoterol is a cost-saving option for the treatment of mild asthma from the perspective of the Canadian public payer compared with low-dose maintenance ICS plus as-needed SABA.

Background Oral corticosteroid (OCS) treatment for severe asthma is associated with substantial disease burden. Thus, OCS dosage reduction is desirable. Relative efficacy of biologics in reducing OCS treatment for severe, uncontrolled asthma is not fully characterized. Objective We performed a matching‐adjusted indirect comparison (MAIC) to assess the relative effects on OCS treatment reduction of three biologic asthma treatments. Methods In MAIC of benralizumab vs. mepolizumab and vs. dupilumab, patient‐level data from the Phase III benralizumab OCS‐sparing trial, ZONDA, were weighted to match treatment effect–modifying patient characteristics in comparator trials. Results After matching adjustment, mean difference between benralizumab and mepolizumab for OCS reduction was 6.08% (95% CI −22.22‐34.38; P = .67) by week 24, and odds ratio of OCS elimination was 2.32 (95% CI 0.48‐11.15; P = .29). A trend in annual asthma exacerbation rate reduction favouring benralizumab over mepolizumab was observed, although it was not statistically significant (rate ratio [RR] = 0.56 [95% CI 0.28‐1.13; P = .11]). Mean difference between benralizumab and dupilumab for OCS reduction was −0.71% (95% CI −20.56‐19.15; P = .94), and odds ratio of OCS elimination was 2.26 (95% CI 0.52‐9.84; P = .28). A non‐significant trend in annual asthma exacerbation rate reduction favouring benralizumab over dupilumab was observed (RR = 0.50 [95% CI 0.20‐1.28; P = .15]). Effective sample size was 49% (72 vs. 148) and 25% (36 vs. 142) of original sample size for MAIC of benralizumab vs. mepolizumab and benralizumab vs. dupilumab, respectively. Conclusions and Clinical Relevance Following patient baseline characteristics matching across clinical trials, benralizumab demonstrated efficacy comparable to mepolizumab and dupilumab for OCS dosage reduction, OCS elimination, and annual exacerbation rate reduction. Comparatively low effective sample sizes indicated substantial differences for patient populations between ZONDA and mepolizumab and dupilumab trials.