UK Healthcare Payer Perspective Research Articles

Rituximab compared to intravenous cyclophosphamide in adults with connective tissue disease-associated interstitial lung disease: the RECITAL RCT

Background Interstitial lung disease frequently complicates systemic autoimmune disorders including scleroderma, idiopathic inflammatory myositis and mixed connective tissue disease, resulting in considerable morbidity and mortality. Based on the results of trials undertaken in scleroderma, cyclophosphamide is the standard of care for individuals with severe or progressive connective tissue disease-associated interstitial lung disease. Observational studies suggest that the anti-CD20 monoclonal antibody, rituximab is an effective rescue therapy in treatment of refractory connective tissue disease-associated interstitial lung disease, but it has not been studied as first-line therapy in clinical trials. Objectives To compare the safety and efficacy of rituximab against that of cyclophosphamide as treatment for individuals with severe, progressive interstitial lung disease associated with scleroderma, idiopathic inflammatory myositis or mixed connective tissue disease. Methods This was a Phase IIb, multicentre, randomised, double-blind, double-dummy study assessing the superiority of rituximab compared with cyclophosphamide, conducted in rheumatology or interstitial lung disease units at 11 UK centres. The study recruited individuals with extensive and/or progressive connective tissue disease-associated interstitial lung disease, excluding those with significant comorbidities, including airflow obstruction. Participants were randomised 1 : 1 to receive either rituximab 1 g given intravenously, twice at an interval of 2 weeks, or intravenous cyclophosphamide given monthly for 6 months at a dose of 600 mg/m2 body surface area. The primary end point for the study was the change in forced vital capacity at 24 weeks. Secondary end points included safety and tolerability, corticosteroid exposure, forced vital capacity change at 48 weeks and patient-reported quality of life. A cost-effectiveness analysis was undertaken to assess the impact of rituximab use in the United Kingdom National Health Service. Results One hundred and one subjects (70 females) with a mean age of 56.3 years were randomised; 51 to rituximab and 50 to cyclophosphamide. Ninety-seven were included in the modified intention-to-treat population for the primary and secondary efficacy analyses (49 in the rituximab group and 48 in the cyclophosphamide group). 38.6% had scleroderma, 44.6% idiopathic inflammatory myositis and 16.8% mixed connective tissue disease. Four subjects withdrew prior to the first dose of therapy (two in each arm). At 24 weeks, both rituximab and cyclophosphamide improved forced vital capacity from baseline [(mean ± standard deviation) 97 ± 234 and 99 ± 329 ml, respectively]. Using an adjusted mixed-effects model corrected for diagnosis and baseline forced vital capacity the difference in forced vital capacity at 24 weeks between rituximab and cyclophosphamide was −40 ml (95% CI −153 to 74 ml), p = 0.49. Other physiological and quality-of-life parameters improved in both arms following treatment but were not statistically significantly different between groups. Numerically fewer adverse events were reported by subjects receiving rituximab. Corticosteroid exposure over the 48 weeks of the trial was numerically less in the rituximab arm [13,291 (±14,657) mg of hydrocortisone equivalent per subject in the cyclophosphamide arm versus 11,469 (±10,041) mg per subject in the rituximab group; these differences did not reach statistical significance]. Limitations of the study include a disproportionate number of subjects being recruited from a single centre and insufficient subjects in each subgroup to determine whether there were treatment differences between individual connective tissue diseases. Based on the results of the trial, from a UK healthcare payer perspective, rituximab is more cost-effective than cyclophosphamide as a treatment for severe or progressive connective tissue disease-interstitial lung disease. Conclusions Rituximab improved forced vital capacity and patient-reported quality of life at 24 weeks but was not superior to cyclophosphamide. Rituximab should be considered as a therapeutic alternative to cyclophosphamide in individuals with connective tissue disease-associated interstitial lung disease requiring systemic therapy. Future work should explore the role of repeated dosing of rituximab and the use of rituximab earlier in the course of connective tissue disease-associated interstitial lung disease. Trial registration This trial is registered as ISRCTN16474148. Funding This award was funded by the National Institute for Health and Care Research (NIHR) Efficacy and Mechanism Evaluation Programme (NIHR award ref: 11/116/03) and is published in full in Efficacy and Mechanism Evaluation; Vol. 11, No. 4. See the NIHR Funding and Awards website for further award information.

Cost-effectiveness of pembrolizumab for previously treated MSI-H/dMMR solid tumours in the UK

Objectives Patients with previously treated microsatellite instability-high (MSI-H)/mismatch repair deficient (dMMR) tumours have limited chemotherapeutic treatment options. Pembrolizumab received approval from the EMA in 2022 for the treatment of colorectal, endometrial, gastric, small intestine, and biliary MSI-H/dMMR tumour types. This approval was supported by data from the KEYNOTE-164 and KEYNOTE-158 clinical trials. This study evaluated the cost-effectiveness of pembrolizumab compared with standard of care (SoC) for previously treated MSI-H/dMMR solid tumours in line with the approved EMA label from a UK healthcare payer perspective. Methods A multi-tumour partitioned survival model was built consisting of pre-progression, progressed disease, and dead health states. Pembrolizumab survival outcomes were extrapolated using Bayesian hierarchical models (BHMs) fitted to pooled data from KEYNOTE-164 and KEYNOTE-158. Comparator outcomes were informed by published sources. Tumour sites were modelled independently and then combined, weighted by tumour site distribution. A SoC comparator was used to formulate the overall cost-effectiveness result with pembrolizumab as the intervention. SoC comprised a weighted average of the comparators by tumour site based on market share. Drug acquisition, administration, adverse events, monitoring, subsequent treatment, end-of-life costs, and testing costs were included. Sensitivity and scenario analyses were performed, including modelling pembrolizumab efficacy using standard parametric survival models. Results Pembrolizumab, at list price, was associated with £129,469 in total costs, 8.30 LYs, and 3.88 QALYs across the pooled tumour sites. SoC was associated with £28,222 in total costs, 1.14 LYs, and 0.72 QALYs across the pooled tumour sites. This yields an incremental cost-effectiveness ratio (ICER) of £32,085 per QALY. Results were robust to sensitivity and scenario analyses. Conclusions This model demonstrates pembrolizumab provides a valuable new alternative therapy for UK patients with MSH-H/dMMR cancer at the cost of £32,085 per QALY, with confidential discounts anticipated to improve cost-effectiveness further.

Calibration of the IQVIA Core Diabetes Model to the stroke outcomes from the SUSTAIN 6 cardiovascular outcomes trial of once-weekly semaglutide

AimsIn the SUSTAIN 6 cardiovascular outcomes trial, once-weekly semaglutide was associated with a statistically significant reduction in major adverse cardiovascular events compared with placebo. To date, no studies have assessed how accurately existing diabetes models predict the outcomes observed in SUSTAIN 6. The aims of this analysis were to investigate the performance of the IQVIA Core Diabetes Model when used to predict the SUSTAIN 6 trial outcomes, to calibrate the model such that projected outcomes reflected observed outcomes, and to examine the impact of calibration on the cost-effectiveness of once-weekly semaglutide from a UK healthcare payer perspective.MethodsThe IQVIA Core Diabetes Model was calibrated to ensure that the projected non-fatal stroke event rates reflected the non-fatal stroke event rates observed in SUSTAIN 6 over a two-year time horizon. Cost-effectiveness analyses of once-weekly semaglutide versus placebo plus standard of care were conducted over a lifetime horizon using the uncalibrated and calibrated models to assess the impact on cost-effectiveness outcomes.ResultsTo replicate the non-fatal stroke event rate in SUSTAIN 6, calibration of the model through the application of relative risks for stroke of 1.07 and 1.65 with once-weekly semaglutide and placebo, respectively, was required. In the long-term cost-effectiveness analysis, the uncalibrated model projected an incremental cost-effectiveness ratio for once-weekly semaglutide versus placebo plus standard of care of GBP 22,262 per quality-adjusted life year (QALY) gained, which fell to GBP 17,594 per QALY gained when the calibrated model was used.ConclusionsThe requirement for calibration to replicate the outcomes observed in SUSTAIN 6 suggests that the reductions in risk of cardiovascular complications observed with once-weekly semaglutide cannot be solely explained by differences in conventional risk factors. Accurate estimation of the risk of diabetes-related complications using methods such as calibration is important to ensure accurate cost-effectiveness analyses are conducted.

Evaluating the cost-utility of intravesical Bacillus Calmette-Guérin versus radical cystectomy in patients with high-risk non-muscle-invasive bladder cancer in the UK

AimsApproximately 75% of bladder cancer (BC) cases present as non-muscle-invasive BC (NMIBC). In patients with high-risk NMIBC, the mainstay treatment is intravesical Bacillus Calmette-Guérin (BCG), with immediate radical cystectomy (RC) as an alternative treatment option. The aim of the present study was to evaluate the cost-utility of BCG versus RC in patients with high-risk NMIBC from the UK healthcare payer perspective.Materials and methodsA six-state Markov model was developed that covered controlled disease, recurrence, progression to muscle-invasive BC, metastatic disease, and death. The model included adverse events of BCG and RC and monitoring and palliative care. Drug costs were obtained from the British National Formulary. Intravesical delivery, RC, and monitoring costs were sourced from the National Tariff Payment System and the literature. Utility data were obtained from the literature. Analyses were run over a 30-year time horizon, with future costs and effects discounted at 3.5% per annum. One-way and probabilistic sensitivity analyses were performed.ResultsThe base case analysis comparing BCG with RC showed that BCG would increase life expectancy by 0.88 years versus RC, from 7.74 to 8.62 years. BCG resulted in an increase of 0.76 quality-adjusted life years (QALYs) versus RC, from 5.63 to 6.39 QALYs. Patients incurred lower lifetime costs if treated with BCG (£47,753) than with RC (£64,264). Cost savings were mainly driven by the lower cost of BCG versus RC, and palliative care costs. Sensitivity analyses showed that results were robust to assumptions.LimitationsThe evidence base informing efficacy estimates of BCG is heterogeneous as different BCG administration schedules were reported in the literature, while incidence and cost data on some BCG-associated adverse events were sparse.ConclusionsIntravesical BCG led to increased QALYs and reduced costs versus RC for patients with high-risk NMIBC from the UK healthcare payer perspective.

Exploratory comparison of Healthcare costs and benefits of the UK's Covid-19 response with four European countries.

BackgroundIn responding to Covid-19, governments have tried to balance protecting health while minimizing gross domestic product (GDP) losses. We compare health-related net benefit (HRNB) and GDP losses associated with government responses of the UK, Ireland, Germany, Spain and Sweden from UK healthcare payer perspective.MethodsWe compared observed cases, hospitalizations and deaths under ‘mitigation’ to modelled events under ‘no mitigation’ to 20 July 2020. We thus calculated healthcare costs, quality adjusted life years (QALYs), and HRNB at £20,000/QALY saved by each country. On per population (i.e. per capita) basis, we compared HRNB with forecast reductions in 2020 GDP growth (overall or compared with Sweden as minimal mitigation country) and qualitatively and quantitatively described government responses.ResultsThe UK saved 3.17 (0.32–3.65) million QALYs, £33 (8–38) billion healthcare costs and £1416 (220–1637) HRNB per capita at £20,000/QALY. Per capita, this is comparable to £1455 GDP loss using Sweden as comparator and offsets 46.1 (7.1–53.2)% of total £3075 GDP loss. Germany, Spain, and Sweden had greater HRNB per capita. These also offset a greater percentage of total GDP losses per capita. Ireland fared worst on both measures. Countries with more mask wearing, testing, and population susceptibility had better outcomes. Highest stringency responses did not appear to have best outcomes.ConclusionsOur exploratory analysis indicates the benefit of government Covid-19 responses may outweigh their economic costs. The extent that HRNB offset economic losses appears to relate to population characteristics, testing levels, and mask wearing, rather than response stringency.

Long-Term Cost-Effectiveness Analyses of Empagliflozin Versus Oral Semaglutide, in Addition to Metformin, for the Treatment of Type 2 Diabetes in the UK

IntroductionInternational guidelines recommend treatment with a sodium-glucose cotransporter-2 (SGLT-2) inhibitor or glucagon-like peptide-1 (GLP-1) receptor agonist for treatment intensification in type 2 diabetes mellitus (T2DM) patients with progression on metformin. In the randomised, controlled, Peptide Innovation for Early Diabetes Treatment (PIONEER) 2 trial, the SGLT-2 inhibitor empagliflozin was compared with the GLP-1 receptor agonist oral semaglutide, in addition to metformin. The aim of the current study was to assess the long-term cost-effectiveness of empagliflozin 25 mg versus oral semaglutide 14 mg, in addition to metformin, for T2DM patients in the UK.MethodsAnalyses were conducted from the UK healthcare payer perspective, using the IQVIA Core Diabetes model, with a time horizon of 50 years. Patients received either empagliflozin or oral semaglutide, in addition to metformin, until Hba1c threshold of 7.5% (58 mmol/mol) was exceeded, following which treatment intensification with insulin glargine in addition to empagliflozin or oral semaglutide plus metformin was assumed. Baseline cohort characteristics and 52-week treatment effects were derived from the PIONEER 2 trial. Treatment effects of empagliflozin and GLP-1 receptor agonists on hospitalisation for heart failure (hHF) were based on the Empagliflozin Comparative Effectiveness and Safety (EMPRISE) real-world study. Utilities, treatment costs and costs of diabetes-related complications were obtained from published sources.ResultsDirect costs for empagliflozin plus metformin were considerably lower than those for oral semaglutide plus metformin (by more than GBP 6000). Compared with oral semaglutide plus metformin, empagliflozin plus metformin was a cost-effective treatment for T2DM patients in all scenarios tested. Probabilistic sensitivity analysis showed cost-effectiveness in > 95% of the iterations using a threshold of 20,000 GBP/QALY.ConclusionEmpagliflozin 25 mg is a cost-effective treatment option versus oral semaglutide 14 mg, when used in addition to metformin, for the treatment of T2DM patients in the UK.Electronic supplementary materialThe online version of this article (10.1007/s13300-020-00883-1) contains supplementary material, which is available to authorized users.

Evaluating the Long-Term Cost-Effectiveness of Once-Weekly Semaglutide Versus Once-Daily Liraglutide for the Treatment of Type2 Diabetes in the UK.

IntroductionOnce-weekly semaglutide 1 mg is a novel glucagon-like peptide-1 receptor agonist (GLP-1 RA) for the treatment of type 2 diabetes that has demonstrated significantly greater reductions in glycated haemoglobin (HbA1c) and body weight than the GLP-1 RA once-daily liraglutide 1.2 mg in the SUSTAIN 10 trial. The present analysis aimed to evaluate the long-term cost-effectiveness of once-weekly semaglutide 1 mg versus once-daily liraglutide 1.2 mg from a UK healthcare payer perspective.MethodsLong-term outcomes were projected using the IQVIA CORE Diabetes Model (version 9.0), with baseline characteristics and treatment effects sourced from SUSTAIN 10. Patients were assumed to initiate treatment with GLP-1 RAs and continue treatment until HbA1c exceeded 7.5%, at which point GLP-1 RAs were discontinued and basal insulin was initiated. Pharmacy costs and costs of complications were measured in 2018 pounds sterling (GBP), with future costs and outcomes discounted at 3.5% per annum. Utilities were taken from published sources.ResultsIn the base-case analysis, once-weekly semaglutide 1 mg was associated with an increase in discounted life expectancy of 0.21 years and discounted quality-adjusted life expectancy of 0.30 quality-adjusted life-years, compared with once-daily liraglutide 1.2 mg. Clinical benefits were achieved at reduced costs, with lifetime cost savings of GBP 140 per patient with semaglutide versus liraglutide, owing to a reduction in diabetes-related complications, in particular cardiovascular disease (mean cost saving of GBP 279 per patient). Therefore, once-weekly semaglutide 1 mg was dominant compared with once-daily liraglutide 1.2 mg. The results of the sensitivity analyses were similar, demonstrating the robustness of the base-case analysis.ConclusionsOnce-weekly semaglutide 1 mg is a cost-effective treatment option versus once-daily liraglutide 1.2 mg, based on the SUSTAIN 10 trial, from a UK healthcare payer perspective.Electronic Supplementary MaterialThe online version of this article (10.1007/s12325-020-01337-7) contains supplementary material, which is available to authorized users.

Evaluation of the long-term cost-effectiveness of once-weekly semaglutide versus dulaglutide for treatment of type 2 diabetes mellitus in the UK.

AimsGlucagon‐like peptide‐1 (GLP‐1) receptor agonists are appealing as glucose‐lowering therapy for individuals with type 2 diabetes mellitus (T2DM) as they also reduce body weight and are associated with low rates of hypoglycaemia. This analysis assessed the long‐term cost‐effectiveness of semaglutide 0.5 and 1 mg vs dulaglutide 1.5 mg (two once‐weekly GLP‐1 receptor agonists) from a UK healthcare payer perspective, based on the head‐to‐head SUSTAIN 7 trial, to inform healthcare decision making.Materials and MethodsLong‐term outcomes were projected using the IQVIA CORE Diabetes Model (version 9.0). Baseline cohort characteristics, changes in physiological parameters and adverse event rates were derived from the 40‐week SUSTAIN 7 trial. Costs to a healthcare payer were assessed, and these captured pharmacy costs and costs of complications. Utilities were taken from published sources.ResultsOnce‐weekly semaglutide 0.5 and 1 mg were associated with improvements in quality‐adjusted life expectancy of 0.04 and 0.10 quality‐adjusted life years, respectively, compared with dulaglutide 1.5 mg. Clinical benefits were achieved at reduced costs, with lifetime cost savings of GBP 35 with once‐weekly semaglutide 0.5 mg and GBP 106 with the once‐weekly semaglutide 1 mg, resulting from fewer diabetes‐related complications due to better glycaemic control. Therefore, both doses of once‐weekly semaglutide were considered dominant vs dulaglutide 1.5 mg (improving outcomes and reducing costs).ConclusionsCompared with treatment with dulaglutide, once‐weekly semaglutide represents a cost‐effective option for treating individuals in the UK with T2DM who are not achieving glycaemic control with metformin, projected to both improve clinical outcomes and reduce costs.

Cost-Utility of an Objective Biochemical Measure to Improve Adherence to Antihypertensive Treatment.

Nonadherence to antihypertensive medications is known to be a major health problem. Novel biochemical analyses using liquid chromatography-tandem mass spectrometry are becoming accepted as a clinically useful objective measure to manage (non)adherence in Hypertension Clinics. Discussion of results from such analyses with patients can significantly improve adherence and blood pressure control. Our objective was to model the cost-effectiveness of performing liquid chromatography-tandem mass spectrometry-based analyses in improving adherence in patients with hypertension. Lifetime cost-utility was assessed from a UK healthcare payer perspective, using a Markov model. Efficacy was based on study findings of lowering blood pressure because of improved adherence to drug treatment. Cost and utilities were derived from literature. The base case cohort consisted of males aged 65 years. Subgroup analyses included varying population sex and age and a subgroup of patients with apparent resistant hypertension. Additionally, univariate and probabilistic sensitivity analyses were performed. Our findings are reported after the Consolidated Health Economic Evaluation Reporting Standards checklist. Per patient, screening resulted in 0.020 incremental quality-adjusted life-years and a negative incremental cost of £495, suggesting the intervention to be dominant compared with care as usual. Targeting younger patients or patients with apparent resistant hypertension would further improve these outcomes. Modeling suggested that screening prevented 518 myocardial infarctions and 305 stroke events in a cohort of 10 000 male hypertensive patients. Using liquid chromatography-tandem mass spectrometry-based biochemical analyses to improve adherence in hypertensive patients is likely to be an effective and cost-saving strategy, especially in patients with apparent resistant hypertension.

Cost-Utility Of Liquid Chromatography -Tandem Mass Spectrometry (LC-MS/MS)-Based Urine Analyses To Improve Adherence To Antihypertensive Treatment

Objectives: In the UK, over 28% of the population are estimated to suffer from hypertension. A high systolic blood pressure (SBP) increases the risk for cardiovascular disease (CVD). While antihypertensive medications, along with lifestyle changes, are deemed effective in combatting hypertension, adherence to drug treatment may not be optimal. Data from UK patient samples show that discussion of the results from LC-MS/MS-based urine analyses with patients can improve adherence significantly. The objective of this study was to determine whether performing LC-MS/ MS-based urine analyses is cost-effective by improving adherence in hypertensive patients. Methods: Cost-utility analysis was performed from a UK healthcare payer perspective over a lifetime horizon. A Markov model was adapted from an existing published model in a UK setting. Hypertensive patients entered the model event-free, but at risk for cardiovascular events. Effectiveness of urine analysis was modelled by lowering the probability of having an event, as a consequence of lowered SBP by improved adherence to drug treatment, as found in the empirical study. Cost and utilities were derived from literature. The base case cohort consisted of males aged 65. Further analysis varied sex and age of the population. Subgroup analysis concerned those with resistant hypertension, and univariate and probabilistic sensitivity analyses were also performed. Results: The intervention resulted in an incremental health benefit of 0.020 quality-adjusted life-years (QALYs) per patient, and incremental cost was -£867 per patient, i.e. the intervention strategy is dominant compared to care as usual. Sensitivity analyses showed that targeting younger patients (aged 45) or only patients with resistant hypertension would increase cost savings and QALY gains. Conclusions: Using LC-MS/MS-based urine analyses to improve adherence in hypertensive patients is an effective and cost-saving strategy, especially in patients with resistant hypertension, since nonadherence is found to be higher in this population.

Evaluating the cost‐effectiveness of laparoscopic adjustable gastric banding versus standard medical management in obese patients with type 2 diabetes in the UK

To evaluate the cost-effectiveness of laparoscopic adjustable gastric banding (LAGB) versus standard medical management (SMM) in obese patients with type 2 diabetes from a UK healthcare payer perspective. A validated computer model of diabetes was used to project outcomes reported from a randomized clinical trial of LAGB versus SMM in obese patients with type 2 diabetes. Two-year follow-up data from the trial were projected over a 40-year time horizon and cost-effectiveness was assessed from the perspective of the National Health Service. Future costs and clinical outcomes were discounted at 3.5% annually and all costs were reported in 2010 pounds sterling. A series of sensitivity analyses were performed. LAGB was associated with benefits in HbA1c, systolic blood pressure, body mass index and serum lipid concentrations, which led to significant increases in discounted life expectancy (an increase of 0.64 years) and quality-adjusted life expectancy (an increase of 0.92 quality-adjusted life years, QALYs) and reduced incidence of diabetes complications relative to SMM. Treatment costs in the LAGB arm increased by 4552 Great British Pounds (GBP), but this was partially offset by cost savings resulting from a reduction in the incidence of all modelled diabetes complications. The incremental cost-effectiveness ratio of GBP 3602 per QALY in the base case fell well below commonly quoted willingness-to-pay thresholds in the UK setting. On the basis of data from a recent randomized controlled trial, LAGB is likely to be considered cost-effective from the healthcare payer perspective when compared with SMM of obesity in patients with type 2 diabetes in the UK setting.

Cost Effectiveness of Quetiapine in Patients with Acute Bipolar Depression and in Maintenance Treatment after an Acute Depressive Episode

Bipolar disorder has a significant impact upon a patient's quality of life, imposing a considerable economic burden on the individual, family members and society as a whole. Several medications are indicated for the acute treatment of mania and depression associated with bipolar disorder as well as for maintenance therapy; however, these have varying efficacy, tolerability and costs. The objective of this study was to develop a new discrete-event simulation model to analyse the long-term consequences of pharmacological therapy for the management of bipolar I and II disorders (acute treatment of episodes of mania and depression as well as maintenance therapy). Probabilities of remission and relapse were obtained from clinical trial data and meta-analyses. Costs (year 2011 values) were assessed from a UK healthcare payer's perspective, and included pharmacological therapy and resource use associated with the treatment of mood events and selected adverse events. The health effects were measured in terms of QALYs. For a patient starting with acute depression or in remission at 40 years of age (which was the average age in the clinical trials), quetiapine 300 mg/day was a cost-effective strategy compared with olanzapine 15 mg/day over a 5-year time frame. With acute bipolar depression as a starting episode, the 5-year medical costs were £323 higher and QALYs were 0.038 higher for quetiapine compared with olanzapine, corresponding to a cost-effectiveness ratio of £8600 per QALY gained. Compared with olanzapine, the results suggest that quetiapine is cost effective as a maintenance treatment for bipolar depression.

Financial impact of a novel pre-eclampsia diagnostic test versus standard practice: a decision-analytic modeling analysis from a UK healthcare payer perspective

Objective:Pre-eclampsia (PE), a leading cause of maternal and perinatal morbidity and mortality, is only detected after symptomatic onset. Early diagnosis may be possible with a new serum test, with resulting clinical and economic benefits versus standard practice. The authors evaluated the financial impact to the UK National Health Service (NHS).Methods:A decision-analytic model was developed in which a cohort of 1,000 pregnant women receiving UK obstetric care was simulated. The economic impact of improved sensitivity and specificity of the novel PE test [Roche Diagnostics, Rotkreuz, Switzerland] over current diagnostic practice was modeled. While there is no specific approved diagnostic test to detect PE, physicians are using a combination of tests including blood pressure, proteinuria, Doppler, serum uric acid, etc. The novel PE test constitutes two novel biomarkers Placenta Growth Factor (PlGF) and soluble fms-like tyrosine kinase-1 (sFlt-1) which can be quantitatively analyzed using an automated system widely available in hospitals or laboratories (Elecsys/Cobas, Roche Diagnostics) and measures the levels of PlGF and sFlt-1 growth factors in pregnant women. The analysis assumed administration of the £31.13 test (the equivalent of 52 Swiss Francs [CHF]) after 20 weeks of gestation as an addition to current practice. True-positive and false-negative patients were assumed to develop mild or severe PE, eclampsia, or death. A hybrid research approach was adopted; when available, data for model inputs were obtained from published literature and public databases. Interviews with obstetricians, laboratory managers, and healthcare payers were used to validate model inputs and fill utilization-related data gaps.Results:The model estimates that the costs associated with managing a typical pregnancy are £1,781 per patient when the new test is used versus £2,726 with standard practice. This represents savings of £945 per pregnant woman, if the test is used as a supplementary diagnostic tool. The savings are attributed to the new test's improved performance and its ability to better classify the pregnant patients.Conclusions:The novel test has the potential to provide substantial cost savings for NHS. Even when the novel test's cost is added to the current cost of care, the benefits exceed the additional cost, driven by the test's ability to reduce the rates of false-positive and false-negative diagnoses compared to current standard of care. Potential study limitations include the use of a pooled average of the individual sensitivities and specificities of currently used tests since no data were available on combination testing, the reliance on clinical trial data versus actual practice, and the use of clinical expert opinion when published data were unavailable.

Cost Effectiveness of Rasagiline and Pramipexole as Treatment Strategies in Early Parkinsonʼs Disease in the UK Setting

Levodopa is the most effective treatment for the symptoms of Parkinson's disease (PD). However, after an initial period of benefit, several limitations become apparent, including motor complications such as dyskinesia. Dyskinesia can severely affect patients' quality of life and increases healthcare resource use. Thus, delaying the need for levodopa, and therefore the onset of levodopa-induced dyskinesia, is important. The aim of this study was to compare the cost effectiveness, from a UK healthcare payer perspective, of two antiparkinsonian treatment strategies in early PD: first-line monotherapy with rasagiline, a novel monoamine oxidase B inhibitor; and the non-ergoline dopamine receptor agonist pramipexole. An economic Markov model was developed as a pragmatic tool to derive comparative information on the effectiveness, utility and costs of these two strategies over a 5-year period. Model input data were obtained from the TEMPO study for rasagiline and from a study by the Parkinson Study Group for pramipexole. Effectiveness outcomes were time to levodopa and time to levodopa-induced dyskinesia. Cost and quality-adjusted life-year (QALY) data were derived from published sources. Rasagiline was the dominant strategy. Compared with pramipexole, use of the rasagiline strategy was estimated to reduce costs by 18% per patient over 5 years and was associated with an additional 10% delay in dyskinesia onset (0.41 years; 95% CI 0.27, 0.55). This strategy was also found to prolong the time to levodopa initiation by 25% through a gain of 0.83 levodopa-free years (95% CI 0.56, 1.1). In addition, use of the rasagiline strategy was found to generate a 5% gain in QALYs over 5 years compared with the pramipexole strategy (3.7 +/- 0.02 vs 3.51 +/- 0.03). Sensitivity analyses confirmed that the model was robust. Rasagiline represents a cost-effective alternative to pramipexole in the treatment of early PD in the UK.

An economic evaluation of NIOX MINO airway inflammation monitor in the United Kingdom

Fractional exhaled nitric oxide (FENO), a marker of eosinophilic airway inflammation, is easily measured by noninvasive means. The objective of this study was to determine the cost-effectiveness of FENO measurement using a hand-held monitor (NIOX MINO), at a reimbursement price of 23 pound sterling, for asthma diagnosis and management in the UK. We constructed two decision trees to compare FENO measurement with standard diagnostic testing and guideline recommendations for management. For asthma diagnosis, we compared FENO measurement with lung function and reversibility testing, bronchial provocation and sputum eosinophil count. For asthma management, we evaluated the impact on asthma control, including inhaled corticosteroid use, exacerbations and hospitalizations, of monitoring with FENO measurement vs symptoms and lung function as in standard care. Resource use and health outcomes were evaluated over a 1-year time frame. Direct costs were calculated from a UK health-care payer perspective (2005 pound sterling). An asthma diagnosis using FENO measurement cost 43 pound sterling less per patient as compared with standard diagnostic tests. Asthma management using FENO measurement instead of lung function testing resulted in annual cost-savings of 341 pound sterling and 0.06 quality-adjusted life-years gained for patients with mild to severe asthma and cost-savings of 554 pound sterling and 0.004 quality-adjusted life-years gained for those with moderate to severe asthma. Asthma diagnosis based on FENO measurement with NIOX MINO alone is less costly and more accurate than standard diagnostic methods. Asthma management based on FENO measurement is less costly than asthma management based on standard guidelines and provides similar health benefits.