Cost-effectiveness of golimumab for the treatment of patients with moderate-to-severe ulcerative colitis in Quebec using a patient level state transition microsimulation

Objective: To conduct cost-effectiveness analyses comparing the addition of golimumab to the standard of care (SoC) for treatment of patients with moderate-to-severe ulcerative colitis (UC) who are refractory to conventional therapies in Quebec (Canada).

Methods: An individual patient state transition microsimulation model was developed to project health outcomes and costs over 10 years, using a payer perspective. The incremental benefit estimates for golimumab were driven by induction response and risk of a flare. Flare risks post-induction were derived for golimumab from the PURSUIT maintenance trial and extension study, while those for SoC were derived from the placebo arms of the Active Ulcerative Colitis Trials (ACT) 1 and 2. Other inputs were derived from multiple sources, including retrospective claims analyses and literature. Costs are reported in 2014 Canadian dollars. A 5% annual discount rate was applied to costs and quality-adjusted life-years (QALYs).

Results: Compared with SoC, golimumab was projected to increase the time spent in mild disease or remission states, decrease flare rates, and increase QALYs. These gains were achieved with higher direct medical costs. The incremental cost-effectiveness ratio for golimumab vs SoC was $63,487 per QALY.

Limitations: The long-term flare projections for SoC were based on the data available from the ACT 1 and 2 placebo arms, as data were not available from the PURSUIT maintenance or extension trial. Additionally, the study was limited to only SoC and golimumab, due to the availability of individual patient data to analyze.

Conclusion: This economic analysis concluded that treatment with golimumab is likely more cost-effective vs SoC when considering cost-effectiveness acceptability thresholds from $50,000–$100,000 per QALY.     

Cost-effectiveness of rivaroxaban versus warfarin for stroke prevention in non-valvular atrial fibrillation in the Japanese healthcare setting

Abstract

Aims: This article aimed to examine the cost-effectiveness of rivaroxaban in comparison to warfarin for stroke prevention in Japanese patients with non-valvular atrial fibrillation (NVAF), from a public healthcare payer’s perspective.

Materials and methods: Baseline event risks were obtained from the J-ROCKET AF trial and the treatment effect data were taken from a network meta-analysis. The other model inputs were extracted from the literature and official Japanese sources. The outcomes included the number of ischaemic strokes, myocardial infarctions, systemic embolisms and bleedings avoided, life-years, quality-adjusted life-years (QALYs), incremental costs and incremental cost-effectiveness ratio (ICER). The scenario analysis considered treatment effect data from the same network meta-analysis.

Results: In comparison with warfarin, rivaroxaban was estimated to avoid 0.284 ischaemic strokes per patient, to increase the number of QALYs by 0.535 per patient and to decrease the total costs by ¥118,892 (€1,011.11) per patient (1 JPY = 0.00850638 EUR; XE.com, 7 October 2019). Consequently, rivaroxaban treatment was found to be dominant compared to warfarin. In the scenario analysis, the ICER of rivaroxaban versus warfarin was ¥2,873,499 (€24,446.42) per QALY.

Limitations: The various sources of data used resulted in the heterogeneity of the cost-effectiveness analysis results. Although, rivaroxaban was cost-effective in the majority of cases.

Conclusion: Rivaroxaban is cost-effective against warfarin for stroke prevention in Japanese patients with NVAF, giving the payer WTP of 5,000,000 JPY.     

Cost-effectiveness of targeted screening for the identification of patients with atrial fibrillation: evaluation of a machine learning risk prediction algorithm

Abstract

Aims: As many cases of atrial fibrillation (AF) are asymptomatic, patients often remain undiagnosed until complications (e.g. stroke) manifest. Risk-prediction algorithms may help to efficiently identify people with undiagnosed AF. However, the cost-effectiveness of targeted screening remains uncertain. This study aimed to assess the cost-effectiveness of targeted screening, informed by a machine learning (ML) risk prediction algorithm, to identify patients with AF.

Methods: Cost-effectiveness analyses were undertaken utilizing a hybrid screening decision tree and Markov disease progression model. Costs and outcomes associated with the detection of AF compared traditional systematic and opportunistic AF screening strategies to targeted screening informed by a ML risk prediction algorithm. Model analyses were based on adults ≥50?years and adopted the UK NHS perspective.

Results: Targeted screening using the ML risk prediction algorithm required fewer patients to be screened (61 per 1,000 patients, compared to 534 and 687 patients in the systematic and opportunistic strategies) and detected more AF cases (11 per 1,000 patients, compared to 6 and 8?AF cases in the systematic and opportunistic screening strategies). The targeted approach demonstrated cost-effectiveness under base case settings (cost per QALY gained of £4,847 and £5,544 against systematic and opportunistic screening respectively). The targeted screening strategy was predicted to provide an additional 3.40 and 2.05 QALYs per 1,000 patients screened versus systematic and opportunistic strategies. The targeted screening strategy remained cost-effective in all scenarios evaluated.

Limitations: The analysis relied on assumptions that include the extended period of patient life span and the lack of consideration for treatment discontinuations/switching, as well as the assumption that the ML risk-prediction algorithm will identify asymptomatic AF.

Conclusions: Targeted screening using a ML risk prediction algorithm has the potential to enhance the clinical and cost-effectiveness of AF screening, improving health outcomes through efficient use of limited healthcare resources.     

Cost-effectiveness of dabigatran etexilate in the prevention of stroke and systemic embolism in patients with atrial fibrillation in Belgium

Abstract

Objective:

To assess the cost-effectiveness of dabigatran etexilate (‘dabigatran’) vs vitamin K antagonists (VKAs) in the Belgian healthcare setting for the prevention of stroke and systemic embolism (SE) in patients with non-valvular atrial fibrillation (AF).

Research design and methods:

A Markov model was used to calculate the cost-effectiveness of dabigatran vs VKAs in Belgium, whereby warfarin was considered representative for the VKA class. Efficacy and safety data were taken from the Randomized Evaluation of Long-Term Anticoagulation Therapy (RE-LY) trial and a network meta-analysis. Local resource use and unit costs were included in the model. Effectiveness was expressed in Quality Adjusted Life-Years (QALYs). The model outcomes were total costs, total QALYs, incremental costs, incremental QALYs and the incremental cost-effectiveness ratio (ICER). The level of International Normalized Ratio (INR) control and the use of other antithrombotic therapies observed in Belgian clinical practice were reflected in two scenario analyses.

Results:

In the base case analysis, total costs per patient were €13,333 for dabigatran and €12,454 for warfarin. Total QALYs per patient were 9.51 for dabigatran and 9.19 for warfarin. The corresponding ICER was €2807/QALY. The ICER of dabigatran was €970/QALY vs warfarin with real-world INR control and €5296/QALY vs a mix of warfarin, aspirin, and no treatment. Results were shown to be robust in one-way and probabilistic sensitivity analyses.

Limitations:

The analysis does not include long-term costs for clinical events, as these data were not available for Belgium. As in any economic model based on data from a randomized clinical trial, several assumptions had to be made when extrapolating results to routine clinical practice in Belgium.

Conclusion:

This analysis suggests that dabigatran, a novel oral anticoagulant, is a cost-effective treatment for the prevention of stroke and SE in patients with non-valvular AF in the Belgian healthcare setting.     

Direct treatment cost of atrial fibrillation in the elderly American population: a Medicare perspective

Abstract

Objective: Although atrial fibrillation (AF) is the most commonly sustained arrhythmia in adults, few studies have examined the direct treatment cost of AF.

Methods: A Medicare database of a 5% random national sample of all beneficiaries was used to identify patients diagnosed with AF in 2003 and to follow them for 1 year after diagnosis. These patients were matched on a 1:1 basis by age, gender and race. The incremental cost of treating AF was calculated with multivariate regression models adjusting for covariates.

Results: In total, 55,260 subjects developed new AF, of which 69% were ≥75 years old, 54% were female and 91% were White. The adjusted mean incremental treatment cost of AF was $14,199 (95% confidence interval $13,201–15,001; p<0.01). Some of this cost was attributable to the incidence of stroke and heart failure at the 1-year post-AF diagnosis. A significantly higher proportion of AF patients experienced stroke (23.1 vs. 13.3%; p<0.01) and heart failure (36.7 vs. 10.4%; p<0.01) compared with Medicare beneficiaries without AF.

Conclusions: Mean incremental direct treatment costs for Medicare beneficiaries with AF were higher than previously reported. Interventions that can reduce the incidence of AF and its complications may also reduce the national economic impact of AF.     

Cost-effectiveness of renal denervation therapy for the treatment of resistant hypertension in The Netherlands

Abstract

Objectives:

Safety and efficacy data for catheter-based renal denervation (RDN) in the treatment of resistant hypertension have been used to estimate the cost-effectiveness of this approach. However, there are no Dutch-specific analyses. This study examined the cost-effectiveness of RDN from the perspective of the healthcare payer in The Netherlands.

Methods:

A previously constructed Markov state-transition model was adapted and updated with costs and utilities relevant to the Dutch setting. The cost-effectiveness of RDN was compared with standard of care (SoC) for patients with resistant hypertension. The efficacy of RDN treatment was modeled as a reduction in the risk of cardiovascular events associated with a lower systolic blood pressure (SBP).

Results:

Treatment with RDN compared to SoC gave an incremental quality-adjusted life year (QALY) gain of 0.89 at an additional cost of €1315 over a patient’s lifetime, resulting in a base case incremental cost-effectiveness ratio (ICER) of €1474. Deterministic and probabilistic sensitivity analyses (PSA) showed that treatment with RDN therapy was cost-effective at conventional willingness-to-pay thresholds (€10,000–80,000/QALY).

Conclusion:

RDN is a cost-effective intervention for patients with resistant hypertension in The Netherlands.     

Cost-effectiveness of percutaneous patent foramen ovale closure as secondary stroke prevention

Objective: Compared to medical therapy alone, percutaneous closure of patent foramen ovale (PFO) further reduces risk of recurrent ischemic strokes in carefully selected young to middle-aged patients with a recent cryptogenic ischemic stroke. The objective of this study was to evaluate the cost-effectiveness of this therapy in the context of the United Kingdom (UK) healthcare system.

Methods: A Markov cohort model consisting of four health states (Stable after index stroke, Post-Minor Recurrent Stroke, Post-Moderate Recurrent Stroke, and Death) was developed to simulate the economic outcomes of device-based PFO closure compared to medical therapy. Recurrent stroke event rates were extracted from a randomized clinical trial (RESPECT) with a median of 5.9-year follow-up. Health utilities and costs were obtained from published sources. One-way and probabilistic sensitivity analyses (PSA) were performed to assess robustness. The model was discounted at 3.5% and reported in 2016 Pounds Sterling.

Results: Compared with medical therapy alone and using a willingness-to-pay (WTP) threshold of £20,000, PFO closure reached cost-effectiveness at 4.2 years. Cost-effectiveness ratios (ICERs) at 4, 10, and 20 years were ?20,951, ?6,887, and ?2,158, respectively. PFO closure was cost-effective for 89% of PSA iterations at year 10. Sensitivity analyses showed that the model was robust.

Conclusions: Considering the UK healthcare system perspective, percutaneous PFO closure in cryptogenic ischemic stroke patients is a cost-effective stroke prevention strategy compared to medical therapy alone. Its cost-effectiveness was driven by substantial reduction in recurrent strokes and patients’ improved health-related quality-of-life.     

The cost-effectiveness and monetary benefits of dabigatran in the prevention of arterial thromboembolism for patients with non-valvular atrial fibrillation in the Netherlands

Background: Atrial fibrillation (AF) causes a significant health and economic burden to the Dutch society. Dabigatran was proven to have at least similar efficacy and a similar or better safety profile when compared to vitamin K antagonists (VKAs) in preventing arterial thromboembolism in patients with AF.

Objective: To evaluate the cost-effectiveness and monetary benefit of dabigatran vs VKAs in Dutch patients with non-valvular AF. Value-based pricing considerations and corresponding negotiations on dabigatran will be explicitly considered.

Methods: The base case economic analysis was conducted from the societal perspective. Health effects and costs were analysed using a Markov model. The main model inputs were derived from the RE-LY trial and Dutch observational data. Univariate, probabilistic sensitivity, and various scenario analyses were performed.

Results: Dabigatran was cost saving compared to VKAs. A total of 4,552 QALYs were gained, and €13,892,288 was saved in a cohort of 10,000?AF patients. The economic value of dabigatran was strongly related to the costs of VKA control that are averted. Notably, dabigatran was cost saving compared to VKAs if annual costs of VKA control exceeded €159 per person, or dabigatran costs were below €2.81 per day.

Conclusion: Dabigatran was cost saving compared to VKAs for the prevention of atrial thromboembolism in patients with non-valvular AF in the Netherlands. This result appeared robust in the sensitivity analysis. Furthermore, volume based reduction of the price in the Netherlands will further increase the monetary benefits of dabigatran.     

The real-world cost-effectiveness of adjuvant trastuzumab in HER-2/neu-positive early breast cancer in Taiwan

Background: Trastuzumab was considered a cost-effective adjuvant treatment for HER 2-positive early breast cancer. Since 2010, the Taiwanese National Health Insurance (NHI) has started to reimburse for 1-year adjuvant treatment. This study aims to provide an updated cost-effectiveness analysis from the NHI perspective, which explores assumptions about long-term cardiac toxicity and treatment benefit of 1-year adjuvant treatment sequentially after chemotherapy.

Methods: A Markov model was used to evaluate the cost-effectiveness of 1-year adjuvant trastuzumab for HER-2/neu positive early breast cancer over a 20-year life-time horizon. A probability sensitivity analysis using Monte Carlo simulation was performed to characterize uncertainties in the expected outcomes, which are expressed as an incremental costs effectiveness ratio (ICER, cost/QALY). A willingness-to-pay threshold of 3-times the per capita gross domestic product was adopted according to the WHO definition. The Taiwan per capita gross domestic product in 2015 was US$22,355; thus, a threshold was considered as NT$2,011,950 (US$67 065, 1USD?=30 NTD in 2015).

Results: The model showed that adjuvant trastuzumab treatment in HER-2/neu positive early breast cancer yielded 1.631 quality-adjusted life-years (QALY) compared with no trastuzumab treatment. The ICER was US $51,863 per QALY gained in the base-case scenario. The Monte Carlo simulation by varying all variables simultaneously demonstrated that the probability of cost-effectiveness at the willingness-to-pay threshold of US$67,065 was 50% for 1-year adjuvant trastuzumab.

Conclusions: From this real-world study, 1-year adjuvant trastuzumab treatment is likely to be a cost-effective therapy for patients with HER-2 positive breast cancer at the willingness-to-pay threshold of 3-times GDP per capita in Taiwan.     

Cost-effectiveness of distributing naloxone to heroin users for lay overdose reversal in Russian cities

Abstract

Objective:

To evaluate the cost-effectiveness of distributing naloxone to illicit opioid users for lay overdose reversal in Russian cities.

Method:

This study adapted an integrated Markov and decision analytic model to Russian cities. The model took a lifetime, societal perspective, relied on published literature, and was calibrated to epidemiologic findings.

Results:

For each 20% of heroin users reached with naloxone distribution, the model predicted a 13.4% reduction in overdose deaths in the first 5 years and 7.6% over a lifetime; on probabilistic analysis, one death would be prevented for every 89 naloxone kits distributed (95% CI?=?32–260). Naloxone distribution was cost-effective in all deterministic and probabilistic sensitivity analyses and cost-saving if resulting in a reduction in overdose events. Naloxone distribution increased costs by US$13 (95% CI?=?US$3–US$32) and QALYs by 0.137 (95% CI?=?0.022–0.389) for an incremental cost of US$94 per QALY gained (95% CI?=?US$40–US$325). In a worst-case scenario where overdose was rarely witnessed and naloxone was rarely used, minimally effective, and expensive, the incremental cost was US$1987 per QALY gained. If national expenditures on drug-related HIV, tuberculosis, and criminal justice were applied to heroin users, the incremental cost was US$928 per QALY gained.

Conclusions:

Naloxone distribution to heroin users for lay overdose reversal is highly likely to reduce overdose deaths in target communities and is robustly cost-effective, even within the constraints of this conservative model.     

The cost-effectiveness of levodopa/carbidopa intestinal gel compared to standard care in advanced Parkinson’s disease

Background: Parkinson’s disease (PD) is an incurable, progressive neurological condition, with symptoms impacting movement, walking, and posture that eventually become severely disabling. Advanced PD (aPD) has a significant impact on quality-of-life (QoL) for patients and their caregivers/families. Levodopa/carbidopa intestinal gel (LCIG) is indicated for the treatment of advanced levodopa-responsive PD with severe motor fluctuations and hyper-/dyskinesia when available combinations of therapy have not given satisfactory results.

Aims: To determine the cost-effectiveness of LCIG vs standard of care (SoC) for the treatment of aPD patients.

Methods: A Markov model was used to evaluate LCIG vs SoC in a hypothetical cohort of 100 aPD patients with severe motor fluctuations from an Irish healthcare perspective. Model health states were defined by Hoehn &; Yahr (H&;Y) scale—combined with amount of time in OFF-time—and death. SoC comprised of standard oral therapy?±?subcutaneous apomorphine infusion and standard follow-up visits. Clinical efficacy, utilities, and transition probabilities were derived from published studies. Resource use was estimated from individual patient-level data from Adelphi 2012 UK dataset, using Irish costs, where possible. Time horizon was 20 years. Costs and outcomes were discounted at 4%. Both one-way and probabilistic sensitivity analyses were conducted.

Results: The incremental cost-effectiveness ratio for LCIG vs SOC was €26,944/quality adjusted life year (QALY) (total costs and QALYs for LCIG vs SoC: €537,687 vs €514,037 and 4.37 vs 3.49, respectively). LCIG is cost-effective at a payer threshold of €45,000. The model was most sensitive to health state costs.

Conclusion: LCIG is a cost-effective treatment option compared with SoC in patients with aPD.     

A long-term analysis evaluating the cost-effectiveness of biphasic insulin lispro mix 75/25 and mix 50/50 versus long-acting basal insulin analogs in the United States

Abstract

Objective:

To evaluate the cost-effectiveness of biphasic insulin lispro mix 75/25 (LM75/25) and mix 50/50 (LM50/50) compared with a long-acting analog insulin (LAAI) regimen from the perspective of a US healthcare payer.

Methods:

A published computer simulation model of diabetes was used to evaluate the cost-effectiveness of LM75/25 and LM50/50 vs a LAAI (insulin glargine) from the perspective of a US healthcare payer. Treatment effects in terms of HbA1c benefits were taken from a recent meta-analysis. Direct medical costs including pharmacy, complication, and patient management costs were obtained from published sources. All costs were expressed in 2010 US dollars and future costs and clinical benefits were discounted at 3% per annum. Sensitivity analyses were performed.

Results:

LM75/25 and LM50/50 were associated with improvements in life expectancy of 0.08 and 0.09 years, improvements in quality-adjusted life expectancy of 0.07 quality-adjusted life years (QALYs) and 0.08 QALYs and increases in cost of US$ 1724 and US$ 1720, respectively, when compared with LAAI.

Limitations:

The base case analysis did not capture mild or serious hypoglycemia on the grounds that the hypoglycemia rate odds ratios failed to reach statistical significance in the meta-analysis. In addition, the baseline cohort characteristics were based on an insulin-naïve population, as opposed to the cohorts in the meta-analysis, which were heterogeneous with regard to insulin treatment history.

Conclusions:

Based on a recently published meta-analysis, biphasic analog insulins are likely to improve clinical outcomes and reduce costs vs LAAIs in the long-term treatment of type 2 diabetes patients in the US.     

Cost-effectiveness of percutaneous closure of a patent foramen ovale compared with medical management in patients with a cryptogenic stroke: from the US payer perspective

Abstract

Aims: To evaluate the cost-effectiveness of percutaneous patent foramen ovale (PFO) closure, from a US payer perspective. Lower rates of recurrent ischemic stroke have been documented following percutaneous PFO closure in properly selected patients. Stroke in patients aged <60?years is particularly interesting because this population is typically at peak economic productivity and vulnerable to prolonged disability.

Materials and methods: A Markov model comprising six health states (Stable after index stroke, Transient ischemic attack, Post-Transient Ischemic Attack, Clinical ischemic stroke, Post-clinical ischemic stroke, and Death) was constructed to evaluate the cost-effectiveness of PFO closure in combination with medical management versus medical management alone. The base-case model employed a 5-year time-horizon, with transition probabilities, clinical inputs, costs, and utility values ascertained from published and national costing sources. Incremental cost-effectiveness ratio (ICER) was evaluated per US guidelines, utilizing a discount rate of 3.0%.

Results: At 5?years, overall costs and quality-adjusted life-years (QALYs) obtained from PFO closure compared with medical management were $16,323 vs $7,670 and 4.18 vs 3.77, respectively. At 5?years, PFO closure achieved an ICER of $21,049, beneficially lower than the conventional threshold of $50,000. PFO closure reached cost-effectiveness at 2.3?years (ICER = $47,145). Applying discount rates of 0% and 6% had a negligible impact on base-case model findings. Furthermore, PFO closure was 95.4% likely to be cost-effective, with a willingness-to-pay (WTP) threshold of $50,000 and a 5-year time horizon.

Limitations: From a cost perspective, our economic model employed a US patient sub-population, so cost data may not extrapolate to other non-US stroke populations.

Conclusion: Percutaneous PFO closure plus medical management represents a cost-effective approach for lowering the risk of recurrent stroke compared with medical management alone.     

A cost-effectiveness analysis of denosumab for the prevention of skeletal-related events in patients with multiple myeloma in the United States of America

Objective: A large, pivotal, phase 3 trial in patients with newly diagnosed multiple myeloma (MM) demonstrated that denosumab, compared with zoledronic acid, was non-inferior for the prevention of skeletal-related events (SREs), extended the observed median progression-free survival (PFS) by 10.7 months, and showed significantly less renal toxicity. The cost-effectiveness of denosumab vs zoledronic acid in MM in the US was assessed from societal and payer perspectives.

Methods: The XGEVA Global Economic Model was developed by integrating data from the phase 3 trial comparing the efficacy of denosumab with zoledronic acid for the prevention of SREs in MM. SRE rates were adjusted to reflect the real-world incidence. The model included utility decrements for SREs, administration, serious adverse events (SAEs), and disease progression. Drug, administration, SRE management, SAEs, and anti-MM treatment costs were based on data from published studies. For the societal perspective, the model additionally included SRE-related direct non-medical costs and indirect costs. The net monetary benefit (NMB) was calculated using a willingness-to-pay threshold of US$150,000. One-way deterministic and probabilistic sensitivity analyses were conducted.

Results: From a societal perspective, compared with zoledronic acid, the use of denosumab resulted in an incremental cost of US$26,329 and an incremental quality-adjusted life-year (QALY) of 0.2439, translating into a cost per QALY gained of US$107,939 and a NMB of US$10,259 in favor of denosumab. Results were sensitive to SRE rates and PFS parameters.

Limitations: Costs were estimated from multiple sources, which varied by tumor type, patient population, country, and other parameters. PFS and overall survival were extrapolated beyond the follow-up of the primary analysis using fitted parametric curves.

Conclusion: Denosumab’s efficacy in delaying or preventing SREs, potential to improve PFS, and lack of renal toxicity make it a cost-effective option for the prevention of SREs in MM compared with zoledronic acid.     

Pharmacoeconomic analysis of paricalcitol and calcitriol in the treatment of secondary hyperparathyroidism in haemodialysis: impact of hospitalisations and survival

Summary

The objective of this study was to evaluate the cost effectiveness of paricalcitol injection compared with calcitriol injection when used to reduce parathyroid hormone levels in patients undergoing haemodialysis. A decision tree was developed to model the 1-year costs and outcomes of therapy for secondary hyperparathyroidism from a US government payer's perspective (2005 US$). Probabilities of hospitalisations and survival with paricalcitol and calcitriol were obtained from published observational studies.

When only drug costs and survival were considered, the incremental cost effectiveness of paricalcitol over calcitriol was $9,900 per life saved. When utilities were included, the incremental cost-effectiveness ratio for paricalcitol compared with calcitriol was $13,200 per quality-adjusted life year. When both drug and hospitalisation costs were included in a cost analysis, paricalcitol treatment was cost saving compared with calcitriol, and when hospitalisation costs were included in both the cost-effectiveness analysis and cost-utility analysis paricalcitol demonstrated first-order dominance, cost savings and cost effectiveness.

This decision analysis demonstrated that paricalcitol injection is both cost effective and cost saving compared with calcitriol injection.     

Within trial cost-utility analysis of disease management program for patients hospitalized with atrial fibrillation: results from the SAFETY trial

Abstract

Background: The potential impact of disease management to optimize quality of care, health outcomes, and total healthcare costs across a range of cardiac disease states is unknown.

Methods: A trial-based cost-utility analysis was conducted alongside a randomized controlled trial of 335 patients with chronic, non-valvular AF (without heart failure; the SAFETY Trial) discharged to home from three tertiary referral hospitals in Australia. A home-based disease management intervention (the SAFETY intervention) that involved community-based AF care including home visits was compared to routine primary healthcare and hospital outpatient follow-up (standard management). Bootstrapped incremental cost-utility ratios were computed based on quality-adjusted life-years (QALYs) and total healthcare costs. Cost-effectiveness acceptability curves were constructed to explore the probability of the SAFETY intervention being cost-effective. Sub-group analyses were performed based on age and sex to determine differential cost-effectiveness.

Results: During median follow-up of 1.75?years, the SAFETY intervention was associated with a non-statistically significant increase in QALYs (0.02 per person) and lower total healthcare costs (–$4,375 per person). Although each of these findings were not statistically significant, the SAFETY intervention was found to be dominant (more effective and cost saving) in 58.8% of the bootstrapped iterations and cost-effective (more effective and gains in QALYs achieved at or below $50,000 per QALY gained) in 61.5% of the iterations. Males and those aged less than 78?years achieved greater gains in QALYs and savings in healthcare costs. The estimated value of perfect information in Australia (the monetized value of removing uncertainty in the cost-effectiveness results) was A$51 million, thus demonstrating the high potential gain from further research.

Conclusions: Compared with standard management, the SAFETY intervention is potentially a dominant strategy for those with chronic, non-valvular AF. However, there would be substantial value in reducing the uncertainty in these estimates from further research.

Trial registration: Australian New Zealand Clinical Trials Registry identifier: ACTRN12610000221055.     

Cost-effectiveness of mechanical thrombectomy for acute ischemic stroke: an Australian payer perspective

Aims: The goal of this study was to assess the cost-effectiveness of mechanical thrombectomy (MT) for acute ischemic stroke (AIS) from an Australian payer perspective.

Methods: This study used a Markov model that employed a life-time time horizon, modeling patients from symptom onset of stroke until end of life. Clinical efficacy and safety data were taken from an individual patient level data (IPD) meta-analysis of clinical studies. The treatment effect of MT compared to usual care was measured by changes in modified Rankin Score (mRS). Post-treatment mRS scores were used to determine short- and long-term stroke care costs. Treatment costs were modeled, with health state utility values determined by literature review. All analyses were conducted using Microsoft Excel.

Results: In comparison to usual care, MT is associated with higher costs ($10,666 per patient) and additional quality-adjusted life years (QALYs) (0.8281 per patient), resulting in an incremental cost per QALY of $12,880. Sensitivity analyses demonstrated the reliability of the base case results across a range of assumptions. The higher cost associated with MT is, to an extent, offset by the cost savings resulting from lower stroke care costs due to improved patient outcomes. The life-time cost savings in terms of stroke care costs are estimated to be more than $8,000 per patient for patients who had received MT in combination with usual care.

Limitations: Stroke care costs based on patient disability/functional level were not available and were derived. As a consequence, long-term care costs for patients with poorer outcomes may be under-estimated. Patient outcomes at 90 days were extrapolated to a lifetime horizon, but this approach was supported by long-term evidence on stroke survival.

Conclusions: Mechanical thrombectomy is a cost-effective treatment option for AIS, with clinical benefits translating to short- and long-term cost benefits. This analysis supports rapid update of stroke care pathways to incorporate this therapy as a treatment option.     

Cost-effectiveness analysis of tissue plasminogen activator in acute ischemic stroke in Iran

Aims: Tissue plasminogen activator (tPA) is used to treat acute ischemic stroke up to 4.5?h after symptom onset. Its cost-effectiveness in developing countries is not specified yet. This study aimed to study cost-effectiveness of tPA in Iran.

Methods: This is a cost-effectiveness analysis from the perspective of the third party payer to compare IV tPA with no tPA of ischemic stroke. A Markov model with a lifetime horizon was used to analyze the costs and outcomes. Cost data were extracted from the 94 patients admitted in two hospitals in Iran. All costs were calculated based on US dollars in 2016. Quality-adjusted life years (QALY) were extracted from previously published literature. Cost-effectiveness was determined by calculating ICER by TreeAge Pro 2011 software.

Results: Lifetime costs of no tPA strategy were higher than tPA ($10,718 in the no tPA group compared with $8,796 in the tPA group). The tPA arm gained 0.20 QALY compared with no tPA. ICER was $8,471 per QALY. ICER value suggests that tPA is cost-effective compared with no tPA.

Limitations: The limitations of the present study are the reliance on calculated QALY value of other countries and difficulty in accessing patients treated with tPA.

Conclusions: The balance of hospitalization and rehabilitation costs and QALYs support the conclusion that treatment with intravenous tPA in the 4.5-h time window is cost-effective from the perspectives of the third party payer and inclusion of tPA in the insurance benefit package being reasonable.     

Cost-effectiveness of a pentavalent human–bovine reassortant rotavirus vaccine for children ≤5 years of age in Taiwan

Abstract

Objective:

A Markov model was used to assess the impact of RV5, a pentavalent (G1, G2, G3, G4, P1A[8]) human bovine (WC3 strain) reassortant rotavirus vaccine, on reducing the healthcare burden and cost associated with rotavirus gastroenteritis (RGE) in Taiwan. Other cost-effectiveness analyses for rotavirus vaccination in industrialized countries have produced varying results depending on the input parameters assumed.

Methods:

Vaccination with RV5 is compared to no vaccination in a hypothetical cohort of Taiwanese children during their first 5 years of life to determine the per dose prices at which vaccination would be cost neutral or provide good value based on established standards from the healthcare (direct medical care costs only) and societal (all RGE-related costs) perspectives. The effects of vaccination on RGE healthcare utilization and days of parental work loss missed are based on results from the Rotavirus Efficacy and Safety Trial.

Results:

Without vaccination there would be 122,526 symptomatic episodes of RGE. Universal vaccination would reduce RGE-related deaths, hospitalizations, emergency department, and outpatient visits by 91.7%, 92.1%, 83.7%, and 73.4%, respectively. The price per dose at which vaccination would be cost-neutral is US$ 21.80 (688 NTD) and US$ 26.20 (827 NTD) from the healthcare and societal perspectives, respectively. At $25 per dose, the cost per QALY gained is US$ 2261 (71,335 NTD) from the healthcare perspective and cost saving from the societal perspective.

Key limitations:

The model only assesses the effect of RV5 on vaccinated children and does not account for herd immunity. However, given that high levels of coverage are anticipated in Taiwan, the effects of herd immunity are likely to be short-term.

Conclusion:

A pentavalent rotavirus vaccination program is likely to substantially reduce the healthcare burden associated with rotavirus gastroenteritis at a cost per QALY ratio within the range defined as cost-effective.     

Long-term cost-consequence analysis of exenatide once weekly vs sitagliptin or pioglitazone for the treatment of type 2 diabetes patients in the United States

Abstract

Objective:

Exenatide once-weekly (ExQW) is a GLP-1 receptor agonist shown to lower glucose and cardiovascular risk factors in patients with type 2 diabetes mellitus (T2DM). The objective of this study was to estimate the clinical benefits and associated economic benefits of treatment with ExQW compared with sitagliptin or pioglitazone in the US.

Methods:

The IMS CORE Diabetes Model, a validated computer simulation model, was used to project lifetime clinical outcomes and complication costs. The costs of glucose-lowering drugs were excluded as not all prices were available. Baseline patient characteristics (mean values: age, 52.5 years; diabetes duration, 6 years; HbA1_c, 8.51%; body mass index, 32.12?kg/m²) and clinical data were derived from a phase 3 clinical trial that compared ExQW with sitagliptin or pioglitazone in T2DM patients. At 6 months, patients treated with ExQW had greater improvements in HbA1_c and body weight than those treated with sitagliptin or pioglitazone. Complication costs were extracted from published sources. Health outcomes and costs were discounted at 3% per year. Sensitivity analyses were performed.

Results:

Over 35 years, and compared with sitagliptin or pioglitazone, ExQW increased life expectancy by, respectively, 0.28 (13.76?±?0.17 vs 13.48?±?0.18) and 0.17 years (13.76?±?0.17 vs 13.59?±?0.17), and quality-adjusted life years by, respectively, 0.28 (9.56?±?0.12 vs 9.28?±?0.12) and 0.24 years (9.56?±?0.12 vs 9.32?±?0.12). ExQW was associated with lower lifetime complication costs: compared with sitagliptin or pioglitazone, ExQW saved, respectively US$2215 (US$55,647?±?2039 vs US$57,862?±?2159) and US$933 (US$55,647?±?2039 vs US$56,580?±?2007) direct cost per patient. Cost-savings resulted mainly from a lower projected cumulative incidence of cardiovascular diseases and neuropathic complications.

Limitations:

Short-term changes in surrogate end-points were used to project lifetime effects on clinical outcomes. Pharmacy costs were excluded from the analyses.

Conclusions:

Over a patient’s lifetime, ExQW was projected to improve health and decrease diabetes-related complication costs compared with sitagliptin or pioglitazone.