Cost-effectiveness of add-on empagliflozin versus standard of care in management of CKD in Malaysia, Thailand and Vietnam - findings from a modelling study assessing an EMPA-KIDNEY eligible population, using CKD progression model.

BACKGROUND AND OBJECTIVES: Nearly one in ten individuals in South-East Asia are estimated to be affected by chronic kidney disease (CKD). The burden of end-stage kidney disease is significant and can be heavy on the healthcare system. The recent EMPA-KIDNEY trial demonstrated a significant reduction in the risk of kidney disease progression or cardiovascular death in patients with CKD with a broad range of kidney function using add-on empagliflozin versus standard of care (SoC) alone. The objective of this study was to estimate the economic benefit of empagliflozin for patients with CKD in Malaysia, Thailand and Vietnam. METHODS: An individual patient level simulation model with an annual cycle that estimates the progression of kidney function and associated risk-factors was employed. Local costs and mortality rates were estimated from a wide range of published literature. A healthcare perspective was used over a 50-year time horizon. RESULTS: The use of add-on empagliflozin versus SoC alone was found to be cost-saving in Malaysia and Thailand and cost-effective (ICER: 77,838,407 Vietnam Dong/QALY vs. a willingness to pay threshold of 96,890,026/QALY) in Vietnam. The bulk of the costs avoided over a lifetime is derived from the prevention or delay of dialysis initiation or kidney transplant - the cost offsets were nearly twice the additional treatment cost. The results were similar in patients with and without diabetes and across broad range of albuminuria. CONCLUSIONS: The use of add-on empagliflozin in a broad population of patients with CKD is expected to be cost-saving in Malaysia and Thailand and cost-effective in Vietnam and will help alleviate the increasing burden of CKD in the region.

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.

Assessment of clinical and economic impact of rivaroxaban plus aspirin vs. aspirin alone as a secondary prophylaxis in patients with chronic and symptomatic peripheral arterial disease in the United States.

AIM: The objective in this study was to assess the clinical and economic implications of the inclusion of rivaroxaban as a secondary prophylaxis in patients with chronic or symptomatic peripheral artery disease (PAD) in the United States (US). METHODS: A cost-consequence model was adapted to evaluate the economic impact of rivaroxaban plus aspirin in a hypothetical 1-million-member health plan. The model inputs were taken from multiple sources: efficacy and safety of rivaroxaban + aspirin vs. aspirin alone were abstracted from COMPASS and VOYAGER randomized clinical trials; the prevalence of chronic and symptomatic PAD and incidence rates of clinical events (major adverse cardiac events [MACE], major adverse limb events [MALE], and major bleeding), were abstracted from the analysis of claims data; healthcare costs of clinical events and wholesale acquisition costs for rivaroxaban were abstracted from the literature and Red Book, respectively (2022 USD). One-way sensitivity analyses and subgroup analyses were also conducted. RESULTS: Over one year, with a 5% uptake of rivaroxaban, the model estimated rivaroxaban + aspirin to reduce 21 MACE/MALE events in the PAD patient population. The reduction in these clinical events offsets the increased risk of major bleeding (16 additional events), demonstrating a positive health benefit of the rivaroxaban addition. These benefits led to a $0.27 incremental cost per member per month (PMPM) to a US plan. The major driver of the incremental cost was the cost of rivaroxaban. In a subgroup of patients with the presence of any high-risk factor (heart failure, diabetes, renal insufficiency, or history of vascular disease affecting two or more vascular beds), the incremental PMPM cost was $0.13. CONCLUSIONS: Rivaroxaban + aspirin was found to provide positive net clinical benefit on the annual number of MACE/MALE avoided, with a modest increase in the PMPM cost.

Cost-effectiveness of volume computed tomography in lung cancer screening: a cohort simulation based on Nelson study outcomes.

OBJECTIVES: This study aimed to evaluate the cost-effectiveness of lung cancer screening (LCS) with volume-based low-dose computed tomography (CT) versus no screening for an asymptomatic high-risk population in the United Kingdom (UK), utilising the long-term insights provided by the NELSON study, the largest European randomized control trial investigating LCS. METHODS: A cost-effectiveness analysis was conducted using a decision tree and a state-transition Markov model to simulate the identification, diagnosis, and treatments for a lung cancer high-risk population, from a UK National Health Service (NHS) perspective. Eligible participants underwent annual volume CT screening and were compared to a cohort without the option of screening. Screen-detected lung cancers, costs, quality-adjusted life years (QALYs), and the incremental cost-effectiveness ratio (ICER) were predicted. RESULTS: Annual volume CT screening of 1.3 million eligible participants resulted in 96,474 more lung cancer cases detected in early stage, and 73,825 fewer cases in late stage, leading to 53,732 premature lung cancer deaths averted and 421,647 QALYs gained, compared to no screening. The ICER was £5,455 per QALY. These estimates were robust in sensitivity analyses. LIMITATIONS: Lack of long-term survival data for lung cancer patients; deficiency in rigorous micro-costing studies to establish detailed treatment costs inputs for lung cancer patients. CONCLUSIONS: Annual LCS with volume-based low-dose CT for a high-risk asymptomatic population is cost-effective in the UK, at a threshold of £20,000 per QALY, representing an efficient use of NHS resources with substantially improved outcomes for lung cancer patients, as well as additional societal and economic benefits for society as a whole. These findings advocate evidence-based decisions for the potential implementation of a nationwide LCS in the UK.

Cost-effectiveness of alternative first- and second-line treatments for benign prostatic hyperplasia in Singapore.

BACKGROUND: Minimally invasive surgical therapies, such as water vapor thermal therapy (WVTT) and prostatic urethral lift (PUL), are typically second-line options for patients in whom medical management (MM) failed but who are unwilling or unsuitable to undergo invasive transurethral resection of the prostate (TURP). However, the incremental cost-effectiveness of WVTT or PUL as first- or second-line therapy is unknown. We evaluated the incremental cost-effectiveness of alternative first- and second-line treatments for patients with moderate-to-severe benign prostatic hyperplasia (BPH) in Singapore to help policymakers make subsidy decisions based on value for money. METHODS: We considered six stepped-up treatment strategies, beginning with MM, WVTT, PUL or TURP. In each strategy, patients requiring retreatment advance to a more invasive treatment until TURP, which may be undergone twice. A Markov cohort model was used to simulate transitions between BPH severity states and retreatment, accruing costs and quality-adjusted life-years (QALYs) over a lifetime horizon. RESULTS: In moderate patients, strategies beginning with MM had similar cost and effectiveness, and first-line WVTT was incrementally cost-effective to first-line MM (33,307 SGD/QALY). First-line TURP was not incrementally cost-effective to first-line WVTT (159,361 SGD/QALY). For severe patients, WVTT was incrementally cost-effective to MM as a first-line treatment (30,133 SGD/QALY) and to TURP as a second-line treatment following MM (6877 SGD/QALY). TURP was incrementally cost-effective to WVTT as a first-line treatment (48,209 SGD/QALY) in severe patients only. All pathways involving PUL were dominated (higher costs and lower QALYs). CONCLUSION: Based on the common willingness-to-pay threshold of SGD 50,000/QALY, this study demonstrates the cost-effectiveness of WVTT over MM as first-line treatment for patients with moderate or severe BPH, suggesting it represents good value for money and should be considered for subsidy. PUL is not cost-effective as a first- nor second-line treatment. For patients with severe BPH, TURP as first-line is also cost-effective.

Benign prostatic hyperplasia (BPH) is a non-cancerous enlargement of the prostate, common among older men. Its symptoms include difficulties with starting and completing urination, incontinence, frequent and urgent need to urinate. Minimally invasive procedures, such as water vapor thermal therapy (WVTT) and prostatic urethral lift (PUL), are typically offered as second-line options to patients for whom medication has failed but who are unwilling or unsuitable to undergo invasive surgery (transurethral resection of the prostate, TURP). However, whether offering these procedures as first-line options represents good value for money (i.e. cost-effectiveness) is an open question. To address this question and inform subsidy decisions in Singapore, we investigated six stepped-up treatment strategies which differ in first- and second-line treatments. For each strategy, we simulated healthcare costs and quality of life for a cohort of moderate and severe BPH patients over their lifetime, considering the possibility of treatment-related adverse effects and multiple rounds of retreatment. The incremental cost of a unit improvement in quality of life for a strategy relative to the next most expensive one was compared against a willingness-to-pay threshold to determine cost-effectiveness. We found that WVTT was cost-effective relative to medication as a first-line treatment for patients with moderate or severe BPH, suggesting it represents good value for money and should be considered for subsidy. PUL was not cost-effective as first- nor second-line treatment. TURP is cost-effective as first-line for severe BPH patients only.

Early exploration of the economic impact of transradial access (TRA) versus transfemoral access (TFA) for neurovascular procedures in Spain.

INTRODUCTION: Transfemoral access (TFA) is the primary access approach for neurointerventional procedures. Transradial access (TRA) is established in cardiology due to its lower complications, yet, it is at its early stages in neuroprocedures. This study performs an early exploration of the economic impact associated with the introduction of TRA in diagnostic and therapeutic neuroprocedures from the Spanish NHS perspective. METHODS: An economic model was developed to estimate the cost and clinical implications of using TRA compared to TFA. Costs considered access-related, complications and recovery time costs obtained from local databases and experts' inputs. Clinical inputs were sourced from the literature. A panel of eight experts from different Spanish hospitals, validated or adjusted the values based on local experience. Hypothetical cohorts of 10,000 and 1000 patients were considered for diagnostic and therapeutic neuroprocedures respectively. Deterministic sensitivity analysis was performed. RESULTS: TRA in diagnostic procedures was associated with lower costs with savings ranging between €486 and €157 depending on the TFA recovery time considered. TRA is estimated to lead to 158 fewer access-site complications. In therapeutic procedures, TRA resulted in 76.4 fewer complications and was estimated to be cost-neutral with an incremental cost of €21.56 per patient despite recovery times were not included for this group. Variation of the parameters in the sensitivity analysis did not change the direction of the results. LIMITATIONS: Clinical data was obtained from literature validated by experts therefore results generalizability is limited. In therapeutic neuroprocedures, there is an experience imbalance between approaches and recovery times were not included hence the total impact is not fully captured. CONCLUSIONS: The early economic model suggests that implementing TRA is associated with reduced costs and complications in diagnostic procedures. In therapeutic procedures, TRA lead to fewer complications and it is estimated to be cost-neutral, however its full potential still needs to be quantified.

Development of a health economic model to evaluate the cost-effectiveness of roxadustat in treating anemia associated with non-dialysis-dependent chronic kidney disease.

BACKGROUND: Treatment for anemia of chronic kidney disease (CKD) largely consists of erythropoiesis-stimulating agents (ESAs) with iron supplementation. Although ESAs are well-established and efficacious, their use has been associated with considerable economic and humanistic burdens. Roxadustat, an oral medication, is a hypoxia-inducible factor prolyl hydroxylase inhibitor that targets multiple causes of CKD and has a similar efficacy and safety profile to ESAs. The cost-effectiveness of this treatment, however, has yet to be investigated. OBJECTIVE: The study objective was to develop a health economic model to evaluate the cost-effectiveness of roxadustat compared with ESAs for treating anemia of non-dialysis-dependent (NDD) CKD. METHODS: A cohort-based model was developed for a hypothetical cohort of 1,000 patients with anemia of NDD CKD, incorporating eight health states, representing the hemoglobin level of each patient. The model was informed by individual patient-level data from the roxadustat global phase 3 clinical trial program. Total and incremental costs as well as quality-adjusted life-years (QALYs) associated with roxadustat versus ESAs were estimated from the perspective of the UK National Health Service. Sensitivity analyses were performed to assess the robustness of the model. Analyses exploring alternative scenarios were also conducted. RESULTS: On a per-person basis, over 1,000 simulations, roxadustat was found to be on average less costly (-£32) and more effective (+0.01 QALYs) than ESAs, with a dominant incremental cost-effectiveness ratio. The probability of cost-effectiveness at a £20,000 per QALY willingness-to-pay threshold from the UK perspective was 67%. CONCLUSION: The model developed may be a useful instrument that, alongside expert clinical opinion, can inform clinical and policy decision-making regarding treatment of anemia of NDD CKD. The model highlights the cost-effectiveness of roxadustat, as well as its potential to have a meaningful impact in reducing the burden of anemia of NDD CKD.

Cost-effectiveness of apixaban and rivaroxaban in thromboprophylaxis of cancer patients treated with chemotherapy in Spain.

BACKGROUND: Apixaban and rivaroxaban are two direct-acting oral anticoagulants (DOACs) recommended for thromboprophylaxis in cancer patients treated with chemotherapy in an ambulatory setting. We aimed to assess the cost-utility of thromboprophylaxis with apixaban and rivaroxaban vs no thromboprophylaxis in ambulatory cancer patients starting chemotherapy with an intermediate-to-high risk of venous thromboembolism (VTE), Khorana score ≥ 2 points. METHODS: A cost-effectiveness analysis was performed from the perspective of Spain's National Health System (NHS) using an analytical decision model in the short-term (180 days) and a Markov model in the long-term (5 years). Transition probabilities were obtained from randomized, double-blind, placebo-controlled clinical trials of apixaban and rivaroxaban in adult ambulatory patients with cancer at risk for VTE, treated with chemotherapy (AVERT and CASSINI trials). The costs (€2,021) were taken from Spanish sources. The utilities of the model were obtained through the EQ-5D questionnaire. Deterministic (base case) and probabilistic (second-order Monte Carlo simulation) analyses were conducted. RESULTS: In the probabilistic sensitivity analysis, apixaban generated a cost per patient of €1,082 ± 187, with a 95% confidence interval (CI) of €713-1,442, while no prophylaxis produced a cost per patient of €1,146 ± 218, with a 95% CI of €700-1,491, with a saving of €64 per patient and a gain of 0.008 QALYs. Likewise, rivaroxaban provided a cost per patient of €993 ± 133, with a 95% CI of €748-1,310, while no prophylaxis produced a cost per patient of €872 ± 152, with a 95% CI of €602-1,250, with an additional expense of €121 per patient and a gain of 0.008 QALYs. CONCLUSIONS: In thromboprophylaxis of cancer patients, the use of apixaban and rivaroxaban generated similar costs compared to non-prophylaxis, without the difference found being statistically significant, with a clinically insignificant QALY gain.

Estimated health economic impact of conducting urine albumin-to-creatinine ratio testing alongside estimated glomerular filtration rate testing in the early stages of chronic kidney disease in patients with type 2 diabetes.

AIM: To estimate the health economic impact of undertaking urine albumin-to-creatinine ratio (UACR) testing versus no UACR testing in early stages of chronic kidney disease (CKD) progression in patients with type 2 diabetes (T2D). METHODS: An economic model, taking a UK healthcare system perspective, estimated the impact of UACR testing on additional costs, clinical benefits measured as prevented dialyses and cardiovascular-related deaths, life years gained (LYg), LYg before kidney failure, and incremental cost-effectiveness ratio (ICER). Sixteen of the 18 Kidney Disease: Improving Global Outcomes (KDIGO) heatmap categories were considered separately, and grouped in health states according to CKD risk. Results were derived for current standard-of-care and emerging CKD therapies. RESULTS: The cohort that adhered to both UACR and estimated glomerular filtration rate (eGFR) testing guidelines in early stages of CKD (n = 1000) was associated with approximately 500 LYg before kidney failure onset; costing approximately £2.5 M. ICERs across the KDIGO heatmap categories were approximately £5,000. LIMITATIONS: This model used data from a comprehensive meta-analysis that was initiated more than 10 years ago (2009). While this was the most comprehensive source identified, recent changes in the treatment landscape, patient population and social determinants of CKD will not be captured. Furthermore, a narrow approach was taken, aligning included costs with UK NHS reference materials. This means that some direct and indirect drivers of costs in late-stage disease have been excluded. CONCLUSIONS: UACR testing in the early stages of CKD is cost effective in T2D patients. Emerging therapies with the potential to slow CKD progression, mean that optimal monitoring through UACR/eGFR testing will become increasingly important for accurate identification and timely treatment initiation, particularly for the highest-risk A3 category.

Cost-effectiveness of tezepelumab in Canada for severe asthma.

AIMS: To assess the cost-effectiveness of tezepelumab as add-on maintenance therapy compared with standard of care (SoC) for the treatment of patients with severe asthma in Canada. MATERIAL AND METHODS: A cost utility analysis was conducted using a Markov cohort model with five health states ("controlled asthma", "uncontrolled asthma", "previously controlled asthma with exacerbation", "previously uncontrolled asthma with exacerbation", and "death"). Tezepelumab plus SoC was compared to SoC (high-dose inhaled corticosteroids plus long-acting beta agonist) using efficacy estimates derived from the NAVIGATOR (NCT03347279) and SOURCE (NCT03406078) trials. The model included the costs of therapy, administration, resource use for disease management, and adverse events. Utility estimates were calculated using a mixed-effects regression analysis of the NAVIGATOR and SOURCE trials. A Canadian public payer perspective was used with a 50-year time horizon, a 1.5% annual discount rate, and the base case analysis was conducted probabilistically. A key scenario analysis assessed the cost-effectiveness of tezepelumab compared with currently reimbursed biologics informed by an indirect treatment comparison. RESULTS: The base case analysis suggested that tezepelumab plus SoC was associated with a quality-adjusted life-year (QALY) gain of 1.077 compared with SoC alone at an incremental cost of $207,101 (2022 Canadian dollars), resulting in an incremental cost-utility ratio of $192,357/QALY. The key scenario analysis demonstrated that tezepelumab was dominant against all currently reimbursed biologics, with higher incremental QALYs (ranging from 0.062 to 0.407) and lower incremental costs (ranging from -$6,878 to -$1,974). Additionally, when compared against currently reimbursed biologics in Canada, tezepelumab had the highest probability of being cost-effective across all willingness-to-pay (WTP) thresholds. CONCLUSION: Tezepelumab provided additional life years and QALYs at additional cost compared with SoC in Canada. In addition, tezepelumab dominated (i.e. more effective, less costly) the other currently reimbursed biologics.

Cost-effectiveness of selective internal radiation therapy with Y-90 resin microspheres for intermediate- and advanced-stage hepatocellular carcinoma in Brazil.

AIMS: Hepatocellular carcinoma (HCC) is a severe condition with poor prognosis that places a significant burden on patients, caregivers, and healthcare systems. Selective internal radiation therapy (SIRT) is a treatment available to patients with HCC which addresses some of the limitations of alternative treatment options. A cost-effectiveness analysis was undertaken into the use of SIRT using Y-90 resin microspheres for the treatment of unresectable intermediate- and late-stage HCC in Brazil. MATERIALS AND METHODS: A partitioned-survival model was developed, including a tunnel state for patients downstaged to receive treatments with curative intent. Sorafenib was the selected comparator, a common systemic treatment in Brazil and for which comparative evidence exists. Clinical data were extracted from published sources of pivotal trials, and effectiveness was measured in quality-adjusted life-years (QALYs) and life-years (LYs). The analysis was conducted from the Brazilian private payer perspective and a lifetime horizon was implemented. Comprehensive sensitivity analyses were conducted. RESULTS: LYs and QALYs were higher for SIRT with Y-90 resin microspheres versus sorafenib (0.27 and 0.20 incremental LYs and QALYs, respectively) and costs were slightly higher for SIRT (R$15,864). The base case incremental cost-effectiveness ratio (ICER) was R$77,602 per QALY. The ICER was mostly influenced by parameters defining the sorafenib overall survival curve and SIRT had a 73% probability of being cost-effective at a willingness-to-pay threshold of R$135,761 per QALY (3-times the per-capita gross domestic product in Brazil). Overall, sensitivity analyses confirmed the robustness of the results indicating that SIRT with Y-90 resin microspheres is cost-effective compared with sorafenib. LIMITATIONS: A rapidly evolving treatment landscape in Brazil and worldwide, and the lack of local data for some variables were the main limitations. CONCLUSIONS: SIRT with Y-90 resin microspheres is a cost-effective option compared with sorafenib in Brazil.

Cost-effectiveness of a novel, non-active implantable device as a treatment for refractory gastro-esophageal reflux disease.

AIMS: Gastro-esophageal reflux disease (GERD) is a common, chronic gastrointestinal condition characterized by heartburn, chest pain, regurgitation, and bloating. The current standard of care includes chronic treatment with proton pump inhibitors (PPIs) or, in selected patients, laparoscopic anti-reflux surgery. RefluxStop is a novel implantable device indicated for GERD patients eligible for laparoscopic surgical treatment. The aim of this analysis was to assess the cost-effectiveness of RefluxStop against available treatment options for GERD. MATERIAL AND METHODS: A Markov model was developed to assess the cost-effectiveness of RefluxStop compared with PPI-based medical management (MM) and two surgical management options, LNF and magnetic sphincter augmentation (MSA, LINX system), in people with GERD. Clinical outcomes and costs were estimated over a lifetime horizon from the UK National Health Service perspective and an annual discount rate of 3.5% was applied. RESULTS: RefluxStop showed favorable surgical outcomes compared with both LNF and MSA. The base case incremental cost-effectiveness ratios compared with MM, LNF, and MSA were £4,156, £6,517, and £249 per QALY gained, respectively. At the UK cost-effectiveness threshold of £20,000 per QALY gained, the probability that RefluxStop was cost-effective against MM, LNF, and MSA was 100%, 93%, and 100%, respectively. LIMITATIONS: The model presented the results of a comparison, with evidence for RefluxStop derived from its single-arm CE mark trial and that for comparators from the literature. The varied clinical care pathway of individual GERD patients was necessarily simplified for modeling purposes, and necessary assumptions were made; however, the model results proved robust to sensitivity analyses. CONCLUSIONS: Introduction of RefluxStop was estimated to extend life expectancy and improve quality-of-life of GERD patients when compared with MM, LNF, and MSA. The results of the cost-effectiveness analysis demonstrated that RefluxStop is highly likely to be a cost-effective treatment option within NHS England.

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.

AIMS: Approximately 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 METHODS: A 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. RESULTS: The 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. LIMITATIONS: The 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. CONCLUSIONS: Intravesical BCG led to increased QALYs and reduced costs versus RC for patients with high-risk NMIBC from the UK healthcare payer perspective.

Intravesical Bacillus Calmette-Guérin (BCG) is a recommended immunotherapy option for patients with high-risk non-muscle invasive bladder cancer (NMIBC) who have undergone transurethral resection of bladder tumor (TURBT). BCG is known for its high efficacy and good safety profile. However, current evidence on its effectiveness against other comparators such as radical cystectomy (RC) is limited, mainly because different BCG schedules are used, particularly with regard to maintenance therapy. Given this lack of evidence, we conducted the first cost-utility analysis which considers adequate BCG therapy relative to RC for the UK. We developed a Markov model that captured the effects of the intravesical BCG and RC on NMIBC, in addition to incidences of adverse events associated with either treatment. Costs of the two treatments, their administration, maintenance, and of treatments for adverse events were modelled alongside the quality-of-life effects of NMIBC, adverse events, and palliative care. We used published clinical data and UK cost data to inform our model. Our results show that BCG was associated with higher quality-adjusted life expectancy than RC and lower total costs from the healthcare system perspective. These results imply that adequate BCG immunotherapy is likely valuable for the National Healthcare System in terms of its effects on patients and indeed cost-saving relative to RC.

Cost-effectiveness of influenza vaccination with a high dose quadrivalent vaccine of the elderly population in Belgium, Finland, and Portugal.

BACKGROUND: Seasonal influenza may result in severe outcomes, resulting in a significant increase of hospitalizations during the winter. To improve the protection provided by the standard dose influenza quadrivalent vaccine (SDQIV), a high-dose vaccine (HDQIV) has been developed specifically for adults aged 60 and older who are at higher risk of life-threatening complications. OBJECTIVES: The aim of this study was to determine the cost-effectiveness of HD QIV vs. SD-QIV in the recommended population of three European countries: Belgium, Finland and Portugal. METHODS: A cost-utility analysis comparing HDQIV vs. SDQIV was conducted using a decision tree estimating health outcomes conditional on influenza: cases, general practitioner and emergency department visits, hospitalizations and deaths. To account for the full benefit of the vaccine, an additional outcome-hospitalizations attributable to influenza-was also evaluated. Demographic, epidemiological and economic inputs were based on the respective local data. HDQIV relative vaccine efficacy vs. SDQIV was obtained from a phase IV efficacy randomized clinical trial. The incremental cost-effectiveness ratios (ICER) were computed for each country, and a probabilistic sensitivity analysis (1,000 simulations per country) was performed to assess the robustness of the results. RESULTS: In the base case analysis, HDQIV resulted in improved health outcomes (visits, hospitalizations, and deaths) compared to SDQIV. The ICERs computed were 1,397, 9,581, and 15,267 €/QALY, whereas the PSA yielded 100, 100, and 84% of simulations being cost-effective at their respective willingness-to-pay thresholds, for Belgium, Finland, and Portugal, respectively. CONCLUSION: In three European countries with different healthcare systems, HD-QIV would contribute to a significant improvement in the prevention of influenza health outcomes while being cost-effective.

Cost-effectiveness of a new ACI technique for the treatment of articular cartilage defects of the knee compared to regularly used ACI technique and microfracture.

AIMS: For patients with cartilage defects of the knee, a new biocompatible and in situ cross-linkable albumin-hyaluronan-based hydrogel has been developed for matrix-associated autologous chondrocyte implantation (M-ACI) - NOVOCART Inject plus (Ninject; TETEC AG, Reutlingen, Germany). We aimed to estimate the potential cost-effectiveness of NInject, that is not available on the market, yet compared to spheroids of human autologous matrix-associated chondrocytes (Spherox; CO.DON GmbH, Leipzig, Germany) and microfracture. MATERIALS AND METHODS: An early Markov model was developed to estimate the cost-effectiveness in the United Kingdom (UK) from the payer perspective. Transition probabilities, response rates, utility values and costs were derived from literature. Since NInject has not yet been launched and no prices are available, its costs were assumed equal to those of Spherox. Cycle length was set at one year and the time horizon chosen was notional patients' remaining lifetime. Model robustness was evaluated with deterministic and probabilistic sensitivity analyses (DSA; PSA) and value of information analysis (VOIA). The Markov model was built using TreeAge Pro Healthcare. RESULTS: NInject was cost-effective compared to microfracture (ICER: £5,147) while Spherox was extendedly dominated. In sensitivity analyses, the ICER exceeded conventional WTP threshold of £20,000 only when the utility value after successful first treatment with NInject was decreased by 20% (ICER: £69,620). PSA corroborated the cost-effectiveness findings of NInject, compared to both alternatives, with probabilities of 60% of NInject undercutting the aforementioned WTP threshold and being the most cost-effective alternative. The VOIA revealed that obtaining additional evidence on the new technology will likely not be cost-effective for the UK National Health Service. LIMITATIONS AND CONCLUSION: This early Markov model showed that NInject is cost-effective for the treatment of articular cartilage defects in the knee, compared to Spherox and microfracture. However, as the final price of NInject has yet to be determined, the cost-effectiveness analysis performed in this study is provisional, assuming equal prices for NInject and Spherox.

The humanistic and economic burden of atopic dermatitis among adults and adolescents in Saudi Arabia.

Aims: Atopic dermatitis (AD) is a chronic skin disease that creates a significant burden to patients and society. There is scarcity in local data about the burden of AD in the Kingdom of Saudi Arabia (KSA). We aimed to fill in this gap and quantify the humanistic and economic burden of AD among adults and adolescents in KSA.Materials and methods: A literature search and local expert interviews were conducted to assess the disease burden. Prevalence values were estimated through the literature. International data about health-related quality of life lost owing to AD was adjusted to age and prevalence in KSA. Direct and indirect costs were calculated using a bottom-up approach. Resource utilization data were collected from local dermatologists through online interviews, and indirect costs were based on absenteeism and presenteeism estimates. Validation meetings were conducted with local experts to adjust the final estimates.Results: The age-standardized health loss per patient due to AD is 0.187 quality-adjusted life-years (QALYs) annually, aggregating to 64 thousand lost QALYs in KSA. The annual average direct cost for a patient with AD was 2924 Saudi Riyal (SAR; 780 USD), totaling 373 million SAR in KSA (99.5 million USD). This value represents 0.2% of the annual health expenditure in KSA. The total productivity loss due to AD was 1.36 billion SAR (363.7 million USD). Overall, the economic burden of AD consumes up to 0.059% of the national gross domestic product.Limitations: Local quality of life and productivity lost data were not available for KSA, so global averages were used, assuming these numbers also apply to KSA.Conclusion: Indirect costs represent a large proportion of AD burden in KSA. The disease has a substantial effect on patient quality of life and social well-being. Alleviating the burden might result in significant savings in resources to society.

Atopic dermatitis is one of the most common skin diseases. Mild cases of the disease cause inflamed and itchy skin, while severe cases may cause painful episodes of itching and cracked skin. Patients with atopic dermatitis and their families suffer lower quality of life as the severity of the disease increases. In countries with hot weather like Saudi Arabia, skin is more susceptible to become dry, so the disease is very prevalent. Therefore, the disease poses a significant quality of life burden as well as an economic burden due to the direct costs of treatment and the indirect costs that arise because patients become non-productive or absent from work or school. Our study aimed to quantify the economic and quality of life burden of atopic dermatitis in Saudi Arabia to understand it's real burden and help decision makers quantify its impact on the patients and society. We conducted a literature search and interviewed local experts to determine estimates of costs and quality of life effects. The results of this study should help in prioritizing treatment disease areas in Saudi Arabia and other countries with similar circumstances.

Moving preinduction cervical ripening to a lower acuity inpatient setting using the synthetic hygroscopic cervical dilator: a cost-consequence analysis for the United States.

BACKGROUND: Leveraging the safety profile of the synthetic hygroscopic cervical dilator (SHCD), one potential way to reduce the burden-of-care provision in the labor-and-delivery unit without compromising safety is to introduce a low-acuity care room (ripening room) for patients undergoing cervical ripening as a part of labor induction at term. METHODS: Implementing a ripening room using SHCDs was compared to scenarios using prostaglandins including a dinoprostone insert (PGE2 insert) or gel (PGE2 gel) and misoprostol given orally (oral PGE1) or vaginally (vaginal PGE1). A theoretical, cost-consequence model was developed to assess costs, staff time, and selected clinical outcomes related to cervical ripening. The model assessed a hypothetical cohort where patients remained in hospital from admission for induction of labor (IOL) to delivery, taking the US labor-and-delivery unit perspective. Model inputs were taken from structured searches of PubMed and ClinicalTrials.gov, published US guidance, and clinical practice. Results are presented as mean (95% credible interval [CrI]). RESULTS: The ripening room using SHCDs cost US$3,210 and required 10.22 hours (h) of nurse time on average per patient. The cost difference to prostaglandins ranged from -$894 (-$2,269 to $398) for PGE2 gel to +$460 (-$1,467 to $1,539) for vaginal PGE1. Mean nurse time was shorter than all prostaglandins, with time savings ranging from -7.05 (-24.55 to 5.73) h for PGE2 insert to -0.97 (-14.69 to 9.59) h for vaginal PGE1. When outcomes of the probabilistic sensitivity analysis were ranked from 1 (best) to 5 (worst), the ripening room using SHCDs ranked 1.94 for costs and 1.97 for nurse time. In a nulliparous population, results improved for the ripening room using SHCDs relative to all prostaglandins. CONCLUSION: In this theoretical study, implementing a ripening room using SHCDs resulted in the lowest time burden and the second lowest costs. The cheapest option for preinduction cervical ripening was vaginal misoprostol.

It is estimated that approximately 20% of deliveries in the USA are submitted to cervical ripening prior to induction of labor to facilitate a vaginal delivery. Cervical ripening can be achieved either by administering a synthetic hormone, called a prostaglandin (e.g. misoprostol or dinoprostone), or by using mechanical means of stretching the cervix (e.g. using the synthetic hygroscopic cervical dilator ­ SHCD). Prostaglandins have been associated with an increased risk of overstimulating uterus contractions such that the person undergoing cervical ripening with prostaglandins requires close monitoring. Each method for cervical ripening has advantages and disadvantages and there is no high-quality evidence to recommend one from the others based on clinical outcomes. In this theoretical study, we estimated the hospital costs and staff time for induction of labor care when using the SHCD in a lower acuity setting within the hospital, without monitoring facilities, in comparison to the patient remaining in the labor-and-delivery room using misoprotol or dinoprostone preparations. Our results suggest that misoprostol resulted in the least expensive option closely followed by the SHCD in a lower acuity setting, both with the potential for notable cost savings when compared to using dinoprostone preparations for cervical ripening. In addition, we associated up to several hours less staff time with the use of the SHCD in a lower acuity setting in comparison to misoprostol and dinoprostone. Patients that were delivering for the first time benefitted more from using the SHCD in a lower acuity setting in comparison to those who had delivered previously.

Cost-utility and cost-benefit analysis of TAVR availability in the US severe symptomatic aortic stenosis patient population.

AIMS: We evaluated the availability of transcatheter aortic valve replacement (TAVR) to determine its value across all severe symptomatic aortic stenosis (SSAS) patients, especially those untreated because of concerns regarding invasive surgical AVR (SAVR) and its impact on active aging. METHODS: We performed payer perspective cost-utility analysis (CUA) and societal perspective cost-benefit analysis (CBA). The CBA's benefit measure is active time: salaried labor, unpaid work, and active leisure. The study population is a cohort of US elderly SSAS patients. We compared a "TAVR available" scenario in which SSAS patients distribute themselves across TAVR, SAVR, and medical management (MM); and a "TAVR not available" scenario with only SAVR and MM. We structured each scenario with a decision-tree model of SSAS patient treatment allocation. We measured the association between health and active time in the US Health and Retirement Study and used this association to impute active time to SSAS patients given their health. RESULTS: The incremental cost-effectiveness ratio (ICER) and rate of return (RoR) of TAVR availability were $8,533 and 395%, respectively. CUA net monetary benefits (NMB) were $212,199 per patient and $43.4 billion population-wide. CBA NMB were $50,530 per patient and $10.3 billion population-wide. LIMITATIONS: Among study limitations were scarcity of evidence regarding key parameters and the lack of long-term survival, health utility, and treatment cost data. Our analysis did not account for TAVR durability, retreatments, and valve-in-valve treatments. CONCLUSION: Across risk-, age-, and treatment-eligibility groups, TAVR is the economically optimal treatment choice. It represents strong value-for-money per patient and population-wide. The vast majority of TAVR value involves raising treatment uptake among the untreated.

Aortic stenosis (AS) is a common and lethal heart disease. Surgical treatment has long been available, but its invasiveness limits uptake. More recently, transcatheter aortic valve replacement (TAVR) has emerged as a treatment alternative. Its minimal invasiveness has significantly increased treatment rates, but economic evaluations omit this benefit, risking undervaluation. We evaluated TAVR in elderly US severe symptomatic AS patients, using payer perspective cost-utility analysis (CUA) and societal perspective cost-benefit analysis (CBA). Both CUA and CBA incorporated TAVR's impact on treatment rates. Given patient preferences for treatment options promoting active aging, our CBA used the value of active time as a benefit measure. We found that CUA/CBA net monetary benefits are $212,199/$50,530 per patient. Across risk-, age-, and treatment-eligibility groups, TAVR is the economically optimal treatment choice over surgery and medical management. It represents strong value-for-money per patient and population-wide. Increased treatment uptake accounts for the vast share of TAVR's value.

Evaluating the cost-utility of a direct transfer to angiosuite protocol within 6 h of symptom onset in suspected large vessel occlusion patients.

INTRODUCTION: A direct transfer to angiosuite (DTAS) protocol has shown to be effective and safe by shortening in-hospital workflows and encouraging long-term outcome benefits. To implement DTAS at a new facility, a large organizational effort is necessary. We performed a cost-utility analysis and budget impact analysis (BIA) of the operation of a new angiosuite, primarily dedicated to stroke patients, that allows facilities to approximate the cost implications of utilizing a DTAS pathway. METHODS: Sixty-one patients who underwent endovascular treatment (EVT) following DTAS were matched for baseline variables to 117 patients who underwent a conventional imaging protocol at a hospital in Catalonia, Spain. An economic model, based on actual data from these patients, was developed to assess the short- and long-term clinical and economic implications of DTAS. In the BIA, the DTAS scenario was gradually implemented for 20% of patients each year until reaching a plateau at 80% of patients in the DTAS pathway. Initial investment and additional organizational costs, €4 million, were taken into consideration to compare the budget impact of the DTAS scenario with no organizational changes over five years. RESULTS: DTAS was associated with better patient functional independence rates (mRS 0-2: 50.9% vs. 41.0%) and a quality-adjusted life-years gain of 0.82 per patient. Despite the additional initial investment, DTAS development was associated with an estimated 10.2% reduction (€14.7 million) of the total costs (€144.5 million). Cost savings were mainly due to long-term associated costs related to patient disability (€13.2 million). LIMITATIONS: The study relies on data obtained from a single-center, and therefore it may be difficult to generalize the findings. CONCLUSIONS: Our economic model predicts that the implementation of a DTAS program is cost-effective compared with no organizational changes. Our model also predicts better clinical outcomes for patients in terms of functional independence and quality-adjusted life years.