The "Big short" of minimally invasive pancreaticoduodenectomy for pancreatic ductal adenocarcinoma. A cost-effectiveness analysis based on randomized trials.

BackgroundLong-term cost-effectiveness analyses of health behaviour interventions to effectively manage type 2 diabetes mellitus (T2DM) in low-income countries are crucial for minimising economic burden and optimising resource allocation. Therefore, this study aimed to estimate the long-term cost-effectiveness of implementing a health behaviour intervention to manage T2DM in Nepal.MethodsA Markov model in combination with a decision tree was developed to compare the costs and outcomes of the health behaviour intervention against usual care among 481 (238-intervention and 243-control) participants from healthcare system and societal perspectives. The model integrates empirical trial data, with published data to inform parameters not collected during the trial. The model estimated costs, quality-adjusted life years (QALYs) and cost-effectiveness over 5 years, 10 years, 20 years, 30 years and a lifetime time horizons with 3% annual discounting. Sub-group, scenarios, both one-way and two-way analyses and probabilistic sensitivity analyses (PSA) were performed to assess the impact of uncertainty in the model under the threshold of 3 times gross domestic product (GDP) per capita (i.e., US $4140) for Nepal.ResultsBase-case analysis with lifetime horizon showed that the health behaviour intervention compared to usual care improved QALYs by 3.88 and increased costs by US $4293 per patient, with an incremental cost-effectiveness ratio (ICER) of US $1106 per QALY gained from a healthcare system perspective. From a societal perspective, QALYs also improved by 3.88 and costs increased by US $4550, with an ICER of US $1173 per QALY gained. Furthermore, the intervention demonstrated ICERs of US $636, US $678, US $637, and US $632 per QALY gained over 5-, 10-, 20-, and 30-year time horizons, respectively, from a healthcare system perspective, and US $719, US $766, US $659, and US $716 per QALY gained from a societal perspective. In the PSA, the probability of the health behaviour intervention being cost-effective was over 57%.ConclusionsThe health behaviour intervention for managing T2DM was cost-effective over a lifetime horizon compared to usual care. To maximise its impact, this intervention should be scaled up nationwide, and future research is warranted to assess the long-term cost-effectiveness across diverse settings in low-income countries.Trial registrationAustralia and New Zealand Clinical Trial Registry (ACTRN12621000531819).Graphical

Axicabtagene ciloleucel (Axi-Cel, 2 × 106 CAR-T cells/kg, single intravenous injection) is a chimeric antigen receptor cell immunotherapy that exhibits favorable clinical efficacy and safety in patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL). However, this treatment is expensive in China. This study aimed to evaluate the cost-effectiveness of Axi-Cel versus salvage chemotherapy for the treatment of R/R DLBCL from the perspective of the Chinese healthcare system. A decision analysis model containing a short-term decision tree and long-term semi-Markov partitioned survival model was developed. The time horizon was 40years and the period from 10 to 40years was included in sensitivity analysis. The model was developed based on data from the ZUMA-1 and SCHOLAR-1 trials. Life years, quality-adjusted life years (QALYs), overall costs, and the incremental cost-effectiveness ratio (ICER) were estimated at a willingness to pay (WTP) threshold of US $31,320 per QALY, which is three times the gross domestic product per capita. The base case analysis revealed that treatment with Axi-Cel is associated with an increased overall cost of US $175,380 and improved effectiveness of 3.43 LYs and 2.61 QALYs compared to salvage chemotherapy, leading to an ICER of US $51,190 per LY and US $67,250 per QALY. The developed model is sensitive to the discount rate, utility of progression-free survival (PFS), and cost of Axi-Cel. The ICER of Axi-Cel was greater than the WTP threshold in the sensitivity and scenario analyses. To achieve cost-effectiveness, the price of Axi-Cel must be reduced by 59.19% to US $71,000. At its current price, Axi-Cel is not likely to be a cost-effective option compared to salvage chemotherapy for adult patients with R/R DLBCL.

BackgroundMobile health (mHealth) technology is increasingly used in disease management. Using mHealth tools to integrate and streamline care has improved clinical outcomes of patients with atrial fibrillation (AF).ObjectiveThe aim of this study was to investigate the potential clinical and health economic outcomes of mHealth-based integrated care for AF from the perspective of a public health care provider in China.MethodsA Markov model was designed to compare outcomes of mHealth-based care and usual care in a hypothetical cohort of patients with AF in China. The time horizon was 30 years with monthly cycles. Model outcomes measured were direct medical cost, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). Sensitivity analyses were performed to examine the robustness of the base-case results.ResultsIn the base-case analysis, mHealth-based care gained higher QALYs of 0.0730 with an incurred cost of US $1090. Using US $33,438 per QALY (three times the gross domestic product) as the willingness-to-pay threshold, mHealth-based care was cost-effective, with an ICER of US $14,936 per QALY. In one-way sensitivity analysis, no influential factor with a threshold value was identified. In probabilistic sensitivity analysis, mHealth-based care was accepted as cost-effective in 92.33% of 10,000 iterations.ConclusionsThis study assessed the expected cost-effectiveness of applying mHealth-based integrated care for AF according to a model-based health economic evaluation. The exploration suggested the potential cost-effective use of mHealth apps in streamlining and integrating care via the Atrial fibrillation Better Care (ABC) pathway for AF in China. Future economic evaluation alongside randomized clinical trials is highly warranted to verify the suggestion and investigate affecting factors such as geographical variations in patient characteristics, identification of subgroups, and constraints on local implementation.

Is SABR Cost-Effective in Oligometastatic Cancer? An Economic Analysis of the SABR-COMET Randomized Trial

Cost-effectiveness analysis in cardiac surgery: A review of its concepts and methodologies

Background: Whether Endovascular Thrombectomy (EVT) is cost-effective in large ischemic core infarcts is unknown. Methods: In the prospective multicenter cohort study of imaging selection study (SELECT), large core was defined as CT ASPECTS < 6 or CTP ischemic core volume (rCBF<30%) ≥ 50 cc. A Markov model estimated costs, quality-adjusted life years (QALYs) and the Incremental Cost-effectiveness Ratio (ICER) of EVT compared to Medical Management (MM) over 20 years life expectancy. The lower and upper willingness to pay (WTP) per QALY were set at $50000 and $100000 and the Net Monetary Benefit (NMB) for EVT were calculated. A probabilistic sensitivity analysis (PSA) and cost-effectiveness acceptability curves (CEAC) assessed EVT cost-effective probability at WTP range values. Results: Of 361 enrolled, 105 had large core on CT or CTP (EVT 62, MM 43). 19 (31%) EVT patients achieved mRS 0-2 vs 6 (14%) MM (aOR: 3.27, 95% CI: 1.11-9.62; P = .03) with a shift towards better mRS (adj cOR: 2.12, 95% CI: 1.05-4.31, P = .04). Over 20 years EVT was associated with $26589 (C.I. $8672- $43978) incremental costs and a gain of 1.18 QALYs (C.I. 0.091- 2.2) per patient. EVT could avert 75 deaths over a theoretical cohort of 1000 patients (MM 861 vs EVT 786) thus the ICER of EVT compared to MM was $22400 per QALY (CI. $10109 - $66140), which is <$50000/QALY, Tab 1. EVT has a higher NMB compared to MM at the lower and upper WTP thresholds (EVT $86,3 and 271,4 million vs MM $53,6-$179,3 million), Tab 2. The PSA confirmed the results (fig 1). The CEAC showed 94% and 97% cost-effectiveness probability of EVT at the lower and upper values respectively of the maximum WTP, fig 2. EVT ICER in SELECT large core ($22400/QALY) was higher but still comparable to those in HERMES ($16882/QALY), DAWN ($7335/QALY) and DEFUSE3 ($14673/QALY), Tab 3. Conclusion: EVT may result in better outcomes and more lives saved in large core patients with higher QALYs, NMB and an acceptable ICER. The results were comparable to other EVT RCTs.

Cost-Effectiveness of Azacitidine and Ivosidenib in Newly Diagnosed Older, Intensive Chemotherapy-Ineligible Patients with IDH1-Mutant Acute Myeloid Leukemia

Deep brain stimulation (DBS) is an effective neurosurgical option for patients with treatment-resistant obsessive-compulsive disorder (OCD). Despite being more costly than neuroablative procedures of comparable efficacy, DBS has gained popularity over the years for its reversibility and adjustability. Although the cost-effectiveness of DBS has been investigated extensively in movement disorders, few economic analyses of DBS for psychiatric disorders exist. In this study, the authors present the first cost-effectiveness analysis of DBS for treatment-resistant OCD in the United States. The authors developed four decision analytical models to compare the cost-effectiveness of DBS with treatment as usual (TAU) for OCD, varying either the device type (i.e., nonrechargeable or rechargeable) or the time horizon (i.e., 3 or 5 years) in each model. Treatment response and complication rates were based on a literature review. Published algorithms were used to convert Yale-Brown Obsessive Compulsive Scale scores into utility scores reflecting improvements in quality of life. Costs were approached from the healthcare sector perspective and were drawn primarily from Medicare facility and physician reimbursement rates. For each model, a Monte Carlo simulation (n = 100,000) and probabilistic sensitivity analysis were performed to estimate the incremental cost-effectiveness ratio (ICER) in US dollars per quality-adjusted life year (QALY). Data from 249 and 265 treatment-resistant OCD patients from the published literature who received DBS and had sufficient follow-up in 3- and 5-year models, respectively, were included. When conventional US willingness-to-pay (WTP) thresholds were used, nonrechargeable DBS models were less cost-effective (3-year ICER: $108,431/QALY; 5-year ICER: $203,202/QALY) and rechargeable DBS models were more cost-effective (3-year ICER: $49,363/QALY; 5-year ICER: $41,495/QALY) than TAU. At a WTP threshold of $100,000/QALY, rechargeable DBS devices were moderately more cost-effective than TAU at 3 and 5 years in 100% of iterations. At a WTP threshold of $50,000/QALY, rechargeable DBS devices were definitively more cost-effective than TAU at 3 and 5 years in 54% and 89% of iterations, respectively. When using WHO WTP conventions, 3- and 5-year nonrechargeable models were cost-effective in 100% and 84% of iterations, and 3- and 5-year rechargeable models were highly cost-effective in 99% and 100% of iterations, respectively. Rechargeable DBS models were cost-effective for treatment-resistant OCD compared with TAU. Nonrechargeable DBS models may be cost-effective, especially with improvement in battery longevity and changes in accepted WTP thresholds.

Background Adding atezolizumab to carboplatin/nab-paclitaxel improved progression-free survival and overall survival in patients with advanced non-squamous non-small-cell lung cancer. However, estimating the economy of atezolizumab/carboplatin/nab-paclitaxel is urgent on account of the high cost of atezolizumab. Objective This study aimed to evaluate the cost-effectiveness of atezolizumab plus carboplatin/nab- paclitaxel for untreated advanced non-squamous non-small-cell lung cancer from the United States payer perspective. Setting This study was based on randomized clinical trial data from the IMpower130 (NCT02367781) published in Lancet Oncology (May 2019). Method A Markov model was constructed to estimate the health expenditure on atezolizumab in combination with carboplatin/nab-paclitaxel for advanced non-small-cell lung cancer treatment. Drug costs were collected from Red Book Wholesale Acquisition Cost, and health state utility values were obtained from the literature. Uncertainty was evaluated via one-way and probabilistic sensitivity analyses. Main outcome measure The main outcomes were cost, life years, quality-adjusted life years, and incremental cost-effectiveness ratio. Results Over a 10-year horizon, atezolizumab/carboplatin/nab-paclitaxel treatment was associated with an expected 1.76 life years and 0.99 quality-adjusted life years compared to the 1.21 life years and 0.67 quality-adjusted life years for carboplatin/nab-paclitaxel alone. Compared to carboplatin/nab-paclitaxel, atezolizumab/carboplatin/nab-paclitaxel produced an incremental cost of $105,617. The resultant incremental cost-effectiveness ratio was $333,199 per quality-adjusted life year, which exceeded the willingness-to-pay threshold of $180,000 per quality-adjusted life year. The price of atezolizumab and utility values were the parameters that greatly impacted the incremental cost-effectiveness ratio. Carboplatin/nab-paclitaxel exhibited 98.6% probability of being a cost-effective treatment option compared to atezolizumab/carboplatin/nab-paclitaxel at a willingness-to-pay of $180,000 per quality-adjusted life year. However, reducing atezolizumab acquisition cost by 43.4% could make atezolizumab/carboplatin/nab-paclitaxel more cost-effective than carboplatin/nab-paclitaxel. Conclusion Adding atezolizumab to carboplatin/nab-paclitaxel was not cost-effective for advanced non-squamous non-small-cell lung cancer in the base-case scenario. Decreasing atezolizumab acquisition cost might enhancethecost-effectiveness.

Funding Acknowledgements Type of funding sources: None. Background Nurse-led self-care interventions (NLSCI) in heart failure (HF), defined as the nurse education delivered to improve the daily patient self-management, are not widely adopted by the health-care systems, even though they are effective to improve outcomes (e.g., mortality, readmission). Moreover, few studies have evaluated whether NLSCI are also cost-effective. Purpose To determine the cost-effectiveness of NLSCI in the context of HF care compared with standard care (care delivered by general practitioner and/or cardiologist). Methods We performed a cost-effectiveness analysis, with a 20-year time horizon, from the perspective of the Italian National Health Service. We developed a Markov model to simulate the progression of a cohort of 1,000 HF patients aged 70 years, who were assumed to alternatively receive a NLSCI after hospital discharge, or usual care. Effectiveness on mortality and on hospitalizations of NLSCI and usual care were extrapolated from a review of randomized control trials. Health-care costs were derived from literature and national formularies. The differences in costs and the differences in Quality Adjusted Life Years (QALY) between the NLSCI and usual care were estimated to present an incremental cost-effectiveness ratio (ICER). A willingness to pay (WTP) threshold of €40,000 per QALY was considered. Probabilistic sensitivity analyses were conducted to test the robustness of results and to estimate a cost-effectiveness acceptability curve. Results Over the 20-year time horizon, NLSCI implied an extra cost of € 1.3 million and a gain of 247 QALYs compared to usual care. This resulted in an ICER of € 5,490/QALY, which is far below the €40,000/QALY WTP threshold. Sensitivity analysis showed that the ICER remains below the WTP threshold in 100% of simulations. Moreover, the cost-effectiveness acceptability curve showed a probability of 80% of being under € 7,500/QALY. Conclusions This study demonstrated that NLSCI represent an affordable solution to support patients with HF as the related extra costs of € 1.3 million is justified by the reduction in mortality and improvement in quality of life. This finding supports the promotion of NLSCI as part of routine care, in order to pursue an optimal allocation of public health expenditures.

To model the cost-effectiveness (CEA) of the use of pregabalin versus usual care (UC) in outpatients with refractory generalised anxiety disorder (GAD) treated in daily practice in mental health settings in Spain. This CEA model used data extracted from a 6-month prospective non-interventional trial: the Amplification of Definition of ANxiety (ADAN) study, which was conducted to determine the cost-of-illness in GAD subjects. Refractory subjects were those who reported persistent symptoms of anxiety and showed suboptimal response in the Hamilton-anxiety scale (HAM-A≥16) after a standard dose regimen of anxiolytics other than pregabalin, alone or in combination, over 6months. The pregabalin arm was documented with data extracted from patients who received pregabalin in the study for the first time, added or replacing the existing therapy. In the UC arm, treatment might include one or more of the following: a serotonin selective reuptake inhibitor, a serotonin-norepinephrine reuptake inhibitor, other anti-depressants, a benzodiazepine or an anti-epileptic drug other than pregabalin. The time horizon of the modelling was 6months in the base-case scenario, and the National Health System perspective was chosen to calculate costs. Effectiveness was expressed as quality-adjusted life years (QALYs) gained, which were derived using the EQ-5D questionnaire, at baseline and end-of-trial visits. Results of the CEA model was expressed as an incremental cost-effectiveness ratio (ICER) per QALY gained. Probabilistic sensitivity analysis using bootstrapping techniques was also carried out to obtain the cost-effectiveness plane and the corresponding acceptability curve. Data from a total of 429 subjects per arm (mean HAM-A score 25.7) meeting eligible criteria for inclusion in CEA modelling were extracted from the original trial. Compared with UC, pregabalin (average dose 218mg/day) was associated with significantly higher QALY gain; 0.1209±0.1030 versus 0.0994±0.0979 (P=0.003), but increased healthcare costs as well; <euro>1,272±1,240 versus <euro>1,070±1,177 (P<0.069) and drug costs <euro>525±252 versus 219±211 (P<0.001), resulting in an ICER of <euro>15,804/QALY (95% CI 6,661; 37,186) for healthcare costs and <euro>15,165/QALY (7,947; 31,754) when drug costs were considered alone. A total of 94% of re-samples fell below the threshold of <euro>30,000 per QALY. This evaluation modelling suggests that pregabalin may be cost-effective in comparison with UC in outpatients with refractory GAD treated in mental healthcare settings in daily practice in Spain.

Aims To evaluate the cost-effectiveness of vibegron compared with other oral pharmacologic therapies as treatment for overactive bladder (OAB). Methods A semi-Markov model with monthly cycles was developed to support a lifetime horizon of vibegron 75 mg from a US commercial payor or Medicare perspective. The model incorporated efficacy (reductions in daily micturitions and urinary incontinence episodes), adverse events, OAB-related comorbidities, drug–drug interactions, anticholinergic burden, and treatment persistence. Direct costs and quality-adjusted life years (QALY) were accumulated over time. The primary outcome was the cost per QALY incremental cost-effectiveness ratio (ICER). One-way (OWSA) and probabilistic sensitivity analyses (PSA) were performed. Results For commercial payors, vibegron was cost-effective at a willingness-to-pay (WTP) threshold of $50,000/QALY versus mirabegron 50 mg (ICER, $9,311) and at a WTP threshold of $150,000/QALY versus mirabegron 25 mg (ICER, $141,957) and versus an anticholinergic basket based on market share (ICER, $118,121). For Medicare, vibegron was cost-effective at a WTP threshold of $50,000/QALY versus mirabegron 50 mg (ICER, $12,154) and at a WTP threshold of $100,000/QALY versus mirabegron 25 mg (ICER, $99,150) and versus an anticholinergic market basket (ICER, $60,756). For commercial payors and Medicare, OWSAs for vibegron versus mirabegron indicated cost-effectiveness was most sensitive to vibegron persistence at 1 and 12 months. PSAs indicated that vibegron was cost-effective versus mirabegron 50 mg 98.6% and 100% of the time at $50,000/QALY for commercial payors and Medicare payors, respectively. Limitations Due to lack of real-world data available on persistence, vibegron was assumed to have the same persistence as mirabegron 50 mg. Long-term efficacy was assumed to be sustained beyond 52 weeks in the absence of clinical trials longer than 52 weeks. Conclusions Vibegron is cost-effective from a commercial payor (WTP threshold $150,000/QALY) and Medicare (WTP threshold $100,000/QALY) perspective when compared with other oral pharmacologic treatments for OAB.

26 Background: Stereotactic body radiotherapy (SBRT) has been proposed for the palliation of painful vertebral bone metastases because higher radiation doses may confer better pain control. A Phase III clinical trial comparing SBRT with single fraction external beam radiotherapy (EBRT) is now ongoing. We performed a cost-effectiveness analysis to compare these strategies. Methods: A Markov model, using a 1-month cycle over a lifetime horizon, was developed to compare the cost effectiveness of SBRT (16 or 18 Gy in 1 fraction) to 8 Gy in 1 fraction of EBRT. Transition probabilities, quality of life utilities, and costs associated with SBRT and EBRT were captured in the model. Costs were based on Medicare reimbursement in 2014. Strategies were compared using the incremental cost effectiveness ratio (ICER), and effectiveness was measured in quality-adjusted life years (QALYs). To account for uncertainty, one-way and probabilistic sensitivity analyses were performed. Strategies were evaluated with a willingness-to-pay (WTP) threshold of $100,000/QALY gained. Results: Base case pain relief after the treatment was assumed as 20% higher in SBRT. Treatment costs for SBRT and EBRT were $9000 and $1087, respectively. In the base case analysis, SBRT resulted in an ICER of $124,552/QALY gained. In one-way sensitivity analyses, results were most sensitive to variation of the utility of unrelieved pain (range: $89,330 to $592,720/QALY gained); the utility of relieved pain post-treatment and median survival were also sensitive to variation. If median survival is ≥11 months (base case estimate: 9 months), SBRT cost &lt;$100,000/QALY gained. Probabilistic sensitivity analysis demonstrated that SBRT was favored in 30% of model iterations at a WTP threshold of $100,000/QALY gained. Conclusions: SBRT for palliation of vertebral bone metastases is not cost-effective compared to EBRT based upon the ICER analysis with the WTP of $100,000/QALY gained. However, if median survival is ≥11 months, SBRT is economically reasonable, suggesting that selective SBRT usage in patients with longer expected survival may be the most cost-effective approach.

Cost-Effectiveness Analysis of Frontline Treatment with Polatuzumab Vedotin in Diffuse Large B-Cell Lymphoma

BackgroundBenmelstobart combined with anlotinib and chemotherapy has demonstrated significant clinical advantages in extending progression-free survival and overall survival compared to chemotherapy alone in patients with extensive-stage small-cell lung cancer (ES-SCLC). This is the first study to assess its cost-effectiveness from both the US payer and Chinese healthcare system perspectives.MethodA Markov state-transition model was utilized for the economic evaluation, reflecting both the perspectives of the US payer and the Chinese healthcare system. Baseline patient demographics and vital clinical data were obtained from the ETER701 trial. Costs and utilities were obtained from open-access databases and published literature. The primary outcomes evaluated were quality-adjusted life years (QALYs), incremental cost-effectiveness ratio (ICER), incremental net health benefit (INHB), and incremental net monetary benefit (INMB). The uncertainties of the model were addressed through probabilistic sensitivity analysis, one-way sensitivity analysis, and scenario analysis.ResultsIn the base-case scenario, adding benmelstobart and anlotinib to chemotherapy increased QALYs by 0.34 at an additional cost of $24,684.07, yielding an ICER of $71,559.84 per QALY. This exceeds the willingness-to-pay (WTP) threshold of $38,042.49 per QALY in China, making the treatment marginally cost-effective, with an INHB of −0.30 QALYs and an INMB of -$11,561.58. In the US, the treatment resulted in a QALY increase of 0.36, but incurred an additional cost of $151,052.04, leading to an ICER of $416,398.56 per QALY, surpassing the US WTP threshold of $150,000. 00.ConclusionThe combination of benmelstobart and anlotinib with chemotherapy is not a cost-effective first-line treatment option for ES-SCLC in either China or the US.