Quality-adjusted Life Years For Patients Research Articles

Cost-Effectiveness Analysis of Empagliflozin for Treatment of Patients With Heart Failure With Reduced Ejection Fraction in the United States.

In the EMPEROR-Reduced trial (Empagliflozin Outcome Trial in Patients with Chronic Heart Failure and a Reduced Ejection Fraction), empagliflozin plus standard of care reduced the composite of cardiovascular death or hospitalization for heart failure versus standard of care in adults with heart failure with reduced ejection fraction. This analysis investigated the cost-effectiveness of the 2 regimens from the perspective of US payors. A Markov cohort model was developed based on Kansas City Cardiomyopathy Questionnaire Clinical Summary Score quartiles and death. Transition probabilities between health states, risk of cardiovascular/all-cause death, hospitalization for heart failure and adverse events, treatment discontinuation, and health utilities were estimated from trial data. Medicare and commercial payment rates were combined for treatment acquisition, acute event management, and disease management. An annual discount rate of 3% was used. Empagliflozin plus standard of care yielded 18% fewer hospitalizations for heart failure and 6% fewer deaths versus standard of care over a lifetime, providing cost-offsets while adding 0.19 life years and 0.19 quality-adjusted life years at an incremental cost of $16 815/patient. The incremental cost-effectiveness ratio was $87 725/quality-adjusted life years gained. Results were consistent across payors, subpopulations, and in deterministic sensitivity analyses. In probabilistic sensitivity analyses, empagliflozin plus standard of care was cost-effective in 3%, 62%, and 80% of iterations at thresholds of $50 000, $100 000, and $150 000/quality-adjusted life years. Empagliflozin plus standard of care may prevent hospitalizations for heart failure, extend life, and increase quality-adjusted life years for patients with heart failure with reduced ejection fraction at an acceptable cost for US payors.

A Markov analysis of azacitidine and venetoclax vs induction chemotherapy for medically fit patients with AML

AbstractAlthough induction chemotherapy (IC) is the standard of care in medically fit patients with newly diagnosed acute myeloid leukemia (AML), limited retrospective data indicate that patients at adverse-risk may benefit from azacytidine and venetoclax (aza-ven). Our goal was to perform a Markov decision analysis to determine whether IC or aza-ven is the optimal induction regimen in this population. Using the TreeAge software, Markov models were created for adverse-risk and intermediate-risk cohorts. A systematic review of the literature informed the transition probabilities and utilities included in the analyses. Our primary outcome was quality-adjusted life years (QALYs) gained over 5 years after diagnosis. Overall, patients at adverse risk treated with IC gained 1.4 QALYs, compared with 2.0 QALYs in patients treated with aza-ven. Patients at adverse risk treated with IC and allogeneic stem cell transplantation (allo-SCT), IC, aza-ven and allo-SCT, or aza-ven gained 2.1, 1.5, 3.0, and 1.9 QALYs, respectively. Meanwhile, patients at intermediate risk treated with IC gained 2.0 QALY, compared with 1.7 QALY in patients treated with aza-ven. Patients at intermediate risk treated with IC and allo-SCT, IC, aza-ven and allo-SCT, and aza-ven gained 2.7, 2.3, 2.6, and 1.8 QALYs, respectively. We have demonstrated that medically fit patients with newly diagnosed adverse-risk AML may benefit from treatment with aza-ven over those treated with IC, whereas IC remains the preferred approach for patients at intermediate risk. Our work challenges the use of the European LeukemiaNet risk classification for patients treated with aza-ven and highlights the need for prospective investigation into aza-ven as induction therapy for medically fit patients.

Cost-effectiveness of finerenone in chronic kidney disease associated with type 2 diabetes in The Netherlands

BackgroundIn the Netherlands, more than one million patients have type 2 diabetes (T2D), and approximately 36% of these patients have chronic kidney disease (CKD). Yearly medical costs related to T2D and CKD account for approximately €1.3 billion and €805 million, respectively. The FIDELIO-DKD trial showed that the addition of finerenone to the standard of care (SoC) lowers the risk of CKD progression and cardiovascular (CV) events in patients with CKD stages 2–4 associated with T2D. This study investigates the cost-effectiveness of adding finerenone to the SoC of patients with advanced CKD and T2D compared to SoC monotherapy.MethodsThe validated FINE-CKD model is a Markov cohort model which simulates the disease pathway of patients over a lifetime time horizon. The model was adapted to reflect the Dutch societal perspective. The model estimated the incremental costs, utilities, and incremental cost-effectiveness ratio (ICER). Sensitivity and scenario analyses were performed to assess the effect of parameter uncertainty on model robustness.ResultsWhen used in conjunction with SoC, finerenone extended time free of CV events and renal replacement therapy by respectively 0.30 and 0.31 life years compared to SoC alone, resulting in an extension of 0.20 quality-adjusted life years (QALYs). The reduction in renal and CV events led to a €6136 decrease in total lifetime costs per patient compared to SoC alone, establishing finerenone as a dominant treatment option. Finerenone in addition to SoC had a 83% probability of being dominant and a 93% probability of being cost-effective at a willingness-to-pay threshold of €20,000.ConclusionBy reducing the risk of CKD progression and CV events, finerenone saves costs to society while gaining QALYs in patients with T2D and advanced CKD in the Netherlands.

First-line nivolumab plus ipilimumab for unresectable MPM in China: a cost-effectiveness analysis

BackgroundThe regimen of nivolumab plus ipilimumab (NI) has been recommended by the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology-Malignant Pleural Mesothelioma (Version 1.2022) and Chinese Guidelines for the Clinical Diagnosis and Treatment of Malignant Pleural Mesothelioma (2021 edition) as the first-line treatment for Malignant Pleural Mesothelioma (MPM). But whether immunotherapy has a financial advantage over conventional chemotherapy (pemetrexed plus cisplatin/carboplatin, C) is uncertain.MethodsBased on survival and safety data from the CheckMate 743 clinical trial (NCT02899299), a partitioned survival model was constructed using TreeAge Pro2022 software. The model cycle was set to 1 month and the study period was 10 years. The output indicators included total cost, quality-adjusted life year (QALY) and incremental cost-effectiveness ratio (ICER). One-way and probabilistic sensitivity analyses were used to assess the robustness of the results, considering only direct medical costs.Results and discussionThe ICER for group NI versus Group C was $375,656/QALY in all randomized patients, $327,943/QALY in patients with epithelioid histology, and $115,495/QALY in patients with non-epithelioid histology. The ICERs of all three different populations all exceeded the willingness-to-pay threshold (three times the per capita gross domestic product of China in 2021). The results of univariate sensitivity analysis showed that the price of pemetrexed and nivolumab had great influence on the analysis results. The results of the probabilistic sensitivity analysis show that the probability of the NI scheme being more economical in all three different populations was 0.What is new and conclusionFrom the perspective of the Chinese healthcare system, in patients with unresectable MPM, NI has no economic advantage over C.

Determining patients with spinal metastases suitable for surgical intervention: A cost-effective analysis.

Both nonoperative and operative treatments for spinal metastasis are expensive interventions. Patients' expected 3-month survival is believed to be a key factor to determine the most suitable treatment. However, to the best of our knowledge, no previous study lends support to the hypothesis. We sought to determine the cost-effectiveness of operative and nonoperative interventions, stratified by patients' predicted probability of 3-month survival. A Markov model with four defined health states was used to estimate the quality-adjusted life years (QALYs) and costs for operative intervention with postoperative radiotherapy and radiotherapy alone (palliative low-dose external beam radiotherapy) of spine metastases. Transition probabilities for the model, including the risks of mortality and functional deterioration, were obtained from secondary and our institutional data. Willingness to pay thresholds were prespecified at $100,000 and $150,000. The analyses were censored after 5-year simulation from a health system perspective and discounted outcomes at 3% per year. Sensitivity analyses were conducted to test the robustness of the study design. The incremental cost-effectiveness ratios were $140,907 per QALY for patients with a 3-month survival probability >50%, $3,178,510 per QALY for patients with a 3-month survival probability <50%, and $168,385 per QALY for patients with independent ambulatory and 3-month survival probability >50%. This study emphasizes the need to choose patients carefully and estimate preoperative survival for those with spinal metastases. In addition to reaffirming previous research regarding the influence of ambulatory status on cost-effectiveness, our study goes a step further by highlighting that operative intervention with postoperative radiotherapy could be more cost-effective than radiotherapy alone for patients with a better survival outlook. Accurate survival prediction tools and larger future studies could offer more detailed insights for clinical decisions.

Biomarkers-Based Cost-Effectiveness of Toripalimab Plus Chemotherapy for Patients with Treatment-Naive Advanced Non-Small Cell Lung Cancer.

This study examined the cost-effectiveness of first-line toripalimab plus chemotherapy (TC) for patients with advanced non-small cell lung cancer (NSCLC), excluding patients with nonsquamous NSCLC and EGFR/ALK mutations. It further analyzed the cost-effectiveness of this strategy in biomarker-based subgroups, all within the context of the Chinese healthcare system. Eighteen Markov models with 21-day Markov cycle lengths and 30-year time horizons were constructed in this study. Clinical effectiveness data were derived from the CHOICE-01 trial. Health state utilities and costs data were obtained from various sources. The primary outputs were the calculation of incremental cost-effectiveness ratios (ICERs), which were then compared to a willingness-to-pay (WTP) threshold of $17,961 per quality-adjusted life-year (QALY). This comparison was used to determine the treatment that offered greater cost-effectiveness. To account for uncertainty in the model, sensitivity analyses were conducted. For the overall patient population, the estimated ICER between first-line TC and placebo plus chemotherapy (PC) was $9445/QALY, significantly lower than the WTP threshold used in the model. In subgroups based on pathologic types, first-line TC had an ICER of $16,757/QALY for patients with nonsquamous NSCLC, slightly below the WTP threshold; first-line TC demonstrated dominance in patients with squamous NSCLC, indicating both better effectiveness and lower costs compared to first-line PC. In biomarkers-based subgroups, first-line TC was dominant over first-line PC in the subgroups with programmed cell death ligand1 (PD-L1) expression ≥ 50% and SMARCA4 mutations. Moreover, first-line TC had ICERs lower than the WTP threshold in other subgroups, except for the subgroup with RB1 mutations. Sensitivity analysis confirmed the robustness of these findings. From the perspective of the Chinese healthcare system, this study's findings suggested that first-line TC represents a cost-effective strategy for patients with advanced NSCLC. However, the cost-effectiveness of first-line TC varied across different subgroups when considering predictive biomarkers.

The Cost-Effectiveness of Sugemalimab Plus Chemotherapy as First-Line Treatment for Metastatic Squamous and Non-squamous NSCLC in China.

Sugemalimab is the first China-developed programmed death-ligand 1 inhibitor that has proved to be effective as a first-line treatment for both metastatic squamous and non-squamous non-small cell lung cancer (NSCLC) when used in combination with chemotherapy. This study compared the cost-effectiveness of sugemalimab plus chemotherapy (sugema + chemo) with placebo plus chemotherapy (placebo + chemo) among metastatic squamous and nonsquamous NSCLC, respectively. Separate Markov models were constructed to generate the cumulative healthcare costs and quality-adjusted life-years (QALYs) associated with two treatment strategies over a 20-year time horizon. Transition probabilities were estimated using survival data reported in the GEMSTONE-302 trial. Health state utilities and costs were derived from published literature, national databases, and local general hospitals. Sensitivity analyses were performed to test the robustness of our conclusions. Compared with first-line placebo + chem, sugema + chemo achieved an incremental cost-effectiveness ratio (ICER) of $57,842/QALY for patients with metastatic squamous NSCLC and achieved an ICER of $78,249/QALY for patients with metastatic non-squamous NSCLC. In our sensitivity analyses of a willingness-to-pay (WTP) threshold of $35,663 per QALY, the first-line sugema + chemo was only cost-effective for patient groups when the price of sugemalimab decreased. Sugema + chemo was not cost-effective as a first-line treatment for either metastatic squamous or metastatic nonsquamous NSCLC in Chinese patients compared with placebo + chemo. However, we found that sugema + chemo would be cost-effective in patients withmetastatic squamous and non-squamous NSCLC when sugemalimab's price was decreased by > 39.0% and 64.8%, respectively.

Economic evaluation of Motor Neuron Diseases: a nationwide cross-sectional analysis in Germany

Background and objectivesMotor Neuron Diseases (MND) are rare diseases but have a high impact on affected individuals and society. This study aims to perform an economic evaluation of MND in Germany.MethodsPrimary patient-reported data were collected including individual impairment, the use of medical and non-medical resources, and self-rated Health-Related Quality of Life (HRQoL). Annual socio-economic costs per year as well as Quality-Adjusted Life Years (QALYs) were calculated.Results404 patients with a diagnosis of Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA) or Hereditary Spastic Paraplegia (HSP) were enrolled. Total annual costs per patient were estimated at 83,060€ in ALS, 206,856€ in SMA and 27,074€ in HSP. The main cost drivers were informal care (all MND) and disease-modifying treatments (SMA). Self-reported HRQoL was best in patients with HSP (mean EuroQoL Five Dimension Five Level (EQ-5D-5L) index value 0.67) and lowest in SMA patients (mean EQ-5D-5L index value 0.39). QALYs for patients with ALS were estimated to be 1.89 QALYs, 23.08 for patients with HSP and 14.97 for patients with SMA, respectively. Cost-utilities were estimated as follows: 138,960€/QALY for ALS, 525,033€/QALY for SMA, and 49,573€/QALY for HSP. The main predictors of the high cost of illness and low HRQoL were disease progression and loss of individual autonomy.ConclusionAs loss of individual autonomy was the main cost predictor, therapeutic and supportive measures to maintain this autonomy may contribute to reducing high personal burden and also long-term costs, e.g., care dependency and absenteeism from work.

Cost-effectiveness of an antibacterial envelope for infection prevention in patients undergoing cardiac resynchronization therapy reoperations in Denmark.

Use of an absorbable antibacterial envelope during implantation prevents cardiac implantable electronic device infections in patients with a moderate-to-high infection risk. Previous studies demonstrated that an envelope is cost-effective in high-risk patients within German, Italian, and English healthcare systems, but these analyses were based on limited data and may not be generalizable to other healthcare settings. A previously published decision-tree-based cost-effectiveness model was used to compare the costs per quality-adjusted life year (QALY) associated with adjunctive use of an antibacterial envelope for infection prevention compared to standard-of-care intravenous antibiotics. The model was adapted using data from a Danish observational two-centre cohort study that investigated infection-risk patients undergoing cardiac resynchronization therapy (CRT) reoperations with and without an antibacterial envelope (n = 1943). We assumed a cost-effectiveness threshold of €34 125/QALY gained, based on the upper threshold used by the National Institute for Health and Care Excellence (£30 000). An antibacterial envelope was associated with an incremental cost-effectiveness ratio (ICER) of €12 022 per QALY in patients undergoing CRT reoperations, thus indicating that the envelope is cost-effective when compared with standard of care. A separate analysis stratified by device type showed ICERS of €6227 (CRT defibrillator) and €29 177 (CRT pacemaker) per QALY gained. Cost-effectiveness ratios were favourable for patients undergoing CRT reoperations in the Danish healthcare system, and thus are in line with previous studies. Results from this study can contribute to making the technology available to Danish patients and align preventive efforts in the pacemaker and ICD area.

Cost-effectiveness analysis of toripalimab plus chemotherapy as the first-line treatment in patients with advanced non-small cell lung cancer (NSCLC) without EGFR or ALK driver mutations from the Chinese perspective.

Objectives: The results of a CHOICE-1 study demonstrated the superior efficacy of toripalimab (anti-PD-1 antibody) plus chemotherapy for patients with advanced non-small cell lung cancer (NSCLC), with a manageable safety profile. This study was performed to evaluate the economic value of this treatment for this patient population from the Chinese payer's perspective. Materials and methods: Basic data were derived from the CHOICE-1 study. Markov models were developed to simulate the process of advanced NSCLC, including the progression-free survival (PFS), progressive disease (PD), and death in intention-to-treat (ITT) populations, as well as patients with squamous or non-squamous NSCLC. The cost was obtained from the local institution, and the value of utilities referred to previous studies. Monte Carlo simulations were performed to depict the probabilistic scatter plots of the incremental cost-effectiveness ratio (ICER) and acceptability curves, aiming to address the uncertainty of model inputs. Results: Compared with standard chemotherapy, toripalimab plus chemotherapy yields an ICER of $21,563 per quality-adjusted life year (QALY) in the ITT population. For patients with squamous NSCLC, comparing the combined therapy with chemotherapy led to an ICER of $18,369 per QALY, while the ICER was $24,754 per QALY in patients with non-squamous NSCLC. With the threshold of willingness to pay we set ($37,653 per QALY), toripalimab plus chemotherapy was cost-effective in these patient populations. Conclusion: For patients with advanced NSCLC, toripalimab plus chemotherapy was an optimal choice as first-line treatment, regardless of histology.

Predicting the Societal Value of Lecanemab in Early Alzheimer's Disease in Japan: A Patient-Level Simulation.

Alzheimer's disease (AD), a neurodegenerative disorder that progresses from mild cognitive impairment (MCI) to dementia, is responsible for significant burden on caregivers and healthcare systems. In this study, data from the large phaseIII CLARITY AD trial were used to estimate the societal value of lecanemab plus standard of care (SoC) versus SoC alone against a range of willingness-to-pay (WTP) thresholds from a healthcare and societal perspective in Japan. A disease simulation model was used to evaluate the impact of lecanemab on disease progression in early AD based on data from the phaseIII CLARITY AD trial and published literature. The model used a series of predictive risk equations based on clinical and biomarker data from the Alzheimer's Disease Neuroimaging Initiative and Assessment of Health Economics in Alzheimer's DiseaseII study. The model predicted key patient outcomes, including life years (LYs), quality-adjusted life years (QALYs), and total healthcare and informal costs of patients and caregivers. Over a lifetime horizon, patients treated with lecanemab plus SoC gained an additional 0.73LYs compared with SoC alone (8.50years vs. 7.77years). Lecanemab, with an average treatment duration of 3.68years, was found to be associated with a 0.91 increase in patient QALYs and a total increase of 0.96 when accounting for caregiver utility. The estimated value of lecanemab varied according to the WTP thresholds (JPY5-15 million per QALY gained) and the perspective employed. From the narrow healthcare payer's perspective, it ranged from JPY1,331,305 to JPY3,939,399. From the broader healthcare payer's perspective, it ranged from JPY1,636,827 to JPY4,249,702, while from the societal perspective, it ranged from JPY1,938,740 to JPY4,675,818. The use of lecanemab plus SoC would improve health and humanistic outcomes with reduced economic burden for patients and caregivers with early AD in Japan.

Health utilities and quality-adjusted life years for patients with amyotrophic lateral sclerosis receiving reldesemtiv or placebo in FORTITUDE-ALS

AimsTo estimate the health utilities and quality-adjusted life years (QALYs) in patients with amyotrophic lateral sclerosis (ALS) receiving reldesemtiv versus placebo in FORTITUDE-ALS.Materials and methodsWe performed a post hoc analysis of clinical trial data from FORTITUDE-ALS (NCT03160898). This Phase IIb, double-blind, randomized, dose-ranging, placebo-controlled, parallel-group, 12-week trial evaluated reldesemtiv in patients with ALS. Health utilities from the five-level version of the EuroQol five-dimensional questionnaire (EQ-5D-5L) were estimated using ALS Functional Rating Scale-Revised (ALSFRS-R) scores collected during the trial. QALYs were estimated using the area under the curve method.ResultsThe full analysis set consisted of 456 patients (reldesemtiv n = 342, placebo n = 114), who received at least one dose of the double-blind study drug, and had ALSFRS-R assessed at baseline and at least one post-baseline assessment. The difference in EQ-5D-5L utility least-squares (LS) mean change from baseline to week 12 for reldesemtiv versus placebo, adjusted for baseline values, was statistically significant (0.03, 95% confidence interval [CI]: 0.01, 0.05; p = .0008). The incremental QALY of reldesemtiv versus placebo adjusted for baseline utility values showed a modest, but statistically significant, difference (0.004, 95% CI: 0.001, 0.007; p = .0058).ConclusionsThis post hoc analysis of FORTITUDE-ALS suggests that reldesemtiv showed a modest but significant benefit in health utilities and QALYs compared with placebo. Future long-term studies that include direct collection of EQ-5D-5L data will be needed to confirm our findings. ClinicalTrials.gov identifierNCT03160898

Cost-effectiveness of idecabtagene vicleucel compared with conventional care in triple-class exposed relapsed/refractory multiple myeloma patients in Canada and France

BackgroundThe chimeric antigen receptor (CAR) T-cell therapy idecabtagene vicleucel (ide-cel) is approved for the treatment of adult patients with relapsed/refractory multiple myeloma (RRMM) who have already received an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have progressed on their last therapy. The objective of this study was to assess the cost-effectiveness of ide-cel versus conventional care in Canada and France.MethodsA partitioned survival model was used to estimate the cost-effectiveness of ide-cel (target dose 450 × 106 CAR T cells) in its approved indication in terms of life-years (LYs), quality-adjusted LYs (QALYs), and costs. Patient-level data from the KarMMa Phase II clinical trial (clinicaltrials.gov NCT03361748) and KarMMa-RW study were used to inform the model; overall and progression-free survival were extrapolated using standard parametric functions after the observed periods. The model adopted Canadian and French societal perspectives over a lifetime horizon. Costs, utilities, discounting (Canada: 1.5%, France: 2.5%), and general population mortality were country-specific.ResultsThe base case demonstrated that ide-cel was associated with more additional LYs (+2.64 and +2.51) and QALYs (+2.31 and +2.54) than conventional care at incremental costs of CAN$588,490 and €392,251 in Canada and France, respectively. The resulting incremental cost-effectiveness ratio (ICER) for ide-cel was $255,245 per QALY in Canada, and €154,593 per QALY in France.ConclusionIde-cel was associated with significant survival improvements in terms of both LYs and QALYs in patients with progressive triple-class-exposed RRMM. The ICER for ide-cel was similar to that of other approved and reimbursed RRMM therapies.

Estimating the Clinical and Economic Impact of Introducing a New Antibacterial into Greek Clinical Practice for the Management of Hospital-Acquired Infections with Limited Treatment Options

Hospital-acquired infections (HAIs) and growing antimicrobial resistance (AMR) represent a significant healthcare burden globally. Especially in Greece, HAIs with limited treatment options (LTO) pose a serious threat due to increased morbidity and mortality. This study aimed to estimate the clinical and economic value of introducing a new antibacterial for HAIs with LTO in Greece. A previously published and validated dynamic model of AMR was adapted to the Greek setting. The model estimated the clinical and economic outcomes of introducing a new antibacterial for the treatment of HAIs with LTO in Greece. The current treatment pathway was compared with introducing a new antibacterial to the treatment sequence. Outcomes were assessed from a third-party payer perspective, over a 10-year transmission period, with quality-adjusted life years (QALYs) and life years (LYs) gained considered over a lifetime horizon. Over the next 10years, HAIs with LTO in Greece account for approximately 1.4 million hospital bed days, hospitalisation costs of more than €320 million and a loss of approximately 403,000LYs (319,000QALYs). Introduction of the new antibacterial as first-line treatment provided the largest clinical and economic benefit, with savings of up to 93,000 bed days, approximately €21 million in hospitalisation costs and an additional 286,000LYs (226,000QALYs) in comparison to the current treatment strategy. The introduction of a new antibacterial was linked to a monetary benefit of €6.8 billion at a willingness to pay threshold of €30,000 over 10years. This study highlights the considerable clinical and economic benefit of introducing a new antibacterial for HAIs with LTO in Greece. This analysis shows the additional benefit when a new antibacterial is introduced to treatment sequences. These findings can be used to inform decision makers to implement policies to ensure timely access to new antibacterial treatments in Greece.

Impact of collateral flow on cost-effectiveness of endovascular thrombectomy.

Acute ischemic stroke patients with large-vessel occlusion and good collateral blood flow have significantly better outcomes than patients with poor collateral circulation. The purpose of this study was to evaluate the cost-effectiveness of endovascular thrombectomy (EVT) based on collateral status and, in particular, to analyze its effectiveness in ischemic stroke patients with poor collaterals. A decision analysis study was performed with Markov modeling to estimate the lifetime quality-adjusted life-years (QALYs) and associated costs of EVT based on collateral status. The study was performed over a lifetime horizon with a societal perspective in the US setting. Base-case analysis was done for good, intermediate, and poor collateral status. One-way, two-way, and probabilistic sensitivity analyses were performed. EVT resulted in greater effectiveness of treatment compared to no EVT/medical therapy (2.56 QALYs in patients with good collaterals, 1.88 QALYs in those with intermediate collaterals, and 1.79 QALYs in patients with poor collaterals), which was equivalent to 1050, 771, and 734 days, respectively, in a health state characterized by a modified Rankin Scale (mRS) score of 0-2. EVT also resulted in lower costs in patients with good and intermediate collaterals. For patients with poor collateral status, the EVT strategy had higher effectiveness and higher costs, with an incremental cost-effectiveness ratio (ICER) of $44,326/QALY. EVT was more cost-effective as long as it had better outcomes in absolute numbers in at least 4%-8% more patients than medical management. EVT treatment in the early time window for good outcome after ischemic stroke is cost-effective irrespective of the quality of collateral circulation, and patients should not be excluded from thrombectomy solely on the basis of collateral status. Despite relatively lower benefits of EVT in patients with poor collaterals, even smaller differences in better outcomes have significant long-term financial implications that make EVT cost-effective.

P10 Modelling Spillover Effects on Informal Carers: The 'Carer QALY Trap'

Caring for patients can impose significant ‘spillover effects’ on the health of informal carers. A review of National Institute for Health and Care Excellence technology appraisals found that carer health-related quality of life (HRQOL) is rarely included in appraisals, and when it is, modelling approaches have been inconsistent. Furthermore, when modelling carer HRQOL, situations can occur whereby a life extension for patients results in an overall reduction in net quality-adjusted life years (QALYs). In other words, for some life-extending treatments, a gain in patient QALYs may be outweighed by a loss in carer QALYs, which may be inconsistent with societal values. The objective of this study was to explore the circumstances through which this issue occurs and consider potential approaches for addressing it.

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

Introduction Ivosidenib (IVO) is an oral inhibitor of mIDH1 that is approved in the USA as monotherapy for pts with mIDH1AML, either relapsed/refractory or newly diagnosed, but older/unfit for intensive therapy. The recent AGILE trial (Montesinos et al. NEJM 2022) randomized newly diagnosed, older/intensive chemotherapy-ineligible mIDH1 AML pts to IVO + AZA vs placebo + AZA and showed improved event-free survival (EFS) and overall survival (OS) in the intervention arm. We sought to understand the health economic implications by conducting a cost-effectiveness analysis. Methods Pts with newly diagnosed mIDH1 AML entered our partitioned survival model at age 75 years and received either IVO/AZA or AZA monotherapy. We used recreated pt-level data derived from the Kaplan-Meier curves and at-risk tables for EFS and OS from the AGILE trial for the IVO/AZA arm to fit loglogistic parametric survival distributions. Hazard ratios (HRs) for EFS and OS were used to derive the EFS and OS curves for the AZA arm, respectively. Medication costs were derived from the average sales price (ASP) for AZA and cytarabine listed by the Centers for Medicare and Medicaid Services or the average wholesale price (AWP) for IVO and venetoclax (Ven), respectively. As ASP ranges between 26-30% below the AWP, we used a 28% discount off the AWP for the base-case scenario and varied this discount during sensitivity analyses between 18% and 38%. Costs and practice patterns of treatment discontinuation or treatment interruption due to adverse events, subsequent treatments, supportive and terminal care, and incidence of complications were derived from the original trial or published literature (Table 1). Costs were adjusted for inflation to 2021 US dollars using the personal consumption expenditure health index. Utilities were derived from the literature and were assumed to be equal between treatment arms for the base case scenario as the quality-of-life assessment were not statistically significantly different for IVO/AZA vs AZA in the AGILE trial at most timepoints. Costs and utilities were modeled over a 30-year time horizon and discounted by 3% annually. Model outputs were used to calculate the incremental cost-effectiveness ratio (ICER) for IVO/AZA, which represents the cost in 2021 US dollars (USD) of each additional quality adjusted life-year (QALY) gained compared to AZA. One-way sensitivity analyses were performed to evaluate the impact of individual parameters on the overall model. Utility values were varied with a 10% range and all other variables were varied across a 50% range. We also performed probabilistic sensitivity analyses, in which we described each parameter using a distribution and performed 10,000 Monte Carlo simulations, each time randomly sampling from the distributions of model inputs. Results In the base case scenario, IVO/AZA and AZA resulted in life-time costs of $652,088 and $242,828, respectively, for an incremental cost of $409,260 with IVO/AZA. With an incremental gain of 2.21 QALYs with IVO/AZA (IVO/AZA: 3.10 QALYs; AZA: 0.89 QALYs), the ICER of IVO/AZA compared to AZA was $185,024/QALY. In one-way sensitivity analyses, only a reduction in the AWP of IVO by 32.4% (from $33,693 to $22,788 per 28-day cycle) would yield a reduction in the ICER to below the conventional willingness-to-pay (WTP) threshold of $150,000/QALY. Figure 1 shows the ten variables with the greatest influence on the ICER demonstrating that variations in any of the variables would not achieve an ICER of less than $150,000/QALY. Probabilistic sensitivity analysis yielded a median ICER of $205,124/QALY (95% CI: $129,344 - $346,479) with AZA favored in 91.2% of 10,000 iterations at a WTP threshold of $150,000/QALY. Conclusion Even in the best-case scenario in favor of the IVO arm following AGILE trial assumptions, we calculated an ICER of $185,024/QALY, which makes IVO/AZA unlikely to be cost-effective compared to AZA alone when applying the traditional WTP threshold of $150,000/QALY in patients with newly diagnosed mIDH1 AML. Only a reduction in the price of IVO by 32.4% lowered the ICER to below $150,000/QALY. The influence of more frequent use of 2nd line IVO in the control arm, comparison against an AZA-Ven control arm, differences in utilities, and extended duration of follow up in the prematurely terminated trial on the ICER warrant additional analyses but are likely to further worsen the cost effectiveness of IVO. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Cost-effectiveness of a centrifugal-flow pump for patients with advanced heart failure in Argentina.

BackgroundCentrifugal-flow pumps are novel treatment options for patients with advanced heart failure (HF). This study estimated the incremental cost-effectiveness ratio (ICER) of centrifugal-flow pumps for patients with advanced HF in Argentina.MethodsTwo Markov models were developed to estimate the cost-effectiveness of a centrifugal-flow pump as destination therapy (DT) in patients with contraindication for heart transplantation, and as bridge-to-transplant (BTT), with a lifetime horizon using the third-party payer Social Security (SS) and Private Sector (PS) perspectives. Clinical, epidemiological, and quality-adjusted life years (QALY) parameters were retrieved from the literature. Direct medical costs were estimated through a micro-costing approach (exchange rate USD 1 = ARS 59.95).ResultsThe centrifugal-flow pump as a DT increased the per patient QALYs by 3.5 and costs by ARS 8.1 million in both the SS and PS, with an ICER of ARS 2.3 million per QALY. Corresponding values for a centrifugal-flow pump as BTT were 0.74 QALYs and more than ARS 8 million, yielding ICERs of ARS 11 million per QALY (highly dependent on waiting times). For the 1, 3, and 5 GDP per QALY thresholds, the probability of a centrifugal-flow pump to be cost-effective for DT/BTT was around 2%/0%, 40%/0%, and 80%/1%, respectively.ConclusionThe centrifugal-flow pump prolongs life and improves the quality of life at significantly higher costs. As in Argentina there is no current explicit cost-effectiveness threshold, the final decision on reimbursement will depend on the willingness to pay in each subsector. Nevertheless, the centrifugal-flow pump as a DT was more cost-effective than as a BTT.

Cost-Effectiveness Analysis of Pembrolizumab in Patients With Advanced Esophageal Cancer Based on the KEYNOTE-181 Study.

Background and PurposeThe KEYNOTE-181 study demonstrated that pembrolizumab for advanced or metastatic esophageal cancer in patients with programmed death ligand-1 (PD-L1) combined positive score (CPS) ≥ 10 had a survival advantage and better tolerability than chemotherapy. However, at the same time, pembrolizumab places an economic burden on patients. This study assessed the cost-effectiveness of pembrolizumab based on the KEYNOTE181 study.Materials and MethodsA three-state Markov model [progression-free survival (PFS), progressive disease (PD), and death] based on data from the KEYNOTE-181 study was used to estimate the incremental cost-effectiveness ratio (ICER) of pembrolizumab versus chemotherapy for advanced or metastatic esophageal cancer. The model evaluates the outcomes from the Chinese society's perspective. Costs, quality-adjusted life-years (QALYs), and the ICER in terms of 2021 US$ per QALY gained, were calculated. one-way and probabilistic sensitivity analyses were performed to evaluate the model robustness.ResultsCompared with chemotherapy, pembrolizumab increased costs by $37,201.68, while gaining 0.23 QALYs, resulting in an ICER of $163,165.26 per QALY in patients with PD-L1 CPS ≥ 10. The ICER is $202,708.62 per QALY and $163,643.19 per QALY in the total population and patients with esophageal squamous cell carcinoma, respectively. The ICER was much higher than the commonly accepted willingness-to-pay threshold ($11,105.8 per QALY). One-way and sensitivity analyses showed that the costs of pembrolizumab and the utility of PD were the crucial factors in determining the ICER, and probabilistic sensitivity analyses demonstrated pembrolizumab is unlikely to be cost-effective at a willingness-to-pay threshold of $11,105.8 per QALY. The result was robust across sensitivity analyses.ConclusionPembrolizumab is not a cost-effective treatment option for the second-line treatment of esophageal cancer from the perspective of Chinese society.

Abstract TP81: Cost Effectiveness Of Cilostazol Added To Aspirin Or Plavix For Secondary Stroke Prevention Following Non-cardioembolic Stroke

Background: The objective of the study is to compare the cost-effectiveness of cilostazol (a selective phosphodiesterase 3 inhibitor) added to aspirin (ASA) or Plavix in patients with non-cardioembolic stroke for secondary stroke prevention. Methods: A Markov model decision tree was used to examine lifetime costs and Quality Adjusted Life Years (QALYs) of patients with non-cardioembolic stroke treated with either single antiplatelet (ASA or Plavix) or with additional cilostazol 100 mg twice daily. Cohorts were followed until all patients died from competing risks, ischemic or hemorrhagic stroke. Input parameters are shown in Table 1. Probabilistic sensitivity analysis using Monte-Carlo simulation was used to model 10 000 cohorts of 10 000 patients. Results: The addition of cilostazol to ASA or Plavix is strongly cost saving (Figure 1). In all 10 000 simulations, the cilostazol strategy resulted in lower health care costs compared to ASA or Plavix alone (mean $14 450 lower per patient, SD $8 466 USD). In 9 998/10 000 simulations, the cilostazol strategy resulted in higher QALYs (mean 0.6016 QALY per patient, SD 0.0704). This result remained robust across a variety of sensitivity analyses varying cost inputs and treatment effects. Conclusion: Based on best available data, the addition of cilostazol to ASA or Plavix for secondary prevention results in significantly reduced healthcare costs and higher lifetime QALYs for patients with non-cardioembolic stroke. Confirmation with high quality data from a randomized trial including a high proportion of non-Asian patients is needed to increase generalizability.