Increase In Quality-adjusted Life Years Research Articles

Transcatheter aortic valve implantation versus surgical aortic valve replacement in Chinese patients with intermediate and high surgical risk for aortic stenosis: a decision analysis on effect, affordability and cost-effectiveness

ObjectiveExamine the cost-effectiveness of transcatheter aortic valve implantation (TAVI) versus surgical aortic valve replacement (SAVR) for Chinese patients with severe aortic stenosis (AS) at intermediate and high surgical risk.DesignA two-phase...

Picking the low-hanging fruit: the cost-effectiveness of opportunistic diabetes screening during tuberculosis contact investigations on the Texas–Mexico border

ObjectiveThere is a known association between type 2 diabetes (diabetes) and tuberculosis (TB), and TB clinics have become hubs for new diabetes diagnosis among active patients with TB. However, despite...

Cost-effectiveness analysis of mindfulness-based cognitive therapy in patients with anxiety disorders in secondary mental health care settings alongside a randomized controlled trial.

Anxiety disorder is one of the most prevalent mental disorders. Mindfulness-based cognitive therapy (MBCT) is effective for treating anxiety disorders. However, no studies have investigated the cost-effectiveness of MBCT for anxiety disorders. We aimed to conduct a cost-effectiveness analysis alongside a randomized controlled trial (RCT) to clarify the cost-effectiveness of MBCT for anxiety disorders. A cost-effectiveness analysis alongside an RCT was conducted for 8 weeks in 40 patients with anxiety disorders at a university hospital. Patients (1) aged 20-75 years; (2) who were diagnosed with panic disorder/agoraphobia or social anxiety disorder based on the Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition, criteria; and (3) who provided written consent were analyzed. The participants were allocated randomly (1:1 ratio) to the augmented MBCT group (i.e., MBCT plus treatment as usual [TAU]) or TAU (waitlist control) group. The cost-effectiveness was assessed using the incremental cost-effectiveness ratio (ICER), which is the ratio of the incremental costs divided by the incremental state-trait anxiety inventory- state (STAI-S), state-trait anxiety inventory- trait (STAI-T), and quality-adjusted life years (QALYs). The QALYs were estimated using The Japanese version of EuroQoL five-dimensional 3-level questionnaire. The unit cost data were derived from the government-regulated fees. This study was conducted from a public healthcare insurance perspective. No discount rates were considered. A total of 38 participants with complete data were included in the analysis. The MBCT was JPY 13,885 more than the cost of TAU and was associated with a STAI-S, STAI-T, and QALY increase of 10.13, 12.00, 0.009 respectively. The ICER were JPY 1,371 (USD13) per STAI-S, JPY 1,157 (USD 11) per STAI-T, and JPY 1,566,357 (USD 14,940) per QALY respectively. MBCT had an 77.5% probability of being cost-effective at a willingness to pay threshold in Japan (JPY 5,000,000 per QALY). The results of the four one-way sensitivity analyses supported the robustness of the base-case analysis findings. Augmented MBCT for anxiety disorders is cost-effective compared with TAU post-treatment from a public healthcare insurance perspective. Future studies should include long-term observations, and analysis from a societal perspective.

US cost-utility model of lenacapavir plus optimized background regimen (OBR) vs fostemsavir plus OBR and ibalizumab plus OBR for people with HIV with multidrug resistance.

Heavily treatment-experienced (HTE) people with HIV (PWH) have limited treatment options owing to multidrug resistance (MDR). Lenacapavir (LEN) is indicated, in combination with other antiretrovirals, for the treatment of adults with MDR HIV-1 experiencing failure of their current antiretroviral regimen because of resistance, intolerance, or safety considerations. To evaluate the cost-utility of LEN in combination with an optimized background regimen (OBR) vs alternative recently approved treatments for HTE PWH, fostemsavir (FTR)+OBR and ibalizumab (IBA)+OBR, for the treatment of PWH with MDR, from a mixed US health care payer perspective. A Markov state-transition model with a lifetime time horizon was developed. Transition probabilities between viral load categories were based on individual participant data from the CAPELLA trial for LEN+OBR and on relative efficacy parameters obtained from indirect treatment comparisons for comparators. Health state utilities were sourced from the literature. Costs included drug acquisition costs, drug administration costs, disease management costs, adverse event costs, AIDS-related event costs, and treatment switching costs and were sourced from red book costs, Medicare and Medicaid fees, and the literature. Costs and outcomes were discounted at 3% annually. The model was used to estimate total and incremental costs, life-years (LYs), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios. A deterministic and a probabilistic sensitivity analysis, as well as scenario analyses, were performed to address elements of uncertainty in the model and to explore the robustness of the results. Over a lifetime time horizon, LEN+OBR was associated with the highest absolute QALYs (9.41) and the greatest number of LYs (12.09) compared with FTR+OBR (QALYs: 8.75; LYs: 11.26) and IBA+OBR (QALYs: 8.36; LYs: 10.78). LEN+OBR was also associated with the lowest total lifetime costs of the 3 interventions (LEN+OBR: $1,441,122 [US dollars]; FTR+OBR: $1,504,986; IBA+OBR: $1,524,396) and therefore was dominant over both comparators in the base case. LEN+OBR remained dominant vs FTR+OBR and IBA+OBR across the range of scenarios tested and LEN+OBR had a 99% probability of being cost-effective compared with FTR+OBR and IBA+OBR in the probabilistic sensitivity analysis at a willingness-to-pay threshold of $50,000/QALY. This economic evaluation demonstrated that LEN+OBR provides meaningful increases in QALYs and LYs, and is dominant over a lifetime time horizon, compared with FTR+OBR and IBA+OBR for the treatment of PWH with MDR in the United States.

Cost-Effectiveness of Hepatocellular Carcinoma Surveillance Strategies in Patients With Compensated Liver Cirrhosis in the United Kingdom

ObjectivesThis study aimed to evaluate the cost-effectiveness (CE) of 4 hepatocellular carcinoma (HCC) surveillance strategies in the United Kingdom, the gender and age with the biomarkers alpha-fetoprotein-L3, alpha-fetoprotein, and des-γ-carboxy prothrombin (GAAD) algorithm, which combines gender (biological sex) and age with Elecsys® biomarker assays, alpha-fetoprotein (AFP) and protein induced by vitamin K absence-II, ultrasound (US), US + AFP and GAAD + US. MethodsA de novo microsimulation state-transition Markov model was developed in Microsoft Excel® from the perspective of the UK National Health Service to calculate life-years, quality-adjusted life-years (QALYs), costs, incremental CE ratios, and net monetary benefits. Parameters were sourced from peer-reviewed published literature, national guidelines, and public cost databases. Sensitivity and scenario analyses were performed to evaluate the impact of parameter and structural uncertainty on the results. ResultsIn a simulated cohort of 100 000 patients, discounted costs and QALYs per patient were £8663 and 6·066 for US, £9095 and 6·076 for US + AFP, £8719 and 6·078 for GAAD alone, and £9114 and 6·086 for GAAD + US. At a CE threshold of £20 000/QALY, GAAD was the most cost-effective strategy; however, although most costly, GAAD + US was the most clinically effective. Sensitivity and scenario analyses indicated that HCC incidence along with costs associated with diagnostic performance influence CE. ConclusionConsidering the cost of US and low incidence of HCC in the United Kingdom, this study suggests that GAAD alone or in combination with US are cost-effective surveillance strategies compared with US and US + AFP. Although GAAD + US showed the highest QALY increase, GAAD alone is considered preferable regarding CE; however, better performance estimates for GAAD + US are needed to confirm.

Highly active antiretroviral therapy is necessary but not sufficient. A systematic review and meta-analysis of mortality incidence rates and predictors among HIV-infected adults receiving treatment in Ethiopia, a surrogate study for resource-poor settings

BackgroundOwing to the introduction of highly active antiretroviral therapy (HAART), the trajectory of mortality and morbidity associated with human immunodeficiency virus (HIV) infection has significantly decreased in developed countries. However, this remains a formidable public health challenge for people living with HIV in resource-poor settings. This study was undertaken to determine the pooled person-time incidence rate of mortality, analyze the trend, and identify predictors of survival among HIV-infected adults receiving HAART.MethodsQuantitative studies were searched in PubMed, Embase, Scopus, Google Scholar, African Journals Online, and Web of Science. The Joana Briggs Institute critical appraisal tool was used to assess the quality of the included articles. The data were analyzed using the random-effects Dersimonian-Laird model.ResultsData abstracted from 35 articles involving 39,988 subjects were analyzed. The pooled person-time incidence rate of mortality (all-cause) was 4.25 ([95% uncertainty interval (UI), 3.65 to 4.85]) per 100 person-years of observations. Predictors of mortality were patients aged ≥ 45 years (hazard ratio (HR), 1.70 [95% UI,1.10 to 2.63]), being female (HR, 0.82 [95% UI, 0.70 to 0.96]), history of substance use (HR, 3.10 [95% UI, 1.31 to 7.32]), HIV positive status non disclosure (HR, 3.10 [95% UI,1.31 to 7.32]), cluster of differentiation 4 + T cell - count < 200 cells/mm3 (HR, 3.23 [95% UI, [2.29 to 4.75]), anemia (HR, 2.63 [95% UI, 1.32 to 5.22]), World Health Organisation classified HIV clinical stages III and IV (HR, 3.02 [95% UI, 2.29 to 3.99]), undernutrition (HR, 2.24 [95% UI, 1.61 to 3.12]), opportunistic infections (HR, 1.89 [95% UI, 1.23 to 2.91]), tuberculosis coinfection (HR, 3.34 [95% UI, 2.33 to 4.81]),bedridden or ambulatory (HR,3.30 [95% UI, 2.29 to 4.75]), poor treatment adherence (HR, 3.37 [95% UI,1.83 to 6.22]), and antiretroviral drug toxicity (HR, 2.60 [95% UI, 1.82 to 3.71]).ConclusionDespite the early introduction of HAART in Ethiopia, since 2003, the mortality rate has remained high. Therefore, guideline-directed intervention of identified risk factors should be in place to improve overall prognosis and increase quality-adjusted life years.

Economic analysis of combined multi-cancer detection blood test and usual care.

e23055 Background: A previous multi-cancer detection blood test (MCDBT) in six prevalent and deadly cancers including colorectal, esophageal, liver, lung, ovarian, and pancreatic cancers, has been reported to decrease late-stage incidence and increase 5-year survival rate. However, the economic cost-effectiveness of integrating the MCDBT with usual care and the factors influencing it have not been investigated. Methods: An interception model was utilized to estimate the stage distribution at the time of cancer diagnosis, while a decision-tree model was developed to assess the clinical and economic outcomes of annual MCDBT for individuals aged 50 to 79 in the United States. The performance metrics for the MCDBT were based on the findings of a previous study called THUNDER. The incidence data were derived from the Surveillance, Epidemiology, and End Results Program (SEER), and the associated costs were obtained from SEER-Medicare. Other input data was estimated based on information available in the published literature. Results: In addition to the usual care, the integration of the MCDBT resulted in a shift of 27.9% in cancer cases from stage III-IV to stage I-II, which led to a decrease in per-cancer treatment cost by $5971 and an increase in quality-adjusted life-years by 0.10 per cancer case. The major factors that influenced the incremental cost-effectiveness ratio (ICER) included price, incidence, specificity, and sensitivity. The MCDBT with a price less than $800 showed cost-effectiveness with an ICER below three times the per capita GDP of the United States in the average-risk population. In a high-risk population with the incidence two-fold higher than the average-risk population, the ICER decreased by 58.3%. When compared the influence of specificity and sensitivity on the ICER of the MCDBT, a 5% increase in specificity resulted a decrease in ICER by 48.8%, while a 5% increase in sensitivity only reduced the ICER by 12.7%, suggesting the importance of high specificity in MCDBT. Conclusions: The MCDBT with a price less than $800 showed cost-effectiveness in the detection of colorectal, esophageal, liver, lung, ovarian, and pancreatic cancers. Price, incidence of the intended-use population, specificity and sensitivity were the major factors that influenced the cost-effectiveness.

Cost-Effectiveness of Diffusion Weighted MRI Versus Planned Second-Look Surgery for Cholesteatoma.

To compare the cost-effectiveness of serial non-echo planar diffusion weighted MRI (non-EP DW MRI) versus planned second look surgery following initial canal wall up tympanomastoidectomy for the treatment of cholesteatoma. A decision-analytic model was developed. Model inputs including residual cholesteatoma rates, rates of non-EP DW MRI positivity after surgery, and health utility scores were abstracted from published literature. Cost data were derived from the 2022 Centers for Medicare and Medicaid Services fee rates. Efficacy was defined as increase in quality-adjusted life year (QALY). One- and 2-way sensitivity analyses were performed on variables of interest to probe the model. Total time horizon was 50 years with a willingness to pay (WTP) threshold set at $50 000/QALY. Base case analysis revealed that planned second-look surgery ($11 537, 17.30 QALY) and imaging surveillance with non-EP DWMRI ($10 439, 17.26 QALY) were both cost effective options. Incremental cost effectiveness ratio was $27 298/QALY, which is below the WTP threhshold. One-way sensitivity analyses showed that non-EP DW MRI was more cost effective than planned second-look surgery if the rate of residual disease after surgery increased to 48.3% or if the rate of positive MRI was below 45.9%. A probabilistic sensitivity analysis at WTP of $50 000/QALY found that second-look surgery was more cost-effective in 56.7% of iterations. Non-EP DW MRI surveillance is a cost-effect alternative to planned second-look surgery following primary canal wall up tympanomastoidectomy for cholesteatoma. Cholesteatoma surveillance decisions after initial canal wall up tympanomastoidectomy should be individualized. V.

Cost-effectiveness analysis of HLA-B*15:02 screening before treatment of epilepsy in Indonesia

IntroductionAdverse skin reactions due to drugs such as Stevens Johnson Syndrome (SJS) and Toxic Epidermal Necrolysis (TEN) occur in 3% of people receiving anti epileptic drugs (AED). Although SJS/TEN has a low incidence, the mortality and morbidity rates are high. Indonesia has not adopted HLA-B*1502 screening prior to administration of carbamazepine (CBZ), although previous studies found a relationship between HLA-B*1502 and SJS/TEN. MethodsA hybrid decision tree and Markov model was developed to evaluate three strategies for treating newly diagnosed focal epilepsy: CBZ direct therapy, levetiracetam (LEV) direct therapy, and therapy based on HLA-B*15:02 test results. From a societal perspective, base case and sensitivity analyses were carried out over a lifetime. ResultsDirect administration of CBZ appears to have a slightly lower average cost than the HLA-B*15:02 allele screening strategy. The increase in quality-adjusted life year (QALY) in HLA-B*15:02 screening before treatment related to the cost difference reached 0.519 with an incremental cost-effectiveness ratio (ICER) of around USD 984 per unit of QALY acquisition. Direct treatment of LEV increased treatment costs by almost USD 2000 on average compared to the standard CBZ strategy. The increase in QALY is 0.834 in direct levetiracetam treatment, with an ICER of around USD 2230 for each QALY processing. ConclusionCalculation of the cost-effectiveness of lifetime epilepsy therapy in this study found that the initial screening strategy with the HLA-B*15:02 test was the most cost-effective.

Cost-Utility Analysis Comparing Pegcetacoplan to Anti-C5 Monoclonal Antibodies in the Treatment of Paroxysmal Nocturnal Hemoglobinuria.

Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease characterized by hemolytic episodes and associated with significant clinical burden. The introduction of C5 inhibitory monoclonal antibodies (C5i) represented a major breakthrough in PNH treatment, effectively reducing intravascular hemolysis (IVH) but showing limited impact on extravascular hemolysis (EVH). In 2021, the C3 inhibitor pegcetacoplan was approved by EMA and recently reimbursed in Italy, which also has the advantages in the reduction of both IVH and EVH, increasing hemoglobin values and simultaneously improving the quality of life and fatigue of patients. A cost-utility analysis was developed to compare pegcetacoplan to C5i (eculizumab and ravulizumab) in the PNH population who remain anemic after treatment with C5i for at least 3 months. The analysis employed a Markov model with a 5-year time horizon whereby patients can transition among 3 PNH health states, adopting the perspective of the Italian NHS. Efficacy data were sourced from the PEGASUS study, with drug prices reflecting ex-factory costs. Additionally, costs associated with resource utilization, adverse events, and complications were estimated based on outpatient and hospital care rates, excluding indirect expenses. Utility and disutility values related to transfusions were also considered, with pegcetacoplan allowing for dose escalation. The cumulative cost of treatment per individual patient at 5 years was estimated to be €1,483,454 for pegcetacoplan, €1,585,763 for eculizumab, and €1,574,826 for ravulizumab. Pegcetacoplan demonstrated a superior increase in quality-adjusted life years (QALYs) compared to both eculizumab (0.51 increase) and ravulizumab (0.27 increase). Furthermore, pegcetacoplan showed a reduction in complication management costs (€22,891 less compared to eculizumab and €22,611 less compared to ravulizumab) and lower transfusion-related expenses (€14,147 less than both C5i treatments). Pegcetacoplan emerged as the dominant strategy in this analysis, being more effective, less expensive and improves quality of life in the analyzed population affected by PNH.

Digital variance angiography in patients undergoing lower limb arterial recanalization: cost-effectiveness analysis within the English healthcare setting.

Aim: Digital variance angiography (DVA) is a recently developed image processing method capable of improving image quality compared with the traditionally used digital subtraction angiography (DSA), among patients undergoing lower limb x-ray angiography. This study aims to explore the potential cost-effectiveness of DVA from an English National Health Service perspective. Materials & methods: A two-part economic model, consisting of a decision tree and a Markov model, was developed to consider the costs and health outcomes associated with the use of DVA as part of current practice imaging, compared with x-ray angiography using standard DSA. The model explored the impact of DVA on the development of acute kidney injury (AKI), chronic kidney disease and radiation-induced cancer over a lifetime horizon. Both deterministic and probabilistic analyses were performed to assess the cost per quality-adjusted life-year (QALY). Results: Base-case results indicate that DVA results in cost savings of £309 per patient, with QALYs also improving (+0.025) over a lifetime. As shown in sensitivity analysis, a key driver of model results is the relative risk (RR) reduction of contrast-associated acute kidney injury associated with use of DVA. The intervention also decreases the risk of carcinoma over a lifetime. Scenario analyses show that cost savings range from £310 to £553, with QALY gains ranging from 0.048 to 0.109 per patient. Conclusion: The use of DVA could result in a decrease in costs and an increase in QALYs over a lifetime, compared with existing imaging practice. The potential for this technology to offer an economically viable alternative to existing image processing methods, through a reduction in contrast media volume and radiation exposure, has been demonstrated.

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.

Cost-effectiveness analysis of olaparib maintenance therapy for BRCA mutation ovarian cancer in the public sector in Malaysia.

Ovarian cancer is one of the most common cancer among women in Malaysia. Patients with ovarian cancer are often diagnosed at an advanced stage. Despite initial response to surgery and chemotherapy, most patients will experience a relapse. Olaparib has been reported have promising effects among BRCA mutated ovarian cancer patients. This study aimed to evaluate the cost-effectiveness of olaparib as a maintenance therapy for BRCA ovarian cancer in Malaysia. We developed a four-state partitioned survival model which compared treatment with olaparib versus routine surveillance (RS) from a Malaysian healthcare perspective. Mature overall survival (OS) data from the SOLO-1 study were used and extrapolated using parametric models. Medication costs and healthcare resource usage costs were derived from local inputs and publications. Deterministic and probabilistic sensitivity analyses (PSA) were performed to explore uncertainties. In Malaysia, treating patients with olaparib was found to be more costly compared to RS, with an incremental cost of RM149,858 (USD 33,213). Patients treated with olaparib increased life years by 3.05 years and increased quality adjusted life years (QALY) by 2.76 (9.45 years vs 6.40 years; 7.62 vs 4.86 QALY). This translated to an incremental cost-effectiveness ratio (ICER) of RM 49,159 (USD10,895) per life year gained and RM54,357 (USD 12,047) per QALY gained, respectively. ICERs were most sensitive to time horizon of treatment, discount rate for outcomes, cost of treatment and health state costs, but was above the RM53,770/QALY threshold. The use of olaparib is currently not a cost-effective strategy compared to routine surveillance based upon the current price in Malaysia for people with ovarian cancer with BRCA mutation, despite the improvement in overall survival.

Cost-Effectiveness of Oral Nirmatrelvir/Ritonavir in Patients at High Risk for Progression to Severe COVID-19 in the United States

ObjectivesNirmatrelvir/ritonavir (NMV/r) is an orally administered antiviral indicated for the outpatient treatment of patients with mild-to-moderate COVID-19 at high risk for disease progression to severe illness. We estimated the cost-effectiveness of NMV/r versus best supportive care for patients with mild-to-moderate COVID-19 at high risk for progression to severe illness from a US health sector perspective. MethodsA cost-effectiveness model was developed using a short-term decision-tree (1 year) followed by a lifetime 2-state Markov model (alive and dead). The short-term decision-tree captured costs and outcomes associated with the primary infection and healthcare utilization; survivors of the short-term decision-tree were followed until death assuming US quality-adjusted life years (QALYs), adjusted in the short-term for survivors of mechanical ventilation. Baseline rate of hospitalization and NMV/r effectiveness were taken from an Omicron-era US real-world study. Remaining inputs were informed by previous COVID-19 studies and publicly available US sources. Sensitivity analyses were conducted for all model inputs to test the robustness of model results. ResultsNMV/r was found to decrease COVID-19 related hospitalizations (−0.027 per infected case) increase QALYs (+0.030), decrease hospitalization costs (−$1110), and increase total treatment cost (+$271), resulting in an incremental cost-effectiveness ratio of $8931/QALY. Results were most sensitive to baseline risk of hospitalization and NMV/r treatment effectiveness parameters. The probabilistic analysis indicated that NMV/r has a >99% probability of being cost-effective at a $100 000 willingness-to-pay threshold. ConclusionsNMV/r is cost-effective vs best supportive care for patients at high risk for severe COVID-19 from a US health sector perspective.

The Cost-Effectiveness of Chin Tuck Against Resistance Compared to Usual Care in Citizens with Oropharyngeal Dysphagia - An Economic Evaluation.

This study aims to evaluate the cost-effectiveness of chin tuck against resistance (CTAR) for citizens suffering from dysphagia compared to the standard municipal treatment in Denmark. A cost-utility analysis, employing a municipal perspective, was conducted using cost data collected alongside clinical data of a randomized controlled trial evaluating the effect of CTAR training for citizens with dysphagia. The composition of the clinical randomized controlled trial, which included citizens with different diagnoses, means that participants had different disease courses. Ninety-two citizens from seven different Danish municipalities were enrolled, of whom 43 received standard care, and 49 received CTAR in addition to standard care. The effect outcome of the economic evaluation was quality-adjusted life years (QALY), estimated using the EQ-5D-5L questionnaire. Individual resource consumption of each citizen was determined based on the use of home care, home nursing care, physio- and occupational therapy, dietitian guidance, and hospital admissions. The incremental costs and QALYs between the intervention group (CTAR in addition to standard care) and standard care group were estimated using regression analysis, and sensitivity analyses were performed to investigate the robustness of the results. The base case analysis showed that the intervention group was dominant compared to the standard care group, with a decrease in incremental costs of £542.38 and an increase in incremental QALYs of 0.0118. All sensitivity analyses demonstrated similar findings as the base case analysis, supporting the robustness of the results. This study found that the intervention group was the dominant alternative, hence being more effective and cost-saving, compared to the standard care group in a Danish municipality perspective with a three-month time horizon. This study adds to the scarce evidence on the cost-effectiveness of CTAR in a Danish clinical setting, but further studies should focus on estimating long-term cost-effectiveness.

Transcatheter aortic valve implantation versus surgical aortic valve replacement in severe aortic stenosis patients at low surgical mortality risk: a cost-effectiveness analysis in Belgium

Background Transcatheter aortic valve implantation (TAVI) with the SAPIEN 3 device has recently shown significant clinical benefits, compared to surgical aortic valve replacement (SAVR), in patients at low risk for surgical mortality (PARTNER 3 trial, NCT02675114). Currently in Belgium, TAVI use is restricted to high-risk or inoperable patients with severe symptomatic aortic stenosis (sSAS). This cost-utility analysis aimed to assess whether TAVI with SAPIEN 3 could lead to potential cost-savings compared with SAVR, in the low-risk sSAS population in Belgium. Methods A previously published, two-stage, Markov-based cost-utility model was used. Clinical outcomes were captured using data from PARTNER 3 and the model was adapted for the Belgian context using cost data from the perspective of the Belgian National Healthcare System, indexed to 2022. A lifetime horizon was chosen. The model outputs included changes in direct healthcare costs, survival and health-related quality of life using TAVI versus SAVR. Results TAVI with SAPIEN 3 provides meaningful clinical and cost benefits over SAVR, in terms of an increase in quality-adjusted life years (QALYs) of 0.94 and cost-saving of €3 013 per patient. While initial procedure costs were higher for TAVI compared with SAVR, costs related to rehabilitation, disabling stroke, treated atrial fibrillation, and rehospitalization were lower. The cost-effectiveness of TAVI over SAVR remained robust in sensitivity analyses. Conclusion TAVI with SAPIEN 3 may offer a meaningful alternative intervention to SAVR in Belgian low-risk patients with sSAS, showing both clinical benefits and cost savings associated with post-procedure patient management.

An economic evaluation of first-line cryoballoon ablation versus antiarrhythmic drug therapy for the treatment of paroxysmal atrial fibrillation from a German healthcare service payer perspective

Abstract Background Three recent randomized controlled trials demonstrated that, in patients with symptomatic paroxysmal atrial fibrillation (PAF), first-line pulmonary vein isolation (PVI) with cryoballoon ablation reduces atrial arrhythmia recurrence when compared to initial antiarrhythmic drug (AAD) therapy. Purpose To evaluate the cost-effectiveness of initial rhythm control therapy from a German healthcare service payer perspective. Methods The cost-effectiveness model (CEM) structure consisted of a hybrid decision tree and Markov model, where the decision tree informed the initial health state allocation in the first cycle of the Markov model and had a one-year time horizon. The Markov model had a 40-year time horizon using a three-month cycle length. Health benefits were expressed in quality-adjusted life years (QALYs). Costs and benefits were discounted at 3.5% p.a. Individual patient-level data from 703 participants with untreated PAF enrolled into Cryo-FIRST (NCT01803438), STOP AF First (NCT03118518) and EARLY-AF (NCT02825979) were used to estimate efficacy, resource use and health-related quality of life parameters. Cost inputs were sourced from diagnosis-related groups and the Institute for the Hospital Remuneration System (InEK). Where parameters could not be derived, inputs were sourced from available published literature or determined through clinical expert opinion. Probabilistic sensitivity analyses were conducted to explore the impact of any assumptions on model outputs. Results In those treated with cryoablation, the three-month rate of AF recurrence was reduced by 46.7% (p&amp;lt;0.001) on average. Similarly, the average monthly rate of receiving an ablation following initial treatment was reduced by 72.8% (p&amp;lt;0.001). Cryoablation was also associated with a 4.3% (p=0.025) increase in health-related quality of life at 12 months, assessed through the standard EQ-5D-3L instrument. There was no difference in the rate of AF resolution in those who failed initial treatment. CEM results are shown in Table 1. Analysis shows that cryoablation is cost-effective, incurring a cost of ∼€1,000 per patient over a lifetime compared to AADs, while offering an increase in QALYs. Cryoablation attains an average ICER of ∼€5,500, with a 94.1% probability of being cost-effective at a willingness-to-pay threshold of €35,000 per QALY gained. Through 5,000 iterations, the probabilistic sensitivity analysis indicates that cryoablation has ∼20% probability of being cost-saving. Individuals are expected to receive a total of ∼1.2 ablations over a lifetime, regardless of initial treatment. Although, those initially treated with cryoablation as opposed to AADs experience a 45% reduction in time spent in AF health states. Conclusion Initial rhythm control with cryoballoon ablation in PAF is a cost-effective treatment option in a German healthcare setting.Table 1:Key results (per patient)

Evaluating Cost-Effectiveness in Using High-Kidney Donor Profile Index Organs

A more granular donor kidney grading scale, the kidney donor profile index (KDPI), has recently emerged in contradistinction to the standard criteria donor/expanded criteria donor framework. In this paper, we built a Markov decision process model to evaluate the survival, quality-adjusted life years (QALY), and cost advantages of using high-KDPI kidneys based on multiple KDPI strata over a 60-month time horizon as opposed to remaining on the waiting list waiting for a lower-KDPI kidney. Data for the model were gathered from the Scientific Registry of Transplant Recipients and the United States Renal Data System Medicare parts A, B, and D databases. Of the 129,024 phenotypes delineated in this model, 65% of them would experience a survival benefit, 81% would experience an increase in QALYs, 87% would see cost-savings, and 76% would experience cost-savings per QALY from accepting a high-KDPI kidney rather than remaining on the waiting list waiting for a kidney of lower-KDPI. Classification and regression tree analysis (CART) revealed the main drivers of increased survival in accepting high-KDPI kidneys were wait time ≥30 months, panel reactive antibody (PRA) <90, age ≥45 to 65, diagnosis leading to renal failure, and prior transplantation. The CART analysis showed the main drivers of increased QALYs in accepting high-kidneys were wait time ≥30 months, PRA <90, and age ≥55 to 65.

The estimated annual financial impact of gene therapy in the United States

Gene therapy is a new class of medical treatment that alters part of a patient’s genome through the replacement, deletion, or insertion of genetic material. While still in its infancy, gene therapy has demonstrated immense potential to treat and even cure previously intractable diseases. Nevertheless, existing gene therapy prices are high, raising concerns about its affordability for U.S. payers and its availability to patients. We assess the potential financial impact of novel gene therapies by developing and implementing an original simulation model which entails the following steps: identifying the 109 late-stage gene therapy clinical trials underway before January 2020, estimating the prevalence and incidence of their corresponding diseases, applying a model of the increase in quality-adjusted life years for each therapy, and simulating the launch prices and expected spending of all available gene therapies annually. The results of our simulation suggest that annual spending on gene therapies will be approximately $20.4 billion, under conservative assumptions. We decompose the estimated spending by treated age group as a proxy for insurance type, finding that approximately one-half of annual spending will on the use of gene therapies to treat non-Medicare-insured adults and children. We conduct multiple sensitivity analyses regarding our assumptions and model parameters. We conclude by considering the tradeoffs of different payment methods and policies that intend to ensure patient access to the expected benefits of gene therapy.

Play brick therapy to aid the social skills of children and young people with autism spectrum disorder: the I-SOCIALISE cluster RCT

BackgroundSocial skills interventions are commonly recommended to help children and young people with autism spectrum disorder develop social skills, but some struggle to engage in these interventions. LEGO®(LEGO System A/S, Billund, Denmark) based therapy, a group social skills intervention, aims to be more interesting and engaging.ObjectiveTo evaluate the clinical effectiveness of LEGO®based therapy on the social and emotional skills of children and young people with autism spectrum disorder in school settings compared with usual support. Secondary objectives included evaluations of cost-effectiveness, acceptability and treatment fidelity.DesignA cluster randomised controlled trial randomly allocating participating schools to either LEGO®based therapy and usual support or usual support only.SettingMainstream schools in the north of England.ParticipantsChildren and young people (aged 7–15 years) with autism spectrum disorder, their parent/guardian, an associated teacher/teaching assistant and a facilitator teacher/teaching assistant (intervention schools only).InterventionSchools randomised to the intervention arm delivered 12 weekly sessions of LEGO®based therapy, which promotes collaborative play and encourages social problem-solving in groups of three children and young people with a facilitator (trained teacher or teaching assistant). Participants received usual support from school and community services. Participants in the control arm received usual support only. Research assistants and statisticians were blind to treatment allocation.Main outcome measureThe social skills subscale of the Social Skills Improvement System (SSIS), completed by the children and young people’s unblinded teacher pre randomisation and 20 weeks post randomisation. The SSIS social skills subscale measures social skills including social communication, co-operation, empathy, assertion, responsibility and self-control. Participants completed a number of other pre- and post-randomisation measures of emotional health, quality of life, loneliness, problem behaviours, academic competence, service resource utilisation and adverse events.ResultsA total of 250 children and young people from 98 schools were randomised: 127 to the intervention arm and 123 to the control arm. Intention-to-treat analysis of the main outcome measure showed a modest positive difference of 3.74 points (95% confidence interval –0.16 to 7.63 points, standardised effect size 0.18;p = 0.06) in favour of the intervention arm. Statistical significance increased in per-protocol analysis, with a modest positive difference (standardised effect size 0.21;p = 0.036). Cost-effectiveness of the intervention was found in reduced service use costs and a small increase in quality-adjusted life-years. Intervention fidelity and acceptability were positive. No intervention-related adverse events or effects were reported.ConclusionsThe primary and pre-planned sensitivity analysis of the primary outcome consistently showed a positive clinical difference, with modest standardised effect sizes of between 0.15 and 0.21. There were positive health economics and qualitative findings, corroborated by the difference between arms for the majority of secondary outcomes, which were not statistically significant but favoured the intervention arm. Post hoc additional analysis was exploratory and was not used in drawing this conclusion. Given the small positive change, LEGO®based therapy for children and young people with autism spectrum disorder in schools should be considered.LimitationsThe primary outcome measure was completed by an unblinded teacher (rather than by the facilitator).Future workThe study team recommends future research into LEGO®based therapy, particularly in school environments.Trial registrationThis trial is registered as ISRCTN64852382.FundingThis award was funded by the National Institute for Health and Care Research (NIHR) Public Health Research programme (NIHR award ref: 15/49/32) and is published in full inPublic Health Research; Vol. 11, No. 12. See the NIHR Funding and Awards website for further award information.