Intensive behavioural interventions based on applied behaviour analysis (ABA) for young children with autism: A cost-effectiveness analysis

Cost-utility analysis of teduglutide compared to standard care in weaning parenteral nutrition support in children with short bowel syndrome

Digital therapeutic care (DTC) programs are unsupervised app-based treatments that provide video exercises and educational material to patients with nonspecific low back pain during episodes of pain and functional disability. German statutory health insurance can reimburse DTC programs since 2019, but evidence on efficacy and reasonable pricing remains scarce. This paper presents a probabilistic sensitivity analysis (PSA) to evaluate the efficacy and cost-utility of a DTC app against treatment as usual (TAU) in Germany. The aim of this study was to perform a PSA in the form of a Monte Carlo simulation based on the deterministic base case analysis to account for model assumptions and parameter uncertainty. We also intend to explore to what extent the results in this probabilistic analysis differ from the results in the base case analysis and to what extent a shortage of outcome data concerning quality-of-life (QoL) metrics impacts the overall results. The PSA builds upon a state-transition Markov chain with a 4-week cycle length over a model time horizon of 3 years from a recently published deterministic cost-utility analysis. A Monte Carlo simulation with 10,000 iterations and a cohort size of 10,000 was employed to evaluate the cost-utility from a societal perspective. Quality-adjusted life years (QALYs) were derived from Veterans RAND 6-Dimension (VR-6D) and Short-Form 6-Dimension (SF-6D) single utility scores. Finally, we also simulated reducing the price for a 3-month app prescription to analyze at which price threshold DTC would result in being the dominant strategy over TAU in Germany. The Monte Carlo simulation yielded on average a €135.97 (a currency exchange rate of EUR €1=US $1.069 is applicable) incremental cost and 0.004 incremental QALYs per person and year for the unsupervised DTC app strategy compared to in-person physiotherapy in Germany. The corresponding incremental cost-utility ratio (ICUR) amounts to an additional €34,315.19 per additional QALY. DTC yielded more QALYs in 54.96% of the iterations. DTC dominates TAU in 24.04% of the iterations for QALYs. Reducing the app price in the simulation from currently €239.96 to €164.61 for a 3-month prescription could yield a negative ICUR and thus make DTC the dominant strategy, even though the estimated probability of DTC being more effective than TAU is only 54.96%. Decision-makers should be cautious when considering the reimbursement of DTC apps since no significant treatment effect was found, and the probability of cost-effectiveness remains below 60% even for an infinite willingness-to-pay threshold. More app-based studies involving the utilization of QoL outcome parameters are urgently needed to account for the low and limited precision of the available QoL input parameters, which are crucial to making profound recommendations concerning the cost-utility of novel apps.

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

SummaryBackgroundTailored smoking cessation interventions, which combine behavioural and pharmaceutical support, are effective in populations with severe mental illness (SMI). We establish the cost-effectiveness of two tailored interventions in the UK: (i) a bespoke smoking cessation intervention (BSCI) versus usual care, and (ii) integrated tobacco cessation and mental health care (IC) versus standard smoking cessation clinic (SCC) referral.MethodsThis economic evaluation was conducted between January 15th 2019 and August 4th 2022. We adapted a Markov model estimating smoking status, healthcare costs and quality-adjusted life years (QALYs) across the lifetime. Intervention effectiveness and costs were obtained from a systematic review and a meta-analysis. We obtained specific parameter values for populations with SMI for mortality, risk of smoking related comorbidities, and health utility. Uncertainty was analysed in deterministic and probabilistic sensitivity analysis (PSA).FindingsThe BSCI was cost-effective versus usual care with an incremental cost-effectiveness ratio (ICER) of £3145 per QALY (incremental costs: £165; incremental QALYs: 0.05). Integrated care was cost-effective versus SCC with an ICER of £6875 per QALY (incremental costs: £292; incremental QALYs: 0.04). The BSCI and IC were cost-effective in 89% and 83% of PSA iterations respectively. The main area of uncertainty related to relapse rates.InterpretationOur findings suggested that the tailored interventions were cost-effective and could increase QALYs and decrease expenditure on treating smoking related morbidities if offered to people with SMI.FundingYork Health Economics Consortium was funded by the 10.13039/100010377National Institute for Health and Care Excellence to produce economic evaluations to inform public health guidelines.

Trials comparing the effects of transfusing RBC units of different storage durations have considered mortality or morbidity as outcomes. We perform the first economic evaluation alongside a full age of blood clinical trial with a large population assessing the impact of RBC storage duration on quality-of-life and costs in critically ill adults. Quality-of-life was measured at 6 months post randomization using the EuroQol 5-dimension 3-level instrument. The economic evaluation considers quality-adjusted life year and cost implications from randomization to 6 months. A generalized linear model was used to estimate incremental costs (2016 U.S. dollars) and quality-adjusted life years, respectively while adjusting for baseline characteristics. Fifty-nine ICUs in five countries. Adults with an anticipated ICU stay of at least 24 hours when the decision had been made to transfuse at least one RBC unit. Patients were randomized to receive either the freshest or oldest available compatible RBC units (standard practice) in the hospital transfusion service. EuroQol 5-dimension 3-level utility scores were similar at 6 months-0.65 in the short-term and 0.63 in the long-term storage group (difference, 0.02; 95% CI, -0.00 to 0.04; p = 0.10). There were no significant differences in resource use between the two groups apart from 3.0 fewer hospital readmission days (95% CI, -5.3 to -0.8; p = 0.01) during follow-up in the short-term storage group. There were no significant differences in adjusted total costs or quality-adjusted life years between the short- and long-term storage groups (incremental costs, -$2,358; 95% CI, -$5,586 to $711) and incremental quality-adjusted life years: 0.003 quality-adjusted life years (95% CI, -0.003 to 0.008). Without considering the additional supply cost of implementing a freshest available RBC strategy for critical care patients, there is no evidence to suggest that the policy improves quality-of-life or reduces other costs compared with standard transfusion practice.

Background: IMPACT (InforMing the PAthway of COPD Treatment, NCT02164513), showed superior exacerbation reduction and lung function improvement with single inhaler, once-daily fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) 100/62.5/22μg vs once-daily FF/VI 100/22μg, for patients with moderate/severe COPD. Objectives: The cost-effectiveness of FF/UMEC/VI vs FF/VI was assessed, from a Canadian public payer perspective. Methods: A validated linked risk equation model (Briggs, Med Decis Making 37;4 2017), which predicts COPD disease progression, associated healthcare costs and health outcomes, was populated with baseline characteristics, efficacy and medication use from IMPACT. Canadian healthcare resource unit costs and drug costs were applied, with future costs and health outcomes discounted at 1.5% annually. Analysis was probabilistic, with a lifetime horizon and outputs including exacerbation rates, costs (2017 CAD), quality-adjusted life years (QALYs) gained and incremental cost effectiveness ratio (ICER) per QALY. Results: Compared with FF/VI, FF/UMEC/VI treatment resulted in fewer moderate and severe exacerbations (10.52 and 3.38 vs 11.13 and 3.48), mean (95% CI) incremental costs and QALYs of $2,598 ($2,010, $3,268) and 0.13 (0.09, 0.18), and an ICER of $19,649 per QALY ($15,406, $26,454). The probability of FF/UMEC/VI being cost-effective vs FF/VI was 100% at a willingness-to-pay threshold of $50,000 per QALY. Results were most sensitive to time horizon, and efficacy of treatment post-discontinuation. Conclusions: FF/UMEC/VI was predicted to improve health outcomes and to be a cost-effective option for treatment of moderate/severe COPD compared with FF/VI, in Canada.

In South Korea, the number of severely obese patients has increased. An economic study comparing bariatric surgery with nonsurgical interventions has not been published for Asia. This study was conducted to evaluate the cost effectiveness of bariatric surgery as compared to nonsurgical interventions for severe obese Korean people. We used the Markov model to compare the lifetime expected costs and quality-adjusted life years (QALYs) between bariatric surgery and nonsurgical interventions from Korean Healthcare system perspectives. Our target cohort consisted of severe obese people defined as having a body mass index of 30-<40 kg/m(2) in South Korea. The starting age of the cohort was 30 years old, and the cycle length was 1 year. Nonsurgical interventions included a physician visit, exercise, diet, and pharmacotherapy. A discount of 5 % was applied in cost and QALY. The incremental cost-effectiveness ratio (ICER) of bariatric surgery compared to nonsurgery interventions was calculated. The cost-utility analysis study indicated that bariatric surgery had US$1,522 incremental costs and 0.86 incremental QALYs as compared to nonsurgical interventions. Through the base case analysis, ICER was US$1,771/QALY. The sensitivity analyses were performed using a variety of assumptions, and the robustness of the study results was also demonstrated. The study indicated that bariatric surgery was a cost-effective alternative to nonsurgical interventions over a lifetime, providing substantial lifetime benefits for severely obese Korean people.

BackgroundAsylum seekers experience large health inequalities and barriers across Europe. Screening for Hepatitis B (HBV), Hepatitis C (HCV) and Tuberculosis (TB) potentially prevents severe disease. We assessed cost-effectiveness of 3 screening strategies for asylum seekers arriving in the UK by small boats.MethodsThree Markov decision-analytic models assessed the cost-effectiveness of ‘systematic screening’ at point of entry (within 3-5 days of arrival) compared to current practice (existing arrangement in primary care) and no screening over 40 years from a health service perspective. Model parameters were sourced from secondary literature, UK government, and NHS sources. We estimated incremental cost per quality-adjusted life year (QALY), incremental cost per case/death averted, and incremental cost-effectiveness ratios (ICERs) with 95% uncertainty intervals. Sensitivity analyses explored the impact of optimistic and pessimistic assumptions on key parameters (i.e., levels of screening uptake, treatment uptake, completion, and efficacy rates).ResultsIn the base-case scenario, for TB, HBV, and HCV, systematic screening is a dominant strategy. compared to the alternatives. Systematic TB screening generated cost savings and QALY gains compared to current practice (cost savings: £222; QALY gains 0.10) and no screening (cost savings: £527; QALY gains 0.36). For HBV, systematic screening led to cost savings and QALY gains compared to current practice (cost savings £111; incremental QALY gains 0.02) and compared to no screening (cost savings £157; QALY gains 0.03). Similarly, for HCV, systematic screening resulted in cost savings (£23 and £33) and QALY gains (0.004 and 0.006) compared to the comparators. Sensitivity analyses led to similar findings.ConclusionsSystematic screening of small boat migrants for TB, HBV, and HCV is cost-effective. Similar health economic analyses can be applied across Europe to inform policy to improve the health of asylum seekers.Key messages• Screening of asylum seekers for TB, HBV & HCV on arrival is likely to be cost-effective in the UK.• Health economic modelling could inform screening policy for asylum seekers entering Europe.

Moderate to high risk medical inpatients are at increased risk of Venous Thromboembolism (VTE). The present study aims to investigate the cost-effectiveness and cost-utility of using Enoxaparin compared to Heparin in VTE prophylaxis in medical inpatients, from Iranian payer's perspective. Decision tree modeling technique was used to evaluate cost-effectiveness and cost-utility of the compared interventions. The main considered outcomes were Life Years Gained (LYG) for Cost-Effectiveness Analysis (CEA) and Quality-Adjusted Life Years (QALY) for Cost-Utility Analysis (CUA). Costs and consequences of the interventions were evaluated for a three-month period and reported as Incremental Cost-Effectiveness Ratios (ICERs). One-way and Probabilistic Sensitivity Analysis (PSA) were conducted to evaluate the robustness of the model due to uncertainty in the input data. In base case scenario (i.e. public tariff), incremental cost was $10.32, and incremental QALY and incremental LYG were 0.0001 and 0.0002 per patients respectively. Base case ICERs were 60,376 USD/QALY and 71,077 USD/LYG per patient. The results of the sensitivity analysis showed the robustness of the model. As the estimated ICER per QALY is more than three times the reported Gross Domestic Product (GDP) per capita by world bank for Iran in 2017 ($5415), the use of Enoxaparin for VTE prophylaxis in medical in patients doesn't seem to be a cost-effective intervention compared to the use of Heparin in Iran.

Utilidad de un policomprimido cardiovascular en el tratamiento de pacientes en prevención secundaria en España: un estudio de coste-efectividad

ObjectiveStratified medicine refers to the use of tests that predict treatment response to drive treatment decisions for individual patient. The pharmacoeconomic implications of this approach in schizophrenia are unknown. We aimed to assess the cost-effectiveness of a hypothetical stratified medicine algorithm (SMA) compared with treatment as usual (TAU), for patients with schizophrenia who failed a first-line antipsychotic.MethodsA decision analytic model with embedded Markov process was constructed, which simulated the health and cost outcomes for patients followed SMA or TAU over a lifetime horizon, from healthcare and social care perspective. In the base-case analysis, sensitivity and specificity of the stratifier were both set as 60%. Extensive sensitivity analyses were conducted to test the impact of uncertainty around the value of important parameters. The primary outcome was the incremental cost per quality-adjusted life year (QALY) gained.ResultsWhen both sensitivity and specificity of the stratified test were set at 60%, SMA appeared to be the optimal strategy as it produces more QALYs and incurs lower costs than TAU. This is robust to all scenarios tested. At a willingness-to-pay threshold of £20,000 per QALY, the probability for SMA to be the optimal strategy is 82.4%.ConclusionsOur results suggest that use of any stratifier with a sensitivity and specificity over 60% is very likely to be cost-effective comparing to TAU, for psychotic patients who failed a first-line antipsychotic. This finding, however, should be interpreted with caution due to lack of evidence for clozapine as a second-line antipsychotic.

BackgroundOlanzapine has been shown to have an additive effect on the three-drug antiemetic therapy consisting of aprepitant, palonosetron, and dexamethasone, in a highly emetogenic cisplatin-containing chemotherapy. Although olanzapine may be more economical than aprepitant or palonosetron, an adequate cost-efficacy analysis has not been conducted.MethodsWe conducted a cost-utility analysis to evaluate the cost-effectiveness of olanzapine use in four-drug antiemetic therapy among Japanese patients. We simulated model patients treated with highly emetogenic cisplatin-containing chemotherapy and developed a decision-analytical model of patients receiving triple antiemetic therapy with or without olanzapine in an inpatient setting. The cost and probabilities of each treatment were calculated from the perspective of the Japanese healthcare payer. The probabilities, utility value, and other costs were obtained from published sources. One-way and probabilistic sensitivity analyses were conducted to examine the influence of each parameter on the model and the robustness of a base-case analysis. Threshold analysis was conducted to determine the cost of olanzapine that would make the incremental cost-effectiveness ratio (ICER) equivalent to the threshold ICER). The threshold incremental cost-effectiveness ratio was set at 5 million Japanese Yen (JPY) per quality-adjusted life-year (QALY) gained.ResultsThe cost was 10,238 JPY in the olanzapine regimen and 9719 JPY in the non-olanzapine regimen. The QALY gained were 0.01065 QALYs and 0.01029 QALYs in the olanzapine and non-olanzapine regimen, respectively. The incremental cost of the olanzapine regimen relative to the non-olanzapine regimen was 519 JPY, and the incremental QALYs were 0.00036 QALY, resulting in an ICER of 1,428,675 JPY per QALY gained. In the one-way sensitivity analysis, the results were most sensitive to the utility value of incomplete control. The probabilistic sensitivity analysis revealed the probability that the ICER was below the willingness-to-pay, and the incremental QALYs was positive was 96.2%. The calculated cost of olanzapine per 5 mg that would make the incremental cost-effectiveness ratio equivalent to the threshold incremental cost-effectiveness ratio was calculated to be 475 JPY.ConclusionsOlanzapine was cost-effective in the four-drug antiemetic therapy for Japanese patients treated with highly emetogenic cisplatin-containing chemotherapy.

This study examines the generalizability of foreign economic evaluations to the Spanish healthcare system. The research aims to describe the cross-country adaptation methods identified in a scoping review of multinational cost-utility analyses and to examine the probability of concordant funding decisions between Spanish and foreign results, as well as to identify factors influencing generalizability. First, a scoping review of multinational studies reporting cost-utility analyses for at least two countries, including Spain, was conducted using MEDLINE, PubMed, Embase and Web of Science in April 2025. Data related to transferability were extracted and a narrative synthesis was performed. Second, a dataset of case comparisons-each defined as a technology against a comparator in a specific population-was developed from the identified studies. Each foreign comparison was matched to its Spanish equivalent within the same study. Incremental cost-effectiveness ratios (ICERs) were converted to 2024 Spanish Euros and compared against a threshold of €30,000 per quality-adjusted life year (QALY). A multilevel logit model was used, with a binary variable indicating decision concordance between Spanish and foreign ICERs/dominance as the dependent variable. We also analysed the distances in the incremental costs and incremental QALYs between countries using a log-normal bivariate model. Country-specific and other study-related factors were considered as independent variables in both models. The review included 57 studies. Most were funded by drug manufacturers and conducted in Europe. The majority of authors did not specify their reasons for selecting countries. All but three studies attempted to use local costs, probabilities and/or epidemiological data. Twelve studies incorporated country-specific utilities. A total of 644 comparisons were analysed; 142 were Spanish results and 502 were foreign results with their Spanish equivalents. The cost-effectiveness plane quadrant of the foreign result matched the Spanish result in 84% of cases. Assuming a threshold of €30,000 per QALY, the funding decisions were the same in 93% of cases. The probability of decision concordance was higher when the study was conducted in a Eurozone country or in the United Kingdom. Sensitivity analysis showed the variability of decisions depending on the selected cost-effectiveness threshold. Similar variables were found as relevant factors explaining the distance in the incremental QALYs analysis. Foreign cost-effectiveness results of those studies analysing drugs from Eurozone countries such as France, Germany, Italy, or from the United Kingdom can often be generalizable and provide meaningful insights for decision making in Spain. However, these findings should not be used as a reason to avoid country-specific studies if they are feasible. Further research is needed to determine if these findings apply to other health technologies. Limitations of the study include the lack of a formal assessment of the methodological quality of the selected studies and the potential risks of bias.

BackgroundVancomycin-resistant enterococci (VRE) are a serious antimicrobial resistant threat in the healthcare setting. We assessed the cost-effectiveness of VRE screening and isolation for patients at high-risk for colonisation on a general medicine ward compared to no VRE screening and isolation from the healthcare payer perspective.MethodsWe developed a microsimulation model using local data and VRE literature, to simulate a 20-bed general medicine ward at a tertiary-care hospital with up to 1000 admissions, approximating 1 year. Primary outcomes were accrued over the patient’s lifetime, discounted at 1.5%, and included expected health outcomes (VRE colonisations, VRE infections, VRE-related bacteremia, and deaths subsequent to VRE infection), quality-adjusted life years (QALYs), healthcare costs, and incremental cost-effectiveness ratio (ICER). Probabilistic sensitivity analysis (PSA) and scenario analyses were conducted to assess parameter uncertainty.ResultsIn our base-case analysis, VRE screening and isolation prevented six healthcare-associated VRE colonisations per 1000 admissions (6/1000), 0.6/1000 VRE-related infections, 0.2/1000 VRE-related bacteremia, and 0.1/1000 deaths subsequent to VRE infection. VRE screening and isolation accrued 0.0142 incremental QALYs at an incremental cost of $112, affording an ICER of $7850 per QALY. VRE screening and isolation practice was more likely to be cost-effective (> 50%) at a cost-effectiveness threshold of $50,000/QALY. Stochasticity (randomness) had a significant impact on the cost-effectiveness.ConclusionVRE screening and isolation can be cost-effective in majority of model simulations at commonly used cost-effectiveness thresholds, and is likely economically attractive in general medicine settings. Our findings strengthen the understanding of VRE prevention strategies and are of importance to hospital program planners and infection prevention and control.

ObjectivesThis study aimed to evaluate the performance of EQ-5D data mapped from SF-12 in terms of estimating cost effectiveness in cost-utility analysis (CUA). The comparability of SF-6D (derived from SF-12) was also assessed.MethodsIncremental quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) were calculated based on two Markov models assessing the cost effectiveness of haemodialysis (HD) and peritoneal dialysis (PD) using utility values based on EQ-5D-5L, EQ-5D using three direct-mapping algorithms and two response-mapping algorithms (mEQ-5D), and SF-6D. Bootstrap method was used to estimate the 95% confidence interval (percentile method) of incremental QALYs and ICERs with 1000 replications for the utilities.ResultsIn both models, compared to the observed EQ-5D values, mEQ-5D values expressed much lower incremental QALYs (range − 14.9 to − 33.2%) and much higher ICERs (range 17.5 to 49.7%). SF-6D also estimated lower incremental QALYs (− 29.0 and − 14.9%) and higher ICERs (40.9 and 17.5%) than did the observed EQ-5D. The 95% confidence interval of incremental QALYs and ICERs confirmed the lower incremental QALYs and higher ICERs estimated using mEQ-5D and SF-6D.ConclusionCompared to observed EQ-5D, EQ-5D mapped from SF-12 and SF-6D would under-estimate the QALYs gained in cost-utility analysis and thus lead to higher ICERs. It would be more sensible to conduct CUA studies using directly collected EQ-5D data and to designate one single preference-based measure as reference case in a jurisdiction to achieve consistency in healthcare decision-making.