Quality-adjusted Life Research Articles

Cost-effectiveness analysis of mHealth applications for depression in Germany using a Markov cohort simulation

AbstractRegulated mobile health applications are called digital health applications (“DiGA”) in Germany. To qualify for reimbursement by statutory health insurance companies, DiGA have to prove positive care effects in scientific studies. Since the empirical exploration of DiGA cost-effectiveness remains largely uncharted, this study pioneers the methodology of cohort-based state-transition Markov models to evaluate DiGA for depression. As health states, we define mild, moderate, severe depression, remission and death. Comparing a future scenario where 50% of patients receive supplementary DiGA access with the current standard of care reveals a gain of 0.02 quality-adjusted life years (QALYs) per patient, which comes at additional direct costs of ~1536 EUR per patient over a five-year timeframe. Influencing factors determining DiGA cost-effectiveness are the DiGA cost structure and individual DiGA effectiveness. Under Germany’s existing cost structure, DiGA for depression are yet to demonstrate the ability to generate overall savings in healthcare expenditures.

Projected Cost Savings With Optimal Medication Adherence in Patients With Cardiovascular Disease Requiring Lipid-Lowering Therapy: A Multinational Economic Evaluation Study.

Poor adherence to chronic cardiovascular treatments can impede targeted clinical outcomes. This study estimates the potential benefits of improving adherence among patients with cardiovascular disease requiring secondary prevention in Mexico, Thailand, and China. We performed Markov model simulation for patients with cardiovascular disease in 3 countries from health care and societal perspectives over a lifetime horizon. Two scenarios were compared: (1) optimal adherence based on a meta-analysis of 51 randomized controlled trials and (2) status quo. The association between adherence and cardiovascular disease outcomes derives from a dose-response meta-analysis of 4 051 338 patients. Outcomes include the accumulated number of cardiovascular events and associated costs in 2022 US dollars, life years, and quality-adjusted life years. Optimal adherence could prevent 42 (95% credible interval [CrI], 29-56) cardiovascular events in Mexico, 34 (95% CrI, 24-50) in Thailand, and 63 (95% CrI, 43-89) in China per 1000 patients over a lifetime. Incremental effectiveness per patient was 0.60 (95% CrI, 0.47-0.74) life-years in Mexico, 0.68 (95% CrI, 0.37-0.94) quality-adjusted life years in Thailand, and 0.93 (95% CrI, 0.44-1.27) quality-adjusted life years in China. Cost savings from societal perspective amounted to $412 (95% CrI, $211-$723), $316 (95% CrI, $187-$541), and $700 (95% CrI, $355-$1144) per patient for Mexico, Thailand, and China, respectively. Findings remained cost saving in deterministic and probabilistic sensitivity analyses. Achieving optimal adherence in patients with cardiovascular disease requiring lipid-lowering therapy saves costs and improves health outcomes in Mexico, Thailand, and China. These findings support national health care systems implementing strategies to improve adherence in these countries.

Cost-Effectiveness of Computer-Assisted Cognitive Behavioral Therapy for Depression Among Adults in Primary Care.

Approximately 1 in 5 adults are diagnosed with depression in their lifetime. However, less than half receive help from a health professional, with the treatment gap being worse for individuals with socioeconomic disadvantage. Computer-assisted cognitive behavioral therapy (CCBT) is an effective and convenient strategy to treat depression; however, its cost-effectiveness in a sociodemographically diverse population remains unknown. To evaluate the cost-effectiveness of clinician-supported CCBT compared with treatment as usual (TAU) in a primary care population with a substantial number of patients with low income, limited computer or internet access, and lack of college education. This economic evaluation was a randomized clinical trial-based cost-effectiveness analysis. The trial was conducted at the Departments of Family and Geriatric Medicine and Internal Medicine at the University of Louisville. Enrollment occurred from June 24, 2016, to May 13, 2019. Participants had mild to moderate depression and were followed up for 6 months after treatment completion. The last follow-up assessment was conducted on January 30, 2020. Statistical analysis was performed from August 2023 to August 2024. CCBT intervention was provided for 12 weeks and included 9 modules ranging from behavioral activation and cognitive restructuring to relapse prevention strategies, supported by telephonic sessions with a clinician, in addition to TAU, which included standard clinical management in primary care. The primary health outcome was quality-adjusted life years (QALYs), estimated using the Short-Form 12 questionnaire (SF-12). The secondary outcome was treatment response, defined as at least 50% improvement in the Patient Health Questionnaire. The intervention cost included sessions with mental health clinicians and the cost of the CCBT software, plus the cost of loaner computer and internet data plan for low-resource households. An incremental cost-effectiveness ratio (ICER) was computed, while adjusting for baseline scores, age, and sex. The cost-effectiveness acceptability curve presented the probability of CCBT being cost-effective for a range of willingness-to-pay values. Among the 175 primary care patients included in this study, 148 (84.5%) were female; 48 (27.4%) were African American, 2 (1.2%) were American Indian or Alaska Native, 4 (2.5%) were Hispanic, 106 (60.5%) were White, and 15 (8.6%) were multiracial; and the mean (SD) age was 47.03 (13.15) years. CCBT was associated with better quality of life and higher chance of treatment response at the posttreatment and 6-month time points, compared with the TAU group. The ICER for CCBT was $37 295 (95% CI, $22 724-$66 546) per QALY, with a probability of 89.4% of being cost-effective at a willingness-to-pay threshold of $50 000/QALY. The ICER per case of treatment response was $3623 (95% CI, $2617-$5377). In this trial-based economic evaluation, CCBT was found to be cost-effective, compared with TAU, in primary care patients with depression. As this study included individuals with low income and with limited internet access who are underrepresented in cost-effectiveness studies, it has important policy implications for addressing unmet needs in sociodemographically diverse populations.

Comparison of clinical outcomes and cost-utility between unilateral biportal endoscopic discectomy and percutaneous endoscopic interlaminar discectomy for single-level lumbar disc herniation: a retrospective matched controlled study.

This study aimed to compare the efficacy and cost-utility of unilateral biportal endoscopy (UBE) versus percutaneous endoscopic interlaminar discectomy (PEID) for the treatment of single-level lumbar disc herniation (LDH). A retrospective analysis was conducted on 99 patients who underwent either UBE (n = 33) or PEID (n = 66) between July 2022 and December 2023 at the Second Xiangya Hospital. Patients were matched 1:2 based on age, sex, and surgery level to ensure comparability. Clinical outcomes were assessed using Visual Analog Scale (VAS), European Quality of Life-5 Dimensions (EQ-5D), and Oswestry Disability Index (ODI) scores, with quality-adjusted life years (QALYs) calculated for cost-utility analysis. Hospitalization costs were analyzed, and the incremental cost-utility ratio (ICER) was determined. Both UBE and PEID groups demonstrated significant postoperative improvements in VAS, EQ-5D, and ODI scores (p < 0.05). The operative time, blood loss and nursing cost were significantly higher for UBE compared to PEID (p < 0.05). UBE has higher gained QALY and overall costs, but the differences are not statistically significant (p = 0.643 for QALY, p = 0.327 for costs). The Incremental Cost-Effectiveness Ratio (ICER) for UBE compared to PEID was calculated to be $354.5 per QALY gained, indicating that for each additional QALY gained through UBE, an additional cost of $354.5 is incurred compared to PEID. In our single-center study conducted in China, both the UBE and PEID procedures have demonstrated comparable short-term efficacy in alleviating pain and improving functional ability in patients with single-level LDH. UBE procedure demonstrates greater cost-utility than the PEID procedure in cost-utility analysis, despite its longer operative time, higher nursing costs and greater blood loss.

Clinical- and Cost-Effectiveness of Liver Disease Staging in Hepatitis C Virus Infection: A Microsimulation Study.

Liver disease assessment is a key aspect of chronic hepatitis C virus (HCV) infection pre-treatment evaluation but guidelines differ on the optimal testing modality given trade-offs in availability and accuracy. We compared clinical outcomes and cost-effectiveness of common fibrosis staging strategies. We simulated adults with chronic HCV receiving care at US health centers through a lifetime microsimulation across five strategies: (1) no staging or treatment (comparator), (2) indirect serum biomarker testing (Fibrosis-4 index [FIB-4]) only, (3) transient elastography (TE) only, (4) staged approach: FIB-4 for all, TE only for intermediate FIB-4 scores (1.45-3.25), and (5) both tests for all. Outcomes included infections cured, cirrhosis cases, liver-related deaths, costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs). We used literature-informed loss to follow-up (LTFU) rates and 2021 Medicaid perspective and costs. FIB-4 alone generated the best clinical outcomes: 87.7% cured, 8.7% developed cirrhosis, and 4.6% had liver-related deaths. TE strategies cured 58.5%-76.6%, 16.8%-29.4% developed cirrhosis, and 11.6%-22.6% had liver-related deaths. All TE strategies yielded worse clinical outcomes at higher costs per QALY than FIB-4 only, which had an ICER of $12 869 per QALY gained compared with no staging or treatment. LTFU drove these findings: TE strategies were only cost-effective with no LTFU. In a point-of-care HCV test-and-treat scenario, treatment without any staging was most clinically and cost-effective. FIB-4 staging alone resulted in optimal clinical outcomes and was cost-effective. Treatment for chronic HCV should not be delayed while awaiting fibrosis staging with TE.

Cost-effectiveness analysis of trifluridine/tipiracil combined with bevacizumab vs. monotherapy for third-line treatment of colorectal cancer

BackgroundThe combination of trifluridine/tipiracil (FTD/TPI) and bevacizumab has demonstrated promising efficacy and safety in the treatment of colorectal cancer (CRC). This study aims to evaluate the cost-effectiveness of trifluridine/tipiracil combined with bevacizumab vs. trifluridine/tipiracil monotherapy as a third-line treatment regimen for colorectal cancer within the Chinese healthcare system, providing an economic basis for clinical application.MethodsBased on data from the SUNLIGHT Phase III clinical trial, a dynamic Markov model was constructed with a cycle length of 4 weeks and a simulation duration of 10 years. Direct medical costs and quality-adjusted life years (QALYs) were calculated. The incremental cost-effectiveness ratio (ICER) was compared with the willingness-to-pay threshold (WTP = ¥268,200.00/QALY) to assess the economic viability of the treatment regimen. One-way sensitivity analysis and probabilistic sensitivity analysis were conducted to verify the robustness of the model results.ResultsThe cost of trifluridine/tipiracil combined with bevacizumab treatment (¥838,492.74) was higher than that of trifluridine/tipiracil monotherapy (¥357,396.97), with greater health benefits (2.45 QALYs vs. 1.54 QALYs). The ICER was ¥527,577.36/QALY, exceeding the willingness-to-pay threshold. One-way sensitivity analysis indicated that drug costs and utility values during the progression-free period significantly impacted model outputs. Probabilistic sensitivity analysis further confirmed the robustness of the results, showing that at a willingness-to-pay threshold of ¥494,000.00, the probability of the combined treatment being cost-effective was 50%.ConclusionTrifluridine/tipiracil combined with bevacizumab, as a third-line treatment for colorectal cancer, does not have a cost-effectiveness advantage compared to trifluridine/tipiracil monotherapy in economic evaluations.

Cost-effectiveness of Implementing a Genotype-Guided De-Escalation Strategy in Patients with Acute Coronary Syndrome.

A genotype-guided P2Y12-inhibitor de-escalation strategy, switching acute coronary syndrome (ACS) patients without a CYP2C19 loss-of-function allele from ticagrelor or prasugrel to clopidogrel, has shown to reduce bleeding risk without affecting effectivity of therapy by increasing ischemic risk. We estimated the cost-effectiveness of this personalized approach compared to standard dual antiplatelet therapy (DAPT; aspirin plus ticagrelor/prasugrel) in the Netherlands. We developed a one-year decision tree based on results of the FORCE-ACS registry, comparing a cohort of ACS patients who underwent genotyping with a cohort of ACS patients treated with standard DAPT. This was followed by a lifelong Markov model to compare lifetime costs, and quality-adjusted life years (QALYs) for a fictional cohort of 1000 patients. The cost-effectiveness analysis was performed from the perspective of the Dutch healthcare system. A genotype-guided de-escalation strategy led to anincrease of 55.30 QALYs and saved €698,286 compared to standard DAPT based on a lifetime horizon. Probabilistic sensitivity analysis showed that the genotype-guided strategy was cost-saving in 93% and increased QALYs in 86% of simulations. The intervention remained cost-effective in the scenario where prices for all P2Y12-inhibitors were equalized. The genotype-guided strategy remained dominant in various other scenario and sensitivity analyses. A genotype-guided de-escalation strategy in patients with ACS was both cost-saving and yielded higher QALYs compared to standard DAPT, highlighting its potential for implementation in clinical practice. Trial registration: ClinicalTrials.gov identifier: NCT03823547.

Exploratory Cost-Utility Analysis of a 37-Gene Panel Versus Usual Care to Guide Therapy for Patients with Intermediate-Risk Myeloid Malignancies.

Genomic risk stratification methods for myeloid malignancies have moved beyond conventional karyotyping and single gene approaches to better define disease behaviour. Next-generation sequencing has been established as the new standard-of-care tool to accurately define prognosis at diagnosis and guide therapy decisions. We aimed to determine the economic value of a 37-gene panel test for informing subsequent care for patients with intermediate-risk myeloid malignancies. We performed an exploratory cost-utility analysis of a 37-gene panel test to inform stem cell transplantation therapy in patients with myeloid malignancies in Queensland, Australia. Clinician surveys provided data on management choice with and without genomics information while both published and individual-level data were used for healthcare costs, quality of life, relapse rates and survival data. We used a decision-analytic cohort model with Markov chains and 5000 simulations to derive the incremental cost per quality-adjusted life year (QALY) gained. Scenario, one-way and probabilistic sensitivity analyses were undertaken to test input variation on the stability of the main findings. Over 10 years, the model predicted mean costs of AU$125,561 for the panel testing strategy and AU$117,045 for usual care, indicating an incremental cost of AU$8516 for panel testing. The corresponding mean QALYs were 4.52 for panel testing and 4.46 for usual care, producing a cost of AU$153,854 per QALY gained. In the Australian system, the likelihood that panel testing would be cost effective was <1 % and would have a more favourable cost-effective profile at a willingness-to-pay of AU$140,000 per QALY gained. Driven by small gains in survival and relapse rates following therapies, genomic panel sequencing for myeloid malignancies in people with intermediate-risk disease is unlikely to be cost effective in Australia.

Abstract 4119874: Cost Effectiveness Analysis of Surgical Strategies Versus Medical Management for Rheumatic Heart Disease in Rwanda

Background: One third of the Rheumatic Heart Disease (RHD) burden is in sub-Saharan Africa. Without valve surgery, 17% of patients die. However, surgery is often deemed too expensive in this region despite the lack of cost-effectiveness analyses to support this assumption. Aim: Assess the cost-effectiveness of surgical strategies for RHD performed in Rwanda. Methods: We built a Markov model to simulate the progression of patients through different health states. We compared mechanical valve replacement, bioprosthetic valve replacement, and valve repair to medical management for patients with severe RHD. Probabilities of transitioning into different health states were derived from regional published studies. Micro-costing data was obtained from the cardiac surgery program to calculate total costs for each health state. Health effects were measured in quality-adjusted life years (QALYs) from the 2019 Global Burden of Disease Study. Sensitivity analysis was performed to account for model uncertainty. We used a willingness-to-pay threshold of three times the GDP per capita of Rwanda (USD$769). Results: All surgical strategies extended life expectancy compared to medical management, which only offered 5.05 QALYs. Mechanical valve replacement surgery was cost-effective with 11.31 QALYs for a lifetime cost of USD$10,721. The incremental cost effectiveness ratio compared to medical management was USD$1,596/QALY, which is below the willingness to pay threshold. Decreasing the initial cost of surgery and probability of long term stroke increased cost-effectiveness. Although bioprosthetic valve replacement and repair provided 6.43 and 5.70 additional QALYs compared to medical management, they had higher lifetime costs than mechanical valve replacement ($19,789.21, $22,477.27). As a result, they were not cost-effective. In the sensitivity analysis, they became cost effective if valve degeneration rates were 11% in 2 years from a baseline of 26% for repair, and 8% in 2 years from baseline of 14% for bioprosthetic valves. Conclusion: This study challenges the prevailing assumption that surgery is not cost-effective in Sub-Saharan Africa. Mechanical valve replacement surgery was most cost-effective. Watchful waiting with medical management is inexpensive but carries an average life expectancy of 5 years. Identifying cost-saving opportunities for mechanical valve replacement surgery and enhancing anticoagulation monitoring can make it even more economically favorable.

Unmet Need in Non-muscle-invasive Bladder Cancer Failing Bacillus Calmette-Guérin Therapy: A Systematic Review and Cost-effectiveness Analyses from the International Bladder Cancer Group.

Non-muscle-invasive bladder cancer (NMIBC) poses a significant clinical challenge, particularly when failing bacillus Calmette-Guérin (BCG) therapy, necessitating alternative treatments. Despite radical cystectomy being the recommended treatment, many patients are unfit or unwilling to undergo this invasive procedure, highlighting the need for effective bladder-sparing therapies. This review aims to summarize and report the evidence on the efficacy and to estimate the costs of bladder-preserving strategies used in NMIBC recurrence after failure of intravesical BCG therapy. We systematically searched online databases for prospective studies investigating intravesical therapy, systemic therapy, or combination of both in patients treated previously with BCG. Owing to significant heterogeneity across the studies, a meta-analysis was inappropriate. A sensitivity analysis was performed in an exploratory manner. We used a decision-analytic Markov model to compare novel U.S. Food and Drug Administration-approved treatments with a 2-yr time horizon. A total of 57 studies published between 1998 and 2024, with 68 unique study arms and consisting of 2589 patients, were identified. The 3-mo overall response rate (ORR) across all studies, complete response rate (CRR) in concomitant carcinoma in situ (CIS) or CIS only disease, and recurrence-free rate (RFR) in papillary disease were estimated to be 52.4% (95% confidence interval [CI]: 45.4-59.2), 52.8% (95% CI: 42.9-62.6), and 26.4% (95% CI: 13.3-45.6), respectively. The 12-mo ORR, CRR, and RFR were estimated to be 78% (95% CI: 52.9-91.8), 27.8% (95% CI: 21.3-35.4), and 25.4% (95% CI: 18.2-34.2), respectively. The progression rate was estimated to be 13% (95% CI: 9-18.2). The mean proportion of patients treated with radical cystectomy was estimated to be 24.7 (range 0-85.7). The reported toxicity grades were overall mild, with a median of 3.4% (range 0-33.3%) participants experiencing a dose limiting toxicity. Compared with using radical cystectomy to treat patients failing BCG therapy, at a willingness-to-pay threshold of 100 000 USD, nadofaragene firadenovec was cost effective, with an incremental cost-effectiveness ratio (ICER) of 10 014 USD per quality-adjusted life year (QALY), while nogapendekin alfa inbakicept was less cost effective than nadofaragene firadenovec (ICER of 44 602 USD per QALY). Pembrolizumab, which dominated, was both less costly and more effective than the other strategies. Salvage bladder-sparing therapies show a response rate of around 50% at 3 mo in patients with NMIBC failing BCG. However, long-term data are heterogeneous. Nevertheless, recently developed agents show promising tumor control activity. In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life. The findings of our review highlight the need for novel, more effective therapeutic strategies. In this study, we reviewed the evidence on the efficacy of bladder-preserving strategies used in patients with bladder cancer recurrence after failing bacillus Calmette-Guérin (BCG) therapy. We found that these strategies show a response rate of around 50% at 3 mo. However, long-term data are heterogeneous. Nevertheless, recently developed agents show promising tumor control activity. In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life.

Cost-Effectiveness of Trastuzumab Deruxtecan in Patients with Unresectable or Metastatic HER2-Low Breast Cancer Who Have Received Prior Chemotherapy.

In 2021, breast cancer affected 75,619 women in Denmark. Approximately 50% of breast cancers are considered human epidermal growth factor receptor2 (HER2)-low. The DESTINY-Breast04 (DB-04) trial led to European Medicines Agency (EMA) approval of trastuzumab deruxtecan (T-DXd) as a treatment for patients with unresectable or metastatic HER2-low breast cancer who have received prior chemotherapy in the metastatic setting or developed disease recurrence during or within 6months of completing adjuvant chemotherapy. Moreover, the Danish Breast Cancer Group guidelines recently included T-DXd as a treatment for HER2-low metastatic breast cancer. This economic evaluation aims to estimate the cost-effectiveness of T-DXd for the approved EMA indication in Denmark. A three-state-progression-free, post-progression, and death-partitioned survival model was developed to estimate the cost-effectiveness of T-DXd versus treatment of physician's choice over a lifetime horizon following the Danish Medicines Council guidelines. Clinical data were gathered from the DB-04 trial, and cost and resource use data were sourced from the literature. Sensitivity and scenario analysis were conducted to explore uncertainty. T-DXd led to 0.78 incremental quality-adjusted life years (QALYs) gained and incurred DKK621,325 in incremental costs compared to the treatment of physician's choice. This resulted in an incremental cost-effectiveness ratio of DKK795,181 per QALY gained, which falls below the willingness-to-pay threshold. Sensitivity and scenario analyses showed the robustness of the deterministic result, with T-DXd remaining cost-effective. Our study demonstrates that T-DXd is a cost-effective treatment for patients with HER2-low unresectable or metastatic breast cancer who have received prior chemotherapy in Denmark.

Cost-effectiveness of midomafetamine-assisted therapy (MDMA-AT) in chronic and treatment-resistant post-traumatic stress disorder of moderate or higher severity: A health-economic model.

To explore the cost-effectiveness of midomafetamine-assisted therapy (MDMA-AT) compared to placebo with therapy (PT) in US healthcare settings. A health state-transition model was used to analyze the cost-effectiveness of MDMA-AT for treating patients with chronic PTSD of moderate or higher severity. Both treatment arms consisted of 3 preparation (90-min), 3 interventional (8-h), and 9 integration (90-min) sessions, lasting ~4 months total. All sessions included psychotherapy, with interventional also including MDMA or placebo. After receiving treatment, patients were distributed across health states of No PTSD (not meeting PTSD diagnostic criteria), Non-Severe PTSD (treatment responders), Severe PTSD (treatment non-responders), and death. Each state had unique healthcare costs and utilities sourced from real-world data analysis and patient data from MDMA-AT clinical trials (including long-term follow-up). The base-case analysis considered the payer's perspective with a 5-year horizon, 3.5% annual cost and effect discounts, and an assumed MDMA medication price of $12,000 per session. Trial-derived utilities and US life tables mortality data were used to calculate quality-adjusted life years (QALY). The main outcome was an incremental cost-effectiveness ratio (ICER) with a $150,000 willingness-to-pay (WTP) threshold. The base-case ICER was $83,845 per QALY. Total direct costs were $64,745 in the MDMA-AT and $33,132 in the PT arms ($31,613 increment). The costs of intervention were $48,376 for MDMA-AT and $12,376 for PT. The highest MDMA medication cost to fit under the WTP threshold was $20,314 per session. Costs related to PTSD healthcare visits and other PTSD treatments were lower with MDMA-AT than PT (-$2,511 and -$1,877 increments, respectively). Utility benefits were higher in MDMA-AT than PT, with 3.691 and 3.314 QALYs generated over 5 years, respectively (0.377 QALY increment). These data suggest MDMA-AT may be a cost-effective treatment compared to PT for patients with chronic PTSD of moderate or higher severity.

Treatment of adult spine deformity: A retrospective comparison of bone morphogenic protein and bone marrow aspirate with bone allograft.

This was a retrospective cohort study. ASD patients were stratified by use of intra-operative BMP (BMP +) or not (BMA + I) and surveyed for the development of complications and mechanical failure. Quality of life gained following the procedure was evaluated using quality-adjusted life years (QALYs). Cost was calculated using the PearlDiver database and CMS definitions. Multivariable analyses (ANCOVA) and logistic regression were used to adjust for confounding. 512 patients were included (60% BMP +). At baseline, BMP + patients were older (62.5 vs 60.8years, p < 0.010). Radiographic and quality-of-life metrics did not differ at follow up timepoints (all p > 0.05). BMP use was associated with higher supplemental rod use (OR: 7.0, 1.9 - 26.2, p = 0.004), greater number of levels fused (OR: 1.1, 1.03 - 1.17, p = 0.003) and greater neurological complications (OR: 5.0, 1.3 - 18.7, p = 0.017). Controlling for rod use and levels fused, BMP use was not associated with a lower risk of mechanical complications (OR 0.3, 95% CI: 0.2 - 3.0, p = 0.353), rod breakage (OR: 3.3, 0.6 - 18.7, p = 0.182) or implant failure (OR: 0.3, 0.04 - 1.51). At 2years, the BMP + cohort exhibited higher overall costs ($108,062 vs $95,144, p = 0.002), comparable QALYs (0.163 vs 0.171, p = 0.65) and higher cost per QALY (p = 0.001) at two years. In this analysis, BMP-2 application was not associated with superior outcomes when compared to a less costly biologic alternative (bone marrow aspirate + cancellous bone chips + i-Factor) following ASD surgery. The use of BMP-2 in ASD surgery appears to have reduced cost-efficacy at two years postoperatively.

QOL-06. OPTIMIZING NEUROFIBROMATOSIS CARE: A COMPREHENSIVE ECONOMIC EVALUATION OF A SPECIALIZED EXPERTISE CENTER’S IMPACT ON PATIENT OUTCOMES

Abstract Neurofibromatosis type 1 (NF1), NF2-related schwannomatosis (NF2) and other schwannomatoses represent a spectrum of genetic disorders characterized by the development of tumors on nerve tissues, profoundly impacting patients’ quality of life and posing complex challenges. Recognizing the imperative for comprehensive care delivery, we undertook to implement a Neurofibromatosis Expertise Center (CE) at the Centre hospitalier de l’Université de Montréal (CHUM), Quebec, Canada. Employing a robust analysis framework, we aimed to assess the economic viability of the CE by quantifying the Quality-Adjusted Life Years (QALYs) gained through centralized multidisciplinary neurofibromatosis care (cost-utility ratio = CAD/QALY gained). Leveraging data from validated questionnaires including the SF-6D, our preliminary analysis revealed significant enhancements in patients’ quality of life across diverse domains, including physical pain, visible cutaneous lesions, cognitive dysfunction, hearing loss, malignancies, psychiatric comorbidities, unemployment, physical disability, and social isolation. Notably, among the respondents (N = 115), patients receiving CE services reported a 12% increase in general health compared to a 25% decline among those without CE services. Moreover, 28% of CE patients reported improved overall health compared to 12% of those without CE services. Using the Work Productivity and Activity Impairment questionnaire (WPAI-GH), we found that cohorts followed in CE for ≥ 1 year (N = 45) experienced less work productivity losses compared to the most recent cohort (N = 32), which presented scores exceeding 6 on a 1-to-10 scale. We hypothesize that the establishment of the CE will yield profound improvements in patient health outcomes and alleviate financial strains on the Ministère de la Santé et des Services Sociaux du Québec (MSSS). Therefore, our next steps include discussing a strategy for funding application to the MSSS to ensure the accessibility of necessary care for patients. Continuing data collection and analysis will also provide a more comprehensive overview of patient characteristics and needs.

Cost-effectiveness analysis of transcatheter aortic valve implantation versus surgical aortic valve replacement in patients with severe aortic stenosis at low risk of surgical mortality in Sweden

Background: Transcatheter aortic valve implantation (TAVI) has shown similar or improved clinical outcomes compared with surgical aortic valve replacement (SAVR) in patients with symptomatic severe aortic stenosis at low risk for surgical mortality. This cost-utility analysis compared TAVI with SAPIEN 3 versus SAVR in symptomatic severe aortic stenosis patients at low risk of surgical mortality from the perspective of the Swedish healthcare system. Methods: A published, two-stage, Markov-based cost-utility model that captured clinical outcomes from the Swedish Web-system for Enhancement and Development of Evidence-based care in Heart disease Evaluated according to Recommended Therapies (SWEDEHEART) registry (2018–2020) was adapted from the perspective of the Swedish healthcare system using local general population mortality, utility and costs data. The model had a lifetime horizon. Model outputs included changes in direct healthcare costs and health-related quality of life from using TAVI as compared with SAVR. Results: TAVI with SAPIEN 3 resulted in lifetime costs per patient of 940,541 Swedish krona (SEK) and lifetime quality-adjusted life years (QALYs) per patient of 7.16, whilst SAVR resulted in lifetime costs and QALYs per patient of 821,380 SEK and 6.81 QALYs, respectively. Compared with SAVR, TAVI offered an incremental improvement of +0.35 QALY per patient at an increased cost of +119,161 SEK per patient over a lifetime horizon, resulting in an incremental cost-effectiveness ratio of 343,918 SEK per QALY gained. Conclusion: TAVI with SAPIEN 3 is a cost-effective option versus SAVR for patients with symptomatic severe aortic stenosis at low risk for surgical mortality treated in the Swedish healthcare setting. These findings may inform policy decisions in Sweden for the management of this patient group.

Cost-effectiveness analysis of lorlatinib and crizotinib in the first-line treatment of anaplastic lymphoma kinase-positive non-small cell lung cancer

ABSTRACT Objectives To assess the economic value of lorlatinib and crizotinib in the first-line treatment of anaplastic lymphoma kinase-positive non-small cell lung cancer at medical insurance negotiation prices from the viewpoint of China’s health system. Methods Based on data from the phase III clinical trial, a three-state partitioned survival model was established. In combination with parameters such as treatment costs, utility values, incidence of adverse reactions, and discount rates, the total incremental cost-effectiveness ratio (ICER) was simulated. Results The results of the fundamental analysis indicated that the patients in the lorlatinib group expended 709,671 yuan more than did the patients in the crizotinib group did but gained 3.09 quality-adjusted life years (QALYs). The ICER value was 229,782.6 yuan/QALY, which was lower than three times the per capita gross domestic product (GDP) of China. Conclusions Compared with crizotinib, lorlatinib may be a cost-effective first-line treatment choice for advanced ALK-positive NSCLC in China.

Populationwide Screening for Chronic Kidney Disease

Sodium-glucose cotransporter-2 (SGLT2) inhibitors have changed clinical management of chronic kidney disease (CKD) and made populationwide screening for CKD a viable strategy. Optimal age of screening initiation has yet to be evaluated. To compare the clinical benefits, costs, and cost-effectiveness of population-wide CKD screening at different initiation ages and screening frequencies. This cost-effectiveness study used a previously published decision-analytic Markov cohort model that simulated progression of CKD among US adults from age 35 years and older and was calibrated to population-level data from the National Health and Nutrition Examination Survey (NHANES). Effectiveness of SGLT2 inhibitors was derived from the Dapagliflozin and Prevention of Adverse Outcomes in Chronic Kidney Disease (DAPA-CKD) trial. Mortality, quality-of-life weights, and cost estimates were obtained from published cohort studies, randomized clinical trials, and US Centers for Medicare & Medicaid Services data. Analyses were performed from June 2023 through September 2024. One-time or periodic (every 10 or 5 years) screening for albuminuria, initiated at ages between 35 and 75 years, with and without addition of SGLT2 inhibitors to conventional CKD therapy (angiotensin-converting enzyme inhibitors/angiotensin receptor blockers). Cumulative incidence of kidney failure requiring kidney replacement therapy (KRT); life years, quality-adjusted life years (QALYs), lifetime health care costs (2024 US currency), and incremental cost-effectiveness ratios discounted at 3% annually. For those aged 35 years, starting screening at age 55 years, and continuing every 5 years through age 75 years, combined with SGLT2 inhibitors, decreased the cumulative incidence of kidney failure requiring KRT from 2.4% to 1.9%, increased life expectancy by 0.13 years, and cost $128 400 per QALY gained. Although initiation of screening every 5 years at age 35 or 45 years yielded greater gains in population-wide health benefits, these strategies cost more than $200 000 per additional QALY gained. The comparative values of starting screening at different ages were sensitive to the cost and effectiveness of SGLT2 inhibitors; if SGLT2 inhibitor prices drop due to patent expirations, screening at age 55 years continued to be cost-effective even if SGLT2 inhibitor effectiveness were 30% lower than in the base case. This study found that, based on conventional benchmarks for cost-effectiveness in medicine, initiating population-wide CKD screening with SGLT2 inhibitors at age 55 years would be cost-effective.

Economic evaluations of technology-based interventions used to provide care support for people with mild dementia or mild cognitive impairment and their caregivers: A systematic review.

The cost-effectiveness of interventions is a key issue owing to the limited resources of healthcare services. To conduct a systematic review of economic evaluations of technology-based healthcare interventions in care support for people with dementia or mild cognitive impairment (MCI) and their caregivers, and of the tools used to assess effectiveness and costs. The following databases were used: PubMed, National Health Service Economic Evaluation Database, and Health Technology Assessment. A total of 207 articles from 2012 to 2024 were identified and then screened. Seventeen studies were included, of which nine were study protocols. Almost half (n = 8) the interventions were multicomponent. The most common components used in the interventions were cognitive stimulation, physical functioning and continuing support. Regarding the efficiency results of these interventions, only three studies provided a full economic evaluation. The most frequent tools in the economic evaluations used to measure effectiveness (measured in quality-adjusted life years) and costs were the European Quality of Life-5 Dimensions and Resource Utilization in Dementia instruments, respectively. Most of the interventions evaluated were cost-effective. However, these results should be interpreted with caution, given the scarcity of the literature, and further economic evaluations of technology-based healthcare interventions for people with mild dementia or MCI care support and their caregivers are therefore needed. Additionally, a meta-analysis could not be performed due to the heterogeneity of the data.

Cost-Effectiveness Analysis of the Use of V116, a 21-Valent Pneumococcal Conjugate Vaccine, in Vaccine-Naïve Adults Aged ≥ 65 Years in the United States.

Given the recent approval and recommendation of V116, a 21-valent pneumococcal conjugate vaccine (PCV), in the United States(US), we evaluated the cost-effectiveness of using V116 versus the 20-valent PCV(PCV20) or the 15-valent PCV(PCV15) in series with the 23-valent pneumococcal polysaccharide vaccine (PPSV23) among adults aged ≥ 65years in the US who had never received a PCV previously. A static multi-cohort state-transition Markov model was developed to estimate the lifetime incremental clinical and economic impact of V116 vs. PCV20 or PCV15 + PPSV23 from the societal perspective. All model inputs were based on published literature and publicly available databases and/or reports. Model outcomes included undiscounted clinical cases: invasive pneumococcal disease (IPD), inpatient and outpatient non-bacteremic pneumococcal pneumonia (NBPP), post-meningitis sequelae (PMS), deaths from IPD and inpatient NBPP, discounted quality-adjusted life years (QALYs) as well as the discounted total cost (in 2023 USD), which consisted of vaccine acquisition and administration costs, direct and indirect costs associated with the disease, and travel costs for vaccination. The final summary measure was the incremental cost-effectiveness ratio (ICER), reported as $/QALY gained. Three percent was used for the annual discounting rate. Based on the inputs and assumptions used, the results indicated that the V116 strategy prevented 27,766 and 32,387 disease cases/deaths and saved $239 million and $1.8 billion in total costs when compared to the PCV20andPCV15 + PPSV23 strategies, respectively, in vaccine-naïve adults aged ≥ 65years. The estimated ICERs were cost saving in both regimens (i.e., V116 vs. PCV20 or vs. PCV15 + PPSV23). The scenario analysis and deterministic and probabilistic sensitivity analyses also demonstrated the robustness of the qualitative results. These results demonstrated that using V116 in adults aged ≥ 65years in the US can prevent a substantial number of PD cases and deaths while remaining highly favorable economically over a wide range of inputs and scenarios.

Cost-effectiveness analysis of trifluridine/tipiracil in the treatment of heavily pretreated metastatic gastric cancer from the perspective of Chinese healthcare system

ObjectivesThe aim of this study was to evaluate the cost-effectiveness of trifluridine/tipiracil (FTD/TPI) for heavily pretreated metastatic gastric cancer from the perspective of the Chinese healthcare system.DesignsBased on the overall...