Lifetime Quality-adjusted Life Years Research Articles

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.

Transcatheter aortic valve implantation with SAPIEN 3 versus surgical aortic valve replacement in patients with symptomatic severe aortic stenosis at low risk of surgical mortality: a cost-utility analysis for Switzerland.

The 2021 European Society of Cardiology Guidelines on valvular heart disease recommend transcatheter aortic valve implantation (TAVI) for patients with symptomatic severe aortic stenosis at low surgical risk and age ≥75 years who are suitable for a transfemoral approach (recommendation class IA) based on two large randomised controlled trials (PARTNER 3 and Evolut Low Risk) comparing transcatheter aortic valve implantation with surgical aortic valve replacement (SAVR). Whether such an approach is cost-effective in Switzerland remains unclear. The aim of this cost-utility analysis was to compare transcatheter aortic valve implantation with SAPIEN 3 versus surgical aortic valve replacement in symptomatic severe aortic stenosis patients at low risk of surgical mortality from the perspective of Swiss compulsory health insurance using data from the PARTNER 3 trial (reflecting specifically the safety and efficacy of the SAPIEN 3 TAVI device). A previously published two-stage Markov-based model that captured clinical outcomes from the PARTNER 3 trial was adapted from the perspective of the Swiss compulsory health insurance system, using local or geographically close general population mortality and utility data, unit costs and medical resource use from publicly available sources and based on expert opinion. The model had a lifetime horizon with a 3% yearly discounting factor. The cost-utility analysis estimated changes in both direct healthcare costs and health-related quality-adjusted life years for transcatheter aortic valve implantation compared with surgical aortic valve replacement in patients with symptomatic severe aortic stenosis at low risk of surgical mortality. Overall, transcatheter aortic valve implantation with SAPIEN 3 resulted in lifetime costs per patient of CHF 79,534 and quality-adjusted life years per patient of 9.64, compared with surgical aortic valve replacement lifetime costs and quality-adjusted life years per patient of CHF 76,891 and 8.96, respectively. Compared with surgical aortic valve replacement, transcatheter aortic valve implantation was estimated to offer an incremental improvement of +0.68 quality-adjusted life years per patient at an increased cost of +CHF 2643 per patient over a lifetime horizon. The incremental cost-effectiveness ratio was CHF 3866 per quality-adjusted life year gained and remained below CHF 50,000 per quality-adjusted life year gained across several sensitivity analyses. This analysis suggests that transcatheter aortic valve implantation using the SAPIEN 3 device is likely to be a highly cost-effective alternative for symptomatic severe aortic stenosis patients at a low risk of surgical mortality, treated in the contemporary Swiss setting. These findings may help to inform a holistic approach when making policy decisions for the management of this patient group.

A cost-effectiveness analysis of stereotactic ablative radiotherapy versus conventionally fractionated radiotherapy in the management of stage 1 non-small-cell lung cancer: Results from the TROG 09.02 CHISEL study.

Stereotactic ablative radiotherapy (SABR) is a standard of care treatment for medically inoperable early-stage non-small-cell lung cancer (NSCLC). The CHISEL trial was a phase 3 randomised controlled trial that compared SABR to conventional radiation therapy (CRT). Using patient-level data, we compared the cost-effectiveness of SABR and CRT for early-stage NSCLC. Data on treatment exposure, outcomes (recurrence, survival) and quality of life (QoL; EORTC QLQ-C30) were sourced from the trial. Quality-adjusted life years (QALYs) were estimated for the trial period using Australian utility weights for the EORTC QLQ-C30-derived QLU-C10D. Costs related to simulation, planning, delivery, verification and post-treatment monitoring were estimated by applying Australian Medicare Benefits Schedule fees. The costs of post-progression therapy and grade ≥3 toxicity were estimated using trial data and relevant literature sources. Cost-effectiveness was investigated as the incremental cost per QALY gained for SABR compared to CRT. Complete QoL data were available for 21 patients: 14 in the SABR arm and 7 in the CRT arm. Mean QALYs discounted at 5% per annum were similar between arms: 12.68 months for SABR and 12.12 months for CRT. The mean costs of delivering SABR and CRT were $4763 and $6817, respectively. The costs of monitoring were similar in both arms, $4856 and $4853 for SABR and CRT. The mean costs of post-progression therapy were $24,572 for SABR and $42,801 for CRT. The mean costs of grade ≥3 toxicity were $809 in the SABR arm and $132 in the CRT arm. Therefore, the total mean cost for SABR over the period of interest was lower for SABR than CRT. Given lower mean costs and numerically higher QALYs for SABR compared with CRT, an incremental cost-effectiveness ratio was not calculated. Compared to CRT, SABR is a cost-effective treatment for early-stage NSCLC as the estimated upfront treatment cost and the cost of subsequent care are lower for SABR for comparable mean QALYs. Assessment of the lifetime QALYs and projections of cost estimation will provide a better indication of the long-term cost-effectiveness of SABR.

Cost-Effectiveness And Health Impact Of Increasing Emergency Department Pediatric Readiness In The US.

The quality of emergency department (ED) care for children in the US is highly variable. The National Pediatric Readiness Project aims to improve survival for children receiving emergency services. We conducted a cost-effectiveness analysis of increasing ED pediatric readiness, using a decision-analytic simulation model. Previously published primary analyses of a nationally representative, population-based cohort of children receiving emergency services at 747 EDs in eleven states provided clinical and cost parameters. From a health care sector perspective, we used a 3percent annual discount rate and quantified lifetime costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). We performed probabilistic, one-way, and subgroup sensitivity analyses. Increasing ED pediatric readiness yields 69,100 QALYs for the eleven-state cohort, costing $9,300 per QALY gained. Achieving high readiness nationally yields 179,000 QALYs at the same ICER (with implementation costs of approximately $260million). Implementing high ED pediatric readiness for all EDs in the US is highly cost-effective.

Cost Analysis of Scleral Buckle and Pars Plana Vitrectomy for Retinal Detachment Surgery.

To compare the cost and utility of scleral buckle (SB) and pars plana vitrectomy (PPV) techniques for repairing moderately complex rhegmatogenous retinal detachment (RRD). A cost-utility analysis was conducted using data from the Primary Retinal Detachment Outcomes Study (PRO) and a study conducted by the author. Total costs, patient utility over a lifetime, and cost per quality-adjusted life year (QALY) were calculated for each surgical procedure. The cost of scleral buckle surgery was €287.93, with an estimated lifetime QALY of 7.49. Costs per QALY were €38.44. According to the PRO study and Belin et al, total costs were $5975, with a lifetime QALY of 5.4 and costs per QALY of $1106. The cost of pars plana vitrectomy (PPV) was €1468.26, with an estimated lifetime QALY of 6.84 and costs per QALY of €214.65. Based on the PRO study and Belin et al, total costs were $8125, with a lifetime QALY of 4.7 and costs per QALY of $2196. Repairing moderately complex RRD presents a highly cost-effective profile for both SB and PPV techniques, well below recommended QALY thresholds. SB demonstrated a slightly more favorable profile compared to PPV.

Assessing the cost-effectiveness of replacing antimetabolites with mTOR inhibitors in heart transplant immunosuppression in China: a network meta-analysis-based economic evaluation.

Although several pharmacoeconomic studies have assessed the cost-effectiveness of maintenance immunosuppressive regimens for heart transplant recipients, economic comparisons between various combination drug therapies remain sparse. This study used an economic evaluation based on network meta-analysis to assess the cost-effectiveness of four immunosuppressive regimens for adult heart transplant recipients in China. We conducted a systematic search for clinical trials in PubMed, Embase, Cochrane Library, Web of Science, China National Knowledge Infrastructure (CNKI), Wanfang Data, and VIP database. A validated Markov model was adapted to reflect the Chinese medical landscape. Four maintenance immunosuppression regimens were considered: tacrolimus/mycophenolate mofetil (TAC/MMF), cyclosporine/mycophenolate mofetil (CSA/MMF), everolimus/cyclosporine (EVL/CSA), and sirolimus/tacrolimus (SRL/TAC). The probabilities of health events were derived from a comprehensive literature review. Direct medical costs, adjusted for 2022 values, were from public documents and websites, while utilities for quality-adjusted life-years (QALYs) were taken from previous studies. Primary outcomes were mean lifetime cost, QALYs, and cost-effectiveness, with a willingness-to-pay (WTP) threshold set at three times China's GDP per capita in 2022. Sensitivity analyses were conducted to test the robustness of the results. The base case analysis identified TAC/MMF as the most cost-effective regimen, producing a mean of 6.31 QALYs per patient at a cost of Chinese Yuan (CNY) 534,182.89. Sensitivity analyses consistently reinforced TAC/MMF as the most cost-effective and robust choice. TAC/MMF is the most cost-effective maintenance immunosuppressive regimen for heart transplant recipients within the Chinese health system. The study findings are reinforced by sensitivity analyses, affirming their robustness amid various uncertainties.

Population health value of being in target: Results from the Canadian Multi-Morbidity Model for Type 2 Diabetes

ObjectiveTo develop a microsimulation model for type 2 diabetes using population-level real-world data. Such a model allows for the synthesis of multiple data sources for comparative effectiveness analysis related to a variety of correlated outcomes. ApproachThe model was built using health state features including sex, age, diabetes duration, laboratory test results, and a history of major acute events. Features update using a cycle length of one month. Events modelled include myocardial infarction, stroke, heart failure, amputation and death. Model outputs include counts of events, lifetime healthcare costs and quality-adjusted life-years (QALYs). We then used the model to calculate the number of events that could be averted if a population with type 2 diabetes achieved treatment targets for HbA1c, blood pressure and LDL-cholesterol for 10 years. ResultsBringing all 60-year-olds with type 2 diabetes into target for 10 years would result in annual event reductions of 87.0 per 100,000 person-years for myocardial infarction, 49.4 for stroke, and 166.8 for heart failure. QALYs would improve by 1,155 per 100,000 patients. For 75-year-olds, annual event reductions would be 178.2, 56.8 and 261.6 per 100,000 person-years, respectively, and QALYs would improve by 2,121 per 100,000 patients. ConclusionsPopulation-level real-world data can be used to develop microsimulation models for type 2 diabetes that estimate long-term event risks, mortality and healthcare costs. This model is capable of comparative effectiveness and cost-effectiveness analysis of novel therapies in diabetes.

Primary HPV screening compared with other cervical cancer screening strategies in women with HIV: a cost-effectiveness study.

To compare the model-predicted benefits, harms, and cost-effectiveness of cytology, cotesting, and primary HPV screening in U.S. women living with HIV (WLWH). We adapted a previously published Markov decision model to simulate a cohort of U.S. WLWH. United States. A hypothetical inception cohort of WLWH. We simulated five screening strategies all assumed the same strategy of cytology with HPV triage for ASCUS for women aged 21 to 29 years. The different strategies noted are for women aged 30 and older as the following: continue cytology with HPV triage, cotesting with repeat cotesting triage, cotesting with HPV16/18 genotyping triage, primary hrHPV testing with cytology triage, and primary hrHPV testing with HPV16/18 genotyping triage. The outcomes include colposcopies, false-positive results, treatments, cancers, cancer deaths, life-years and costs, and lifetime quality-adjusted life-years. Compared with no screening, screening was cost-saving, and > 96% of cervical cancers and deaths could be prevented. Cytology with HPV triage dominated primary HPV screening and cotesting. At willingness-to-pay thresholds under $250,000, probabilistic sensitivity analyses indicated that primary HPV testing was more cost-effective than cotesting in over 98% of the iterations. Our study suggests the current cytology-based screening recommendation is cost-effective, but that primary HPV screening could be a cost-effective alternative to cotesting. To improve the cost-effectiveness of HPV-based screening, increased acceptance of the HPV test among targeted women is needed, as are alternative follow-up recommendations to limit the harms of high false-positive testing.

Modeling First-Line Daratumumab Use for Newly Diagnosed, Transplant-Ineligible, Multiple Myeloma: A Cost-Effectiveness and Risk Analysis for Healthcare Payers.

This study aimed to assess the cost-effectiveness of two regimens regarded as the standard of care for the treatment of newly diagnosed, transplant-ineligible multiple myeloma in Singapore: (1) daratumumab, lenalidomide, and dexamethasone and (2) bortezomib, lenalidomide, and dexamethasone. Additionally, it aimed to explore potential strategies to manage decision uncertainty and mitigate financial risk. A cost-effectiveness analysis from the healthcare system perspective was conducted using a partitioned survival model to estimate lifetime costs and quality-adjusted life years (QALYs) associated with daratumumab-based treatment and the bortezomib-based regimen. The analysis used data from the MAIA and SWOG S0777 trials and incorporated local real-world data where available. Sensitivity analyses were performed to evaluate the robustness of the findings, and a risk analysis was conducted to analyze various payer strategies in terms of their payer strategy and uncertainty burden (P-SUB), which account for the decision uncertainty and the additional cost of choosing a suboptimal intervention. The incremental cost-effectiveness ratio (ICER) for daratumumab, lenalidomide, and dexamethasone (DRd) compared with bortezomib, lenalidomide, and dexamethasone (VRd) was US $90,364 per QALY gained. The results were sensitive to variations in survival for DRd, postprogression treatment costs, cost of hospice care, and hazard ratio for progression-free survival. The scenarios explored indicated that structural assumptions, such as the time horizon of the analysis, significantly influenced the results due to uncertainties arising from immature trial data and treatment efficacy over time. Among the various payer strategies compared, an upfront price discount for daratumumab emerged as the best approach with the lowest P-SUB at US $14,708. In conclusion, this study finds that daratumumab as a first-line treatment for myeloma exceeds the cost-effectiveness threshold considered in this evaluation. An upfront price reduction is the recommended strategy to manage uncertainties and mitigate financial risks. These findings highlight the importance of targeted payer strategies to address specific types and sources of uncertainty.

Cost-effectiveness of early vs delayed use of abemaciclib combination therapy for patients with high-risk hormone receptor-positive/human epidermal growth factor receptor 2-negative early breast cancer.

Abemaciclib was newly approved for hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) high-risk early breast cancer (EBC). Clinical guidelines recommended abemaciclib as the first-line treatment for HR+/ HER2- EBC (early use) or HR+/ HER2- metastatic breast cancer (MBC) (delayed use). To compare the cost-effectiveness of early vs delayed use of abemaciclib for treatment of HR+/HER2- high-risk EBC. Early use was defined as combined abemaciclib and endocrine therapy as first-line therapy for EBC, followed by treatment with fulvestrant for MBC. Delayed use was defined as endocrine therapy for EBC, followed by combined abemaciclib and fulvestrant therapy for MBC. A 5-state model was developed to estimate lifetime costs, life-years (LYs), and quality-adjusted life-years (QALYs) of hypothetical patients with HR+/ HER2- EBC from a third-party US payer's perspective. Key clinical and safety data were derived from the monarchE and MONARCH 2 clinical trials. Costs, utilities, and disutility values of adverse events were obtained from the literature. We calculated the incremental cost-effectiveness ratio (ICER) of early vs delayed abemaciclib use and compared it with a willingness-to-pay (WTP) threshold of $100,000 per LY or QALY. Deterministic and probabilistic sensitivity analyses (PSAs) were performed to test the robustness of the base-case model. Base-case analysis showed early use yielded 21.08 LYs and 17.93 QALYs for $586,213 and delayed use yielded 11.14 LYs and 9.38 QALYs for $157,576. The ICER of early vs delayed use was $43,136/LY and $50,104/QALY, which was cost-effective at the WTP threshold of $100,000. The PSA result indicated that a 94.6% likelihood of early use (vs delayed use) was cost-effective at the WTP threshold of $100,000 per QALY. This study suggests that giving abemaciclib in the early stage rather than waiting until patients develop metastatic disease (current standard of care in MBC) is a cost-effective strategy.

The UK resuscitative endovascular balloon occlusion of the aorta in trauma patients with life-threatening torso haemorrhage: the (UK-REBOA) multicentre RCT.

The most common cause of preventable death after injury is haemorrhage. Resuscitative endovascular balloon occlusion of the aorta is intended to provide earlier, temporary haemorrhage control, to facilitate transfer to an operating theatre or interventional radiology suite for definitive haemostasis. To compare standard care plus resuscitative endovascular balloon occlusion of the aorta versus standard care in patients with exsanguinating haemorrhage in the emergency department. Pragmatic, multicentre, Bayesian, group-sequential, registry-enabled, open-label, parallel-group randomised controlled trial to determine the clinical and cost-effectiveness of standard care plus resuscitative endovascular balloon occlusion of the aorta, compared to standard care alone. United Kingdom Major Trauma Centres. Trauma patients aged 16 years or older with confirmed or suspected life-threatening torso haemorrhage deemed amenable to adjunctive treatment with resuscitative endovascular balloon occlusion of the aorta. Participants were randomly assigned 1 : 1 to: standard care, as expected in a major trauma centre standard care plus resuscitative endovascular balloon occlusion of the aorta. Primary: Mortality at 90 days. Secondary: Mortality at 6 months, while in hospital, and within 24, 6 and 3 hours; need for haemorrhage control procedures, time to commencement of haemorrhage procedure, complications, length of stay (hospital and intensive care unit-free days), blood product use. Health economic: Expected United Kingdom National Health Service perspective costs, life-years and quality-adjusted life-years, modelled over a lifetime horizon. Case report forms, Trauma Audit and Research Network registry, NHS Digital (Hospital Episode Statistics and Office of National Statistics data). Ninety patients were enrolled: 46 were randomised to standard care plus resuscitative endovascular balloon occlusion of the aorta and 44 to standard care. Mortality at 90 days was higher in the standard care plus resuscitative endovascular balloon occlusion of the aorta group (54%) compared to the standard care group (42%). The odds ratio was 1.58 (95% credible interval 0.72 to 3.52). The posterior probability of an odds ratio > 1 (indicating increased odds of death with resuscitative endovascular balloon occlusion of the aorta) was 86.9%. The overall effect did not change when an enthusiastic prior was used or when the estimate was adjusted for baseline characteristics. For the secondary outcomes (3, 6 and 24 hours mortality), the posterior probability that standard care plus resuscitative endovascular balloon occlusion of the aorta was harmful was higher than for the primary outcome. Additional analyses to account for intercurrent events did not change the direction of the estimate for mortality at any time point. Death due to haemorrhage was more common in the standard care plus resuscitative endovascular balloon occlusion of the aorta group than in the standard care group. There were no serious adverse device effects. Resuscitative endovascular balloon occlusion of the aorta is less costly (probability 99%), due to the competing mortality risk but also substantially less effective in terms of lifetime quality-adjusted life-years (probability 91%). The size of the study reflects the relative infrequency of exsanguinating traumatic haemorrhage in the United Kingdom. There were some baseline imbalances between groups, but adjusted analyses had little effect on the estimates. This is the first randomised trial of the addition of resuscitative endovascular balloon occlusion of the aorta to standard care in the management of exsanguinating haemorrhage. All the analyses suggest that a strategy of standard care plus resuscitative endovascular balloon occlusion of the aorta is potentially harmful. The role (if any) of resuscitative endovascular balloon occlusion of the aorta in the pre-hospital setting remains unclear. Further research to clarify its potential (or not) may be required. This trial is registered as ISRCTN16184981. This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 14/199/09) and is published in full in Health Technology Assessment; Vol. 28, No. 54. See the NIHR Funding and Awards website for further award information.

Osteochondroplasty with or without labral repair is more cost-effective than arthroscopic lavage with or without labral repair for treatment of young adults with femoroacetabular impingement: A cost-utility analysis based on data from a randomized controlled trial

ObjectivesThe objective of this study was to conduct a cost–utility analysis of osteochondroplasty with or without labral repair compared to arthroscopic lavage with or without labral repair for femoroacetabular impingement (FAI) from a Canadian public payer perspective. MethodsA Markov model was constructed to compare the lifetime quality-adjusted life years (QALYs) and costs of the two treatment strategies. The target population was surgical FAI patients aged 36 years. The primary data source was patient-level data from the Femoroacetabular Impingement Randomised Controlled Trial, which evaluated the efficacy of the surgical correction of FAI via arthroscopic osteochondroplasty with or without labral repair compared to arthroscopic lavage with or without labral repair in Canada. Long-term data were extrapolated using a generalized gamma model. The primary outcome was the incremental cost-effectiveness ratio, calculated by dividing the difference in costs by the difference in QALYs between osteochondroplasty and lavage, with or without labral repair. Probabilistic sensitivity analyses and one-way sensitivity analyses were used to characterize uncertainty of model parameters and assumptions. ResultsOver a lifetime horizon, osteochondroplasty, with or without labral repair, had a greater expected benefit (0.63 QALYs gained per patient) and lower costs ($955.89 saved per patient), as compared with lavage with or without labral repair. Probabilistic sensitivity analyses demonstrated that the probability of osteochondroplasty, with or without labral repair, being cost-effective was 90.5% at a commonly used willingness-to-pay threshold of $50,000/QALY in Canada. Across all one-way sensitivity analyses, osteochondroplasty with or without labral repair remained a cost-effective option. ConclusionOver a lifetime time horizon, osteochondroplasty, with or without labral repair, is a cost-effective treatment strategy for young adults with FAI. Future research involving real-word data is needed to further validate these findings. Level of evidenceIII.

Comparing the effectiveness and cost-effectiveness of alternative type 2 diabetes monitoring intervals in resource limited settings.

Type 2 diabetes (T2D) represents a growing disease burden in South Africa. While glycated haemoglobin (HbA1c) testing is the gold standard for long-term blood glucose management, recommendations for HbA1c monitoring frequency are based on expert opinion. This study investigates the effectiveness and cost-effectiveness of alternative HbA1c monitoring intervals in the management of T2D. A Markov model with three health states (HbA1c <7%, HbA1c ≥ 7%, Dead) was used to estimate lifetime costs and quality-adjusted life years (QALYs) of alternative HbA1c monitoring intervals among patients with T2D, using a provider's perspective and a 3% discount rate. HbA1c monitoring strategies (three-monthly, four-monthly, six-monthly and annual tests) were evaluated with respect to the incremental cost-effectiveness ratio (ICER) assessing each comparator against a less costly, undominated alternative. The scope of costs included the direct medical costs of managing diabetes. Transition probabilities were obtained from routinely collected public sector HbA1c data, while health service utilization and health-related-quality-of-life (HRQoL) data were obtained from a local cluster randomized controlled trial. Other parameters were obtained from published studies. Robustness of findings was evaluated using one-way and probabilistic sensitivity analyses. A South African indicative cost-effectiveness threshold of USD2665 was adopted. Annual and lifetime costs of managing diabetes increased with HbA1c monitoring, while increased monitoring provides higher QALYs and life years. For the overall cohort, the ICER for six-monthly vs annual monitoring was cost-effective (USD23 22.37 per QALY gained), whereas the ICER of moving from six-monthly to three-monthly monitoring was not cost effective (USD6437.79 per QALY gained). The ICER for four-monthly vs six-monthly monitoring was extended dominated. The sensitivity analysis showed that the ICERs were most sensitive to health service utilization rates. While the factors influencing glycaemic control are multifactorial, six-monthly monitoring is potentially cost-effective while more frequent monitoring could further improve patient HrQoL.

Cost-Effectiveness of Adjuvant Abemaciclib and Ribociclib in High-Risk Hormone Receptor-Positive Early Breast Cancer: An Indian Perspective.

Incorporating adjuvant cyclin-dependent kinase (CDK) 4/6 inhibitors abemaciclib and ribociclib along with endocrine therapy has been shown to improve invasive disease-free survival (iDFS) for hormone receptor-positive (HR+) human epidermal receptor 2-negative (HER2-) early breast cancer (EBC). This study assesses the cost-effectiveness of this strategy, along with adjuvant aromatase inhibitors from an Indian perspective. A Markov chain model evaluated the cost-effectiveness of abemaciclib and ribociclib with letrozole compared with letrozole alone for HR+/HER2- EBC from a payer perspective in India. Key measures included lifetime quality-adjusted life-years (QALY), life-years (LY), and total costs. This study explores two scenarios for effectiveness: a best-case (BC) scenario, where the benefit of CDK4/6 inhibitors in improving iDFS lasts a lifetime, and a worst-case (WC) scenario, where benefits disappear after 5 years. Probabilistic sensitivity analyses (PSA) were used to account for simulation uncertainty. In the BC scenario, abemaciclib added 2.17 QALY and 4.96 LY, incurring ₹2,317,957.7 ($27,756.65 in US dollars [USD]) in additional costs. However, the incremental cost-effectiveness ratio (ICER) for abemaciclib exceeded India's willingness-to-pay threshold in the BC and WC scenarios. In the BC scenario, ribociclib added 0.98 QALY and 2.58 LY with added costs of ₹1,711,504.32 ($20,494.6 USD). The ICER for ribociclib also surpassed India's threshold in both scenarios. PSA showed that neither drug was cost-effective at the current market prices in either BC/WC scenario. The cost of abemaciclib and ribociclib needs to be reduced by at least 78.61% and 87.19%, respectively, to be cost-effective in the BC scenario. The combination of adjuvant abemaciclib or ribociclib with letrozole is not cost-effective for HR+/HER2- EBC in India in either the BC or WC scenario.

Cost-Effectiveness of Biosimilars vs Leflunomide in Patients With Rheumatoid Arthritis

Among patients with rheumatoid arthritis (RA) who had an inadequate response to methotrexate, a treatment sequence initiated with biosimilar disease-modifying antirheumatic drugs (DMARDs) provides better clinical efficacy compared with conventional synthetic DMARDs recommended by current treatment guidelines; but its cost-effectiveness evidence remains unclear. To evaluate the cost-effectiveness of the treatment sequence initiated with biosimilar DMARDs after failure with methotrexate vs leflunomide and inform formulary listing decisions. This economic evaluation's cost-effectiveness analysis was performed at a Hong Kong public institution using the Markov disease transition model to simulate the lifetime disease progression and cost for patients with RA, using monetary value in 2022. Scenario and sensitivity analyses were performed to test the internal validity of the modeling conclusion. Participants included patients diagnosed with RA from 2000 to 2021 who were retrieved retrospectively from local electronic medical records to generate model input parameters. Statistical analysis was performed from January 2023 to March 2024. The model assesses 3 competing treatment sequences initiated with biosimilar infliximab (CT-P13), biosimilar adalimumab (ABP-501), and leflunomide; all used in combination with methotrexate. Lifetime health care cost and quality-adjusted life-years (QALYs) of the simulated cohort. In total, 25 099 patients with RA were identified (mean [SD] age, 56 [17] years; 19 469 [72.7%] women). In the base-case analysis, the lifetime health care cost and QALYs for the treatment sequence initiated with leflunomide were US $154 632 and 14.82 QALYs, respectively; for biosimilar infliximab, they were US $152 326 and 15.35 QALYs, respectively; and for biosimilar adalimumab, they were US $145 419 and 15.55 QALYs, respectively. Both biosimilar sequences presented lower costs and greater QALYs than the leflunomide sequence. In the deterministic sensitivity analysis, the incremental cost-effectiveness ratio (US$/QALY) comparing biosimilar infliximab sequence vs leflunomide sequence and biosimilar adalimumab sequence vs leflunomide sequence ranged from -15 797 to -8615 and -9088 to 10 238, respectively, all below the predefined willingness-to-pay threshold (US $48 555/QALY gain). In the probabilistic sensitivity analysis, the probability of treatment sequence initiated with leflunomide, biosimilar infliximab, and biosmilar adalimumab being cost-effective out of 10 000 iterations was 0%, 9%, and 91%, respectively. In this economic evaluation study, the treatment sequences initiated with biosimilar DMARDs were cost-effective compared with the treatment sequence initiated with leflunomide in managing patients with RA who experienced failure with the initial methotrexate treatment. These results suggest the need to update clinical treatment guidelines for initiating biosimilars immediately after the failure of methotrexate for patients with RA.

Estimating Risk Factor Time Paths Among People with Type 2 Diabetes and QALY Gains from Risk Factor Management.

Most type 2 diabetes simulation models utilise equations mapping out lifetime trajectories of risk factors [e.g. glycated haemoglobin (HbA1c)]. Existing equations, using historic data or assuming constant risk factors, frequently underestimate or overestimate complication rates. Updated risk factor time path equations are needed for simulation models to more accurately predict complication rates. (1) Update United Kingdom Prospective Diabetes Study Outcomes Model (UKPDS-OM2) risk factor time path equations; (2) compare quality-adjusted life-years (QALYs) using original and updated equations; and (3) compare QALY gains for reference case simulations using different risk factor equations. Using pooled contemporary data from two randomised trials EXSCEL and TECOS (n = 28,608), we estimated: dynamic panel models of seven continuous risk factors (high-density lipoprotein cholesterol, low density lipoprotein cholesterol, HbA1c, haemoglobin, heart rate, blood pressure and body mass index); two-step models of estimated glomerular filtration rate; and survival analyses of peripheral arterial disease, atrial fibrillation and albuminuria. UKPDS-OM2-derived lifetime QALYs were extrapolated over 70 years using historical and the new risk factor equations. All new risk factor equation predictions were within 95% confidence intervals of observed values, displaying good agreement between observed and estimated values. Historical risk factor time path equations predicted trial participants would accrue 9.84 QALYs, increasing to 10.98 QALYs using contemporary equations. Incorporating updated risk factor time path equations into diabetes simulation models could give more accurate predictions of long-term health, costs, QALYs and cost-effectiveness estimates, as well as a more precise understanding of the impact of diabetes on patients' health, expenditure and quality of life. ClinicalTrials.gov NCT01144338 and NCT00790205.

Exploring the Cost-Effectiveness of Newborn Screening for Metachromatic Leukodystrophy (MLD) in the UK.

Metachromatic leukodystrophy (MLD) is a fatal inherited lysosomal storage disease that can be detected through newborn bloodspot screening. The feasibility of the screening assay and the clinical rationale for screening for MLD have been previously demonstrated, so the aim of this study is to determine whether the addition of screening for MLD to the routine newborn screening program in the UK is a cost-effective use of National Health Service (NHS) resources. A health economic analysis from the perspective of the NHS and Personal Social Services was developed based on a decision-tree framework for each MLD subtype using long-term outcomes derived from a previously presented partitioned survival and Markov economic model. Modelling inputs for parameters related to epidemiology, test characteristics, screening and treatment costs were based on data from three major UK specialist MLD hospitals, structured expert opinion and published literature. Lifetime costs and quality-adjusted life years (QALYs) were discounted at 1.5% to account for time preference. Uncertainty associated with the parameter inputs was explored using sensitivity analyses. This health economic analysis demonstrates that newborn screening for MLD is a cost-effective use of NHS resources using a willingness-to-pay threshold appropriate to the severity of the disease; and supports the inclusion of MLD into the routine newborn screening programme in the UK.

Cost-effectiveness of a multicomponent-adherence intervention in fracture liaison services

ABSTRACT Background This study aims to assess the lifetime cost-effectiveness of a multi-component adherence intervention (MCAI), including a patient decision aid and motivational interviewing, compared to usual care in patients with a recent fracture attending fracture liaison services (FLS) and eligible for anti-osteoporosis medication (AOM). Research design and methods Data on AOM initiation and one-year persistence were collected from a quasi-experimental study conducted between 2019 and 2023 in two Dutch FLS centers. An individual level, state-transition Markov model was used to simulate lifetime costs and quality-adjusted life years (QALYs) with a societal perspective of MCAI vs usual care. One-way and probabilistic sensitivity analyses were conducted including variation in additional FLS and MCAI costs (no MCAI cost in baseline). Results MCAI was associated with gain in QALYs (0.0012) and reduction in costs (-€16) and is therefore dominant. At the Dutch willingness-to-pay threshold of €50,000/QALY, MCAI remained cost-effective when increasing costs of the FLS visit or the yearly maintenance cost for MCAI up to +€60. Probabilistic sensitivity analysis demonstrated MCAI to be dominant in 54% of the simulations and cost-effective in 87% with a threshold of €50,000/QALY. Conclusions A MCAI implemented in FLS centers may lead to cost-effective allocation of resources in FLS care, depending on extra costs.

Cost-effectiveness of adjuvant abemaciclib and ribociclib in high-risk, hormone receptor–positive, early breast cancer in India.

e12516 Background: Incorporating adjuvant cyclin-dependent kinase (CDK) 4/6 inhibitors abemaciclib and ribociclib along with endocrine therapy has been shown to improve invasive disease-free survival (iDFS) for hormone receptor-positive (HR+) human epidermal receptor 2 negative (HER2-) early breast cancer (EBC). This study assesses the cost-effectiveness of this strategy, along with adjuvant aromatase inhibitors (AI) from an Indian perspective. Methods: A Markov chain model evaluated the cost-effectiveness of abemaciclib and ribociclib with letrozole compared to letrozole alone for HR+/HER2- EBC from a payer perspective in India. Key measures included lifetime quality-adjusted life years (QALY), life-years (LY), and total costs. This study explores two scenarios for effectiveness: a best-case (BC) scenario, where the benefit of CDK4/6 inhibitors in improving iDFS lasts a lifetime, and a worst-case (WC) scenario, where benefits disappear after 5 years. Probabilistic sensitivity analyses (PSA) were used to account for simulation uncertainty. Results: In the BC scenario, abemaciclib added 2.17 QALY and 4.96 LY, costing $27,756.65 more. Ribociclib in the BC scenario added 0.98 QALY and 2.58 LY, with added costs of $20,494.6. The incremental cost-effectiveness ratio for abemaciclib and ribociclib surpassed the willingness to pay threshold for in India in both scenarios (Table). At current market prices there is 0% probability of cost-effectiveness of abemaciclib and ribociclib. The cost of abemaciclib and ribociclib needs to be reduce by 78.61% and 87.19%, respectively to achieve cost-effectiveness. Conclusions: The combination of adjuvant abemaciclib or ribociclib with letrozole is not cost-effective for HR+/HER2- EBC in India in either the best or worst-case scenario.[Table: see text]

Clinical and economic benefit of genomic testing strategies to guide the treatment of patients with HR+/HER2- breast cancer in the US.

10584 Background: Multiple testing technologies are available to inform treatment decisions for hormone receptor positive (HR+) and human epidermal growth factor receptor 2 negative (HER2-) early-stage breast cancer, including tumor profiling and germline genetic testing (GGT). The 21-gene assay provides an estimate of distant recurrence risk and potential clinical benefit of chemotherapy. GGT identifies pathogenic variants which inform lifetime cancer risk as well may inform therapy options. This study explored the potential synergy of utilizing both tests by assessing the cost-effectiveness of this strategy compared to no testing from a societal perspective. Methods: A health economic model was developed using best practices to assess a patient cohort with HR+/HER2-, node-negative (N0) or node-positive (N1) breast cancer that was guided to surgical and adjuvant treatment by GGT ( ATM, BRCA1/2, CDH1, CHEK2, PALB2, PTEN,and TP53) and the 21-gene assay. Treatment effectiveness was informed by clinical trials (TAILORx and RxPONDER); costs for treatment, monitoring, and health utilities were sourced from literature. Clinical expert opinion and guidelines informed current US clinical practice. The base case analysis evaluated lifetime total costs, life-years, and quality-adjusted life years (QALYs) versus no testing. Scenario and sensitivity analyses were performed to evaluate model robustness. Results: The testing strategy was dominant (lower cost and higher QALYs gained) compared to no testing leading to a QALY improvement of 0.286 and cost savings of $16,463 in the N0 population (Table) and a 0.099 QALY improvement and $299 cost savings in the N1 population. Scenario analyses indicated that while the 21-gene assay was the primary driver of results, there were synergistic effects when tests were combined. Sensitivity analysis supported cost-effectiveness at most willingness-to-pay thresholds and identified the probability of chemotherapy and rate of distant recurrence as the most impactful model inputs. Conclusions: Using a testing strategy including both the 21-gene assay and GGT enables optimization of treatment decisions in early HR+/HER2- breast cancer, improving patient outcomes at reduced costs. [Table: see text]