Incremental Cost-utility Ratio Research Articles

Cost-Effectiveness and Economic Burden Analyses on All First-Line Treatments of Chronic Lymphocytic Leukemia

ObjectivesSeveral chemoimmunotherapy and targeted treatment regimens are approved as front-line therapies in chronic lymphocytic leukemia. We estimated for the 10-year cost-effectiveness of these treatment regimens and the economic burden of following the estimated risk-stratified 21 040 patients with chronic lymphocytic leukemia diagnosed in 2020 for 10 years. MethodsA Markov model with 7 exclusive health states was specified over a 10-year time horizon. Treatment effectiveness inputs were obtained from a novel network meta-analysis on the progression-free survival, overall survival curves, and time to next treatment. Costs and utilities inputs were included for each health state for each treatment and discounted at 3.0%/year. Life-years (LYs) and quality-adjusted LYs (QALYs) for each treatment were determined. Using the lowest cost regimen as reference, the incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratio (ICUR) were estimated. The 10-year per-patient cost was determined by risk status and by initial treatment. ResultsVenetoclax-plus-obinutuzumab was the lowest cost regimen, hence the reference. Superior in effectiveness to all chemoimmunotherapies, it was cost saving. With the highest effectiveness gains at 6.26 LYs and 5.01 QALYs and despite being the most expensive regimen ($1 298 638 per patient), acalabrutinib-plus-obinutuzumab yielded the best ICER ($409 343/LY gained) and ICUR ($501 236/QALY gained). The remaining ICERs of targeted therapies ranged from $512 101/LY gained to $793 236/LY gained and the ICURs from $579 737/QALY gained to $869 300/QALY gained. The 10-year postdiagnosis low/high (venetoclax-plus-obinutuzumab/acalabrutinib-plus-obinutuzumab) economic burden ranges were $42 690 to $98 665 for low-risk, $141 339 to $326 660 for intermediate-risk, and $273 650 to $632 453 for high-risk patients. ConclusionsCompared with venetoclax-plus-obinutuzumab, chemoimmunotherapies are associated with less health benefits at higher cost. The targeted therapies achieve greater benefits at higher cost.

Reverse Total Shoulder Arthroplasty Is the Most Cost-effective Treatment Strategy for Proximal Humerus Fractures in Older Adults: A Cost-utility Analysis.

Proximal humerus fractures are the second-most common fragility fracture in older adults. Although reverse total shoulder arthroplasty (RTSA) is a promising treatment strategy for proximal humerus fractures with favorable clinical and quality of life outcomes, it is associated with much higher, and possibly prohibitive, upfront costs relative to nonoperative treatment and other surgical alternatives. (1) What is the cost-effectiveness of open reduction internal fixation (ORIF), hemiarthroplasty, and RTSA compared with the nonoperative treatment of complex proximal humerus fractures in adults older than 65 years from the perspective of a single-payer Canadian healthcare system? (2) Which factors, if any, affect the cost-effectiveness of ORIF, hemiarthroplasty, and RTSA compared with nonoperative treatment of proximal humerus fractures including quality of life outcomes, cost, and complication rates after each treatment? This cost-utility analysis compared RTSA, hemiarthroplasty, and ORIF with the nonoperative management of complex proximal humerus fractures in adults older than 65 years over a lifetime time horizon from the perspective of a single-payer healthcare system. Short-term and intermediate-term complications in the 2-year postoperative period were modeled using a decision tree, with long-term outcomes estimated through a Markov model. The model was initiated with a cohort of 75-year-old patients who had a diagnosis of a comminuted (three- or four-part) proximal humerus fractures; 90% of the patients were women. The mean age and gender composition of the model's cohort was based on a systematic review conducted as part of this analysis. Patients were managed nonoperatively or surgically with either ORIF, hemiarthroplasty, or RTSA. The three initial surgical treatment options of ORIF, hemiarthroplasty, and RTSA resulted in uncomplicated healing or the development of a complication that would result in a subsequent surgical intervention. The model reflects the complications that result in repeat surgery and that are assumed to have the greatest impact on clinical outcomes and costs. Transition probabilities and health utilities were derived from published sources, with costs (2020 CAD) sourced from regional costing databases. The primary outcome was the incremental cost-utility ratio, which was calculated using expected quality-adjusted life years (QALYs) gained and costs. Sensitivity analyses were conducted to explore the impact of changing key model parameters. Based on both pairwise and sequential analysis, RTSA was found to be the most cost-effective strategy for managing complex proximal humerus fractures in adults older than 65 years. Compared with nonoperative management, the pairwise incremental cost-utility ratios of hemiarthroplasty and RTSA were CAD 25,759/QALY and CAD 7476/QALY, respectively. ORIF was dominated by nonoperative management, meaning that it was both more costly and less effective. Sequential analysis, wherein interventions are compared from least to most expensive in a pairwise manner, demonstrated ORIF to be dominated by hemiarthroplasty, and hemiarthroplasty to be extendedly dominated by RTSA. Further, at a willingness-to-pay threshold of CAD 50,000/QALY, RTSA had 66% probability of being the most cost-effective treatment option. The results were sensitive to changes in the parameters for the probability of revision RTSA after RTSA, the treatment cost of RTSA, and the health utilities associated with the well state for all treatment options except ORIF, although none of these changes were found to be clinically realistic based on the existing evidence. Based on this economic analysis, RTSA is the preferred treatment strategy for complex proximal humerus fractures in adults older than 65 years, despite high upfront costs. Based on the evidence to date, it is unlikely that the parameters this model was sensitive to would change to the degree necessary to alter the model's outcome. A major strength of this model is that it reflects the most recent randomized controlled trials evaluating the management of this condition. Therefore, clinicians should feel confident recommending RTSA for the management of proximal humerus fractures in adults older than 65 years, and they are encouraged to advocate for this intervention as being a cost-effective practice, especially in publicly funded healthcare systems wherein resource stewardship is a core principle. Future high-quality trials should continue to collect both clinical and quality of life outcomes using validated tools such as the EuroQOL-5D to reduce parameter uncertainty and support decision makers in understanding relevant interventions' value for money. Level III, economic and decision analysis.

Intravenous iron for the treatment of iron deficiency anemia in China: a patient-level simulation model and cost-utility analysis comparing ferric derisomaltose with iron sucrose

Objectives Two intravenous (IV) iron formulations, ferric derisomaltose (FDI) and iron sucrose (IS), are currently available for the treatment of iron deficiency anemia (IDA) in China. Clinical studies have demonstrated that FDI has an improved efficacy and safety profile versus IS, while requiring fewer infusions to correct iron deficits. Based on these findings, the present study evaluated the costs and benefits of FDI and IS for the treatment of IDA, from a healthcare system and societal perspective in China. Methods A patient-level model was developed to project time to hematological response and incidence of cardiovascular adverse events and hypersensitivity reactions (HSRs) associated with FDI and IS over 5 years. Costs included iron acquisition, administration, and adverse event/HSR treatment costs, based on published studies, fee schedules, and a physician survey. Health state utilities associated with adverse events, HSRs, and the number of infusions were obtained from the literature and a time trade-off survey. Results From a healthcare system perspective, FDI was associated with incremental costs of RMB 1,934 (purchasing power parity USD 462) and incremental quality-adjusted life expectancy of 0.078 quality-adjusted life-years (QALYs) versus IS, yielding an incremental cost-utility ratio of RMB 24,901 (USD 5,949) in the base case scenario. From a societal perspective, FDI was associated with reduced total costs and therefore dominant versus IS. Limitations Limitations included the absence of clinical data specific to China and insufficient data to model persistence with treatment. Conclusions This was the first cost-utility analysis comparing FDI and IS for the treatment of IDA in China. Based on a patient-level model, FDI was found to improve quality of life and reduce administration and adverse events costs relative to IS. Using the 2020 Chinese gross domestic product per capita of RMB 72,447 (USD 17,307) as a cost-effectiveness threshold, FDI would be considered cost-effective in China.

MO375: The Health Economic Impact of Accurately Diagnosing CKD With and Without a UACR Test at Different Points in the Pathway of Disease Progression

Abstract BACKGROUND AND AIMS In the Kidney Disease Improving Global (KDIGO) guidelines, it is recommended that an accurate chronic kidney disease (CKD) diagnosis is based on repeated positive test results for estimated glomerular filtration rate (eGFR) and urine albumin-to-creatine ratio (UACR). While eGFR indicates kidney function decline, UACR identifies kidney damage, predicts CKD progression, and indicates risk for cardiovascular events. UACR testing is, therefore, a critical component of identifying CKD patients early. There is a lack of evidence that demonstrates the economic impact of early CKD diagnosis; hence, a cost-effectiveness model was built to demonstrate the potential clinical and economic benefit of undertaking UACR tests in those with early-stage CKD. METHOD The KDIGO heatmap quadrants were organised into a Markov model with three levels of severity (moderate, high, and very high CKD risk) over a lifetime horizon, for a cohort of 60-year-old patients in the base-case. Costs, life-years (LYs), quality adjusted life-years (QALYs), and the associated incremental cost-utility ratios (ICURs) were calculated (discounted at 3.5%). Costs consisted of testing, general practitioner (GP) visit, nephrologist visit, and treatment costs. Data inputs were based on published data from the literature and validated by clinicians. RESULTS Hypothetical comparisons between a 1000-patient cohort who had UACR tests undertaken in the early stages of CKD, predicted higher LYs and QALYS for patients in the KDIGO heatmap quadrants of G3aA1, G1A2, G2A2, G3aA2, G1A3, G2A3 and G3aA3 compared with a 1000-patient cohort who did not have UACR tests. By identifying individuals in the early stages of CKD, treatment can be initiated earlier, reducing the number of patients progressing to late-stage CKD and ultimately offsetting the more expensive treatment costs associated with late-stage CKD. Overall, ICURs for all quadrants were less than the UK's willingness to pay threshold of £20 000 per QALY. CONCLUSION Preliminary results suggest that undertaking regular UACR tests early in patients with CKD is a cost-effective strategy, as this allows an individual to initiate the appropriate treatment, resulting in slower disease progression. This is an important consideration for clinicians and policymakers due to the suboptimal UACR testing rates globally. Funding: Bayer AG

Minimally Invasive Surgery for Spontaneous Intracerebral Hematoma. Real-Life Implementation Model and Economic Estimation.

ObjectiveSpontaneous intracerebral hemorrhage is characterized by high fatality outcomes, even under best medical treatment. Recently, minimally invasive surgical (MIS) evacuation of the hematoma has shown promising results and may soon be implemented in the clinical practice. Hereby, we intended to foresee the logistic requirements for an early hematoma evacuation protocol, as well as to evaluate in a real-life implementation model the cost-utility of the two main MIS techniques for hemorrhagic stroke (catheter evacuation plus thrombolysis and neuroendoscopic aspiration).MethodsData were obtained from the pool of hemorrhagic-stroke patients admitted to our institution during an annual period (2020–2021) and contrasted to the reported results in published trials of MIS techniques. Potential candidates for surgical treatment were identified according to the inclusion/exclusion criteria established in these trials. Then, a cost-utility analysis was performed, which explored the incremental cost per unit of health gained with a given treatment. The treatment effect was measured by differences in modified Rankin Score, and subsequently converted to quality-adjusted life years (QALY).ResultsOf the 137 patients admitted to our center with supratentorial spontaneous intracerebral hemorrhage in a 1-year period, 17 (12.4%) were potential candidates for the catheter evacuation plus thrombolysis technique (Minimally Invasive Surgery with Thrombolysis in Intracerebral Hemorrhage Evacuation trial, MISTIE III criteria) and 59 (43.0%) for the neuroendoscopic aspiration technique (Dutch Intracerebral Hemorrhage Surgery Trial Pilot Study, DIST criteria). The incremental cost-utility ratio was € 76,533.13 per QALY for the catheter-based evacuation and € 60,703.89 per QALY for the endoscopic-based technique.ConclusionAround 12–43% of patients admitted to hospital with spontaneous hemorrhagic stroke could be potential candidates to MIS early evacuation of the cerebral hematoma. In our real-life implementation model, the cost-utility analysis favored the neuroendoscopic evacuation over the catheter aspiration technique. Further studies are advisable as new data from the ongoing randomized trials becomes available.

Cost-Utility Analysis of STN1013001, a Latanoprost Cationic Emulsion, versus Other Latanoprost Formulations (Latanoprost) in Open-Angle Glaucoma or Ocular Hypertension and Ocular Surface Disease in France.

Purpose To investigate the cost utility of STN1013001, a latanoprost cationic emulsion, versus Latanoprost in patients with open-angle glaucoma or ocular hypertension (OAG/OHT) and concomitant ocular surface disease (OSD) in France. Methods An early Markov model, including 7 health states and a 1-year cycle length, was developed to estimate the cost utility of STN1013001 versus Latanoprost from the French health system perspective over a 5-year time horizon. The model was populated with pooled data (treatment adherence, quality of life, disease progression, and resource utilization) collected, via a questionnaire, from a convenience sample of 5 French glaucoma specialists. Remaining data were retrieved from published sources. Half-cycle correction and 2.5% real social discount rate were applied to costs (in €2020), life years saved (LYS), and quality-adjusted life years (QALYs). The incremental cost-utility ratio (ICUR) was contrasted against the informal willingness-to-pay (WTP) range for incremental LYS or QALY gained (€30,000–€50,000) suggested for France. One-way and probabilistic sensitivity analyses tested the robustness of the baseline ICUR. Results Over a 5-year time horizon, STN1013001 resulted in an incremental 0.35 QALYs gained at an incremental cost of €7.39 compared to Latanoprost, resulting in an ICUR of €21.26. This is well below the lower limit of the unofficial WTP range proposed for France. Sensitivity analyses confirmed the robustness of the baseline results. Conclusion Once on the market, STN1013001 will provide the French health system with a cost-effective treatment versus Latanoprost for OAG/OHT + OSD patients. These results should be confirmed by future economic evaluations carried out alongside empirical trials.

Cost-effectiveness of indocyanine green angiography in postmastectomy breast reconstruction

Mastectomy skin flap necrosis is a major complication of skin- or nipple-sparing mastectomy. Indocyanine green angiography (ICGA) is a novel technology that can identify flaps at risk of necrosis, but there is paucity of cost-effectiveness data particularly in the Australian context. We evaluated its cost-effectiveness in breast reconstruction surgery. Single-institution retrospective study of 295 implant-based breast reconstructions using ICGA compared with 228 reconstructions without ICGA from 2015 to 2020. Costs were calculated using Medicare item numbers and micro-costing analysis. Break-even point analysis determined the number needed to break-even. Cost-utility analysis compared probabilities of ischaemic complications and utility estimates derived from surveys of surgeons to fit into a decision model. There were 295 breast reconstructions using ICGA with a total cost of AU$164,657. The average cost of treating an ischaemic complication was AU$21,375. Use of ICGA reduced the ischaemic complication rate from 14.9% to 8.8%. Ischaemic complications were prevented in 18 breasts resulting in gross cost savings of AU$384,745 and net savings of AU$220,088. Three hundred eighteen cases using ICGA are needed to break-even. The decision model demonstrated a baseline cost difference of AU$1,179, a quality-adjusted life-years (QALY) difference of 1.77, and an incremental cost-utility ratio (ICUR) of AU$656 per QALY favouring ICGA. Routine use of ICGA during implant-based breast reconstruction is a cost-effective intervention for the reduction of ischaemic complications in the Australian setting. ICGA use was associated with a gain of 1.77 additional years of perfect health at a cost of AU$656 more per year.

Cost-Utility of the CDK 4/6 Inhibitors for Postmenopausal Women With Luminal Advanced Breast Cancer in Brazil

Several trials have demonstrated the benefit of the CDK 4/6 inhibitors for postmenopausal women with luminal advanced breast cancer. This research aims to compare the cost-utility of the CDK 4/6 inhibitors in patients with no history of resistance to endocrine therapy. A Markov model was constructed to estimate the incremental cost per quality-adjusted life-years (QALYs) of treatments from the Brazilian public health system perspective over a lifetime horizon (30 years) with 5% annual discount rate for both benefits and costs. Efficacy parameters were extracted from the pivotal studies. Costs were based on open data from the Brazilian Ministry of Health. The utilities were calculated according to the overall population preferences from a British study. Deterministic and probabilistic sensitivity analyses evaluated the robustness of the results. The most cost-effective drug was ribociclib (US$50 748/QALY), followed by abemaciclib (US$64 052/QALY) and palbociclib (US$65 289/QALY). The univariate analysis showed that the incremental cost-utility ratio (ICUR) was mainly sensitive to the overall survival hazard ratio. The one thousand-probabilistic simulation showed that all ICUR values were above classical thresholds such as 1 to 3 gross domestic product (GDP) per capita per QALY. Even though there is no established willingness to pay threshold in Brazil, the estimated ICUR for CDK 4/6 inhibitors is >6 times the Brazilian GDP per capita (GDP per capita= US$5694.73), which might be a barrier to their inclusion in the Brazilian public health system.

Cost Utility of Prasugrel in Postangioplasty Diabetic Patients

ObjectivesTo prevent thrombotic events after angioplasty, current guidelines recommend dual antiplatelet therapy with aspirin and thienopyridine. Clopidogrel is the only thienopyridine currently available in the Brazilian National Health System. The purpose of this study was to determine the cost-effectiveness of prasugrel, an alternative thienopyridine, compared with clopidogrel in patients with acute coronary syndrome and diabetes mellitus who underwent angioplasty. MethodsA state-transition Markov model was created to simulate the progression of diabetic patients after angioplasty. The model had a lifetime horizon and discounted outcomes at a 5% annual rate. The risks of myocardial infarction and death were calculated using data from the diabetes subgroup, and the risks of bleeding were calculated using data from the overall group from the Trial to Assess Improvement in Therapeutic Outcomes by Optimizing Platelet Inhibition With Prasugrel Thrombolysis in Myocardial Infarction 38 trial. Direct costs were estimated using official Brazilian open data. Quality of life values were obtained through literature search. Univariate and multivariate sensitivity analyses were performed. ResultsPrasugrel was associated with more quality-adjusted life-years (QALYs) (5.03 vs 4.94) and higher costs (US$975.11 vs US$575.97), resulting in an incremental cost-utility ratio (ICUR) of US$4303.86/QALY. In one-way sensitivity analysis, the costs of prasugrel had the greatest impact on ICUR, followed by the initial age entering the cohort. In the probabilistic sensitivity analysis, all ICUR values simulations were less than one Brazilian gross domestic product per capita/QALY (US $5802.86). ConclusionsGiven the appealing economic profile, the clinical debate between reducing the risk of myocardial infarction and increasing the risk of bleeding may overcome economic concerns in the Brazilian National Health System.

Cost-effectiveness and value of information analyses of Bruton's tyrosine kinase inhibitors in the treatment of relapsed or refractory mantle cell lymphoma in the United States.

BACKGROUND: Ibrutinib, acalabrutinib, and zanubrutinib have shown improvements in efficacy and safety over conventional chemoimmunotherapy in refractory or relapsed mantle cell lymphoma (R/R MCL). OBJECTIVE: To evaluate the comparative cost-effectiveness of the Bruton's tyrosine kinase inhibitors (BTKIs) and estimate the expected value of (partial) perfect information (EV[P]PI) in terms of net health benefits (NHBs) and net monetary benefits (NMBs) forgone. METHODS: Using a two-state Markov model (progression-free; progression or death), we estimated in base-case and probabilistic sensitivity analyses (PSAs) the incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) of, respectively, progression-free survival (PFS) life-years (PFLYs) and PFS quality-adjusted LY (PFQALY) gained (g) against 3-year and 5-year time horizons. A willingness-to-pay threshold of $150,000/PFQALY was used to assess the probability of being cost-effective in the PSA. EVPI was calculated from the respective NHBs and NMBs. RESULTS: Compared with ibrutinib, acalabrutinib yielded a 3-year ICER of $90,571 (PSA = $88,588)/PFLYg and ICUR of $117,098 ($110,063)/PFQALYg, whereas zanubrutinib yielded a 3-year ICER of $58,422 ($58,907)/PFLYg and ICUR of $73,027 ($73,634)/PFQALYg. The corresponding 5-year estimates were ICER of $73,918 ($74,189)/PFLYg and ICUR of $90,512 ($90,844)/PFQALYg for acalabrutinib and ICER of $48,641 ($48,732)/PFLYg and ICUR of $61,612 ($63,727)/PFQALYg for zanubrutinib. Compared with zanubrutinib, treatment with acalabrutinib yielded a 3-year ICER of $144,633 ($134,964)/PFLYg and ICUR of $197,227 ($166,109)/PFQALYg; the corresponding 5-year estimates were $117,579 ($118,161)/PFLYg and $136,144 ($136,818)/PFQALYg. The EVPI/patient was an NHB of 0.036 PFQALYs and NMB of $3,602 forgone, resulting in a population EVPI of $134,766,957 forgone. The EVPPIs/patient for effectiveness were NHB of 0.015 PFQALYs and NMB of $1,479, with corresponding values of 0.032 and $3,187 for costs and 0.015 and $1,519 for health-related quality of life forgone. CONCLUSIONS: This early cost-effectiveness analysis based on phase I/II clinical trials of BTKIs in R/R MCL suggests an additional PFS benefit for second-generation BTKIs compared with ibrutinib. However, the relative uncertainty due to the lack of direct trial evidence may lead to an opportunity cost or lost health benefits if the current evidence is adopted to compare between these products. Additional evidence is needed to address the relative efficacy of the BTKIs. DISCLOSURES: A. McBride serves on speakers bureaus for Coherus BioSciences and Merck. He is now at Bristol-Myers Squibb in a position unrelated to this study. I. Abraham is joint equity owner in Matrix45. By company policy, owners and employees are prohibited from owning equity in client and sponsor organizations (except through mutual funds or other independently administered collective investment instruments), contracting independently with client and sponsor organizations, or receiving compensation independently from such organizations. Matrix45 provides similar services to biopharmaceutical, diagnostics, and medical device companies on a nonexclusivity basis. Of relevance to this article, Matrix45 has not provided any services to this study. I. Abraham is the quantitative methods editor of JAMA Dermatology and deputy editor-in-chief of the Journal of Medical Economics. The remaining authors have no relevant financial or nonfinancial interests to disclose.

Cost-Effectiveness Analysis of Fracture Liaison Services Compared with Standard of Care in the Secondary Prevention of Fragility Fractures in Spain.

PurposeTo assess the cost-effectiveness of a Fracture Liaison Service (FLS) compared with standard care for the secondary prevention of fragility fractures in Spain.MethodsPatients with osteoporosis and an initial fragility fracture who were candidates to initiate osteoporosis treatment (mean age 65 years, 90.7% female) were included in the model. Disease progression was simulated with a Markov model through seven health states (with and without osteoporosis treatment, subsequent hip, vertebral, forearm and humerus fracture, and death). A time horizon of 10 years and a 6-month duration per cycle was set. Clinical, economic, and quality of life parameters were estimated from the literature and Spanish clinical practice. Resource use and treatment patterns were validated by an expert panel. The Spanish National Health System (SNS) perspective was adopted, taking direct costs (€; 2020) into account. Effectiveness was measured in life-years gained (LYG) and quality-adjusted life years gained (QALYs). A discount rate of 3% was applied to costs and outcomes. The uncertainty of the parameters was assessed using deterministic, scenario and probabilistic sensitivity analyses (1000 iterations).ResultsSetting up a FLS for the secondary prevention of fragility fractures in Spain would provide better osteoporosis treatment initiation and persistence. This would reduce subsequent fragility fractures, disutilities and deaths. FLS would have greater clinical benefits (0.008 and 0.082 LYG and QALY gained per patient, respectively) and higher costs (€563.69 per patient) compared with standard care, leading to an incremental cost-utility ratio of €6855.23 per QALY gained over the 10 years horizon. The sensitivity analyses showed limited dispersion of the base case results, corroborating their robustness.ConclusionFrom the SNS perspective and considering Spanish willingness-to-pay thresholds, the introduction of FLS for the secondary prevention of fragility fractures would be a cost-effective strategy.

Cost-effectiveness and cost-utility of traditional and telemedicine combined population-based age-related macular degeneration and diabetic retinopathy screening in rural and urban China

SummaryBackgroundTo assess the cost-effectiveness and cost-utility of a population-level traditional and telemedicine combined age-related macular degeneration (AMD) and diabetic retinopathy (DR) screening program in rural and urban China.MethodsDecision-analytic Markov models were conducted to evaluate the costs and benefits of traditional and telemedicine combined AMD and DR screening from a societal perspective. A cohort of all participants aged 50 years old and above was followed through a total of 30 1-year Markov cycles. Separate analyses were performed for rural and urban settings. Relevant parameters such as the prevalence of AMD and DR, transition probability, compliance with screening and treatment, screening sensitivity, specificity, utility, and mortality were collected from published studies specific to China, other Asian counties' studies, or unpublished data sources such as the National Committee for the Prevention of Blindness. Costs of screening, full examination, and treatment come from the real medical environments and unified pricing of Beijing Municipal Medical Insurance Bureau. Primary outcomes were incremental cost-utility ratios (ICURs) using quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs) using years of blindness avoided. One-way deterministic and simulated probabilistic sensitivity analyses were conducted to reflect uncertainty.FindingsUnder the status quo, the total expected medical costs for a 50-year-old patient with AMD or DR were $869·59 and $1,514·18 in rural and urban settings, respectively. Both traditional and telemedicine screening were highly cost-effective. In rural settings, ICURs were $191 (95% confidence interval [CI]: $66 to $239) and $199 (95% CI: $-12 to $217), and ICERs were $2,436 (95% CI: $1,089 to $3,254) and $2,441 (95% CI: $1,452 to $3,900) for traditional and telemedicine screening separately. Even more surprising, both screening strategies dominated no screening in urban settings. Our results were insensitive and robust to extensive sensitivity analyses. Among all acceptable screening intervals (from 1 to 5 years), annual screening could not only produce biggest benefits but also keep ICERs less than three times and one time the per capita gross domestic product (GDP) in rural and urban settings separately. When compared with traditional screening, ICERs of telescreening were less than three times the per capita GDP in rural settings ($2,559 to $8,809) and less than one time the per capita GDP in urban settings (less than $5,564), annual telescreening produced the biggest benefits, it could avert 119 and 270 years of blindness in rural and urban areas separately when 100,000 people were screened.InterpretationWe performed decision-analytic Markov models for combined AMD and DR screening in rural and urban China, and the results showed that population-level combined screening for AMD and DR is likely to be highly cost-effective in both rural and urban China for people over 50 years old. Optimal screening may have an interval of every year based on teleophthalmology platforms. In the future, China should pay more attention to chronic eye diseases and the government should establish a sound chronic disease management system and make every patient enjoy equal medical services.FundingNational Natural Science Foundation of China, NSFC (82171051); the Major Innovation Platform of Public Health & Disease Control and Prevention, Renmin University of China and Beijing Nova program (Z191100001119072).

Cost Utility of Vaccination Against COVID-19 in Brazil

ObjectivesThe objective of this study was to evaluate the cost-utility of the Oxford, CoronaVac, and Janssen COVID-19 vaccines from the perspective of the Brazilian public health system. MethodsA total of 3 microsimulation models were constructed with individual data to evaluate the 3 vaccines. The simulation contains 7 transition states that are related to the natural history of the disease. The model with a daily cycle has a time horizon of 1 year and uses data from 289 days of the pandemic. The analysis was conducted from the perspective of the Brazilian public health system considering direct medical costs. For the model inputs, outpatient and hospital databases were used with information on treated patients stratified by age. Information on mortality was also stratified based on patients' age in the mortality database (SIM). The efficacy of vaccines to reduce the likelihood of patients becoming ill was evaluated independently for each vaccine. Information on the quality of life of patients in outpatient or hospital treatment and the sequelae resulting from the disease were extracted from the literature. The main outcome of the analysis was quality-adjusted life-years (QALYs). ResultsThe vaccines showed incremental cost-utility ratios ranging from R$−23 161.3/QALY (Oxford) to R$17 757.85/QALY (CoronaVac). The older the population, the lower was the incremental cost-utility ratio. Given a willingness-to-pay threshold of R$17 586/QALY, all the vaccines were considered cost-effective in the probabilistic sensitivity analysis. ConclusionsThe results of the analysis by age group can help in the preparation of a vaccination prioritization plan.

Clinical and economic impact of \u2018ROS1-testing\u2019 strategy compared to a \u2018no-ROS1-testing\u2019 strategy in advanced NSCLC in Spain

BackgroundDetection of the ROS1 rearrangement is mandatory in patients with advanced or metastatic non-small cell lung cancer (NSCLC) to allow targeted therapy with specific inhibitors. However, in Spanish clinical practice ROS1 determination is not yet fully widespread. The aim of this study is to determine the clinical and economic impact of sequentially testing ROS1 in addition to EGFR and ALK in Spain.MethodsA joint model (decision-tree and Markov model) was developed to determine the cost-effectiveness of testing ROS1 strategy versus a no-ROS1 testing strategy in Spain. Distribution of ROS1 techniques, rates of testing, positivity, and invalidity of biomarkers included in the analysis (EGFR, ALK, ROS1 and PD-L1) were based on expert opinion and Lungpath real-world database. Treatment allocation depending on the molecular testing results was defined by expert opinion. For each treatment, a 3-states Markov model was developed, where progression-free survival (PFS) and overall survival (OS) curves were parameterized using exponential extrapolations to model transition of patients among health states. Only medical direct costs were included (€ 2021). A lifetime horizon was considered and a discount rate of 3% was applied for both costs and effects. Both deterministic and probabilistic sensitivity analyses were performed to address uncertainty.ResultsA target population of 8755 patients with advanced NSCLC (non-squamous or never smokers squamous) entered the model. Over a lifetime horizon, the ROS1 testing scenario produced additional 157.5 life years and 121.3 quality-adjusted life years (QALYs) compared with no-ROS1 testing scenario. Total direct costs were increased up to € 2,244,737 for ROS1 testing scenario. The incremental cost-utility ratio (ICUR) was 18,514 €/QALY. Robustness of the base-case results were confirmed by the sensitivity analysis.ConclusionsOur study shows that ROS1 testing in addition to EGFR and ALK is a cost-effective strategy compared to no-ROS1 testing, and it generates more than 120 QALYs in Spain over a lifetime horizon. Despite the low prevalence of ROS1 rearrangements in NSCLC patients, the clinical and economic consequences of ROS1 testing should encourage centers to test all advanced or metastatic NSCLC (non-squamous and never-smoker squamous) patients.

Cost-Effectiveness of Brexucabtagene Autoleucel versus Best Supportive Care for the Treatment of Relapsed/Refractory Mantle Cell Lymphoma following Treatment with a Bruton's Tyrosine Kinase Inhibitor in Canada.

For patients with Mantle Cell Lymphoma (MCL), there is no recognized standard of care for relapsed/refractory (R/R) disease after treatment with a Bruton’s tyrosine kinase inhibitor (BTKi). Brexucabtagene autoleucel (brexu-cel) represents a promising new treatment modality in MCL. We explored whether brexu-cel was cost-effective for the treatment of R/R MCL. We developed a partitioned survival mixture cure approach to model the costs and outcomes over a lifetime horizon. The clinical data were derived from the ZUMA-2 clinical trial. The costs were estimated from the publicly available Canadian databases, published oncology literature, and pan-Canadian Oncology Drug Review economic guidance reports. The health state utilities were sourced from the ibrutinib submission to the National Institute for Health and Care Excellence for R/R MCL and supplemented with values from the published oncology literature. In the base case over a lifetime horizon, brexu-cel generated an incremental 9.56 life-years and an additional 7.03 quality-adjusted life-years compared to BSC, while associated with CAD 621,933 in additional costs. The resultant incremental cost-utility ratio was CAD 88,503 per QALY gained compared with BSC. Based on this analysis, we found brexu-cel to be a cost-effective use of healthcare resources relative to BSC for treatment of adult patients with R/R MCL previously treated with a BTKi in Canada, though additional research is needed to confirm these results using longer follow-up data.

Effectiveness and Cost-Utility Analysis of Different Doses of Irinotecan Plus Bevacizumab in Patients With Metastatic Colorectal Cancer: A Long-Term and Prospective Cohort Study.

ObjectivePatients with metastatic colorectal cancer (mCRC) had oncological benefits with irinotecan dose escalation of FOLFIRI regimen combined with bevacizumab according to UGT1A1 genotypes in our previous study. In the current study, we performed a quality of life (QOL) outcome evaluation and cost-utility analysis of different irinotecan dose regimens in patients with mCRC.Materials and MethodsWith inverse probability-of-treatment weighting (IPTW) matching on all covariates, 75 patients with dose escalation of irinotecan (study group) and 121 patients with the recommended dose of irinotecan (control group) were recruited between October 2015 and December 2019. The QOL outcome measures were Functional Assessment of Cancer Therapy-Colorectal, Beck Anxiety Inventory, Beck Depression Inventory, and SF-36; cost-utility outcome measures were medical direct costs, quality-adjusted life-years (QALYs), and incremental cost-utility ratios (ICURs).ResultsAll mCRC patients exhibited a significant decrease in both emotional wellbeing and depression from pretherapeutic period to posttherapeutic 6th month (P < 0.05); however, from the posttherapeutic 1st year to the 2nd year, improvement in most QOL measures was significantly better in the study group than in the control group (P < 0.05). Over a 2-year time period, the study group had higher total medical direct costs than the control group (US$ 54,742 ± 14,013 vs. US$ 54,608 ± 9,673) and higher average QALYs gained (1.88 vs. 1.65), with an ICUR of US$ 583 per QALY gained.ConclusionFor patients with mCRC, irinotecan dose escalation appeared cost-effective with considerable QOL improvements during the study period. Further randomized, multi-institutional controlled trials are warranted to corroborate these results.

A multicenter study to evaluate the disease burden and health economics of inpatients with multiple sclerosis in China

To assess the disease burden and health economics of inpatients with multiple sclerosis (MS) in China by evaluating the direct, indirect, and intangible costs. A total of 863 patients were included for a cross-sectional retrospective study in 50 centers. The direct economic burden was measured by the cost of hospitalization and out-of-hospital application drugs, and the indirect economic burden was measured by the human capital method. The disability-adjusted life year (DALY) was used to express the intangible economic burden. Cost-utility analysis (CUA) using DALYs as indicators of health benefits was performed by calculating the incremental cost-utility ratio. The mean direct economic burden/year, daily medication expenses/year, DALY, indirect economic burden, and indirect economic burden/year were 27,655.57 Yuan, 17,944.97 Yuan, 10.89 Yuan, 512,041.7 Yuan, and 11,299.85 Yuan, respectively. For the study period of two years, the direct economic burden, daily medication expense, and indirect economic burden were 48.6%, 31.5%, and 19.85% of the total economic burden, respectively. Disease burden and the number of episodes of remission were not statistically significant (p>0.001). The direct economic burden and total economic burden of the disease-modifying therapy (DMT) group were higher than those of the non-DMT group, but DALYs had no statistical significance (p>0.001). CUA showed that inpatients with MS in the DMT group received a DALY every time compared with the non-DMT group. The DALY losses are concentrated in young and middle-aged Chinese people. In this two-year study, CUA prompted the application of DMT drugs to increase the economic burden and DALYs. However, follow-up time is still short, and further follow-up observation is required.

Effectiveness of a multi-faceted intervention to deprescribe proton pump inhibitors in primary care: protocol for a population-based, pragmatic, cluster-randomized controlled trial

BackgroundInappropriately using proton pump inhibitors (PPI) is associated with severe adverse drug reactions and may have major consequences on healthcare costs. Deprescribing (the process by which a healthcare professional supervises the withdrawal of an inappropriate medication, to manage polypharmacy and improve outcomes) should be considered when an inappropriate PPI prescription is identified. Deprescribing interventions directed solely to prescribers have limited efficacy and are rarely targeted to patients. The aim of this trial is to assess the efficacy of a multi-faceted intervention with patients and general practitioners (GPs) to deprescribe PPI.MethodsWe will conduct a pragmatic, cluster-randomized, population-based, controlled trial in two regions of Western France. GPs with practices with over 100 patients, and their adult patient to whom over 300 defined daily doses (DDD) of PPIs have been dispensed in the year before baseline will be included. A total of 1300 GPs and 33,000 patients will be cluster-randomized by GPs practices. Three arms will be compared: i) a multi-faceted intervention associating a) a patient education brochure about PPI deprescribing sent directly to patients (the brochure was designed using a mixed-methods study), and b) a personalized letter with the Bruyere’s PPI deprescribing algorithm sent to their respective GPs, or ii) a single intervention where only the GPs received the letter and algorithm, or iii) no intervention.The primary outcome will be PPI deprescribing, defined as the proportion of patients achieving at least a 50% decrease in the amount of PPI dispensed to them (DDD/year) at 12 months compared to baseline. Secondary outcomes will include incremental cost-utility ratio (using EQ-5D-5L scale and National Health Insurance’s database), acid rebound (using the Gastroesophageal Reflux Disease Impact Scale), and the patients’ attitudes towards deprescribing (using the French rPATD).DiscussionBased on previous trials, we anticipate more than 10% “successful PPI deprescribing” in the multi-faceted intervention compared to the single intervention on GPs and the control arm. The study has been funded through a national grant and will be launched in autumn 2020, for early results by the end of 2022.Trial registrationClinicaltrials.gov NCT04255823; first registered on February 5, 2020.

Abstract P3-16-03: Clinical and pharmacoeconomic analyses of CDK4/6 inhibitors use in stage IV breast cancer females in the state of Qatar: A comparative retrospective observational study with cost effectiveness and cost utility analyses

Abstract Introduction: Cyclin-dependent kinase 4 and 6 (CDK4/6) inhibitors are indicated in the first-line treatment of hormonal receptor positive and HER-2 negative (HR+/HER2- negative) metastatic breast cancer (MBC). Although phase III randomized controlled trials (RCTs) proved their clinical efficacy, they can increase healthcare expenditure. To date, there are no observational studies to validate the clinical findings of the existed RCTs. In addition, only a few pharmacoeconomic evaluations were published regarding the two common CDK4/6 inhibiting agents, palbociclib and ribociclib to evaluate their financial burden. Objectives: To evaluate the clinical efficacy of palbociclib and ribociclib in the local settings in Qatar. Moreover, to conduct a thorough pharmacoeconomic analysis for the two medications and compare them in terms of their cost-effectiveness and cost-utility. Methodology: A retrospective observational study on HR+/HER-2 negative stage IV breast cancer patients receiving palbociclib or ribociclib in Qatar was conducted, followed by a comparative pharmacoeconomic analysis. The primary outcomes were progression-free survival (PFS) and overall-survival (OS). Safety profiles of both medications were also evaluated. Clinical data of all MBC in Qatar were collected from the National Center for Cancer Care and Research (NCCCR) from January 2017 to December 2019 using Cerner ® system. To evaluate the long-term costs-effectiveness, a 10-year within-cycle corrected Markov’s model was developed. The Markov’s model consisted of three main health states: progression-free (PFS), progressed disease (PD), and death. Incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratio (ICUR) were generated and compared to a three gross-domestic-product (3 GDP) per capita. Model costs were obtained from the department of accounting and finance in the NCCCR, transition probabilities were calculated from individual patient data obtained in the clinical phase, and utilities were summarized from the published literature. Deterministic and probabilistic sensitivity analyses were carried out. Modeling was conducted via TreeAge Pro ® software. Results: There was no statistically significant difference between palbociclib and ribociclib in PFS; median PFS time was 17.85 versus 13.55 months respectively; p&amp;gt; 0.05. Similarly, there was no statistically significant difference in OS (29.82 versus 31.72 months; p&amp;gt;0.05). For the pharmacoeconomic analysis, ribociclib dominated palbociclib in yielding an ICER of - 83,090.88 QAR per life-year gained, and ICUR of -31,868.06 QAR per quality-adjusted life-year gained. The results remained robust in all cases of the deterministic sensitivity analyses suggesting ribociclib is more cost-effective than palbociclib. Taking all combined uncertainties into account, the overall confidence in the base-case conclusion was around 60%. Conclusions: Both treatment strategies have similar efficacy and safety profiles. Nonetheless, ribociclib is a more cost-effective option than palbociclib based on the base-case results and based on the addressed uncertainties related to the model inputs. Citation Format: Nour Hisham Al-Ziftawi, Shereen Elazzazy, Mohammed Fasihul Alam, Asrul Akmal Shafie, Anas Hamad, Salha Bujassoum, Mohamed Izham Ibrahim. Clinical and pharmacoeconomic analyses of CDK4/6 inhibitors use in stage IV breast cancer females in the state of Qatar: A comparative retrospective observational study with cost effectiveness and cost utility analyses [abstract]. In: Proceedings of the 2021 San Antonio Breast Cancer Symposium; 2021 Dec 7-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2022;82(4 Suppl):Abstract nr P3-16-03.

Cost Effectiveness of Endovascular Revascularisation vs. Exercise Therapy for Intermittent Claudication Due to Iliac Artery Obstruction

To compare cost effectiveness of endovascular revascularisation (ER) and supervised exercise therapy (SET) as primary treatment for patients with intermittent claudication (IC) due to iliac artery obstruction. Cost utility analysis from a restricted societal perspective and time horizon of 12 months. Patients were included in a multicentre randomised controlled trial (SUPER study, NCT01385774, NTR2648) which compared effectiveness of ER and SET. Health status and health related quality of life (HRQOL) were measured using the Euroqol 5 dimensions 3 levels (EQ5D-3L) and VascuQol-25-NL. Incremental costs were determined per allocated treatment and use of healthcare during follow up. Effectiveness of treatment was determined in quality adjusted life years (QALYs). The difference between treatment groups was calculated by an incremental cost utility ratio (ICER). Some 240 patients were included, and complete follow up was available for 206 patients (ER 111 , SET 95). The mean costs for patients allocated to ER were €4 031 and €2 179 for SET, a mean difference of €1 852 (95% bias corrected and accelerated [bca] bootstrap confidence interval 1 185 - 2 646). The difference in QALYs during follow up was 0.09 (95% bcaCI 0.04 - 0.13) in favour of ER. The ICER per QALY was €20 805 (95% bcaCI 11 053 - 45 561). The difference in VascuQol sumscore was 0.64 (95% bcaCI 0.39 - 0.91), again in favour of ER. ER as a primary treatment, results in slightly better health outcome and higher QALYs and HRQOL during 12 months of follow up. Although these differences are statistically significant, clinical relevance must be discussed due to the small differences and relatively high cost of ER as primary treatment.