Modelling the Costs of Sublingual Immunotherapy versus Subcutaneous Immunotherapy Based on Clinical Appointments and Impacts of Patient Travel in Sweden.

Aim: In Sweden, allergy immunotherapy (AIT) is available as either subcutaneous immunotherapy (SCIT) injections or sublingual immunotherapy (SLIT) tablets and is used to treat moderate-severe allergic rhinitis (AR). This study sought to determine direct and indirect annual costs stemming from treatment-related travel, appointments, waiting times and medication costs, before exploring likely CO2 emission-related cost-savings for 20,330 patients receiving SCIT or SLIT-tablets in Sweden. Methods: A model was developed in Python to capture each category of costs in the target patient population. Absenteeism costs arising from treatment-related travel were determined by obtaining average hourly pay data from Swedish Government sources. Absenteeism costs were also calculated for 30-minute post-dose observation times, which occurred during one clinical appointment for SLIT patients, and all clinical appointments for SCIT patients. Clinical appointment costs were obtained from healthcare price lists for Sweden. Medication costs were retrieved from the Pharmaceutical Specialities in Sweden (Fass) website, and treatment doses required for SCIT and SLIT-tablets were determined based on product labels and previously-calculated dosage regimes. High-cost protection and reimbursement scheme payment caps were applied when determining patient appointment and medication costs, respectively, and when identifying financial burdens for individual payers. Results: Mean total annual costs for SCIT were Swedish Krona (SEK) 604.1 million (m), with clinical appointments contributing the largest share of these costs (52.7%), followed by medication (34.4%), travel-related absenteeism (8.9%), waiting time-related absenteeism (2.7%) and private transportation (1.3%). Mean total annual costs for SLIT-tablets were SEK 336.2m. Medication contributed the most to these costs (72.3%), followed by clinical appointments (22.7%), travel-related absenteeism (3.8%), waiting time-related absenteeism (0.6%) and private transportation (0.6%). Conclusion: For patients with moderate-severe AR receiving AIT in Sweden, SLIT-tablets displayed large potential cost savings to patients, the healthcare system, and the government, whilst possessing reduced societal costs of carbon emissions relative to SCIT.

High costs, low quality of life, reduced survival, and room for improving treatment: an analysis of burden and unmet needs in glioma.

Gliomas are a group of heterogeneous tumors that account for substantial morbidity, mortality, and costs to patients and healthcare systems globally. Survival varies considerably by grade, histology, biomarkers, and genetic alterations such as IDH mutations and MGMT promoter methylation, and treatment, but is poor for some grades and histologies, with many patients with glioblastoma surviving less than a year from diagnosis. The present review provides an introduction to glioma, including its classification, epidemiology, economic and humanistic burden, as well as treatment options. Another focus is on treatment recommendations for IDH-mutant astrocytoma, IDH-mutant oligodendroglioma, and glioblastoma, which were synthesized from recent guidelines. While recommendations are nuanced and reflect the complexity of the disease, maximum safe resection is typically the first step in treatment, followed by radiotherapy and/or chemotherapy using temozolomide or procarbazine, lomustine, and vincristine. Immunotherapies and targeted therapies currently have only a limited role due to disappointing clinical trial results, including in recurrent glioblastoma, for which the nitrosourea lomustine remains the de facto standard of care. The lack of treatment options is compounded by frequently suboptimal clinical practice, in which patients do not receive adequate therapy after resection, including delayed, shortened, or discontinued radiotherapy and chemotherapy courses due to treatment side effects. These unmet needs will require significant efforts to address, including a continued search for novel treatment options, increased awareness of clinical guidelines, improved toxicity management for chemotherapy, and the generation of additional and more robust clinical and health economic evidence.

Cost-utility analysis of ferric derisomaltose versus ferric carboxymaltose in patients with inflammatory bowel disease and iron deficiency anemia in England.

AIMS: Anemia is the most common extraintestinal complication of inflammatory bowel disease (IBD), with approximately half of cases caused by iron deficiency (ID). Intravenous iron is the preferred ID anemia (IDA) treatment where oral iron is contraindicated, ineffective or not tolerated, or where ID correction is urgent. The objective was to evaluate the cost-utility of ferric derisomaltose (FDI) versus ferric carboxymaltose (FCM) in patients with IBD and IDA in England, in whom IV iron treatment is preferred. MATERIALS AND METHODS: A patient-level simulation model was developed, capturing quality of life (QoL) differences based on SF-36v2 data from the PHOSPHARE-IBD randomized controlled trial, monitoring and incidence of post-infusion hypophosphatemia, and number of iron infusions required. Analyses were conducted over a five-year time horizon from the Department of Health and Social Care (DHSC) perspective, with healthcare provider and societal perspectives adopted in separate analyses. Future costs and effects were discounted at 3.5% per annum and one-way and probabilistic sensitivity analyses were performed. RESULTS: FDI increased quality-adjusted life expectancy by 0.075 QALYs versus FCM from 2.57 QALYs to 2.65 QALYs per patient. Patients receiving FDI required 1.63 fewer iron infusions over the five-year time horizon, driving infusion-related cost savings of GBP 496 per patient (GBP 2,188 versus GBP 1,692) from the DHSC perspective. Costs of monitoring and treating hypophosphatemia after FCM were GBP 226, yielding total savings of GBP 722 per patient (GBP 2,414 versus GBP 1,692) over the five-year time horizon. FDI also led to reduced costs versus FCM in the societal and provider analyses and was therefore the dominant intervention across all three perspectives. LIMITATIONS: The analysis did not capture patient adherence, hypophosphatemic osteomalacia, or fractures. CONCLUSIONS: Results showed that FDI improved patient QoL and reduced direct healthcare expenditure versus FCM in patients with IBD and IDA in England.

Ferric derisomaltose (FDI) is an intravenous iron approved for the treatment of clinically diagnosed iron deficiency in the United Kingdom (UK), and can be an important therapeutic option for patients with inflammatory bowel disease (IBD), who require regular and rapid iron replenishment. Ferric carboxymaltose (FCM) is the sole alternative intravenous iron formulation available in the UK, but is associated with reduced blood phosphate levels, potentially causing fatigue and weakening of the bones. We conducted an economic analysis to weigh the costs and clinical outcomes associated with FDI and FCM in the UK, for patients with IBD and iron deficiency anemia (IDA). The main clinical difference we investigated was reduced blood phosphate levels, which occurred more often after FCM than FDI. We also incorporated recent quality of life data from a clinical study, and calculated the number of infusions (and associated costs) of each iron formulation, that patients would require over five years. Clinical data were obtained from published medical literature, while cost data came from UK sources including the 2022/2023 National Tariff Payment System and the British National Formulary. Our model showed that FDI was associated with quality of life improvements, fewer overall infusions per treatment course, and reduced costs compared to FCM, from the English Department of Health and Social Care perspective, the societal perspective, and the perspective of individual healthcare providers (namely NHS Trusts) within NHS England. FDI is therefore likely to represent the best value intravenous iron for the treatment of IDA with IBD in the UK.

Cost-utility of real-time continuous glucose monitoring versus self-monitoring of blood glucose in people with insulin-treated Type II diabetes in France.

Aim: Clinical trials and real-world data for Type II diabetes both show that glycated hemoglobin (HbA1c) levels and hypoglycemia occurrence can be reduced by real-time continuous glucose monitoring (rt-CGM) versus self-monitoring of blood glucose (SMBG). The present cost-utility study investigated the long-term health economic outcomes associated with using rt-CGM versus SMBG in people with insulin-treated Type II diabetes in France. Materials & methods: Effectiveness data were obtained from a real-world study, which showed rt-CGM reduced HbA1c by 0.56% (6.1 mmol/mol) versus sustained SMBG. Analyses were conducted using the IQVIA Core Diabetes Model. A French payer perspective was adopted over a lifetime horizon for a cohort aged 64.5 years with baseline HbA1c of 8.3% (67 mmol/mol). A willingness-to-pay threshold of €147,093 was used, and future costs and outcomes were discounted at 4% annually. Results: The analysis projected quality-adjusted life expectancy was 8.50 quality-adjusted life years (QALYs) for rt-CGM versus 8.03 QALYs for SMBG (difference: 0.47 QALYs), while total mean lifetime costs were €93,978 for rt-CGM versus €82,834 for SMBG (difference: €11,144). This yielded an incremental cost-utility ratio (ICUR) of €23,772 per QALY gained for rt-CGM versus SMBG. Results were particularly sensitive to changes in the treatment effect (i.e., change in HbA1c), annual price and quality of life benefit associated with rt-CGM, SMBG frequency, baseline patient age and complication costs. Conclusion: The use of rt-CGM is likely to be cost-effective versus SMBG for people with insulin-treated Type II diabetes in France.

A European Cost-Utility Analysis of the MiniMed™ 780G Advanced Hybrid Closed-Loop System Versus Intermittently Scanned Continuous Glucose Monitoring with Multiple Daily Insulin Injections in People Living with Type 1 Diabetes.

Background: Advanced hybrid closed-loop (AHCL) automated insulin delivery systems are the most effective therapy in terms of assisting people with type 1 diabetes (T1D) to achieve glycemic targets; however, the cost can represent a barrier to uptake. In this study, a cost-utility analysis of the MiniMed™ 780G AHCL system (MM780G) versus intermittently scanned continuous glucose monitoring (is-CGM) plus multiple daily insulin injections (MDI) in people with T1D not achieving glycemic goals was performed across six European countries. Methods: Clinical input data were sourced from the ADAPT trial. Assuming a baseline HbA1c of 9.04%, HbA1c reductions of 1.54% for AHCL and 0.2% for is-CGM+MDI were modeled. The analyses were performed from a payer perspective over a time horizon of 40 years and an annual discount rate of 3% was applied. Results: Across all countries, the use of AHCL was projected to result in an incremental gain in quality-adjusted life expectancy of >2 quality-adjusted life years (QALYs) versus is-CGM+MDI. Lifetime direct costs were higher with AHCL resulting in incremental cost-utility ratios for AHCL versus is-CGM+MDI ranging from EUR 11,765 per QALY gained in Austria to EUR 43,963 per QALY gained in Italy. Conclusions: For people with T1D managed with is-CGM+MDI not achieving glycemic targets, initiation of the MM780G system was projected to improve long-term clinical outcomes; however, due to differences in health care costs between countries, the health economic outcomes differed. In all included countries, AHCL is likely to be cost-effective relative to is-CGM+MDI for people not achieving glycemic goals with is-CGM+MDI. The ADAPT trial is registered with ClinicalTrials.gov, NCT04235504.

Cost-utility of real-time continuous glucose monitoring versus self-monitoring of blood glucose in people with insulin-treated Type 2 diabetes in Canada.

Aim: Clinical trials and real-world data for Type 2 diabetes have shown that real-time continuous glucose monitoring (rt-CGM) lowers glycated hemoglobin (A1c) and reduces hypoglycemia relative to self-monitoring of blood glucose (SMBG). This analysis examined the long-term health and economic outcomes associated with using rt-CGM versus SMBG in people with insulin-treated Type 2 diabetes in Canada. Materials & methods: Clinical data were sourced from a real-world study, in which rt-CGM reduced A1C by 0.56% versus continued SMBG. The analysis was performed using the IQVIA Core Diabetes Model, from a Canadian payer perspective over a lifetime horizon for a cohort aged 65 years with an A1C of 8.3% at baseline. Future costs and clinical outcomes were discounted at 1.5% annually. Results: Projected total mean lifetime costs were CAD 207,466 for rt-CGM versus CAD 189,863 for SMBG (difference: CAD 17,602) and projected mean quality-adjusted life expectancy was 9.97 quality-adjusted life years (QALYs) for rt-CGM versus 9.02 QALYs for SMBG (difference: 0.95 QALYs), resulting in an incremental cost-utility ratio (ICUR) of CAD 18,523 per QALY gained for rt-CGM versus SMBG. Findings were sensitive to changes in the A1C treatment effect, annual cost and quality of life benefit associated with using rt-CGM, SMBG frequency, and baseline age, but ICURs remained below CAD 50,000 per QALY in all analyses. Conclusion: For people in Canada with insulin-treated Type 2 diabetes and poor glycemic control, use of rt-CGM is likely to be cost-effective relative to SMBG.

Cost-utility of real-time continuous glucose monitoring versus self-monitoring of blood glucose and intermittently scanned continuous glucose monitoring in people with type 1 diabetes receiving multiple daily insulin injections in Denmark.

AIMS: To determine the cost-effectiveness of the Dexcom G6 real-time continuous glucose monitoring (rt-CGM) system compared with both the self-monitoring of blood glucose (SMBG) and the Abbott FreeStyle Libre 1 and 2 intermittently scanned CGM (is-CGM) devices in people with type 1 diabetes receiving multiple daily insulin injections in Denmark. MATERIALS AND METHODS: The analysis was performed using the IQVIA Core Diabetes Model, which associates rt-CGM use with glycated haemoglobin reductions of 0.6% and 0.36% based on data from the DIAMOND and ALERTT1 trials, respectively, compared with SMBG and is-CGM use. The analysis was performed from the payer perspective over a 50-year time horizon; future costs and clinical outcomes were discounted at 4% per annum. RESULTS: The use of rt-CGM was associated with an incremental gain of 1.37 quality-adjusted life years (QALYs) versus SMBG. Total mean lifetime costs were Danish Krone (DKK) 894 535 for rt-CGM and DKK 823 474 for SMBG, resulting in an incremental cost-utility ratio of DKK 51 918 per QALY gained versus SMBG. Compared with is-CGM, the use of rt-CGM led to a gain of 0.87 QALYs and higher mean lifetime costs resulting in an incremental cost-utility ratio of DKK 40 879 to DKK 34 367 per QALY gained. CONCLUSIONS: In Denmark, the rt-CGM was projected to be highly cost-effective versus both SMBG and is-CGM, based on a willingness-to-pay threshold of 1× per capita gross domestic product per QALY gained. These findings may help inform future policies to address regional disparities in access to rt-CGM.

Evaluating the cost-utility of intravesical Bacillus Calmette-Guérin versus radical cystectomy in patients with high-risk non-muscle-invasive bladder cancer in the UK.

AIMS: Approximately 75% of bladder cancer (BC) cases present as non-muscle-invasive BC (NMIBC). In patients with high-risk NMIBC, the mainstay treatment is intravesical Bacillus Calmette-Guérin (BCG), with immediate radical cystectomy (RC) as an alternative treatment option. The aim of the present study was to evaluate the cost-utility of BCG versus RC in patients with high-risk NMIBC from the UK healthcare payer perspective. MATERIALS AND METHODS: A six-state Markov model was developed that covered controlled disease, recurrence, progression to muscle-invasive BC, metastatic disease, and death. The model included adverse events of BCG and RC and monitoring and palliative care. Drug costs were obtained from the British National Formulary. Intravesical delivery, RC, and monitoring costs were sourced from the National Tariff Payment System and the literature. Utility data were obtained from the literature. Analyses were run over a 30-year time horizon, with future costs and effects discounted at 3.5% per annum. One-way and probabilistic sensitivity analyses were performed. RESULTS: The base case analysis comparing BCG with RC showed that BCG would increase life expectancy by 0.88 years versus RC, from 7.74 to 8.62 years. BCG resulted in an increase of 0.76 quality-adjusted life years (QALYs) versus RC, from 5.63 to 6.39 QALYs. Patients incurred lower lifetime costs if treated with BCG (£47,753) than with RC (£64,264). Cost savings were mainly driven by the lower cost of BCG versus RC, and palliative care costs. Sensitivity analyses showed that results were robust to assumptions. LIMITATIONS: The evidence base informing efficacy estimates of BCG is heterogeneous as different BCG administration schedules were reported in the literature, while incidence and cost data on some BCG-associated adverse events were sparse. CONCLUSIONS: Intravesical BCG led to increased QALYs and reduced costs versus RC for patients with high-risk NMIBC from the UK healthcare payer perspective.

Intravesical Bacillus Calmette-Guérin (BCG) is a recommended immunotherapy option for patients with high-risk non-muscle invasive bladder cancer (NMIBC) who have undergone transurethral resection of bladder tumor (TURBT). BCG is known for its high efficacy and good safety profile. However, current evidence on its effectiveness against other comparators such as radical cystectomy (RC) is limited, mainly because different BCG schedules are used, particularly with regard to maintenance therapy. Given this lack of evidence, we conducted the first cost-utility analysis which considers adequate BCG therapy relative to RC for the UK. We developed a Markov model that captured the effects of the intravesical BCG and RC on NMIBC, in addition to incidences of adverse events associated with either treatment. Costs of the two treatments, their administration, maintenance, and of treatments for adverse events were modelled alongside the quality-of-life effects of NMIBC, adverse events, and palliative care. We used published clinical data and UK cost data to inform our model. Our results show that BCG was associated with higher quality-adjusted life expectancy than RC and lower total costs from the healthcare system perspective. These results imply that adequate BCG immunotherapy is likely valuable for the National Healthcare System in terms of its effects on patients and indeed cost-saving relative to RC.

A Cost-Utility Analysis of SQ® Tree SLIT-Tablet versus Placebo in the Treatment of Birch Pollen Allergic Rhinitis from a Swedish Societal Perspective.

Background and Aims: Allergic rhinitis (AR) is an immunoglobulin E antibody-mediated inflammatory condition that arises in response to inhaled allergens such as pollen. Pollens from trees in the birch homologous group are the most common allergenic tree pollens in Northern and Central Europe and North America. SQ® Tree SLIT-Tablet (ITULAZAX®) is a sublingual immunotherapy tablet indicated for moderate-to-severe AR and/or conjunctivitis induced by pollen from the birch homologous group. The present analysis evaluated the cost-utility of treating adults with AR with SQ Tree SLIT-Tablet versus placebo, both in combination with symptom-relieving medications, from a Swedish societal perspective. Methods: A model was developed to evaluate changes in cost and quality of life associated with using SQ Tree SLIT-Tablet relative to placebo in an adult population of individuals with AR. The model captured costs associated with symptom-relieving medications, healthcare professional interactions, SQ Tree SLIT-Tablet, and indirect costs arising from absenteeism and reduced workplace productivity. The analysis was conducted over 10 years with costs captured in 2021 Swedish Krona (SEK) and future costs and effects discounted at 3% per annum. One-way and probabilistic sensitivity analyses were conducted. Results: Treatment with SQ Tree SLIT-Tablet resulted in an improvement of 0.041 quality-adjusted life years (QALYs) over 10 years versus placebo. From a Swedish societal perspective, costs increased by SEK 9077 over the same period, resulting in an incremental cost-utility ratio of SEK 223,445 per QALY gained. One-way sensitivity analysis showed that the model was most sensitive to assumptions around the disease-modifying effect of SQ Tree SLIT-Tablet. Conclusion: SQ Tree SLIT-Tablet improved quality of life in moderate-to-severe AR and/or conjunctivitis induced by pollen from the birch homologous group in Sweden, with only a modest increase in societal costs over a medium-term time horizon, representing good value for money at a willingness-to-pay threshold of SEK 700,000 per QALY.

Advances in Delivery of Selective Internal Radiation Therapy (SIRT): Economic and Logistical Effects of Same-Stay Work-Up and Procedure in the Treatment of Unresectable Liver Tumors in England.

INTRODUCTION: Selective internal radiation therapy (SIRT) is a targeted method of treatment for unresectable liver tumors in which radiation therapy is directly delivered to the tumor(s) via the hepatic vasculature. Successful outcomes with SIRT are dependent on the specific vasculature of the liver and tumor, and the patient therefore needs to attend a "work-up" to map the hepatic vasculature prior to the SIRT procedure. Recent advances in SIRT delivery have enabled same-day or same-stay work-up and procedure, requiring only one hospital visit rather than two. We aimed to evaluate the economic, travel time, and transport-related environmental impact of a new brachytherapy device delivery program, the order-map-treat (OMT) program, in patients with unresectable hepatocellular carcinoma (HCC) in England. METHODS: A healthcare resource group (HRG)-based analysis of costs from a national payer (Department of Health and Social Care, DHSC) perspective was conducted assuming that, with OMT, patients would have to attend hospital only once for both the SIRT work-up and procedure versus twice without OMT. Patient travel time and CO2 emissions were then estimated by identifying the SIRT center closest to the centroid of each clinical commissioning group (CCG) and calculating straight-line distances with a "detour index" to capture the effect of indirect routes via road or rail. RESULTS: It was estimated that 856 patients per annum would be eligible for SIRT treatment for unresectable HCC in England. OMT would be anticipated to save GBP 2842 per patient versus performing SIRT without OMT. Furthermore, across all patients with HCC eligible for SIRT in England, OMT would avoid 74,500 km of travel, 2299 h of travel time, and 13.9 metric tons of patient transport-related CO2 emissions annually. CONCLUSION: OMT reduces the number of hospital visits required for SIRT by 50%, resulting in financial savings from the DHSC perspective, time savings from the patient perspective, and reduced CO2 emissions arising from patient transport.

ITULAZAX® versus Alutard SQ® in the treatment of allergic rhinitis induced by pollen from the birch homologous group: A cost-minimization modeling analysis from the Danish societal perspective.

BACKGROUND AND AIMS: Allergic rhinitis (AR) is an inflammatory disorder triggered by an allergic immune response to inhaled allergens. Birch pollen is the major allergenic tree pollen in parts of Europe. ITULAZAX® is a sublingual immunotherapy tablet for the treatment of adults with moderate-to-severe AR and/or conjunctivitis induced by pollen from the birch homologous group. The aim was to compare the costs of treating AR with ITULAZAX® versus subcutaneous ALUTARD SQ® Betula verrucosa (ALUTARD SQ®) from a Danish societal perspective. METHODS: A cost-minimization model was developed to capture costs of allergy immunotherapy (AIT), interactions with healthcare professionals (HCPs) in three different care settings (general practice, allergy specialist, and hospital), and indirect costs arising from absenteeism and presenteeism. The cost-minimization analysis was conducted over a 3-year time horizon with costs reported in 2021 Danish Kroner (DKK) and Euros (EUR) based on the European Central Bank 365-day average exchange rate. One-way sensitivity analyses were performed. RESULTS: The base case analysis showed that the total cost of treatment over 3 years was estimated to be DKK 49,117 (EUR 6598) per patient with ALUTARD SQ®, compared with DKK 30,996 (EUR 4164) with ITULAZAX®, reflecting a cost saving of DKK 18,121 (EUR 2434) per patient with ITULAZAX® over 3 years. Over the 3-year time horizon, costs of AIT were predicted to increase by DKK 17,928 (EUR 2408) with ITULAZAX®, while costs of interactions with HCPs were predicted to decrease by DKK 22,528 (EUR 3027) versus ALUTARD SQ®, more than offsetting the increased cost of ITULAZAX®. CONCLUSIONS: Given the equivalent effectiveness of the two AIT products, and the cost savings with ITULAZAX® versus ALUTARD SQ® from a Danish societal perspective, ITULAZAX® should be considered as a cost-saving alternative to ALUTARD SQ® for the treatment of birch pollen-induced moderate-to-severe AR in adults.

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.

Ferric derisomaltose (FDI) was approved in February 2021 for the treatment of iron deficiency anemia (IDA) in China and allows for fast iron correction in one visit with a good safety profile. The current standard of care in China is iron sucrose (IS). Clinical and economic decision-making can benefit from having longer-term projections on the benefits and costs of new medications relative to the current standard of care, which is why we conducted the first cost-utility analysis of FDI and IS for China. We developed a patient-level model that captured the effects of the iron formulations on IDA, in addition to incidences of adverse events and hypersensitivity reactions (HSRs) associated with either formulation. Costs of the iron formulations, their administration, and of treatments for adverse events and HSR were modeled alongside the quality of life effects of IDA, adverse events, HSRs, and iron infusions. We used published clinical data and Chinese cost data to inform our model. Our results show that FDI was associated with higher quality-adjusted life expectancy than IS, regardless of the perspective of the analysis, and higher total costs from the healthcare system perspective. From a societal perspective, FDI was associated with lower costs due to reduced travel and waiting time and smaller productivity losses given there were fewer appointments. These results imply that FDI is likely good value for money for the healthcare system and indeed cost-saving for society relative to IS, which has so far been the most widely used IV iron treatment in China.

The PRIME Type 2 Diabetes Model: a novel, patient-level model for estimating long-term clinical and cost outcomes in patients with type 2 diabetes mellitus.

BACKGROUND AND AIMS: The growing burden of diabetes mellitus and recent progress in understanding cardiovascular outcomes for type 2 diabetes (T2D) patients continue to make the disease a priority for healthcare decision-makers around the world. Our objective was to develop a new, product-independent model capable of projecting long-term clinical and cost outcomes for populations with T2D to support health economic evaluation. METHODS: Following a systematic literature review to identify longitudinal study data, existing T2D models and risk formulae for T2D populations, a model was developed (the PRIME Type 2 Diabetes Model [PRIME T2D Model]) in line with good practice guidelines to simulate disease progression, diabetes-related complications and mortality. The model runs as a patient-level simulation and is capable of simulating treatment algorithms and risk factor progression, and projecting the cumulative incidence of macrovascular and microvascular complications as well as hypoglycemic events. The PRIME T2D Model can report clinical outcomes, quality-adjusted life expectancy, direct and indirect costs, along with standard measures of cost-effectiveness and is capable of probabilistic sensitivity analysis. Several approaches novel to T2D modeling were utilized, such as combining risk formulae using a weighted model averaging approach that takes into account patient characteristics to evaluate complication risk. RESULTS: Validation analyses comparing modeled outcomes with published studies demonstrated that the PRIME T2D Model projects long-term patient outcomes consistent with those reported for a number of long-term studies, including cardiovascular outcomes trials. All root mean squared deviation (RMSD) values for internal validations (against published studies used to develop the model) were 1.1% or less and all external validation RMSDs were 3.7% or less. CONCLUSIONS: The PRIME T2D Model is a product-independent analysis tool that is available online and offers new approaches to long-standing challenges in diabetes modeling and may become a useful tool for informing healthcare policy.HIGHLIGHTSThe PRIME Type 2 Diabetes (T2D) Model is a new, product-independent simulation model.The model offers new approaches to long-standing challenges in diabetes modeling.PRIME T2D Model projects outcomes consistent with those from clinical trials.The model is designed to be a useful tool for informing healthcare policy in T2D.

Effects of Trial Population Selection on Quality of Life and Healthcare Decision-Making: A Systematic Review and Example in the Treatment of Hepatocellular Carcinoma with Radioembolization.

BACKGROUND AND AIMS: Quality of life is among the most important considerations in the treatment of hepatocellular carcinoma (HCC), arguably second only to overall survival. Measuring and modeling patient quality of life is also crucial in the evaluation of the cost-effectiveness of health interventions. In the present study, we aimed to identify cost-utility analyses comparing selective internal radiation therapy (SIRT) with systemic therapy in patients with unresectable HCC and to compare the modeled incremental quality of life differences between the two therapies. METHODS: A systematic literature review was conducted. PubMed, EMBASE, the Cochrane Library, and health technology assessment agency websites were searched to identify cost-utility studies of SIRT versus systemic therapies in the treatment of HCC. Key characteristics of the studies, modeled populations and incremental quality of life outcomes were extracted from the included studies. RESULTS: The systematic literature review retrieved 1140 studies, of which four were ultimately included. Hand searches then identified two distinct analyses, and an updated version of one of the four studies identified initially. From these seven studies, 18 analyses were included. Analyses using data from the overall trial populations reported incremental quality-of-life estimates spanning -0.09 to +0.28 quality-adjusted life years (QALYs), with that range expanding to -0.09 to +0.60 QALYs when also considering post hoc sub-group analyses. CONCLUSION: The wide range of incremental QALYs, with substantial differences between overall trial populations and subgroups, illustrates the impact that the choice of target population may have on the relative quality of life outcomes of the compared interventions, which may in turn affect clinical decision-making. The small differences also highlight both the importance of reporting measures of dispersion around the findings, and the limitations of the incremental cost-effectiveness ratio (ICER) for assessing the relative cost-effectiveness of interventions that are predicted to result in similar quality-of-life outcomes.

An Economic Analysis of Ferric Derisomaltose versus Ferric Carboxymaltose in the Treatment of Iron Deficiency Anemia in Patients with Inflammatory Bowel Disease in Norway, Sweden, and Finland.

BACKGROUND AND AIMS: Iron deficiency anemia (IDA) is a common sequela of inflammatory bowel disease (IBD), arising from the combined effects of gastrointestinal blood loss and reduced iron absorption. Given this, intravenous (IV) iron should be considered as the first-line treatment in patients with clinically active IBD. The present study evaluated the budget impact of administering IV iron with ferric derisomaltose (FDI) versus ferric carboxymaltose (FCM) in patients with IDA and IBD in Norway, Sweden, and Finland. METHODS: A cohort level model of iron need was developed using a bivariate distribution of hemoglobin and bodyweight based on observational data from a multi-country Scandinavian study of patients with IDA and IBD. The base case analysis was conducted over 5 years in patients with IDA with mean bodyweight of 75.4 kg (SD 17.5 kg) and hemoglobin levels of 10.77 g/dL (SD 1.43 g/dL). Infusion costs were modeled using diagnosis-related groups. Sensitivity analyses were performed around different patient characteristics, care settings, and retreatment frequencies, and probabilistic sensitivity analyses were conducted. RESULTS: Using FDI required 1.25 infusions to correct the mean iron deficit, compared with 1.64 infusions with FCM. In Norway, the per-patient cost of iron replenishment over 5 years was estimated to be NOK20,767 with FCM versus NOK15,799 with FDI, reflecting a cost saving of NOK4,968 or 23.9%. In Finland, costs were projected to decrease from EUR3075 with FCM to EUR2339 with FDI, reflecting a cost saving of EUR736 per patient. In Sweden, costs decreased from SEK27,760 with FCM to SEK21,119 with FDI. CONCLUSION: Using FDI in place of FCM resulted in a substantial reduction in the number of infusions required to correct iron deficits in patients with IDA and IBD. The reduction in infusions was accompanied by substantial cost savings relative to FCM over 5 years across all three Nordic countries evaluated.

Lower Drug Cost of Successfully Treating Patients with Type 2 Diabetes to Targets with Once-Weekly Semaglutide versus Once-weekly Dulaglutide in Japan: A Short-Term Cost-Effectiveness Analysis.

INTRODUCTION: In the head-to-head trial (SUSTAIN 7), the novel, injectable, once-weekly GLP-1 analogue semaglutide showed superiority in both glycemic outcomes and body weight reduction, compared with once-weekly dulaglutide in the treatment of type 2 diabetes (T2D). However, no economic evaluation using these data has yet been conducted in the Japanese setting. The objective of this analysis was to assess the short-term cost-effectiveness in Japan of once-weekly semaglutide 0.5 mg (the approved maintenance dose in Japan) compared with once-weekly dulaglutide 0.75 mg (the only licensed dose in Japan) over a 1-year period using Japanese cost data. METHODS: Responder endpoints were obtained from the SUSTAIN 7 trial to assess the cost of successfully treating patients to these targets ("cost of control"). Responder endpoint definitions consisted of single, dual, and triple composite endpoints related to glycemic control, body weight, and hypoglycemia outcomes. The cost of treatment was accounted from a healthcare payer perspective, capturing drug costs only. RESULTS: Treatment with once-weekly semaglutide 0.5 mg was associated with a lower cost and a lower cost per patient treated to target for all endpoints, compared with once-weekly dulaglutide 0.75 mg. For each JPY 1 spent on bringing patients to target with once-weekly semaglutide 0.5 mg, JPY 1.58, JPY 1.44, JPY 1.60, JPY 2.10, and JPY 2.33 would need to be spent on once-weekly dulaglutide 0.75 mg to achieve an equivalent outcome for endpoints of HbA1c ≤ 6.5%, HbA1c < 7.0%, HbA1c < 7.0% without hypoglycemia, and no weight gain, weight loss ≥ 5%, and ≥ 1.0% HbA1c reduction and ≥ 3.0% weight loss, respectively. CONCLUSIONS: These findings suggest that once-weekly semaglutide is a cost-effective treatment option compared with once-weekly dulaglutide for patients with T2D in Japan. In the future, this finding should be extrapolated to traditional long-term cost-effectiveness analysis, using common outcomes such as quality-adjusted life years.

A patient-level cost-effectiveness analysis of iron isomaltoside versus ferric carboxymaltose for the treatment of iron deficiency anemia in the United Kingdom.

Objectives: Intravenous iron is the recommended treatment for patients with iron deficiency anemia (IDA) where oral iron is ineffective or rapid iron replenishment is required. Two high-dose, rapid-administration intravenous iron formulations are currently available in the UK: iron isomaltoside 1000/ferric derisomaltose (IIM) and ferric carboxymaltose (FCM). An indirect treatment comparison (ITC) recently showed that improvement from baseline hemoglobin was significantly larger with IIM than FCM. The objective was to use the ITC findings to evaluate the cost-effectiveness of IIM versus FCM from the UK healthcare payer perspective.Methods: A patient-level simulation model was developed in R to evaluate the cost per patient experiencing hematological response with IIM versus FCM. The model generated a simulated cohort from parametric distributions of baseline hemoglobin and bodyweight. Changes in hemoglobin were modeled based on data from the ITC, covaried with baseline hemoglobin based on patient-level data from a randomized controlled trial. Posological models of the iron formulations were developed based on the summaries of product characteristics. UK-specific costs were based on healthcare resource groups.Results: The proportion of patients experiencing hematological response was 9.0% higher with IIM relative to FCM (79.0% versus 70.0%), based on modeling of clinically realistic, correlated distributions of baseline hemoglobin and change from baseline hemoglobin. The mean number of infusions needed to administer the required dose was 1.92 with FCM, versus 1.38 with IIM, resulting in costs of £637 and £457 per treated patient with FCM and IIM respectively, corresponding to respective costs of £910 and £579 per responder.Conclusions: The analysis showed that using IIM rather than FCM in patients with IDA was dominant and would reduce the number of iron infusions required to correct iron deficiency, thereby reducing the costs associated with IDA treatment and simultaneously increasing the proportion of patients with IDA experiencing a clinically meaningful hematological response.

A cost analysis of SIR-Spheres yttrium-90 resin microspheres versus tyrosine kinase inhibitors in the treatment of unresectable hepatocellular carcinoma in France, Italy, Spain and the UK.

Background and aims: A wide range of treatment options are available for hepatocellular carcinoma (HCC), including systemic treatment with tyrosine kinase inhibitors (TKIs) such as sorafenib and lenvatinib, immunotherapies, locoregional therapies such as selective internal radiation therapy (SIRT) and treatments with curative intent such as resection, radiofrequency ablation and liver transplantation. Given the substantial economic burden associated with HCC treatment, the aim of the present analysis was to establish the cost of using SIRT with SIR-Spheres yttrium-90 (Y-90) resin microspheres versus TKIs from healthcare payer perspectives in France, Italy, Spain and the United Kingdom (UK).Methods: A cost model was developed to capture the costs of initial systemic treatment with sorafenib (95%) or lenvatinib (5%) versus SIRT in patients with HCC in Barcelona Clinic Liver Cancer (BCLC) stages B and C. A nested Markov model was utilized to model transitions between progression-free survival (PFS), progression and death, in addition to transitions between subsequent treatment lines. Cost and resource use data were identified from published sources in each of the four countries.Results: Relative to TKIs, SIRT with SIR-Spheres Y-90 resin microspheres were found to be cost saving in all four country settings, with the additional costs of the microspheres and the SIRT procedure being more than offset by reductions in drug and drug administration costs, and treatment of adverse events. Across the four country settings, total cost savings with SIR-Spheres Y-90 resin microspheres fell within the range 5.4-24.9% and SIRT resulted in more patients ultimately receiving treatments with curative intent (4.6 vs. 1.4% of eligible patients).Conclusion: SIR-Spheres Y-90 resin microspheres resulted in cost savings relative to TKIs in the treatment of unresectable HCC in all four country settings, while increasing the proportion of patients who become eligible for treatments with curative intent.

Development of a Resource Impact Model for Clinics Treating Pre-Operative Iron Deficiency Anemia in Ireland.

INTRODUCTION: Intravenous (IV) iron is typically the preferred treatment for patients with iron deficiency anemia (IDA) who cannot tolerate or absorb oral iron, or who require fast replenishment of iron stores pre-operatively. Several IV iron formulations are available with different dosing characteristics affecting infusion speed and maximum dose. The aim was to develop a resource impact model to calculate the cost of establishing an IV iron clinic and model resource impact of different IV irons to inform clinicians and service providers implementing innovative pre-operative IV iron services in Ireland. METHODS: A resource impact tool was developed to model resource utilization and IDA treatment costs. Two fast-administration, high-dose formulations of IV iron are available in Ireland: iron isomaltoside 1000/ferric derisomaltose (IIM) and ferric carboxymaltose (FCM). The tool modeled clinic throughput based on their different dosing characteristics in a specific IDA population, capturing fixed overheads, variable costs, clinic income from private and publicly-funded patients, and savings associated with IV iron. RESULTS: Based on a 70:30 split between public and private patients in a new pre-operative service with capacity for 12 infusion slots weekly, IIM would facilitate correction of iron deficits in 474 patients annually, resulting in a net annual clinic balance of €42,736 on income of €159,887 and net costs of €117,151. FCM would facilitate treatment of 353 patients, resulting in a net annual clinic balance of €36,327 on income of €116,050 and costs of €79,722, a difference of €6408 and 121 patients treated in favor of using IIM over FCM. CONCLUSION: Based on this provider-perspective analysis, IIM would maximize clinic throughput relative to other IV iron formulations, allowing clinicians in Ireland to optimize their current service provision and expenditure, and model the impact of introducing IV iron clinics for pre-operative patients with IDA.