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.

Disutilities Associated with Intravenous Iron Infusions: Results from a Time Trade-off Survey and Diminishing Marginal Utility Model for Treatment Attributes in China.

Purpose: Treatment process attributes can affect health state utilities associated with therapy. For intravenous iron, used to treat iron deficiency and iron deficiency anemia, research into process attributes is still lacking. This study estimated utilities associated with process attributes for intravenous iron infusions. Methods: An online survey including seven health state vignettes and time trade-off tasks was administered to participants, who were not patients living with iron deficiency or iron deficiency anemia, from a Chinese online panel. Vignettes used an identical description of iron deficiency and iron deficiency anemia but differed in the annual number of infusions, infusion duration, and infusion-associated risk of hypophosphatemic osteomalacia. Disutilities and their rate of change as the number of infusions increased were examined using a power model. Results: The survey was completed by 1091 participants. The highest utilities were observed for one annual infusion of 15-30 minutes or 30-60 minutes, without risk of hypophosphatemic osteomalacia (0.754 and 0.746, respectively). In comparison, more infusions and infusions with a risk of hypophosphatemic osteomalacia were associated with lower utilities. Utility continued to decrease, but at a diminishing rate, as the annual number of infusions increased, with utility decrements of 0.006 and 0.002, respectively, when going from zero to one and from four to five infusions per year. All marginal disutilities were small (values <0.01). Conclusion: This study suggested that treatment attributes of intravenous iron infusions affect health state utilities. Using intravenous iron formulations that allow for fewer and shorter infusions without the risk of hypophosphatemic osteomalacia can reduce the number of visits required and increase patients' quality of life.

Intermediate and high-risk non-muscle-invasive bladder cancer: an overview of epidemiology, burden, and unmet needs.

Bladder cancer ranks among the most common cancers globally. At diagnosis, 75% of patients have non-muscle-invasive bladder cancer (NMIBC). Patients with low-risk NMIBC have a good prognosis, but recurrence and progression rates remain high in intermediate- and high-risk NMIBC, despite the decades-long availability of effective treatments for NMIBC such as intravesical Bacillus Calmette-Guérin (BCG). The present review provides an overview of NMIBC, including its burden and treatment options, and then reviews aspects that counteract the successful treatment of NMIBC, referred to as unmet treatment needs. The scale and reasons for each unmet need are described based on a comprehensive review of the literature, including insufficient adherence to treatment guidelines by physicians because of insufficient knowledge, training, or access to certain therapy options. Low rates of lifestyle changes and treatment completion by patients, due to BCG shortages or toxicities and adverse events as well as their impact on social activities, represent additional areas of potential improvement. Highly heterogeneous evidence for the effectiveness and safety of some treatments limits the comparability of results across studies. As a result, efforts are underway to standardize treatment schedules for BCG, but intravesical chemotherapy schedules remain unstandardized. In addition, risk-scoring models often perform unsatisfactorily due to significant differences between derivation and real-world cohorts. Reporting in clinical trials suffers from a lack of consistent outcomes reporting in bladder cancer clinical trials, paired with an under-representation of racial and ethnic minorities in many trials.

A systematic literature review and meta-analysis of the incidence of serious or severe hypersensitivity reactions after administration of ferric derisomaltose or ferric carboxymaltose.

BACKGROUND: Intravenous iron is the preferred treatment for patients with iron deficiency anemia in a variety of clinical situations. Although uncommon, administration of modern IV iron formulations can result in hypersensitivity reactions (HSRs) and, rarely, anaphylactic or anaphylactoid reactions. AIM: The objective of the present study was to systematically review the literature to identify and analyze data on the incidence of HSRs after administration of ferric derisomaltose (FDI) or ferric carboxymaltose (FCM). METHOD: A prospectively-registered systematic literature review was conducted to identify prospective randomized controlled trials comparing FDI and FCM with other intravenous iron formulations or oral iron. Searches were conducted in PubMed (including MEDLINE), EMBASE, and the Cochrane Library in November 2020. The relative incidence of serious or severe HSRs occurring on the day or day after dosing of intravenous iron, recorded under the standardized Medical Dictionary for Regulatory Activities query for anaphylactic reaction. RESULTS: Data were obtained from seven randomized controlled trials of FCM (N = 2683) and ten of FDI (N = 3474) enrolling 10,467 patients in total. The number of patients experiencing any serious or severe HSR event was 29/2683 (1.08%) with FCM versus 5/3474 with FDI (0.14%). Bayesian inference of proportions showed the event rates to be significantly lower with FDI relative to FCM. CONCLUSION: HSR events were uncommon with both intravenous iron formulations; however, the present study showed a significantly lower incidence of HSRs with FDI relative to FCM. Further large-scale, head-to-head trials of the iron formulations would be required to confirm this finding.

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.

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.

Modeling Chronic Kidney Disease in Type 2 Diabetes Mellitus: A Systematic Literature Review of Models, Data Sources, and Derivation Cohorts.

INTRODUCTION: As novel therapies for chronic kidney disease (CKD) in type 2 diabetes mellitus (T2DM) become available, their long-term benefits should be evaluated using CKD progression models. Existing models offer different modeling approaches that could be reused, but it may be challenging for modelers to assess commonalities and differences between the many available models. Additionally, the data and underlying population characteristics informing model parameters may not always be evident. Therefore, this study reviewed and summarized existing modeling approaches and data sources for CKD in T2DM, as a reference for future model development. METHODS: This systematic literature review included computer simulation models of CKD in T2DM populations. Searches were implemented in PubMed (including MEDLINE), Embase, and the Cochrane Library, up to October 2021. Models were classified as cohort state-transition models (cSTM) or individual patient simulation (IPS) models. Information was extracted on modeled kidney disease states, risk equations for CKD, data sources, and baseline characteristics of derivation cohorts in primary data sources. RESULTS: The review identified 49 models (21 IPS, 28 cSTM). A five-state structure was standard among state-transition models, comprising one kidney disease-free state, three kidney disease states [frequently including albuminuria and end-stage kidney disease (ESKD)], and one death state. Five models captured CKD regression and three included cardiovascular disease (CVD). Risk equations most commonly predicted albuminuria and ESKD incidence, while the most predicted CKD sequelae were mortality and CVD. Most data sources were well-established registries, cohort studies, and clinical trials often initiated decades ago in predominantly White populations in high-income countries. Some recent models were developed from country-specific data, particularly for Asian countries, or from clinical outcomes trials. CONCLUSION: Modeling CKD in T2DM is an active research area, with a trend towards IPS models developed from non-Western data and single data sources, primarily recent outcomes trials of novel renoprotective treatments.

The clinical effects of new treatments and their costs are often evaluated over a longer time frame than is possible in clinical trials by using computer simulation models. As new treatments are becoming available to treat chronic kidney disease, including in patients with type 2 diabetes, chronic kidney disease models may be used to inform clinical and economic decisions regarding these new treatment options. In the present study, we identified 49 published simulation models of chronic kidney disease used in populations with type 2 diabetes, and reviewed their structures and the data sources they used. The models focused mostly on disease states and outcomes associated with albuminuria (a condition in which the protein albumin is found in the urine) and end-stage kidney disease. Model structures with five disease states, including a kidney disease-free state, three kidney disease states, and death, were the most common. Relatively few models used glomerular filtration rates (a common measure of kidney function) or captured the possibility of an improvement in chronic kidney disease. Important data sources for many models were patient registries, cohort studies, and clinical trials, most conducted several decades ago in high-income countries with a high proportion of White participants. Several models developed in the past 5 years, particularly for Asian countries, instead relied largely or exclusively on country-specific data. In parallel, several individual patient simulations were recently developed from large outcomes trials for new treatments, including from trial subgroups covering specific geographical settings or ethnicities, shortly after trial publication.

Mixture Cure Models in Oncology: A Tutorial and Practical Guidance.

Novel cancer therapies are associated with survival patterns that differ from established therapies, which may include survival curves that plateau after a certain follow-up time point. A fraction of the patient population is then considered statistically cured and subject to the same mortality experience as the cancer-free general population. Mixture cure models have been developed to account for this characteristic. As compared to standard survival analysis, mixture cure models can often lead to profoundly different estimates of long-term survival, required for health economic evaluations. This tutorial is designed as a practical introduction to mixture cure models. Step-by-step instructions are provided for the entire implementation workflow, i.e., from gathering and combining data from different sources to fitting models using maximum likelihood estimation and model results interpretation. Two mixture cure models were developed to illustrate (1) an "uninformed" approach where the cure fraction is estimated from trial data and (2) an "informed" approach where the cure fraction is obtained from an external source (e.g., real-world data) used as an input to the model. These models were implemented in the statistical software R, with the freely available code on GitHub. The cure fraction can be estimated as an output from ("uninformed" approach) or used as an input to ("informed" approach) a mixture cure model. Mixture cure models suggest presumed estimates of long-term survival proportions, especially in instances where some fraction of patients is expected to be statistically cured. While this type of model may initially seem complex, it is straightforward to use and interpret. Mixture cure models have the potential to improve the accuracy of survival estimates for treatments associated with statistical cure, and the present tutorial outlines the interpretation and implementation of mixture cure models in R. This type of model will likely become more widely used in health economic analyses as novel cancer therapies enter the market.

Cost-effectiveness of physical activity interventions in cancer survivors of developed countries: a systematic review.

PURPOSE: Physical activity has been shown to improve survival and quality of life of cancer patients. Due to differences in patient populations, healthcare settings, and types of intervention, cost-effectiveness analyses of physical activity interventions in cancer survivors are difficult to compare. Available evidence from breast cancer survivor research has shown inconsistent results, and transfer of results to other types of cancer is not straightforward. This paper systematically reviewed current evidence on the cost-effectiveness of physical activity interventions in cancer survivors independent of cancer type compared to usual care or another experimental intervention. METHODS: The literature search was conducted in seven databases and enhanced by a search for gray literature. Eligible studies were restricted to developed countries and assessed using the CHEERS, CHEC, and PHILIPS checklists. The study protocol was pre-published in PROSPERO. RESULTS: Seven studies, five cost-utility, and two combined cost-utility/cost-effectiveness analyses fully met the inclusion criteria. They covered eight different types of cancer and various interventions. The cost-effectiveness analyses were of moderate to high methodological quality. A high probability of cost-effectiveness was reported in two analyses. One intervention appeared to be not cost-effective, and one to be cost-effective only from an organizational perspective. Three other analyses reported a cost-effectiveness better than US$ 101,195 (€ 80,000) per QALY gained. CONCLUSIONS: Physical activity interventions in cancer survivors of developed countries were cost-effective in some but not all clinical trials reviewed. IMPLICATIONS FOR CANCER SURVIVORS: Cost-effectiveness of physical activity interventions appear to depend upon the intensity of the activity.

Value For Money In The Treatment Of Patients With Type 2 Diabetes Mellitus: Assessing The Long-Term Cost-Effectiveness Of IDegLira Versus iGlarLixi In Italy.

OBJECTIVE: Italian treatment guidelines for type 2 diabetes mellitus (T2DM) target good glycemic control but acknowledge the associated risk of hypoglycemia. Unlike traditional antidiabetic therapies, modern treatment options such as fixed-ratio combinations of basal insulin and glucagon-like peptide 1 receptor agonists are associated with improved glycemic control, reduced body weight and low risk of hypoglycemia. The cost-effectiveness of the fixed-ratio combinations of basal insulin and glucagon-like peptide 1 receptor agonists IDegLira and iGlarLixi was assessed for Italy in patients with T2DM uncontrolled on basal insulin, to evaluate how short-term clinical benefits translate into long-term health economic outcomes. METHODS: The IQVIA CORE Diabetes Model was used to project clinical and economic outcomes over patient lifetimes. Treatment effects were sourced from an indirect treatment comparison. The analysis captured direct medical costs (expressed in 2017 Euros) from the perspective of the Italian National Health Service (NHS) and patient-related quality of life. Sensitivity analyses were performed. RESULTS: IDegLira was associated with gains of 0.09 life years and 0.13 quality-adjusted life years (QALYs) relative to iGlarLixi, due to a lower cumulative incidence and delayed onset of diabetes-related complications. IDegLira was associated with an incremental cost of EUR 930 over patient lifetimes, leading to an incremental cost-effectiveness ratio of EUR 7,386 per QALY gained. CONCLUSION: Over the lifetime of patients with T2DM uncontrolled on basal insulin, IDegLira was associated with improved clinical outcomes at higher costs relative to iGlarLixi. At a willingness-to-pay threshold of EUR 30,000 per QALY gained, IDegLira was considered to be cost-effective versus iGlarLixi from the perspective of the Italian NHS.

Cost-Effectiveness Analysis of the MiniMed 670G Hybrid Closed-Loop System Versus Continuous Subcutaneous Insulin Infusion for Treatment of Type 1 Diabetes.

BACKGROUND: Hybrid closed-loop (HCL) systems combine continuous glucose monitoring with continuous subcutaneous insulin infusion (CSII) to continuously self-adjust basal insulin delivery. Relative to CSII, HCL improves glycemic control and reduces the risk of hypoglycemia but has higher acquisition costs. The aim of this analysis was to assess the cost-effectiveness of the MiniMed™ 670G HCL system versus CSII in people with type 1 diabetes (T1D) in Sweden. METHODS: Cost-effectiveness analysis, from a societal perspective, was performed over patient lifetimes using the IQVIA CORE Diabetes Model. Clinical data were sourced from a study comparing the MiniMed 670G system with CSII in people with T1D. Cost data, expressed in 2018 Swedish krona (SEK), were obtained from Swedish reference prices and published literature. RESULTS: The MiniMed 670G system was associated with a quality-adjusted life-year (QALY) gain of 1.90 but higher overall costs versus CSII, leading to an incremental cost-effectiveness ratio (ICER) of SEK 164,236 per QALY gained. Use of the HCL system resulted in a lower cumulative incidence of diabetes-related complications. Higher HCL system acquisition costs were partially offset by reduced complication costs and productivity losses. In people with T1D poorly controlled at baseline, the MiniMed 670G system was associated with 2.25 incremental QALYs versus CSII, yielding an ICER of SEK 15,830 per QALY gained. CONCLUSIONS: The MiniMed 670G system was associated with clinical benefits and quality-of-life improvements in people with T1D relative to CSII. At a willingness-to-pay threshold of SEK 300,000 per QALY gained, this HCL system likely represents a cost-effective treatment option for people with T1D in Sweden.

Treating Patients with Type 2 Diabetes Mellitus Uncontrolled on Basal Insulin in the Czech Republic: Cost-Effectiveness of IDegLira Versus iGlarLixi.

INTRODUCTION: Few patients with type 2 diabetes mellitus (T2DM) achieve recommended glycemic control targets in the Czech Republic. Novel therapies, such as fixed-ratio combinations of basal insulin plus glucagon-like peptide-1 receptor agonists, may contribute to better glycemic control. In the analysis presented here, the present analysis assessed the long-term cost-effectiveness of two fixed-ratio combinations, IDegLira (insulin degludec/liraglutide) and iGlarLixi (insulin glargine/lixisenatide), for the treatment of patients with T2DM inadequately controlled with basal insulin from a healthcare payer perspective in the Czech Republic. METHODS: A cost-effectiveness analysis was performed over patient lifetimes using the IQVIA CORE Diabetes Model. Treatment effects were obtained from an indirect treatment comparison as no head-to-head data for IDegLira versus iGlarLixi are currently available. IDegLira was compared with two iGlarLixi pens (100 U/mL insulin glargine + 33 µg/mL and 50 µg/mL of lixisenatide, respectively). Direct medical costs associated with pharmaceutical interventions, screening and diabetes-related complications were captured. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: IDegLira was associated with gains in life expectancy of 0.11 years and in quality-adjusted life expectancy of 0.14 quality-adjusted life-years (QALYs) versus iGlarLixi, due to a lower cumulative incidence and delayed onset of diabetes-related complications. IDegLira was also associated with higher projected costs due to higher acquisition costs; however, these were partially offset by cost savings from avoided complications. IDegLira was associated with incremental cost-effectiveness ratios of Czech Koruna (CZK) 695,998 and CZK 348,323 per QALY gained versus iGlarLixi pens containing 33 and 50 µg/mL of lixisenatide, respectively. These ratios were below the commonly used willingness-to-pay threshold of CZK 1,200,000 per QALY gained. CONCLUSION: The present analysis indicated that IDegLira was associated with clinical benefits relative to iGlarLixi over patient lifetimes and was likely to be cost-effective in the treatment of patients with T2DM uncontrolled on basal insulin in the Czech Republic. FUNDING: Novo Nordisk. Plain language summary is available for this article.

Nasal Glucagon Versus Injectable Glucagon for Severe Hypoglycemia: A Cost-Offset and Budget Impact Analysis.

BACKGROUND: Severe hypoglycemic events (SHEs) in patients with diabetes are associated with substantial health care costs in the United States (US). Injectable glucagon (IG) is currently available for treatment of severe hypoglycemia but is associated with frequent handling errors. Nasal glucagon (NG) is a novel, easier-to-use treatment that is more often administered successfully. The economic impact of this usability advantage was explored in cost-offset and budget impact analyses for the US setting. METHODS: A health economic model was developed to estimate mean costs per SHE for which treatment was attempted using NG or IG, which differed only in the probability of treatment success, based on a published usability study. The budget impact of NG was projected over 2 years for patients with type 1 diabetes (T1D) and type 2 diabetes treated with basal-bolus insulin (T2D-BB). Epidemiologic and cost data were sourced from the literature and/or fee schedules. RESULTS: Mean costs were $992 lower if NG was used compared with IG per SHE for which a user attempted treatment. NG was estimated to reduce SHE-related spending by $1.1 million and $230 000 over 2 years in 10 000 patients each with T1D and T2D-BB, respectively. Reduced spending resulted from reduced professional emergency services utilization as successful treatment was more likely with NG. CONCLUSIONS: The usability advantage of NG over IG was projected to reduce SHE-related treatment costs in the US setting. NG has the potential to improve hypoglycemia emergency care and reduce SHE-related treatment costs.

IS THE EUNETHTA HTA CORE MODEL® FIT FOR PURPOSE? EVALUATION FROM AN INDUSTRY PERSPECTIVE.

OBJECTIVES: The HTA Core Model® was developed to improve the transferability of health technology assessment (HTA) between settings. The model has been used by HTA agencies but is also of interest to manufacturers, for improving internal evidence generation and communicating with other HTA stakeholders. To establish if the model is fit for purpose from an industry perspective, the pharmaceutical company Roche, collaborating with the European Network for HTA (EUnetHTA), conducted an assessment of the model. METHODS: A questionnaire was developed to evaluate all assessment elements in the HTA Core Model v2.0 for their usefulness in meeting payers' evidence needs and demonstrating value. The questionnaire was completed by country affiliate teams working in evidence generation and reimbursement submissions for pharmaceuticals. Survey results were discussed in workshops to ensure consistency and alignment between teams. RESULTS: The questionnaire was completed by six teams. An additional team from global pricing and market access participated in workshops. Model domains pertaining to the health problem and current technology use, technology description, clinical effectiveness, and economic value were considered most important because they meet payers' evidence needs. Overall, the model was considered useful to improve the efficiency of HTA evidence generation, share evidence internally, and communicate value to payers and HTA agencies. CONCLUSIONS: From an industry perspective, the HTA Core Model provides a useful framework and common terminology for efficient generation of transferable HTA evidence. The timeliness, efficiency, and transparency of HTA processes could be improved by a more standardized approach to HTA across settings.

Liraglutide Versus Lixisenatide: Long-Term Cost-Effectiveness of GLP-1 Receptor Agonist Therapy for the Treatment of Type 2 Diabetes in Spain.

INTRODUCTION: Glucagon-like peptide-1 (GLP-1) receptor agonists are used successfully in the treatment of patients with type 2 diabetes as they are associated with low hypoglycemia rates, weight loss and improved glycemic control. This study compared, in the Spanish setting, the cost-effectiveness of liraglutide 1.8 mg versus lixisenatide 20 µg, both GLP-1 receptor agonists, for patients with type 2 diabetes who had not achieved glycemic control targets on metformin monotherapy. METHODS: The IMS CORE Diabetes Model was used to project clinical outcomes and costs, expressed in 2015 Euros, over patient lifetimes. Baseline cohort data and treatment effects were taken from the 26-week, open-label LIRA-LIXI™ trial (NCT01973231). Treatment and management costs of diabetes-related complications were retrieved from published sources and databases. Future benefits and costs were discounted by 3% annually. Sensitivity analyses were conducted. RESULTS: Compared with lixisenatide 20 µg, liraglutide 1.8 mg was associated with higher life expectancy (14.42 vs. 14.29 years), higher quality-adjusted life expectancy [9.40 versus 9.26 quality-adjusted life years (QALYs)] and a reduced incidence of diabetes-related complications. Higher acquisition costs resulted in higher total costs for liraglutide 1.8 mg (EUR 42,689) than for lixisenatide 20 µg (EUR 42,143), but these were partly offset by reduced costs of treating diabetes-related complications (EUR 29,613 vs. EUR 30,636). Projected clinical outcomes and costs resulted in an incremental cost-effectiveness ratio of EUR 4113 per QALY gained for liraglutide 1.8 mg versus lixisenatide 20 µg. CONCLUSIONS: Long-term projections in the Spanish setting suggest that liraglutide 1.8 mg is likely to be cost-effective compared with lixisenatide 20 µg in type 2 diabetes patients who have not achieved glycemic control targets on metformin monotherapy. Liraglutide 1.8 mg presents a clinically and economically attractive treatment option in the Spanish setting.