Systematic Literature Review of Studies Reporting Measures of Functional Outcome or Quality of Life in People with Negative Symptoms of Schizophrenia.

Aim: Negative symptoms of schizophrenia (NSS) have been linked with poor functional outcomes. A literature review was performed to identify instruments used to assess functional outcomes and quality of life in clinical trials and observational studies conducted in groups of people with NSS. Methods: Literature search strings were designed using Medical Subject Headings combined with free-text terms and searches were performed using the PubMed, Embase and the Cochrane Library databases. For inclusion, articles were required to be published as full-text articles, in English, over the period 2011-2021, include at least one group or treatment arm of people with NSS and report either functional outcomes or quality of life (QoL). Results: Literature searches identified a total of 3,268 unique hits. After two rounds of screening, 37 publications (covering 35 individual studies) were included in the review. A total of fourteen different instruments were used to assess functional outcomes and eleven different instruments were used to assess QoL. In studies in people with NSS, the most frequently used functional outcome measures were the Personal and Social Performance scale and the Global Assessment of Functioning. The most frequently used QoL instruments included the Manchester Short Assessment of Quality of Life, the Heinrich Carpenter Quality of Life Scale, the Schizophrenia Quality of Life Scale and the EQ-5D. Conclusion: A large number of measures have been used to assess functional outcomes and QoL in people with NSS, these include both generic and condition-specific as well as both interviewer-administered and self-reported instruments.

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.

Evaluation of the Long-Term Cost-Effectiveness of the Dexcom G6 Continuous Glucose Monitor versus Self-Monitoring of Blood Glucose in People with Type 1 Diabetes in Canada.

BACKGROUND: The Dexcom G6 real-time continuous glucose monitoring (RT-CGM) system is one of the most sophisticated RT-CGM systems developed to date and became available in Canada in 2019. A health economic analysis was performed to determine the long-term cost-effectiveness of the Dexcom G6 RT-CGM system versus SMBG in adults with type 1 diabetes (T1D) in Canada. METHODS: The analysis was performed using the IQVIA Core Diabetes Model. Based on clinical trial data, patients with mean baseline HbA1c of 8.6% were assumed to have a HbA1c reduction of 1.0% with RT-CGM versus 0.4% reduction with SMBG. RT-CGM was also associated with a quality of life (QoL) benefit owing to reduced incidence of hypoglycemia, reduced fear of hypoglycemia (FoH) and elimination of fingerstick testing. Direct medical costs were sourced from published literature, and inflated to 2019 Canadian dollars (CAD). RESULTS: Dexcom G6 RT-CGM was projected to improve mean quality-adjusted life expectancy by 2.09 quality-adjusted life years (QALYs) relative to SMBG (15.52 versus 13.43 QALYs) but mean total lifetime cots were CAD 35,353 higher with RT-CGM (CAD 227,357 versus CAD 192,004) resulting in an incremental cost-effectiveness ratio (ICER) of CAD 16,931 per QALY gained. Sensitivity analyses revealed that assumptions relating to the QoL benefit associated with reduced FoH and the elimination of fingerstick testing with RT-CGM as well as SMBG usage and change in HbA1c were the key drivers of cost-effectiveness. CONCLUSION: For adults with T1D in Canada, RT-CGM is associated with improved glycemic control and QoL benefits owing to a reduced FoH and elimination of the requirement for fingerstick testing and over a lifetime time horizon is cost-effective relative to SMBG.

Long-Term Cost-Effectiveness the Dexcom G6 Real-Time Continuous Glucose Monitoring System Compared with Self-Monitoring of Blood Glucose in People with Type 1 Diabetes in France.

INTRODUCTION: The aim was to determine the long-term cost-effectiveness of the Dexcom G6 real-time continuous glucose monitoring (RT-CGM) system versus self-monitoring of blood glucose (SMBG) in adults with type 1 diabetes (T1D) in France. METHODS: The analysis was performed using the IQVIA Core Diabetes Model and utilized clinical input data from the DIAMOND clinical trial in adults with T1D. Simulated patients were assumed to have a mean baseline HbA1c of 8.6%, and those in the RT-CGM arm were assumed to have a HbA1c reduction of 1.0% compared with 0.4% in the SMBG arm. A quality of life (QoL) benefit associated with a reduced fear of hypoglycemia (FoH) and elimination of the requirement for fingerstick testing in the RT-CGM arm was also applied. RESULTS: The G6 RT-CGM system was associated with an incremental gain in quality-adjusted life expectancy of 1.38 quality-adjusted life years (QALYs) compared with SMBG (10.64 QALYs versus 9.23 QALYs). Total mean lifetime costs were 21,087 euros higher with RT-CGM (148,077 euros versus 126,990 euros), resulting in an incremental cost-effectiveness ratio of 15,285 euros per QALY gained. CONCLUSIONS: In France, based on a willingness-to-pay threshold of 50,000 euros per QALY gained, the use of the G6 RT-CGM system is cost-effective relative to SMBG for adults with long-standing T1D, driven primarily by improved glycemic control and the QoL benefit associated with reduced FoH and elimination of the requirement for fingerstick testing.

Cost-effectiveness of lutetium (177Lu) oxodotreotide vs everolimus in gastroenteropancreatic neuroendocrine tumors in Norway and Sweden.

BACKGROUND: Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) represent a relatively rare and heterogenous group of tumors. Currently available treatment options for patients with progressive GEP-NETs include lutetium (177Lu) oxodotreotide (177Lu-Dotatate) and everolimus [as well as sunitinib for patients with pancreatic NETs (P-NETs)]. AIM: To perform a health economic analysis to determine the cost-effectiveness of 177Lu-Dotatate compared with everolimus in patients with unresectable or metastatic midgut-NETs or P-NETs in both Sweden and Norway. METHODS: Simulations were performed using a three-state partitioned survival model and analyses were performed separately for patients with midgut-NETs and P-NETs. Clinical input data were sourced from an indirect comparison that utilized survival data from clinical trials of 177Lu-Dotatate and everolimus. The analyses were performed from the healthcare payer perspective over a time horizon of 20 years. For Sweden, future costs and clinical outcomes were discounted at 3% per annum. For Norway, a discount rate of 4% per annum was applied. RESULTS: For Sweden, improved survival outcomes and higher lifetime costs with 177Lu-Dotatate resulted in an incremental cost-effectiveness ratio (ICER) of SEK 391194 per quality-adjusted life year (QALY) gained for midgut NETs and SEK 16764 per QALY gained for P-NETs for 177Lu-Dotatate compared with everolimus. For Norway, the corresponding ICERs were NOK 244444 per QALY gained and NOK 106451 per QALY gained, respectively. One-way sensitivity analyses revealed that the results were most sensitive to changes in drug acquisition costs and health state utility values. CONCLUSION: In both Sweden and Norway, from a healthcare provider perspective, 177Lu-Dotatate is likely to be considered cost-effective relative to everolimus for the treatment of patients with unresectable or metastatic, progressive midgut-NETs or P-NETs.

Long-term Cost-Effectiveness of Dexcom G6 Real-time Continuous Glucose Monitoring Versus Self-Monitoring of Blood Glucose in Patients With Type 1 Diabetes in the U.K.

OBJECTIVE: A long-term health economic analysis was performed to establish the cost-effectiveness of real-time continuous glucose monitoring (RT-CGM) (Dexcom G6) versus self-monitoring of blood glucose (SMBG) alone in U.K.-based patients with type 1 diabetes (T1D). RESEARCH DESIGN AND METHODS: The analysis used the IQVIA CORE Diabetes Model. Clinical input data were sourced from the DIAMOND trial in adults with T1D. Simulations were performed separately in the overall population of patients with baseline HbA1c ≥7.5% (58 mmol/mol), and a secondary analysis was performed in patients with baseline HbA1c ≥8.5% (69 mmol/mol). The analysis was performed from the National Health Service health care payer perspective over a lifetime time horizon. RESULTS: In the overall population, G6 RT-CGM was associated with a mean incremental gain in quality-adjusted life expectancy of 1.49 quality-adjusted life years (QALYs) versus SMBG (mean [SD] 11.47 [2.04] QALYs versus 9.99 [1.84] QALYs). Total mean (SD) lifetime costs were also pounds sterling (GBP) 14,234 higher with RT-CGM (GBP 102,468 [35,681] versus GBP 88,234 [39,027]) resulting in an incremental cost-effectiveness ratio of GBP 9,558 per QALY gained. Sensitivity analyses revealed that the findings were sensitive to changes in the quality-of-life benefit associated with reduced fear of hypoglycemia and avoidance of fingerstick testing as well as the HbA1c benefit associated with RT-CGM use. CONCLUSIONS: For U.K.-based T1D patients, the G6 RT-CGM device is associated with significant improvements in clinical outcomes and, over patient lifetimes, is a cost-effective disease management option relative to SMBG on the basis of a willingness-to-pay threshold of GBP 20,000 per QALY gained.

Impact of Stigma on People Living with Chronic Hepatitis B.

BACKGROUND: People with chronic infectious diseases such as hepatitis B can face stigma, which can influence everyday life as well as willingness to engage with medical professionals or disclose disease status. A systematic literature review was performed to characterize the level and type of stigma experienced by people infected with hepatitis B virus (HBV) as well as to identify instruments used to measure it. METHODS: A literature review was performed using the PubMed, Embase and Cochrane Library databases to identify studies describing HBV-related stigma. For inclusion, articles were required to be published in full-text form, in English and report quantitative or qualitative data on HBV-related stigma that could be extracted. RESULTS: A total of 23 (17 quantitative and 6 qualitative) articles examined HBV-related stigma. The scope of the review was global but nearly all identified studies were conducted in countries in the WHO Southeast Asia or Western Pacific regions or within immigrant communities in North America. Several quantitative studies utilized tools specifically designed to assess aspects of stigma. Qualitative studies were primarily conducted via patient interviews. Internalized and social stigma were common among people living with chronic HBV . Some people also perceived structural/institutional stigma, with up to 20% believing that they may be denied healthcare and up to 30% stating they may experience workplace discrimination due to HBV. CONCLUSION: HBV-related stigma is common, particularly in some countries in Southeast Asia and the Western Pacific region and among Asian immigrant communities, but is poorly characterized in non-Asian populations. Initiatives are needed to document and combat stigma (particularly in settings/jurisdictions where it is poorly described) as well as its clinical and socioeconomic consequences.

Cost-Effectiveness of Sensor-Augmented Insulin Pump Therapy Versus Continuous Insulin Infusion in Patients with Type 1 Diabetes in Turkey.

Background and Aims: Sensor-augmented pump therapy (SAP) combines continuous glucose monitoring with continuous subcutaneous insulin infusion (CSII). SAP is costlier than CSII but provides additional clinical benefits relative to CSII alone. A long-term cost-effectiveness analysis was performed to determine whether SAP is cost-effective relative to CSII in patients with type 1 diabetes (T1D) in Turkey. Methods: Analyses were performed in two different patient cohorts, one with poor glycemic control at baseline (mean glycated hemoglobin 9.0% [75 mmol/mol]) and a second cohort considered to be at increased risk of hypoglycemic events. Clinical input data and direct medical costs were sourced from published literature. The analysis was performed from a third-party payer perspective over patient lifetimes and future costs and clinical outcomes were discounted at 3.5% per annum. Results: In both patient cohorts, SAP was associated with a gain in quality-adjusted life expectancy but higher costs relative to CSII (incremental gain of 1.40 quality-adjusted life years [QALYs] in patients with poor baseline glycemic control and 1.73 QALYs in patients at increased risk of hypoglycemic events). Incremental cost-effectiveness ratios for SAP versus CSII were TRY 76,971 (EUR 11,612) per QALY gained for patients with poor baseline glycemic control and TRY 69,534 (EUR 10,490) per QALY gained for patients at increased risk for hypoglycemia. Conclusions: SAP is associated with improved long-term clinical outcomes versus CSII, and in Turkey, SAP is likely to represent good value for money compared with CSII in T1D patients with poor glycemic control and/or with frequent severe hypoglycemic events.

Cost-effectiveness of bevacizumab plus paclitaxel versus paclitaxel for the first-line treatment of HER2-negative metastatic breast cancer in specialist oncology centers in France.

BACKGROUND: Evidence from clinical trials suggests that the addition of bevacizumab to chemotherapy in the first-line treatment of patients with HER2-negative metastatic breast cancer improves progression-free survival (PFS) but not overall survival (OS). However, a retrospective analysis of real-world data from the French Comprehensive Cancer Centers (FCCC) through the Epidemiological Strategy and Medical Economics (ESME) Research Program, suggested that in this setting, the addition of bevacizumab may confer a significant benefit in terms of both PFS and OS. A cost-effectiveness analysis was performed to determine the cost-effectiveness of bevacizumab plus paclitaxel versus paclitaxel alone in the first-line treatment of HER2-negative metastatic breast cancer at specialist oncology centers in France. METHODS: The analysis was performed using a three-state Markov model and clinical input data from N = 3426 HER2-negative metastatic breast cancer patients treated with bevacizumab plus paclitaxel or paclitaxel alone. The analysis was performed from a third party payer perspective over a 10-year time horizon; future costs and clinical outcomes were discounted at 4% per annum. RESULTS: In the overall population, the addition of bevacizumab to paclitaxel led to incremental gain of 0.72 life years and 0.48 quality-adjusted life years (QALYs) relative to paclitaxel alone. The incremental lifetime cost of the addition of bevacizumab was EUR 27,390, resulting in an incremental cost-effectiveness ratio (ICER) of EUR 56,721 per QALY gained for bevacizumab plus paclitaxel versus paclitaxel alone. In a subgroup of triple negative patients the ICER was EUR 66,874 per QALY gained. CONCLUSIONS: The analysis indicated that the combination of bevacizumab plus paclitaxel is likely to be cost-effective compared with paclitaxel alone for the first-line treatment of HER2-negative metastatic breast cancer in specialized oncology centers in France.

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.

Cost-Effectiveness of Continuous Subcutaneous Insulin Infusion Versus Multiple Daily Injections in Patients with Poorly Controlled Type 2 Diabetes in Finland.

INTRODUCTION: Although primarily utilized in type 1 diabetes, continuous subcutaneous insulin infusion (CSII) represents a useful treatment alternative for patients with type 2 diabetes who are unable to achieve good glycemic control despite optimization of multiple daily injections (MDI). The aim of the analysis reported here was to investigate the long-term cost-effectiveness of CSII versus MDI in type 2 diabetes patients with poor glycemic control in Finland. METHODS: The IQVIA CORE Diabetes Model was used to make long-term projections of the clinical and economic outcomes associated with CSII use in type 2 diabetes, based on clinical input data from the OpT2mise trial, which showed that CSII was associated with a 1.1% decrease in glycated hemoglobin (HbA1c) in patients with poor glycemic control at baseline. The analysis was performed from a societal perspective and the time horizon was that of patient lifetimes. Future costs and clinical outcomes were discounted at 3% per annum. RESULTS: Continuous subcutaneous insulin infusion was associated with a gain in quality-adjusted life expectancy of 0.32 quality-adjusted life-years (QALYs) compared with MDI (8.15 vs. 7.83 QALYs, respectively), as well as higher mean lifetime costs, resulting in an incremental cost-effectiveness ratio of Euro (EUR) 47,834 per QALY gained for CSII versus MDI. The higher treatment costs in the CSII group were partly mitigated by a 15% reduction in diabetes-related complication costs. Sensitivity analyses demonstrated that CSII was most cost-effective in patients with the highest baseline HbA1c values. CONCLUSION: In Finland, CSII is likely to represent a cost-effective treatment alternative for patients with type 2 diabetes with poor glycemic control despite optimization of MDI. In such patients, CSII is associated with improved clinical outcomes relative to MDI, with the higher acquisition costs partly offset by a lower lifetime incidence and cost of diabetes-related complications. FUNDING: Medtronic International Sàrl.

Cost-effectiveness of sensor-augmented insulin pump therapy vs continuous subcutaneous insulin infusion in patients with type 1 diabetes in the Netherlands.

AIM: The aim of this study was to perform a cost-effectiveness analysis to establish the cost-effectiveness of sensor-augmented pump therapy (SAP) with automated insulin suspension vs continuous subcutaneous insulin infusion (CSII) alone in patients with type 1 diabetes in the Netherlands. PATIENTS AND METHODS: The analysis was performed using the IQVIA CORE Diabetes Model (CDM) in two different patient cohorts: one with suboptimal glycemic control at baseline (mean age 27 years, mean baseline HbA1c 8.0% [64 mmol/mol]) and the other at increased risk of hypoglycemic events (mean age 18.6 years, mean baseline HbA1c 7.5% [58 mmol/mol]). Clinical input data were sourced from published literature, and the analysis was performed from the societal perspective. RESULTS: In patients with suboptimal baseline glycemic control, SAP improved quality-adjusted life expectancy by 1.77 quality-adjusted life years (QALYs) vs CSII (15.54 QALYs vs 13.77 QALYs) with higher lifetime costs (EUR 189,855 vs EUR 150,366), resulting in an incremental cost-effectiveness ratio (ICER) of EUR 22,325 per QALY gained. In this cohort, sensitivity analyses showed that the influence of SAP on fear of hypoglycemia (FoH) and baseline HbA1c were key drivers of results. In patients at increased risk of hypoglycemia, the gain in quality-adjusted life expectancy with SAP vs CSII was 2.16 QALYs (16.70 QALYs vs 14.53 QALYs) with higher lifetime costs (EUR 204,013 vs EUR 171,032) leading to an ICER of EUR 15,243 per QALY gained. In this patient group, findings were most sensitive to changes in assumptions relating to the incidence of severe hypoglycemic events in the CSII arm. CONCLUSION: For type 1 diabetes patients in the Netherlands who do not achieve target HbA1c levels or who experience frequent severe hypoglycemic events on CSII, switching to SAP is likely to be cost-effective.

Cost-Effectiveness Analysis of Sensor-Augmented Insulin Pump Therapy with Automated Insulin Suspension Versus Standard Insulin Pump Therapy in Patients with Type 1 Diabetes in Sweden.

INTRODUCTION: In Sweden an estimated 10,000 people with type 1 diabetes use continuous subcutaneous insulin infusion (CSII). Sensor-augmented pump therapy (SAP) is associated with higher acquisition costs but provides additional clinical benefits (e.g. reduced rate of hypoglycemic events) over and above that of CSII alone. The aim of the analysis was to assess the cost-effectiveness of SAP with automated insulin suspension relative to CSII alone in two different groups of patients with type 1 diabetes in Sweden. METHODS: Cost-effectiveness analyses were performed using the QuintilesIMS CORE Diabetes Model, with clinical and economic input data derived from published literature. Separate analyses were performed for patients at increased risk of hypoglycemia and for patients with uncontrolled glycated hemoglobin (HbA1c) at baseline. Analyses were performed from a societal perspective over a lifetime time horizon. Future costs and clinical outcomes were discounted at 3% per annum. RESULTS: SAP with automated insulin suspension was associated with an incremental gain in quality-adjusted life expectancy versus the CSII of 1.88 quality-adjusted life years (QALYs) in patients at high risk of hypoglycemia and of 1.07 QALYs in patients with uncontrolled HbA1c at baseline. Higher lifetime costs for SAP with automated insulin suspension resulted in projected incremental cost-effectiveness ratios for the SAP with automated insulin suspension versus CSII of Swedish Krona (SEK) 139,795 [euros (EUR) 14,648] per QALY gained for patients at increased risk for hypoglycemia and SEK 251,896 (EUR 26,395) per QALY gained for patients with uncontrolled HbA1c. In both groups, SAP with automated insulin suspension also reduced the incidence of diabetes-related complications relative to CSII. CONCLUSIONS: In Sweden, SAP with automated insulin suspension likely represents a cost-effective treatment option relative to CSII for the management of patients with type 1 diabetes with a history of severe hypoglycemic events or patients who struggle to achieve good glycemic control despite the use of CSII. FUNDING: Medtronic International Trading Sàrl.

Evaluating health-related quality of life in type 1 diabetes: a systematic literature review of utilities for adults with type 1 diabetes.

BACKGROUND AND AIMS: Type 1 diabetes is a chronic condition associated with micro- and macrovascular complications that have a notable impact on health-related quality of life, the magnitude of which can be quantified via the use of utility values. The aim of this review was to conduct a systematic literature review to identify and compare published health state utility values for adults with type 1 diabetes both, with and without diabetes-related complications. METHODS: Literature searches of the PubMed, EMBASE, and Cochrane Library databases were performed to identify English language studies on adults with type 1 diabetes, published from 2000 onward, reporting utility values for patients with or without diabetes-related complications or assessing the impact of changes in HbA1c or body mass index on quality of life. For inclusion, studies were required to report utilities elicited using validated methods. RESULTS: A total of 20 studies were included in the final review that included utility values elicited using the EuroQuol five dimensions questionnaire (n=9), 15D questionnaire (n=2), Quality of Well-Being scale (n=4), time trade-off (n=3), and standard gamble (n=2) methods. For patients with no complications, reported utility values ranged from 0.90 to 0.98. Complications including stroke (reported disutility range, -0.105 to -0.291), neuropathy (range, -0.055 to -0.358), and blindness (range, -0.132 to -0.208) were associated with the largest decrements in utility values. The magnitude of utility values and utility decrements was influenced by the assessment method used. CONCLUSION: Complications lead to impaired health-related quality of life in patients with type 1 diabetes, the magnitude of which is influenced by the method used to determine utilities. There is currently a lack of utility data for certain complications of type 1 diabetes, meaning that many economic evaluations have relied on a combination of type 1 and type 2 diabetes utilities, despite differences between the conditions and populations, or type 1 diabetes-specific utilities derived from different instruments.

Review of the Clinical and Economic Burden of Antibody-Mediated Rejection in Renal Transplant Recipients.

UNLABELLED: Antibody-mediated rejection (AbMR) is a leading cause of late graft loss in kidney transplant recipients, accounting for up to 60% of late graft failures. AbMR manifests as two distinct phenotypes: the first occurs in the immediate post-transplant period in sensitized patients; the second occurs in the late post-transplant period and has been associated with non-adherence to immunosuppression. The present review summarizes the current treatment options for AbMR, its clinical and economic burden, and approaches for reducing the risk of AbMR. While AbMR is typically refractory to treatment with corticosteroids, there are numerous other approaches focused on removal, inhibition or neutralization of donor-specific antibodies, or inhibition of complement-mediated allograft damage. AbMR treatment is generally expensive with one US study reporting costs of USD 49,000-155,000 per episode. However, leaving AbMR untreated puts patients at high risk of capillaritis, microangiopathy, necrosis and graft failure, which may ultimately result in much greater costs associated with a return to dialysis. Given the barriers to treatment, which include the high cost and the fact that pharmacologic treatments are currently used off-label, prevention of AbMR is important, with improvement in patient adherence to immunosuppression a key strategic approach that may be worthy of further evaluation. FUNDING: Astellas Pharma EMEA Limited.

Long-term health economic benefits of sensor-augmented pump therapy vs continuous subcutaneous insulin infusion alone in type 1 diabetes: a U.K. perspective.

AIMS/HYPOTHESIS: Continuous subcutaneous insulin infusion (CSII) is an important treatment option for type 1 diabetes patients unable to achieve adequate glycemic control with multiple daily injections (MDI). Combining CSII with continuous glucose monitoring (CGM) in sensor-augmented pump therapy (SAP) with a low glucose-suspend (LGS) feature may further improve glycemic control and reduce the frequency of hypoglycemia. A cost-effectiveness analysis of SAP + LGS vs. CSII plus self-monitoring of blood glucose (SMBG) was performed to determine the health economic benefits of SAP + LGS in type 1 diabetes patients using CSII in the U.K. METHODS: Cost-effectiveness analysis was performed using the CORE diabetes model. Treatment effects were sourced from the literature, where SAP + LGS was associated with a projected HbA1c reduction of -1.49% vs. -0.62% for CSII, and a reduced frequency of severe hypoglycemia. The time horizon was that of patient lifetimes; future costs and clinical outcomes were discounted at 3.5% and 1.5% per annum, respectively. RESULTS: Projected outcomes showed that SAP + LGS was associated with higher mean quality-adjusted life expectancy (17.9 vs. 14.9 quality-adjusted life years [QALYs], SAP + LGS vs. CSII), and higher life expectancy (23.8 vs. 21.9 years), but higher mean lifetime direct costs (GBP 125,559 vs. GBP 88,991), leading to an incremental cost-effectiveness ratio (ICER) of GBP 12,233 per QALY gained for SAP + LGS vs. CSII. Findings of the base-case analysis remained robust in sensitivity analyses. CONCLUSIONS/INTERPRETATION: For UK-based type 1 diabetes patients with poor glycemic control, the use of SAP + LGS is likely to be cost-effective compared with CSII plus SMBG.

Cost-Effectiveness of Sensor-Augmented Pump Therapy with Low Glucose Suspend Versus Standard Insulin Pump Therapy in Two Different Patient Populations with Type 1 Diabetes in France.

BACKGROUND: Sensor-augmented pump therapy (SAP) provides a useful adjunct relative to continuous subcutaneous insulin infusion (CSII) alone. It can provide early warning of the onset of hyperglycemia and hypoglycemia and has the functionality to suspend insulin delivery if sensor glucose levels fall below a predefined threshold. The aim was to assess the cost-effectiveness of SAP with low glucose suspend (LGS) versus CSII alone in type 1 diabetes. MATERIALS AND METHODS: Cost-effectiveness analysis was performed using the CORE Diabetes Model, using published clinical input data. The analysis was performed in two cohorts: one with uncontrolled glycated hemoglobin at baseline and one at elevated risk for hypoglycemic events. The analysis was conducted from a healthcare payer perspective over a lifetime time horizon; future costs and clinical outcomes were discounted at 4% per annum. RESULTS: In patients with uncontrolled glycated hemoglobin at baseline, SAP + LGS resulted in improved discounted quality-adjusted life expectancy (QALE) versus CSII (10.55 quality-adjusted life-years [QALYs] vs. 9.36 QALYs) but higher mean lifetime direct costs (€84,972 vs. €49,171) resulting in an incremental cost-effectiveness ratio (ICER) of €30,163 per QALY gained. In patients at elevated risk for hypoglycemia, the ICER was €22,005 per QALY gained for SAP + LGS versus CSII as lifetime costs were higher (€88,680 vs. €57,097), but QALE was also higher (18.46 QALYs vs. 18.30 QALYs). CONCLUSIONS: In France, projected improvements in outcomes with SAP + LGS versus CSII translated into an ICER generally considered as good value for money, particularly in patients who experience frequent and/or problematic hypoglycemic events.

Achieving sustained virologic response in hepatitis C: a systematic review of the clinical, economic and quality of life benefits.

BACKGROUND: The goal of chronic hepatitis C treatment is to remove the virus to avoid progression of HCV-related disease. Sustained virologic response (SVR) is the most widely used efficacy endpoint in clinical studies of hepatitis C, and represents the eradication of HCV from the body. The aim of the current review was to examine the long-term clinical, economic and quality of life benefits associated with achieving SVR. METHODS: A systematic literature review was performed using the PubMed, EMBASE and Cochrane library databases to identify articles examining the clinical, economic and quality of life benefits associated with SVR, published in English language from 2002-2013. For inclusion studies were required to enroll ≥100 patients and to report clinical endpoints including hepatocellular carcinoma, overall- or liver-related mortality, or progression of disease/complications (e.g. portal hypertension, esophageal varices). Review of economic studies on cost/cost-effectiveness of achieving SVR were focused on studies assessing boceprevir/telaprevir plus pegIFN and ribavirin as this represents the current standard of care in several jurisdictions worldwide. Quality of life evidence was required to use validated quality of life instruments and provide a quantitative analysis of the impact of SVR versus no treatment or treatment failure. RESULTS: SVR is durable with late relapse rates over 4-5 year periods being in the range of 1-2%. Patients who achieve SVR frequently demonstrate some regression of fibrosis/cirrhosis and have a substantially reduced risk for hepatocellular carcinoma (relative risk [RR] 0.1-0.25), liver-related mortality (RR 0.03-0.2) and overall mortality (RR 0.1-0.3) in comparison with no treatment or treatment failure. In the 5 years post-treatment, medical costs for patients achieving SVR are 13-fold lower than patients not achieving SVR. Patients who achieve SVR also have health state utility values that are 0.05 to 0.31 higher than non-responders to treatment. CONCLUSIONS: SVR represents the fundamental goal of antiviral treatment for patients infected with chronic HCV, so as to reduce risk of liver disease progression. Achievement of SVR has implications beyond those of clearing viral infection; it is associated with improved long-term clinical outcomes, economic benefits and improved health-related quality of life.

Project Baiterek: A Patient Access Program to Improve Clinical Outcomes and Quality of Life in Children with Type 1 Diabetes in Kazakhstan.

Diabetes is a key driver in the rise of noncommunicable diseases globally. It causes expensive and burdensome short- and long-term complications, with both an economic and social impact. In many countries, however, access to care and disease management in type 1 diabetes is suboptimal, increasing the risk for complications. In 2011, Project Baiterek was initiated as a collaborative effort between the Kazakhstan Ministry of Health, industry (Medtronic Plc), local physicians, and the Diabetes Association of the Republic of Kazakhstan to enhance patient access to continuous subcutaneous insulin infusion (CSII) therapy. It was the first countrywide project to provide equity and universal access to insulin pump therapy among children with type 1 diabetes, increasing pump use from zero to two-thirds of this population in less than 3 years. The project also involved instigating longitudinal data collection, and long-term clinical outcomes continue to be monitored. Here, we provide an overview of the clinical, quality-of-life, and economic outcomes to date associated with providing CSII therapy to children with type 1 diabetes in Kazakhstan. Initial clinical data show that CSII therapy improved clinical outcomes and quality of life for patients entered into the program and that CSII therapy was cost-effective relative to multiple daily injection therapy. The positive outcomes of Project Baiterek provide a template for similar patient access programs in other settings, and its framework could be adapted to initiatives to change health care infrastructures and standards of care for other noncommunicable diseases.

Cardiovascular risk profiles in Type 2 diabetes and the impact of geographical setting.

Cardiovascular (CV) disease is a leading morbidity and mortality in Type 2 diabetes (T2DM). Previous studies have shown geographic differences in the prevalence of CV and renal diseases. A literature review of longitudinal (≥5 years) studies including ≥1000 T2DM patients and reporting CV endpoints was performed to compare risk profiles. Key differences between geographies included a relatively higher prevalence of microalbuminuria in East Asian relative to North American and European patients, which in turn is an important CV risk factor. Patients from East Asia also have a relatively higher incidence of stroke and lower incidence of coronary heart disease. Overall, there are differences in CV risk in T2DM patients between different regions and that long-term studies from Africa, the Middle East and Latin America are lacking.