Outcomes of Patients with COPD Treated with ICS/LABA Before and After Initiation of Single-Inhaler Triple Therapy with Fluticasone Furoate/Umeclidinium/Vilanterol (FF/UMEC/VI).

INTRODUCTION: Triple therapy (fluticasone furoate/umeclidinium/vilanterol; FF/UMEC/VI) has been shown to improve symptoms and reduce exacerbations in patients with chronic obstructive pulmonary disease (COPD) and a history of exacerbations. This real-world study compared exacerbation rates and healthcare resource utilization (HCRU) before and after initiation of FF/UMEC/VI in patients with COPD previously treated with inhaled corticosteroid (ICS)/long-acting ß2-agonist (LABA). METHODS: This retrospective cohort study included commercial and Medicare Advantage with Part D administrative claims data from September 01, 2016, to March 31, 2020, of patients diagnosed with COPD. The index date was the date of the first FF/UMEC/VI claim (September 2017-March 2019). The 12 months prior to index (baseline) were used to assess patient characteristics and outcomes; the 12 months following index (follow-up) were used to assess study outcomes. All patients had ≥ 30 consecutive days' supply of any ICS/LABA dual therapy during the 12 months prior to FF/UMEC/VI initiation. Subgroup analyses included patients with ≥ 30 consecutive days' supply of budesonide/formoterol (BUD/FORM) during baseline. Analyses of patients with ≥ 1 COPD exacerbation during baseline were reported as well. RESULTS: The overall population included 1449 patients (mean age 70.75 years; 54.18% female), of whom 540 were patients in the BUD/FORM subgroup. Significantly fewer patients experienced any exacerbation during follow-up versus baseline (overall population 53.49% vs 62.59%; p < 0.001; BUD/FORM subgroup 55.00% vs 62.41%; p = 0.004). Effects on exacerbation reduction were more pronounced among patients with ≥ 1 exacerbation during baseline. Lower COPD-related HCRU was observed during the follow-up compared with baseline for both the overall population and the BUD/FORM subgroup. CONCLUSION: Patients with COPD treated with ICS/LABA during baseline, including patients specifically treated with BUD/FORM and those with a history of ≥ 1 exacerbation, had fewer COPD exacerbations and lower COPD-related HCRU after initiating FF/UMEC/VI.

Real-World Disease Burden and Healthcare Resource Utilization Among Patients with COPD and Asthma Using Triple Therapy (FF/UMEC/VI) in the United States.

Purpose: Chronic obstructive pulmonary disease (COPD) and asthma are associated with chronic inflammation of the respiratory tract; despite some overlap of symptoms, they are considered separate disorders. Triple therapy is recommended for patients with COPD and asthma whose symptoms remain uncontrolled despite dual therapy. There are limited real-world studies evaluating outcomes among patients with COPD and asthma who are receiving inhaled triple therapy. This United States (US)-based real-world study aimed to evaluate clinical and economic outcomes among patients with COPD and asthma receiving single-inhaler triple therapy (fluticasone furoate/umeclidinium/vilanterol [FF/UMEC/VI]). Patients and Methods: Retrospective pre-post study using claims data from the Optum Clinformatics® database. Patients with COPD and asthma were indexed on the first date of FF/UMEC/VI prescription (1 October 2017-31 March 2019). Each patient acted as their own control. Patients were required to have continuous health plan enrollment for 12 months prior to (pre-treatment) and following (post-treatment) index. Exacerbations, all-cause and COPD-related healthcare resource utilization, and costs were compared before and after FF/UMEC/VI initiation. Results: Overall, 2743 patients were included (mean age: 71 years; 64% female). Cardiovascular disease was the most prevalent comorbidity during both the pre- and post-treatment periods (90% for both periods). There was a lower proportion of patients with ≥1 COPD exacerbation or ≥1 asthma exacerbation post-treatment versus pre-treatment (51% vs 57%, p<0.0001, and 22% vs 32%, p<0.0001, respectively). Fewer patients had ≥1 all-cause office visit post-treatment versus pre-treatment (99.3% vs 99.7%, p=0.0329); more patients had ≥1 COPD-related office visit post-treatment versus pre-treatment (89.6% vs 87.5%, p=0.0035). Total all-cause healthcare costs were significantly higher post-treatment versus pre-treatment ($72,809 vs $63,734, p<0.0001). The driver of increased costs appeared to be primarily non-COPD-related (COPD-related costs: post-treatment $27,779 vs pre-treatment $25,081, p=0.0062). Conclusion: FF/UMEC/VI reduced exacerbations among patients with COPD and asthma in a real-world setting in the US.

Real-World Study of Single-Inhaler Triple Therapy with Fluticasone Furoate/Umeclidinium/Vilanterol on Asthma Control in the US.

Purpose: Real-world asthma control data among patients initiating fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) are limited. This study assessed rescue medication use and asthma-related exacerbations in patients with asthma before and after initiating single-inhaler FF/UMEC/VI using administrative claims data. Patients and Methods: This retrospective, pre-post cohort study analyzed data from the IQVIA PharMetrics Plus database (September 18, 2016‒March 31, 2020). Patients aged ≥18 years that had ≥1 dispensing of single-inhaler FF/UMEC/VI 100/62.5/25 mcg (first dispensing = index date), ≥12 months of continuous health insurance enrollment prior to (pre-treatment) and following (post-treatment) FF/UMEC/VI initiation and ≥1 diagnosis of asthma during the pre-treatment period or on the index date were included. The primary endpoint was the number of oral corticosteroid (OCS) dispensings per patient per year during pre- and post-treatment periods. Secondary endpoints included asthma-related exacerbation rates and short-acting ß2-agonist (SABA) use. Comparisons between pre- and post-treatment periods were made using risk and rate ratios. Results: Overall, 890 patients with asthma initiating treatment with FF/UMEC/VI were included. The most recently dispensed controller medications prior to FF/UMEC/VI initiation were inhaled corticosteroids/long-acting ß2-agonists (33.5%) and leukotriene modifiers (33.0%). Patients had a 29% reduction in the number of OCS dispensings (rate ratio [95% confidence interval (CI)]: 0.71 [0.65, 0.77], P < 0.001) during post-treatment versus pre-treatment, with a 23% reduction in the proportion of patients with ≥1 OCS dispensing post-treatment (risk ratio [95% CI]: 0.77 [0.73, 0.82], P < 0.001). Significant reductions in rates (rate ratio [95% CI]) of asthma-related exacerbations (0.59 [0.52, 0.67], P < 0.001) and SABA use (0.80 [0.74, 0.86], P < 0.001) were also observed. Conclusion: In this real-world study, patients with asthma had significantly lower OCS use, asthma-related exacerbations, and SABA use following treatment initiation with FF/UMEC/VI compared with their pre-treatment period. These results suggest better asthma control following initiation of FF/UMEC/VI in a routine clinical practice setting.

Glycemic and Economic Outcomes in Elderly Patients with Type 2 Diabetes Initiating Dulaglutide Versus Basal Insulin in a Real-World Setting in the United States: The DISPEL-Advance Study.

INTRODUCTION: Treatments like glucagon-like peptide-1 receptor agonists carry low hypoglycemia risk and are recommended for elderly patients with type 2 diabetes (T2D), while some routine treatments, like insulin, increase hypoglycemia risk. The DISPEL-Advance (Dulaglutide vs Basal InSulin in Injection Naïve Patients with Type 2 Diabetes: Effectiveness in ReaL World) study compared glycemic outcomes, healthcare resource utilization, and costs in elderly patients with T2D who initiated treatment with dulaglutide versus those initiating treatment with basal insulin. METHODS: This observational, retrospective cohort study used data from the Optum Research Database. Medicare Advantage patients (≥ 65 years) with T2D were assigned to dulaglutide or basal insulin cohorts based on pharmacy claims and propensity score matched on demographic and baseline characteristics. Change in HbA1c, 12-months follow-up HbA1c, and follow-up all-cause and diabetes-related healthcare resource utilization and costs were compared. RESULTS: Propensity score matching yielded well-balanced cohorts with 1891 patients each (mean age: dulaglutide, 72.09 years; basal insulin, 72.56 years). The dulaglutide cohort had significantly greater mean HbA1c reduction from baseline to follow-up than basal insulin cohort (- 0.95% vs - 0.69%; p < 0.001). The dulaglutide cohort had significantly lower mean all-cause and diabetes-related medical costs (all-cause: $8306 vs $12,176; diabetes-related: $4681 vs $7582 respectively; p < 0.001) and lower mean all-cause total costs ($18,646 vs $20,972, respectively; p = 0.007) than basal insulin cohort. The dulaglutide cohort had significantly lower all-cause and diabetes-related total costs per 1% change in HbA1c than basal insulin cohort (all-cause: $19,729 vs $30,334; diabetes-related: $12,842 vs $17,288, respectively; p < 0.001). CONCLUSIONS: Elderly patients with T2D initiating dulaglutide had greater HbA1c reduction, lower mean all-cause medical and total costs, lower diabetes-related medical costs, and lower total all-cause and diabetes-related costs per 1% change in HbA1c than patients initiating basal insulin. Future studies assessing medications that do not increase hypoglycemia risk could help inform therapeutic strategies in elderly patients.

The Association Between Sustained HbA1c Control and Long-Term Complications Among Individuals with Type 2 Diabetes: A Retrospective Study.

INTRODUCTION: The prevalence of type 2 diabetes (T2D) represents a rising burden in the US and worldwide, with the condition shown to be associated with relatively large human and economic costs. Part of the reason for such high costs associated with T2D is that the condition is often accompanied by additional health-related complications. The goal of this research is to examine the association between glycemic control and diabetes-related complications for individuals with T2D. METHODS: The Optum Clinformatics® Data Mart (CDM) database from 2007 to 2020 was used to identify adults with T2D. Individuals were classified as having sustained glycemic control (all hemoglobin A1c [HbA1c] < 7%) or poor glycemic control (all HbA1c ≥ 7%) over the 5-year post-period, and diabetes-related complications were identified based upon the Diabetes Complications Severity Index. Multivariable analyses examined the association between sustained glycemic control and diagnosis of a diabetes-related complication in the post-period. RESULTS: Maintaining HbA1c < 7% over the 5-year post-period, compared to maintaining HbA1c ≥ 7%, was associated with reduced odds of the diabetes-related complications of cardiovascular disease (odds ratio [OR] = 0.76, 95% confidence interval [CI] 0.61-0.94), metabolic disease (OR = 0.37, 95% CI 0.22-0.600), neuropathy (OR = 0.62, 95% CI 0.45-0.84), nephropathy (OR = 0.81, 95% CI 0.69-0.94), and peripheral vascular disease (OR = 0.52, 95% CI 0.33-0.83). There was no statistically significant association between sustained glycemic control and cerebrovascular disease. CONCLUSIONS: Sustained glycemic control was found to be associated with significant reductions in the odds of being diagnosed with diabetes-related complications over a 5-year post-period.

Understanding the impact of five major determinants of health (genetics, biology, behavior, psychology, society/environment) on type 2 diabetes in U.S. Hispanic/Latino families: Mil Familias - a cohort study.

BACKGROUND: In the United States (U.S.), the prevalence of both diagnosed and undiagnosed type 2 diabetes (T2D) is nearly twice as high among Mexican-origin Hispanic/Latino adults compared to non-Hispanic Whites. Rates of diabetes-related complications, e.g., acute stroke and end-stage renal disease, are also higher among Hispanic/Latino adults compared to their non-Hispanic/Latino White counterparts. Beyond genetic and biological factors, it is now recognized that sociocultural influences are also important factors in determining risk for T2D and the associated complications. These influences include ethnicity, acculturation, residence, education, and economic status. The primary objective of this study is to determine the influence of the 5 major determinants of human health (genetics, biology, behavior, psychology, society/environment) on the burden of T2D for Latino families. To achieve this objective, Mil Familias (www.milfamilias.sansum.org/) is establishing an observational cohort of 1000 Latino families, with at least one family member living with T2D. METHODS: Specially trained, bilingual Latino/a community health workers (Especialistas) recruit participant families and conduct research activities. Each individual family member will contribute data annually on over 100 different variables relating to their genetics, biology, psychology, behavior, and society/environment, creating a Latino-focused biobank ("Living Information Bank"). This observational cohort study is cross-sectional and longitudinal. Participants are divided into 4 groups: adults age ≥ 18 years with and without T2D, and children age ≥ 7 and < 18 years with and without T2D. Study activities take place through encounters between families and their Especialista. Encounters include screening/enrollment, informed consent, health promotion assessment, laboratory tests, questionnaires, physical activity monitoring, and reflection. DISCUSSION: By creating and providing the framework for the Cohort Establishment study, we intend to inform new approaches regarding equity and excellence in diabetes research and care. We will examine the complex set of factors that contribute to the burden of diabetes in Latino families and assess if cardio-metabolic disease risks go beyond the traditional biological and genetic factors. Breaking the code on the interplay of cardio-metabolic risk factors may help not only this fast growing segment of the U.S. population, but also other high-risk populations. TRIAL REGISTRATION: Study retrospectively registered at ClinicalTrials.gov (NCT03830840), 2/5/2019 (enrollment began 2/1/2019).

Development of the influence, motivation, and patient activation in diabetes (IMPACT-D™) measure.

AIMS: We sought to create a new research and clinical instrument -the Influence and Motivation for Patient ACTivation in Diabetes care (IMPACT-D™) - to measure the degree to which patients with type 2 diabetes (T2DM) value health and believe they can influence it. METHODS: Candidate items were generated via a literature review, expert opinion, and qualitative interviews and focus groups with T2DM patients in Chicago, IL and Chapel Hill, NC. Psychometric testing guided by item response theory was conducted among an online panel of 500 English-speaking adults with T2DM. Differential item functioning analyses evaluated item performance across key participant characteristics. To determine construct validity, IMPACT-D™ scores were compared to other general measures of personality and patient activation. A second study among 300 patients recruited from two internal medicine clinics further assessed associations between the IMPACT-D™ and health outcomes. Cognitive interviews confirmed patient understanding of IMPACT-D™ items and instructions. RESULTS: Exploratory factor analyses revealed a single-factor solution that included 6 items. The IMPACT-D™ demonstrated adequate reliability (α = 0.72) and moderate construct validity with patient activation (r = 0.51, p < 0.001) and personality-conscientiousness subscales (r = 0.29, p < 0.001). Higher scores on the IMPACT-D™ were associated with better physical health (r = 0.17, p = 0.003) and lower severity of depressive (r = -0.35, p < 0.001) and anxiety (r = -0.28, p < 0.001) symptoms. There were no significant differences by blood pressure (r = -0.0021, p = 0.9) or haemoglobin A1c (r = -0.069p = 0.2). CONCLUSIONS: The IMPACT-D holds potential for use in both clinical care and research applications. Future studies should evaluate how to best operationalize its use for both settings.

Development of the Diabetes Injection Device Experience Questionnaire (DID-EQ) and Diabetes Injection Device Preference Questionnaire (DID-PQ).

BACKGROUND: Previous research has examined patient perceptions of insulin injection devices. However, injectable medications other than insulin are increasingly used to treat type 2 diabetes, including GLP-1 receptor agonists. No patient-reported outcome (PRO) instruments have been developed taking into account the experiences of patients using newer injection devices, which are often different from devices used for insulin. Therefore, the purpose of this qualitative study was to develop two draft PRO instruments focusing on patients' experiences with these newer injection devices (one instrument assessing perceptions of a single injection device, and another assessing preferences between two devices). METHODS: Questionnaire development proceeded in six steps: literature review, interviews with six device experts, concept elicitation interviews with patients (N = 32), preliminary translatability assessment, cognitive interviews with patients (N = 20), and final translatability assessment. RESULTS: Literature review and expert interviews were conducted to inform a concept elicitation interview guide. In concept elicitation in the US, UK, and Germany, patients with type 2 diabetes reported a range of injection features that influenced their perceptions of non-insulin injection devices (e.g., requirements for preparation of the medication/device, issues related to the needle, ease-of-use, portability). Two draft "item pools" were developed based on the literature review, expert interviews, and concept elicitation results. In cognitive interviews, patients recommended minor revisions and indicated that the draft instruments were generally clear, comprehensible, and relevant to their experience with non-insulin injectable medication. The instruments were refined based on the cognitive interviews and translatability assessment, resulting in two questionnaires. CONCLUSIONS: The various steps of qualitative research support the content validity of these new PRO instruments, which are the first developed specifically to assess perceptions of non-insulin injection delivery systems. Despite some overlap with insulin-focused questionnaires, the new instruments are distinct from previous instruments (omitting content that would not be relevant to patients receiving non-insulin injectable treatment, while including content that is not included in the insulin focused instruments). This qualitative research yielded two draft questionnaires that are grounded in patient perceptions and ready for psychometric validation studies with larger samples of patients with type 2 diabetes.

Psychometric evaluation of the Diabetes Injection Device Experience Questionnaire (DID-EQ) and Diabetes Injection Device Preference Questionnaire (DID-PQ).

BACKGROUND: Previous research has examined patient perceptions of insulin injection devices. However, a range of injectable medications other than insulin are now used to treat type 2 diabetes. No patient-reported outcome (PRO) instruments have been developed taking into account the perceptions of patients using newer injection devices, which are often different from those used in the past. Therefore, the primary purpose of this study was to evaluate a new PRO instrument focusing on patients' experiences with injection devices, including those used for newer treatments such as GLP-1 receptor agonists. METHODS: Patients with T2D treated with non-insulin injectable medications were recruited via advertisements and six clinical sites in the US. All participants completed the draft Diabetes Injection Device - Experience Questionnaire (DID-EQ) and additional measures administered for validity assessment. Participants who had experience with two non-insulin injection devices also completed the draft Diabetes Injection Device - Preference Questionnaire (DID-PQ). Analyses focused on item reduction (item performance, exploratory factor analysis), reliability, and validity. RESULTS: One hundred fourty two patients (mean age = 63.0y; 56.3% female) participated. Item reduction yielded a 10-item version of the DID-EQ, including a 7-item Device Characteristics subscale and three global items assessing satisfaction, ease of use, and convenience of the injection device. The DID-EQ demonstrated good internal consistency reliability (Cronbach's alpha of Device Characteristics subscale = 0.80) and 7-day test-retest reliability (ICCs: 0.92 for Device Characteristics subscale; 0.65 to 0.91 for the three global items). Construct validity was demonstrated via correlations with previously validated instruments (e.g., correlations with the DTSQ treatment satisfaction subscale ranged from 0.56 to 0.60, all p < 0.0001; correlations with the TRIM-D Device ranged from 0.63 to 0.77, all p < 0.0001). Descriptive analyses of the DID-PQ were conducted with a subset of 27 participants who were able to use it to compare two devices. CONCLUSIONS: This psychometric evaluation supports the reliability and validity of the DID-EQ, while providing initial information on the performance of the DID-PQ. These brief questionnaires complement measures of treatment efficacy and provide a more thorough picture of patients' experiences with non-insulin injectable treatments for type 2 diabetes.

Patient perceptions of injection devices used with dulaglutide and liraglutide for treatment of type 2 diabetes.

OBJECTIVE: Liraglutide and dulaglutide have demonstrated similar glycemic efficacy and safety. However, they differ in treatment administration and injection devices. The purpose of this study was to examine and compare patient perceptions of the injection devices used with liraglutide and dulaglutide. METHODS: Patients with type 2 diabetes treated with liraglutide or dulaglutide were recruited from across the US. Patients completed the Diabetes Injection Device Experience Questionnaire (DID-EQ) to rate their current injection device. Patients who had experience with both treatments also completed the Diabetes Injection Device Preference Questionnaire (DID-PQ) to report preferences between the two devices. ANCOVAs were conducted to compare DID-EQ scores between dulaglutide and liraglutide patients, while controlling for covariates. Descriptive statistics are presented for preferences reported on the DID-PQ. RESULTS: A total of 404 patients were recruited from 49 states (mean age = 60.7 years; 54.0% female; 204 liraglutide; 200 dulaglutide). Mean DID-EQ item scores for both treatments were high, ranging from 3.48 to 3.90 on a 4 point scale. ANCOVAs found significantly higher scores for dulaglutide than liraglutide on DID-EQ global items assessing ease of use (3.82 vs. 3.73, p = .040) and convenience (3.79 vs. 3.66, p = .004). Among the 58 patients who had used both devices, more patients reported a preference for the dulaglutide device than the liraglutide device on every item of the DID-PQ. CONCLUSIONS: High DID-EQ scores indicate positive perceptions of both the liraglutide and dulaglutide injection devices. The dulaglutide device was associated with slightly higher scores for ease of use and convenience than the liraglutide device.

Health state utilities associated with attributes of weekly injection devices for treatment of type 2 diabetes.

BACKGROUND: Glucagon-like peptide-1 (GLP-1) receptor agonists are often recommended as part of combination therapy for type 2 diabetes when oral medication does not result in sufficient glycemic control. Several GLP-1 receptor agonists are available as weekly injections. These medications vary in their injection delivery systems, and these differences could impact quality of life and treatment preference. The purpose of this study was to estimate utilities associated with attributes of injection delivery systems for weekly GLP-1 therapies. METHODS: Participants with type 2 diabetes in the UK valued health states in time trade-off interviews. The health states (drafted based on literature, device instructions for use, and clinician interviews) had identical descriptions of type 2 diabetes, but differed in description of the treatment process. One health state described oral treatment, while six others described oral treatment plus a weekly injection. The injection health states varied in three aspects of the treatment administration process: requirements for reconstituting the medication (i.e., mixing the medication prior to the injection), waiting during medication preparation, and needle handling. Every participant valued all seven health states. RESULTS: A total of 209 participants completed interviews (57.4% male; mean age = 60.4y). The mean utility of the oral treatment health state was 0.89. All injection health states had significantly (p < 0.01) lower utilities ranging from 0.86 to 0.88. Differences among health state utilities suggest that each administration requirement had a small but measureable disutility: -0.004 (reconstitution), -0.004 (needle handling), -0.010 (reconstitution, needle handling), and -0.020 (reconstitution, waiting, needle handling). CONCLUSIONS: Findings suggest it is feasible to use the TTO method to quantify preferences among injection treatment processes. It may be useful to incorporate these utility differences into cost-utility models comparing weekly injectable treatments for patients with type 2 diabetes.

Health-related quality of life burden of nonalcoholic steatohepatitis: a robust pragmatic literature review.

OBJECTIVE: Nonalcoholic steatohepatitis (NASH) is a form of chronic liver disease (CLD): patients have an increased risk of developing cirrhosis, liver failure, and complications (e.g. hepatocellular carcinoma). NASH has a high clinical burden, and likely impairs patients' health-related quality of life (HRQoL), but there are currently no licensed therapies. The objective of this robust pragmatic literature review was to identify and describe recent studies on the HRQoL burden of NASH from the patient perspective. METHODS: English-language primary research studies were identified that measured HRQoL in adults with NASH (population-based studies or clinical trials of pharmacological therapy). Searches were conducted in the following bibliographical databases: MEDLINE, EMBASE, Cochrane Database of Systematic Reviews (CDSR), Cochrane Central Register of Controlled Trials (CENTRAL), Database of Abstracts of Reviews of Effects (DARE), and Health Technology Assessment Database (HTA). Abstracts from selected congresses (2015/2016) were hand searched. Articles were assessed for relevance by two independent reviewers, and HRQoL data were extracted. RESULTS: A total of 567 de-duplicated abstracts were identified, and 20 full-text articles were reviewed. Eight studies were included: five quantitative, two interventional, and one qualitative. The quantitative and interventional studies measured HRQoL using the Short-Form 36 (SF-36) and the Chronic Liver Disease Questionnaire (CLDQ), and the qualitative study involved focus groups and individual interviews. Overall, the studies showed that NASH affects HRQoL, especially physical functioning, with many patients reporting being fatigued. In quantitative studies, overall, patients with NASH had a reduced HRQoL versus normative populations and nonalcoholic fatty liver disease (NAFLD) patients, but not versus chronic liver diseases. A longitudinal study showed that when weight loss was achieved, HRQoL improvement over 6 months was greater in patients with NASH versus NAFLD. Qualitative research suggested that, in addition to fatigue, other symptoms are also burdensome, having a broad negative impact on patients' lives. The impact of pharmacological treatment on HRQoL was explored in only two included studies. CONCLUSIONS: HRQoL is impaired in patients with NASH. Patients experience a range of symptoms, especially fatigue, and the impact on their lives is broad. Further research is needed to understand the HRQoL burden of NASH (e.g. assessing NASH-specific impacts not captured by SF-36 and CLDQ) and the impact of future NASH therapies on HRQoL.

Evaluating preferences for profiles of GLP-1 receptor agonists among injection-naïve type 2 diabetes patients in the UK.

OBJECTIVE: To use a discrete choice experiment (DCE) to evaluate preferences for the actual treatment features and overall profiles of two injectable glucagon-like peptide-1 receptor agonists (dulaglutide and liraglutide) among patients with type 2 diabetes mellitus (T2DM) in the UK. METHODS: In-person interviews were conducted in the UK to administer a DCE to patients with self-reported T2DM, naïve to treatment with injectable medications. The DCE examined six attributes of T2DM treatment each described by two levels: "dosing frequency," "hemoglobin A1c change," "weight change," "type of delivery system," "frequency of nausea," and "frequency of hypoglycemia." Part-worth utilities were estimated using random effects logit models and were used to calculate relative importance (RI) values for each attribute. A chi-square test was used to determine differences in preferences for dulaglutide versus liraglutide profiles. RESULTS: A total of 243 participants [mean age: 60.5 (standard deviation 10.9) years; 76.1% male; mean body mass index: 29.8 (standard deviation 5.4) kg/m(2)] completed the study. RI values for the attributes in rank order were: "dosing frequency" (41.6%), "type of delivery system" (35.5%), "frequency of nausea" (10.4%), "weight change" (5.9%), "hemoglobin A1c change" (3.6%), and "frequency of hypoglycemia" (3.0%). Significantly more participants preferred the dulaglutide profile (83.1%) compared with the liraglutide profile (16.9%; P<0.0001). CONCLUSION: This study elicited patients' preferences for attributes and levels representing the actual characteristics of two specific glucagon-like peptide-1 medications. In this context, dosing frequency and type of delivery system were most important, accounting for over 75% of the RI. While previous studies have identified efficacy as highly important in T2DM medication decisions, this study suggests that when differences in efficacy between medications are small, other treatment features (eg, dosing frequency and delivery system) are of much greater importance to patients.

Utility values in diabetic kidney disease: a literature review.

OBJECTIVE: To summarize the published literature on utilities for health states associated with diabetic kidney disease (DKD), including end-stage renal disease (ESRD). RESEARCH DESIGN AND METHODS: A literature review was conducted (MEDLINE, MEDLINE in process, EMBASE, NHS EED, HEED, CEA Registry, EconLit, RePEc, and HTA) to identify relevant articles published between January 2000 and July 2013. Results were assessed for relevance by two reviewers in line with the study protocol. MAIN OUTCOME MEASURES: For eligible studies, data extracted included patient population, health states, methods used to elicit utility values, and the source of the preference values. RESULTS: Twelve studies satisfied the inclusion criteria. They reported various utility and/or disutility scores for different DKD health states using a range of patient populations, utility approaches, and sources of preference values. The most common study country was the USA. Most of the studies collected data at one time point, but two had a longitudinal design. Three different utility instruments - EuroQoL (EQ-5D), Quality of Well-Being Scale (QWB), and 15-dimensional (15D) - were used to elicit utilities indirectly. The Time Trade-Off (TTO) approach was used in one study; another undertook a meta-analysis of published utility studies. Utilities were identified for different health states including DKD, ESRD-no dialysis, ESRD-dialysis, and transplant. One study reported utilities for patients by type of transplant. There was variation in values for the same health state between studies, and none of the studies reported utilities for the different stages of DKD. In the studies that undertook a comparison, utility values for those with DKD were generally found to be lower than those without DKD. CONCLUSIONS: This literature review highlights that at present utility scores (or disutility penalties) exist for relatively few health states in DKD. Further studies are needed to produce accurate and comprehensive utility scores that differentiate between different DKD health states.

Field testing the ENSEMBLE Minimum Dataset: performance of an instrument to address heterogeneity of treatment effects.

AIM: To assess the ability of ENterprising SElective Multi-instrument BLend for hEterogeneity analysis (ENSEMBLE) Minimum Dataset instrument dimensions to discriminate among subgroups of patients expected to have differential outcomes. MATERIALS & METHODS: Patients with Type 2 diabetes, knee osteoarthritis, ischemic heart disease or heart failure completed a survey designed to represent three dimensions (health, personality and behavior). Health-related outcomes and utilization were investigated using claims data. Discriminant validity and associations between the dimensions and outcomes were assessed. RESULTS: A total of 2625 patients completed the survey. The dimensions discriminated 50-100% of the outcome levels across disease cohorts; behavior dimension scores did not differ significantly among the healthcare utilization level subgroups in any disease cohort. CONCLUSION: ENSEMBLE Minimum Dataset dimensions discriminated health-related outcome levels among patients with varied diseases.

Heterogeneity of response to biologic treatment: perspective for psoriasis.

Psoriasis treatment responses are affected by patient characteristics. However, the literature does not contain reviews of factors that affect the response to biologic therapies. We therefore performed a comprehensive literature search to identify papers describing demographic, lifestyle, and clinical factors associated with response to biologic drug therapy in psoriatic patients. We found that age, gender, ethnicity, alcohol consumption, smoking, geographic location, age at diagnosis, duration and severity of psoriasis, and baseline C-reactive protein levels did not consistently affect response to biologic psoriasis therapy. However, increased body mass index (BMI) appears to adversely affect responses. It might therefore be valuable to include BMI as a stratification variable in future studies of psoriasis therapies and to consider a patient's weight or BMI when selecting a systemic psoriasis treatment.

Comprehensive review of factors implicated in the heterogeneity of response in depression.

BACKGROUND: Heterogeneity in overall response and outcomes to pharmacological treatment has been reported in several depression studies but with few sources that integrate these results. The goal of this study was to review the literature and attempt to identify nongenetic factors potentially predictive of overall response to depression treatments. METHODS: A comprehensive review of the literature from the last 10 years was performed using three key databases (PubMed, EMBASE, and Cochrane). All relevant studies that met the inclusion criteria were selected and scored for their levels of evidence using the NICE scoring method. A subjective assessment of the strength of evidence for each factor was performed using predefined criteria. RESULTS: Our broad search yielded 76 articles relevant to treatment heterogeneity. Sociodemographic factors, disease characteristics, and comorbidities were the most heavily researched areas. Some of the factors associated with more favorable overall response include being married, other social support, and low levels of baseline depressive symptoms. Evidence relating to baseline disease severity as a factor predictive of antidepressant response was particularly convincing among the factors reviewed. The presence of comorbid anxiety and pain contributed to worse antidepressant treatment outcomes. CONCLUSIONS: Several factors either predictive of or associated with overall response to antidepressant treatment have been identified. Inclusion of factors predictive of response in the design of future trials may help tailor treatments to depression patients presenting to the average clinical practice, resulting in improved outcomes.

Review of treatment response in rheumatoid arthritis: assessment of heterogeneity.

OBJECTIVE: Rheumatoid arthritis (RA) is a chronic, systemic, progressive, inflammatory disorder. The primary goals of treatment in RA are to reduce the signs and symptoms of disease, prevent progression of joint damage and improve patients' physical function. Patients with different sociodemographic characteristics, varying degrees of severity of illness, and comorbidities tend to exhibit differential response to treatment. The purpose of this review was to identify a broad set of factors that are associated with and/or predictive of RA treatment response and determine those that warrant further research. RESEARCH DESIGN AND METHODS: A comprehensive review of the literature from the last 10 years was performed using three key databases (PubMed, EMBASE, and Cochrane). All relevant articles that met the inclusion/exclusion criteria were selected and scored for their levels of evidence using the National Institute of Clinical Excellence (NICE) scoring method. Data on study design, interventions and treatment outcomes were abstracted using a structured abstraction table. RESULTS: A total of 30 articles were included in the review and data abstraction. Besides gender, baseline clinical variables such as C-reactive protein level, erythrocyte sedimentation rate, measures of disease activity, and Health Assessment Questionnaire scores (based on five patient-centered dimensions) were consistently associated with treatment response over time. CONCLUSIONS: This comprehensive literature review identified several factors associated with treatment response which might be valuable to include as relevant measures in future studies of RA treatment. Inclusion of these factors, particularly those in the clinical and sociodemographic domains, in the design of future trials will further the understanding that ultimately may help clinicians deliver targeted treatment to community practice RA patients, thus resulting in improved patient outcomes.