Real-world Canagliflozin Utilization: Glycemic Control Among Patients With Type 2 Diabetes Mellitus-A Multi-Database Synthesis.

PURPOSE: Randomized controlled trials have found that treatment of type 2 diabetes mellitus with canagliflozin, a sodium glucose co-transporter 2 inhibitor, is associated with significant reductions in glycosylated hemoglobin (HbA1c) levels. However, very few studies have evaluated the effectiveness of sodium glucose co-transporter 2 inhibitors in a real-world context. This data synthesis aims to examine the demographic characteristics and glycemic control among patients treated with canagliflozin in clinical practice, using results obtained from 2 US-specific retrospective administrative claims databases. METHODS: Data included in the synthesis were derived from 2 large claims databases (the Optum Research Database and the Inovalon MORE(2) Registry, Research Edition) and were obtained from 3 recently published retrospective observational studies of adult patients with type 2 diabetes mellitus who were treated with canagliflozin. Two of the studies used the Optum database (3-month and 6-month follow-up) and 1 study used the Inovalon database (mean follow-up of 4 months). Patient demographic characteristics, clinical characteristics, treatment utilization, and achievement of glycemic goals at baseline and after canagliflozin treatment were evaluated across the 3 studies. Results were assessed using univariate descriptive statistics. FINDINGS: Baseline demographic characteristics were generally similar between the Optum and Inovalon cohorts. Mean baseline HbA1c was 8.7% in the Optum and 8.3% in the Inovalon cohort. Seventy-five percent of the Optum (3-month study) cohort and 74% of the Inovalon cohort used 2 or more antihyperglycemic agents. During follow-up, in both cohorts, the proportion of patients who achieved tight glycemic control (HbA1c <7.0%) more than doubled, while the proportion who had poor control (HbA1c ≥9.0%) decreased by approximately 50%. Among patients who had baseline HbA1c ≥7.0%, 21% of the Optum cohort and 24% of the Inovalon cohort achieved tight glycemic control (HbA1c <7.0%), and the proportion of patients achieving HbA1c <8.0% more than doubled in both cohorts (from 30% to 61% in the Optum cohort, and from 33% to 69% in the Inovalon cohort). IMPLICATIONS: This synthesis of real-world data from 2 large patient databases suggests that treatment of type 2 diabetes mellitus with canagliflozin is associated with significant and consistent improvements in glycemic control. Patients with varying HbA1c control and multiple antihyperglycemic agent use were able to lower their HbA1c levels with canagliflozin treatment. Additional studies with longer follow-up would be beneficial to evaluate the durability of the real-world effectiveness of canagliflozin.

The value of specialty pharmaceuticals - a systematic review.

OBJECTIVES: Novel specialty biopharmaceuticals hold promise for patients living with complex and chronic conditions. However, high research and development costs, special handling, and other necessary enhancements to patient support programs all contribute to frequently higher prices for these products. This study sought to assess the value of specialty pharmaceuticals through an examination of the clinical, functional, and economic benefits of these treatments for the top 3 disease areas by pharmaceutical spend: rheumatoid arthritis (RA), multiple sclerosis (MS), and breast cancer (BC). STUDY DESIGN: Systematic literature review. METHODS: A systematic review of market research and cost-effectiveness articles was conducted for each disease area to assess clinical, functional, and economic outcomes associated with specialty medicine treatments versus the previous standard of care. RESULTS: All RA clinical (American College of Rheumatology) and functional (Health Assessment Questionnaire) outcome articles were classified as positive. The median cost-effectiveness ratio was $38,900 per quality-adjusted life year (QALY). All MS clinical outcome (relapse rate) articles were positive. The MS functional outcome (Expanded Disability Status Scale) findings were less conclusive. The median cost-effectiveness ratio was $248,000 per QALY. The majority of BC articles yielded statistically inconclusive results for survival. All functional outcome (Quality of Life Questionnaire- Core 30) articles were positive. The median cost-effectiveness ratio was $51,900 per QALY. CONCLUSIONS: Novel specialty therapies hold promise for arresting disease progression and improving quality of life for the 3 conditions associated with the highest specialty pharmaceutical spend. These findings demonstrate a strong value proposition for specialty pharmaceuticals, and suggest even greater potential individual patient benefit with consideration of patient heterogeneity.