Impact of Elexacaftor/Tezacaftor/Ivacaftor on Healthcare Resource Utilization and Associated Costs Among People With Cystic Fibrosis in the US: A Retrospective Claims Analysis.

Introduction: Cystic fibrosis (CF) is a life-limiting genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is a CFTR modulator (CFTRm) that targets the underlying cause of CF. Based on safety and efficacy demonstrated in clinical trials, ELX/TEZ/IVA is approved in the US for the treatment of CF in people aged ≥ 2 years who have ≥ 1 F508del-CFTR mutation or a CFTR mutation that is responsive to ELX/TEZ/IVA based on in vitro data.

EFFECT OF ELEXACAFTOR/TEZACAFTOR/IVACAFTOR ON ANNUAL RATE OF LUNG FUNCTION DECLINE IN PEOPLE WITH CYSTIC FIBROSIS.

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) with ≥ 1 F508del-CFTR allele in Phase 3 clinical trials. ELX/TEZ/IVA treatment led to improved lung function, with increases in percent predicted forced expiratory volume in 1 second (ppFEV) and Cystic Fibrosis Questionnaire-Revised respiratory domain score. Here, we evaluated the impact of ELX/TEZ/IVA on the rate of lung function decline over time by comparing changes in ppFEV in participants from the Phase 3 trials with a matched group of people with CF from the US Cystic Fibrosis Foundation Patient Registry not eligible for cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy.

Non-respiratory health-related quality of life in people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor.

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) heterozygous for F508del and a minimal function mutation (F/MF) or homozygous for F508del (F/F) in two pivotal Phase 3 trials, significantly improving percentage predicted forced expiratory volume in 1 second, Cystic Fibrosis Questionnaire-Revised, Respiratory Domain (CFQ-R RD) scores, and sweat chloride concentration. Here, we analyzed the 11 non-respiratory domains (non-RDs) of the CFQ-R, which assess general health-related quality of life (i.e.

Disease burden in people with cystic fibrosis heterozygous for F508del and a minimal function mutation.

Background: People with cystic fibrosis (CF) heterozygous for F508del-CFTR and a minimal function CFTR mutation (F/MF) that results in no CFTR protein or results in CFTR protein that is not responsive to tezacaftor, ivacaftor, and tezacaftor/ivacaftor in vitro comprise a sizeable percentage of the US CF population. This retrospective, cross-sectional, observational study aimed to characterize CF burden in this subpopulation.

Methods: People ≥2 years of age in the US CF Foundation Patient Registry with a CF diagnosis, F/MF genotype, and ≥1 encounters in 2017 were included.

The Trend of High-Dose Insulin Usage Among Patients with Diabetes in the UK: A Retrospective Study.

Introduction: Many patients with diabetes may require high-dose insulin treatment to achieve target HbA1c level, but the prevalence, disease burden, and patient characteristics of the population remain unclear. We therefore investigated people with insulin-treated diabetes in the UK from 2009 to 2013, who were prescribed high daily doses (> 200 units/day).

Methods: A retrospective analysis was conducted using the UK primary care electronic dataset from the Clinical Practice Research Datalink (CPRD).

Carbon stocks, sequestration, and emissions of wetlands in south eastern Australia.

Nontidal wetlands are estimated to contribute significantly to the soil carbon pool across the globe. However, our understanding of the occurrence and variability of carbon storage between wetland types and across regions represents a major impediment to the ability of nations to include wetlands in greenhouse gas inventories and carbon offset initiatives. We performed a large-scale survey of nontidal wetland soil carbon stocks and accretion rates from the state of Victoria in south-eastern Australia-a region spanning 237,000 km and containing >35,000 temperate, alpine, and semi-arid wetlands.

Treating Type 1 Diabetes Mellitus with a Rapid-Acting Analog Insulin Regimen vs. Regular Human Insulin in Germany: A Long-Term Cost-Effectiveness Evaluation.

Objective: The aim of the present study was to evaluate the cost effectiveness of rapid-acting analog insulin relative to regular human insulin in adults with type 1 diabetes mellitus in Germany.

Methods: The PRIME Diabetes Model, a patient-level, discrete event simulation model, was used to project long-term clinical and cost outcomes for patients with type 1 diabetes from the perspective of a German healthcare payer. Simulated patients had a mean age of 21.

Patient-reported Outcomes in Patients with Type 2 Diabetes Treated with Dulaglutide Added to Titrated Insulin Glargine (AWARD-9).

Purpose: This 28-week, randomized, double-blind study compared a once-weekly injection of dulaglutide 1.5 mg to placebo, both added to titrated once-daily insulin glargine (with or without metformin), in patients with type 2 diabetes mellitus and inadequate glycemia control (control defined as hemoglobin A, ≥7% and ≤10.5%).

Behaviours, thoughts and perceptions around mealtime insulin usage and wastage among people with type 1 and type 2 diabetes mellitus: A cross-sectional survey study.

Aims: People with diabetes who use mealtime insulin (MTI) were surveyed about insulin wastage and injection habits when insufficient insulin remains in a disposable prefilled pen/cartridge to administer a full dose in a single injection.

Methods: Cross-sectional, online, self-reported survey of MTI usage/wastage behaviour in 400 adults with type 1 (n=120) or type 2 (n=280) diabetes mellitus administering >20units/day of MTI via 100units/ml prefilled pens/cartridges for ⩾1month, conducted in France, Germany, Italy and UK.

Results: Participants' mean±standard deviation age was 54.

Basal-bolus Therapy in Patients with Type 2 Diabetes Mellitus in the UK: Patient Characteristics, Treatment Patterns and the Effect of Switching to Premixed Insulin.

Introduction: Increasing emphasis is being placed on insulin use among patients with type 2 diabetes mellitus (T2DM). Basal-bolus (BB) therapy is regarded as the gold standard, but a high frequency of injections and the general complexity of this therapy are seen as barriers in real-world practice. Here we describe the characteristics and treatment patterns of patients with T2DM receiving BB in the UK, with specific focus on those switching to a simplified regimen of premixed insulin.

Attributes Influencing Insulin Pen Preference Among Caregivers and Patients With Diabetes Who Require Greater Than 20 Units of Mealtime Insulin.

Background: This study compared patient preference for Humalog® KwikPen™ 200 units/mL (insulin lispro; hereafter, IL 200 pen; Eli Lilly and Company, Indianapolis, IN) versus the Humalog KwikPen 100 units/mL (insulin lispro; hereafter, IL 100 pen; Eli Lilly and Company, Indianapolis, IN) in patients with diabetes requiring >20 units of mealtime insulin and diabetes caregivers. This study also determined which attributes had the greatest influence on pen preference selection.

Methods: In this 2-period, crossover, simulated-use study, 106 participants were randomized to 1 of 8 sequences that varied the pen order (IL 100 pen or IL 200 pen) and dosing order (15 units = low dose or 50 units = high dose) for a total of 4 simulated injections.

Insulin dosing and outcomes among commercially insured patients with type 2 diabetes in the United States.

Purpose: The purpose of this study was to examine costs, resource use, adherence, and hypoglycemic events among patients with type 2 diabetes mellitus (T2DM) treated with increasing doses of 100-U/mL (U-100) insulin regimens.

Methods: Data from Truven's Health Analytics Commercial Claims and Encounters database from January 1, 2008, through January 31, 2011, were used. Regressions were used to examine the associations among costs, resource use, adherence, and receipt of a hypoglycemic event and index dose of insulin.

Dosing of U-100 insulin and associated outcomes among Medicare enrollees with type 1 or type 2 diabetes.

Objective: To examine costs, resource utilization, adherence, and hypoglycemic events among various doses of U-100 insulin regimens among elderly patients (age ≥65 years) diagnosed with diabetes.

Methods: Truven Health Analytics Medicare databases from January 1, 2008 through December 31, 2011 were utilized. General linear models with a gamma distribution and log link were used to examine costs, while logistic and negative binomial regressions were used to examine resource utilization and hypoglycemic events.

Safe and Effective Use of the Once Weekly Dulaglutide Single-Dose Pen in Injection-Naïve Patients With Type 2 Diabetes.

Background: This 4-week, phase 3b, multicenter, open-label, single-arm, outpatient study demonstrated the safe and effective use of the dulaglutide single-dose pen containing 0.5 mL of placebo for subcutaneous injection in injection-naïve adult patients with type 2 diabetes (T2D), with A1C ≤ 8.5% (69 mmol/mol), BMI ≥ 23 kg/m2 and ≤ 45 kg/m(2).

Insulin use in long term care settings for patients with type 2 diabetes mellitus: a systematic review of the literature.

Objective: To summarize currently available data about insulin therapy in patients with diabetes mellitus (DM), focusing on patients with type 2 DM (T2DM), in long term care (LTC) settings.

Data Sources: Ovid Medline, EMBASE, Cochrane Library databases, and United Kingdom National Health Service (NHS) Economic Evaluation Database, last accessed on November 12, 2012.

Study Eligibility Criteria: We included studies that reported insulin use in patients with T2DM, and studies with combined samples of patients with type 1 DM or T2DM, that were conducted in LTC settings.

Utilities associated with subcutaneous injections and intravenous infusions for treatment of patients with bone metastases.

Introduction: Although cost-utility models are often used to estimate the value of treatments for metastatic cancer, limited information is available on the utility of common treatment modalities. Bisphosphonate treatment for bone metastases is frequently administered via intravenous infusion, while a newer treatment is administered as a subcutaneous injection. This study estimated the impact of these treatment modalities on health state preference.

Health state utilities for skeletal-related events secondary to bone metastases.

Introduction: Patients with bone metastases often experience skeletal-related events (SREs). Although cost-utility models are used to examine treatments for metastatic cancer, limited information is available on utilities of SREs. The purpose of this study was to estimate the disutility of four SREs: spinal cord compression, pathological fracture, radiation to bone, and surgery performed to stabilize a bone.

Patient-reported outcome instruments used to assess pain and functioning in studies of bisphosphonate treatment for bone metastases.

Purpose: When treating metastatic bone disease, relief of bone pain is often a key outcome. Because pain cannot be quantified with objective clinical measures, patient-reported outcome (PRO) measures are required to assess patients' subjective experience. The goal of the current review was to examine measures used to assess pain, as well as the impact of pain on functional status and health-related quality of life (HRQL), in trials of bisphosphonates for the treatment of bone metastases.

Preferences related to attention-deficit/hyperactivity disorder and its treatment.

Objectives: A growing body of literature has highlighted the importance of considering patient preferences as part of the medical decision-making process. The purpose of the current review was to identify and summarize published research on preferences related to attention-deficit/hyperactivity disorder (ADHD) and its treatment, while suggesting directions for future research.

Methods: A literature search identified 15 articles that included a choice-based assessment of preferences related to ADHD.

Utilities and disutilities for attributes of injectable treatments for type 2 diabetes.

Introduction: Although cost-utility models are frequently used to estimate treatment outcomes for type 2 diabetes, utilities are not available for key attributes of injectable treatments. The purpose of this study was to identify the utility or disutility of three injection-related attributes (dose frequency, dose flexibility, injection site reaction) that may influence patient preference.

Methods: Patients with type 2 diabetes in Scotland completed standard gamble (SG) interviews to assess the utility of hypothetical health states and their own current health state.