Physician-reported Clinical Unmet Needs, Burden and Treatment Patterns of Paediatric Psoriasis Patients: A US and EU Real-world Evidence Study.

This study is a retrospective analysis using data collected from the Adelphi Paediatric Psoriasis Disease-Specific Programme cross-sectional survey. Despite being treated for their psoriasis, a substantial proportion of paediatric patients presented with moderate (18.3%) or severe (1.

Health Care Utilization and Costs in Patients With Migraine Who Have Failed Previous Preventive Treatments.

Objective: To characterize health care utilization (HCU) and associated costs among patients with migraine categorized by the number of preventive treatment failures (TFs; 1 TF, 2 TFs, and 3+ TFs) using real-world data.

Methods: This retrospective analysis identified adults with incident migraine diagnosis in the IBM MarketScan Commercial and Medicare Supplemental database between January 1, 2011, and June 30, 2015. TF was defined in the 2 years after the first migraine diagnosis period.

Effectiveness of erenumab and onabotulinumtoxinA on acute medication usage and health care resource utilization as migraine prevention in the United States.

Migraine is a common neurological disease that can have a substantial impact on patients' lives and on society. Erenumab, a fully human monoclonal antibody that targets the calcitonin gene-related peptide receptor, was specifically developed for migraine prevention. The efficacy of erenumab has been established in several clinical trials; however, the real-world comparative effectiveness of erenumab has not been fully investigated.

Impact of erenumab on acute medication usage and health care resource utilization among migraine patients: a US claims database study.

Background: Migraine is one of the leading causes of disability worldwide. Erenumab is a fully human monoclonal antibody that targets the calcitonin gene-related peptide (CGRP) receptor. This study aimed to evaluate real-world evidence on the impact of erenumab on acute medication usage and health care resource utilization (HCRU) among migraine patients.

Real-World Trends in Characteristics of Patients with Migraine Newly Initiated on Erenumab in the USA: A Retrospective Analysis.

Introduction: This retrospective analysis aimed to characterize patients with migraine initiating erenumab and the shifting or trend of patient characteristics over time in a real-world setting.

Methods: Adult patients with at least one erenumab written prescription/administration between May 1, 2018 and September 30, 2019 were identified from the Optum De-identified Electronic Health Record (EHR) database (index date = date of the first erenumab prescription/administration). Patient demographics and characteristics, acute and preventive medications used prior to initiation of erenumab, and the initial prescriber specialty were examined.

Uncontrolled asthma across GINA treatment steps 2 - 5 in a large US patient cohort.

Objective: Despite advances in treatment, asthma remains uncontrolled in many patients, with increased risk of exacerbation and associated healthcare resource utilization (HCRU). We describe patient characteristics, exacerbations, asthma control, and HCRU using GINA treatment step (GS) as a proxy for asthma severity. .

Employee and Employer Benefits From a Migraine Management Program: Disease Outcomes and Cost Analysis.

Objective: To assess the impact of a migraine management program offered as a complimentary service by a company within its corporate well-being program.

Background: Migraine imposes a substantial burden on patients, families, employers, and societies. As migraine primarily affects working-age adults, this has important implications for both employees and employers.

The Humanistic and Economic Burden of Migraine in Europe: A Cross-Sectional Survey in Five Countries.

Introduction: Prior studies have estimated the burden of migraine in patients suffering from ≥ 4 monthly headache days (MHDs), but the burden experienced by migraineurs suffering from one to three (1-3) MHDs is unknown. The aim of this study was to examine the incremental burden of migraine in terms of health-related quality of life (HRQoL), impairments to work and daily activities, and healthcare resource utilization (HRU) in five European countries (France, Germany, Italy, Spain, and the UK (EU5]), by comparing migraineurs with ≥ 4 MHDs and migraineurs with 1-3 MHDs.

Methods: The sample for this retrospective cross-sectional study was collected from the 2017 National Health and Wellness Survey (N = 62,000).

Evaluation of exacerbations and blood eosinophils in UK and US COPD populations.

Background: Blood eosinophil counts and history of exacerbations have been proposed as predictors of patients with chronic obstructive pulmonary disease (COPD) who may benefit from triple therapy (inhaled corticosteroid, long-acting β-agonist and long-acting muscarinic antagonist).

Methods: In a retrospective cohort analysis we examined the profiles of COPD patients from the UK Clinical Practice Research Datalink (CPRD) and US Optum Clinformatics™ Data Mart (Optum) databases with reference to exacerbation frequency and blood eosinophil distribution.

Results: Of the 31,437 (CPRD) and 383,825 (Optum) patients with COPD, 15,364 (CPRD) and 139,465 (Optum) met the eligibility criteria and were included.

Associations Between Headache-Free Days and Patient-Reported Outcomes Among Migraine Patients: A Cross-Sectional Analysis of Survey Data in Europe.

Introduction: Migraine imposes a substantial burden on patients, society, and healthcare systems. This study aimed to assess the associations between the number of headache-free days (HFDs) and health-related quality of life (HRQoL), work productivity and activity impairment (WPAI), and healthcare resource utilization (HRU) in patients with migraine in the EU5 (France, Germany, Italy, Spain, and the United Kingdom).

Methods: This retrospective cross-sectional study collected survey responses from adults aged ≥ 18 years from the 2017 National Health and Wellness Survey (N = 62,000).

Burden of Migraine in Europe Using Self-Reported Digital Diary Data from the Migraine Buddy© Application.

Introduction: Migraine is a neurological disease characterized by recurring attacks that can cause severe disabling pain. This study described the burden of migraine as reported by individuals with migraine in the real world using a mobile application.

Methods: A retrospective, cross-sectional analysis was conducted using data captured through the Migraine Buddy© smartphone application from adult, self-diagnosed individuals with migraine in 17 European countries.

Effect of interferon beta-1a subcutaneously three times weekly on clinical and radiological measures and no evidence of disease activity status in patients with relapsing-remitting multiple sclerosis at year 1.

Background: In the PRISMS study, interferon beta-1a subcutaneously (IFN β-1a SC) reduced clinical and radiological disease burden at 2 years in patients with relapsing-remitting multiple sclerosis. The study aimed to characterize efficacy of IFN β-1a SC 44 μg and 22 μg three times weekly (tiw) at Year 1.

Methods: Exploratory endpoints included annualized relapse rate (ARR), 3-month confirmed disability progression (1-point Expanded Disability Status Scale increase if baseline was < 6.

Patients' perspective on the burden of migraine in Europe: a cross-sectional analysis of survey data in France, Germany, Italy, Spain, and the United Kingdom.

Background: Migraine is a distinct neurological disease that imposes a significant burden on patients, society, and the healthcare system. This study aimed to characterize the incremental burden of migraine in individuals who suffer from ≥4 monthly headache days (MHDs) by examining health-related quality of life (HRQoL), impairments to work productivity and daily activities, and healthcare resource utilization (HRU) in the EU5 (France, Germany, Italy, Spain, United Kingdom).

Methods: This retrospective cross-sectional study used data from the 2016 National Health and Wellness Survey (NHWS; N = 80,600).

Predictive value of early magnetic resonance imaging measures is differentially affected by the dose of interferon beta-1a given subcutaneously three times a week: an exploratory analysis of the PRISMS study.

Background: On-treatment magnetic resonance imaging lesions may predict long-term clinical outcomes in patients receiving interferon β-1a. This study aimed to assess the effect of active T2 and T1 gadolinium-enhancing (Gd+) lesions on relapses and 3-month confirmed Expanded Disability Status Scale (EDSS) progression in the PRISMS clinical trial.

Methods: Exploratory analyses assessed whether active T2 and T1 Gd + lesions at Month 6, or active T2 lesions at Month 12, predicted clinical outcomes over 4 years in PRISMS.

Early MRI results and odds of attaining 'no evidence of disease activity' status in MS patients treated with interferon β-1a in the EVIDENCE study.

Introduction: 'No evidence of disease activity' (NEDA) is increasingly used as a treatment target with disease-modifying drugs for relapsing multiple sclerosis.

Methods: This post-hoc analysis of the randomised EVIDENCE trial compared interferon beta-1a injected subcutaneously three times weekly (IFN β-1a SC tiw) with interferon β-1a injected intramuscularly once weekly (IFN β-1a IM qw) on NEDA and clinical activity-free (CAF) status. The influence of the frequency of magnetic resonance imaging (MRI) scanning on NEDA and the effect of baseline T1 gadolinium-enhancing (Gd+) lesions on NEDA and CAF were also investigated.

Secondary Use of Data: Non-Interventional Study Best Practices in Planning and Protocol Development.

Well established guidelines already exist that address best practices for Non-Interventional Study (NIS) design and methods. These guidelines provide advice on things to consider while designing a study and developing a protocol, but do not necessarily capture specific details related to the implementation of NIS. The intent of this paper is to propose a best practice for conducting secondary use of data NIS.

Minimization of CYP2D6 Polymorphic Differences and Improved Bioavailability via Transdermal Administration: Latrepirdine Example.

Purpose: Transdermal delivery has the potential to offer improved bioavailability by circumventing first-pass gut and hepatic metabolism. This study evaluated the pharmacokinetics of oral immediate release and transdermal latrepirdine in extensive and poor CYP2D6 metabolizers (EM/PM).

Methods: Latrepirdine transdermal solution was prepared extemporaneously.

The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: item reduction and further validation.

Purpose: The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire is a patient and parent-completed disease-specific instrument used in Hunter syndrome (mucopolysaccharidosis II), a rare paediatric progressive multi-systemic lysosomal storage disease. The objective of this study was to shorten the number of items of the Questionnaire to reduce response burden while maintaining its content validity.

Methods: Data collected in a clinical trial were used.

Steady-state pharmacokinetics of sirolimus in stable adult Chinese renal transplant patients.

This open-label, nonrandomized study was conducted to evaluate the steady-state pharmacokinetics of sirolimus in 24 stable Chinese renal transplant patients receiving daily oral maintenance doses of sirolimus (1-4 mg). Repeated trough and serial whole blood sirolimus concentrations over a 24-hour dosing interval were collected and assayed using high-performance liquid chromatography with tandem mass spectrometry (HPLC/MS/MS). Non-compartmental analysis (NCA) was employed to calculate sirolimus pharmacokinetic parameters.

The impact of Hunter syndrome (mucopolysaccharidosis type II) on health-related quality of life.

Background: Hunter syndrome (mucopolysaccharidosis type II (MPS II)) is a rare metabolic disease that can severely compromise health, well-being and life expectancy. Little evidence has been published on the impact of MPS II on health-related quality of life (HRQL). The objective of this study was to describe this impact using the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) questionnaire and a range of standard validated questionnaires previously used in paediatric populations.

Pharmacokinetics, safety, and tolerability of maraviroc in HIV-negative subjects with impaired renal function.

Purpose: This open-label, nonrandomized, parallel-group study was conducted to explore the pharmacokinetics, safety, and tolerability of maraviroc in renally impaired subjects.

Methods: Subjects with normal renal function; mild, moderate, or severe renal impairment; or end-stage renal disease (ESRD) (n = 6 per group) were enrolled. Subjects with normal function (period 1), severe impairment, and ESRD received a single 300 mg dose of maraviroc.