Development and content validation of patient-reported outcomes tools for ulcerative colitis and Crohn's disease in adults with moderate-to-severe disease.

Background: Ulcerative colitis (UC) and Crohn's disease (CD) are associated with a range of symptoms that adversely affect health-related quality of life. This research aimed to develop and validate two patient-reported outcome (PRO) tools to assess signs and symptoms in patients with moderate-to-severe UC or CD.

Methods: PRO-UC and PRO-CD Diaries were developed in accordance with US Food and Drug Administration (FDA) recommendations.

The patient journey to diagnosis and treatment of congenital sucrase-isomaltase deficiency.

Purpose: Congenital sucrase-isomaltase deficiency (CSID) is a rare genetic disorder characterized by a deficiency of the sucrase-isomaltase (SI) enzyme complex within the brush border membrane of the small intestine. Mutations in the SI gene result in abnormal synthesis and/or incorrect transport of the SI enzyme. Patients with CSID generally have reduced sucrase activity, but levels of isomaltase activity range from absent to almost normal.

Patient perspectives on the pathway to psoriatic arthritis diagnosis: results from a web-based survey of patients in the United States.

Background: There are limited real-world data on the diagnostic experiences of patients with psoriatic arthritis (PsA), including medical care sought and potential barriers to diagnosis. We aim to describe patient experiences related to receiving a PsA diagnosis.

Methods: Ours was a mixed-method, 2-phase study.

Healthcare provider perspectives on diagnosing and treating adults with attention-deficit/hyperactivity disorder.

: This study examined adult attention-deficit/hyperactivity disorder (ADHD) screening and management patterns among healthcare provider (HCP) subgroups. : An online survey of US-based HCPs (neurologists, n = 200; nurse practitioners [NPs], n = 100; psychiatrists, n = 201; primary care physicians [PCPs], n = 201) was conducted from May to June 2017. The survey assessed issues relating to adult ADHD screening and management and HCP perceptions of factors influencing patient choice of pharmacotherapy.

The Patient Perspective: Unmet Treatment Needs in Adults With Attention-Deficit/Hyperactivity Disorder.

Objective: To characterize impairments in daily life experienced by pharmacologically treated adults with attention-deficit/hyperactivity disorder (ADHD) versus adults without ADHD and to identify unmet needs in ADHD treatment from the perspective of adults with ADHD.

Methods: Adults with ADHD taking prescription medication for ≥ 6 months and adults without ADHD agreed to participate in a cross-sectional online survey during December 2016. Participants with ADHD were stratified by their current ADHD medication: long-acting (LA) once daily, short-acting (SA) ≤ 2 times/d, and augmenters (AU; LA > 1 time/d, SA > 2 times/d, or LA plus SA).

Real-World Patient Experience on the Path to Diagnosis of Ankylosing Spondylitis.

Introduction: We describe the journey to diagnosis of ankylosing spondylitis (AS) from the patient perspective and examine differences in this journey by sex.

Methods: US adults aged ≥ 18 years with a self-reported AS diagnosis were recruited online through CreakyJoints, a patient support community, and ArthritisPower, a patient research registry. Respondents completed a web-based survey on sociodemographics, disease burden, and diagnosis history.

A Conceptual Model of Angelman Syndrome and Review of Relevant Clinical Outcomes Assessments (COAs).

Background: Angelman syndrome (AS) is a rare, neurological genetic disorder for which no clinical outcomes assessments (COAs) or conceptual models (CM) have been developed.

Objective: This study aimed to identify symptoms and impacts relevant and important in this patient population and develop a conceptual model of AS, and to evaluate the content validity of selected COA instruments with potential for inclusion in clinical studies of AS to capture treatment benefit.

Methods: For both concept elicitation (CE) and cognitive interviews (CI), caregivers of children, adolescents, and adults with AS and clinicians with AS experience were targeted.

Development of the pediatric daily ulcerative colitis signs and symptoms scale (DUCS): qualitative research findings.

Background: The purpose of this study is to develop patient-reported (PRO) and observer-reported (ObsRO) outcome measures of ulcerative colitis (UC) signs/symptoms in children aged 5-17 with mild/moderate UC. The daily ulcerative colitis signs and symptoms scale (DUCS) was developed in two phases. Phase I involved concept elicitation interviews with patients and healthcare providers, review of website posts and item generation.

Discrete Choice Experiment Attribute Selection Using a Multinational Interview Study: Treatment Features Important to Patients with Type 2 Diabetes Mellitus.

Introduction: Methods for discrete choice experiment (DCE) attribute and attribute-level selection have not yet been firmly established and are rarely reported in detail. This paper describes a qualitative study designed to inform the development of a DCE survey designed to examine preferences for glucagon-like peptide-1 receptor agonist (GLP-1RA) treatments among patients with type 2 diabetes mellitus.

Methods: The study involved a literature review, interviews with clinical experts, and interviews with GLP-1RA-experienced (i.

Glucagon-Like Peptide-1 Receptor Agonist Treatment Attributes Important to Injection-Experienced Patients with Type 2 Diabetes Mellitus: A Preference Study in Germany and the United Kingdom.

Introduction: This study assessed the relative importance of treatment-related attributes in influencing patient preferences for glucagon-like peptide-1 receptor agonists (GLP-1RAs) among injection-experienced type 2 diabetes mellitus (T2DM) patients in Germany and the United Kingdom.

Methods: T2DM patients experienced with injecting once-weekly (QW) exenatide or once-daily (QD) liraglutide completed an online discrete-choice experiment (DCE) survey. Patients chose between hypothetical blinded treatment profiles reflecting attributes of GLP-1RAs.

Glucagon-like Peptide-1 Receptor Agonist Treatment Attributes Important to Injection-Naïve Patients with Type 2 Diabetes Mellitus: A Multinational Preference Study.

Introduction: Glucagon-like peptide-1 receptor agonists (GLP-1RAs) differ in efficacy, side effects, dosing frequency, and device-related attributes. This study assessed the relative importance of treatment-related attributes in influencing preferences for GLP-1RAs among injection-naïve patients with type 2 diabetes mellitus (T2DM).

Methods: Injection-naïve T2DM patients from five countries completed a Web-based discrete choice experiment (DCE) survey.

The Caregiver Perspective on Paediatric ADHD (CAPPA) survey: Understanding sociodemographic and clinical characteristics, treatment use and impact of ADHD in Europe.

Background: Attention-deficit/hyperactivity disorder (ADHD) is a neurodevelopmental disorder that affects the lives of patients and their families. The Caregiver Perspective on Paediatric ADHD (CAPPA) survey was conducted to evaluate the burden associated with ADHD in Europe and to identify unmet needs. Here, we describe sociodemographic and clinical characteristics, treatment use and impact of ADHD.