This study explored patient and clinician perspectives on a new fixed-dose combination of macitentan tadalafil (M/T FDC) in once-daily single tablet for treatment pulmonary arterial hypertension (PAH). Qualitative semi-structured interviews were conducted during the open-label period global, phase 3 A DUE clinical trial that evaluated M/T FDC. subset enrolled patients (N = 26) site investigators 18 clinicians) interviewed. Patients received four tablets double-blind could be one three arms...

Abstract Background Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, genetic leading to impaired lysosomal function and neurodegeneration causing serious morbidity shortened life expectancy. The Clinical Severity Scale (NPCCSS) a 17 domain, disease-specific, clinician-reported outcome measure of severity progression. An abbreviated 5-domain NPCCSS scale has been developed (measuring Ambulation, Swallow, Cognition, Speech, Fine Motor Skills) the reliability established....

Objectives: This randomized, double-blind, placebo-controlled, multicenter, 2-period crossover study (two 6-week treatment periods separated by a 2-week washout period) evaluated the efficacy and safety of pregabalin (150 to 300 mg/d) for pain on walking in patients with painful diabetic peripheral neuropathy (DPN) who experienced while walking. Methods: Co-primary endpoints were: (1) mean score (last 7 daily diary scores, 0 10 numeric rating scale at end each (2) DPN (0 immediately after 50...

Abstract Background Robust outcome measures are needed to assess and monitor the impact of chronic low back pain (CLBP) on physical functioning. The Roland Morris Disability Questionnaire (RMDQ) is a well-established measure designed capture impacts everyday functioning, with particular emphasis It has documented evaluation psychometric properties. However, there no qualitative evidence confirm content validity tool, nor have changes made for electronic administration been debriefed in...

Sleep disturbance is a common experience in fibromyalgia (FM). The field lacks sleep specific patient reported outcome (PRO) measure developed and validated FM population. study objective to gain an in-depth understanding of develop PRO it.Research involved the following stages: 1) A literature review conducted identify key concepts associated with measures that have been used assess this; 2) Qualitative interviews therapeutic area experts; 3) Focus groups patients who experienced...

Abstract Background Classic Galactosemia is a rare, autosomal recessive disease in which galactose not metabolized properly due to severe deficiency/absence of the galactose-1-phosphate uridylyltransferase (GALT) enzyme, converting an aberrant and toxic metabolite, galactitol. Newborn screening timely galactose-restricted diet can resolve acute symptoms decrease fatalities. However, despite this, significant chronic, progressive morbidities remain have real impact upon daily life. To better...

Abstract Background Niemann-Pick disease type C (NPC) is a debilitating condition that impacts patients’ and caregivers’ quality of life (QOL) reduces the patient’s expectancy. Since there little qualitative research from perspective patients family caregivers, this study explored impact NPC on daily lives to understand burden disease. Results A survey caregivers for with adult (n = 49; patient age: 13 months–65 years) assessed severity, importance symptoms, how symptoms impacted activities...

The Multidimensional Daily Diary of Fatigue-Fibromyalgia-17 instrument (MDF-Fibro-17) has been developed for use in fibromyalgia (FM) clinical studies and includes 5 domains: Global Fatigue Experience, Cognitive Fatigue, Physical Motivation, Impact on Function. Psychometric properties the MDF-Fibro-17 needed to demonstrate appropriateness using this are presented. analyses were conducted evaluate factor structure, reliability, validity, responsiveness data from a Phase 2 study FM patients (N...

Fibromyalgia (FM), a disorder characterized by chronic widespread pain and tenderness, affects greater than five million individuals in the United States alone. Patients experience multiple symptoms addition to pain, among them, fatigue is one of most bothersome disabling. There growing body literature suggesting that multidimensional concept. Currently, our knowledge, no Patient Reported Outcome (PRO) measure FM-related meets Food Drug Administration (FDA) requirements support product label...

Objective: Collecting data that helps evaluate different types of pain may improve physicians' decision-making with regard to treatment selection and on-going monitoring patients. To date, no chronic assessments have been widely implemented in primary care. The aim this study was psychometrically validate the electronic Chronic Pain Questions (eCPQ) a care setting.Research design methods: All men women ≥18 years arriving at two similar clinics southeastern Michigan were invited participate....

Abstract Background Classic Galactosemia (CG) is a rare, autosomal recessive condition. Newborn screening and timely galactose-restricted diet can resolve acute symptoms decrease fatalities, but significant chronic, progressive morbidities remain significantly impact daily life. The objective of this study was to better understand the burden disease in children adults with CGs describe how evolve over time. Methods A total 49 individuals CG from United States (US) were included qualitative...

To explore, from the perspective of Study Partners (SPs; eg, caregivers) clinical trial participants with autism spectrum disorder (ASD), any changes experienced in socialization and communication over trial, how these manifested, impact had on autistic individual, SP, family. This helps interpret whether outcomes were meaningful.Interviews conducted SPs individuals ASD, without intellectual disability, 2 trials: 86 children (aged 5-12 years) or adolescents 13-17 who took part aV1ation...

The VinelandTM Adaptive Behavior Scale is often used in autism spectrum disorder (ASD) trials. Composite Score (VABS-ABC) the standardized overall score (the average of Socialization, Communication and Daily Living skills domains), 2-Domain (VABS-2DC) a novel outcome measure (average Socialization domains). A within-person meaningful change threshold (MCT) has not been established for VABS-2DC. This paper presents quantitative qualitative interpretation what constitutes these scores to...

Introduction: Fatigue is an important fibromyalgia (FM) symptom, but existing measures of fatigue are unlikely to meet regulatory standards for clinical trial use. We describe the development and validation Daily Diary Symptoms—Fibromyalgia (DFS-Fibro), a 24-hour recall, patient-reported outcome (PRO) measure in FM that administered electronically (ePRO). Methods: There were 3 phases work: 1) item generation based on concept elicitation interviews with patients, relevance confirmed by expert...

Classic Galactosemia (CG) is an autosomal recessive rare metabolic disease resulting in inability to metabolize the simple sugar galactose. Newborn screening and implementation of a galactose-restricted diet have decreased mortality acute symptoms during perinatal period.

e23196 Background: Clinical Outcome Assessment (COA) conceptual gap analyses for oncology are complex and time consuming. Artificial Intelligence may efficiently reduce to completion of such analyses. We aimed assess two AI models’ performance literature screening identify relevant qualitative research. also compared accuracy run-time both models. Methods: manually curated a dataset title/abstract (n = 1,700 study references) across 17 landscape reviews. Among these, 11 reviews 951 spanning...

Severe hypertriglyceridemia (sHTG) is a rare condition, complicated by episodes of acute pancreatitis (AP), which can cause pain and/or life-threatening multi-organ dysfunction. Currently, there are no disease-specific patient-reported outcome (PRO) measures evaluating symptoms or dietary impact for this condition. The objective study was to explore and impacts sHTG AP develop new capture the condition using patient language. In-depth, semi-structured concept elicitation interviews were...

The patient burden of completing large numbers patient-reported outcome (PRO) items is often a concern; particularly when PROs must be completed daily, or at multiple timepoints over long studies. However, as ePRO and mPRO (technology that utilizes patients’ personal tablets smartphones) methods advance, PRO completion becomes quicker easier. How does it actually take patients to complete ePROs? burdensome do find completion? allows collection the time taken set items. We summarise data from...

201 Background: To explore the emotional response of men with a recent metastatic Castration Resistant Prostate Cancer (mCRPC) diagnosis, burden monitoring their condition prior to additional treatment for metastasis (“watch and wait”), impact on caregivers. Methods: In-depth, qualitative, one-to-one interviews 25 diagnosis mCRPC from US (n = 4), France 12) Germany 9). Additional were conducted 12 participants’ primary support person (PSP) - friend/family member who provided participants...