Psychometric evaluation of the Adelphi Adherence Questionnaire (ADAQ©) in a type 2 diabetes mellitus population as a new measure of adherence and the complex factors influencing adherence in routine care.

Aims: To evaluate the psychometric properties of the Adelphi Adherence Questionnaire (ADAQ©) as an adherence measure in a diverse type 2 diabetes mellitus (T2DM) population in routine clinical practice.

Materials And Methods: Data were drawn from the Adelphi T2DM Disease Specific Programme™, a survey of physicians and adults with T2DM consulting in the United States, February-May 2021. Participants completed the ADAQ and single questions on medication satisfaction and convenience.

A literature review exploring whether meaningful change threshold estimates for patient-reported outcome (PRO) measures vary depending on baseline severity.

Purpose: Meaningful change thresholds for patient-reported outcome (PRO) measures may differ according to patients' baseline disease severity. This review aimed to explore methods used in the peer-reviewed literature to assess the relationship between baseline severity and meaningful change thresholds and the nature of this relationship.

Methods: A structured literature review was conducted to identify relevant abstracts published between 2018-2022 reporting estimation of PRO meaningful change thresholds by baseline disease severity.

Psychometric validation of the Chronic Ocular Pain Questionnaire (COP-Q).

Background: The Chronic Ocular Pain Questionnaire (COP-Q) is a newly developed patient-reported outcome (PRO) measure intended to assess symptoms and impacts associated with Chronic Ocular Surface Pain (COSP). This study assessed the psychometric properties of the COP-Q to determine the adequacy of the COP-Q as a 'fit-for-purpose' instrument to derive trial endpoints for future clinical studies in COSP.

Methods: Patients with COSP completed the COP-Q daily for four weeks on an electronic, touch-screen, tablet device as part of a longitudinal, observational study in the United States (N = 124).

Qualitative In-trial Interviews: Methods, Challenges, and Best Practice.

Qualitative in-trial interviews with clinical trial participants are a means of providing rich, in-depth patient experience data to supplement and complement data captured by clinical outcome assessments and other clinical trial efficacy endpoints. Such in-trial interview data can be used to build understanding of disease and treatment experiences, evaluate content validity of clinical outcome assessments, aid interpretation of scores and meaningful changes, inform trial design feasibility and operational considerations, and provide supportive evidence regarding safety, efficacy, and effectiveness. Despite the rapid growth of in-trial interviews as part of clinical development programs in the pharmaceutical industry in recent years, published guidelines regarding the methods, conduct, and implementation of in-trial interviews are scarce.

Development and Validation of a Patient-Reported Outcome Measure of the Impact of Chronic Hand Eczema on Health-Related Quality of Life: the Hand Eczema Impact Scale (HEIS).

Introduction: Chronic Hand Eczema (CHE) is an inflammatory skin disease that causes significant impact on health-related quality of life (HRQoL). The Hand Eczema Impact Scale (HEIS) is a new patient-reported outcome (PRO) measure designed to assess the impact of CHE on key domains of HRQoL. This study aimed to develop and evaluate content and psychometric validity of the HEIS.

Psychometric evaluation of the Adelphi Adherence Questionnaire (ADAQ©) in adults with osteoarthritis.

Background: Medication non-adherence is a common issue in chronic illness. The World Health Organization has recognized a need for a valid and reliable method of measuring adherence to understand and mitigate non-adherence. This study aimed to psychometrically evaluate the English version of the Adelphi Adherence Questionnaire (ADAQ©), a questionnaire designed to assess patient-reported medication adherence across multiple therapy areas, in patients with Osteoarthritis (OA).

Evaluation of validity, reliability and ability to detect change for the Hand Eczema Severity Index (HECSI) and evaluation of HECSI-75 and HECSI-90 as within-patient responder definitions.

Background: The Hand Eczema Severity Index (HECSI) is a Clinician-Reported Outcome measure of the severity of hand eczema (HE).

Objectives: This study aimed to evaluate the validity, reliability and ability to detect change of the HECSI, and the HECSI-75 and HECSI-90 as responder definitions.

Methods: Analyses were performed using data from a sample of n = 258 patients with Chronic Hand Eczema (CHE) from a Phase 2b, randomised, double-blind, vehicle-controlled trial of delgocitinib cream, pooled across treatment groups.

Improving Assessment of Adherence Behaviors and Drivers: Targeted Literature Review and Concept Elicitation Interviews in Multiple Countries and Disease Populations.

Purpose: Medication adherence is crucial for achieving clinical goals. Medication adherence drivers and behaviors were explored across multiple conditions, countries, and medication schedules/modalities to develop a conceptual model of medication adherence, which could later be used to support development of a patient-reported outcome (PRO) measure of adherence.

Patients And Methods: Targeted review of qualitative literature identified important medication adherence concepts.

Psychometric evaluation of the near activity visual questionnaire presbyopia (NAVQ-P) and additional patient-reported outcome items.

Background: The Near Visual Acuity Questionnaire Presbyopia (NAVQ-P) is a patient-reported outcome (PRO) measure that was developed in a phakic presbyopia population to assess near vision function impacts. The study refined and explored the psychometric properties and score interpretability of the NAVQ-P and additional PRO items assessing near vision correction independence (NVCI), near vision satisfaction (NVS), and near vision correction preference (NVCP).

Methods: This was a psychometric validation study conducted using PRO data collected as part of a Phase IIb clinical trial (CUN8R44 A2202) consisting of 235 randomized adults with presbyopia from the US, Japan, Australia, and Canada.

Validation of the Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE): a new clinician reported outcome measure of CHE severity.

The Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE) is a novel Clinician-Reported Outcome measure that allows investigators to assess cross-sectional CHE global disease severity using clinical characteristics of erythema, scaling, lichenification/hyperkeratosis, vesiculation, oedema, and fissures as guidelines for overall severity assessment. This study aimed to evaluate the psychometric properties of the IGA-CHE for use as an outcome measure in CHE clinical trials and clinical practice. Psychometric analyses were performed using data from a sample of 280 patients with moderate to severe CHE from a phase 3 trial of delgocitinib cream, pooled across treatment groups.

Development and Psychometric Validation of a Patient-Reported Outcome Measure to Assess the Signs and Symptoms of Chronic Hand Eczema: The Hand Eczema Symptom Diary (HESD).

Introduction: Chronic Hand Eczema (CHE) is an inflammatory skin disease of the hands. The Hand Eczema Symptom Diary (HESD) is a new patient-reported outcome measure of worst severity of core CHE signs/symptoms. This study aimed to evaluate content and psychometric validity of the HESD.

Qualitative Research to Understand the Patient Experience and Evaluate Content Validity of the Chronic Ocular Pain Questionnaire (COP-Q).

Introduction: Chronic ocular surface pain (COSP) is described as a persistent, moderate-to-severe pain at the ocular surface lasting more than 3 months. Symptoms of COSP have a significant impact on patients' vision-dependent activities of daily living (ADL) and distal health-related quality of life (HRQoL). To adequately capture patient perspectives in clinical trials, patient-reported outcome (PRO) measures must demonstrate sufficient evidence of content validity in the target population.

Assessing asthma symptoms in children: qualitative research supporting the development of the Pediatric Asthma Diary-Child (PAD-C) and Pediatric Asthma Diary-Observer (PAD-O).

Background: Pediatric asthma has been identified by regulators, clinicians, clinical trial sponsors, and caregivers as an area in need of novel fit-for-purpose clinical outcome assessments (COAs) developed in accordance with the U.S. Food and Drug Administration's (FDA's) regulatory guidance for evaluating clinical benefit in treatment trials.

The Amblyopia Quality of Life (AmbQoL): Development and Content Validation of a Novel Health-Related Quality of Life Instrument for Use in Adult and Pediatric Amblyopia Populations.

Introduction: Amblyopia is a neurodevelopmental vision disorder, characterized by poor vision in one or both eyes. Given the lack of existing clinical outcome assessments (COA) considered fit-for-purpose for amblyopia clinical trials, this study developed new COAs to assess amblyopia symptoms and health-related quality of life (HRQoL) impacts in adult and pediatric amblyopia populations that conform with best practice standards and regulatory guidelines.

Methods: Findings from a targeted qualitative literature review informed the development of three versions of the new Amblyopia Quality of Life Questionnaire (AmbQoL): a patient-reported outcome measure (PRO) for individuals aged 13 years and older, a PRO for children aged 9-12 years, and an observer-reported outcome measure (ObsRO) for caregivers of children aged 4-8 years.

Comparison of Literature Review, Social Media Listening, and Qualitative Interview Research Methods in Generating Patient-Reported Symptom and Functional Impact Concepts of Presbyopia.

Introduction: To compare the insights obtained about the experience of individuals with presbyopia (age-related impaired near vision) across three different sources of qualitative data: a structured targeted literature review, a social media listening (SML) review, and qualitative concept elicitation (CE) interviews with individuals with presbyopia and healthcare professionals (HCPs). The number of concepts identified, depth of data, cost and time implications, and value of the patient insights generated were explored and compared for each method.

Methods: Keyword searches in bibliographic databases and review of abstracts identified 120 relevant publications; in-depth targeted literature review of the qualitative studies identified key symptoms/functioning concepts.

A qualitative study to explore the symptoms and impacts of Crohn's disease and to develop the Crohn's Disease Diary.

Purpose: To explore symptoms and disease impacts of Crohn's disease and to develop a new patient-reported outcomes (PRO) measure according to industry best practices.

Methods: A conceptual model of relevant symptoms experienced by patients with Crohn's disease was developed following a literature review. Three rounds of combined qualitative semi-structured concept elicitation and cognitive debriefing interviews with 36 patients (≥ 16 years) with Crohn's disease and 4 clinicians were conducted to further explore the most commonly reported and most bothersome symptoms to patients.

Content validation of a caregiver diary to monitor severity and recovery of pediatric patients with respiratory syncytial virus infection.

Background: Respiratory Syncytial Virus (RSV) is a leading cause of hospitalization and serious respiratory illness in infants/young children. The objectives of this study were to (1) identify important RSV-related signs of illness in infants that were observed by the parent/caregiver of the child and (2) assess content validity and usability of the Pediatric RSV Electronic Severity and Outcomes Rating System (PRESORS) to monitor signs of RSV-related illness.

Methods: Review of medical literature identified signs of pediatric RSV-related illness in PRESORS.

Qualitative study to characterize patient experience and relevance of patient-reported outcome measures for patients with metastatic synovial sarcoma.

Background: The outlook for patients with metastatic synovial sarcoma (mSS) is poor. Better understanding of patient experience in this setting, beyond clinical measures, may guide improvements in management. Validated patient-reported outcome (PRO) instruments specific to many types of cancer exist, but for rare cancers this is often not the case.

Understanding the visual function symptoms and associated functional impacts of phakic presbyopia.

Background: Presbyopia is defined as the age-related deterioration in the ability to focus on close objects, causing difficulty with near vision tasks. The study aim was to understand the lived experience of phakic presbyopia and identify all relevant visual function symptoms and associated functional impacts.

Methods: Fifty individuals with clinician-confirmed phakic presbyopia (US n = 30, France n = 10, Germany n = 10) and seven healthcare professionals (HCPs) participated in in-depth, face-to-face, qualitative concept elicitation interviews.

Evaluation of the content validity of patient-reported outcome (PRO) instruments developed for use with individuals with phakic presbyopia, including the Near Activity Visual Questionnaire-presbyopia (NAVQ-P) and the near vision correction independence (NVCI) instrument.

Background: Presbyopia is the age-related deterioration in the ability to focus on close objects. In order to develop a patient-reported outcome (PRO) instrument to assess near vision functioning, the Near Activity Visual Questionnaire (NAVQ) was adapted to incorporate modern technology (e.g.