Experience of switching from a daily to a less frequent administration of injection treatments.

Background: Daily injections of recombinant human growth hormone are the standard of care to treat growth failure due to pediatric growth hormone deficiency (GHD). While effective, daily injections are burdensome and can compromise adherence. In recent years, novel injection treatments requiring less frequent administration for growth hormone deficiency (GHD) have been developed.

Development and content validation of the Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQ): patient-reported and caregiver-reported outcome measures.

Background: Evolving regulatory guidelines recommend routine assessment of the acceptability of pediatric oral medicines throughout clinical development processes. However, such assessment is problematic owing to a lack of standard methods or criteria that define acceptability for children and their caregivers. This research aimed to identify the attributes of acceptability for targeted oral formulation types that are important to children, and to develop content-valid patient- and caregiver-reported outcome acceptability measures for use in the context of clinical drug development.

A novel patient-reported outcomes instrument assessing the side effects of peanut oral immunotherapy.

Background: Patients treated with peanut oral immunotherapy (OIT) may experience adverse reactions, particularly during up-dosing.

Objective: To develop the Side Effects of Peanut Oral Immunotherapy Diary (SEPOD), an electronic questionnaire assessing the daily side effects of peanut OIT in clinical trials.

Methods: Content and design of the SEPOD were informed by empirical literature review and meetings with 3 allergy-immunology experts.

Factors Driving Patient Preferences for Growth Hormone Deficiency (GHD) Injection Regimen and Injection Device Features: A Discrete Choice Experiment.

Introduction: The daily injection burden of recombinant human growth hormone (r-hGH) replacement therapy to treat growth hormone deficiency (GHD) may reduce compliance and limit treatment benefit. Research is needed to evaluate patient preferences for GHD injection regimen and device features.

Objective: Quantitatively evaluate factors driving preferences for r-hGH injection regimen and device features among pediatric (3-17 years, and caregivers) and adult (≥25 years) patients with GHD using a discrete choice experiment (DCE) approach.

Exploring and Leveraging Known Resources to Support Pediatric Clinical Outcomes Assessment (COA) Development.

Background: Best practices for pediatric clinical outcomes assessment (COA) development rely on guidelines that have been developed for adult populations. While some useful resources are available to support pediatric COA development, this information has primarily come from within the measurement development field. To our knowledge, no research has explored the experiences of professionals from other disciplines who interact with children on a routine basis.

Content Validity for the VVSymQ Instrument: A New Patient-Reported Outcome Measure for the Assessment of Varicose Veins Symptoms.

Background: Varicose veins are common and can impact patients' quality of life, but consensus regarding the evaluation of varicose vein symptoms is lacking and existing measures have limitations.

Objective: This research aimed to develop and establish the content validity of a new electronic patient-reported outcome (PRO) measure, the VVSymQ instrument, to assess symptoms of superficial venous insufficiency (varicose veins) in clinical trials.

Methods: The development of the VVSymQ instrument began with qualitative interviews with patients based on the symptom domain of the VEINES-QOL/Sym, an existing PRO instrument for chronic venous disorders of the leg.

Cognitive Testing of an Electronic Version of the Faces Pain Scale-Revised with Pediatric and Adolescent Sickle Cell Patients.

Background: Patient diaries and pain scales can capture the course and complications of pain managed at home in children. The Faces Pain Scale-Revised (FPS-R) is a validated scale showing reliability in children, but it has not been validated in children with sickle cell disease (SCD).

Objective: The purpose of this study was to evaluate comprehension and usability of an electronic modified version of the FPS-R among pediatric patients with SCD.

Psychometric Evaluation of a New Patient-Reported Outcome (PRO) Symptom Diary for Varicose Veins: VVSymQ(®) Instrument.

Objective: To evaluate the psychometric properties of the VVSymQ(®) instrument, a new 5-item patient-reported outcome (PRO) measure for symptoms of varicose veins.

Method: The VVSymQ(®) electronic daily diary was administered to outpatients who received routine treatment for varicose veins (N = 40). Compliance with diary administration and item score variability, reliability, construct validity, sensitivity to change, and clinically meaningful change were evaluated.

Practical Considerations for the Use of Clinical Outcome Assessments (COAs) in Pediatric Clinical Research: Examples From Pediatric Gastroenterology.

Clinical outcome assessments (COAs), including patient-reported outcome (PRO) measures, are routinely used in drug development and other clinical research initiatives to assess the impact of treatment on patient health and well-being. The FDA Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims (2009), the European Medicines Agency's Reflection Paper on the Regulatory Guidance for the Use of Health-Related Quality of Life Measures in the Evaluation of Medicinal Products (2005), and the International Society for Pharmacoeconomics and Outcomes Research PRO Good Research Practices for the Assessment of Children and Adolescence Task Force (2013) outline key considerations and good measurement principles that are relevant to the selection and use of COAs in a pediatric population. However, challenges remain in the appropriate selection and use of COAs to assess treatment benefit in pediatric clinical research.

The VVSymQ® instrument: Use of a new patient-reported outcome measure for assessment of varicose vein symptoms.

Introduction: No existing patient-reported outcome instrument focuses solely on assessment of varicose veins symptoms that are bothersome to patients.

Methods: The VVSymQ® instrument is a five-item patient-reported outcome that assesses symptoms most important to patients with varicose veins (heaviness, achiness, swelling, throbbing and itching). This paper describes how the VVSymQ® instrument was incorporated into an electronic daily diary to monitor key outcomes over time and capture treatment benefit in two randomized, controlled, phase 3 clinical trials.

Development and preliminary validation of the Adult Asthma Adherence Questionnaire™.

Background: Asthma medication adherence is related to better asthma outcomes, but identification of suboptimal patient adherence behavior is not standardized in clinical settings.

Objective: [corrected] The purpose of this study was to develop a practical questionnaire that reflects nonadherence risk and identifies potential adherence barriers.

Methods: A questionnaire that included 20 potential adherence questions was completed by 420 adult patients with asthma who filled a prescription for an inhaled corticosteroid (ICS) and a short-acting beta agonist (SABA) in the previous 6 months.

Item-level psychometric properties for a new patient-reported psoriasis symptom diary.

Objectives: This research evaluated the psychometric properties of a new Psoriasis Symptom Diary, identified diary responder definitions for use in determining whether a patient has experienced clinically meaningful change, and refined diary item content for use in future clinical trials.

Methods: The Psoriasis Symptom Diary was administered in a phase 2 clinical trial of AIN457 to US adult outpatients (N = 172) with physician-diagnosed moderate to severe chronic plaque-type psoriasis. Participant compliance with daily diary administration and item score variability, reliability, construct and discriminant validity, sensitivity to change, and interpretation were all evaluated.

Single-item electronic administration of the SF-36v2 Health Survey.

Purpose: To evaluate the psychometric properties and factor structure of a computerized electronic version of the SF-36v2 Health Survey (SF-36v2) with items administered one-per-page versus the traditional grid format used in the paper-and-pencil version in a sample of physician-diagnosed headache patients.

Methods: Patients (N = 180) completed the SF-36v2 administered as part of a broader study of health outcomes. Scaling assumptions, reliability, factor structure, and the tool's ability to discriminate between headache pain severity groups were examined.

Cognitive testing and readability of an item bank for measuring the impact of headache on health-related quality of life.

Background: Patient-reported outcomes (PRO) measures should be valid and accessible to a wide audience.

Objective: Cognitive item testing and readability studies were conducted to evaluate how adult headache sufferers (N = 9) understood and responded to the Headache Impact Test (HIT™) item bank, a PRO measure for headache that serves as the source of item content for the HIT-6™ (a widely used six-item short-form measure of headache impact with more than 30 language translations), and the Dynamic Health Assessment Headache Impact Test (DYNHA® HIT™) [a computerized adaptive test (CAT) of headache impact].

Methods: During cognitive interviews, participants were asked to 'think aloud' as they read survey instructions, completed items, and formulated responses.

A computerized adaptive version of the SF-36 is feasible for clinic and Internet administration in adults with HIV.

DYNHA SF-36 is a computerized adaptive test version of the SF-36 Health Survey. The feasibility of administering a modified DYNHA SF-36 to adults with HIV was evaluated with Johns Hopkins University Moore (HIV) Clinic patients (N=100) and Internet consumer health panel members (N=101). Participants completed the DYNHA SF-36, modified to capture seven health domains [(physical function (PF), role function (RF, without physical or emotional attribution), bodily pain (BP), general health, vitality (VT), social function (SF), mental health (MH)], and scored to produce two summary components [Physical Component Summary (PCS), Mental Component Summary (MCS)].

Development of a computerized adaptive test to assess health-related quality of life in adults with asthma.

Objective: The purpose of this research was to calibrate an item bank for a computerized adaptive test (CAT) of asthma impact on health-related quality of life (HRQOL), test CAT versions of varying lengths, conduct preliminary validity testing, and evaluate item bank readability.

Methods: Asthma Impact Survey (AIS) bank items that passed focus group, cognitive testing, and clinical and psychometric reviews were administered to adults with varied levels of asthma control. Adults self-reporting asthma (N = 1106) completed an Internet survey including 88 AIS items, the Asthma Control Test, and other HRQOL outcome measures.

Heuristic evaluation and usability testing of a computerized patient-reported outcomes survey for headache sufferers.

Objective: The aim of this study was to evaluate usability of a prototype tablet PC-administered computerized adaptive test (CAT) of headache impact and patient feedback report, referred to as HEADACHE-CAT.

Materials And Methods: Heuristic evaluation specialists (n = 2) formed a consensus opinion on the application's strengths and areas for improvement based on general usability principles and human factors research. Usability testing involved structured interviews with headache sufferers (n = 9) to assess how they interacted with and navigated through the application, and to gather input on the survey and report interface, content, visual design, navigation, instructions, and user preferences.

A Computerized Asthma Outcomes Measure Is Feasible for Disease Management.

OBJECTIVE: To develop and test an online assessment referred to as the ASTHMA-CAT (computerized adaptive testing), a patient-based asthma impact, control, and generic health-related quality of life (HRQOL) measure. STUDY DESIGN: Cross-sectional pilot study of the ASTHMA-CAT's administrative feasibility in a disease management population. METHODS: The ASTHMA-CAT included a dynamic or static Asthma Impact Survey (AIS), Asthma Control Test, and SF-8 Health Survey.

Using Qualitative Research to Inform the Development of a Comprehensive Outcomes Assessment for Asthma.

BACKGROUND: Qualitative research can inform the development of asthma patient-reported outcome (PRO) measures and user-friendly technologies through defining measurement constructs, identifying potential limitations in measurement and sources of response error, and evaluating usability. OBJECTIVE: The goal of the current study was to inform the development of a comprehensive asthma PRO assessment with input from patients and clinical experts. METHOD: Self-reported adult asthma sufferers recruited from a 3,000 member New England-area research panel participated in either one of three focus groups (N=21) or individual cognitive item debriefing interviews (N=20) to discuss how asthma impacts their health-related quality of life (HRQOL), and provide feedback on a preliminary set of asthma impact survey items and prototype patient report.

Using qualitative research to inform the development of a comprehensive outcomes assessment for asthma.

Background: : Qualitative research can inform the development of asthma patient-reported outcome (PRO) measures and user-friendly technologies through defining measurement constructs, identifying potential limitations in measurement and sources of response error, and evaluating usability.

Objective: : To inform the development of a comprehensive asthma PRO assessment with input from patients and clinical experts.

Methods: : Self-reported adult asthma patients recruited from a 3000-member New England area research panel participated in either one of three focus groups (n = 21) or individual cognitive item debriefing interviews (n = 20) to discuss how asthma impacts their health-related quality of life (HR-QOL), and provide feedback on a preliminary set of asthma impact survey items and prototype patient reports.