Experience and impact of stigma in people with chronic hepatitis B: a qualitative study in Asia, Europe, and the United States.

Background: People with chronic hepatitis B (CHB) commonly experience social and self-stigma. This study sought to understand the impacts of CHB-related stigma and a functional cure on stigma.

Methods: Adults with CHB with a wide range of age and education were recruited from 5 countries and participated in 90-minute qualitative, semi-structured interviews to explore concepts related to CHB-associated stigma and its impact.

Upscaling DAC Hubs with Wind Energy and CO Mineral Storage: Considerations for Large-Scale Carbon Removal from the Atmosphere.

Continued fossil fuel emissions will increase CO concentrations in the atmosphere and could require removal of 10 Gt of CO per year or more to reach IPCC global climate goals. Large-scale construction of direct air capture (DAC) hubs to scrub CO from the atmosphere paired with geological storage is a prominent approach to potentially meet this target. We consider one location for theoretical scale-up of a DAC hub: the Kerguelen plateau in the Southern Indian Ocean which has high-potential renewable energy resources (wind) and large volumes of basalt rock for mineral storage.

The value of co-creating a clinical outcome assessment strategy for clinical trial research: process and lessons learnt.

Background: In support of UCB pharmaceutical research programs, the aim of this research was to implement a novel process for patient involvement in a multidisciplinary research group to co-create a clinical outcome assessment strategy to accurately reflect the experience of people living with early-stage Parkinson's. Patient experts were an integral part of the decision-making process for patient-reported outcome (PRO) research and instrument development.

Methods: In partnership with two patient organizations (Parkinson's UK and the Parkinson's Foundation), 6 patient experts were recruited into a multidisciplinary research group alongside clinical, patient engagement and involvement, regulatory science, and outcome measurement experts.

An Updated Review of Food and Drug Administration Warning and Untitled Letters for Clinical Outcome Assessment Claims Between 2013 and 2021.

Objectives: We aimed to ascertain the frequency and types of Food and Drug Administration (FDA)-identified clinical outcome assessment (COA) violations in US pharmaceutical promotional materials from 2013 to 2021 by updating the review by Symonds et al (covering the period 2006-2012 for patient-reported outcomes [PRO] measures), expanding to infringements across all types of COAs, and grouping by efficacy or overall patient experience.

Methods: Warning letters (WLs) and untitled letters (ULs) issued by the FDA's Office of Prescription Drug Promotion were reviewed for COA violations and classified by their type: (1) COA measure not fit for purpose, (2) issue with study design or interpretation of results, and (3) inadequate statistical analysis.

Results: From 2013 to 2021, the FDA issued 22 WLs and 65 ULs.

Recommendations on the Selection, Development, and Modification of Performance Outcome Assessments: A Good Practices Report of an ISPOR Task Force.

In evaluating the clinical benefit of new therapeutic interventions, it is critical that the treatment outcomes assessed reflect aspects of health that are clinically important and meaningful to patients. Performance outcome (PerfO) assessments are measurements based on standardized tasks actively undertaken by a patient that reflect physical, cognitive, sensory, and other functional skills that bring meaning to people's lives. PerfO assessments can have substantial value as drug development tools when the concepts of interest being measured best suit task performance and in cases where patients may be limited in their capacity for self-report.

Psychometric Validation and Meaningful Change Thresholds of the New Nasal Polyposis Symptom Diary.

Objectives: The Nasal Polyposis Symptom Diary (NPSD) is a novel and short patient-reported outcome (PRO) tool specifically developed to assess important and relevant symptoms reported by patients with chronic rhinosinusitis with nasal polyposis (CRSwNP). We evaluated the psychometric properties of 4 predefined NPSD-derived scores intended to support symptom-improvement assessments of investigational therapies for inclusion in product labeling.

Methods: Five hundred eighteen patients with severe CRSwNP from a Phase III clinical trial (NCT03401229) completed the NPSD, comprising 11 items: 8 symptom-specific, 2 symptom-impact, and 1 optional medication-compliance.

Psychometric evaluation of the Symptoms of Infection with Coronavirus-19 (SIC): results from a cross-sectional study and a phase 3 clinical trial.

Background: The Symptoms of Infection with Coronavirus-19 (SIC) is a 30-item patient-reported outcome (PRO) measure scored by body system composites to assess signs/symptoms of coronavirus disease 2019 (COVID-19). In addition to cross-sectional and longitudinal psychometric evaluations, qualitative exit interviews were conducted to support the content validity of the SIC.

Methods: In a cross-sectional study, adults diagnosed with COVID-19 in the United States completed the web-based SIC and additional PRO measures.

Development and early qualitative evidence of two novel patient-reported outcome instruments to assess daily functioning in people with early-stage Parkinson's.

Background: Previous research on concepts that are important to people living with early-stage Parkinson's indicated that 'functional' slowness, fine motor skills, and subtle gait abnormalities are cardinal concepts that are not comprehensively captured by existing patient-reported outcome (PRO) instruments that are used in clinical practice and research to assess symptoms and daily functioning within this patient population. We sought to develop novel PRO instruments to address this unmet need.

Methods: PRO instrument development was led by a multidisciplinary research group, including people living with Parkinson's (termed 'patient experts'), as well as patient engagement and involvement, regulatory science, clinical, and outcome measurement experts.

A global examination of clinical ladder programs - A synthesis of commonalities and opportunities for standardization.

Background: Clinical ladder programs (CLPs) have been part of the nursing profession for nearly 4 decades. However, the structure and implementation of CLPs vary widely throughout healthcare systems. CLPs are a valuable factor in nurse retention and employee satisfaction initiatives, but globally replicating and measuring their impact is difficult due to lack of standardization.

Development of a novel patient-reported outcome measure to assess signs and symptoms of COVID-19.

Background: Given the urgent need for vaccines and treatments for coronavirus disease 2019 (COVID-19), the Symptoms of Infection with Coronavirus-19 (SIC), a comprehensive, patient-reported outcome (PRO) measure of signs and symptoms associated with COVID-19, was developed in full alignment with current US regulatory guidance to support evaluations of vaccines and treatments in development.

Methods: An initial version of the SIC was developed to address concepts identified through a targeted literature review and consultation with experts in infectious diseases and clinicians routinely managing COVID-19 in a hospital setting. A qualitative study was conducted in sites in the United States among 31 participants aged ≥ 18 years who were English-speaking and willing and able to provide informed consent and a self-reported history by telephone or online method.

Patient Experience in Early-Stage Parkinson's Disease: Using a Mixed Methods Analysis to Identify Which Concepts Are Cardinal for Clinical Trial Outcome Assessment.

Introduction: Qualitative research on patient experiences in early-stage Parkinson's disease (PD) is limited. It is increasingly acknowledged that clinical outcome assessments used in trials do not fully capture the range of symptoms/impacts that are meaningful to people with early-stage PD. We aimed to conceptualize the patient experience in early-stage PD and identify, from the patient perspective, those cardinal symptoms/impacts which might be more useful to measure in clinical trials.

Developing a conceptual model of symptoms and impacts in progressive fibrosing interstitial lung disease to evaluate patient-reported outcome measures.

Background: An understanding of the experience of patients with progressive fibrosing interstitial lung disease (PF-ILD) is needed to select appropriate patient-reported outcome measures (PROMs) to evaluate treatment effect in clinical trials.

Methods: A systematic literature review was conducted to develop a preliminary conceptual model of the symptoms experienced by patients with PF-ILD and the impacts the disease has on them. An online survey and consensus meetings were then conducted with 12-14 stakeholders (patients, clinicians, regulatory and payer advisors) to refine the conceptual model and critically appraise how key concepts should be measured by PROMs.

Measuring the patient experience of chronic rhinosinusitis with nasal polyposis: qualitative development of a novel symptom diary.

Background: This qualitative study assessed the experience of patients with chronic rhinosinusitis with nasal polyposis (NP) to inform the development of a novel symptom diary for clinical study use.

Methods: Concept elicitation and cognitive interviews were conducted with patients who had a physician-verified diagnosis of NP and a history of intranasal corticosteroid use. Concepts were identified via open-ended and follow-up questions.

Development and content validation of a symptom assessment for eosinophilic gastritis and eosinophilic gastroenteritis in adults and adolescents.

Background: A patient reported outcome (PRO) instrument with evidence of validity and reliability for assessing symptoms of eosinophilic gastritis (EG) and eosinophilic gastroenteritis (EGE) is needed to measure treatment benefit in clinical trials. The aim of this research is to develop an EG/EGE symptom PRO instrument for patients aged 12 and above.

Methods: The Symptom Assessment for Gastrointestinal Eosinophilic Diseases (SAGED) was developed through a literature review, discussions with expert clinicians, and concept elicitation and cognitive debriefing interviews with patients.

Dataset of characteristic remanent magnetization and magnetic properties of early Pliocene sediments from IODP Site U1467 (Maldives platform).

This data article describes data of magnetic stratigraphy and anisotropy of isothermal remanent magnetization (AIRM) from "Magnetic properties of early Pliocene sediments from IODP Site U1467 (Maldives platform) reveal changes in the monsoon system" [1]. Acquisition of isothermal magnetization on pilot samples and anisotropy of isothermal remanent magnetization are reported as raw data; magnetostratigraphic data are reported as characteristic magnetization (ChRM).

Creating a personalized evaluation framework for patient-reported outcomes: an illustration using the EQ-5D visual analogue scale.

Background: This paper outlines the creation of an intuitive, personalized evaluation framework for Patient-Reported Outcomes, using the EQ-5D visual analog scale (VAS) as an illustration.

Methods: A draft framework asked patients to divide and label the EQ-5D-VAS into different levels or categories of health. Comprehension of the framework and patient-defined health level labels, and how they map onto the EQ-5D-VAS, were tested through in-person, semi-structured interviews with individuals self-reporting cardiovascular disease.

Measurement Equivalence of Patient-Reported Outcome Measures Migrated to Electronic Formats: A Review of Evidence and Recommendations for Clinical Trials and Bring Your Own Device.

A growing number of clinical trials employ electronic media, in particular smartphones and tablets, to collect patient-reported outcome data. This is driven by the ubiquity of the technology, and an increased awareness of associated improvements in data integrity, quality and timeliness. Despite this, there remains a lingering question relating to the measurement equivalence of an instrument when migrated from paper to a screen-based format.

Mode of delivery in pregnancies with gastroschisis according to delivery institution.

We aim to compare the mode of delivery in pregnancies with gastroschisis delivered in nonacademic institutions with those delivered in an academic center. Chart review from 2008 to 2015 was performed. Cesarean delivery rate (CDR), attempted vaginal delivery rate (AVR), planned cesarean rate (PCR) and adverse neonatal outcomes were compared among pregnancies with gastroschisis delivered in nonacademic hospitals with those delivered in an academic institution.

Fit for purpose and modern validity theory in clinical outcomes assessment.

Purpose: The US Food and Drug Administration (FDA), as part of its regulatory mission, is charged with determining whether a clinical outcome assessment (COA) is "fit for purpose" when used in clinical trials to support drug approval and product labeling. In this paper, we will provide a review (and some commentary) on the current state of affairs in COA development/evaluation/use with a focus on one aspect: How do you know you are measuring the right thing? In the psychometric literature, this concept is referred to broadly as validity and has itself evolved over many years of research and application.

Review: After a brief introduction, the first section will review current ideas about "fit for purpose" and how it has been viewed by FDA.

Clinician-Reported Outcome Assessments of Treatment Benefit: Report of the ISPOR Clinical Outcome Assessment Emerging Good Practices Task Force.

A clinician-reported outcome (ClinRO) assessment is a type of clinical outcome assessment (COA). ClinRO assessments, like all COAs (patient-reported, observer-reported, or performance outcome assessments), are used to 1) measure patients' health status and 2) define end points that can be interpreted as treatment benefits of medical interventions on how patients feel, function, or survive in clinical trials. Like other COAs, ClinRO assessments can be influenced by human choices, judgment, or motivation.

The abrupt onset of the modern South Asian Monsoon winds.

The South Asian Monson (SAM) is one of the most intense climatic elements yet its initiation and variations are not well established. Dating the deposits of SAM wind-driven currents in IODP cores from the Maldives yields an age of 12. 9 Ma indicating an abrupt SAM onset, over a short period of 300 kyrs.

Focusing on Core Patient-Reported Outcomes in Cancer Clinical Trials: Symptomatic Adverse Events, Physical Function, and Disease-Related Symptoms.

Cancer clinical trials have relied on overall survival and measures of tumor growth or reduction to assess the efficacy of a drug. However, benefits are often accompanied by significant symptomatic toxicities. The degree to which a therapy improves disease symptoms and introduces symptomatic toxicity affects how patients function in their daily lives.