Patient-Reported Outcome Consortium Research Articles

Desenvolvimento da Versão Portuguesa do Questionário ESF-1: Rastreio para Síndrome Metabólica

Introduction Metabolic Syndrome is a cluster of alterations, a consequence of a continuum of insulin resistance and chronic inflammation, being a predisposing factor for the development of several pathologies, including Type 2 Diabetes Mellitus and cardiovascular diseases. Due to the challenges inherent to organizing screenings in large populations, and the fact that recent biochemical parameters are not always available in different contexts, there is a tendency to use simple and cost-effective techniques, such as quick-administered questionnaires. The advantages relate to the ease of administration, the relatively short time, the need for blood samples and lower associated costs. There are several validated instruments for metabolic syndrome screening, although none are yet used in Portugal. One of those available is the “Encuesta de Identificación de Sujetos Metabólicamente Comprometidos en Fase-I”, and the present work aims to produce a linguistically and culturally adapted version for the Portuguese language, maintaining the conceptual equivalence of the original. Methodology For the translation, the procedures standardized by the Patient Reported Outcome Consortium were followed (Preparation, Translation, Reconciliation, Back-translation, Harmonization, Review, Cognitive Interview, Analysis, Final Review). Critical analysis The translated tool was applied to a convenience sample of the target population consisting of five individuals, and it was easy to understand and apply, with good acceptability, with only the need for minor adjustments to two questions. Conclusion This tool can be applied in a clinical and/or community setting for metabolic syndrome screening, proving to be of special interest in occupational health services, since it’s in the workplace that the population spends most of their adult life, optimizing resources, and promoting increasingly early referral and treatment. Furthermore, the healthier the workers, the more satisfied and productive they are. Keywords: Metabolic Syndrome; screening tool; Occupational Health.

Comparing patient global impression of severity and patient global impression of change to evaluate test–retest reliability of depression, non-small cell lung cancer, and asthma measures

PurposeScore reproducibility is an important measurement property of fit-for-purpose patient-reported outcome (PRO) measures. It is commonly assessed via test–retest reliability, and best evaluated with a stable participant sample, which can be challenging to identify in diseases with highly variable symptoms. To provide empirical evidence comparing the retrospective (patient global impression of change [PGIC]) and current state (patient global impression of severity [PGIS]) approaches to identifying a stable subgroup for test–retest analyses, 3 PRO Consortium working groups collected data using both items as anchor measures.MethodsThe PGIS was completed on Day 1 and Day 8 + 3 for the depression and non-small cell lung cancer (NSCLC) studies, and daily for the asthma study and compared between Day 3 and 10. The PGIC was completed on the final day in each study. Scores were compared using an intraclass correlation coefficient (ICC) for participants who reported “no change” between timepoints for each anchor.ResultsICCs using the PGIS “no change” group were higher for depression (0.84 vs. 0.74), nighttime asthma (0.95 vs. 0.53) and daytime asthma (0.86 vs. 0.68) compared to the PGIC “no change” group. ICCs were similar for NSCLC (PGIS: 0.87; PGIC: 0.85).ConclusionWhen considering anchor measures to identify a stable subgroup for test–retest reliability analyses, current state anchors perform better than retrospective anchors. Researchers should carefully consider the type of anchor selected, the time period covered, and should ensure anchor content is consistent with the target measure concept, as well as inclusion of both current and retrospective anchor measures.

Recommendations for the Electronic Migration and Implementation of Clinician-Reported Outcome Assessments in Clinical Trials

ObjectivesAlthough best practices from electronic patient-reported outcome (PRO) measures are transferable, the migration of clinician-reported outcome (ClinRO) assessments to electronic modes requires recommendations that address their unique properties, such as the user (eg, clinician), and complexity associated with programming of clinical content. Faithful migration remains essential to ensuring that the content and psychometric properties of the original scale (ie, validated reference) are preserved, such that clinicians completing the ClinRO assessments interpret and respond to the items the same way regardless of data collection mode. The authors present a framework for how to “faithfully” migrate electronic ClinRO assessments for successful deployment in clinical trials. MethodsCritical Path Institute’s Electronic PRO Consortium and PRO Consortium convened a consensus panel of representatives from member firms to develop recommendations for electronic migration and implementation of ClinRO assessments in clinical trials based on industry standards, regulatory guidelines where available, and relevant literature. The recommendations were reviewed and approved by all member firms from both consortia. Consensus RecommendationsStandard, minimal electronic modifications for ClinRO assessments are described. This article also outlines implementation steps, including planning, startup, electronic clinical outcome assessment system development, training, and deployment. The consensus panel proposes that functional clinical testing by a clinician or clinical outcome assessment expert, as well as copyright holder review of screenshots (if possible) are sufficient to support minimal modifications during migration. Additional evidence generation is proposed for modifications that deviate significantly from the validated reference.

Clinically Meaningful Outcomes in Early Alzheimer Disease: A Consortia-Driven Approach to Identifying What Matters to Patients.

Numerous statistically derived composite measures have recently been proposed as clinical outcome assessments (COAs) for clinical trials in the early stages of Alzheimer disease. Critical Path Institute's Coalition Against Major Diseases (CAMD) advanced a proposed statistically derived composite measure to regulatory agencies with the goal of qualifying it as a COA for pre-dementia trials. In response to FDA's requirement to demonstrate that proposed COAs are meaningful to patients, this project aimed to identify the most important cognition-related concerns patients and informants report early in the disease and determine how this information maps to what is assessed by several statistically derived composite measures. Leveraging qualitative research completed by Critical Path Institute's Patient-Reported Outcome Consortium, CAMD utilized a summary report that included frequency grids of reported concerns of amnestic mild cognitive impairment patients and their informants, as well as the narrative transcripts from focus groups. Transcripts were reviewed and analyzed to identify which cognitive domains the patient- and informant-reported concerns mapped onto. The results were then compared to see how well these cognitive domains were represented in various statistically derived composite measures. The patient- and informant-reported concerns primarily mapped to the cognitive domains of episodic memory and, secondarily, orientation and language. Depending on the specified composite, there were varying levels of alignment between their subcomponents and these cognitive domains. Through secondary analyses of existing qualitative data, this study examined several statistically derived composite measures and found that they generally capture cognitive domains that reflect aspects of day-to-day functioning that patients and informants consider meaningful.

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 purpose of this paper is to summarize proceedings from a panel presentation at the Critical Path Institute's 2015 Annual PRO Consortium Workshop. This paper underscores the importance of considering children's specific needs and the numerous challenges faced when developing and implementing well-defined and reliable COAs in pediatric clinical trials evaluating medical products, and describes some approaches to addressing these unique needs and challenges.

Patient-Reported Outcomes in Cancer Drug Development and US Regulatory Review

Data reported directly by patients about how they feel and function are rarely included in oncology drug labeling in the United States, in contrast to Europe and to nononcology labeling in the United States, where this practice is more common. Multiple barriers exist, including challenges unique to oncology trials, and industry's concerns regarding cost, logistical complexities, and the Food and Drug Administration's (FDA's) rigorous application of its 2009 guidance on the use of patient-reported outcome (PRO) measures. A panel consisting of representatives of industry, FDA, the PRO Consortium, clinicians, and patients was assembled at a 2014 workshop cosponsored by FDA to identify practical recommendations for overcoming these barriers. Key recommendations included increasing proactive encouragement by FDA to clinical trial sponsors for including PROs in drug development programs; provision of comprehensive PRO plans by sponsors to FDA early in drug development; promotion of an oncology-specific PRO research agenda; development of an approach to existing ("legacy") PRO measures, when appropriate (focused initially on symptoms and functional status); and increased FDA and industry training in PRO methodology. FDA has begun implementing several of these recommendations.

The Patient-Reported Outcome (PRO) Consortium: Lessons Learned Along the Path to PRO Instrument Qualification.

Established in 2008, the Patient-Reported Outcome (PRO) Consortium is a collaboration among the US Food and Drug Administration's Center for Drug Evaluation and Research, the Critical Path Institute, the pharmaceutical/biotechnology industry, and other stakeholders. The purpose of the consortium is to qualify PRO instruments through the Center for Drug Evaluation and Research's drug development tool qualification process for use as clinical trial endpoints to support drug approval and product labeling claims. The PRO Consortium has made notable progress toward collaborative development of PRO instruments in the following areas: asthma, mild cognitive impairment, depression, functional dyspepsia, irritable bowel syndrome, non-small cell lung cancer, and rheumatoid arthritis. This progress has come with considerable challenges, including navigating a new and evolving regulatory initiative, gaining consensus on key issues, and maintaining communication and engagement in a precompetitive environment. The purpose of this paper is to describe some of the challenges and lessons learned since the creation of the PRO Consortium in hopes that this information may provide direction and insight for similar collaborations.

Development of a patient-reported outcome (PRO) measure for assessing non-small cell lung cancer (NSCLC) symptoms in clinical trials: Interim report from the PRO Consortium’s NSCLC Working Group (WG).

e17620 Background: In collaboration with the FDA, the Critical Path Institute - PRO Consortium NSCLC WG is developing a new PRO measure to assess treatment (tx) benefit in advanced NSCLC trials and...