Search

BackgroundChronic obstructive pulmonary disease (COPD) is a progressive chronic disease characterized by airflow obstruction that leads to shortness of breath and substantial negative impacts on health-related quality of life (HRQL). The course of COPD includes periodic acute exacerbations that require changes in treatment and/or hospitalizations. This study was designed to examine the responsiveness of Patient-Reported Outcomes Measurement Information System® (PROMIS®) measures to changes associated with COPD exacerbation recovery.MethodsA longitudinal analysis using mixed-effects models was conducted of people who were enrolled while stable (n = 100) and those who experienced an acute exacerbation (n = 85). PROMIS (physical function, pain interference, pain behavior, fatigue, anxiety, depression, anger, social roles, discretionary social activities, Global Health, dyspnea severity and dyspnea functional limitations) and COPD-targeted HRQL measures were completed at baseline and at 12 weeks.ResultsWe administered PROMIS measures using computer adaptive testing (CAT), followed by administration of any remaining short form (SF) items that had not yet been administered by CAT. Examination of the difference between group differences from baseline to 12 weeks in the stable and exacerbation groups revealed that the exacerbation group changed (improved) significantly more than the stable group in anxiety (p

PurposeThe Patient-Reported Outcomes Measurement Information System (PROMIS(®)) initiative was developed to advance the methodology of PROs applicable to chronic diseases. Chronic obstructive pulmonary disease (COPD) is a progressive chronic disease associated with poor health. This study was designed to examine the correlation of PROMIS health-related quality of life (HRQOL) scales and clinical measures among COPD patients.MethodsA cross-sectional analysis was conducted comparing patients who were stable (n = 100) with those currently experiencing a COPD exacerbation (n = 85). All PROMIS measures for adults available at the time of the study (2008), disease-targeted and other HRQOL instruments, health literacy, percent predicted FEV1, and a 6-min walk distance were assessed when patients were considered clinically stable.ResultsStable COPD patients reported significantly (p ≤ 0.05) better health-related quality of life on PROMIS domains than patients experiencing an exacerbation. PROMIS domain scores were significantly (p ≤ 0.01) correlated with each of legacy measures. Six-min walk scores were most highly correlated with the PROMIS physical function domain scores (r = 0.53) followed by the fatigue (r = -0.26), social (r = 0.24) and to a lesser extent depression (r = -0.23), and anxiety (r = -0.22) domain scores. Percent predicted FEV1 score was significantly associated with PROMIS physical function scores (r = 0.27).ConclusionThis study provides support for the validity of the PROMIS measures in COPD patients.

Background: To develop content validity of a comprehensive patient-reported outcome (PRO) measure following current best scientific methodology to standardize assessment of influenza (flu) symptoms in clinical research.Methods: Stage I (Concept Elicitation): 1:1 telephone interviews with influenza-positive adults (≥18 years) in the US and Mexico within 7 days of diagnosis. Participants described symptom type, character, severity, and duration. Content analysis identified themes and developed the draft Flu-PRO instrument. Stage II (Cognitive Interviewing): The Flu-PRO was administered to a unique set of influenza-positive adults within 14 days of diagnosis; telephone interviews addressed completeness, respondent interpretation of items and ease of use.Results: Samples: Stage I: N = 46 adults (16 US, 30 Mexico); mean (SD) age: 38 (19), 39 (14) years; % female: 56%, 73%; race: 69% White, 97% Mestizo. Stage II: N = 34 adults (12 US, 22 Mexico); age: 37 (14), 39 (11) years; % female: 50%, 50%; race: 58% White, 100% Mestizo.Symptoms: Symptoms identified by >50%: coughing, weak or tired, throat symptoms, congestion, headache, weakness, sweating, chills, general discomfort, runny nose, chest (trouble breathing), difficulty sleeping, and body aches or pains. No new content was uncovered during Stage II; participants easily understood the instrument.Conclusions: Results show the 37-item Flu-PRO is a content valid measure of influenza symptoms in adults with a confirmed diagnosis of influenza. Research is underway to evaluate the suitability of the instrument for children and adolescents. This work can form the basis for future quantitative tests of reliability, validity, and responsiveness to evaluate the measurement properties of Flu-PRO for use in clinical trials and epidemiology studies. [ABSTRACT FROM AUTHOR]

Objectives: As the role and importance of patient-reported outcomes (PROs) increase, the validity and reliability of PRO measures come under greater scientific and regulatory scrutiny. One key issue is selecting the 'most appropriate' recall period for capturing PROs in clinical trials. This paper draws on survey research, health-specific literature, and results from clinical trials to summarize factors that can influence recall and provide guidance on selecting an optimal recall period. Methods: We conducted a systematic review of six databases and additional literature drawn from bibliographies of the selected articles. Results: Six major factors can influence recall; these can be classified into two broad areas: characteristics of the recalled phenomenon (recency, attributes, complexity) and context or meaning of the recalled phenomenon (salience, patient experience, mood). Results of different recall periods for three classes of PROs are presented: health behaviors, symptoms, and health-related quality of life. We present findings on the effect of alternative recall periods for three commonly used PROs. Finally, we propose a heuristic model to link the concept under investigation with an optimal recall period. Conclusions: No single recall period is best for all measures or all phenomena. The recall period must correspond to the characteristics of the phenomenon of interest and the purpose of the assessment. Recall period is an issue of internal validity. An incorrect recall period introduces measurement error that may reduce the chances of detecting a treatment effect. Researchers should consider recall period as seriously as they do other measurement properties. [ABSTRACT FROM AUTHOR]

Innovations in medical device technology have greatly expanded the range of therapeutic options available to physicians and their patients. The understanding of treatment effects from the patient’s perspective is an essential component of a comprehensive assessment of any new therapy, including medical devices. The term “patient-reported outcomes” (PROs) has been growing in use to refer to a cluster of variables such as health-related quality of life, symptoms, physical functioning, psychological well-being, treatment satisfaction, and treatment preferences. As in drug trials, the use of PROs in device evaluation has several methodological challenges, ranging from general concerns about interpretation, to more specific issues related to study design and regulatory approval (use of PROs as primary end points, incorporation in labeling, and product promotion). Successful approaches for integrating PROs into device evaluation trials include the careful selection of appropriate, interpretable PRO end points, accounting for possible confounding factors, and the use of alternatives to placebo-controlled trial designs, such as single-arm pre–post, observational, and registry studies, when the use of placebo control groups is not feasible. This article discusses the potential value and difficulties in measuring PROs in device studies. [ABSTRACT FROM AUTHOR]

Abstract: The importance of content validity in developing patient reported outcomes (PRO) instruments is stressed by both the US Food and Drug Administration and the European Medicines Agency. Content validity is the extent to which an instrument measures the important aspects of concepts developers or users purport it to assess. A PRO instrument measures the concepts most relevant and important to a patient''s condition and its treatment. For PRO instruments, items and domains as reflected in the scores of an instrument should be important to the target population and comprehensive with respect to patient concerns. Documentation of target population input in item generation, as well as evaluation of patient understanding through cognitive interviewing, can provide the evidence for content validity. Part 1 of this task force report covers elicitation of key concepts using qualitative focus groups and/or interviews to inform content and structure of a new PRO instrument. Building on qualitative interviews and focus groups used to elicit concepts, cognitive interviews help developers craft items that can be understood by respondents in the target population and can ultimately confirm that the final instrument is appropriate, comprehensive, and understandable in the target population. Part 2 details: 1) the methods for conducting cognitive interviews that address patient understanding of items, instructions, and response options; and 2) the methods for tracking item development through the various stages of research and preparing this tracking for submission to regulatory agencies. The task force report''s two parts are meant to be read together. They are intended to offer suggestions for good practice in planning, executing, and documenting qualitative studies that are used to support the content validity of PRO instruments to be used in medical product evaluation. [Copyright &y& Elsevier]

Background: Patient-reported outcome (PRO) instruments are used to evaluate the effect of medical products on how patients feel or function. This article presents the results of an ISPOR task force convened to address good clinical research practices for the use of existing or modified PRO instruments to support medical product labeling claims. The focus of the article is on content validity, with specific reference to existing or modified PRO instruments, because of the importance of content validity in selecting or modifying an existing PRO instrument and the lack of consensus in the research community regarding best practices for establishing and documenting this measurement property. Methods: Topics addressed in the article include: definition and general description of content validity; PRO concept identification as the important first step in establishing content validity; instrument identification and the initial review process; key issues in qualitative methodology; and potential threats to content validity, with three case examples used to illustrate types of threats and how they might be resolved. A table of steps used to identify and evaluate an existing PRO instrument is provided, and figures are used to illustrate the meaning of content validity in relationship to instrument development and evaluation. Results & Recommendations: Four important threats to content validity are identified: unclear conceptual match between the PRO instrument and the intended claim, lack of direct patient input into PRO item content from the target population in which the claim is desired, no evidence that the most relevant and important item content is contained in the instrument, and lack of documentation to support modifications to the PRO instrument. In some cases, careful review of the threats to content validity in a specific application may be reduced through additional well documented qualitative studies that specifically address the issue of concern. Conclusion: Published evidence of the content validity of a PRO instrument for an intended application is often limited. Such evidence is, however, important to evaluating the adequacy of a PRO instrument for the intended application. This article provides an overview of key issues involved in assessing and documenting content validity as it relates to using existing instruments in the drug approval process. [ABSTRACT FROM AUTHOR]