Interpreting patient-reported outcomes from clinical trials in COPD: a discussion

Paul W Jones,1,2 Stephen Rennard,3,4 Maggie Tabberer,5 John H Riley,2 Mitra Vahdati-Bolouri,2 Neil C Barnes2,6 1Institute for Infection and Immunity, University of London, London, 2Global Respiratory Franchise, GlaxoSmithKline, Uxbridge, UK; 3Division of Pulmonary, Critical Care, Sleep and Allergy,...

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Main Authors: Jones PW, Rennard S, Tabberer M, Riley JH, Vahdati-Bolouri M, Barnes NC
Format: Article
Language:English
Published: Dove Medical Press 2016-12-01
Series:International Journal of COPD
Subjects:
Online Access:https://www.dovepress.com/interpreting-patient-reported-outcomes-from-clinical-trials-in-copd-a--peer-reviewed-article-COPD
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author Jones PW
Rennard S
Tabberer M
Riley JH
Vahdati-Bolouri M
Barnes NC
author_facet Jones PW
Rennard S
Tabberer M
Riley JH
Vahdati-Bolouri M
Barnes NC
author_sort Jones PW
collection DOAJ
description Paul W Jones,1,2 Stephen Rennard,3,4 Maggie Tabberer,5 John H Riley,2 Mitra Vahdati-Bolouri,2 Neil C Barnes2,6 1Institute for Infection and Immunity, University of London, London, 2Global Respiratory Franchise, GlaxoSmithKline, Uxbridge, UK; 3Division of Pulmonary, Critical Care, Sleep and Allergy, Nebraska Medical Center, Omaha, NE, USA; 4Clinical Discovery Unit, AstraZeneca, Cambridge, 5Global R&D, GlaxoSmithKline, Uxbridge, 6William Harvey Institute, Bart’s and the London School of Medicine and Dentistry, London, UK Abstract: One of the challenges faced by the practising physician is the interpretation of patient-reported outcomes (PROs) in clinical trials and the relevance of such data to their patients. This is especially true when caring for patients with progressive diseases such as COPD. In an attempt to incorporate the patient perspective, many clinical trials now include assessments of PROs. These are formalized methods of capturing patient-centered information. Given the importance of PROs in evaluating the potential utility of an intervention for a patient with COPD, it is important that physicians are able to critically interpret (and critique) the results derived from them. Therefore, in this paper, a series of questions is posed for the practising physician to consider when reviewing the treatment effectiveness as assessed by PROs. The focus is on the St George’s Respiratory Questionnaire for worked examples, but the principles apply equally to other symptom-based questionnaires. A number of different ways of presenting PRO data are discussed, including the concept of the minimum clinically important difference, whether there is a ceiling effect to PRO results, and the strengths and weaknesses of responder analyses. Using a worked example, the value of including a placebo arm in a study is illustrated, and the influence of the study on PRO results is considered, in terms of the design, patient withdrawal, and the selection of the study population. For the practising clinician, the most important consideration is the importance of individualization of treatment (and of treatment goals). To inform such treatment, clinicians need to critically review PRO data. The hope is that the questions posed here will help to build a framework for this critical review. Keywords: patient-centered outcomes research, St George’s Respiratory Questionnaire, COPD, data interpretation, statistical
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spelling doaj.art-1a921a7f66024d529c8fdef0e9b32e352022-12-21T18:31:43ZengDove Medical PressInternational Journal of COPD1178-20052016-12-01Volume 113069307830367Interpreting patient-reported outcomes from clinical trials in COPD: a discussionJones PWRennard STabberer MRiley JHVahdati-Bolouri MBarnes NCPaul W Jones,1,2 Stephen Rennard,3,4 Maggie Tabberer,5 John H Riley,2 Mitra Vahdati-Bolouri,2 Neil C Barnes2,6 1Institute for Infection and Immunity, University of London, London, 2Global Respiratory Franchise, GlaxoSmithKline, Uxbridge, UK; 3Division of Pulmonary, Critical Care, Sleep and Allergy, Nebraska Medical Center, Omaha, NE, USA; 4Clinical Discovery Unit, AstraZeneca, Cambridge, 5Global R&D, GlaxoSmithKline, Uxbridge, 6William Harvey Institute, Bart’s and the London School of Medicine and Dentistry, London, UK Abstract: One of the challenges faced by the practising physician is the interpretation of patient-reported outcomes (PROs) in clinical trials and the relevance of such data to their patients. This is especially true when caring for patients with progressive diseases such as COPD. In an attempt to incorporate the patient perspective, many clinical trials now include assessments of PROs. These are formalized methods of capturing patient-centered information. Given the importance of PROs in evaluating the potential utility of an intervention for a patient with COPD, it is important that physicians are able to critically interpret (and critique) the results derived from them. Therefore, in this paper, a series of questions is posed for the practising physician to consider when reviewing the treatment effectiveness as assessed by PROs. The focus is on the St George’s Respiratory Questionnaire for worked examples, but the principles apply equally to other symptom-based questionnaires. A number of different ways of presenting PRO data are discussed, including the concept of the minimum clinically important difference, whether there is a ceiling effect to PRO results, and the strengths and weaknesses of responder analyses. Using a worked example, the value of including a placebo arm in a study is illustrated, and the influence of the study on PRO results is considered, in terms of the design, patient withdrawal, and the selection of the study population. For the practising clinician, the most important consideration is the importance of individualization of treatment (and of treatment goals). To inform such treatment, clinicians need to critically review PRO data. The hope is that the questions posed here will help to build a framework for this critical review. Keywords: patient-centered outcomes research, St George’s Respiratory Questionnaire, COPD, data interpretation, statisticalhttps://www.dovepress.com/interpreting-patient-reported-outcomes-from-clinical-trials-in-copd-a--peer-reviewed-article-COPDPatient-Centered Outcomes ResearchSt George's Respiratory QuestionnaireChronic Obstructive Pulmonary DiseaseData InterpretationStatistical
spellingShingle Jones PW
Rennard S
Tabberer M
Riley JH
Vahdati-Bolouri M
Barnes NC
Interpreting patient-reported outcomes from clinical trials in COPD: a discussion
International Journal of COPD
Patient-Centered Outcomes Research
St George's Respiratory Questionnaire
Chronic Obstructive Pulmonary Disease
Data Interpretation
Statistical
title Interpreting patient-reported outcomes from clinical trials in COPD: a discussion
title_full Interpreting patient-reported outcomes from clinical trials in COPD: a discussion
title_fullStr Interpreting patient-reported outcomes from clinical trials in COPD: a discussion
title_full_unstemmed Interpreting patient-reported outcomes from clinical trials in COPD: a discussion
title_short Interpreting patient-reported outcomes from clinical trials in COPD: a discussion
title_sort interpreting patient reported outcomes from clinical trials in copd a discussion
topic Patient-Centered Outcomes Research
St George's Respiratory Questionnaire
Chronic Obstructive Pulmonary Disease
Data Interpretation
Statistical
url https://www.dovepress.com/interpreting-patient-reported-outcomes-from-clinical-trials-in-copd-a--peer-reviewed-article-COPD
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