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Review
. 2020 Jul 23:15:1789-1800.
doi: 10.2147/COPD.S235845. eCollection 2020.

Patient-Reported Outcomes (PROs) in COPD Clinical Trials: Trends and Gaps

Nuzhat Afroz  1 , Florian S Gutzwiller  2 , Alex J Mackay  3   4 , Christel Naujoks  2 , Francesco Patalano  5 , Konstantinos Kostikas  6   7
Affiliations
Review

Patient-Reported Outcomes (PROs) in COPD Clinical Trials: Trends and Gaps

Nuzhat Afroz et al. Int J Chron Obstruct Pulmon Dis. .

Abstract

Key characteristics of chronic obstructive pulmonary disease (COPD) that significantly affect health-related quality of life (HRQoL) include chest symptoms, dyspnea, cough, sputum production, and exacerbations. Additional areas of impact are sleep, fatigue, emotional well-being, social functioning, and coping. Patient-reported outcomes (PROs) are essential to evaluate symptoms, impact of symptoms on activities of daily living, and treatment response. This review summarizes COPD-specific PRO endpoints from randomized controlled trials of approved and commonly used COPD drugs. A search conducted in "ClinicalTrials.gov" to identify COPD clinical trials (only completed Phase III and IV) incorporating PRO endpoints yielded a total of 104 clinical trials for inclusion in this analysis. Both symptom-based and HRQoL-specific PRO measures were reported. Several COPD-specific PRO measures are available; however, the St. George's Respiratory Questionnaire (SGRQ) and the Baseline and Transition Dyspnea Indexes (BDI/TDI) were reported in the majority of the studies. Results reflected a gap in terms of full coverage of key impacted areas from a patient's perspective. Methodological issues identified in this review related to scoring of instruments require careful consideration, as these challenges may limit the complete assessment of drug benefits. Selection of PRO measures aligned with the expected treatment benefit of a drug in a clinical trial should reflect patients' perspective holistically.

Keywords: Patient-Focused Drug Development; chronic obstructive pulmonary disease; clinical trials; endpoints; patient-reported outcomes; randomized controlled trials.

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Conflict of interest statement

Nuzhat Afroz is an employee of Novartis Healthcare Pvt. Ltd., Hyderabad, India. Florian S. Gutzwiller, Francesco Patalano, and Christel Naujoks are employees and shareholders of Novartis Pharma AG, Basel, Switzerland. Konstantinos Kostikas was an employee of Novartis Pharma AG, Basel, Switzerland, at the time of execution of this analysis, and reports grants and personal fees from AstraZeneca, Chiesi, Boehringer-Ingelheim, ELPEN, Novartis, and GSK, outside the submitted work; Alex J. Mackay was a European Respiratory Society fellow with Industry working with Novartis Pharma AG, Basel, Switzerland, at the time of execution of this analysis, and is currently an employee of AstraZeneca; they also report personal fees from Pfizer, outside the submitted work. The authors report no other possible conflicts of interest in this work.

Figures

Figure 1
Figure 1
Overview of impact of COPD symptoms on HRQoL. Notes: Data from these studies.– Clinical endpoints, symptom diaries, and EXACT were used to capture exacerbations. Cost of treatment includes both direct and indirect costs, but this aspect was not captured in this review. Abbreviations: EXACT, Exacerbations of Chronic Pulmonary Disease ToolHRQoL, health-related quality of life.
See this image and copyright information in PMC

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