Observational studies assessing the pharmacological treatment of obstructive lung disease: strengths, challenges and considerations for study design

Jørgen Vestbo* (Corresponding Author), Christer Janson, Javier Nuevo, David Price

*Corresponding author for this work

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6 Citations (Scopus)
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Randomised controlled trials (RCTs) are the gold standard for evaluating treatment efficacy in patients with obstructive lung disease. However, due to strict inclusion criteria and the conditions required for ascertaining statistical significance, the patients included typically represent as little as 5% of the general obstructive lung disease population. Thus, studies in broader patient populations are becoming increasingly important. These can be randomised effectiveness trials or observational studies providing data on real-world treatment effectiveness and safety data that complement efficacy RCTs.

In this review we describe the features associated with the diagnosis of asthma and chronic obstructive pulmonary disease (COPD) in the real-world clinical practice setting. We also discuss how RCTs and observational studies have reported opposing outcomes with several treatments and inhaler devices due to differences in study design and the variations in patients recruited by different study types. Whilst observational studies are not without weaknesses, we outline recently developed tools for defining markers of quality of observational studies. We also examine how observational studies are capable of providing valuable insights into disease mechanisms and management and how they are a vital component of research into obstructive lung disease.

As we move into an era of personalised medicine, recent observational studies, such as the NOVEL observational longiTudinal studY (NOVELTY), have the capacity to provide a greater understanding of the value of a personalised healthcare approach in patients in clinical practice by focussing on standardised outcome measures of patient-reported outcomes, physician assessments, airway physiology, and blood and airway biomarkers across both primary and specialist care.
Original languageEnglish
Article number00044-2020
Number of pages16
JournalERJ Open Research
Issue number4
Early online date13 Oct 2020
Publication statusPublished - 13 Oct 2020

Bibliographical note

Editorial support under the direction of the authors was provided by Richard Knight, CMC Connect, McCann Health Medical Communications, and funded by AstraZeneca in accordance with Good Publication Practice guidelines. The first draft of the manuscript was written in three sections by J. Vestbo, C. Janson and D. Price. Editorial support specifically for D. Price was provided by Antony Hardjojo of the Observational and Pragmatic Research Institute, Singapore. J. Vestbo is supported by the NIHR Manchester BRC.


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