Abstract
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.
Abstract
Observational studies can support RCTs in influencing clinical practice in the field of obstructive lung disease https://bit.ly/36YWu0W
Footnotes
Conflict of interest: J. Vestbo reports personal fees from AstraZeneca for co-chairing the NOVELTY study, from which the idea for this article arose, during the conduct of the study; honoraria for presenting and/or advising on COPD from AstraZeneca, an unconditional biomarker grant and honoraria for presenting and/or advising on COPD from Boehringer Ingelheim, honoraria for presenting and/or advising on COPD from Chiesi, an honorarium for advising on COPD from GSK, and honoraria for presenting and/or advising on COPD fees from Novartis, outside the submitted work; and his son is an employee of Chiesi (Denmark).
Conflict of interest: C. Janson has nothing to disclose.
Conflict of interest: J. Nuevo is an employee of AstraZeneca.
Conflict of interest: D. Price reports grants from AKL Research and Development Ltd, personal fees from Amgen, grants and personal fees from AstraZeneca, grants and personal fees from Boehringer Ingelheim, grants from British Lung Foundation, grants and personal fees from Chiesi, grants and personal fees from Circassia, personal fees from Cipla, personal fees from GlaxoSmithKline, personal fees from Kyorin, personal fees from Merck, grants and personal fees from Mylan, grants and personal fees from Mundipharma, grants and personal fees from Napp, grants and personal fees from Novartis, grants and personal fees from Pfizer, grants and personal fees from Regeneron Pharmaceuticals, grants from Respiratory Effectiveness Group, grants and personal fees from Sanofi Genzyme, grants and personal fees from Teva, grants and personal fees from Theravance, grants from UK National Health Service, grants and personal fees from Zentiva (Sanofi Generics), non-financial support from Efficacy and Mechanism Evaluation programme, non-financial support from Health Technology Assessment, outside the submitted work; stock/stock options from AKL Research and Development Ltd, which produces phytopharmaceuticals; and owns 74% of the social enterprise Optimum Patient Care Ltd (Australia and UK) and 74% of Observational and Pragmatic Research Institute Pte Ltd (Singapore).
Support statement: The development of this manuscript was funded by AstraZeneca. Funding information for this article has been deposited with the Crossref Funder Registry.
- Received January 28, 2020.
- Accepted June 1, 2020.
- Copyright ©ERS 2020
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