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Perspective
Pragmatic randomised controlled trials in COPD and asthma: how to guide clinical practice
  1. Nawar Diar Bakerly1,2,
  2. Kirill Nikitin3,
  3. Neil G Snowise4,
  4. Glenn Cardwell3,
  5. Daryl Freeman5,
  6. Ravijyot Saggu6 and
  7. Anthony De Soyza7
  1. 1School of Biological Sciences, Manchester Metropolitan University, Manchester, UK
  2. 2Salford Royal, NHS Foundation Trust, Salford, UK
  3. 3Respiratory Department, GSK, Brentford, UK
  4. 4Institute of Pharmaceutical Science, Faculty of Life Sciences and Medicine, King's College, London, UK
  5. 5Norfolk Community Health and Care, Woodlands House, Norwich, UK
  6. 6Pharmacy Department, University College Hospital, NHS Foundation Trust, London, UK
  7. 7Population and Health Sciences Institute, Newcastle University, Newcastle, UK
  1. Correspondence to Professor Anthony De Soyza; anthony.de-soyza{at}newcastle.ac.uk

Abstract

The use of real-world evidence (RWE) studies, including pragmatic randomised controlled trials (RCTs; randomised RWE studies), to aid the development of treatment guidelines, is gradually becoming a mainstay within clinical practice. RWE is an integral part of patient-driven decision-making and offers important value to add complimentary evidence to traditional RCTs; these provide a more well-rounded view of the benefits to patient-reported outcomes and improve the external validity of a given treatment versus findings from traditional RCTs alone. Discussions in recent scientific workshops explored the importance of pragmatic RCTs in optimising guideline development and patient care in chronic obstructive pulmonary disease (COPD) and asthma. The Salford Lung Study in patients with COPD (NCT01551758) and asthma (NCT01706198) were the world’s first prelicence pragmatic RCTs that compared novel investigational treatments with existing COPD and asthma treatments and, more recently (2021), RWE studies have been used by the American Thoracic Society and the US Food and Drug Administration to support the approval of an immunosuppressant drug in patients receiving lung transplants. This highlights the importance of RWE data in supporting clinical guideline development and emphasises the advantages for the use of pragmatic RCTs in guiding clinical practice.

  • Asthma Guidelines
http://creativecommons.org/licenses/by-nc/4.0/

This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.

https://doi.org/10.1136/bmjresp-2022-001303

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    Footnotes

    • Collaborators Not applicable.

    • Contributors All authors participated in at least one of the scientific workshops and contributed to the drafting and or critical review of the manuscript and approved the final version for submission.

    • Funding GSK-sponsored scientific workshops.

    • Competing interests NDB reports grants and personal fees from GSK, Novartis and Almirall/AstraZeneca, and congress attendance fees from Boehringer-Ingelheim. KN and GC are employees of and shareholders in GSK. NGS is a former employee of GSK. RS reports previous honoraria/sponsorship for consultancy work, lecturing and educational events unrelated to this workshop from GSK, Cogora, Pharman, Chiesi, Boehringer Ingelheim, Teva, AstraZeneca and the Centre for Pharmacy Postgraduate Education, and is a member of the National Institute for Health and Care Excellence COPD guidelines committee and the British Thoracic Society specialist advisory group. ADS reports grants, personal fees and other financial activities from AstraZeneca, Bayer, GSK, Gilead, Novartis, Pfizer, Teva and Chiesi.

    • Provenance and peer review Not commissioned; externally peer reviewed.