Real-world data and randomised controlled trials: The Salford Lung Study
Real-world data and randomised controlled trials: The Salford Lung Study
Traditional efficacy double-blind randomised controlled trials (DBRCTs) measure the benefit a treatment produces under near-ideal test conditions in highly selected patient populations; however, the behaviour of patients and investigators in such trials is highly controlled, highly compliant and adherent, and non-representative of routine clinical practice. Pragmatic effectiveness trials measure the benefit a treatment produces in patients in everyday “real-world” practice. Ideally, effectiveness trials should recruit patients as similar as possible to those who will ultimately be prescribed the medicine, and create freedom within the study design to allow normal behaviours of patients and healthcare professionals (HCPs) to be expressed. The Salford Lung Study (SLS) was a world-first, prospective, phase III, pragmatic randomised controlled trial (RCT) programme in patients with chronic obstructive pulmonary disease and asthma to evaluate the effectiveness of a pre-licensed medication (fluticasone furoate/vilanterol) in real-world practice using electronic health records and through collaboratively engaging general practitioners and community pharmacists in clinical research. The real-world aspect of SLS was unique, requiring careful planning and attention to the goals of maximising the external validity of the trials while maintaining scientific rigour and securing suitable electronic processes for proper interpretation of safety data. Key learnings from SLS that may inform the design of future pragmatic effectiveness RCTs include: (1) ensuring the trial setting and operational infrastructure are aligned with routine clinical care; (2) recruiting a broad patient population with characteristics as close as possible to patients in routine clinical practice, to maximise the generalisability and applicability of trial results; (3) ensuring that patients and HCPs are suitably engaged in the trial, to maximise the chances of successful trial delivery; and (4) careful study design, incorporating outcomes of value to patients, HCPs, policymakers and payers, and using pre-planned analyses to address scientifically valid research hypotheses to ensure robustness of the trial data.
Asthma, Chronic obstructive pulmonary disease, Effectiveness, Fluticasone furoate/vilanterol, Primary care, Randomised controlled trial, Real world, Salford Lung Study, Usual care
977-997
Leather, David A.
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Jones, Rupert
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Woodcock, Ashley
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Vestbo, Jørgen
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Jacques, Loretta
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Thomas, Mike
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1 March 2020
Leather, David A.
ac7e6999-7d5e-41b8-bd08-63a83d784600
Jones, Rupert
05544cdd-8933-45cf-ab4f-50f2ab3dfd77
Woodcock, Ashley
b13cf094-8318-42ef-b8ed-2f4b8d8770f3
Vestbo, Jørgen
6298f58b-134a-4dc5-b560-2b7eb261965f
Jacques, Loretta
e8745267-4b6e-429b-be54-1db019fb6099
Thomas, Mike
997c78e0-3849-4ce8-b1bc-86ebbdee3953
Leather, David A., Jones, Rupert, Woodcock, Ashley, Vestbo, Jørgen, Jacques, Loretta and Thomas, Mike
(2020)
Real-world data and randomised controlled trials: The Salford Lung Study.
Advances in Therapy, 37 (3), .
(doi:10.1007/s12325-019-01192-1).
Abstract
Traditional efficacy double-blind randomised controlled trials (DBRCTs) measure the benefit a treatment produces under near-ideal test conditions in highly selected patient populations; however, the behaviour of patients and investigators in such trials is highly controlled, highly compliant and adherent, and non-representative of routine clinical practice. Pragmatic effectiveness trials measure the benefit a treatment produces in patients in everyday “real-world” practice. Ideally, effectiveness trials should recruit patients as similar as possible to those who will ultimately be prescribed the medicine, and create freedom within the study design to allow normal behaviours of patients and healthcare professionals (HCPs) to be expressed. The Salford Lung Study (SLS) was a world-first, prospective, phase III, pragmatic randomised controlled trial (RCT) programme in patients with chronic obstructive pulmonary disease and asthma to evaluate the effectiveness of a pre-licensed medication (fluticasone furoate/vilanterol) in real-world practice using electronic health records and through collaboratively engaging general practitioners and community pharmacists in clinical research. The real-world aspect of SLS was unique, requiring careful planning and attention to the goals of maximising the external validity of the trials while maintaining scientific rigour and securing suitable electronic processes for proper interpretation of safety data. Key learnings from SLS that may inform the design of future pragmatic effectiveness RCTs include: (1) ensuring the trial setting and operational infrastructure are aligned with routine clinical care; (2) recruiting a broad patient population with characteristics as close as possible to patients in routine clinical practice, to maximise the generalisability and applicability of trial results; (3) ensuring that patients and HCPs are suitably engaged in the trial, to maximise the chances of successful trial delivery; and (4) careful study design, incorporating outcomes of value to patients, HCPs, policymakers and payers, and using pre-planned analyses to address scientifically valid research hypotheses to ensure robustness of the trial data.
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e-pub ahead of print date: 11 January 2020
Published date: 1 March 2020
Additional Information:
Funding Information:
Editorial support in the development of this article (in the form of writing assistance, collating author comments, assembling tables/figures, grammatical editing, and referencing) was provided by Emma Landers, PhD, at Gardiner-Caldwell Communications (Macclesfield, UK) and was funded by GlaxoSmithKline plc.
Funding Information:
David A. Leather has employment with, and stock/share ownership in, GlaxoSmithKline plc. Loretta Jacques has employment with, and stock/share ownership in, GlaxoSmithKline plc. Rupert Jones has grants from AstraZeneca and GlaxoSmithKline plc.; personal fees from AstraZeneca, Boehringer Ingelheim, Chiesi, GlaxoSmithKline plc., Novartis and Nutricia. Ashley Woodcock has received speaker fees and expenses from GlaxoSmithKline plc.; advisory board member for, and expenses from, Chiesi; personal fees from Axalbion and Reacta Biotech; Chair of Medicines Evaluation Unit. Jørgen Vestbo has received research grant from Boehringer Ingelheim; honoraria from AstraZeneca, Boehringer Ingelheim, Chiesi, GlaxoSmithKline plc., and Novartis for consulting/presenting. Mike Thomas has received speaker fees from Aerocrine, GlaxoSmithKline plc., and Novartis; honoraria from Aerocrine, Boehringer Ingelheim, GlaxoSmithKline plc., MSD, Novartis, and Pfizer. Mike Thomas is a recent a member of the BTS SIGN Asthma guideline steering group and the NICE Asthma Diagnosis and Monitoring guideline development group. Rupert Jones was supported by the National Institute for Health Research Collaboration for Leadership in Applied Health Research and Care South West Peninsula (NIHR CLAHRC South West Peninsula). Ashley Woodcock and Jørgen Vestbo are supported by the National Institute of Health Research Biomedical Centre in Manchester (NIHR Manchester BRC).
Publisher Copyright:
© 2020, The Author(s).
Keywords:
Asthma, Chronic obstructive pulmonary disease, Effectiveness, Fluticasone furoate/vilanterol, Primary care, Randomised controlled trial, Real world, Salford Lung Study, Usual care
Identifiers
Local EPrints ID: 437562
URI: http://eprints.soton.ac.uk/id/eprint/437562
ISSN: 0741-238X
PURE UUID: a5f0dda6-ac32-4b36-94f7-acfcfbc6476a
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Date deposited: 05 Feb 2020 17:32
Last modified: 16 Mar 2024 06:25
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Author:
David A. Leather
Author:
Rupert Jones
Author:
Ashley Woodcock
Author:
Jørgen Vestbo
Author:
Loretta Jacques
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