Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases
Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases
Background: fibrotic interstitial lung diseases (fILDs) are a heterogeneous group of lung diseases associated with significant morbidity and mortality. Despite a large increase in the number of clinical trials in the last 10 years, current regulatory-approved management approaches are limited to two therapies that prevent the progression of fibrosis. The drug development pipeline is long and there is an urgent need to accelerate this process. This manuscript introduces the concept and design of an innovative research approach to drug development in fILD: a global Randomised Embedded Multifactorial Adaptive Platform in fILD (REMAP-ILD).
Methods: description of the REMAP-ILD concept and design: the specific terminology, design characteristics (multifactorial, adaptive features, statistical approach), target population, interventions, outcomes, mission and values, and organisational structure.
Results: the target population will be adult patients with fILD, and the primary outcome will be a disease progression model incorporating forced vital capacity and mortality over 12 months. Responsive adaptive randomisation, prespecified thresholds for success and futility will be used to assess the effectiveness and safety of interventions. REMAP-ILD embraces the core values of diversity, equity, and inclusion for patients and researchers, and prioritises an open-science approach to data sharing and dissemination of results.
Conclusion: by using an innovative and efficient adaptive multi-interventional trial platform design, we aim to accelerate and improve care for patients with fILD. Through worldwide collaboration, novel analytical methodology and pragmatic trial delivery, REMAP-ILD aims to overcome major limitations associated with conventional randomised controlled trial approaches to rapidly improve the care of people living with fILD.
Connective tissue disease associated lung disease, Hypersensitivity pneumonitis, Idiopathic pulmonary fibrosis, Interstitial Fibrosis, Rheumatoid lung disease
788-795
Kawano-Dourado, Leticia
51a09a78-ee86-4264-9a05-28c8d640fc1c
Kulkarni, Tejaswini
cab47c14-5782-49fb-bc7e-a32be6556648
Ryerson, Christopher J.
d7904e80-fa84-4514-95b3-4f24acbcce1f
Fletcher, Sophie
71599088-9df7-4d4a-8570-aef773ead0fe
16 July 2024
Kawano-Dourado, Leticia
51a09a78-ee86-4264-9a05-28c8d640fc1c
Kulkarni, Tejaswini
cab47c14-5782-49fb-bc7e-a32be6556648
Ryerson, Christopher J.
d7904e80-fa84-4514-95b3-4f24acbcce1f
Fletcher, Sophie
71599088-9df7-4d4a-8570-aef773ead0fe
Kawano-Dourado, Leticia, Kulkarni, Tejaswini and Ryerson, Christopher J.
,
et al. and REMAP-ILD consortium
(2024)
Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases.
Thorax, 79 (8), .
(doi:10.1136/thorax-2023-221148).
Abstract
Background: fibrotic interstitial lung diseases (fILDs) are a heterogeneous group of lung diseases associated with significant morbidity and mortality. Despite a large increase in the number of clinical trials in the last 10 years, current regulatory-approved management approaches are limited to two therapies that prevent the progression of fibrosis. The drug development pipeline is long and there is an urgent need to accelerate this process. This manuscript introduces the concept and design of an innovative research approach to drug development in fILD: a global Randomised Embedded Multifactorial Adaptive Platform in fILD (REMAP-ILD).
Methods: description of the REMAP-ILD concept and design: the specific terminology, design characteristics (multifactorial, adaptive features, statistical approach), target population, interventions, outcomes, mission and values, and organisational structure.
Results: the target population will be adult patients with fILD, and the primary outcome will be a disease progression model incorporating forced vital capacity and mortality over 12 months. Responsive adaptive randomisation, prespecified thresholds for success and futility will be used to assess the effectiveness and safety of interventions. REMAP-ILD embraces the core values of diversity, equity, and inclusion for patients and researchers, and prioritises an open-science approach to data sharing and dissemination of results.
Conclusion: by using an innovative and efficient adaptive multi-interventional trial platform design, we aim to accelerate and improve care for patients with fILD. Through worldwide collaboration, novel analytical methodology and pragmatic trial delivery, REMAP-ILD aims to overcome major limitations associated with conventional randomised controlled trial approaches to rapidly improve the care of people living with fILD.
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Accepted/In Press date: 6 February 2024
e-pub ahead of print date: 6 March 2024
Published date: 16 July 2024
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© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.
© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.
Keywords:
Connective tissue disease associated lung disease, Hypersensitivity pneumonitis, Idiopathic pulmonary fibrosis, Interstitial Fibrosis, Rheumatoid lung disease
Identifiers
Local EPrints ID: 495828
URI: http://eprints.soton.ac.uk/id/eprint/495828
ISSN: 0040-6376
PURE UUID: 8b3d5f3c-a31a-4833-be20-4382178d3e80
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Date deposited: 25 Nov 2024 17:36
Last modified: 18 Mar 2025 03:17
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Contributors
Author:
Leticia Kawano-Dourado
Author:
Tejaswini Kulkarni
Author:
Christopher J. Ryerson
Author:
Sophie Fletcher
Corporate Author: et al.
Corporate Author: REMAP-ILD consortium
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