Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme)
Abstract Background MACRO (Defining best Management for Adults with Chronic RhinOsinusitis) is an NIHR-funded programme of work designed to establish best practice for adults with chronic rhinosinusitis (CRS). The 7-year programme comprises three consecutive workstreams, designed to explore NHS care...
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2019-04-01
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Online Access: | http://link.springer.com/article/10.1186/s13063-019-3318-3 |
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record_format |
Article |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Helen Blackshaw Jane Vennik Carl Philpott Mike Thomas Caroline Eyles James Carpenter Caroline S. Clarke Steve Morris Anne Schilder Valerie Lund Paul Little Stephen Durham Spiros Denaxas Elizabeth Williamson David Beard Jonathan Cook Steffi Le Conte Kim Airey Jim Boardman Claire Hopkins |
spellingShingle |
Helen Blackshaw Jane Vennik Carl Philpott Mike Thomas Caroline Eyles James Carpenter Caroline S. Clarke Steve Morris Anne Schilder Valerie Lund Paul Little Stephen Durham Spiros Denaxas Elizabeth Williamson David Beard Jonathan Cook Steffi Le Conte Kim Airey Jim Boardman Claire Hopkins Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme) Trials Expert panel Mixed methods Consensus Decision-making Chronic rhinosinusitis |
author_facet |
Helen Blackshaw Jane Vennik Carl Philpott Mike Thomas Caroline Eyles James Carpenter Caroline S. Clarke Steve Morris Anne Schilder Valerie Lund Paul Little Stephen Durham Spiros Denaxas Elizabeth Williamson David Beard Jonathan Cook Steffi Le Conte Kim Airey Jim Boardman Claire Hopkins |
author_sort |
Helen Blackshaw |
title |
Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme) |
title_short |
Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme) |
title_full |
Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme) |
title_fullStr |
Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme) |
title_full_unstemmed |
Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme) |
title_sort |
expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the macro programme) |
publisher |
BMC |
series |
Trials |
issn |
1745-6215 |
publishDate |
2019-04-01 |
description |
Abstract Background MACRO (Defining best Management for Adults with Chronic RhinOsinusitis) is an NIHR-funded programme of work designed to establish best practice for adults with chronic rhinosinusitis (CRS). The 7-year programme comprises three consecutive workstreams, designed to explore NHS care pathways through analysis of primary and secondary data sources, and to undertake a randomised controlled trial to evaluate a longer-term course of macrolide antibiotics and endoscopic sinus surgery for patients with CRS. A number of outstanding elements still required clarification at the funding stage. This paper reports an expert panel review process designed to agree and finalise the MACRO trial design, ensuring relevance to patients and clinicians whilst maximising trial recruitment and retention. Methods An expert panel, consisting of the MACRO Programme Management Group, independent advisors, and patient contributors, was convened to review current evidence and the mixed-method data collected as part of the programme, and reach agreement on MACRO trial design. Specifically, agreement was sought for selection of macrolide antibiotic, use of orally administered steroids, inclusion of CRS phenotypes (with/without nasal polyps), and overall trial design. Results A 12-week course of clarithromycin was agreed as the main trial comparator due to its increasing use as a first- and second-line treatment for patients with CRS, and the perceived need to establish its role in CRS management. Orally administered steroids will be used as a rescue medication during the trial, rather than routinely either pre or post trial randomisation, to limit any potential effects on surgical outcomes and better reflect current UK prescribing habits. Both CRS phenotypes will be included in a single trial to ensure that the MACRO trial is both pragmatic and generalisable to primary care. A modified, three-arm trial design was agreed after intense discussions and further exploratory work. Inclusion criteria were amended to ensure that the patients recruited would be considered eligible for the treatment offered in the trial due to having already received appropriate medical therapy as deemed suitable by their ENT surgeon. A proposed 6-week run-in period prior to randomisation was removed due to the new criteria prior to randomisation. Conclusion The expert panel review process resulted in agreement on key elements and an optimal design for the MACRO trial, considered most likely to be successful in terms of both recruitment potential and ability to establish best management of patients with CRS. |
topic |
Expert panel Mixed methods Consensus Decision-making Chronic rhinosinusitis |
url |
http://link.springer.com/article/10.1186/s13063-019-3318-3 |
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doaj-a8b27b2e0d0f4d7d899698f6c2c391462020-11-25T02:52:37ZengBMCTrials1745-62152019-04-012011910.1186/s13063-019-3318-3Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme)Helen Blackshaw0Jane Vennik1Carl Philpott2Mike Thomas3Caroline Eyles4James Carpenter5Caroline S. Clarke6Steve Morris7Anne Schilder8Valerie Lund9Paul Little10Stephen Durham11Spiros Denaxas12Elizabeth Williamson13David Beard14Jonathan Cook15Steffi Le Conte16Kim Airey17Jim Boardman18Claire Hopkins19evidENT, Ear Institute, University College LondonPrimary Care and Populations Sciences, Faculty of Medicine, University of SouthamptonNorwich Medical School, University of East AngliaPrimary Care and Populations Sciences, Faculty of Medicine, University of SouthamptonPrimary Care and Populations Sciences, Faculty of Medicine, University of SouthamptonLondon School of Hygiene and Tropical MedicineResearch Department of Primary Care and Population Health, University College LondonDepartment of Applied Health Research, University College LondonevidENT, Ear Institute, University College LondonevidENT, Ear Institute, University College LondonJames Paget University Hospital NHS Foundation TrustFaculty of Medicine, Imperial College LondonFarr Institute, University College LondonFarr Institute, University College LondonSurgical Interventional Trials Unit, University of OxfordSurgical Interventional Trials Unit, University of OxfordSurgical Interventional Trials Unit, University of OxfordevidENT, Ear Institute, University College LondonFifth Sense, Sanderum HouseGuy’s and St. Thomas’ NHS Foundation TrustAbstract Background MACRO (Defining best Management for Adults with Chronic RhinOsinusitis) is an NIHR-funded programme of work designed to establish best practice for adults with chronic rhinosinusitis (CRS). The 7-year programme comprises three consecutive workstreams, designed to explore NHS care pathways through analysis of primary and secondary data sources, and to undertake a randomised controlled trial to evaluate a longer-term course of macrolide antibiotics and endoscopic sinus surgery for patients with CRS. A number of outstanding elements still required clarification at the funding stage. This paper reports an expert panel review process designed to agree and finalise the MACRO trial design, ensuring relevance to patients and clinicians whilst maximising trial recruitment and retention. Methods An expert panel, consisting of the MACRO Programme Management Group, independent advisors, and patient contributors, was convened to review current evidence and the mixed-method data collected as part of the programme, and reach agreement on MACRO trial design. Specifically, agreement was sought for selection of macrolide antibiotic, use of orally administered steroids, inclusion of CRS phenotypes (with/without nasal polyps), and overall trial design. Results A 12-week course of clarithromycin was agreed as the main trial comparator due to its increasing use as a first- and second-line treatment for patients with CRS, and the perceived need to establish its role in CRS management. Orally administered steroids will be used as a rescue medication during the trial, rather than routinely either pre or post trial randomisation, to limit any potential effects on surgical outcomes and better reflect current UK prescribing habits. Both CRS phenotypes will be included in a single trial to ensure that the MACRO trial is both pragmatic and generalisable to primary care. A modified, three-arm trial design was agreed after intense discussions and further exploratory work. Inclusion criteria were amended to ensure that the patients recruited would be considered eligible for the treatment offered in the trial due to having already received appropriate medical therapy as deemed suitable by their ENT surgeon. A proposed 6-week run-in period prior to randomisation was removed due to the new criteria prior to randomisation. Conclusion The expert panel review process resulted in agreement on key elements and an optimal design for the MACRO trial, considered most likely to be successful in terms of both recruitment potential and ability to establish best management of patients with CRS.http://link.springer.com/article/10.1186/s13063-019-3318-3Expert panelMixed methodsConsensusDecision-makingChronic rhinosinusitis |