Novel method to select meaningful outcomes for evaluation in clinical trials
Background A standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and th...
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| Format: | Article |
| Language: | English |
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BMJ Publishing Group
2021-01-01
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| Series: | BMJ Open Respiratory Research |
| Online Access: | https://bmjopenrespres.bmj.com/content/8/1/e000877.full |
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| author | Yue Wu Charlie McLeod Richard Norman Andre Schultz Steven Mascaro Steve Webb Tom Snelling Alan Smyth Christopher Blyth Jamie Wood Siobhain Mulrennan Sue Morey Mitch Messer Kate Spaapen Matthew Stoneham Owen Woodberry |
| author_facet | Yue Wu Charlie McLeod Richard Norman Andre Schultz Steven Mascaro Steve Webb Tom Snelling Alan Smyth Christopher Blyth Jamie Wood Siobhain Mulrennan Sue Morey Mitch Messer Kate Spaapen Matthew Stoneham Owen Woodberry |
| author_sort | Yue Wu |
| collection | DOAJ |
| description | Background A standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and the health intervention being studied. Causal network diagrams may help researchers identify outcomes that are both clinically meaningful and likely to be causally dependent on the intervention, and endpoints that are, in turn, causally dependent on those outcomes. We aimed to (1) develop a generalisable method for selecting outcomes and endpoints in trials and (2) apply this method to select outcomes for evaluation in a trial investigating treatment strategies for pulmonary exacerbations of cystic fibrosis (CF).Methods We conducted a series of online surveys and workshops among people affected by CF. We used a modified Delphi approach to develop a consensus list of important outcomes. A workshop involving domain experts elicited how these outcomes were causally related to the underlying pathophysiological processes. Meaningful outcomes were prioritised based on the extent to which each outcome captured separate rather than common aspects of the underlying pathophysiological process.Results The 10 prioritised outcomes were: breathing difficulty/pain, sputum production/clearance, fatigue, appetite, pain (not related to breathing), motivation/demoralisation, fevers/night sweats, treatment burden, inability to meet personal goals and avoidance of gastrointestinal symptoms.Conclusions This proposed method for selecting meaningful outcomes for evaluation in clinical trials may improve the value of research as a basis for clinical decisions. |
| format | Article |
| id | doaj-art-b1ced55873094843a3c44b0908650904 |
| institution | Kabale University |
| issn | 2052-4439 |
| language | English |
| publishDate | 2021-01-01 |
| publisher | BMJ Publishing Group |
| record_format | Article |
| series | BMJ Open Respiratory Research |
| spelling | doaj-art-b1ced55873094843a3c44b09086509042024-11-24T09:30:09ZengBMJ Publishing GroupBMJ Open Respiratory Research2052-44392021-01-018110.1136/bmjresp-2021-000877Novel method to select meaningful outcomes for evaluation in clinical trialsYue Wu0Charlie McLeod1Richard Norman2Andre Schultz3Steven Mascaro4Steve Webb5Tom Snelling6Alan Smyth7Christopher Blyth8Jamie Wood9Siobhain Mulrennan10Sue Morey11Mitch Messer12Kate Spaapen13Matthew Stoneham14Owen Woodberry153 Anhui Province Key Laboratory of Cognition and Neuropsychiatric Disorders, Hefei, Anhui, China2 School of Medicine, University of Western Australia, Nedlands, Western Australia, Australia9 School of Population Health, Faculty of Health Sciences, Curtin University, Perth, Western Australia, AustraliaDepartment of Respiratory Medicine, Perth Children`s Hospital, Nedlands, Western Australia, Australia5 Clayton School of IT, Monash University, Clayton, Victoria, AustraliaSchool of Population Health and Preventive Medicine, Monash University, Clayton, Victoria, AustraliaMenzies School of Health Research, Casuarina, Northern Territory, AustraliaChild Health Obstetrics and Gynaecology, University of Nottingham, Nottingham, UKAneurin Bevan University Health Board, Newport, Gwent, UKDepartment of Rehabilitation and Human Performance, Icahn School of Medicine at Mount Sinai, New York, New York, USARespiratory Medicine, Sir Charles Gairdner Hospital, Nedlands, Western Australia, AustraliaRespiratory Medicine, Sir Charles Gairdner Hospital, Nedlands, Western Australia, AustraliaWesfarmers Centre of Vaccines and Infectious Diseases, Telethon Kids Institute, Perth, Western Australia, AustraliaConsumer advocate, Perth, Western Australia, AustraliaConsumer advocate, Melbourne, Victoria, AustraliaBayesian Intelligence, Upwey, Victoria, AustraliaBackground A standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and the health intervention being studied. Causal network diagrams may help researchers identify outcomes that are both clinically meaningful and likely to be causally dependent on the intervention, and endpoints that are, in turn, causally dependent on those outcomes. We aimed to (1) develop a generalisable method for selecting outcomes and endpoints in trials and (2) apply this method to select outcomes for evaluation in a trial investigating treatment strategies for pulmonary exacerbations of cystic fibrosis (CF).Methods We conducted a series of online surveys and workshops among people affected by CF. We used a modified Delphi approach to develop a consensus list of important outcomes. A workshop involving domain experts elicited how these outcomes were causally related to the underlying pathophysiological processes. Meaningful outcomes were prioritised based on the extent to which each outcome captured separate rather than common aspects of the underlying pathophysiological process.Results The 10 prioritised outcomes were: breathing difficulty/pain, sputum production/clearance, fatigue, appetite, pain (not related to breathing), motivation/demoralisation, fevers/night sweats, treatment burden, inability to meet personal goals and avoidance of gastrointestinal symptoms.Conclusions This proposed method for selecting meaningful outcomes for evaluation in clinical trials may improve the value of research as a basis for clinical decisions.https://bmjopenrespres.bmj.com/content/8/1/e000877.full |
| spellingShingle | Yue Wu Charlie McLeod Richard Norman Andre Schultz Steven Mascaro Steve Webb Tom Snelling Alan Smyth Christopher Blyth Jamie Wood Siobhain Mulrennan Sue Morey Mitch Messer Kate Spaapen Matthew Stoneham Owen Woodberry Novel method to select meaningful outcomes for evaluation in clinical trials BMJ Open Respiratory Research |
| title | Novel method to select meaningful outcomes for evaluation in clinical trials |
| title_full | Novel method to select meaningful outcomes for evaluation in clinical trials |
| title_fullStr | Novel method to select meaningful outcomes for evaluation in clinical trials |
| title_full_unstemmed | Novel method to select meaningful outcomes for evaluation in clinical trials |
| title_short | Novel method to select meaningful outcomes for evaluation in clinical trials |
| title_sort | novel method to select meaningful outcomes for evaluation in clinical trials |
| url | https://bmjopenrespres.bmj.com/content/8/1/e000877.full |
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