@article {McLeode000877, author = {Charlie McLeod and Richard Norman and Jamie Wood and Siobhain Mulrennan and Sue Morey and Andr{\'e} Schultz and Mitch Messer and Kate Spaapen and Matthew Stoneham and Yue Wu and Alan Smyth and Christopher Blyth and Steve Webb and Steven Mascaro and Owen Woodberry and Tom Snelling}, title = {Novel method to select meaningful outcomes for evaluation in clinical trials}, volume = {8}, number = {1}, elocation-id = {e000877}, year = {2021}, doi = {10.1136/bmjresp-2021-000877}, publisher = {Archives of Disease in childhood}, abstract = {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.No data are available. Data is available largely as hard copy survey responses and is therefore not available.}, URL = {https://bmjopenrespres.bmj.com/content/8/1/e000877}, eprint = {https://bmjopenrespres.bmj.com/content/8/1/e000877.full.pdf}, journal = {BMJ Open Respiratory Research} }