Improving the Methdology for Patient-Centred Trials in Cystic fibrosis (IMPACT-CF)

This thesis explores the optimal selection of meaningful outcomes and endpoints for late stage trials designed to inform practice and policy, using trials of pulmonary exacerbations in people with cystic fibrosis (CF) as an exemplar. Herein I present (i) the outcomes of importance to people with lived experience of CF and the methods for measurement of these outcomes, (ii) a novel method for optimising the selection of outcomes for late phase trials and (iii) a weighted patient-reported outcome measure for application as one measure of treatment success in intervention trials of pulmonary exacerbations in people with CF.