Discrete choice experiment (DCE) to quantify the influence of trial features on the decision to participate in cystic fibrosis (CF) clinical trials.

Engaging people with cystic fibrosis (CF) in clinical trials is critical to improving outcomes for this fatal disease. Following extensive exploration of engagement in CF trials we believe six key concepts require a quantitative understanding of their influence in the current CF trials landscape including how controversial issues like placebos, washouts, stipend provision and location of trial visits are viewed by the CF community and how these might be modified depending on the type of medicine being investigated and the mechanism of access to the drug on trial completion. We have designed and will administer an online discrete choice experiment to elicit and quantify preferences of people with CF for these trials' attributes and estimate the relative importance of an attribute when choosing to participate in a trial. The cross-sectional data generated will be explored using conditional multinomial logit model. Mixed logit models such as the random-parameters logit and a latent class models will be used to explore preference heterogeneity. To determine the relative importance of an attribute, the difference between the attribute level with the highest preference weight and the level with the lowest preference weight will be calculated. Imperial College London Joint Research Compliance Office has granted ethical approval for this study. Patient consent will be sought following full explanation. No identifying information will be collected. Dissemination will be via international conferences, peer-review publication and patient accessible forums. Major CF trials networks have agreed to incorporate our findings into their review process, meaning our results can realistically influence and optimise CF trial delivery. CRD42020184886.

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Déclaration de conflit d'intérêts

Competing interests: RD, MB, FK and SM have no competing interests to declare. JCD has served on advisory boards and participated in clinical trial leadership, educational activities and grant review board activities for a number of pharma companies active in CF clinical trials: Vertex, PTI, Galapagos, AbbVie, AlgiPharma, Chiesi, Enterprise, Teva, Ionis, Eloxx, Roche and Gilead. JSE has provided consultancy and advice for Vertex, Celtaxsys, Corbus, Ionis in clinical trial design and delivery. He also holds an EU Innovative medicines Initiative grant with Novartis, Polyphor and Alaxia.