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

Dobra RA, Boeri M, Elborn S, Kee F, Madge S, Davies JC

BMJ open, 2021

Abstract

INTRODUCTION: 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.

METHODS AND ANALYSIS: 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.

ETHICS AND DISSEMINATION: Imperial College London Joint Research Compliance Office has granted ethical approval for this study. Patient consent will be sought following full explanation. No identifying…

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Evidence Hub reads this as a cross-sectional, done in people. Its evidence level is 4 of 5 (Low · observational). Observational designs can show an association but not prove cause.

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