Characterising burden of treatment in cystic fibrosis to identify priority areas for clinical trials

Davies, Gwyneth; Rowbotham, Nicola J; Smith, Sherie; Elliot, Zoe C; Gathercole, Katie; Rayner, Oli; Leighton, Paul A; Herbert, Sophie; Duff, Alistair Ja; Chandran, Suja; Daniels, Tracey; Nash, Edward F; Smyth, Alan R

Abstract

In a recent James Lind Alliance Priority Setting Partnership in cystic fibrosis (CF) the top priority clinical research question was: “What are effective ways of simplifying the treatment burden of people with CF?” We aimed to summarise the lived experience of treatment burden and suggest research themes aimed at reducing it. An online questionnaire was co-produced and responses subjected to quantitative and thematic analysis. 941 survey responses were received (641 from lay community). People with CF reported a median of 10 (interquartile range: 6–15) current treatments. Seven main themes relating to simplifying treatment burden were identified. Treatment burden is high, extending beyond time taken to perform routine daily treatments, with impact varying according to person-specific factors. Approaches to communication, support, evaluation of current treatments, service set-up, and treatment logistics (obtaining/administration) contribute to burden, offering scope for evaluation in clinical trials or service improvement.

Citation

Davies, G., Rowbotham, N. J., Smith, S., Elliot, Z. C., Gathercole, K., Rayner, O., Leighton, P. A., Herbert, S., Duff, A. J., Chandran, S., Daniels, T., Nash, E. F., & Smyth, A. R. (2020). Characterising burden of treatment in cystic fibrosis to identify priority areas for clinical trials. Journal of Cystic Fibrosis, 19(3), 499-502. https://doi.org/10.1016/j.jcf.2019.10.025

• Journal Article Type: Article
• Acceptance Date: Oct 29, 2019
• Online Publication Date: Nov 14, 2019
• Publication Date: 2020-05
• Deposit Date: Nov 5, 2019
• Publicly Available Date: Nov 15, 2020
• Journal: Journal of Cystic Fibrosis
• Print ISSN: 1569-1993
• Electronic ISSN: 1873-5010
• Publisher: Elsevier
• Peer Reviewed: Peer Reviewed
• Volume: 19
• Issue: 3
• Pages: 499-502
• DOI: https://doi.org/10.1016/j.jcf.2019.10.025
• Keywords: Treatment burden; cystic fibrosis; clinical trial; co-production; priority setting
• Public URL: https://nottingham-repository.worktribe.com/output/3058224
• Publisher URL: https://www.sciencedirect.com/science/article/pii/S1569199319309610
• Contract Date: Nov 5, 2019