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. 2020 May;19(3):499-502.
doi: 10.1016/j.jcf.2019.10.025. Epub 2019 Nov 15.

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

Gwyneth Davies  1 , Nicola J Rowbotham  2 , Sherie Smith  2 , Zoe C Elliot  3 , Katie Gathercole  4 , Oli Rayner  5 , Paul A Leighton  6 , Sophie Herbert  2 , Alistair Ja Duff  7 , Suja Chandran  8 , Tracey Daniels  9 , Edward F Nash  10 , Alan R Smyth  2
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Free article

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

Gwyneth Davies et al. J Cyst Fibros. 2020 May.
Free article

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.

Keywords: Clinical trial; Co-production; Cystic fibrosis; Priority setting; Treatment burden.

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Conflict of interest statement

Declaration of Competing Interest A Duff reports personal fees and non-financial support from Chiesi pharmaceuticals, personal fees from Novartis pharmaceuticals, outside the submitted work. A Smyth reports grants from Vertex, personal fees from Vertex and support for educational meetings from Teva, outside the submitted work; In addition, A Smyth has a patent ‘Alkyl quinolones as biomarkers of Pseudomonas aeruginosa infection and uses thereof’ issued. N Rowbotham reports non-financial support from Teva, outside the submitted work. T Daniels reports personal fees from Profile pharma, personal fees from Gilead, personal fees from Chiesi, other from Teva, personal fees from Vertex, outside the submitted work. All other authors have no other conflict of interest to declare.

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