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

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

doi:10.1016/j.jcf.2019.10.025

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

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

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

78 Citations (Scopus)

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.

Original language	English
Pages (from-to)	499-502
Number of pages	4
Journal	Journal of Cystic Fibrosis
Volume	19
Issue number	3
Early online date	14 Nov 2019
DOIs	https://doi.org/10.1016/j.jcf.2019.10.025
Publication status	Published - May 2020
Externally published	Yes

Bibliographical note

Funding Information:
Members of the James Lind Alliance CF2 steering group include: Brownlee K, Collins S, Daniels T, Davies G, Duff AJA, Elliot ZC, Gathercole K, Hurley MN, Leighton PA, Rayner O, Rowbotham NJ, Smith S, Chandran S, Nash EF, Smyth AR (Chair), Wilson P. We would like to thank PwCF and their families, and the multi-disciplinary professional CF community for taking part in this survey. This work was funded by the UKCF Trust , and the University of Nottingham . G Davies was supported an NIHR Academic Clinical Lectureship at UCL. N Rowbotham is an NIHR Academic Clinical Fellow at University of Nottingham.

Funding Information:
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.

Publisher Copyright:
© 2019 European Cystic Fibrosis Society

Keywords

Clinical trial
Co-production
Cystic fibrosis
Priority setting
Treatment burden

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Pulmonary and Respiratory Medicine

Access to Document

10.1016/j.jcf.2019.10.025Licence: Other

Cite this

Davies, G., Rowbotham, N. J., Smith, S., Elliot, Z. C., Gathercole, K., Rayner, O., Leighton, P. A., Herbert, S., Duff, A. JA., 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

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title = "Characterising burden of treatment in cystic fibrosis to identify priority areas for clinical trials",

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",

author = "Gwyneth Davies and Rowbotham, \{Nicola J.\} and Sherie Smith and Elliot, \{Zoe C.\} and Katie Gathercole and Oli Rayner and Leighton, \{Paul A.\} and Sophie Herbert and Duff, \{Alistair JA\} and Suja Chandran and Tracey Daniels and Nash, \{Edward F.\} and Smyth, \{Alan R.\}",

note = "Funding Information: Members of the James Lind Alliance CF2 steering group include: Brownlee K, Collins S, Daniels T, Davies G, Duff AJA, Elliot ZC, Gathercole K, Hurley MN, Leighton PA, Rayner O, Rowbotham NJ, Smith S, Chandran S, Nash EF, Smyth AR (Chair), Wilson P. We would like to thank PwCF and their families, and the multi-disciplinary professional CF community for taking part in this survey. This work was funded by the UKCF Trust , and the University of Nottingham . G Davies was supported an NIHR Academic Clinical Lectureship at UCL. N Rowbotham is an NIHR Academic Clinical Fellow at University of Nottingham. Funding Information: 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 {\textquoteleft}Alkyl quinolones as biomarkers of Pseudomonas aeruginosa infection and uses thereof{\textquoteright} 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. Publisher Copyright: {\textcopyright} 2019 European Cystic Fibrosis Society",

year = "2020",

month = may,

doi = "10.1016/j.jcf.2019.10.025",

language = "English",

volume = "19",

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AU - Davies, Gwyneth

AU - Rowbotham, Nicola J.

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AU - Elliot, Zoe C.

AU - Gathercole, Katie

AU - Rayner, Oli

AU - Leighton, Paul A.

AU - Herbert, Sophie

AU - Duff, Alistair JA

AU - Chandran, Suja

AU - Daniels, Tracey

AU - Nash, Edward F.

AU - Smyth, Alan R.

N1 - Funding Information: Members of the James Lind Alliance CF2 steering group include: Brownlee K, Collins S, Daniels T, Davies G, Duff AJA, Elliot ZC, Gathercole K, Hurley MN, Leighton PA, Rayner O, Rowbotham NJ, Smith S, Chandran S, Nash EF, Smyth AR (Chair), Wilson P. We would like to thank PwCF and their families, and the multi-disciplinary professional CF community for taking part in this survey. This work was funded by the UKCF Trust , and the University of Nottingham . G Davies was supported an NIHR Academic Clinical Lectureship at UCL. N Rowbotham is an NIHR Academic Clinical Fellow at University of Nottingham. Funding Information: 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. Publisher Copyright: © 2019 European Cystic Fibrosis Society

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N2 - 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.

AB - 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.

KW - Clinical trial

KW - Co-production

KW - Cystic fibrosis

KW - Priority setting

KW - Treatment burden

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DO - 10.1016/j.jcf.2019.10.025

M3 - Article

C2 - 31735561

AN - SCOPUS:85075426248

SN - 1569-1993

VL - 19

SP - 499

EP - 502

JO - Journal of Cystic Fibrosis

JF - Journal of Cystic Fibrosis

IS - 3

ER -

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

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

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