Loading...
Examining patient and professional perspectives in the UK for gene therapy in haemophilia
Woollacott, Ione Morgan, George Chowdary, Pratima O'Hara, Jamie Franks, Bethany van Overbeeke, Eline Dunn, Nicola Michelsen, Sissel Huys, Isabelle Martin, Antony
Woollacott, Ione
Morgan, George
Chowdary, Pratima
O'Hara, Jamie
Franks, Bethany
van Overbeeke, Eline
Dunn, Nicola
Michelsen, Sissel
Huys, Isabelle
Martin, Antony
Citations
Altmetric:
Advisors
Editors
Other Contributors
EPub Date
Publication Date
2022-04-19
Submitted Date
Collections
Files
Loading...
Article - VoR
Adobe PDF, 3.03 MB
Other Titles
Abstract
With the development of gene therapy for people with haemophilia (PWH), it is important to understand how people impacted by haemophilia (PIH) and clinicians prioritise haemophilia treatment attributes to support informed treatment decisions. To examine the treatment attribute preferences of PIH and clinical experts in the United Kingdom (UK) and to develop a profile of gene therapy characteristics fit for use in future discrete choice experiments (DCEs). Semi-structured interviews were conducted with PIH (n = 14) and clinical experts (n = 6) who ranked pre-defined treatment attributes by importance. Framework analysis was conducted to identify key themes and treatment attributes; points were allocated based on the rankings. Synthesis of results by a multidisciplinary group informed development of a profile of gene therapy characteristics for use in future research. Key themes identified by PIH and clinical experts included patient relevant features and the importance of 'informed decision making'. The six top-ranked treatment attributes were 'effect on factor level' (79 points), 'uncertainty regarding long-term risks' (57 points), 'impact on daily life' (41 points), 'frequency of monitoring' (33 points), 'impact on ability to participate in physical activity' (29 points), and 'uncertainty regarding long-term benefits' (28 points). The final treatment characteristics were categorised as therapeutic option, treatment effectiveness, safety concerns, impact on self-management and quality of life (role limitations). We identified several gene therapy characteristics important to PIH and clinicians in the UK. These characteristics will be used in a future DCE to further investigate patient preferences for gene therapy. [Abstract copyright: © 2022 John Wiley & Sons Ltd.]
Citation
Woollacott, I., Morgan, G., Chowdary, P., O'Hara, J., Franks, B., van Overbeeke, E., Dunn, N., Michelsen, S., Huys, I., Martin, A., Cawson, M., Brownrigg, J., Winburn, I., & Thomson, J. (2022). Examining patient and professional perspectives in the UK for gene therapy in haemophilia. Haemophilia, 28(4), 588-609. https://doi.org/10.1111/hae.14572
Publisher
Journal
Haemophilia
Research Unit
PubMed ID
PubMed Central ID
Type
Article
Language
Description
Series/Report no.
ISSN
1351-8216
EISSN
1365-2516
ISBN
ISMN
Gov't Doc
Test Link
Sponsors
Funder: Pfizer