Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

Witkop, M; Morgan, G; O'Hara, J; Recht, M; Buckner, TW; Nugent, D; Curtis, R; O'Mahony, B; Skinner, MW; Mulhern, B; Cawson, M; Ali, TM; Sawyer, EK; Li, N

Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

Witkop, M Morgan, G O'Hara, J Recht, M Buckner, TW Nugent, D Curtis, R O'Mahony, B Skinner, MW Mulhern, B

Cawson, M Ali, TM Sawyer, EK Li, N

Permalink

Publisher:: Wiley
Publication Type:: Journal Article
Citation:: Haemophilia, 2021
Issue Date:: 2021-07-26

Open Access

Copyright Clearance Process

Recently Added
In Progress
Open Access

This item is open access.

Adobe PDF

Download full textAdobe PDF (1.07 MB)

View on publisher's site

View statistics

Full metadata record

Field	Value	Language
dc.contributor.author	Witkop, M
dc.contributor.author	Morgan, G
dc.contributor.author	O'Hara, J
dc.contributor.author	Recht, M
dc.contributor.author	Buckner, TW
dc.contributor.author	Nugent, D
dc.contributor.author	Curtis, R
dc.contributor.author	O'Mahony, B
dc.contributor.author	Skinner, MW
dc.contributor.author	Mulhern, B https://orcid.org/0000-0003-3656-8063
dc.contributor.author	Cawson, M
dc.contributor.author	Ali, TM
dc.contributor.author	Sawyer, EK
dc.contributor.author	Li, N
dc.date.accessioned	2021-08-30T00:59:08Z
dc.date.available	2021-07-15
dc.date.available	2021-08-30T00:59:08Z
dc.date.issued	2021-07-26
dc.identifier.citation	Haemophilia, 2021
dc.identifier.issn	1351-8216
dc.identifier.issn	1365-2516
dc.identifier.uri	http://hdl.handle.net/10453/150254
dc.description.abstract	<h4>Introduction</h4>Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments.<h4>Aim</h4>We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences patients provide for gene therapy attributes.<h4>Methods</h4>We surveyed male adults with haemophilia in the United States recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform using an online survey over 4 months in 2020/21. Participants indicated preferences for gene therapy attributes including dosing frequency/durability, effect on annual bleeding, uncertainty related to side effects, impact on daily activities, impact on mental health, and post-treatment requirements. The relative importance of each attribute was analysed overall and for subgroups based on haemophilia type and severity.<h4>Results</h4>A total of 183 males with haemophilia A (n = 120) or B (n = 63) were included. Half (47%) had severe haemophilia; most (75%) were White. Overall, participants gave effect on bleeding rate the greatest relative importance (31%), followed by dose frequency/durability (26%), uncertainty regarding safety issues (17%), and impact on daily activities (11%). Dose frequency/durability had the greatest importance for those with haemophilia B (35%).<h4>Conclusion</h4>People with haemophilia prioritised reduced bleeding and treatment burden; the former was more important in haemophilia A and the latter in haemophilia B, followed by safety and impact on daily life in this DCE of gene therapy attributes. These findings and differences can inform clinical and health policy decisions to improve health equity for people with haemophilia.
dc.format	Print-Electronic
dc.language	eng
dc.publisher	Wiley
dc.relation.ispartof	Haemophilia
dc.relation.isbasedon	10.1111/hae.14383
dc.rights	info:eu-repo/semantics/openAccess
dc.subject	1103 Clinical Sciences
dc.subject.classification	Cardiovascular System & Hematology
dc.title	Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment
dc.type	Journal Article
utslib.location.activity	England
utslib.for	1103 Clinical Sciences
pubs.organisational-group	/University of Technology Sydney
pubs.organisational-group	/University of Technology Sydney/Faculty of Business
pubs.organisational-group	/University of Technology Sydney/Strength - CHERE - Centre for Health Economics Research and Evaluation
utslib.copyright.status	open_access	*
dc.date.updated	2021-08-30T00:59:07Z
pubs.publication-status	Published

Abstract:

Introduction

Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments.

Aim

We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences patients provide for gene therapy attributes.

Methods

We surveyed male adults with haemophilia in the United States recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform using an online survey over 4 months in 2020/21. Participants indicated preferences for gene therapy attributes including dosing frequency/durability, effect on annual bleeding, uncertainty related to side effects, impact on daily activities, impact on mental health, and post-treatment requirements. The relative importance of each attribute was analysed overall and for subgroups based on haemophilia type and severity.

Results

A total of 183 males with haemophilia A (n = 120) or B (n = 63) were included. Half (47%) had severe haemophilia; most (75%) were White. Overall, participants gave effect on bleeding rate the greatest relative importance (31%), followed by dose frequency/durability (26%), uncertainty regarding safety issues (17%), and impact on daily activities (11%). Dose frequency/durability had the greatest importance for those with haemophilia B (35%).

Conclusion

People with haemophilia prioritised reduced bleeding and treatment burden; the former was more important in haemophilia A and the latter in haemophilia B, followed by safety and impact on daily life in this DCE of gene therapy attributes. These findings and differences can inform clinical and health policy decisions to improve health equity for people with haemophilia.

Please use this identifier to cite or link to this item:

http://hdl.handle.net/10453/150254