Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.
advanced therapy
attitudes
gene therapies
haemophilia
interviews
opinion
preference
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916
Informations de publication
Date de publication:
Jan 2021
Jan 2021
Historique:
received:
02
04
2020
revised:
19
10
2020
accepted:
20
10
2020
pubmed:
9
11
2020
medline:
25
9
2021
entrez:
8
11
2020
Statut:
ppublish
Résumé
Exploring patient perceptions regarding gene therapies may provide insights about their acceptability to patients. To investigate opinions of people with haemophilia (PWH) regarding gene therapies. Moreover, this study aimed to identify patient-relevant attributes (treatment features) that influence PWH's treatment choices. Semi-structured individual interviews were conducted with Belgian PWH, types A and B. A predefined interview guide included information sections and open, attribute ranking and case questions. Qualitative data were organized using NVivo 12 and analysed following framework analysis. Sum totals of scores obtained in the ranking exercise were calculated per attribute. In total, 20 PWH participated in the interviews. Most participants demonstrated a positive attitude towards gene therapy and were very willing (40%; n = 8) or willing (35%; n = 7) to receive this treatment. The following five attributes were identified as most important to PWH in making their choice: annual bleeding rate, factor level, uncertainty of long-term risks, impact on daily life, and probability that prophylaxis can be stopped. While participants were concerned about the uncertainty regarding long-term safety, most participants were less concerned about uncertainty regarding long-term efficacy. This qualitative study showed that most PWH have a positive attitude towards gene therapy and that besides efficacy, safety and the related uncertainties, also impact on daily life is important to patients. The identified patient-relevant attributes may be used by regulators, health technology assessment bodies and payers in their evaluation of gene therapies for haemophilia. Moreover, they may inform clinical trial design, pay-for-performance schemes and real-world evidence studies.
Identifiants
pubmed: 33161616
doi: 10.1111/hae.14190
pmc: PMC7894464
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
129-136Subventions
Organisme : Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project
Informations de copyright
© 2020 The Authors. Haemophilia published by John Wiley & Sons Ltd.
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