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Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.


ABSTRACT:

Introduction

Exploring patient perceptions regarding gene therapies may provide insights about their acceptability to patients.

Objective

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.

Methods

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.

Results

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.

Conclusions

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.

SUBMITTER: van Overbeeke E 

PROVIDER: S-EPMC7894464 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature

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Publications

Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.

van Overbeeke Eline E   Michelsen Sissel S   Hauber Brett B   Peerlinck Kathelijne K   Hermans Cedric C   Lambert Catherine C   Goldman Michel M   Simoens Steven S   Huys Isabelle I  

Haemophilia : the official journal of the World Federation of Hemophilia 20201107 1


<h4>Introduction</h4>Exploring patient perceptions regarding gene therapies may provide insights about their acceptability to patients.<h4>Objective</h4>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.<h4>Methods</h4>Semi-structured individual interviews were conducted with Belgian PWH, types A and B. A predefined interview guide includ  ...[more]

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