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Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.
van Overbeeke, Eline; Michelsen, Sissel; Hauber, Brett; Peerlinck, Kathelijne; Hermans, Cedric; Lambert, Catherine; Goldman, Michel; Simoens, Steven; Huys, Isabelle.
Afiliación
  • van Overbeeke E; Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium.
  • Michelsen S; Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium.
  • Hauber B; Health Preference Assessment, RTI Health Solutions, Research Triangle Park, NC, USA.
  • Peerlinck K; Haemophilia Center, UZ Leuven, Leuven, Belgium.
  • Hermans C; Haemophilia Clinic, St-Luc University Hospital, Brussels, Belgium.
  • Lambert C; Haemophilia Clinic, St-Luc University Hospital, Brussels, Belgium.
  • Goldman M; Institute for Interdisciplinary Innovation in healthcare, Université libre de Bruxelles, Brussels, Belgium.
  • Simoens S; Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium.
  • Huys I; Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium.
Haemophilia ; 27(1): 129-136, 2021 Jan.
Article en En | MEDLINE | ID: mdl-33161616
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.
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Hemofilia A Tipo de estudio: Health_technology_assessment / Prognostic_studies / Qualitative_research Límite: Humans Idioma: En Revista: Haemophilia Asunto de la revista: HEMATOLOGIA Año: 2021 Tipo del documento: Article País de afiliación: Bélgica

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Hemofilia A Tipo de estudio: Health_technology_assessment / Prognostic_studies / Qualitative_research Límite: Humans Idioma: En Revista: Haemophilia Asunto de la revista: HEMATOLOGIA Año: 2021 Tipo del documento: Article País de afiliación: Bélgica