An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families

An Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their Families

This study programme aims to examine the real-world experience and impact of gene therapy in a diverse community of people and families affected by haemophilia who have been or will be exposed to gene therapy.

This is a prospective observational multiple cohort qualitative research study to be conducted among diverse groups within the haemophilia community whose lives may have been impacted by gene therapy.

The study is designed to allow English-speaking patients and their families to tell their own life stories through narrative accounts. The narratives represent a true sharing of experiences and offer insight into how these patients and families cope with haemophilia.

Inclusion Criteria:

• People with haemophilia A or B who consented to and have undergone gene therapy in the early dose-finding studies and a member of their family
• People with haemophilia A or B who consented to a gene therapy trial following the results of the early studies and a member of their family
• People with haemophilia A or B who consented to a gene therapy trial but who withdrew, were withdrawn from, or were ineligible for the study, and a member of their family
• People with haemophilia A or B who are definitely not interested in or unaware of gene therapy and a member of their family
• People with haemophilia A or B who are interested in but have not been offered gene therapy
• Those who have given written consent to be in the study
• All participants will be ≥16 years.

Exclusion Criteria:

• Participants will be excluded if they do not speak English (for the interviews) or do not consent to be in the study.