The Exigency Study

 

The Exigency study programme aims to examine the real-world experience and impact of gene therapy in a diverse community of people, families and health care professionals affected by haemophilia. It will also explore the expectations that patients, families and health care professionals in the UK have of gene therapy and its position in contemporary and future haemophilia management.

Gene therapy is a novel approach to the management of haemophilia likely to gain regulatory approval in the coming months. It is likely to have a major impact on the lives of people with haemophilia and their families, and also on the healthcare professionals who care for them.

The Exigency study aims to gain a greater understanding of:

• The “real life” experience of people who have undergone gene therapy
• The impact of gene therapy on the extended family
• The impact of being ineligible for treatment
• The impact of being withdrawn from treatment and how this might affect the individuals’ ongoing attitude to their haemophilia care
• The educational and support needs of health care providers regarding gene therapy.

This is a non-interventional study being conducted by Haemnet with Oxford University Hospitals NHS Foundation Trust. All data will be gathered by means of structured face-to-face interviews or focus groups conducted by trained professionals. Interviews and focus groups may be conducted in person or by video call. Each interview will be recorded and transcribed. Transcripts will then be anonymised to ensure confidentiality, and analysed using accepted methodologies.

We hope to interview people over the age of 16 years who fall into the following three groups:

• People with haemophilia who consented to and have undergone gene therapy, together with a family member (spouse/partner or sibling)
• Those with haemophilia who have not undergone gene therapy either because they have not been offered gene therapy, are definitely not interested in or are unaware of gene therapy, or because they have consented to a gene therapy trial but withdrew/were withdrawn from it, or were found to be ineligible for the study, together with a family member (spouse/partner or sibling).

If you would like to take part in the study, please click on the link that best describes you in the list above. This will download a more detailed participant information sheet. If you would then still like to take part, please send an email Dr Kate Khair, our Director of Research, who will confirm your eligibility. Then, after you have signed and returned a participant consent form, our researchers will be in touch with you to arrange an interview.

Once the study has been completed, we will post a summary of the results on this page, and will send copies to all of these who have taken part.