Katharine Dormandy Haemophilia Centre & Thrombosis Unit, Ground floor, Royal Free Hospital, Pond Street, London NW3 2QG, UK. Email: firstname.lastname@example.org
The investigators behind the first gene therapy trial with adeno-associated virus 8 (AAV8) Factor IX appointed a patient ombudsperson to help ensure participants were able to give truly informed consent. The experiences and challenges of the ombudsperson, who met with the first six UK-based patients, are described. It was stressed to potential participants that altruism, rather than any expectation of clinical benefit, should be the primary motivation to taking part. At the same time a sober assessment of the potential risks to their safety needed to be made.
The Journal of Haemophilia Practice is published by Haemnet.
Haemnet is a registered charity that brings together and gives a voice to haemophilia nurses, physiotherapists and allied health care professionals, providing forums for collaborative research, educational activities and support.