<abstract xmlns="http://www.w3.org/1999/xhtml"><p> The investigators behind the first gene therapy trial with adenoassociated 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.</p></abstract>

The investigators behind the first gene therapy trial with adenoassociated 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.

Patient advocacy helps patients weigh up gene therapy trial risk/benefits

The Journal of Haemophilia Practice

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

{"article-title":"Patient advocacy helps patients weigh up gene therapy trial risk/benefits"}

The investigators behind the first gene therapy trial with adenoassociated virus 8 (AAV8) Factor IX appointed a patient ombudsperson to help ensure...

Patient advocacy helps patients weigh up gene therapy trial risk/benefits

Article Category: Gene Therapy

Published Online: Apr 21, 2018

Page range: 6 - 8

DOI: https://doi.org/10.17225/jhp.00040

Keywords
haemophilia, haemophilia B, Christmas Disease, gene therapy, factor IX, informed consent

© 2018

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

Patient advocacy helps patients weigh up gene therapy trial risk/benefits

Article Category: Gene Therapy

Published Online: Apr 21, 2018

Page range: 6 - 8

DOI: https://doi.org/10.17225/jhp.00040

Keywordshaemophilia, haemophilia B, Christmas Disease, gene therapy, factor IX, informed consent

© 2018

This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.

Keywords
haemophilia, haemophilia B, Christmas Disease, gene therapy, factor IX, informed consent