Risk-benefit assessment for Gene Therapy in Haemophilia ft. Declan Noone, EHC President
Haemcast20 Joulu 2021

Risk-benefit assessment for Gene Therapy in Haemophilia ft. Declan Noone, EHC President

Declan Noone, President of the European Haemophilia Consortium (EHC) joins Haemcast hosts, Dr Kate Khair and Luke Pembroke to discuss the complexities surrounding risk-benefit assessment when making a decision around gene therapy. From considering the patients' and healthcare professional perspectives to the regulators and payers, thorough risk-benefit assessment processes will be required in order to make informed decisions about gene therapy for haemophilia. But what is needed from the community to support these decision making processes? How can we ensure equal access and opportunity to gene therapy in the future? How do we begin to try quantifying the benefits of gene therapy without focusing solely on annual bleed rates and factor levels? Listen in to hear the thoughts of our experts in this final episode of Haemcast for 2021.

Thank you to all of our listeners throughout the year for tuning in and supporting Haemcast. The feedback we have received has been amazing and confirmed that it serves as a valuable resource for the community. If you want to help supporting us, please do leave us a review and rating on the platform you listen on e.g. Apple podcasts, Spotify and be sure to share Haemcast with your colleagues and friends.

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Quality of life in the balance: Helen Tate on living with Factor V deficiency

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