4th Gene Therapy Round Table webinar covers challenges and opportunities


The third and final webinar of the 4th World Federation of Hemophilia (WFH) Gene Therapy Round Table (GTRT) was held on September 16, 2021, and featured a number of experts discussing the challenges and opportunities presented by gene therapy.

The first presentation of the webinar was given by Radoslaw Kaczmarek, WFH Coagulation Product Safety, Supply, and Access (CPSSA) Committee chair, and covered latest news about gene therapy clinical trials. This was followed by a discussion by Leonard Valentino, MD, on how to help people with hemophilia thrive by bringing a shared decision making (SDM) approach to hemophilia management. Declan Noone, President of the European Haemophilia Consortium (EHC), provided an update on the development of the WFH SDM tool. Breakout groups then allowed participants to provide expert feedback on the details of the SDM tool, with each group discussing different components of the tool.

The second session of the webinar focused on paying and reimbursing for gene therapy for hemophilia. Glenn Pierce, MD, PhD, WFH Vice President, Medical, discussed the costs of gene therapy and other considerations beyond price. Edmund Pezalla, MD, and Anja Schiel, PhD, then presented perspectives from the U.S.A and Europe on the challenges of developing novel payment methods for gene therapy.

To find out more about the WFH GTRT series, please click here.

To learn more about the latest news about gene therapy clinical trials, please see the video below—available in English, with additional voiceover versions in French, Spanish, Arabic and Russian (coming soon)—where Glenn Pierce and Steve Pipe, MD, discuss the gene therapy information that was shared at the ISTH Virtual Congress.