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Speakers cover the need for long-term data on the safety and efficacy of gene therapy

Tuesday’s session, “Critical need for harmonized post-marketing surveillance in gene therapy” looked at the urgent need for a mechanism to collect long-term data on the safety, variability, and durability of efficacy of gene therapy. Peter Marks of the U.S. Food and Drug Administration (FDA) and Caroline Voltz on the European Medicines Agency (EMA) talked about the issue in depth.

The session also featured Michael Recht, who discussed the importance of collaborating with other databases and harmonization of patient data, and Barbara Konkle who elaborated on the WFH Gene Therapy Registry initiative and how it aims to fill the need for long-term follow data in gene therapy.

To watch sessions from the WFH Virtual Summit – Connecting the Global Bleeding Disorders Community, please click here.