Year: 2020
Language: English
Author(s): World Federation of Hemophilia
With regulatory approval of the first gene therapy product expected, there is an urgent need for a mechanism to collect long-term data on safety and variability and durability of efficacy. There will be elements required by regulators for post marketing surveillance and additional data needed to enhance our understanding of gene therapy outcomes and their impact on the lives of people with hemophilia.
In this session from the WFH 2020 Virtual Summit, Barbara Konkle, Michael Recht, Caroline Voltz and Peter Marks discuss efforts underway by the American Thrombosis and Hemostasis Network and the World Federation of Hemophilia to collect long-term harmonized data and considerations of the European and US regulatory agencies, which will inform ongoing data collection.
The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH recommends that you contact your physician or local treatment centre. Before administering any products, the WFH urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.
The WFH does not promote any particular pharmaceutical product and any mention of any commercial brand in this presentation is strictly for educational purposes.
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