EMA workshop on the challenges in drug development, regulation and clinical practice in hemoglobinopathies
EventHumanMedicines
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- The original event time is:
- Pirmadienis, Liepa 1, 2024 - 14:00 - 18:00 Amsterdam time (CEST)
Data
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Vieta
- OnlineEuropean Medicines Agency, Amsterdam, the Netherlands
Event summary
This workshop is organised in order to have a multi-stakeholder’s perspectives on sickle disease and thalassaemia before initiating the drafting of new guidelines for both diseases. This is in line with the haematology work plan for 2024.
The aims of the workshop are:
- to present the epidemiology and disease background in adults and children with sickle cell and thalassemia, the current international treatment guidelines, the unmet medical need, and the overview of the authorised medicines/treatments in the EU and in US for sickle cell and thalassemia;
- to present the challenges in treatment/drug development from a clinicians’ perspective with regards to study design and endpoints used in clinical trials as well as the introduction of new therapies such as gene therapy;
- to present additional perspectives from health technology assessment bodies, bioethics for gene editing, and the use of registries in these diseases.