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 and the overview of the authorised medicines/treatments in the EU and in US for sickle cell and
• 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 (including gene editing products).