EMA workshop on the challenges in drug development, regulation and clinical practice in hemoglobinopathies

• Date:
  Monday, 01 July 2024 , 14:00 - 18:00 Amsterdam time (CEST)
• Location:
  Online
  European 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.

Video recording

Documents

Meeting report - Workshop on the challenges in drug development, regulation and clinical practice in haemoglobinopathies

Agenda - Workshop on the challenges in drug development, regulation and clinical practice in haemoglobinopathies

Presentation - Patients and clinical perspectives and management of Thalassemia (Ali Taher)

Presentation - Management of, and unmet needs in, Sickle Cell disorders (John Porter)

Presentation - Challenges in treatment/drug development from a clinicians’ perspective with regards to study design and endpoints used in clinical trials for management of paediatric Sickle Cell disease (Raffaella Colombatti, Mariane De Montalembert)

Presentation - EMA workshop on the challenges in drug development, regulation and clinical practice in hemoglobinopathies (A Loris Brunetta)

Presentation - Approved medicines in Sickle Cell Disease and Thalassemia diseases and regulatory considerations by FDA CDER (Patricia Ann Oneal)

Presentation - Regulatory considerations CBER (Megha Kaushal)

Presentation - Authorised medicines and regulatory considerations by EMA (Johanna Lähteenvuo)

Presentation - OPEN and other collaborative procedures (Radhouane Cherif)

Presentations - Bioethics considerations on gene editing (Laurence Lwoff)

Presentation - Hemoglobinopathies HTA challenges (Anja Schiel)

Presentation - Experience from registries (María del Mar Mañú Pereira)

Presention - Challenges in drug development for haemoglobinopathies (Antonella Isgrò)