Clinical requirements for medicines intended for the treatment of sickle cell disease

This guideline aims at providing guidance on general principles of the clinical development programme of medicinal products for the treatment of sickle cell disease (SCD). This guideline focuses on the confirmatory phase 3 trials investigating safety and efficacy and serving as the main basis for benefit risk assessment of these products.

Keywords: Sickle Cell Disease (SCD), vaso-occlusive crises (VOC), haemolysis, haemolytic anaemia, transfusion

Current version

Draft guideline on the clinical requirements for medicines intended for the treatment of sickle cell disease

Draft: consultation open Consultation dates: 24/09/2026 to 31/01/2027 Reference Number: EMA/CHMP/HAEMWP/351950/2024 Summary:

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