Clinical requirements for medicines intended for the treatment of thalassaemia
HumanScientific guidelines
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This guideline aims at providing guidance on general principles of the clinical development programme of medicinal products for the treatment of α and β-thalassaemia. 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 medicines.
Keywords: α-thalassaemia, β-thalassaemia, transfusion-dependent, non-transfusiondependent, iron overload