Clinical efficacy and safety: haematology and blood products (including biotech alternatives)

The European Medicines Agency's scientific guidelines on the clinical evaluation of blood products help medicine developers prepare marketing authorisation applications for human medicines.

For a complete list of scientific guidelines currently open for consultation, see Public consultations.

Haemophilia

Human coagulation factor VIII & human coagulation factor IX

Guidelines

• Clinical investigation of recombinant and human plasma-derived factor VIII products
• Clinical investigation of recombinant and human plasma-derived factor IX products
• Core summary of product characteristics for human plasma-derived and recombinant coagulation factor VIII products
• Core summary of product characteristics for human plasma-derived and recombinant coagulation factor IX products

Reflection papers

• Immune tolerance induction in haemophilia A patients with inhibitors

Non replacement therapies 

• Clinical requirements for non replacement therapy in haemophilia A and B

Other coagulation factors

• Clinical investigation of human plasma-derived von Willebrand factor products
• Core summary of product characteristics for human fibrinogen products
• Core summary of product characteristics for human plasma coagulation factor VII products
• Core summary of product characteristics for human plasma derived von Willebrand factor
• Core summary of product characteristics for human prothrombin complex products

Chronic primary immune thrombocytopenia

• Clinical development of medicinal products intended for the treatment of chronic primary immune thrombocytopenia
• Need for a guideline on the clinical development of thrombopoetin receptor agonists for the treatment of chronic immune (idiopathic) thrombocytopenic purpura

Haematopoietic growth factors

• Endpoints in clinical studies with haematopoietic growth factors for mobilisation of autologous stem cells

Specific immunoglobulins

• Clinical investigation of human anti-D immunoglobulin for intravenous and/or intramuscular use
• Clinical investigation of hepatitis B immunoglobulins
• Core summary of products characteristics for human anti-D immunoglobulin for intramuscular use
• Core summary of product characteristics for human anti-D immunoglobulin for intravenous use
• Core summary of product characteristics for human plasma-derived hepatitis B immunoglobulin for intramuscular use
• Core summary of product characteristics for human plasma-derived hepatitis B immunoglobulin for intravenous use
• Core summary for product characteristics for human rabies immunoglobulin for intramuscular use
• Core summary of product characteristics for human tetanus immunoglobulin for intramuscular use
• Core summary of product characteristics for human tick-borne encephalitis immunoglobulin for intramuscular use
• Core summary of product characteristics for human varicella immunoglobulin for intramuscular use

Normal immunoglobulins

• Clinical investigation of human normal immunoglobulin for intravenous administration (IVIg)
• Clinical investigation of human normal immunoglobulin for subcutaneous and/or intramuscular administration (SCIg/IMIg)
• Core summary of product characteristics for human normal immunoglobulin for intravenous administration (IVIg)
• Core summary of product characteristics for human normal immunoglobulin for subcutaneous and intramuscular use

Other

• Clinical investigation of plasma-derived antithrombin products
• Clinical investigation of plasma-derived fibrin sealant/haemostatic products
• Core summary for product characteristics for human albumin solution
• Core summary of product characteristics for human plasma derived antithrombin
• Core summary of product characteristics for plasma-derived fibrin sealant/haemostatic products

General

• Warning on transmissible agents in summary of product characteristics and package leaflets for plasma-derived medicinal products

Clinical efficacy and safety: blood and blood-forming organs

The European Medicines Agency's scientific guidelines on the clinical evaluation of human medicines used in conditions affecting the blood and blood-forming organs help medicine developers prepare marketing authorisation applications.

For a complete list of scientific guidelines currently open for consultation, see Public consultations.

Guidelines

• Clinical development of medicinal products intended for the treatment of chronic primary immune thrombocytopenia

Points to consider

• Endpoints in clinical studies with haematopoietic growth factors for mobilisation of autologous stem cells

Concept paper

• Need for a guideline on the clinical development of thrombopoetin receptor agonists for the treatment of chronic immune (idiopathic) thrombocytopenic purpura