This guideline describes the information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor IX products is made for use in the treatment and prevention of bleeding in patients with haemophilia B. The guideline covers clinical investigations to be conducted pre- and post-marketing authorisation. Guidance is also provided for authorised products where a significant change in the manufacturing process has been made.
Keywords: Recombinant factor IX, plasma-derived factor IX, efficacy, safety, immunogenicity, inhibitor, thrombogenicity, anaphylactic reactions, potency assays
Current effective version
Guideline on the clinical investigation of recombinant and human plasma-derived factor IX products - Revision 2
English (EN) (396.49 KB - PDF)
Questions and answers on the revision of the guidelines on clinical investigation of recombinant and human plasma-derived factor VIII products (EMA/CHMP/BPWP/144533/2009 Rev. 2) and FIX products (EMA/CHMP/BPWP/144552/2009 Rev. 2)
English (EN) (136.85 KB - PDF)
Revision 2
Guideline on the clinical investigation of recombinant and human plasma-derived factor IX products - Revision 2
English (EN) (396.49 KB - PDF)
Overview of comments on 'Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 2'
English (EN) (405.39 KB - PDF)
Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 2
English (EN) (575.99 KB - PDF)
Revision 1
Guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 1
This guideline describes the information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor IX products is made for use in treatment and prevention of bleeding in patients with haemophilia B.
English (EN) (347.64 KB - PDF)
Overview of comments received on ' Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 1'
English (EN) (140.82 KB - PDF)
Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 1
This guideline describes the information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor IX products is made for use in treatment and prevention of bleeding in patients with haemophilia B. The guideline covers clinical investigations to be conducted pre- and post-marketing authorisation. Guidance is also provided for authorised products where a significant change in the manufacturing process has been made.
English (EN) (284.4 KB - PDF)
First version
Guideline on clinical investigation of recombinant and human plasma-derived factor IX products
This guideline describes the information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor IX products is made for use in treatment and prevention of bleeding in patients with haemophilia B. The guideline covers clinical investigations to be conducted pre- and post-marketing authorisation. Guidance is also provided for authorised products where a significant change in the manufacturing process has been made.
English (EN) (295.22 KB - PDF)
Draft guideline on the clinical investigation of recombinant and human plasma-derived factor IX products
English (EN) (241.89 KB - PDF)
Superseded documents
Draft guideline on the clinical Investigation of Recombinant Factor VIII and IX Products - Superseded
English (EN) (237.44 KB - PDF)
Concept paper on the revision of notes for guidance on the clinical investigation of human plasma derived and recombinant factor VIII and IX products and the corresponding Core SPCs - Revision 1 - Superseded
English (EN) (34.15 KB - PDF)
Note for guidance on the clinical investigation of human plasma-derived factor VIII and IX products - Superseded
English (EN) (114.39 KB - PDF)