Clinical investigation of recombinant and human plasma-derived factor IX products - Scientific guideline

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

Adopted Reference Number: EMA/CHMP/BPWP/144552/2009 Rev. 2 Legal effective date: 30/06/2024

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)

Revision 2

Guideline on the clinical investigation of recombinant and human plasma-derived factor IX products - Revision 2

Adopted Reference Number: EMA/CHMP/BPWP/144552/2009 Rev. 2 Legal effective date: 30/06/2024

Overview of comments on 'Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 2'

Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 2

Draft: consultation closed Consultation dates: 03/12/2018 to 30/06/2019 Reference Number: EMA/CHMP/BPWP/144552/2009 rev. 2 Corr.1

Revision 1

Guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 1

Adopted Reference Number: EMA/CHMP/BPWP/144552/2009 Rev.1 Corr. 1 Legal effective date: 01/09/2015 Summary:

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.

Overview of comments received on ' Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 1'

Reference Number: EMA/CHMP/BPWP/111667/2015

Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products - Revision 1

Draft: consultation closed Consultation dates: 06/01/2015 to 07/02/2015 Reference Number: EMA/CHMP/BPWP/144552/2009 rev 1 Summary:

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.

First version

Guideline on clinical investigation of recombinant and human plasma-derived factor IX products

Adopted Reference Number: EMA/CHMP/BPWP/144552/2009 Legal effective date: 01/02/2012 Summary:

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.

Draft guideline on the clinical investigation of recombinant and human plasma-derived factor IX products

Draft: consultation closed Reference Number: EMEA/CHMP/BPWP/144552/2009

Superseded documents

Draft guideline on the clinical Investigation of Recombinant Factor VIII and IX Products - Superseded

Draft: consultation closed Reference Number: CPMP/BPWG/1561/99 rev.1

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

Reference Number: EMEA/CHMP/BPWG/189106/2004

Note for guidance on the clinical investigation of human plasma-derived factor VIII and IX products - Superseded

Adopted Reference Number: CPMP/BPWG/198/95 rev.1 Legal effective date: 01/04/2001