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

This document covers clinical investigations to be conducted pre- and post-marketing authorisation for recombinant or human plasma-derived factor VIII products that are intended to use in the treatment and prevention of bleeding in patients with haemophilia A.

Keywords: Recombinant factor VIII, plasma-derived factor VIII, efficacy, safety, immunogenicity, inhibitor, potency assays

Document history - Revision 2

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

Adopted Reference Number: EMA/CHMP/BPWP/144533/2009 Legal effective date: 01/02/2019

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

Draft: consultation closed Consultation dates: 30/10/2017 to 31/01/2018 Reference Number: EMA/CHMP/BPWP/144533/2009 Rev. 2 Summary:

This guideline describes the information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor VIII products is made for use in treatment and prevention of bleeding in patients with haemophilia A. The guidance 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.

Concept paper on revision of guidelines on the clinical investigation and core SmPC of recombinant and human plasma-derived factor VIII products (EMA/CHMP/BPWP/144533/2009 rev. 1) (EMA/CHMP/BPWP/1619/1999 rev. 2)

Draft: consultation closed Consultation dates: 01/08/2016 to 30/09/2016 Reference Number: EMA/CHMP/BPWP/383118/2016 Summary:

The proposed guideline will replace guidelines on the clinical investigation and core SmPC of recombinant and human plasma-derived factor VIII products(EMA/CHMP/BPWP/144533/2009 rev. 1) (EMA/CHMP/BPWP/1619/1999 rev. 2).

Document history - Revision 1

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

Adopted Reference Number: EMA/CHMP/BPWP/144533/2009 rev. 1 Legal effective date: 01/05/2016 Summary:

This guideline describes the information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor VIII products is made for use in treatment and prevention of bleeding in patients with haemophilia A.

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

Draft: consultation closed Consultation dates: 01/06/2015 to 01/07/2015 Reference Number: EMA/CHMP/BPWP/144533/2009 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 VIII products is made for use in treatment and prevention of bleeding in patients with haemophilia A. The guidance 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.

Document history - First version

Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products - First version

Adopted Reference Number: EMA/CHMP/BPWP/144533/2009 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 VIII products is made for use in treatment and prevention of bleeding in patients with haemophilia A. The guidance 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 VIII products - First version

Draft: consultation closed Reference Number: EMEA/CHMP/BPWP/144533/2009

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 Recombinant Factor VIII and IX Products - Superseded

Adopted Reference Number: CPMP/BPWG/1561/99 Legal effective date: 01/04/2001