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
English (EN) (492.58 KB - PDF)
Draft guideline on clinical investigation of recombinant and human plasma-derived factor VIII products - Revision 2
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.
English (EN) (378.86 KB - PDF)
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)
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).
English (EN) (92.85 KB - PDF)
Document history - Revision 1
Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII 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 VIII products is made for use in treatment and prevention of bleeding in patients with haemophilia A.
English (EN) (332.13 KB - PDF)
Draft guideline on the clinical investigation of recombinant and human plasma-derived factor VIII 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 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.
English (EN) (330.21 KB - PDF)
Document history - First version
Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products - First version
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.
English (EN) (302.35 KB - PDF)
Draft guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products - First version
English (EN) (192.53 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 Recombinant Factor VIII and IX Products - Superseded
English (EN) (111.4 KB - PDF)
Related content
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)
- Clinical efficacy and safety: blood products (including biotech alternatives)
- Guideline on core SmPC for human plasma derived and recombinant coagulation factor VIII products
- Directive 2001/83/EC
- Paediatric Regulation (EC) 1901/2006
- Points to consider for assessors - New factor VIII and factor IX products: potency determination for labelling and assays for testing post-infusion samples