Consultation open for draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products

By William Chin, PhD, Scientific Coordinator, EUCRAF

The Blood Products Working Party (BPWP) at EMA has opened the draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products for use in the treatment and prevention of bleeding in patients with haemophilia B, for public consultation from the 6 January to 7 February 2015. This guideline covers the following topics:

  • Information to be documented when an application for a marketing authorisation for recombinant or human plasma-derived factor IX products is made
  • Clinical investigations to be conducted pre- and post-marketing authorisation
  • Significant change in the manufacturing process 
  • Adverse events
  • Safety with respects to viruses and other transmissble agents
  • Immunogenicity
  • Thrombogenicity
  • Efficacy in previous treated and untreated patients as well as in children
  • Risk management plan 

Source:

1. Scientific guideline: Draft guideline on clinical investigation of recombinant and human plasma-derived factor IX products, draft: consultation open, 6 January 2015