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.
The guideline covers clinical investigations to be conducted pre- and post-marketing authorisation. In general, quality aspects are outside the scope of this guideline. The document has to be read in conjunction with the introduction and general principles (4) and Annex I to Directive 2001/83/EC as amended, as well as the Paediatric Regulation (EC) 1901/2006 as amended.
The guideline includes efficacy aspects, safety aspects, information for marketing authorisation as well as aspects for changes in the manufacturing process.
Further information is available in the "Guideline on clinical investigation of recombinant and human plasma-derived factor IX products".