Division Haematology / Transfusion Medicine
Core tasks include the assessment of blood components for transfusion, haematopoietic stem cells derived from bone marrow, peripheral blood and cordblood, non-substantially manipulated stem cells – which as human somatic cell therapeutics belong to the group of advanced therapy medicinal products (ATMPs) – and medicinal products made from human plasma and their bioengineered analogues. The division oversees all the stages in the blood product development cycle, from early advice and the approval of clinical trials through to marketing authorisation and subsequent procedures – supported by testing-based research. The division’s experts represent the PEI in a highly committed manner on the national and international relevant committees, e.g. of the World Health Organization (WHO), the European Medicines Agency (EMA) and the European Directorate for the Quality of Medicines (EDQM).
Head of Division’s Research Group
The research team constantly advances scientific expertise in the area of blood through independent research. In addition to basic research topics, the focus is also on aspects related to testing, including the further development of methods and safety-relevant parameters for measuring coagulation factors and blood components such as platelet concentrates.
German Haemophilia Registry and Reporting
The division makes, as the operator of the clinical haemophilia registry (DHR), an important contribution to ensuring the transparency and safety of supply for haemophilia patients. The DHR reflects the realities of clinical treatment and enables long-term (longitudinal) recording of patient data. In future, it is also intended that the DHR will document novel treatment options, such as the use of monoclonal antibodies or gene therapies. It reports in accordance with Section 21 of the German Transfusion Act (TFG) record the manufacture and consumption of blood products in order to guarantee the transparency of supply and demand and to highlight trends in the supply situation. The results are published in annual reports.
Coagulation Products I Section
The section coordinates all European and national procedures for plasma proteins and bioengineered analogues. The section’s responsibilities also include the assessment of clinical and non-clinical studies and of SPCs and package leaflets. In order to help shape the scientific requirements on an international level, the section is actively involved in the Blood Products Working Party (BPWP) of the European Medicines Agency (EMA), which defines the European specifications for the clinical testing of blood products with regard to marketing authorisation.
Coagulation Products II Section
The section assesses the quality of plasma-derived blood products and bioengineered analogues. The section’s core tasks also include the assessment and the lead coordination of the plasma master file (PMF) at the PEI. PMFs are fundamental documents which ensure that plasma pools meet European standards. The section’s international networking is strengthened by the fact that its experts are members and chairs of relevant committees at the EDQM and EMA.
Batch Release Blood Products, Logistics Section
The section determines, in the laboratory, quality and safety-relevant parameters of plasma-derived blood products in the context of official batch testing. It reviews and evaluates the manufacturing documents to ensure that the marketing authorisations comply with European standards. In addition, samples of the starting material, the plasma pool, are tested experimentally for relevant viral markers. The European Commission, via the European network of Official Medicines Control Laboratories (OMCLs), prescribes centralised testing procedures, thus guaranteeing consistent quality and efficacy.
Transfusion Medicine Section
The section processes the marketing authorisation procedures of blood components for transfusion. It is constantly developing the procedures, the details of which are coordinated in consultation with representatives from the professional associations. They draw up sample forms and document templates in order to harmonise and simplify procedures.
The section’s responsibilities also include the marketing authorisation or approval of haematopoietic stem cells derived from peripheral blood, cord blood and bone marrow – also in the form of ATMPs.