The Haematology, Cell and Gene Therapy Division accompanies all steps in the life cycle of the medicinal products it supervises, from providing scientific advice, to approving clinical trials, up to authorisation or approval. This is supplemented by federal batch testing and release as a prerequisite for placing blood products on the market after authorisation, as well as inspections.
The division is responsible for:
In addition, the division regularly collects, evaluates, and publishes relevant information on the clinical use and supply situation of the medicinal products within its area of responsibility.
Another focus of the division’s tasks is scientific and regulatory advice to manufacturers and developers from industry and science, as well as advice to policymakers, because ATMPs in particular often require specific strategies and adapted solutions for successful development.
Experts from the division represent the Institute on relevant national and international committees.
The division's research accompanying trials and basic research on relevant and current aspects of biomedical therapy development complements its technical expertise in the regulatory field.
This section evaluates the clinical and non-clinical trials of all medicinal products under the responsibility of the Haematology, Cell and Gene Therapy Division within the framework of all national and European regulatory procedures.
This section evaluates the production and quality of blood products and their recombinant versions as well as gene therapy medicinal products within the framework of all national and European procedures. Another central task is the scientific coordination and evaluation of the plasma master files at the Paul-Ehrlich-Institut.
This section also supports the competent state authorities in inspections for compliance with Good Manufacturing Practice (GMP) with regard to the state of the art in science and technology in the manufacture of blood products and gene therapy medicinal products.
This section tests quality and safety-relevant parameters of blood products made from plasma in the laboratory as part of the legally required official control authority batch release (OCABR). It also examines and evaluates the manufacturing documents to ensure compliance with the marketing authorisation and with European standards. In addition, the section coordinates the experimental testing of plasma pools for relevant viral markers. Recombinantly produced analogues and centrally authorised medicinal products are tested within the framework of Centrally Authorised Product (CAP) testing procedures.
Alongside its tasks related to batch testing and release of blood products, the section conducts basic research.
The office of the German Haemophilia Registry (Deutsches Hämophilieregister, DHR) is located within the "Reporting" Section.
Further tasks of this section are the collection and evaluation of reports according to legal requirements, e.g. the Transfusion Act, the Transplantation Act or the Preimplantation Regulation. A modular register platform is maintained and continuously developed to collect these reports.
The section is responsible for blood components for transfusion, haematopoietic stem cells, somatic cell therapeutics and tissue engineering products that are not genetically modified, as well as human tissue preparations. It evaluates all submitted data on the quality of these medicinal products and, in consultation with professional societies, prepares model forms and document specifications in order to harmonise and simplify regulatory procedures. All products for which the section is responsible are monitored throughout their life cycle.
This section brings together the division’s research groups on transposition and genome engineering, stem cell safety, xenogeneic medicinal products, and the Genomics Core Facility. Joint projects with experts from the other disciplines of the division on various research topics ensure synergies between research and regulation.
Updated: 13.08.2024
