Masterarbeit

Tissue or Blood Product - Regulatory requirements concerning the manufacture and clinical use of human hematopoietic stem cells in Germany ***

Dr. Mathias Krech (2013)

Summary
Language: German

Therapeutic use of human hematopoietic stem cells obtained from bone marrow, peripheral blood or cord blood is part of the current standard therapy for several hematopoietic, genetic and immunologic diseases. Collection and use of hematopoietic stem cells depends on the concept of therapy as well as on the availability of the stem cell source for autologous, allogeneic directed and allogeneic undirected donation. Therapeutic use of stem cell preparation relies on stringent quality requirements of the product and of the manufacturing process. Each stem cell preparation is to be manufactured, tested and released for clinical use as an individual batch.
In Germany, human hematopoietic stem cell products are regulated by complex drug regulatory legislation. Complexity arises from the legal definition of stem cells from cord blood and apheresis-derived blood as a blood product, while bone marrow derived hematopoietic stem cells is considered a tissue. This differentiation within the hematopoietic stem cells has been recently questioned, since it is not justified by medical reasons and significantly increases the regulatory and administrative burdens of the manufacture and clinical use, as well as of authorization and inspection by the involved authorities.

The present master thesis gives an overview of the current regulatory framework for hematopoietic stem cells in Germany and describes the specific pharmaceutical legislation and regulatory requirements on stem cells from bone marrow, apheresis-derived blood and cord blood. By way of introduction, the standard procedures established in Germany for collection and manufacture of hematopoietic stem cells are outlined. Recent results on manufacturing development and transplantation activities in Germany are presented based on retrospective analyses of statistical data from PEI reports pursuant to §21 German Transfusion Act and §8d German Transplant Act, and the yearly statistical summaries of the Deutsches Register für Stammzelltransplantation. The focus of the regulatory considerations are the product specific rules, procedures and competences for the manufacture, import/export and the placing on the market of hematopoietic stem cells as well as the mandatory report and notification requirements to the hemo/tissue-vigilance system. Regarding the heterogeneous guidelines for stem cell preparations from cord blood, bone marrow and apheresis-derived blood, the requisites for a uniform legal framework for hematopoietic stem cells is being discussed.

Pages: 71, Annexes: 5