title: [Authorization of tissue and blood stem cell preparations for hematopoietic reconstitution: Documentation of clinical and nonclinical data in a central expert report].
abstract: In order to address the European Directive 2004/23 on human tissues and cells, the authorization obligation for tissue and blood stem cell preparations was introduced (§ 21a AMG) in the year 2007 in the German medicinal products act. Stem cell transplantation for hematopoietic reconstitution has been in use for decades and is well established for the treatment of many malignancies. The manufacture of stem cell preparations varies, but in terms of hematopoietic reconstitution, different products are intended for the same indication. Taking these aspects into account, it was considered inappropriate that every single applicant should provide their own documentation, including an expert report on clinical and nonclinical data. Consequently, the idea came up to create a central expert report, to which all applicants could refer and would include relevant clinical and nonclinical data according to current knowledge. A central expert report was therefore generated, called the "Gemeinsame Stellungnahme der Fachgesellschaften Deutsche Gesellschaft für Transfusionsmedizin und Immunhämatologie (DGTI), Deutsche Gesellschaft für Hämatologie und Onkologie (DGH) und Gesellschaft für Pädiatrische Onkologie und Hämatologie (GPOH)". Applicants are allowed to refer to this central expert report provided their stem cell product is comparable with the cell preparations included in the report. In order to represent current knowledge, the content of the central expert report was already reworked once, but should be updated regularly.
authors.affiliation: Paul-Ehrlich-Institut, Paul-Ehrlich-Straße 51-59, 63225, Langen, Deutschland. ute.vahlensieck@pei.de.
authors.forename: Ute
authors.initials: U
authors.lastname: Vahlensieck
authors.affiliation: Paul-Ehrlich-Institut, Paul-Ehrlich-Straße 51-59, 63225, Langen, Deutschland.
authors.forename: Susanne
authors.initials: S
authors.lastname: Poley-Ochmann
authors.affiliation: Paul-Ehrlich-Institut, Paul-Ehrlich-Straße 51-59, 63225, Langen, Deutschland.
authors.forename: Anneliese
authors.initials: A
authors.lastname: Hilger
authors.affiliation: Paul-Ehrlich-Institut, Paul-Ehrlich-Straße 51-59, 63225, Langen, Deutschland.
authors.forename: Margarete
authors.initials: M
authors.lastname: Heiden
authors.affiliation: Paul-Ehrlich-Institut, Paul-Ehrlich-Straße 51-59, 63225, Langen, Deutschland.
authors.forename: Rainer
authors.initials: R
authors.lastname: Seitz
mesh_terms: D064987:Cell- and Tissue-Based Therapy / Q000592:standards*; D003625:Data Collection / Q000331:legislation & jurisprudence* / Q000592:standards*; D004282:Documentation / Q000592:standards*; D005060:Europe; D005104:Expert Testimony / Q000331:legislation & jurisprudence*; D018380:Hematopoietic Stem Cell Transplantation / Q000331:legislation & jurisprudence* / Q000592:standards*; D006801:Humans; D009313:National Health Programs / Q000331:legislation & jurisprudence*; D061214:Patient Safety; D011785:Quality Assurance, Health Care / Q000331:legislation & jurisprudence*; D011786:Quality Control
keywords: Authorization of tissue and blood stem cell preparations; European Directive 2004/23 on human tissues and cells; Hematopoietic stem cell transplantation; Hematopoietic stem cells