Regulatory requirements for clinical trial and marketing authorisation application for cell-based medicinal products

Bundesgesundheitsblatt, Gesundheitsforschung, Gesundheitsschutz
P SalmikangasR Maciulaitis

Abstract

The new era of regenerative medicine has led to rapid development of new innovative therapies especially for diseases and tissue/organ defects for which traditional therapies and medicinal products have not provided satisfactory outcome. Although the clinical use and developments of cell-based medicinal products (CBMPs) could be witnessed already for a decade, robust scientific and regulatory provisions for these products have only recently been enacted. The new Regulation for Advanced Therapies (EC) 1394/2007 together with the revised Annex I, Part IV of Directive 2001/83/EC provides the new legal framework for CBMPs. The wide variety of cell-based products and the foreseen limitations (small sample sizes, short shelf life) vs. particular risks (microbiological purity, variability, immunogenicity, tumourigenicity) associated with CBMPs have called for a flexible, case-by-case regulatory approach for these products. Consequently, a risk-based approach has been developed to allow definition of the amount of scientific data needed for a Marketing Authorisation Application (MAA) of each CBMP. The article provides further insight into the initial risk evaluation, as well as to the quality, non-clinical, and clinical requirements of...Continue Reading

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Citations

Nov 19, 2015·Kidney International·James A Hutchinson, Edward K Geissler
Jun 24, 2016·Stem Cells Translational Medicine·Gaia BarazzettiAlexandre Mauron
Mar 12, 2020·Ocular Immunology and Inflammation·Claudia Eugenia Duran MerinoAna María Rodríguez

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