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

Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2010 Jan;53(1):24-9. doi: 10.1007/s00103-009-0991-5.

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 CBMPs. Special somatic cell therapies designed for active immunotherapy are also addressed.

MeSH terms

  • Cell Transplantation / legislation & jurisprudence*
  • Clinical Trials as Topic / legislation & jurisprudence*
  • Europe
  • Gene Transfer Techniques
  • Genetic Therapy / legislation & jurisprudence*
  • Guidelines as Topic
  • Humans
  • Marketing of Health Services / legislation & jurisprudence*
  • Quality Assurance, Health Care / legislation & jurisprudence
  • Tissue Engineering / legislation & jurisprudence*