Regulatory requirements for clinical trial and marketing authorisation application for cell-based medicinal products.
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.
Salmikangas
P
P
National Agency for Medicines, Mannerheimintie 103b, FI-00301, Helsinki. Paula.salmikangas@nam.fi
Flory
E
E
Reinhardt
J
J
Hinz
T
T
Maciulaitis
R
R
D017690:Cell Transplantation / Q000331:legislation & jurisprudence*; D002986:Clinical Trials as Topic / Q000331:legislation & jurisprudence*; D005060:Europe; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000331:legislation & jurisprudence*; D017408:Guidelines as Topic; D006801:Humans; D008389:Marketing of Health Services / Q000331:legislation & jurisprudence*; D011785:Quality Assurance, Health Care / Q000331:legislation & jurisprudence; D023822:Tissue Engineering / Q000331:legislation & jurisprudence*