Current challenges in the development of novel cell-based medicinal products

Current challenges in the development of novel cell-based medicinal products

Article on Current challenges in the development of novel cell-based medicinal products, 

by Paula Salmikangas, Finnish Medicines Agency, Helsinki, Finland; Patrick Celis, European Medicines Agency, London, UK. (From http://www.topra.org/)

Abstract of the article is----

The scientific knowledge in cell and molecular biology and rapid development in biomaterial technology have fostered the development of new innovative therapies especially for diseases and tissue/organ defects for which traditional therapies have not provided satisfactory outcomes. The progress in research and development has been most rapid in cellular therapies, where almost 250 new cell-based medicinal products (CBMPs) have been in clinical studies in the EU during the past five years. While regulatory requirements for quality, safety and efficacy apply to these innovative products as for all other medicinal products, their demonstration is associated with very specific challenges related to the manufacture, nonclinical and clinical testing, as well as administration of the products. On the other hand, these therapies also involve certain risks that may be more difficult to mitigate and manage than with other medicinal products (eg, immunotoxicity, problems in viral and TSE safety, tumourigenicity, insertional mutagenesis). The situation may be especially challenging for autologous products, for which extensive testing may not be possible due to restrictions related to small sample sizes and short shelf-lives. The wide variety of different products and possible indications has called for a risk-based approach, which companies may use to justify the amount of data in their applications for marketing authorisations.Whereas the legal and regulatory framework for advanced therapies has enhanced the development of cell-based medicinal products, it has also created an uncertain atmosphere among developers due to the divergent national interpretations of article 28 of Regulation (EC) No.1394/2007 (the so-called hospital exemption). The European Medicines Agency (EMA) and the Committee for Advanced Therapies (CAT) are aware of the main current obstacles and challenges faced by CBMP developers, and the possibilities for increased and early dialogue with the regulatory authorities – to manage these regulatory and scientific challenges – are addressed in this article. 

Reference: Current challenges in the development of novel cell-based medicinal products