Cell therapy medicinal product regulatory framework in Europe and its application for MSC-based therapy development

Janis Ancans

doi:10.3389/fimmu.2012.00253

Abstract

Advanced therapy medicinal products (ATMPs), including cell therapy products, form a new class of medicines in the European Union. Since the ATMPs are at the forefront of scientific innovation in medicine, specific regulatory framework has been developed for these medicines and implemented from 2009. The Committee for Advanced Therapies (CAT) has been established at the European Medicines Agency (EMA) for centralized classification, certification and evaluation procedures, and other ATMP-related tasks. Guidance documents, initiatives, and interaction platforms are available to make the new framework more accessible for small- and medium-sized enterprises, academia, hospitals, and foundations. Good understanding of the centralized and national components of the regulatory system is required to plan product development. It is in the best interests of the cell therapy developers to utilize the resources provided starting with the pre-clinical stage. Whilst there have been no mesenchymal stem cell (MSC)-based medicine authorizations in the EU, three MSC products have received marketing approval in other regions since 2011. The information provided on the regulatory requirements, procedures, and initiatives is aimed at facilitating MSC-based medicinal product development and authorization in the EU.

Highlights

The scientific progress and advances in the biotechnology sector have led to the development of therapies which are based on the use of living cells, recombinant genetic material, and in vitro engineered tissue
For cells to be classified as medicinal products they have to fulfill at least one of the following conditions: the cells have been subject to substantial manipulation and/or these cells are not intended for use for the same essential function
“Guideline on the risk-based approach according to Annex I, part IV of Directive 2001/83/EC applied to Advanced Therapy Medicinal Products” is an important advanced therapy medicinal products (ATMPs) guidance document which has been published for public discussion and will be finalized by the end of 2012

Summary

Introduction

The scientific progress and advances in the biotechnology sector have led to the development of therapies which are based on the use of living cells, recombinant genetic material, and in vitro engineered tissue. The European pharmaceutical regulatory framework is applied, in particular the requirements of the Directive 2001/83/EC and the Regulation (EC) N◦ 726/2004, the Regulation (EC) N◦ 1394/2007 with regard to the ATMPs. The EMA is responsible for the centralized procedure for medicines and the Committee for Advanced Therapies (CAT) evaluates ATMP submissions.

Results

Conclusion