Abstract

Regulatory Pathways to the Market

Timo Faltus, Investigator, TRM Leipzig

The European market is a financially strong market for pharmaceuticals. One of the reasons for that is that there is a well-developed health care system in the European Union. The financial capacity in the health care sector and the fact of a big market for new medicinal therapies make the European market interesting for the introduction of new pharmaceuticals such as (stem) cell based therapies. However, the legal hurdles to get a new (stem) cell based medicinal product into that market are high and complex. The presentation illustrates the European legal system for research and market approval in the field of (stem-) cell based products. One of the major statutes in that field is the so called ATMP-Regulation, which regulates Advanced Therapy Medicinal Products, such as gene therapy products, somatic cell products, and tissue engineered products. Any product which falls within the scope of the ATMP-regulation must undergo a centralized European market approval regime by the European Medicines Agency (EMA) in London. However, this practice of the regular centralized market approval can be very costly and may be oversized for small and medium sized companies which are the major developer of (stem) cell based products. Therefore, there are a number of financial amenities for small and medium sized companies as well as exemptions and shortcuts from the requirement for a centralized market authorization. The presentation therefore will explain these amenities as well as the requirements to enter the European market without a centralized market approval. Due to current trends in the field of cell based therapies the presentation will focus on the regulation of autologous therapy concepts.