Masterstudiengang "Drug Regulatory Affairs"
Master-Thesis
Novel biomedical products: Conventional biologicals or ATMPs ***
Dr. Gabriele Noffz (Abschlußjahr: 2011)
Language: English
Biotechnology has evolved a broad variety of divergent medicinal products including hormones and blood factors as well as vaccines and monoclonal antibodies for chronic, acute and rare diseases, such as cancer, hepatitis C, diabetes, multiple sclerosis and rheumatoid arthritis. Many of those biological medicinal products are either regulated through Regulation (EC) No. 726/2004 or Directive 2001/83/EC depending on the manufacturing process used or their application. In some cases they are authorised nationally. However, a harmonised regulation covering all biological products does not exist. Such medicinal products are indicated as conventional biologics in this master thesis.
New emerging fields of biotechnology are therapies based on gene therapy, somatic cell therapy or tissue engineering. Such innovative therapeutics are summarised under the term advanced therapy medicinal products (ATMP). ATMPs offer new opportunities for treatment of rare or previously untreated diseases, early detection of dysfunctions of the human body or others, e.g. skin replacement products.
In 2007 a regulation for ATMPs, Regulation (EC) No. 1394/2007, has been adopted to overcome the lack of a clear and coherent legal framework and to boost research in this field. The intention was to harmonise the regulation of these therapies and to adapt technical specifications to ATMP products. The regulation involves a classification procedure for these products. This is important for companies in case the classification of the case is ambiguous as the application of ATMP requires more data regarding quality and safety compared to other biological products.
The aim of the master thesis is to give an overview of the classification and demarcation of ATMPs as compared to other biological medicinal products. The criteria defined for the classification of ATMPs are mainly established in the Regulation (EC) No. 1394/2007 and the consolidated Directive 2001/83/EC demonstrating that the classification of ATMPs and thus, the demarcation of specialised therapeutic biological products to other biological products have a significant impact on regulatory requirements. Key aspects of the regulatory requirements are highlighted. Significance and limitations of this classification are discussed, especially regarding clarity and suitability of the classification criteria, the limitation of the ATMPs to the three classification groups gene therapy medicinal products, somatic cell medicinal products and tissue engineered products and the impact of the demarcation in view of health protection and health market. Furthermore, the differences of ATMPs and biotechnological medicinal products justifying the question of a demarcation are discussed.