Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

„Regulatory framework for the marketing authorisation of medicinal products for human use containing or consisting of genetically modified organisms – comparison of requirements in Europe and the United States“

Dr. Christiane Pering (Abschlußjahr: 2006)

Summary

The development of genetic engineering techniques in the 1970s (eg, in vitro nucleic acid processes and technology-aided cell fusions), with the consequent ability to produce genetically modified organisms (GMOs) and transgenic species, necessitated the development of additional guidelines and regulations that would allow development and application of the “new” biotechnology, while minimising the risks to human health and safety, and avoiding environmental damage.

The European Communitys regulatory framework for biotechnology was designed in the late 1980s in order to provide the necessary legislation to ensure adequate protection of health and the environment, while at the same time creating the internal market for biotechnological products. On the basis of the scientific knowledge of the early 1980s and the limited practical experience of that time with GMOs especially in industrial applications, “horizontal legislation” was introduced in Europe based on the process by which products were produced, eg, utilization of rDNA techniques in a variety of industries, which included contained use of genetically modified micro-organisms, deliberate release into the environment of genetically modified organisms, and the protection of workers from risks related to exposure to biological agents at work. In addition “product” legislation (medicinal products, additives used in animal nutrition, plant protection products, novel foods, seeds) as well as legislation on intellectual property protection also form part of this framework.

Regulatory oversight of biotechnology has been in place in the US for longer than in most other parts of the world. The first environmental release of a GMO occurred in 1983 following the approval of the National Institutes of Health (NIH). This approval of a field test of “ice-minus” bacteria used for preventing frost damage on strawberries sparked a heated controversy, challenging the NIH decision and questioning the ability of federal agencies to address hazards to ecosystems in light of the uncertainties. As a consequence the Coordinated Framework for Regulation of Biotechnology was introduced in 1986, describing the basic network of Federal agency jurisdiction for evaluating both research and products developed using modern biotechnology. With it, the US adopted early on a product-based, vertical, preventive regulatory approach, starting with research and development activities until authorization to market of the new products under the roof of one single federal agency.

Despite harmonization efforts on the international level and extensive exchange of scientific evidence between regulators for the past 25 years regarding the development of new gene therapy products, differences remain regarding the requirements to obtain a marketing authorisation for GMOs in medicinal products in the different regions of the world, depending on the influence of local views on economical, ethical and environmental aspects on the respective regulatory settings.

In this thesis an overview is provided on the current legislation in place in Europe and the US to obtain a marketing authorization for medicinal products containing or consisting of GMOs. Furthermore the similarities and differences between the two regions are described and an outlook on future regulatory activities in this area is given.

Overall, care has to be taken that the adoption of a new and more and more strict legislation in most instances triggered by safety concerns or uncertainties on the overall implications of a new therapeutic product does not result in hurdles very difficult to overcome and associated with a substantial rise in R & D costs. This could eventually lead to a significant decline in R & D activities in the field of human gene therapies, because in many instances diseases of high public interest but of limited commercial attractiveness are the target.

Pages: 72