Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Early Market Access - Regulatory Environment in the EU and the USA and Essential Requirements within the Pharmaceutical Company ***

Dr. Diane Seimetz (Abschlußjahr: 2002)

Drug development times have expanded enormously over the past and it seems as if the more we learn from science, the more new questions arise, resulting in increasing regulatory requirements. Today pharmaceutical industry is one of the most regulated industries in the world. For decades now drugs have significantly contributed to public health by preventing or
treating many diseases. Despite this progress there are still a considerable number of patients for which there is no or no sufficient treatment available. Pharmaceutical industry is faced with increasing pressure to discover new compounds quickly, to efficiently move drugs through clinical testing and to achieve peak sales rapidly. It is therefore in the interest of both, patients and pharmaceutical industry, to have in place a consistent and reliable regulatory environment to rapidly and effectively develop, control and approve meaningful drugs.

The present master thesis identifies, analyses and compares the regulatory environment in the EU and the USA for early market access of drugs with high therapeutic value and outlines the essential requirements within the pharmaceutical company. In the EU the centralized procedure with the option for accelerated evaluation and the granting of a marketing authorization under exceptional circumstances is identified to have a direct impact on early market access of such drugs. Besides these mechanisms on EU level some member states, e.g. Germany, have national regulations in place aiming to accelerate the market access of drugs with high therapeutic value. In the US the fast track development programs consisting of meetings, written correspondence, review programs, and dispute resolution are successfully implemented to facilitate the development and to accelerate the market access of drugs intended for the treatment of serious and life-threatening diseases. Promising drugs that are not designated as “fast track” may also benefit from individual programs such as priority review or ccelerated approval contributing to early market access. Both regions have at different stages in the drug development process and during the review phase regulatory mechanisms in place that allow for early market access of drugs, particularly for those that are intended to treat serious or life-threatening diseases and which address an unmet medical need. This is confirmed by the regulatory procedures of several drugs analyzed in this thesis. However, at present the US mechanisms are codified, more formalized and seem to work with higher efficiency. When comparing the regulatory procedures of products that were submitted almost simultaneously in both regions, it turns out that the overall approval time is shorter in the USA than in the EU. The reasons for this are argued.

The EU regulations are currently under review. The changes that are planned within the Review 2001 to promote drug development and early market access in the EU are presented and discussed. The regulatory environment as such is a major contributor to early market access. However, this external system alone does not make a promising drug come to market early. It is at least likewise important to have in place an effective environment in the pharmaceutical company that allows for accelerated development to optimize the benefit from the regulatory environment.

Thus, general considerations for accelerated drug development within the pharmaceutical company are made with having also regard to the role of regulatory affairs.

Pages: 46