Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Marketing authorisation of medicinal products used to prevent the toxicity of chemical, biological, radiological or nuclear substances (MA of Anti-CBRN Drugs) ***

Dr. Boris Mey (Abschlußjahr: 2005)

Comparison of the respective regulations and guidelines in place in the United States of America, in the European Union, in Germany and in France.

Summary

Language: English

The possible use of chemical, biological, radiological or nuclear (CBNR) agents within battlefield or terrorist scenarios has risen significantly since the terrorist attacks in the US in autumn 2001. Worldwide international and multinational organisations and institutions as well as many governments launched immediately major activities and protection measures related to CBNR threats to improve public health security and preparedness. In order to respond adequately in an emergency following CBNR attacks worldwide new, safe, and approved medicinal products are needed for the prophylaxis and treatment of the diseases potentially caused by CBNR agents to remedy the anomaly of either being up to now incapable to respond adequately to certain threats by fault of suitable medicinal products or to avoid/reduce the up to now inevitable off-label or non-authorised use of medicines. Whereas in the US enormous efforts are undertaken to close this gap of capabilities in the public health system, the actions and resources launched by the European Community and its Member States are rather reluctant. In Germany, in France and meanwhile also in the US the off-label and non-licensed use of drugs in CBNR emergencies can be authorised to assure a controlled marketing and a controlled administration of such drugs.

In the present study the current regulatory situation in the US in comparison to the EU and its Member States Germany and France is analysed and discussed with specific regard to Anti-CBNR drugs. To counter the insufficient availability, accessibility and distribution of approved Anti-CBNR drugs in emergencies, specific measures are needed to encourage the research, development and marketing of such drugs and to make them accessible in a sufficient quality and quantity to the may be concerned population. Based on the specific characteristics and difficulties related to Anti-CBNR drugs proposals for the further improvement of the existing pharmaceutical legislation are drawn up. The designation of Anti-CBNR drugs as a particular category of drugs is described as a may be suitable measure to facilitate and encourage the supply with such drugs.

Since year 2000 seven new medicinal products and six new medical devices have been approved for Anti-CBNR use in the US. The approval of these drugs was based on the existing regulations developed to foster the marketing authorisation (MA) of drugs for the prophylaxis/treatment of life threatening (rare) diseases/conditions as for example the rules for orphan drugs as well as for fast track and accelerated approvals. Additional regulations like the meanwhile enacted animal efficacy rule and the respective guidance for industry are analysed.

In the EU up to now Anti-CBNR medicines have to be administered mainly without MA (emergency use, off label use, etc.) or based on non-harmonised national MA. In the course of the review process of the European pharmaceutical legislation in 2004 many regulatory innovations have and will be introduced which will have a positive impact on the development and marketing of Anti-CBNR drugs as for example the introduction of accelerated assessment procedures, the introduction of a temporary MA in connection with the imposition of specific obligations and the support of small and medium-sized enterprises in the field of pharmaceuticals.

The German Standard MA system for substances without patent protection should be extended to all EU Member States to put the MA agencies into the position to produce or to take over and maintain MA for essential medicines of public interest on their own.

With regard to the International Conference on Harmonisation (ICH) proposals for new tripartite harmonised guidelines (ethics in clinical trials; realisation of the animal efficacy rule; production of drugs in emergency situations) and for the amendment/update of existing guidelines (ICH guidelines M3(M), E6 and E 8) are made.

Besides creating a favourable regulatory environment, it is the duty of the State to take precautions within the framework of civil protection and disaster control and towards high risk personal as for example the personal of its military forces. It is the responsibility of governmental bodies/institutions to invest in the build up, remuneration and renewal of national stockpiles of Anti-CBNR drugs. A steady investment in this domain will encourage automatically private institutions and companies to develop and market new medicines. Alternatively, in case of very limited financial resources, new strategies and programmes under the lead of the agencies responsible for the MA might mitigate the inadequate supply with Anti-CBNR drugs.

Pages: 50, Annexes:34