Many seriously ill people are depending on continual research for new drug therapies for their disease. Typically, such a development takes several years from the discovery of a promising compound in a research laboratory until it is commercially available for the treatment of patients. But in cases of a disabling or life-threatening disease, when no alternative therapies are available, the time needed for full development and authority review may be too long to provide these patients with the benefit of the new compound. Consequently, many of these patients are willing to accept the greater risk associated with a treatment using an unlicensed medicinal product and also their treating physicians are taking a different approach in balancing the risks of a possibly beneficial treatment with the consequences of disease progression.

The aim of this thesis is to present an overview of the different possibilities patients suffering from a serious disease have, in order to gain access to yet unlicensed medicinal products. The countries in focus are Germany and France, as the two major pharmaceutical markets within in the EU and the United States as the biggest pharmaceutical market in the world.

The results of this thesis show that the national provisions governing the use of unlicensed medicinal products differ significantly in Germany, France and the USA. Whereas in the USA, the relevant mechanisms have developed consecutively according to arising needs in the course of time, France has established a consistent regulation in order to meet the needs of desperately ill patients. Although the European legislation allows the implementation of such procedures in each Member State, Germany is still missing an explicit mechanism to allow patients access to investigational drugs for treatment use.

The "Evaluation of the operation of Community procedures for the authorisation of medicinal products" carried out by CMS Cameron McKenna and Andersen Consulting on behalf of the European Commission indicated that guidance on Community level regarding this topic would be highly appreciated by all concerned parties in the EU.

Therefore, proposals are currently discussed, which equip the European Agency for the Evaluation of Medicinal Products with the competence to set standards for compassionate use programmes, which are to be applied by the EU Member States within their own administrative framework. The intention of this regulation is to ensure a greater equity between patients in Europe regarding the access to new drug therapies.

Pages: 31