Masterarbeit

The repurposing of already marketing drugs to be used in the field of rare diseases: challenges, advantages and regulatory strategy in the European Union

Olga Borsuk (2015)

Summary
Language: English
In the last three decades, the general public has become aware of issues concerning rare diseases such as lack of medicinal products, physicians and researches. The limited number of the patients suffering from rare diseases impeded the development of medicines, as return of investments and profits were implausible. To solve these issues, a legal regulatory basis was required.
The regulatory framework has significantly promoted the development of medicinal products indicated for the treatment of rare diseases, namely, orphan medicines. However, it has been insufficient to address this concern.
Therefore, strategies that may bring a medicinal product more rapidly to the market are crucial. One of such approach is drug repurposing, which allows using known compounds for the new therapeutic areas.  This strategy reduces the medicine development period, as some preclinical and clinical researches have already been performed.
On the other hand, drug repurposing in the field of rare diseases meets the difficulties, which may obstruct this strategy. Even though there are critical issues, this approach presents a progressive paradigm for a regulatory strategy in the pharmaceutical industry.
This thesis focuses on interesting regulatory approaches that have been applied successfully in order to repurpose medicines for the treatment of rare diseases in the EU in the period from 2004 to 2014.
The first part of the thesis deals with complexity of issues regarding rare diseases, the EU legislation in the field of orphan medicinal products, general considerations with respect to drug repurposing as well as critical aspects concerning intellectual and commercial property rights.
The second part provides cases of the repurposed medicines. A background of this strategy, a brief outlook concerning etiology and pathogenesis of corresponding diseases, a mode of action of the substances in question, regulatory actions as well as challenges connected with this approach are discussed.
Pages: 56, Annexes: 1, Pages: 5