Legal Regulation of Orphan Drugs

The existence of orphan (rare) diseases is a serious medical, social and economic problem. This is because the rarity of these diseases naturally leads to low levels of demand both in the sales of medicines and in scientific research, as a result of which patients often do not receive the necessary treatment, which leads to disability or death in the case of certain diseases. Unfortunately, the current legal regime for orphan drugs does not take into account all the features of this problem. The study analyzed the norms of Russian and foreign (US and EU) legislation, identified problems relevant to the Russian legal regime of orphan medicines, and identified possible ways to solve them. In the first Chapter, "Establishing a special legal regime for orphan drugs as a way to solve the problem of orphan diseases", an analysis of the concept and phenomenon of orphan diseases, General legislative provisions in Russia and abroad in relation to orphan diseases and medicines, highlighting problematic aspects of Russian legislation at this level, and finally, a review of the history of the development of orphan legislation in Russia and abroad was conducted. The second Chapter "providing patients with orphan medicines" contains a detailed review of the current Russian legal regime of orphan medicines, which for the most part consists of a set of rules for providing these drugs to patients. In particular, the analysis of regulatory regulation in this area, including the specifics of practical implementation of existing standards, identification of existing problems and suggestions of ways to solve them, was carried out. In the third Chapter, measures that can be taken to improve the orphan legislation in the Russian Federation were formulated. At the beginning of the Chapter, the importance of encouraging pharmaceutical companies to improve patient well-being was explained.