Effect of EU Regulations on Innovation and Marketing Authorisation of Orphan Drugs for Children

R. Charitha*, J. Praveen Kumar¹, E. Vijay Kumar^2
*Krishna Teja Pharmacy College, Tirupati, A.P., India
¹Associate Professor, Krishna Teja Pharmacy College, Tirupati, A.P., India
²Assistant Professor, Srinivasa Institute of Pharmaceutical Sciences, Proddatur, A.P., India

A B S T R A C T
No single definition for orphan diseases exists worldwide, but it is generally a disease that affects a small portion of the world population. Despite the development of science and pharmaceutical technology, the number of rare diseases for which no treatment is available is estimated between 4.000 and 5.000 worldwide.  The analysis of the finances that are required for research, development and manufacturing of orphan drugs suggest that these drugs are perhaps the most expensive drugs produced by the pharmaceutical industry. The main objective of orphan regulations is to encourage pharmaceutical companies to begin the process of researching and developing of new drugs intended for treatment of orphan diseases. The first legal framework for defining the rules for the marketing authorization of the orphan drugs was presented by the Food and Drug Administration (FDA) in the 1983. In European Union, the regulation for orphan drugs was introduced by the European Medicines Agency (EMA) in1999. The FDA orphan drugs regulation is based on three laws, while the EU legislation is covered by six regulations and two additional guidelines. The detailed overview of the FDA and EMA orphan drugs regulatory requirements showed that both regulatory authorities provide shortened registration procedure, allow exception from payment of certain fees, provide protocol assistance and stimulate processes of a parallel application for orphan designation. The differences could be seen in the period of market exclusivity, tax incentives and source of the grants.
Keywords: Incentives, orphan (rare) diseases, orphan drugs, orphan drugs regulation