ContentsThis study about orphan drugs policies aims to provide the European Parliament with background information for the establishment of a European Policy on orphan drugs.
According to the World Health Organization, there are around 5,000 rare diseases. They affect a small part of the population, yet they constitute a real public health issue since patients are suffering from the lack of treatment for their particular disease. Tropical diseases are also missing efficacious and safe treatments.
The necessary drugs for treating rare diseases are not available mainly because of profitability issues for the Pharmaceutical Industry. Yet, patients and their representative associations need to obtain better care and safe and efficacious treatments. Therefore, the availability of economic incentives is required to encourage pharmaceutical and biotechnology companies develop such orphan drugs. In alleviating the costs of developing orphan drugs, these incentives will have a positive impact on the development of biotechnology and small and medium pharmaceutical companies. In addition, mainstream pharmaceutical industry are also likely to extend their activities towards orphan drugs.
A comparative study of the existing orphan drugs policies in the USA, in Japan, in Singapore and in Australia, shows that policies based on market exclusivity, R&D incentives and protocol assistance seem to have a very positive effect on the emergence of appropriate treatments for rare diseases. For example, the Orphan Drug Act in the USA has allowed the designation of around 900 new substances as orphan drugs.
The European Commission’s regulation proposal sets a prevalence criterion for orphan drug designation just between US and Japanese criteria. The 10 years’ market exclusivity included in the text is probably the most important stimulus for pharmaceutical companies to develop orphan drugs. Protocol assistance and waiving of registration fees are also interesting for small and medium companies.
The extension of the orphan drug status to tropical disease treatments (in Europe only) will facilitate the emergence of new important medicines, even if it has a marginal impact on drug financing in the developing world.
Finally, Patients Associations support the implementation of the regulation which will have a positive impact on the emergence of a European orphan drugs supply.