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What are orphan drugs?

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They orphan drugs are those used in the diagnosis, prevention or treatment of rare diseases or little common, those with a very low prevalence, affecting not more than five people per 10,000 inhabitants. Their special characteristics make it necessary that health authorities secured public funds to encourage research and development of these drugs.
According to the World Health Organization (who) estimates, there are about five thousand diseases considered rare - 80% genetic - and it is estimated that 7% of the world population could be any of these diseases, which in the majority of cases take a long time to be diagnosed, so it is not easy to determine its real incidence, although in Europe could be about 30 million affected.
People suffering from these diseases so frequent have the same rights to a safe and effective treatment to patients with common diseases; However, develop therapies for them is a great challenge that private companies cannot afford without help.
The research that is required to develop a drug and check its safety and effectiveness before distribute it - that can last an average of eight years - has very high costs and for the drugs orphans not could pay for itself with its marketing, to ir led each of them to a very small segment of the population. The number of people that you may check the effectiveness of the drug also is very low, and clinical trials should be done in very distant centers together.

Research and development of orphan drugs


Research and development of orphan drugs only is possible with the intervention of the public health system , aid to private enterprise. In countries such as the United States three decades ago -Orphan Drug Act- measures were established with the aim of promoting the research of targeted therapies to treat rare diseases.
In the European Union also came into effect in the year 2000, legislation which lays down some basic measures to encourage research, development and marketing of these drugs, as the commercial exclusivity in the European Community during the ten years following the granting of permission marketing - implying that during that period similar products - you can sell, various tax breaks and grants to researchers.
To access these and other privileges, the drug must first be designated as an orphan and, to do so, it must be ordered - with appropriate scientific justification on their use - to the Committee for medicinal products orphans (COMP) of the European Medicines Agency (EMA), an organization that also provides scientific advice to companies interested in developing a specific drug.
The fact that a product is designated as orphan does not mean it will subsequently be approved its use, since it must demonstrate that it meets the criteria of safety, efficacy and quality that are required so that your marketing is authorized. You can use some drugs whose safety and effectiveness have already been verified, however, although remain in pilot phase, thanks to emergency procedures that approve its use temporarily.
Article contributed for educational purposes
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