2.1. Figures of rarity
“A rare disease is a disease that occurs infrequently or rarely in the general population”. In order to be considered as rare, each specific disease cannot affect more than a limited number of people out of the whole population, defined in Europe as less than 1 in 2,000 citizens (EC Regulation on Orphan Medicinal Products). This figure can also be expressed as 500 rare disease patients out of 1 million citizens. While 1 out of 2,000 seems very few, in a total population of 459 million citizens this could mean as many as 230,000 individuals for each rare disease. It is important to underline that the number of rare disease patients varies considerably from disease to disease, and that most people represented by the statistics in this field suffer from even rarer diseases, affecting only one in 100,000 people or less. Most rare diseases do only affect some thousands, hundreds or even a couple of dozens patients. These “very rare diseases” make patients and their families particularly isolated and vulnerable. It is worth noting that most cancers, including all cancers affecting children, are rare diseases.
Despite the rarity of each rare disease, it is always surprising for the public to discover that according to a well-accepted estimation, “about 30 million people have a rare disease in the 25 EU countries”3, which means that 6% to 8% of the total EU population are rare disease patients. This figure is equivalent to the combined populations of the Netherlands, Belgium and Luxembourg. Quoting from the Background Paper on Orphan Diseases for the “WHO Report on.
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