Rare Diseases – Information and Diagnostics

Rare diseases are those diseases that occur in less than 5 in 10,000 members of a given population. These are diseases that usually have a chronic and severe course, most often genetically determined, in half of the cases manifesting themselves in childhood. On the other hand, the number of rare diseases described so far is large - it is about 6 thousand, which means that a total of about 6-8% of the population suffers from them. In Poland, this is about 2.3-3 million people. This is a serious and so far underestimated health problem.

Accurate diagnosis of these diseases is not always possible, because most doctors have not had the chance to meet them before due to their rare occurrence. Therefore, the path to establishing a diagnosis for many patients is long and these diseases are diagnosed at a very advanced stage. As diagnostic methods are improved, and above all, modern therapies are available, knowledge about rare diseases is constantly expanding, and their awareness among doctors and society is also growing.

6-8%

suffering from a rare disease

50%

concerns children

7 000

rare diseases are known

only 10%

rare diseases have treatment

~7 years

takes an average diagnostic process from the first
symptoms

only 1 in 8

patients with
rare diseases are diagnosed
and treated appropriately

Drugs used in the treatment of these diseases, according to the terminology and registration procedure in the EU, are classified as orphan drugs (orphan medical products) due to the fact that their use concerns a very small group of patients. They are usually very expensive.

For the medical community, rare diseases are a significant challenge for two reasons: their severity and chronic nature, and expensive diagnostics and therapy. They often affect children, in many cases leading to permanent physical and mental impairment and even death. Patients and their families have difficulty obtaining consent to finance the therapy, and for economic reasons, access to expensive drugs is limited.

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If you suspect you have a rare disease and need help diagnosing it, we’re here to help. Learn how to access our support and start your journey to answers.

Have you been experiencing disturbing, and still unexplained, symptoms for over half a year?

Do you or your doctor suspect a rare disease?

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