Leki sieroce jako pomoc w wyobcowaniu spowodowanym chorobami rzadkimi
Orphan drugs as an aid in alienation caused by rare diseases

Summary/Abstract: Medicines needed to treat rare diseases are referred to as orphan drugs. A rare disease is defined as a condition that affects 5 in 10,000 people. Most often, the cause of rare diseases is genetic. It is estimated that the cost of producing and introducing one drug for treatment is about $1 billion. The income of pharmaceutical companies depends on the sale of the drug, which is why it is „not profitable” for the companies to produce drugs used to treat rare diseases, which affect very few people around the world. Orphan drugs that are available for treatment are very expensive. The annual cost of therapy for one child may even exceed PLN 1 million. Diagnosis of rare diseases is longterm, on average it takes up to 5 years, there were cases that it lasted even 30 years. The suffering of a person suffering from a rare disease is often associated with a sense of loneliness, helplessness, exclusion from society. The lack of orphan drugs or access to them, which is difficult for financial reasons, has very far-reaching consequences. It affects the structure of the family, its finances and functioning in society. Help in alienation caused by a rare disease seems to be simple, access to orphan drugs for children with rare diseases. This aid, which is limited only by financial considerations, would prevent suffering for many children and families.

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