Rare Diseases - Orphan Drugs

Rare diseases pose a major challenge to healthcare systems due to the difficulties in diagnosing and treating them. Rare diseases are usually defined as those that affect fewer than 5 per 10 000 people in a given population. Their diagnosis is difficult and can take years, and rare diseases are often severe, chronic and life-threatening. It is estimated that between 2 and 3 million people suffer from rare diseases in Poland. The small number of patients suffering from these conditions compared to the number of patients suffering from more common diseases makes drug development costly and unprofitable for the pharmaceutical industry. High production costs and a limited market effectively discourage pharmaceutical companies from investing in the development of so-called orphan drugs. If a drug is already produced, it is often the only one available to treat a particular rare disease, and so is extremely expensive, which significantly limits availability to patients,