Orphan medicinal products

Orphan medicinal products are intended for the analysis, prevention or treatment of dangerous or very serious conditions that affect no more than 5 in 10,000 people in the European Union. Patients suffering from rare diseases deserve the same quality of treatment as other patients within the European Union. Given the small numbers of patients affected by rare diseases, the pharmaceutical industry has been unwilling in the past to invest in the research and development of medicinal products to treat them. The EU introduced new legislation in 2000 with the aim of providing incentives for the development of medicines for rare diseases (so-called orphan medicinal products).

  • Orphan designation
  • Medicines for rare diseases

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