A disease or disorder is defined as rare in Europe when it affects less than 1 in 2,000 citizens. These drugs are called “orphan” because under normal market conditions the pharmaceutical industry has little interest in developing and marketing products intended for only a small number of patients. For drug companies, the extremely high cost of bringing a medicinal product to market would not be recovered by the expected sales of the product. As a result the potential market for new drug treatment is also small and the drug companies industry would actually incur a financial loss. Therefore governments and rare disease patient organisations such as EURORDIS advocate for economic incentives to encourage drug companies to develop and market medicines for rare disease treatment.
Orphan medicines legislation
Orphan medicines legislation provides incentives to pharmaceutical companies to develop and market medicinal products to treat rare diseases. A ground-breaking precedent was set by the United States in 1983 when the Orphan Drug Act came into effect, which was followed by Japan and Australia in the 1990s. In Europe, the European Parliament adopted the EU Regulation on Orphan Medicinal Products (Regulation EC n° 141/2000) on 16 December 1999. EURORDIS, representing the rare disease patient community in Europe, actively advocated for the adoption of this legislation. Following the adoption of the regulation, the Committee for Orphan Medicinal Products (COMP) was created within the European Medicines Agency (EMA) to review the applications for medicinal products seeking an orphan designation.
The Committee for Orphan Medicinal Products (COMP) was created as part of Regulation (EC) No 141/2000 (the Orphan Drug Regulation) in order to review applications for an orphan designation. Medicinal products intended for rare diseases can receive an orphan designation based on the following criteria:
- • The product is intended for an indication with a prevalence not exceeding 5 in 10 000 persons in the EU
- • The disease is life-threatening, seriously debilitating or a serious and chronic condition
- • No satisfactory method of diagnosis, prevention or treatment of the condition is already authorised in the EU. If any method exists, then the medicinal product has to demonstrate that it provides a significant benefit compared to the product for which orphan designation is being requested