Presentation (CTI)

OBJECTIVES: In the EU, there are several incentives for companies to develop pharmaceutical treatments for rare diseases. An orphan drug designation (ODD) can be granted when (1) the new treatment is intended for the treatment, prevention, or diagnosis of a disease that is life-threatening or chronically debilitating, (2) the prevalence of the condition in the community must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development, and (3) no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition. These conditions still need to apply at the time of marketing authorization. We investigated how many pharmaceuticals approved by the European Commission (EC) did or did not maintain their ODD at time of approval.
METHODS: Data of pharmaceuticals approved by the EC since 2015 were retrieved from the Union Register of Medicinal Products for Human Use on 05/05/2025. Data of ODD were retrieved from the European Medicines Agency (EMA) website on 14/05/2025 and verified on 02/06/2025.
RESULTS: Since 2015, 898 products were approved by EMA, of which 684 never had ODD, 3 received ODD after first approval and 18 ODD were withdrawn prior to first approval. ODD was confirmed at approval for 161 products, withdrawn by the company for 29, and not confirmed for 3.
CONCLUSIONS: Most (161/193; 83.4%) products with prior ODD kept their status with approval, only 16.6% (32/193) did not maintain ODD (most common reason: condition of significant benefit not proven/met). At the time of approval, there is still no satisfactory treatment for many rare diseases, or a significant benefit of the new medicine is proven.