Celebrating 10 Years of the Orphan Drug Regulation in Europe

The European Orphan Drug Regulation celebrates its 10th Anniversary this year ? an opportunity to reflect on the experiences gained and the challenges ahead.

¡°Overall the Regulation can be considered a success. It has led to medicinal products coming to market for rare conditions previously untreatable, transforming the lives of the sick and dependent, as well as improving quality of life and survival¡±, says Lesley Greene, a former EURORDIS President and current member of the Committee for Orphan Medicinal Products (COMP).

It has contributed to boosting research, development and marketing of orphan medicinal products in Europe. Since its adoption, more than 1,000 applications for orphan drug status have been reviewed and the number of new applications is rising every year. In 10 years, 728 have been designated as ¡®orphan drugs¡¯ and 60 have received marketing authorisation. Of this, around 2.5-2.6 million patients potentially stand to benefit. The consolidated trend indicates that an average of 10 new orphan medicines will be approved for marketing every year in EU over the next 5 to 10 years.