Orphan Drugs Continue to Proliferate in Europe

Feb 06, 2014

No less than 11 out of 81 medicines recommended for marketing authorization in 2013 by the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) were intended for the treatment of rare diseases. The comparable figures were 8 in 2012 and 4 in 2011, according to an EMA press release issued on 3 February.

Developers of orphan medicines are now making much better use of the support tools offered by the Agency and the European Orphan Regulation, noted Bruno Sepodes, Chair of the Committee for Orphan Medicinal Products (COMP), the EMA body responsible for recommending orphan designation of medicines for rare diseases.

“12 years after the European Orphan Regulation came into force, it is undoubtedly serving its purpose, with more and more orphan medicines reaching patients with rare diseases each year," he explained. "The joint efforts of the EMA committees, including the COMP, the Pediatric Committee and the CHMP, in providing scientific advice to companies during the development of their medicines, have been a driving force behind this success."
 
The volume of applications for orphan designation submitted to the Agency has increased by more than 22% since 2009, and the number of companies seeking protocol assistance increased from 82 requests in 2012 to 108 requests in 2013. Micro, small or medium-sized enterprises (SME) are particularly active in this area, accounting for around 40% of the applications for an orphan designation.
 
“In 2014, we will continue to review and adapt our internal procedures. This will help us to better meet the needs of patients, with whom the Committee has been working in close contact since its creation, and to keep up-to-date with the scientific knowledge on rare diseases,” Sepodes said. “Another priority will be to continue to actively contribute to scientific publications and scientific debate in our area.”