Working together on both sides of the Atlantic
To accelerate the pace of orphan drug development
In 2006, Eurordis initiated a comparative analysis of EU and US designated and marketed orphan drugs. 
The US have had an early start with the 1983 Orphan Drug Act, compared with the 1999 EU Regulation on Orphan Medicinal Products. They therefore offer sufficient experience and data to conduct an in-depth analysis of the dynamics of orphan drug development that can be applied to their European counterpart. To this end, Eurordis approached the OOPD (Office of Orphan Products Development) at the FDA (US Food and Drug Administration). The work was performed by Fabrizia Bignami (Therapeutic Development Officer at Eurordis), François Faurisson (Clinical Research Advisor at Eurordis), Tan Nguyen (FDA), Spiros Vamvakas (EMEA1) and two students from ESSEC business school (France). This partnership between Eurordis, the FDA and the EMEA was unprecedented and paved the way forward for more cooperation. 'Europe started fostering orphan drug development after the US,' says Fabrizia Bignami, dbut we wanted to make sure that we were not late in terms of therapeutic development. We decided it was time to analyse how the European orphan drug system has performed since its inception in 1999.' 'We also wanted to analyse the differences in outcomes in the designation procedures between the EU and US systems,' adds François Faurisson. 'We had in mind that some synergies might even be possible.'
The data analysed in the study include, among others, EU-US comparisons of:
- Rate of orphan drug designations granted and not granted (by type of product, by clinical field, etc.)
- The reasons for granting or not granting designation requests
- Rate of marketing authorisations granted and not granted
- Time from designation to market authorisation
- Countries of origin of designated- and market authorised- orphan drug sponsors and sponsor holdings
The results of the study were presented during the 4th workshop of the Eurordis Round Table of Companies (ERTC) entitled "Common drugs for common needs: the EU vs US approach to orphan medicinal product development", which took place in Barcelona in June 2006. 45 people attended (a third from the US), including some members of the COMP2 (EMEA), industry representatives and regulators such as Marlene Haffner and Tan Nguyen from the FDA, and Agnès Saint Raymond from the EMEA. 'It was such a positive, co-operative and constructive meeting!' said Agnès Saint Raymond at the time. The outcomes of this workshop are very important for the rare disease community on both sides of the Atlantic, with an agreement on the necessity for a global - or at least transatlantic - approach to orphan drug development. A few months later, this harmonisation process between the COMP/EMEA and the OOPD/FDA is formally on the agenda of the COMP.
What is proposed is to:
- Harmonise the format and content of orphan drug applications at the EMEA and FDA
- Speed up parallel designation, while keeping respective specificities of the EU and US regulations
- Seek converging requirements for Protocol Assistance, Marketing Authorisation and post- marketing studies by the FDA and the EMEA
The results of the comparative study and the ensuing proposals were presented at the EPPOSI meeting in Madrid in October 2006 and at the COMP November 2006 meeting. 'I am confident that the EU-US convergence in orphan drug designation, with a unique, harmonised application format and parallel procedure, can be achieved by 2008,' says Yann Le Cam, CEO of Eurordis. 'Such a move is to be celebrated. There is goodwill from all parties involved. If successful, parallel designation is a lever for more EU / US converging regulatory requirements, more global development, higher success rate, and earlier access to innovative orphan drugs for rare disease patients, both in Europe and the US.'
1 European Medicines Agency
2 Committee for Orphan Medicinal Products
This article was previously published in the January 2007 issue of our newsletter.
Author: Jerome Parisse-Brassens
Photo credits: medicine © Anita Patterson Peppers; other photos © Eurordis
