03 August, 2018

Did you know the EMA has simplified the orphan drug designation application process?

By Andrea Sisneros

Find me on:

Every month, Envigo’s pharmaceutical regulatory team keeps track of the key changes, influences and opinions that impact the drug development process around the globe.  This month, the team discuss recent changes to the way in which the European Medicines Agency (EMA) has simplified the submission of applications for orphan drug designation.


Orphan-drugs-featured-imageOrphan drug designation changes at the EMA

The regulatory landscape in Europe is complicated by the specific needs of the 28 member states, so anything that seeks to harmonize and simplify drug development in the region is welcomed.  On June 19th this year, the EMA launched a new, secure portal for pre- and post- orphan designation activities for sponsors. 

Orphan drugs, i.e. drugs developed specifically to treat rare medical conditions, have become increasingly popular to developers. Named ‘Iris’, the EMA portal has been designed to save time for the preparation and submission of applications.

Iris has been designed to permit sponsors to check the status of applications in real-time, and receive automatic notifications when progress status changes. Iris replaces the current submission process (Eudralink), which will be phased out completely by 19th September this year.  Consequently, sponsors are being encouraged to adopt the new system now.

Iris also eliminates the need for sponsors to notify the EMA of their intent to file an application for an orphan drug.  Instead, the EMA simply asks that sponsors submit orphan designation applications a few days ahead of the published submission deadlines.

Learn more about the new orphan drug designation process. 

Simplified does not mean simple

There are a lot of reasons why orphan drug indications are an attractive proposition, for healthcare providers and drug developers alike.  For small or medium-sized enterprises, these include reduced fees for protocol assistance and marketing-authorization applications, an opportunity for inspections before authorization, applications for changes to marketing authorizations made after approval, reduced annual fees and 10 years market exclusivity.

Not surprisingly, the number of orphan applications to the EMA has steadily increased in recent years, rising from 548 between the years 2000 and 2005, to 1151 between the years 2011 and 2015.

However, of those applications granted an orphan drug designation, a high proportion are failing during development: authors of a recent report in the BMJ analyzing designations between the year 2000 and 2017 found 28% had failed during development. While a good proportion of these (84%) were abandoned during the development process, 15% failed at marketing authorization because of efficacy/safety issues, insufficient data, quality issues, regulatory issues on trials, and commercial reasons.

The attrition rates reported here underline the need to for an inter-disciplinary development team, experienced not only at producing quality safety data, but also at contextualizing study results and devising follow-on studies to satisfy regulator demands.

A special note for non-EU orphan drug developers

There is a temptation for non-EU based drug developers to assume that filing with the FDA makes applications easily transferable to the EMA.  This is not always true, and with that in mind, we recommend designing regulatory strategies to sit along side your drug development plans, ensuring your drug gets the best chance of being one of the few that make it through to treating those patients due to the rarity of their disease may not have adequate treatment specially for them.

If you're in the process of planning and conducting preclinical studies to move into the next development stage, read our article 'How to plan and conquer the drug development process in the early stages'.

Click me



Category // Pharmaceutical development, Regulatory affairs, European Medicines Agency, Food and drug administration