Regulatory support for development of orphan medicines: virtual workshop from the European Medicines Agency

A live online event from the European Medicines Agency on 30 November combined short talks with Q & A sessions and was intended to encourage early and efficient interactions with  regulators by highlighting pre-marketing support in medicine development for treating rare diseases.

The meeting aimed in particular to explain existing tools relevant to early product development strategy. 

These include:​

• orphan designation;
• protocol assistance;
• the PRIority MEdicines (PRIME) scheme.

Stakeholders were given an opportunity to express their views in relation to early medicine development and regulatory interactions. ​

The workshop was targeted at small to medium enterprise medicine developers, academia, patients, healthcare professionals and European Reference Networks (ERNs), often at the forefront of medicine development to treat rare and neglected diseases.

High level points include:

There are estimated to be 6-8000 orphan conditions of which around half concern diseases with an incidence of less than 1 in 10000.

183 orphan medical products have been registered in the EU since 2000. ~120 of these have active orphan status for treating ~130 orphan diseases ie several have indications for treating more than one disease.

Around 2 in 3 orphan products are based on conventional chemical treatments and 1 in 3 involve advanced therapeutic approaches.

The EMA supports SMEs and Academia in orphan drug development through a dedicated contact point, regulatory, administrative and procedural support and engaging with EU bodies and industry stakeholders.

Of the ~2000 SMEs registered with the EMA, 23% are working on orphan medical products.

A video recording will be available after the event on the EMA website.