Workshop on regulatory support for development of orphan medicines

On Monday, 30 November, EMA is hosting a workshop to discuss the benefits and impact of early regulatory interactions and incentives for the development of medicines for rare diseases.

The purpose of the workshop is to encourage early dialogue between developers and regulators in order to facilitate the development of innovative medicines in neglected disease areas. The workshop is targeted at small and medium-sized enterprises (SMEs), academia, patients, healthcare professionals and European Reference Networks (ERNs) who are often at the forefront of medicine development in rare and neglected diseases.

There are over 6,000 rare diseases that affect about 30 million people in the European Union (EU). Treating patients with rare diseases is often difficult because there are often none or only few treatment options available. This represents a huge unmet medical need and a significant public health challenge.

The 


PDF iconagenda for the workshop

, which will be held virtually due to the ongoing pandemic, is available. The workshop can be followed via live broadcast on the event page.

Workshop participants will be asked to provide their feedback and views using an interactive polling platform. Participation in this workshop is subject to prior application. To submit an expression of interest, please complete the online form by 30 November at 08:00 CET.

EMA offers incentives to encourage companies to research and develop medicines for rare diseases that otherwise would not be developed. Through orphan designation, protocol assistance and the PRIority MEdicines (PRIME) scheme, EMA provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients’ unmet medical needs. Early interaction with regulators also helps medicine developers understand regulatory requirements and generate the robust data needed to draw conclusions on the medicines’ benefits and risks.

The outcomes of the workshop and presentations will be published on the event page after the meeting.