The Orphan Medicines Office of the European Medicines Agency (EMA) is hosting an interactive webinar on Rare Disease Day on 29 February 2024 from 16:00 to 17:30, to answer questions that developers of products for rare diseases or researchers in this field might have on orphan designations and orphan medicines development. Agenda is available here.

The event is aimed to patients, healthcare professionals, academia and industry representatives.

Registration is open until 23 February 2024.

The webinar will feature:

  • short presentations on the background of orphan designation and the benefits it has brought to patients
  • a live question-and-answer session enabling participants to ask their questions on orphan medicines development to a panel of regulatory experts

Participants will have the opportunity to submit their questions in advance of the meeting. Questions should be sent to by 23 February 2024.

The event will be also broadcast and recorded. The videos and presentations will be made available on the EMA website after the event.

Read more.

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