Within the European Joint Programme for Rare diseases, EATRIS and the EMA's Regulatory Science and Innovation Task Force and the Orphan Office, have joined forces to develop this tutorial for academic medicines developers who would like to apply for an Orphan Drug Designation (ODD). In this video tutorial, we will highlight the benefits of an ODD, walk you through the different steps of the process, and provide you with information to evaluate your readiness to apply. We will also explain how to access IRIS, the Regulatory & Scientific Information Management Platform, which is the online secure portal to submit applications to EMA. We will guide you through the application form, how to submit it in IRIS and how the evaluation process of an orphan designation works. We will also explain what is protocol assistance and what questions can be discussed. And finally, we will address how to manage post-designation activities and where to find useful information to build your drug development strategy.
Timecode:
00:00 - Introduction
01:11 - What are the benefits of an orphan designation
02:40 - Are you and your product ready for an ODD?
04:06 - How can you access IRIS?
08:18 - How to write the scientific part of your application?
17:57 - How does the evaluation process of an Orphan Designation work?
19:42 - How can EMA support academic sponsors during Protocol Assistance?
21:49- How to discuss the medicine development for paediatric patients?
25:27 - Closing remarks
This video has been developed through funding from the EJP-RD project. The European Joint Programme on Rare Diseases is an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N°825575