BlueReg (BR) was engaged to assist a start-up company with the preparation of their orphan drug designation application dossier in Europe and to provide regulatory support during the procedure. 

Challenges:

• Timelines: the client contacted BR a short time ahead of the planned submission date requesting a review and update of their draft ODD dossier. At this time, the client had been in contact with the EMA but had not followed up them for some time.
• Regulatory context: at the time the project started, a new EU guideline laying down new requirements for ODD, in particular to justify significant benefit over existing therapies that would directly impact this application, was issued.