Support for Orphan Drug Designation (ODD) in Europe
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.
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.