Business Case detail

Scientific Advice - Orphan Designated Drug

Project: Scientific Advice - Orphan Designated Drug


The client was a US-based Biopharmaceutical company that had recently received FDA approval. Their aim was to enter Europe and then Japan in waves.


The product was considered to be less likely of acceptance in Europe due to its composition. The client believed that there was not sufficient relevant detail in the European Union guidelines to be safe in making an application. Moreover, they had concerns about the time and cost and were unaware of all the EU framework as they had no base in Europe.

Solutions we gave:

We began by performing an analysis of the technical parts of the dossier to see whether the current requirements in Europe could be sufficiently considered “acceptable” to go forward and submit an application to the EMA under a Centralized Procedure. We believed, upon reflection, that there were sufficient arguments to justify this application, but we wanted to ensure that any risk was minimized by requesting Protocol Assistance with EMA.  

We worked closely with the client to frame the correct questions for the meeting.  We prepared the client for the meeting and attended on behalf of the client.


The client received positive feedback and, with the support of our regulatory team, we managed to update the dossier so that it was suitable for submission, based upon the responses that we had received.  We managed the procedure using our PV system PSMF, translating all documents within the designated 5 days being a liaison with Europe.  Finally, we managed the global and local pharmacovigilance for the product after approval.