Benefit assessment orphan drug

Client Situation

Our client was in the process of obtaining the approval for an oncological drug for which the orphan designation could be considered certain. The client commissioned us to prepare the benefit dossier including accompanying strategic advice.

Our Solution

We prepared the complete benefit dossier for our client. The project started when the application for approval was submitted and took just over a year to complete. We conducted weekly teleconferences and several workshops to coordinate with the client. In this project, the analyses were carried out by our client’s global biometrics team. We drew up the statistical analysis plan and prepared the results according to the dossier requirements. We identified the communication of unmet medical needs as a key strategic component, and accordingly we presented this prominently in the dossier. As the indication was restricted in the course of the approval procedure, we changed the contents of the dossier at short notice, in particular the section on the epidemiology in module 3. Subsequently, we also supported our client in the preparation of the written statement and the hearing.

Client Benefit

We prepared a dossier with which our client was highly satisfied; especially the quality of the medical writing was praised. The minor additional benefit granted by the G-BA as a very good result considering the available data.