Benefit assessment orphan drug

Client Situation

The client was in the midst of securing approval for an oncological drug, expected to receive orphan designation. co.value was hired to prepare the AMNOG-specific benefit dossier and provide strategic advice.

Our Solution

During the approval application, co.value completed the entire benefit dossier for the client. In this project, the analyses were carried out by our client’s global biometrics team. co.value created the statistical analysis plan and tailored the results to meet dossier requirements. We identified the communication of unmet medical needs as a key strategic component, and accordingly we presented this prominently in the dossier.  Adjustments were made to the dossier content, notably in the epidemiology section of module 3, due to a change in the indication during the approval process. co.value also assisted in preparing the written statement and the hearing.

Client Benefit

The client was highly satisfied with the dossier, particularly praising the quality of medical writing.  The G-BA granted a minor additional benefit, which was considered a very good outcome based on the available data.