German early benefit assessments of orphan drugs under full dossier conditions

Market access in Germany provides several advantages for orphan drugs in the course of the early benefit assessment, since the additional benefit is already granted by law on the basis of the marketing authorization and its substantiating studies. However, this no longer applies if the drug's turnovers has exceeded 50 million Euro over the past 12 months leading to additional requirements along the benefit assessment (full dossier requirements). In this case, the additional benefit is not legally assured and must be demonstrated in comparison to the corresponding appropriate comparative therapy and taking a higher level of evidence into account.
The objective is to analyze the already completed procedures regarding their underlying evidence and the additional benefit granted by IQWiG and G-BA as well as the impact on the subsequent price negotiations.