Orphan drugs in Germany

Durch das Gesetz für mehr Sicherheit in der Arzneimittelversorgung (GSAV), welches am 28. Juni 2019 vom Bundesrat verabschiedet wurde, überarbeiten wir momentan dieses White Papers. Die Version, die sie hier herunterladen können, enthält den Stand von 2018 und ist teilweise nicht mehr aktuell. Bitte kontaktieren Sie uns bei weiteren Fragen direkt.

The AMNOG, the legislative framework governing the market access of innovative prescription drugs in Germany, has implicated a paradigm shift in the examination of the value of a pharmaceutical and in the setting of an appropriate reimbursement level for the product. Based on the evidence of available study data, the additional benefit over the existing standard of care is assessed by the most powerful German health authority, the G-BA. Depending on the classification of the benefit category, the pharmaceutical manufacturer negotiates the final refunded price with the umbrella organization of the German statutory health insurances. 

For orphan drugs, there are specific challenges that the manufacturer has to meet. Due to the randomness of the disease, subpopulations are small and study data are scarce. This leads to a considerable uncertainty about the perceived value of the drug and, thus, to intense and sometimes tough negotiations with high rebates in the end. 

Pharmaceutical companies planning to introduce new orphan drugs into the German market should prepare well in advance a sound and stringent strategy to optimize the performance throughout the entire market access processes. This white paper elaborates on all relevant issues and describes important recommendations for manufacturers facing these challenges.