Orphan drugs in Germany

The Law for more safety in the provision of medicinal products (Gesetz für mehr Sicherheit in der Arzneimittelversorgung, GSAV) passed the German Bundesrat on June 28th, 2019. Therefore, we are currently revising this white paper. You can download the 2018 white paper here, but it is partially not up to date. Please contact us for further questions.

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.