OBJECTIVES: Survey of the benefit assessment of orphan drugs in Germany and analysis, whether adjustments are necessary for the upcoming AMNOG reform. Comparison with France and England should help to identify current differences and to determine whether certain processes could be adapted for Germany.

METHODS: Results of the benefit assessments for Zolgensma^® were analyzed and compared to the beforementioned countries. National health technology/reimbursement agency websites were assessed. Zolgensma^® was chosen because it is a high-priced single-use gene therapy, has generated much discussion, and was the first orphan drug in Germany to be mandated by the Federal Joint Committee (G-BA) to collect data accompanying its use. Expert interviews with different Stakeholders (Industry, Payer, HTA Agencies) were conducted.

RESULTS: Analysis of Zolgensma^®: Availability: Germany February 2020, France May 2020, England March 2021. Benefit assessment: Germany: no additional benefit; France: for SMA type I, II; ASMR 3, (moderate clinical added benefit); England: NICE recommendation: Only for a specific proportion of the patient population. Expert interviews: Germany: First implementation of a data collection accompanying the application of Zolgensma^®. Experts agree that there must be changes in the reform. A deficit of approximately EUR 50 billion in the German SHI system requires adjustments around orphan drugs. Specifically, reduce EUR 50 million sales threshold to 20 million, reduce free pricing from 12 to 6 months. France: pioneer in terms of creating reference centers (for (ultra-)rare diseases); capability to identify and treat these diseases relatively early on. England: introduction of a new fund - Innovative Medicines Fund (IMF) - to finance innovative (non-cancer) medicines; volume: £340 million.

CONCLUSIONS: Adjustments from other countries are difficult to implement. Structural and normative differences lead to different additional benefit assessments. Adjustments of AMNOG for orphan drugs are necessary and planned (referendum draft of the Ministry of Health).