OBJECTIVES: Orphan drugs (ODs) have privileges in the early benefit assessment (EBA) and the subsequent price negotiation in Germany. The added benefit (AB) is acknowledged by law, no appropriate comparator therapy (ACT) is defined. If the sales of the OD exceed a sales volume threshold of 30m€ (50m€ until Nov 2022), the product must undergo a regular EBA. The objective of this study was to evaluate which factors influence the change in annual therapy costs (ATC) for the SHI between a drug’s EBA as an OD and its subsequent regular EBA procedure.

METHODS: The results of all EBAs due to exceeding the sales threshold with completed price negotiations by March 2023 were analyzed. The analysis of the change in ATC was carried out at active ingredient level. For this purpose the ATC of the subpopulations were weighted by the size of the subpopulations according to the G-BA resolution. A linear regression was used to analyze the change in ATC using change of the AB (cAB), patient numbers, availability of an active ACT (not only BSC) and quantifiable AB in OD procedure as independent variables.

RESULTS: A total of 17 drugs were analyzed. In 3 of 17 cases an AB for the whole population of the OD was granted after reassessment. In 7 cases there was an AB for at least a subpopulation and in 7 cases there was no AB granted at all. The ATC after exceedance changed between -43% and +14%. Linear regression analysis (adjusted R²=19%) showed that cAB had a significant influence on ATC (p=0,03) whereas the other variables had not (p>0,05).

CONCLUSIONS: The significant impact of cAB on ATC is in line with the objective of AMNOG that the reimbursement amount of drugs should depend on their AB.