PRO94 Outcomes of Benefit Reassessments of Orphan Drugs in Germany after Exceedance of the 50-Million-EURO Revenue Threshold

Abstract

For orphan drugs in Germany, an added benefit is granted by law within the early benefit assessment. However, this privilege no longer applies if the drug’s turnovers exceed 50 million euro within 12 months, leading to a benefit reassessment based on a comparison against an appropriate comparative therapy. The objective of the current study is to evaluate the development of an orphan drug’s added benefit as well as the negotiated price rebate after reassessment. Outcomes of all early benefit assessments of orphan drugs that were reassessed by April 2020 due to the exceedance of the 50-million-euro revenue threshold were analyzed. The number and size of subpopulations and the respective granted added benefit as well as the resulting negotiated rebate were determined across assessments. In 6 out of 9 cases, an orphan drug was granted an added benefit for at least one subpopulation after reassessment. However, the mean proportion of patients with an added benefit among the total population shrunk from 100% to 43%. In the first assessment, the added benefit had been considered non-quantifiable for an average of 56% of the total patient population. This proportion amounted to only 3% in the reassessment, while the added benefit of 40% of the total population was quantified as considerable. Nevertheless, the rebate after reassessment maintained identical for only one drug. In all other cases the rebate increased by additional 3% to 14%. The reassessment of an orphan drug due to exceedance of the 50-million-euro threshold in Germany is associated with a high risk of a reduction of the proportion of patients with a granted added benefit resulting in an increased rebate on the drug’s price. Therefore, a careful evaluation of potential risks and benefits of exceeding the 50-million-euro threshold is crucial to prevent harmful outcomes of the reassessment.