Outcomes of Benefit Reassessments of Orphan Drugs in Germany After Exceedance of the Lowered Sales Threshold of 30 Million Euros

OBJECTIVES: In German HTA, an added benefit is granted by law for orphan drugs until the drug’s turnover exceeds a legally defined threshold within 12 months, leading to a reassessment against an appropriate comparator therapy (ACT). As part of the SHI-Financial Stabilization Act, this threshold was lowered from 50 million to 30 million euros. The objective of this study is to analyze the effects of this reduction on the benefit assessment outcomes and the price level of reassessed orphan drugs.

METHODS: Orphan drugs that have exceeded the 50-million-euro threshold were compared with those that have exceeded the 30-million-euro threshold by June 2024. The number of orphan drugs, the time until the relevant threshold was exceeded, the granted added benefit and the resulting price level compared to the ACT were examined.

RESULTS: Out of 33 orphan drugs, 19 exceeded the 50-million-euro threshold and 14 the 30-million-euro threshold. The added benefit was on average better after the exceedance of the 50-million-euro compared to the 30-million-euro threshold: 47% vs. only 9% of orphan drugs with an at least considerable added benefit and 42% vs. 63% with at highest no added benefit. After exceeding the 50-million-euro threshold, the average negotiated annual therapy costs of orphan drugs with an ACT without patent protection exceeded the costs of the ACT many times over while the average price level was significantly below the average costs of patent-protected ACTs. The same could be expected for prices after exceeding the 30-million-euro threshold.

CONCLUSIONS: The risk of downgrading the added benefit category due to a reassessment has appeared to be higher since the lowering of the sales threshold. Strict application of the guardrails according to the SHI-Financial Stabilization Act could lead to a drastic price drop after reassessment particularly for orphan drugs with a patent-protected ACT.