Presentation (CTI)

OBJECTIVES: During early benefit assessment in Germany, orphan drugs (ODs) are initially granted an added benefit by law while no appropriate comparator therapy (ACT) is defined. However, once annual revenue exceeds €30 million, the marketing authorization holder must prove an added benefit versus an ACT defined by the Federal Joint Committee (G-BA) during a reassessment. This threshold was reduced from €50 million to €30 million on December 1, 2022. This study aimed to evaluate the impact of the reduced revenue threshold on time to reassessment, added benefit outcomes, and subsequent discounts.
METHODS: Reassessments of ODs completed up to May 22, 2026, were extracted from the Pharm-Analytics database. The observation period was set at 36 months per revenue threshold, based on the start date of the respective G-BA reassessment. For each reassessment, the highest level of added benefit per procedure was considered for further analysis.
RESULTS: A total of 41 ODs underwent reassessment after exceeding the revenue threshold. Since the reduction of the revenue threshold from €50 million to €30 million, the proportion of reassessments has doubled, rising from 11 % to 22 %. Concurrently, the average time to reassessment decreased from 46.5 months to 39.1 months. In contrast, the proportion of ODs losing their added benefit during reassessment remained at high levels (79 % vs. 74 %). This resulted in substantial price discounts of 26.9 % and 23.5 % for the €50 million and €30 million thresholds, respectively, compared with 7.9 % and 3.5 % for ODs maintaining their added benefit.
CONCLUSIONS: Reducing the revenue threshold has led to earlier and more frequent reassessments. Many ODs lose their added benefit during reassessment due to a lack of evidence demonstrating superiority over the ACT defined by the G-BA. This is associated with substantial discounts. Successful reassessment requires solid evidence and a well-considered pricing strategy.