OBJECTIVES: Orphan drugs are exempt from regular Health Technology Assessment (HTA) and benefit assessment procedures in Germany due to their 'non-quantifiable benefit,' giving manufacturers a favourable position in AMNOG price negotiations. However, this privilege is revoked when an orphan drug's annual turnover exceeds €30 million (previously €50 million), triggering a reassessment of its added benefit rating and subsequent price renegotiation without orphan drug privileges. This study investigates the time to reassessment for orphan drugs in Germany and the impact of this reassessment on their benefit assessment outcomes.

METHODS: Gemeinsamer Bundesausschuss (G-BA) benefit assessment decisions were searched, and a comprehensive analysis conducted regarding the time to reassessment for orphan drugs and the impact of this reassessment on their benefit assessment outcome. Trends in the ratings of reassessed orphan drugs were examined, and the potential implications of these trends were considered.

RESULTS: A total of 20 orphan drugs across 38 indications underwent reassessment by the G-BA after exceeding the annual turnover limit of €30 or €50 million between June 2019 to May 2024. Among these, 13 (65%) lost their added benefit rating, that is, they received either a ‘no additional benefit rating’ (13 [61.9%]), or a ‘lesser benefit rating’ (1 [4.4%]). Notably, 11 (55%) of these drugs had multiple indications, which likely contributed to the increase in revenue required to exceed the turnover limit. The average time to reassessment was approximately 49.9 months. Price changes across the sample varied, with an average price reduction of 19.52% (range: 0%, 61.77%).

CONCLUSIONS: The reassessment of orphan drugs in Germany, triggered by exceeding a certain annual turnover limit, impacts their benefit ratings and prices. A majority of reassessed drugs lost their added benefit rating, which has the potential to lead to substantial price reductions. This highlights the need for manufacturers to strategically plan for this reassessment.