OBJECTIVES: The use of placebo-controlled trials typically satisfies regulatory requirements and feasibility assessments but are not always suitable for HTA. This has downstream effects on reimbursement negotiations within European. This analysis aims to understand the impact of non-comparative data on Federal Joint Committee (G-BA) benefit assessments of non-orphan drugs and the subsequent impact on the post-AMNOG price.

METHODS: Published benefit assessments from 2021 onwards were extracted from the G-BA website and reviewed to identify drugs with: a first indication launch; a population <25,000 without orphan drug designation; no comparative data (or where comparative data was deemed inappropriate); completed AMNOG pricing negotiations.

RESULTS: In total, 9 drugs were identified. In the absence of appropriate comparative data, 100% of these assessments resulted in a “no added benefit” rating with an average list price decrease of 29.9% following negotiations. Five products provided indirect treatment comparisons (ITCs), 3 of which were accepted by the G-BA but did not have a significant outcome. Additionally, 2 products withdrew from the market due to the “no added benefit” rating or an inability to achieve a price agreement at the end of the free-pricing period.

CONCLUSIONS: Although, the G-BA will accept adjusted ITCs, their stringent methodological requirements often result in rejection of comparative analysis and a resultant “no added benefit” rating. However, ITCs continue to be needed in other EU HTA processes and are important for manufacturers to conduct. Overall, it is essential for manufacturers to consider the access and pricing implications of non-comparative data for non-orphan drugs when designing clinical trial. Manufacturers are beginning to consult much earlier in the clinical development process to consider improved evidence generation plans that satisfy both regulatory and HTA requirements, ultimately leading to improved patient access.