Presentation (CTI)

OBJECTIVES: In Germany, orphan drugs are exempt from the obligation to show an additional benefit over the appropriate comparator (AC) if annual sales are below €30 MN; initially, an additional benefit is considered as proven with marketing authorization and at least categorized as non-quantifiable. If the sales limit is exceeded, re-assessment against an AC defined by the G-BA follows and the reimbursement price is re-negotiated. This research aims to analyze the impact of a re-assessment on the price and to identify influencing factors.
METHODS: Publicly available G-BA documents on the initial and re-assessment of orphan drugs are systematically reviewed considering study outcomes, extent and probability of additional benefit, costs of the AC and population size. The extracted findings are compared to the initial and re-negotiated price published in the LauerTaxe. Based on this pre-post-comparison, price changes are analyzed and influencing factors are identified.
RESULTS: By June 15, 2025, 33 products were re-assessed. In 18 cases, no additional benefit could be proven after re-assessment, leading to 5-60% discount on the initial price. The size of the discount essentially depends on the costs of the respective AC. In 8 cases, the additional benefit could be maintained, still leading to up to 22% discount. However, study outcomes demonstrating an improved additional benefit can justify mark-ups on the initial price of 6-21% (7 cases).
CONCLUSIONS: If the required AC is not met in the clinical trials, re-assessment represents a risk, at least at the subpopulation level, and results in non-proven additional benefit. In contrast, if the AC is reflected in the clinical trials, re-assessment offers the opportunity to present further data and thus to maintain or even improve the initial assessment. The change in the reimbursement price in the form of a discount or mark-up largely follows the re-assessment outcome.