Presentation (CTI)

OBJECTIVES: In Germany, benefit assessment of medicinal products conducted by the Federal Joint Committee (G-BA) defines the reimbursable patient population and thus form the basis for subsequent price negotiations. The target population presented in German early benefit assessment is therefore a critical link between clinical evidence and real-world healthcare provision. Deviations between market authorisation holder (MAH)-submitted and G-BA-derived estimates therefore directly impacts pricing outcomes. This analysis evaluated the discrepancies between MAH-submitted and G-BA-derived population estimates and identified key drivers of observed deviations.
METHODS: All benefit assessments completed between July 1 and November 15, 2025, were included. The analysis comprised (i) a conceptual description of the multi-stage derivation process and the data sources commonly used, as well as (ii) a systematic evaluation of the G BA’s acceptance, deviations, and underlying justifications.
RESULTS: Data from 53 benefit assessments were analyzed. In a subset of procedures, the MAH and the G-BA applied different definitions for the target population or its subpopulations - potentially reflecting strategic considerations, though the impact on price negotiations cannot be determined from publicly available information.Moreover, even in cases where both parties applied the same definitions, notable discrepancies in estimated target populations were identified in a considerable proportion of assessments. The most frequent driver of G-BA revisions was consistency with preceding assessments. Further reasons comprised the use of data from the MAH’s written statement, or calculations by the Institute for Quality and Efficiency in Health Care.
CONCLUSIONS: Given the complexity and strategic relevance of target population derivation for price negotiations, this analysis highlights that G-BA frequently adjusts the estimates and anchors decisions in previous appraisals. For MAH, this underscores the importance of early validation of data sources, transparent assumptions, and alignment with established decision-making patterns - a process that may become even more critical in the context of the EU Joint Clinical Assessment.