OBJECTIVES: German health technology assessment (HTA) bodies IQWiG and G-BA examine study outcomes by categorizing them into four patient-relevant benefit dimensions: mortality, morbidity, health-related quality of life (HRQoL) and safety. In oncology, efficacy is mainly determined by overall survival (mortality) and the incidence of adverse effects. In contrast, the importance of patient-reported outcomes (PROs) reviewed as part of the categories morbidity and HRQoL remains uncertain. Objective of this research is to understand whether and how PROs impact G-BAs benefit rating decision for oncology drugs.

METHODS: The AMNOG database of Pharm-Analytics GmbH was used to identify relevant procedures. Authorized oncological non-orphan procedures initiated between 01/2020 and 02/2024 based on at least one randomized control trial were considered. Relevant documentation of the selected procedures was searched on the publicly available website of the G-BA. Data on study characteristics and PRO operationalization in dossiers, recognition, criticism and opinions by healthcare stakeholders were collected. IBM SPSS Statistics Version 29.0.2.0 (20) was used for statistical analyses including Spearman correlation.

RESULTS: 64 procedures were identified that met the selection criteria. Review of cases where study data analyses were accepted by G-BA for all benefit dimensions (N=41) showed high significant correlations (p<0.001) of G-BA benefit rating and the assessment of mortality (correlation coeffcient r_S=0.733) and HRQoL (r_S=0.538). Correlation analysis of 31 cases in which the benefit assessment of the morbidity dimension was exclusively based on PROs revealed a significant (p<0.01) correlation between the total additional benefit rating ascribed by the G-BA and the benefit dimensions of mortality (r_S=0.766), morbidity (r_S=0.511), and HRQoL (r_S=0.499).

CONCLUSIONS: In oncology, study outcomes of the mortality dimension have the greatest impact on the overall added benefit rating by G-BA. However, PRO-based study outcomes were also shown to be positively associated with the benefit rating when study data is analyzed appropriately.