Presentation (CTI)

OBJECTIVES: In the German Health Technology Assessment (HTA) process, the demonstration of added benefit relies on patient-relevant outcomes such as mortality, morbidity, health-related quality of life (HRQoL), and safety. Patient-reported outcomes (PROs) are essential for capturing aspects from the patient perspective, including health status, functioning, symptoms, and HRQoL. This abstract aims to outline key challenges and methodological requirements for the inclusion of PROs in German HTA submissions.
METHODS: We reviewed current requirements and past assessments by the German Federal Joint Committee (G-BA) to identify typical methodological hurdles. Emphasis was placed on the operationalization of PROs, endpoint selection, and common issues such as missing data or the lack of survival information at the time of benefit assessment. Our evaluation builds on practical experience with HTA dossiers submitted in Germany.
RESULTS: The absence of HRQoL data, especially in safety-relevant contexts with adverse events (AEs), often limits the justification of an added benefit. When AEs indicate harm and survival data are unavailable, it becomes particularly challenging to support a positive assessment. This issue is frequently raised by the G-BA. We identified recurring challenges in the inclusion of PROs and provide illustrative examples from previous submissions.
CONCLUSIONS: PROs play a critical role in patient-centered benefit assessment, yet their implementation faces various practical and methodological obstacles. Our findings support the feasibility of systematically integrating PROs into the HTA process and clinical trials. We recommend targeted strategies to enhance the quality and relevance of PRO data, thereby improving their utility for regulatory decision-making.