In the case of chronic diseases, health issues often do not occur until far in the future. For this reason, the goal of treatments is usually to prevent late and subsequent complications. An effect on those endpoints lying in the future would require studies of long duration, which cannot be available at the time a drug is approved. While surrogate endpoints, which offer the possibility of demonstrating effects at an early stage, have long been accepted and established by the regulatory authorities, they are only accepted in the context of the benefit assessment if complex validation studies are provided that demonstrate the direct relationship between surrogate and patient-relevant endpoint. However, such validation takes just as long as the direct use of endpoints that lie far in the future.

Thus, while the AMNOG per se already poses hurdles that are difficult to overcome, the GKV-Finanzstabilisierungsgesetz (GKV-FinStG), which came into force last year, now additionally breaks with its basic rule that a proven added benefit leads to a higher reimbursement price than for the inferior comparator therapy and thus additionally devalues the benefit categories “minor” and “non-quantifiable”.

The presentation aims to address these specific challenges while providing thought-provoking ideas for the development of the system.