PNS144 HOW THE RESULTS OF THE AMNOG EARLY BENEFIT ASSESSMENT (EBA) IN GERMANY HAVE DEVELOPED OVER TIME

Abstract

Since 2011, newly marketed drugs undergo an EBA in which the Federal Joint Committee (GBA) assesses the drug’s additional benefit (AB) compared to an appropriate comparator. A post-hoc segmentation of a therapeutic indication into patient populations (PP), e.g. by pre-treatment or disease severity, is possible. Specific requirements regarding study design and data analysis often constitute obstacles for pharmaceutical companies that submit a dossier in order to prove the AB. This research aims to quantify as to how the success rate of EBAs has evolved over time. All 276 EBAs finished until December 31st, 2017 were considered in this analysis. The number of PPs awarded with the highest category of AB was counted and descriptively analyzed with regard to category of AB and year. From 2011 to 2017 an increase regarding both positive (at least non-quantifiable AB) and negative results (no or smaller AB) was determined which is attributable to the rising number of EBAs per se. Between 2011 and 2014 the proportion of PPs with a positive result vs. a negative result was roughly evenly distributed. However, from 2015 onwards a rise in the proportion of positive vs. negative results became apparent. The proportion of PPs with a non-quantifiable AB among positive results has risen during the years whereas the proportion of PPs with a minor AB has declined. There is no clear picture regarding the other positive AB categories. With increasing positive EBA results, a better understanding of the methodological issues of the EBA can be assumed. However, the increasing number of marketing authorizations for orphan drugs which entail a positive EBA result by law might have played a relevant role. EBA-tailored study designs and early coordination with the GBA in this respect will likely increase the chances of positive EBA results.