PHP293 - ORPHAN DRUGS IN GERMANY - LESSONS LEARNED FROM AMNOG, BEST AND WORST PRACTICES AND STRATEGIC IMPLICATIONS

Abstract

AMNOG has implicated a paradigm shift in the examination of the value of a pharmaceutical and in the setting of an appropriate reimbursement level. Depending on the classification of the benefit category, the pharmaceutical manufacturer negotiates the final refunded price with the payer organization GKV-SV. For orphan drugs, there are specific challenges. Due to the randomness of the disease, subpopulations are small and study data are scarce. This leads to uncertainty about the perceived value of the drug and, thus, to intense and sometimes tough negotiations resulting in high rebates. Pharmaceutical companies planning to introduce new orphan drugs should therefore prepare a sound and stringent strategy well in advance. This research elaborates on arising issues and describes important recommendations for manufacturers facing these challenges. G-BA resolutions and its reasonings for all procedures of orphan drugs were retrieved up until May 31st, 2018 from the G-BA homepage. Based on these published documents, active substance, therapeutic area, indication, extent of additional benefit and negotiated rebate were extracted. To date, 72 benefit assessment procedures of orphan drugs have been carried out, 39 in oncological indications and 15 in indications of metabolic diseases. In approximately 60% of all assessed drugs the G-BA granted a non-quantifiable additional benefit. Only a subpopulation of one drug was assessed with a major benefit. The range of negotiated rebates varies between 14.7% to 72.0%. Since the implementation of AMNOG, the number of benefit assessments of orphan drugs has steadily increased with a peak in 2016. Indications in the therapeutic areas of oncology and metabolic diseases are particularly well represented. Based on the analysis it can be assumed that benefit assessments for orphan drugs become more challenging, in particular for oncological drugs.