Presentation (CTI)

OBJECTIVES: AMNOG provides a process to negotiate reimbursed prices for new drugs based on established additional benefits. While AMNOG softens some assessment criteria for ODs at launch, ODs have to undergo GKV rebate negotiations. Our research aims to identify key impact factors on negotiated GKV rebates for OD.
METHODS: The RAre Disease Assessment Review database (RADAR) is a proprietary CRA OD-database. For Germany, it includes publicly available information on indication, prevalence rates, G-BA assessments, reimbursed list/ net unit prices as published in Lauer Taxe, calculated annual cost based on SmPC dosing, and negotiated GKV rebates. It includes 164 ODs that received EMA Marketing Authorization between July 2013 and September 2024. The analysis included ODs with available information on published list price at launch, G-BA assessment outcome, GKV-rebate, and negotiated reimbursement price. For those 115 ODs that fulfilled all analysis criteria, we tested the impact of GBA-assessment outcome, clinical trial comparator, prevalence, indication, and manufacturer-set list price on negotiated GKV-rebate. Impact size was measured as deviation from mean/median rebate.
RESULTS: Overall mean and median GKV-rebate were calculated with 22.5%. Factors most contributing to higher (+) or lower (-) GKV rebates vs. mean for ODs are GBA-assessment outcomes considerable (-44%), non-quantifiable (+10%), and no added benefit (+25%), disease prevalence above (+13%) / below (-12%) median, oncology indication (+15%), and manufacturer set list price above €500,000 annual cost (+20%).
CONCLUSIONS: Our results allow manufacturers to engage more informed in GKV negotiations when linking OD product characteristics to historic negotiation results: AMNOG seems to work as intended, also for ODs. GBA assessments seem to translate into rebates. Despite orphan designation, population size has significant impact on rebate size. Oncology ODs seem to be scrutinized more, potentially due to treatment alternatives in adjacent indications. Perceived high manufacturer-set list prices seem to lead to higher GKV-rebates.