This is the second report in a three-part series presenting Germany-specific data from CRA’s proprietary Rare Disease Assessment Review (RADAR) database.
RADAR contains all orphan drugs (ODs) that received European Medicines Agency (EMA) marketing authorisation between July 1, 2013, and September 30, 2024. For this analysis, we focused on 115 ODs that met the following criteria: (1) they were registered by the manufacturer with list prices freely set in the Lauer Taxe pharmaceuticals database; (2) all underwent assessment by the German Federal Joint Committee (G-BA); and (3) GKV (the statutory health insurance system) negotiations were finalized, with rebates and net prices published in Lauer Taxe (for more information on these ODs, please see Part 1 of this series).
Although RADAR includes nearly 12 years of German data, our initial approach was a cross-sectional, static analysis. In this second report, we conducted a longitudinal (dynamic) analysis to evaluate how the German Medicines Market Reorganisation Act (AMNOG) has influenced manufacturers’ price setting over time and the negotiation of GKV rebates for drugs with orphan designation.
We performed this analysis by dividing all OD launches into two six-year periods: 2013–2018 and 2019–2024. Within each period, we examined the progression of the mean (standard deviation, SD) annual cost based on manufacturer-set list prices and negotiated rebates for the ODs’ first-listed indicated use in Lauer Taxe.
