The European Orphan Medicinal Products Regulation (EC) No 141/2000 encourages developing treatments for rare diseases by offering incentives like fee reductions and extended market exclusivity. However, due to limited patient populations, high drug prices are often necessary to ensure a return on investment.
Germany’s AMNOG introduced in 2011 follows health technology assessments (HTAs) with reimbursement price negotiations and some exceptions for orphan drugs (ODs).
The German Federal Joint Committee’s (G-BA) benefit assessment acknowledges OD’s benefits by defaulting to a non-quantitative benefit.
The analysis in this report (the first in a three-part series) uses German data from RADAR, a proprietary CRA database encompassing EMA Marketing Authorisations (MA), indications, prevalence rates, HTA outcomes, prices, treatment costs, and negotiated rebates for ODs approved between July 1, 2013, and September 30, 2024.
