Preisregulierung durch Nutzenbewertung bei seltenen Erkrankungen

Abstract

The aim of the study is a detailed investigation of the population-normalized treatment costs of orphan drugs. For this purpose, a study was already conducted in 2022 to examine the treatment costs of orphan drugs for the years 2005 to 2021. The calculations have now been validated as part of a study update, using the identical sample size. The update enabled an inflation adjustment based on the price index of the year 2022, as well as complete treatment costs after AMNOG assessment and price negotiation for all orphan drugs. The analysis shows that the AMNOG also fulfills its function as a price regulation instrument for orphan drugs. Since the introduction of AMNOG, the inflation-adjusted normalized treatment costs for orphan drugs have fallen both in terms of the launch price (minus 25%) and the reimbursement amount (minus 6%).