OBJECTIVES: All benefit assessments of pharmaceutical products since the introduction of the Act on the Reform of the Market for Medicinal Products (AMNOG) in 2011 are accessible on the website of the Federal Joint Committee. Aim of this study was to examine the frequency and trend of the incorporation of Real World Evidence (RWE) studies into German AMNOG dossiers for the estimation of prevalence and incidence of the underlying disease as well as the quantification of target populations (TP).

METHODS: All published AMNOG dossiers until 31 December 2019 were included. The epidemiological section of the dossiers was screened for the inclusion of RWE studies using Statutory Health Insurance claims data, registry data, surveys, and/or other data sources (e.g. chart reviews) to determine the underlying disease and TP. Trends for the frequency of integration of respective data sources within the period 2011 to 2019 were assessed.

RESULTS: A total of 570 TP were identified within 258 AMNOG dossiers with active compounds. 253 (44.4%) TP were quantified using RWE studies. Oncologic (37.2%) and metabolic (28.1%) diseases were most prominent indication areas, followed by infectious diseases (7.4%). Most frequent RWE sources were other data (49.4%) and claims data (27.7%). After an increase until 2013 (65.9%), a downward trend regarding the integration of RWE studies in German AMNOG dossiers was observable until 2019 (27.1%). While in the first years of the German benefit assessment primarily other data sources (2013: 72.4%) were used to generate RWE, in 2019 the frequency of other data (38.5%), claims data (34.6%) and surveys (30.8%) was almost balanced.

CONCLUSIONS: In contrast to previous years, proportionately fewer RWE studies were initiated to support AMNOG dossiers in 2019. Simultaneously, the importance of individual data sources for generating RWE has become more balanced over time highlighting various opportunities for supporting epidemiological considerations.