Application-accompanying data in benefit assessment

The aim of the project was to identify recommendations for evidence generation and evaluation by means of in-use data in the benefit assessment of orphan drugs and medicinal products.

With the GSAV, the G-BA was authorized to require application-accompanying data collection and evaluation for the purpose of benefit assessment for orphan drugs and drugs with conditional approval or approval granted under special conditions. According to the intention of the legislator, non-randomized studies and thus lower certainty of results are also to be accepted.

Based on an overview of the current handling of non-randomized studies on the part of relevant organizations as well as an overview of methodological evaluation procedures, recommendations were formulated regarding evidence generation and evaluation, which serve the objective of being able to generate usable data for a quantifiable additional benefit and thus improve the evidence situation.


Bleß, H.: „Anwendungsbegleitende Daten in der Nutzenbewertung – Empfehlungen zur Evidenzgenerierung und -auswertung“, in: „Monitor Versorgungsforschung“ (01/20), S. 47-54, doi: 10.24945/MVF.01.20.1866-0533.2202