Anwendungsbegleitende Datenerhebungen für die Nutzenbewertung von Arzneimitteln in Deutschland: Warum, wann und wie?

Abstract

For newly authorised medicinal products with new active substances, a benefit assessment is carried out at the time of their market access in Germany. The subject of this assessment is the question of the additional benefit of the new active substance compared to the current standard therapy (the “appropriate comparator”) with regard to patient-relevant outcomes. Orphan drugs (medicinal products for rare diseases) are treated separately. For these, the additional benefit is considered proven with market access, regardless of the actual data. Only with annual sales of at least 30 million euros a regular assessment will be conducted. In this case, data for comparison with an appropriate comparator has to be submitted. An analysis shows that in such regular benefit assessments, an additional benefit cannot be proven for orphan drugs in more than half of the cases. In 2019, in recognition of the often insufficient evidence base for orphan drugs, the „Anwendungsbegleitende Datenerhebung“ (AbD: collection of routine practice data) was established as a further component of the benefit assessment. Accordingly, the Federal Joint Committee (G-BA) can demand an AbD for orphan drugs as well as for medicinal products with a conditional marketing authorisation or marketing authorisation under exceptional circumstances. The pharmaceutical company is responsible for conducting the AbD. The AbD has to generate data on the new active substance as well as meaningful data for comparison with the appropriate comparator. The AbD is to be conducted as a study without randomisation, and the G-BA has to determine concrete specifications on the methodology and patient-relevant endpoints.The first step of the AbD is the preparation of a study protocol (SP) and a statistical analysis plan (SAP) under the responsibility of the company. These documents are checked with regard to their content-related and methodological suitability for conducting the AbD including the associated evaluation of the data. The adjustment for confounders represents a central methodological aspect of an AbD. The SPs and SAPs submitted so far had significant deficits in this regard. There is a substantial need for development in this area.

Keywords

• benefit assessment
• orphan drugs
• non-randomized controlled trials
• registries