OBJECTIVES: Gene therapies (GT) are highly innovative medicinal products which have the potential to heal patients from a life-threatening disease with a single application. In this work, we aim to give an overview on the benefit assessment of those GT currently available on the German market. In addition, we intend to give first insights regarding the new instrument of routine practice data collection and evaluation (anwendungsbegleitende Datenerhebung, AbD), which can be imposed by the Federal Joint Committee (G-BA) to evaluate innovative medicines.

METHODS: We searched the database of the G-BA for benefit assessment procedures on GT and assessed their outcome as well as whether AbD was requested. Additionally, we searched the database of the Institute for Quality and Efficiency in Health Care (IQWiG) for AbD concepts and evaluated these regarding study methodology.

RESULTS: Out of nine procedures on GT, the added benefit of six GT was non-quantifiable. Two GT achieved a quantifiable, considerable or higher rating; one GT showed no added benefit. Five GT assessments were temporary. For two GT assessed, AbD have been already requested. Moreover, G-BA requested AbD for one GT before market entry. The first ongoing AbD with a GT is performed as a register study, and the analysis of the implementation so far showed a considerable delay to the intended timeline. Apart from AbD concepts for single medicinal products, the IQWiG has recently published a novel concept for a specific class of GT which calls for platform studies.

CONCLUSIONS: The G-BA reacts to an often poor evidence base by restricting market access for GT. AbD is increasingly imposed and may become standard in Germany for GT evaluation. Besides register studies, the concept of platform studies is being developed and may be applied to assess new classes of GT and other innovative medicines in the future.