Application-Related Data Collection During Early Benefit Assessments in Germany: An Analysis of Current Procedures

OBJECTIVES: In Germany, the Act for Greater Safety in Drug Supply empowered the Federal Joint Committee (G-BA) in 2019 to require manufacturers to conduct application-related data collection (AbD) as part of the benefit assessment, mainly in case of initially missing evidence. As the number of new approvals of advanced therapy medicinal products and orphan drugs increases, evidence gaps appear for the initial assessment, which are to be closed by data from AbD, while providing access for patients. We aimed to analyze which AbDs were commissioned so far, in which process steps the procedures are and which challenges appeared.

METHODS: We conducted a retrospective quantitative content analysis. Benefit assessments of IQWiG/G-BA and other data on AbD (e.g. statistical analysis plans (SAPs), submitted protocols) from the G-BA homepage were used. The extraction took place until 31 March 2024.

RESULTS: To date, 13 procedures have been initiated to request an AbD. In two cases, an AbD is currently running. The respective therapies can be divided into three therapeutic areas: neurodegenerative diseases, oncological diseases and diseases of the blood and blood-forming organs. On average, 300 days passed from the start of the procedure to the request for an AbD. During the first review of the protocols and SAPs prepared by the company, a total of 61 deficiencies were identified. Most of them were found in the categories PICO, identification and collection of confounders and their adjustment, and quality and completeness of data collection.

CONCLUSIONS: Conducting AbDs is time-consuming and complex due to the individual requirements for data collection. The establishment of the process of conducting AbDs is still in its early stages. However, the generation of data close to care has the potential to improve the evidence for quantifying the additional benefit of a drug and to close possible evidence gaps.