OBJECTIVES: Evidence transfers are permitted in Germany since 2017 under the AMNOG procedure. Since then, for pediatric drugs, evidence from non-pediatric trials may be used to delineate an added benefit. We aimed to investigate how many evidence transfers have been submitted to G-BA since 2017 and for what reasons they were rejected or accepted. In addition, we aimed to compare decisions by G-BA with EMA decisions

METHODS: We conducted a retrospective quantitative content analysis. Benefit assessments of IQWiG/G-BA and European Public Assessment Reports (EPAR) of EMA were used. The handling of evidence transfers of both institutions and reasons for rejection/acceptance were extracted starting from 2017 until December 2023.

RESULTS: 62 documents (35 benefit assessments and 27 EPARs) were included. The company applied for added benefits for all drugs in the assessments. The handling of the evidence transfers varied: IQWiG accepted an added benefit based on evidence transfers in 2 and the G-BA in 13 of the 35 cases. The main reasons for acceptance of an evidence transfer are the fulfillment of evidence transfer criteria (e.g. comparable populations) and the assessments of EMA. Similarly, non-fulfillment of these criteria is the reason against evidence transfer. Further reasons are methodological inaccuracies and lack of evidence. EMA accepted evidence transfer in 18 of the 27 or, extrapolated, in 25 of the 35 approval procedures.

CONCLUSIONS: There are considerable differences as to the acceptance of evidence transfers between IQWiG and G-BA and between G-BA and EMA. The differences of IQWiG and G-BA have an impact on the added benefit assessments and thus on the pricing of pediatric medicines. The different assessments of G-BA and EMA highlight the need for aligned and transparent criteria for evidence transfers. Currently, it is hard to anticipate the success or failure of evidence transfers.