OBJECTIVES: In 2020, legislation was passed in Germany that allows the G-BA to request real-world evidence (RWE) be collected to support a future renewed benefit assessment. This is particularly relevant for products that receive expedited EC approval (e.g., orphan medicines, conditional approvals, approval under exceptional circumstances, advanced therapy medicinal products [ATMPs]), because evidence available for the benefit assessment at launch is immature or insufficient for payers to derive meaningful conclusions around value. This research compares characteristics and outcomes of therapies assessed and summarises key learnings for future therapies.

METHODS: Publicly available G-BA decisions were identified from www.g-ba.de and key information extracted (June 2023).

RESULTS: Six products have completed the assessment of requirement for data collection, with two more currently being assessed. Data collection itself has begun for just one of the products – onasemnogene abeparvovec. All eight products are EMA approved orphan medicines and six are ATMPs. All products launched with single-arm pivotal trials and are lacking direct comparative data.

All six data requests specify that a non-randomised comparison with the current standard of care should be carried out; 50% of products require a specific comparator whilst the other 50% use patient-specific comparators (i.e., clinician’s choice). The G-BA named their preferred primary data source for each product and request data collected be in a study specific . The duration of requested data collection ranges from 24 to 60 months and, depending on study duration, two or three interim analyses are requested.

CONCLUSIONS: Manufacturers with orphan medicines eligible for benefit assessment using single-arm pivotal studies are highly likely to be mandated for further data collection from the G-BA. This potential barrier also presents the opportunity to demonstrate additional benefits through post-launch RWE collection. Further research is needed to establish if this data collection can be utilised to secure better HTA outcomes and enable price-renegotiation.