Presentation (CTI)

OBJECTIVES: The German Early Benefit Assessment of medicinal products for pediatric use is challenging due to limited clinical evidence in children and adolescents. In 2024, Pick et al. defined minimum criteria and proposed a decision algorithm for successful evidence transfer. This study aimed to verify these criteria and expand the understanding of evidence transfers from adult to pediatric populations.
METHODS: An internal AMNOG database was queried for procedures involving evidence transfer between 2018 and 2026. Procedures were included if evidence transfer from adult to pediatric populations was conducted by either the HTD or the G-BA. The consistency of the decision algorithm proposed by Pick et al. with G‑BA decisions was evaluated in previously unassessed procedures. Factors associated with successful evidence transfer, including those described by Pick et al., were examined. Logistic regression analyses were performed to identify factors influencing successful evidence transfer.
RESULTS: Of 59 identified procedures, 34 procedures were included in the final evaluation, including 7 previously unassessed by Pick et al. Among 30 non-orphan procedures, 14 evidence transfers were accepted (46.7%), whereas all 4 transfers in orphan procedures were rejected. In 6 of 7 previously unassessed procedures, the decision algorithm was consistent with the G‑BA decision. A backward logistic regression identified factors therapeutic breakthrough, transferable endpoints, comparable populations and mechanism of action as significantly associated with the acceptance of an evidence transfer (p<0.1).
CONCLUSIONS: The decision algorithm proposed by Pick et al. was verified. Therapeutic breakthrough, transferable endpoints, comparable populations and mechanism of action are key drivers for successful evidence transfer.