Presentation (CTI)

OBJECTIVES: This analysis explores how international and national oncology guidelines recommend off-label use of therapies in new lines of treatment, with a focus on supporting clinical evidence and other contributing factors influencing guideline adoption. It draws insights from historical analogues to better understand the dynamics of off-label reimbursement decisions.
METHODS: Oncology drugs with a minimum of two launches for the same indication, each in a different line of therapy, between January 2013 and December 2024 were analysed across the EU4, Switzerland and Canada. Reimbursed population was compared against ESMO as well as national oncology guidelines to identify divergences. The study tracked timelines of reimbursement decisions, publication of supporting clinical data, and issuance of off-label guideline recommendations.
RESULTS: The cross-referencing identified several analogues with off-label guideline recommendations, including Lenvima in second-line (2L+) ICI-eligible advanced HCC, Abraxane in 2L+ PD-L1 ≥50% metastatic NSCLC, and Tukysa in 2L HER2+ metastatic breast cancer patients with active brain metastases. In all cases, the therapies were repositioned off-label due to the emergence of more clinically effective alternatives. Guideline updates typically followed pivotal clinical data within 12 months. Off-label use was often limited to specific patient sub-populations characterized by unmet needs, compelling evidence, or absence of approved alternatives
CONCLUSIONS: Although reimbursed population define national funding pathways, off-label use continues to be recommended in both international and national guidelines based on evolving clinical evidence. These recommendations persist even without formal label updates or reimbursement revisions. This analysis underscores the nuanced nature of clinical positioning and highlights how emerging data for new therapies can significantly influence treatment pathways.