Presentation (CTI)

OBJECTIVES: Post-market safety assessment of a newly launched therapy is critical for providing early insights into its safety profile in real-world settings compared to clinical trial data. This real-world data (RWD) study evaluated the safety profile of a novel therapy recently approved as combination therapy for a hematologic cancer.
METHODS: Komodo Healthcare Map®, a nationally representative US claims database, was used to obtain a randomly selected patient cohort with ≥1 medical or pharmacy claim for the novel therapy between August 2022 and September 2025. Medical claims were used to assess the frequency of common (i.e., occurring in ≥20% of patients) adverse events (AEs) reported during the key Phase 3 clinical trial (which had a median follow-up of 28.2 months), including dry eyes (51%), photophobia (47%), and drug-induced thrombocytopenia (69%). The background rates of these AEs were also investigated.
RESULTS: The analysis included 149 patients (mean [SD] age 73.7 [9.34] years and 51.7% male; similar to the trial population). The median observational period pre- and post-initiation of the novel therapy was 78 months and 25 months, respectively. In the post-initiation period, 42% (n=62), 3% (n=4), and 19% (n=28) of patients had ≥1 claim for dry eyes, photophobia, and drug-induced thrombocytopenia respectively - all lower than their respective proportions reported during the trial. Notably, ≥1 claim was also observed in the pre-initiation period for dry eyes (73% - 45/62) and thrombocytopenia (50% - 14/28).
CONCLUSIONS: This study highlights the potential for RWD use in post-market safety assessments, including providing insight into background AE rates in the indicated population prior to therapy initiation. The noted discrepancies in AE frequencies in the RWD cohort and trial subjects might be due to various factors, including variations in the method of outcome identification (ICD-10 codes in RWD vs. clinical examination in trials) and possible reporting/surveillance bias during the trial period.