Geographical and baseline clinical characteristics of participants enrolled in hepatocellular carcinoma (HCC) trials: Analysis of US FDA approvals

4115 Background: Globalization of drug development is increasing, with reliance on data from patients accrued to large, randomized studies across continents. In addition to appropriate racial/ethnic representation, trials submitted to support FDA approvals should reflect the etiology and treatment patterns of HCC in the US. We conducted a pooled analysis of patient-level data from the HCC trials supporting approved marketing applications in the US to characterize geographical and baseline variables in clinical trials. Methods: Datasets from applications which received FDA approval for HCC between 2010-2022 were identified. The following data was abstracted: country, baseline disease characteristics (median age, HCC etiology, extent of tumor, and Child-Pugh score), and prior treatments received. Geographical region was recoded using categories observed by the United Nations M49 standard, based on country of enrollment reported in the datasets. Results: Variables that were defined differently across studies included region of enrollment, some categories of etiology, and prior therapy. Overall, the rate of HBV etiology was higher than that observed among patients with HCC in the US (20%), while the rate of HCV etiology was lower than in the US (50%). Conclusions: Variability in patient populations exist across trials submitted to FDA based on the regions that were targeted for enrollment and eligibility criteria. Global trials may be appropriate to support drug approvals for HCC; however, to ensure results are applicable to US patients, sponsors should select sites that represent a similar population with respect to racial/ethnic representation, disease etiology, and treatment patterns. [Table: see text]