A Real‐World Observational Cohort of Patients with Hepatocellular Carcinoma: Design and Rationale for TARGET‐HCC.

Challenge

HCC is a complex, multidisciplinary disease with limited prospective real-world data on treatment patterns, outcomes, and the performance of novel systemic therapies in patients not represented in registration trials, creating critical gaps in natural history understanding for drug developers and regulators.

Solution

The TARGET-HCC cohort was designed as a multi-site, international longitudinal registry enrolling HCC patients at academic and community oncology and hepatology centers in the US and Europe, with medical record abstraction, PRO collection, and a biospecimen repository.

Impact

Establishing the TARGET-HCC registry created the foundational infrastructure for all subsequent HCC treatment pattern, outcomes, and safety studies, directly enabling partnerships with Genentech, Merck, and other oncology sponsors developing systemic HCC therapies.