Published On: 6/29/2021

Liver International Publishes Hepatocellular Carcinoma RWE Review

Hepatocellular carcinoma (HCC) is a deadly form of cancer on the rise in the United States. The high rates are party due to the large number of patients living with advanced hepatitis C virus (HCV) infection as well as an increasing number of people with NAFLD (known as “non-alcoholic fatty liver disease”).^{1, 2, 3}

New treatments for HCC are being studied, usually within the context of phase 2 and 3 clinical trials that have strict inclusion and exclusion criteria. However, the recent Liver International (doi.org/10.1111/liv.14864) review highlighted several sources of real-world evidence (RWE) based on data from patients with HCC and the use of RWE for this disease. Among the highlighted RWE sources were longitudinal observational studies, including Target RWE’s HCC study TARGET-HCC (NCT02954094), which helps inform the natural history of disease progression and use of new therapies/treatment approaches in usual clinical care settings. Target RWE’s study design and real-world evidence methodology for TARGET-HCC was also recently published in Hepatology Communications in December 2020.

“As real-world data are collected from electronic health records, claims databases, and pharmacy records of HCC patients, we have the opportunity to learn more about the disease and how existing treatments perform in real-world settings over time,” said Andrea R. Mospan, PhD, RAC, Senior Director of Scientific & Medical Affairs, Target RWE. “TARGET-HCC allows partners to quickly assess the safety and effectiveness data needed to inform healthcare decisions as new therapies and treatments are introduced.”

Over 1,800 patients with HCC have been enrolled in TARGET-HCC and are being followed over the course of several years. Study participants are observed across 67 academic/community sites in the United States and Europe. As highlighted in the Liver International study publication, the most common liver diseases associated with HCC included HCV infection, followed by NAFLD/NASH, alcohol-related liver disease, and hepatitis B. Patients in the study were mostly white, male, and had a median age of 64 years and had cirrhosis.

Real-world longitudinal observational studies like TARGET-HCC can contribute to understanding the long-term safety and effectiveness of these agents as well as provide a better understanding of the progression of disease.

 

¹Lok AS, Seeff LB, Morgan TR, et al. Incidence of hepatocellular carcinoma and associated risk factors in hepatitis C-related advanced liver disease. Gastroenterology 2009;136:138-48.
²Younossi Z, Stepanova M, Ong JP, et al. Nonalcoholic steatohepatitis is the fastest growing cause of hepatocellular carcinoma in liver transplant candidates. Clinical Gastroenterology and Hepatology 2019;17:748-55. e3.
³Yang JD, Hainaut P, Gores GJ, Amadou A, Plymoth A, Roberts LR. A global view of hepatocellular carcinoma: trends, risk, prevention and management. Nature reviews Gastroenterology & hepatology 2019:1-16.