Real-world evidence in hepatocellular carcinoma

Real-world evidence includes all health-related information, such as electronic health records, insurance claims, pharmacy records and wearables that are obtained outside of clinical trials. These data can provide critical insights into the natural history of disease and evaluate the safety and effectiveness of treatment regimens used in clinical practice. Real-world data have been applied to varying degrees by global regulatory agencies to inform and expedite many phases of drug development and help refine the use of therapeutic regimens after marketing, especially in populations that are under-represented in registration trials. For the management of hepatocellular carcinoma, early detection provides the best chance for curative therapies, whose success has been evaluated in numerous cohorts. The availability of novel systemic therapies, including kinase inhibitors and immunotherapies, has provided new treatment options and improved survival in patients with advanced stage hepatocellular carcinoma. Real-world longitudinal observational studies can help understand the long-term safety and effectiveness of these agents.

Automated summary

Real-world evidence includes health-related information obtained outside of clinical trials, which can provide insights into disease progression and treatment effectiveness. Global regulatory agencies have utilized real-world data to inform drug development and refine therapeutic regimens post-marketing, especially in underrepresented populations. In the management of hepatocellular carcinoma, early detection offers the best chance for curative therapies, while novel systemic treatments have improved survival rates. Longitudinal observational studies in real-world settings can help assess the long-term safety and effectiveness of these therapies.