Closing the Gap Between Pre-Clinical Efficacy Testing & Human Clinical Trials Practices in Fibrotic NASH: The Key to Successful Translation

Time: 12:00 pm
day: Day 1 Stream 2 AM


• Understanding why in diseases associated with progressive fibrosis such as NASH, there is a fundamental methodological discordance between efficacy testing in animal models of liver fibrosis and clinical trials in humans. These include treating too early and mild disease in animals and relying on “surrogate” endpoints that are not reflecting the therapeutic goal in chronic liver disease in humans.
• Exploring existing and new pre-clinical animal models should undergo continuous and systematic improvement in parallel with clinical development effort: refinement, standardization, optimization, and “humanization” of efficacy end-points.
• Understanding the fidelity of the disease models should be improved by moving towards treating more advanced diseases, longer treatment window, and assessing efficacy via long-term outcomes such as portal hypertension, progression to cirrhosis and its sequelae.