Online Program

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All Times EDT

Friday, September 25
Fri, Sep 25, 3:30 PM - 4:45 PM
Virtual
Challenges and Recommendations in Using Biomarker/Surrogate Endpoints for the Accelerated Approval

Statistical Considerations for NASH Trials (301224)

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*Weiya Zhang, FDA 

Keywords: NASH

Nonalcoholic steatohepatitis (NASH) is one of the most common etiologies of chronic liver disease and is estimated to affect approximately 17 million people in the U.S. Since NASH may progress to cirrhosis and may require liver transplantation, the disease represents a clear unmet need for treatment; however, except for weight loss no effective treatment currently exists. To assist sponsors in the development of safe and effective drugs and biologics to reduce the burden of this disease, the FDA has issued separate draft guidance documents regarding developing drugs for treatment of two NASH subpopulations. We will focus on the noncirrhotic NASH with liver fibrosis guidance (https://www.fda.gov/media/119044/download). This draft guidance addresses clinical trials for noncirrhotic NASH with moderate hepatic fibrosis and recommends use of histologic endpoints to support marketing applications on the accelerated approval path (Subpart H). Histologic resolution of NASH is reasonably likely to predict clinical benefit. Before initiating their trials, sponsors are encouraged to discuss specific trial design and their statistical analysis plan with the Agency. In this presentation, the speaker will give an overview of statistical considerations for conducting clinical trials for noncirrhotic NASH and moderate liver fibrosis intended to support accelerated approval.