Leveraging Clinical Pharmacology to Optimize Drug Development for Nonalcoholic Steatohepatitis (NASH) and Cholestatic Liver Diseases


FDA Convening

Leveraging Clinical Pharmacology to Optimize Drug Development for Nonalcoholic Steatohepatitis (NASH) and Cholestatic Liver Diseases


Event Description

There is significant unmet medical need in the prevention and treatment of NASH, primary biliary cholangitis (PBC), and primary sclerosing cholangitis (PSC). Despite the increasing prevalence of NASH and its potential to cause severe liver-related morbidity and mortality, there are currently no drugs approved for its treatment. There are also no FDA-approved therapies for PSC and limited therapeutic options approved for the treatment of PBC. Recognizing that the variability in etiologies, natural histories, and pathophysiologies of NASH and cholestatic liver diseases are major impediments to ongoing drug development, the U.S. Food and Drug Administration (FDA) released two draft guidances to assist industry in the clinical development of drugs for the treatment of NASH with liver fibrosis and with compensated cirrhosis.

To further assist in drug development for NASH and cholestatic liver disease, Duke-Margolis and the FDA are convening this public meeting to discuss clinical pharmacology-driven considerations and promising approaches to optimizing drug safety and efficacy. Discussion will encompass (1) the impact of liver dysfunction on pharmacokinetics, pharmacodynamics, patient safety, and patient outcomes; (2) emerging biomarkers, including non-invasive biomarkers, and their utility in the early-phase development of drugs for NASH and cholestatic liver disease; (3) challenges associated with optimizing clinical trial design, including the adequate characterization of pharmacologic effect, selection of study population, and bridging of trial endpoints to clinically meaningful outcomes and; (4) clinical pharmacology approaches to optimizing the safety and efficacy of new drugs.

Funding for this meeting was made possible in part by a cooperative agreement from the U.S. Food and Drug Administration Center for Drug Evaluation and Research. The views expressed in written meeting materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.

Duke-Margolis Team


Sarah Sheehan, MPA

Managing Associate
Senior Team Member

Image of Nicholas Harrison

Nicholas R. Harrison, MPH, MA

Senior Policy Analyst


Adam Kroetsch, MPP

Research Director, Biomedical Innovation and Regulatory Policy
Senior Team Member