FDA Public Workshop: Clinical Pharmacology in Drug Development for Liver Diseases

FDA Announces Public Workshop on Clinical Pharmacology in Drug Development for Nonalcoholic Steatohepatitis (NASH) and Cholestatic Liver Diseases on December 9, 2019

The U.S. Food and Drug Administration (FDA), in collaboration with the Duke Margolis Center for Health Policy, will host a public workshop entitled “Leveraging Clinical Pharmacology to Optimize Drug Development for Nonalcoholic Steatohepatitis (NASH) and Cholestatic Liver Diseases” on December 9, 2019.

There is significant unmet medical need in the prevention and treatment of nonalcoholic steatohepatitis (NASH), primary biliary cholangitis, and primary sclerosing cholangitis. Despite its increasing prevalence and potential to cause severe liver-related morbidity (including liver cancer) and mortality, there are currently no drugs approved for the treatment of NASH. Recognizing that the complex etiologies, natural histories, and pathophysiologies of NASH and other cholestatic liver diseases are major impediments to ongoing drug development, the FDA released two draft guidances to assist industry in the clinical development of drugs for the treatment of NASH with liver fibrosis (https://go.usa.gov/xpqsX) and with compensated cirrhosis (https://go.usa.gov/xpqsN).

To further assist in drug development for NASH and cholestatic liver disease indications, FDA and Duke-Margolis are convening this public meeting to discuss clinical pharmacology-driven considerations and approaches to optimize drug safety and efficacy for these indications. Discussion will encompass:

• The impact of liver dysfunction on drug pharmacokinetics, pharmacodynamics, patient safety and patient outcomes;
• Emerging biomarkers, including non-invasive biomarkers, and their utility in the early-phase development of drugs for NASH and cholestatic liver disease;
• Challenges associated with optimizing clinical trial design, including adequate characterization of pharmacologic effect, selection of study population, and bridging of trial endpoints to clinical meaningfulness; and
• Clinical pharmacology approaches for optimizing drug development programs as well as the safety and efficacy of new drugs.

The date, time, location, and other details of the workshop are as follows:

Date: December 9, 2019

Time: 9:00 am to 4:45 pm

Location: Sheraton Silver Spring Hotel (Magnolia Ballroom), 8777 Georgia Avenue, Silver Spring, Maryland 20910

Persons interested in attending this public workshop in-person or via webcast can register online at https://events.r20.constantcontact.com/register/eventReg?oeidk=a07egnu5br5a1b5f0d9&oseq=&c=&ch=. Registration for this public workshop is free.

For further information, contact: Duke-Margolis Events, Duke-Robert J. Margolis, MD, Center for Health Policy, margolisevents@duke.edu.