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Agenda

Speaker Biographies

Day 1 Recording: Sept 18

Day 2 Recording: Sept 19

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ABOUT THIS EVENT

FDA is hosting this workshop to update key stakeholders on the state-of-the-art use of biomarkers and noninvasive tests (NITs) based on recent advances in nonalcoholic steatohepatitis (NASH)/metabolic dysfunction associated steatohepatitis (MASH) clinical trials. Drug development for NASH/MASH with liver fibrosis has increased substantially; and there is great interest in developing NITs to detect the presence of fibrosis, and to accurately classify different stages of fibrosis as well as cirrhosis. Candidate NITs include both blood tests (circulating biomarkers) and imaging tests.

In recent years, most data regarding use of NITs have been collected from NASH/MASH clinical trials. However, there are limited published data for use of NITs across the spectrum of the affected population that would be encountered in routine clinical care, including patients without fibrosis.

This workshop will assist the FDA in identifying current knowledge gaps for using NITs as diagnostic biomarkers and reasonably likely surrogates, as well as provide a framework for additional data that are needed to fill these knowledge gaps. Ultimately, the FDA seeks to learn whether expert stakeholders have evidence to indicate currently available NITs are adequate to meet the Agency evidentiary standard for assessing primary evidence of clinical efficacy.

The primary focus of this workshop is “non-cirrhotic NASH/MASH population with advanced (i.e., Stage 2 or Stage 3) liver fibrosis”. The workshop will not address the use of biomarkers for treatment trials in cirrhosis due to NASH/MASH, however, the workshop will discuss identification of “progression to cirrhosis” using biomarkers.

INTENDED AUDIENCE

Stakeholders in NASH clinical trials, including clinicians, clinical trial investigators, industry, regulators, and patients.

OBJECTIVES

• Review the currently accepted surrogate endpoints for NASH/MASH marketing trials
• Emphasize the importance of completing clinical benefit trials both to fulfill regulatory requirements for full traditional drug approval and to validate biomarkers
• Investigate new techniques to assist in interpretation of histopathology – artificial intelligence-based reads - its strengths and limitations
• Review the current data supporting the use of NITs as “diagnostic biomarkers”
• Review the current data supporting the use of NITs as biomarkers of “treatment response”
• Summarize current use of NITs in clinical practice
• Stimulate further investigations for NIT use in NASH/MASH clinical trials

FDA RESOURCES

• Guidance for Industry: Noncirrhotic Nonalcoholic Steatohepatitis With Liver Fibrosis: Developing Drugs for Treatment

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