S4-E41 - Reviewing the FDA's NIT Workshop

Send us Fan MailOn September 18-19, the FDA hosted a workshop to update key stakeholders on the state-of-the-art use of biomarkers and noninvasive tests (NITs) based on recent advances in NASH/metabolic dysfunction associated steatohepatitis (MASH) clinical trials. In this episode, Surfing NASH  reviews highlights from the workshop in three seperate interview sessions with guests Naim Alkhouri, Laurent Castera and Veronica Miller. Each guest participated in some form at the meeting and shares slightly differing but incredibly insightful perspectives with Surfers Jörn Schattenberg, Louise Campbell and Roger Green. Naim elaborates on the limitations of current pathways and the possibilities of combination NITs and conditional approval. Laurent discusses "inflammation as disease a driver, liver fibrosis as a killer and steatosis as a marker." Veronica shares thoughts around collaboration and expanding this conversation on NITs to move the field forward. Additionally, plenty more ideas are explored as this is a very fascinating and pivotal workshop which covers a range of topics on NITs with presentations by the some of the field's most innovative and knowledgable contributors. Listen to the full episode to gain a richer understanding than can be described in this summary. If you have questions or comments around the workshop, NITs, drug development or any other themes addressed in this episode, we kindly ask that you submit reviews wherever you download the discourse. Alternatively, you can write to us directly at [email protected]. Stay Safe and Surf On! [Meeting Information from the FDA Website]Use of Biomarkers for Diagnosing and Assessing Treatment Response in Noncirrhotic NASH TrialsDrug development for NASH/MASH with liver fibrosis has increased substantially and there is growing 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.