Washington DC, Tuesday, February 27, 2018 

The Liver Forum is pleased to announce the “Early View” publication of its second manuscript, Case Definitions for Inclusion and Analysis of Endpoints in Clinical Trials for NASH through the Lens of Regulatory Science,published in the peer-reviewed journal Hepatology. The development of this manuscript included input and contributions from key opinion leaders in the field of non-alcoholic steatohepatitis (NASH), and was led by Dr. Mohammad Shadab Siddiqui (Virginia Commonwealth University), Dr. Stephen Harrison (Pinnacle Clinical Research), and Dr. Arun Sanyal (Virginia Commonwealth University). The published article is available at: https://doi.org/10.1002/hep.29607.

Dr. Veronica Miller, Executive Director of the Forum for Collaborative Research and The Liver Forum, commented on the significance of this article, noting that "the publication of this article is significant because although there are many drugs in various stages of development, there are currently no approved therapies to treat NASH. Drug development for NASH is challenged in part due to the inconsistent definitions for disease status of patients at baseline, making trial data difficult to interpret and compare. The purpose of this article was to address this challenge by building a scaffold for current and future case definitions of NASH stages."

Another goal of the manuscript is to "improve regulatory science, with the ultimate goal of providing patients with better therapeutic options than what is currently available," said Dr. Mohammad Shadab Siddiqui, Assistant Professor of Medicine at Virginia Commonwealth University. "NASH is now the leading indication for liver transplantation among new waitlist registrants, and there are no approved therapies."

This publication is an output of the Liver Forum’s Case Definitions Working Group, which was created to address the need for standardized case definitions that could be applied uniformly across NASH clinical trials and conform with regulatory standards. Experts from academia, regulatory agencies, pharmaceutical and diagnostic organizations, and patient advocacy organizations were invited to join the working group and participate in a consensus building process. The end result of this process was the development of recommendations for the “best” definitions from a regulatory science perspective, understanding that those definitions may change over time as new data becomes available.

“A key unmet need in the field is the lack of uniform case definitions for inclusion in clinical trials and the assessment of therapeutic response, which makes it virtually impossible to compare the results of one trial to the next,” said Dr. Arun Sanyal, academic co-chair of the Liver Forum and Professor of Medicine at Virginia Commonwealth University. “This manuscript represents a multi-stakeholder effort to resolve this barrier and involved input from the U.S. Food and Drug Administration and the European Medicines Agency, as well as a diverse group of academic investigators and industry researchers. It is expected that the case definitions provided in this manuscript will form the backbone of clinical trial development for NASH going forward and will thus be transformational for this field.”

Dr. Sanyal noted, “the success of the Case Definitions Working Group establishes the paradigm of such multi-stakeholder approaches to resolving scientific barriers and accelerating drug development for this common and increasingly important disease.”

About the Liver Forum
The Liver Forum is an initiative of the Forum for Collaborative Research, part of the University of California, Berkeley School of Public Health. The Liver Forum was launched in 2014 in response to the Trial Designs and Endpoints for Liver Disease Secondary to Nonalcoholic Fatty Liver Disease meeting sponsored in 2013 by the U.S. Food and Drug Administration and the American Association for the Study of Liver Disease.

The Liver Forum aims to advance the regulatory science for the treatment of non-alcoholic steatohepatitis and liver fibrosis by providing an independent and neutral platform for ongoing multi-stakeholder dialogue to identify and address gaps in the field. Neutrality and objectivity is ensured through representation and active engagement of scientific experts from all stakeholder groups, including academia, industry, patient community and regulatory agencies.

Contact Information
Katherine Greene, MPH
Senior Research Associate
Forum for Collaborative Research
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Twitter: @LiverForum