NASH News August 2020

Fatty liver disease (FLD) and non-alcoholic steatohepatitis (NASH) are increasing in prevalence worldwide, creating a major global public health crisis. To adequately educate patients, practitioners and policy makers, there is a need to collect, curate and share relevant information. NASH News, published on behalf of the Global Liver Institute’s NASH Council, intends to meet that need and to facilitate collaboration across the emerging NASH community on a monthly basis.

We would appreciate your feedback and content contributions. Please contact nash@globalliver.org

Patients Insist on Reliability, Transparency, & Engagement in the Regulatory Process

The U.S. Food and Drug Administration has undergone significant restructuring over the course of the past several months, including the creation of a new Division of Hepatology and Nutrition separate from the Division of Gastroenterology, now both under the Office of Immunology and Inflammation. This has been an opportunity for recruitment of additional expertise and organization of technological and other resources to accelerate the full and timely review of new therapies for liver diseases. Unfortunately, that is not was has occurred in NASH, with FDA actions derailing both potential NASH medications and procedures, leading patient advocates to believe that staff across the agency may be misunderstanding or underestimating the seriousness of NASH, the value of stopping progression of this condition to cirrhosis, severe complications, or cancer, and the significant unmet need for NASH therapeutics. Additionally, and with implications beyond NASH, the manner of decision making seems paternalistic, without engagement with or even explanation to patient advocates particularly on issues of value of endpoints previously agreed to and appropriate benefit/risk balance.

As someone who has served as a patient representative to the FDA more than a decade, serving on multiple advisory committees and participating in workshops and other activities, I understand the hard work and dedication that FDA brings to the challenges of therapeutic evaluation and regulation to ensure that medications and procedures are both effective and safe, particularly during a global pandemic, however, patients are dying every day from NASH. That did not stop during the pandemic. In fact, covid is demonstrating the increased risk for NASH patients at this time.

GLI has been working with the Fatty Liver Foundation, NASH Knowledge, and other advocates in the U.S. to bring the community concerns to FDA and with the European Liver Patients Association and Liver Patients International to ensure that regulatory agencies in Europe also adhere to principles of transparency and patient engagement. We will continue to press all regulatory agencies involved in reviewing medications for NASH and other liver diseases on the seriousness of these conditions, the vital need for innovation, the value of the patient perspective, and agency accountability to the patient community.

Watch our GLI Live episode on the Urgency of NASH Drug Development for more on this issue.

Donna R. Cryer, JD
President & CEO
Global Liver Institute

CMS and CDC to modernize the ICD-10-CM codes for FY 2021 by specifically adding K74.01 (early fibrosis/F1 or F2), and K74.02 (advanced fibrosis/F3)

In March of 2019, GLI requested that CMS update the ICD-10-CM and create specific codes for hepatic fibrosis distinguishing early from advanced disease, along with a note for these to be coded as NASH. GLI would also like to thank the other members of the NASH community who submitted comments of support to CMS and CDC about this change. This vital update would have not been possible without the NASH community unifying behind a single initiative.
These revisions will enable liver fibrosis to be classified more accurately and more consistently with current clinical perspectives and documentation, and will enable enhanced tracking and research into progression of disease and the impact of treatment. These 2021 ICD-10-CM codes are to be used for discharges occurring from October 1, 2020 through September 30, 2021 and for patient encounters occurring from October 1, 2020 through September 30, 2021.

Announcing the International NASH Day 2020 Report

International NASH Day 2020 was a global success thanks to our remarkable panelists, our 80 partners in 26 countries, AASLD and EASL. Read the report to learn more about our partners' virtual events, social media campaigns and materials.

Read the 2020 IND Report and watch a replay of the panels!

GLI LIVE Weekly

Please join the Global Liver Institute Wednesdays at 12 pm EDT for a regular Facebook Live event. Donna Cryer, Founder, and CEO of GLI will take coronavirus questions from liver patients, caregivers, and other community members. We’re here to help you stay informed.

Beyond the Biopsy

This September GLI will launch a Beyond the Biopsy (BTB) initiative to provide surround-sound and direct advocacy support to advocate for a greater adoption of non-invasive diagnostics at a national level whether directly through legislative, regulatory or coverage/reimbursement changes.

This initiative launches with a national radio tour on August 31 and then moves online with virtual panels focusing on three states - Colorado, Massachusetts, and New York - with leading experts, policymakers, and patient advocates. The virtual events conclude September 30th with a discussion on the state of and prospects for noninvasive diagnostics.

August Radio Media Tour

August 31st: GLI Radio Media Tour

September 2020: GLI LIVE Beyond the Biopsy Panels Schedule

Sept: 9th: GLI LIVE: Beyond the Biopsy, The Colorado Panel
Sept 16th: GLI LIVE: Beyond the Biopsy, The Massachusetts Panel
Sept 23rd: GLI LIVE: Beyond the Biopsy, The New York Panel
Sept. 30th: GLI LIVE: Beyond the Biopsy, in Review

Patient Perspective

This month, the Patient Perspective is from Kim Martinez, a GLI Advanced Advocacy Academy alumni. Kimberly discovered she had NASH when she lost weight to become a living kidney donor for her older brother, Paul.

If you could change one thing about how the healthcare system treats patients, especially those with liver disease, what would it be?

Early screening for liver problems should be as common as screenings for diabetes. Then, [once] liver problems are diagnosed, education for the patient is pivotal. ...They need to be aware of what stage of liver disease they have [and] what can be done by changing their lifestyle. I believe the liver patient should not only have a gastroenterologist but also be referred to a hepatologist.

I wish I would have been told that cirrhosis could be caused by things other than drinking or drug abuse. I was clueless, and no one told me [that] being heavy my whole life was most likely the reason I developed cirrhosis. I only found out my diagnosis, NASH, from my discharge papers. I found out what NASH was by looking it up on Google. I definitely would have benefited from point-of-care patient counseling.

Read more from Kim at GLI’s International NASH Day Patient Stories page.

GLI Partner Highlight

For this edition of NASH News, GLI is spotlighting one of our newest members of the NASH Council and Lifestyle Working group, the American College of Sports Medicine (ACSM).

The American College of Sports Medicine has more than 50,000 members and certified professionals strong from 90 countries around the globe. Representing 70 occupations within the sports medicine field, ACSM is the only organization that offers a 360-degree view of the profession.

The American College of Sports Medicine advances and integrates scientific research to provide educational and practical applications of exercise science and sports medicine. From academicians to students and from personal trainers to physicians, the association of sports medicine, exercise science, and health and fitness professionals is dedicated to helping people worldwide live longer, healthier lives.

Check out ACSM’s newly released educational material called Being Active When You Have NAFLD (also in Spanish) and Being Active When You Have Chronic Liver Disease (also in Spanish).

Updates

A blood-based biomarker panel (NIS4) for non-invasive diagnosis of non-alcoholic steatohepatitis and liver fibrosis: a prospective derivation and global validation study

In this prospective derivation and global validation study, blood samples, clinical data, and liver biopsy results from three independent cohorts with suspected NAFLD were used to develop and validate a non-invasive blood-based diagnostic test, called NIS4.

Patients’ emotions matter in the regulation of medicines

Marco Greco, President of the European Patients’ Forum, writes that factual information is essential, but numbers alone are not enough to assess human health or to understand why a patient is not adherent to their treatment, or why a patient decides (or not) to accept a certain level of risk. Emotions represent a complementary “set of data” that can help include the “real world experience” within the regulatory process, and, although interpreting this information is not always easy, it is worthwhile, rewarding and ultimately results in more patient-relevant regulatory outcomes.

Attribution of Nonalcoholic Steatohepatitis as an Etiology of Cirrhosis for Clinical Trials Eligibility: Recommendations From the Multi-stakeholder Liver Forum

Nonalcoholic fatty liver disease has emerged as a major public health threat and it is dynamic in its natural history of disease progression. A major challenge in conducting clinical trials for NASH cirrhosis is an accurate case definition for trial eligibility. It may be straightforward in clinical practice to attribute NASH as the likely cause of cirrhosis; however, the stringent case definitions required to standardize enrollment across clinical trials of investigational drugs or lifestyle interventions are currently lacking for NASH-related cirrhosis.

This article summarizes the work of the NASH Cirrhosis Working Group of the Liver Forum to specifically focus on case definitions that would allow attribution of NASH as the etiology for compensated cirrhosis.

Nonalcoholic Fatty Liver Disease and Alcoholic Liver Disease are Major Drivers of Liver Mortality in the United States

In this article from Hepatology Communications, the researchers see liver diseases remain a major public health challenge in the United States. Since 2007, mortality due to HCC and cirrhosis has been increasing in this country. Although there is a downward trend in liver‐related mortality and YLL data due to viral hepatitis, there are substantial increases related to both NAFLD and ALD. Given the increasing global trends in the epidemiology of these liver diseases, the burden of liver disease is expected to increase. In this context, it is critical that policy makers, providers, payers, and patients focus on developing national policies and multiprong strategies to deal with the burden of liver disease in the United States.

Merck licences NASH drug efinopegdutide from Hanmi

Merck has signed an exclusive licensing agreement with Hanmi Pharmaceutical to develop, manufacture, and commercialize efinopegdutide to treat nonalcoholic steatohepatitis (NASH). The drug’s safety and efficacy have been assessed in various Phase I and Phase II clinical studies, including severely obese individuals with and without type 2 diabetes.

NASH: An Introduction On The Most Severe Non-Alcoholic Fatty Liver Disease

In this piece from GLI Board member Brian Harvey, MD, PhD, NASH is considered in light of the U.S. Food and Drug Administration process for approval and recent adjustments to the process made during the current COVID-19 pandemic. Unfortunately, there are currently no FDA-approved drugs or biologics specifically indicated for the treatment of NASH patients, but change may soon be on the horizon. Multiple organizations have begun their pipeline development on drugs and treatments competing to be first to market. Phase III-IV clinical trials are also in progress, showing promising results in patients against a placebo.

Diabetes More Than Doubles Risk of Advanced Liver Disease

Type 2 diabetes increases the risk of severe liver disease in the context of non-alcoholic fatty liver disease (NAFLD), according to a recent systematic review and meta-analysis study. The team found that type 2 diabetes was linked to a more than twofold jump in the likelihood of experiencing advanced liver disease. Obesity was associated with liver disease to a lesser extent than diabetes. Lipid abnormalities and hypertension were both independently linked to liver disease, though the number of studies that looked at these factors was small.

Researchers Find Promising Therapy to Fight Epidemic of Liver Disease

In an effort to combat a growing worldwide epidemic of Nonalcoholic Fatty Liver Disease, scientists have discovered a new target and a new therapy that has shown promising results in preclinical mouse models, according to researchers at the University of Colorado Anschutz Medical Campus.

Researchers focused on Methylation-Controlled J Protein (MCJ) as a target for NASH. This protein lives in the mitochondria, the engine of the cells, where lipids are burned in the liver. MCJ acts as a brake on the metabolic activity of the mitochondria. Patients with NAFLD often have higher MCJ levels in their livers. MCJ is not critical for life under normal conditions so researchers decided to eliminate this metabolic brake in the liver to increase fat burning and minimize the accumulation of lipids and the development of fibrosis. They used Small Interfering RNA to silence MCJ in the liver. This is an emerging therapeutic approach that has shown success in treating some liver diseases.

Surfing the NASH Tsunami Podcast adds Patient Contributor

Drug developers, investors, researchers, and corporate executives wrestle weekly to understand what is happening in commercial development of NASH medications. Join Global Liver Institute Founder and CEO, Donna Cryer, as she becomes a regular panelist on the weekly Surfing the NASH Tsunami podcast - the world's leading podcast for NASH and Fatty Liver Disease.

Along with Donna, join Hepatology researcher and Key Opinion Leader Stephen Harrison, M.D., C-suite veteran Peter Traber, M.D., Founder and Medical Director of Tawazun Health Louise Campbell MSc, and Forecasting and Pricing guru Roger Green, M.B.A. as they discuss issues affecting the evolving NASH market from their own unique perspectives on the Surfing the NASH Tsunami podcast.

If you listen on Apple Podcasts, don’t forget to leave your review and rating there. What you say could ultimately help someone with fatty liver disease find hope and help!

GLI is Hiring!

Communications Manager for NASH

The Communications Manager for NASH (Nonalcoholic Steatohepatitis, an advanced form of fatty liver disease with global prevalence estimated at 25%) will advance GLI’s leadership position in patient-driven advocacy by increasing the visibility, reach, and collaborations for GLI’s initiatives in NASH, specifically the GLI NASH Council, International NASH Day, the Beyond the Biopsy Campaign, and NASH as a driver of liver cancer. See more: Communications Manager for NASH

Video Editing Specialist

GLI is looking for a video editor with their own equipment to edit footage from virtual and in-person events and manage GLI’s YouTube channel. This is a contracted, part-time position. See more: Video Editing Specialist