NASH News February 2021
GLI’s Response to FDA Briefing on NASH Drug Development
The Food and Drug Administration (FDA) held an informative briefing at the end of January titled “Regulatory Perspectives for Development of Drugs for Treatment of NASH” (view presentation). We are pleased that the FDA has followed through on our 2020 line of dialogue and provided a response to many of our previously communicated concerns around acceptable endpoints, biomarkers, non-invasive diagnostics, and accelerated approval. Read our August 2020 statement.
While we appreciate the FDA’s acknowledgement of the seriousness of nonalcoholic steatohepatitis (NASH), we still see a lack of urgency. It is critical for the FDA to not underplay the value of stopping progression. As the FDA stated in last week’s presentation, we need more fully developed evidence on the rate of progression in patients, but the assumption of length of use (chronic) used to balance safety is still not necessarily the answer. To fill this gap in knowledge, the FDA must consider the perspective of patients with NASH and explicitly invite patient input on safety tolerance and benefit-risk acceptability.
Our critical message to the FDA remains the same — delay has a risk. Doing nothing has a risk. Striving for perfection in the midst of complexity has a risk. Our patients are risking their disease progressing to stages with life-threatening complications, like cirrhosis and liver cancer, and potentially death.
We also understand that it is the responsibility of sponsors to prove scientific advancement and reasonable evidence of efficacy. It is also necessary, however, for a complementary relationship to exist between regulators and sponsors to aid their respective efforts to support patients. The field needs a set threshold or level of acceptance provided by regulators to lead to safe and effective approved therapies.
On top of this, it was disheartening to hear the Division of Hepatology and Nutrition (DHN) stance on nonalcoholic fatty liver disease (NAFLD). While we understand that therapy approvals may fall within another division of the FDA, it is still disappointing to hear the lack of willingness to collaborate and explore NAFLD solutions.
As the voice of the liver patient community, including those with NASH, we patiently await the release of the FDA’s updated guidance later this year. We hope that the FDA will expand upon and clarify the Agency’s viewpoints even further. The lives of liver patients depend on it.
Donna R. Cryer, JD
President & CEO
Global Liver Institute
GLI Updates
Become a Partner or Sponsor for International NASH Day - June 10, 2021
Global Liver Institute (GLI) is pleased to announce that the 2021 International NASH Day (#NASHday) will be held on June 10, 2021! Please join us in a global effort to raise awareness about NASH and the actions that people can take to address this life-threatening disease worldwide.
The application process is now open for organizations to become partners and organize virtual events along the 2021 theme, NASH Around the World! Among a number of benefits, partners may use the #NASHday logo and multilingual NAFLD/NASH educational materials and apply for small program-support grants. In light of the ongoing COVID-19 pandemic, we encourage partners to organize events in a virtual format again this year. To become a partner, please submit the application and partner agreement forms by March 31, 2021. Organizations seeking funding must also submit the budget request form, as detailed on the webpage, by March 31, 2021.
We are now also accepting sponsorships for #NASHday as our gracious sponsors are critical to financing #NASHday awareness campaigns around the world. Sponsorship levels range from “Contributor” to “Platinum” as our top level. Please contact Richard Gelula for more information.
Last year, we transformed #NASHday into an engaging multi-platform experience, convening 80 partner organizations from 26 countries and more than 25 remarkable international speakers (read the 2020 NASH Day Summary Report [PDF]). We hope you will consider becoming a #NASHday partner or sponsor in 2021 and help us continue to drive global awareness.
U.S. NASH Action Plan: Recommendations for Patient Advocacy Organizations
In our January NASH News, we shared GLI’s U.S. NASH Action Plan [PDF], which includes a set of actionable recommendations for the full spectrum of groups involved in NASH prevention and treatment, including: patients and caregivers, clinicians, patient advocacy organizations, medical societies, industry, policymakers, regulators, health systems, and health insurance payers. In each edition of NASH News, we’ll highlight recommendations for each group of NASH stakeholders. This month, we’re highlighting some of the recommendations for patient advocacy organizations:
Education: Collaborate with physician (medical school and continuing) education providers on materials, including patient-provider communications in NASH.
Diagnosis: Participate and support research in non-invasive diagnostics and biomarker validation initiatives.
Patient Management/Treatment: Collaborate on the development of patient activation and engagement tools to support successful patient navigation of health systems and treatment plan adherence.
Policy Effort/Legislation: Conduct briefings and other advocacy activities to ensure NASH patient and community perspectives are represented in relevant legislative packages that address correlated conditions or conditions that increase the risk of serious health outcomes, such as COVID-19, diabetes, and obesity.
As a patient advocacy organization, GLI is supporting these areas through a number of initiatives, such as the 2021 NASH Diagnostics Forum™. Read more about how we’re implementing the U.S. NASH Action Plan on our NASH Council webpage.
NASH Council Member Listening Tour: Sign Up to Connect with GLI Staff
GLI would like to connect with each NASH Council member to learn more about our members’:
Activities regarding NASH (including communications, education, and events/conferences).
Specific interests in engaging with the NASH Council.
Ways GLI can enhance communication with NASH Council members.
Please select the time-slot that works best for you to meet via Google Meets with Whitney Gray, NASH Communications Manager, and Jeff McIntyre, NASH Program Director. Whitney will follow up with members to ensure each member has a timeslot. Email NASH@globalliver.org to inquire about membership in the NASH Council.
GLI Announces 2021 Board of Directors
GLI is pleased to announce newly elected members to its 14-member board of directors. Donna Cryer shares, “We are delighted to announce the members of GLI's 2021 Board of Directors who bring a diverse and expansive set of expertise and networks to continue our growth trajectory as we scale to meet the needs of liver patients worldwide." Patients and patient family members now comprise one-half of the board, which includes five returning members and nine newly elected directors. Victor J. Reyes, MBA, Managing Director, Human Capital, Deloitte Consulting LLP, was elected as Board Chair. View our social media channels for weekly highlights of our Board members. Read the full announcement.
GLI Presents to Health Care Providers from U.S. Department of Veterans Affairs
Donna Cryer led a virtual presentation with health care providers from the U.S. Department of Veterans Affairs on February 2, titled, The Fierce Urgency of Now: How the VA is Uniquely Equipped to Lead in Addressing the Challenges of NASH and Why It Must. The presentation focused on patient experiences in NASH and how clinicians can partner to increase appropriate screening, diagnoses, and management.
Donna emphasized the opportunity that doctors have to start conversations with their patients on NASH, diabetes, and metabolic disease. She encouraged providers to seek to “navigate this journey together” with their patients. GLI looks forward to continuing to engage with providers working with veterans and other patient groups. View the presentation [PDF].
This Is MedTech Article Featuring Donna Cryer
Donna Cryer was recently interviewed for an article shared on the blog platform, This Is MedTech, an initiative by MedTech Europe to share stories of people who have had their lives changed thanks to health and medical technologies. “There used to be so little information, but now there is a constellation of ‘wearables’ and digital scales, and more visual imaging of organ function to supplement lab work,” shares Donna in the article. She adds, “This makes me so excited to shine a spotlight on innovation, to share with others that these things are possible and available.” Read the article. Then, learn more about the GLI initiative, Beyond the Biopsy, supporting the use of non-invasive diagnostics, such as imaging technology.
Upcoming Events
February 19 - 2021 NASH Diagnostics Forum™. Invitation-only event for a multidisciplinary panel of experts. Learn more.
March 4 - NASH Council Policy Working Group. Contact Andrew Scott for more information.
May TBD - Full NASH Council Meeting. Contact NASH@globalliver.org for more information.
June 10 - International NASH Day. To become a partner and hold an event, submit an application online by March 31. Contact Richard Gelula for sponsorship information.
September 20-24 - Advanced Advocacy Academy. Contact Jen DelGrande for more information.
Patient Perspective
How Do Patients Feel and What Is Their Outlook?
The excerpt below is taken from a patient story featured on our International NASH Day website. Read the full patient’s full story.
“Today, I am alive and grateful. I was diagnosed in 2009 with Stage 4 NASH with Cirrhosis. After having a liver biopsy in early 2011, I was referred to [a medical center in the U.S.]. My first visit with my Hepatologist is one I nor my wife will ever forget. He had viewed all of my tests and gave me the news that I only had 2-3 years to live without a liver transplant. I needed to lose over 200 pounds to even qualify for a transplant and he didn’t think I would live long enough to lose enough weight…
I started my new eating plan without exercising and I was losing weight and muscle mass. After losing the first 50 pounds, I began walking with my grandson and our little dog. We were walking 2-3 miles when my grandson would tell me he was getting tired. But when I would get tired, he would say, “Come on Poppy, you can go further!” He wouldn’t allow me to quit or miss even one day unless I was sick.”
GLI Partner Highlight
American Gastroenterological Association
GLI is pleased to welcome the American Gastroenterological Association (AGA) as one of the newest members of the GLI NASH Council. AGA includes more than 16,000 members from around the globe who are involved in all aspects of the science, practice, and advancement of gastroenterology. View AGA guidelines on liver diseases. Learn more on the AGA website.
Nonalcoholic Fatty Liver Disease: Another Leap Forward - Article by Dr. Manal F. Abdelmalek
In 2020, substantial advances in research occurred regarding NAFLD mechanisms, diagnostics, and treatment. Key developments included the identification of a cellular and tissue signature to provide new insights into pathophysiology, advancements in non-invasive diagnostics, and the publication of interim results of the first phase III trial to demonstrate improvement in hepatic fibrosis. Read more in this article, Nonalcoholic fatty liver disease: another leap forward, from Dr. Manal F. Abdelmalek, Duke University School of Medicine.
Research & Development
Utility of Multiparametric CT for Identification of High-Risk NAFLD
In a study recently published in the American Journal of Roentgenology, researchers sought to evaluate the utility of laboratory and computed tomography (CT) metrics in identifying patients with high-risk NAFLD. Patients with biopsy-proven NAFLD who underwent CT within one year of biopsy were included. Study authors concluded that FIB-4 and multiple CT findings can identify patients with high-risk NAFLD (advanced fibrosis or cirrhosis), but the presence of NASH is elusive on CT. Read the full article.
Study Reveals Links Between Fatty Liver Disease and Liver Cancer
A new study at Washington University School of Medicine in St. Louis indicates that an RNA-binding protein involved in regulating lipid levels in the liver and blood promotes the development and progression of fatty liver disease and liver cancer in mice. Senior Investigator Nicholas O. Davidson, MD, director of the university’s Division of Gastroenterology, shared, “It’s been difficult to determine how liver cancer develops and why some people with fatty liver disease progress while others don’t because we haven’t had a good way to model nonalcoholic fatty liver disease in the laboratory. These new findings allow us to connect some of the dots linking fatty liver disease to cancer.” Read more in the news release.
Clinical Care
Advanced Liver Fibrosis Is Common in Patients With Type 2 Diabetes Followed in the Outpatient Setting: The Need for Systematic Screening
In a study recently shared in the February 2021 issue of Diabetes Care, a total of 561 patients with type 2 diabetes mellitus (T2D) attending primary care or endocrinology outpatient clinics and unaware of having NAFLD were recruited and invited to be screened by elastography for steatosis and fibrosis by controlled attenuation parameter (≥274 dB/m) and liver stiffness measurement (LSM; ≥7.0 kPa), respectively. Researchers found that moderate-to-advanced fibrosis (F2 or higher), an established risk factor for cirrhosis and overall mortality, affects at least one out of six (15%) patients with T2D. These results support the American Diabetes Association guidelines to screen for clinically significant fibrosis in patients with T2D with steatosis or elevated alanine aminotransferase (ALT). Read the full article.
Survey Gauges Willingness of Current and At-Risk Liver Disease Patients to Participate in Trials
SubjectWell, a company involved in patient recruitment for clinical trials, fielded a survey in November 2020 to better understand the attitudes of patients diagnosed or at risk of NAFLD or NASH toward clinical research and why these patients might consider a clinical trial (read the full findings). Researchers presented 561 at-risk patients above the age of 50 who have T2D with a questionnaire. Among 74 people diagnosed with NAFLD or NASH and 467 people who were undiagnosed, the findings included the following details (Note: Use caution when interpreting findings due to small sample size):
57% of the undiagnosed respondents reported being willing to participate in a trial for a disease they were at high-risk of developing.
72% of respondents diagnosed with NAFLD reported being willing to participate in clinical research related to their condition.
61% of patients diagnosed with NASH reported being willing to participate
Current Clinical Trials
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