Patient-Driven Transformation is Key to NASH Clinical Trials
There is not a day that passes that I do not receive a call to help recruit for a clinical trial. The efforts to reach out to patients seem ad hoc, scattershot, and an afterthought to rescue research and recruitment plans that fail to resonate when faced with the realities of a competitive trials landscape in a disease state with low awareness, engagement, and connection to the care centers that would normally serve as research sites. Although new practice-based research sites are standing up and even some patient advocacy organizations are implementing screening initiatives that contain an aspect of connection to research, the current model of clinical trial recruitment is unsustainable and ill-equipped to help patients or further the research needed to address NASH at the scale and in the time that we need to get multiple treatments approved to meet the urgent patient needs.
There are currently 1,028 studies in NASH that are listed or registered on ClinicalTrials.gov, with a smaller subset in NAFLD, recruiting right now. Each study—whether for drug, device, or diagnostic—seeks 30 to 200 patients. If we average about 100 patients per trial, that’s around 25,000 NAFLD/NASH patients needed immediately. On a recent episode of the Surfing the NASH Tsunami podcast entitled Increasing Diversity In The Clinical Trial Patient Population, I invited Medidata Vice President Alicia Staley, knowRx Chief Medical Officer Dr. James Powell, and GLI NASH Program Director Jeff McIntyre to discuss how we could meet the audacious challenge to fully enroll every single NASH clinical trial.
The collective assessment of the group was best stated by Alicia Staley, who is also a cancer-survivor and founder of the #bcsm social media movement: “Don’t invest in the trial; invest in the community.”
The economic cost of standing up and taking down trial infrastructure is well-documented. Less well appreciated are the costs of not laying down a foundation of awareness, education, and trust-building activities, strengthening the patient advocacy and patient communities well before recruitment for an individual trial begins.
Investing in our communities, that is the liver community writ large, and also the disproportionately impacted populations such as Hispanic/Latinx, can remove the transactional aspect of a clinical trial and enable a culture of collaboration to thrive. Creating an ecosystem that is receptive for NASH patient participation includes education and community conversation around NASH, a patient-provider relationship rooted in trust, followed by a connection to clinical trials as an option for furthering care. Systematic improvements, such as the ability to query across siloed patient registries and EHRs, will reduce time and money spent on recruitment efforts, allowing more time to enhance the understanding of NASH disease management.
Every person with the risk factors for NASH or diagnosed with NAFLD/NASH should hear repetitively in places they already go and trust that participating in the NASH clinical trial is considered a normal part of care, particularly at this point in the field, with no FDA- or EMA- approved drug. Every doctor has to be prepared to have that same conversation with them, whether it’s their primary care doctor, the endocrinologist or gastroenterologist, or certainly their hepatologist. Community health fairs, church meetings, and outreach for COVID-19 vaccination or social benefit discussions can have information about NASH clinical trials available for people.
To do our part to achieve this, GLI will be adding an in-depth session of becoming a community research advocate as part of our upcoming fifth annual Advanced Advocacy Academy (A3) (Sept 21-23). GLI will train the next generation of liver and community research advocates, who will join over 160 A3 alumni from 36 U.S. states and territories and 13 countries. A3 2021 offers the opportunity to learn from experts in the fields of patient advocacy, clinical trials, drug development, state and federal legislation, media/communications, and liver health and disease.
For more information on how to apply or support A3 to empower liver patient advocates, improve clinical trials, and create a better future for everyone connected to liver disease, please contact us at a3@globalliver.org or the links provided.
Donna R. Cryer, JD
President & CEO
Global Liver Institute
GLI News
Global Liver Institute Applauds FDA and CDC Actions to Approve Third COVID-19 Vaccine Dose for Immunocompromised Individuals
Global Liver Institute applauded dual actions from U.S. government health agencies: U.S. Food and Drug Administration approval of an additional COVID-19 mRNA vaccine dose for organ transplant recipients, liver cancer survivors, and other immunocompromised or immunosuppressed individuals, followed by a unanimous vote of recommendation for an additional dose from the Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices
GLI Updates
GLI Nutrition App with NutriStyle
In conjunction with International NASH Day 2021, GLI and NutriStyle Inc. announced a personalized nutrition app for people living with liver disease, diabetes, and other chronic conditions or who want to maintain good liver health. The app will create personalized meal plans to meet the specific requirements set out by GLI nutritional advisors for people with NASH or a general interest in liver health. The development reflects the importance of nutrition in preventing and managing liver diseases and related conditions. Visit NutriStyle to learn more.
NASH Core Curriculum: A Comprehensive Online Resource Center
GLI, in collaboration with Clinical Care Options, launched the CME course, NASH Core Curriculum: A Comprehensive Online Resource Center, to improve clinician understanding of foundational concepts in NASH diagnosis, management, and emerging pharmacologic treatment strategies. Resources are developed primarily for clinicians, including advanced practice clinicians, in hepatology, gastroenterology, endocrinology, and primary care. The full curriculum includes CME/CE-certified video modules, ClinicalThought™ expert commentaries, and downloadable presentations and resources.
The most recent module shared online focuses on making the diagnosis of NASH and features Wing-Kin Syn, MBChB, PhD, FACP, FRCP, as the module presenter. NASH Core Curriculum is supported by educational grants from Gilead Sciences and Novo Nordisk.
#OctoberIs4livers
#OctoberIs4livers is the first and only global program dedicated to public awareness, policy, and practice needs to prevent and manage liver cancer while educating and empowering those living with and impacted by liver cancer.
Designed to span the globe to improve health outcomes for liver cancer patients, the #OctoberIs4Livers program aims to rally stakeholders in creative and impactful ways to support the countless men, women, and loved ones who have heard the terrifying words “you have liver cancer.” We have something for everyone! You bring the passion; we’ll give you the tools to change the world for liver patients. Join us today!
Reaching At-Risk Patients Through COVID-19 Vaccination Sites
GLI launched a collaborative effort with Summit Clinical Research to promote NASH awareness in the context of liver health and COVID vaccination via partnerships through city vaccination sites and direct patient education. Providers will be giving people who have just received their COVID-19 vaccination our new resource, After Your COVID-19 Vaccination, Take the Next Step for Your Liver. Please contact NASH@globalliver.org if you would like to share this resource through a COVID-19 vaccination site.
U.S. NASH Action Plan: Recommendations for Medical Societies
In our January NASH News, we shared GLI’s U.S. NASH Action Plan, 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 medical societies:
Education: Develop and offer NASH-related medical school curricula.
Diagnosis: Convene a consensus conference to drive the acceleration of the adoption of noninvasive diagnostics and the simplification/streamlined version for integration into primary care and diverse (high-low resource) clinical settings.
Patient Management/Treatment: Collaborate with health systems on the development of meaningful quality measures for the diagnosis and management of NAFLD and NASH appropriate to each stage of disease.
Policy Effort/Legislation: Advocate for coverage/reimbursement coding changes and raise awareness of recent NASH coding changes that have occurred (International Classification of Diseases, Tenth Revision, Clinical Modification [ICD-10-CM] and Current Procedural Terminology [CPT]).
Surfing the NASH Tsunami
Catch the latest episodes of Surfing the NASH Tsunami podcast, which regularly features guest appearances by GLI staff such as Donna Cryer, GLI President and CEO; Andrew Scott, GLI Director of Policy; Jeff McIntyre, NASH Program Director; and Livia Alimena, EU Office Director.
GLI LIVE
After a summer break, GLI LIVE returns this fall. Join Global Liver Institute President and CEO Donna Cryer in conversation with key opinion leaders, policymakers, researchers, and patients as they address current issues important to liver patients. Join GLI LIVE with your questions on Facebook every Wednesday at 12 pm ET.
Upcoming Events
September 2: NASH Council Policy Working Group. Contact Andrew Scott for more information
September 21-23: Advanced Advocacy Academy. Contact Gina Bartes for more information
October: #OctoberIs4livers Contact Ivy Ahmed for further information
November 4: NASH Patient-Focused Drug Development (PFDD) Symposium. Contact NASH@globalliver.org
November TBD: Beyond the Biopsy (en français). Contact NASH@globalliver.org
December TBD: NASH Council Meeting. Contact NASH@globalliver.org
June 9, 2022 - International NASH Day. Contact Livia Alimena for more information
Patient Perspective
Caregiver and Patient Advocate
Nancy P. is a caregiver and patient advocate for her mother and thousands of patients and families who have suffered from NASH misdiagnosis. Nancy’s mother was wrongfully diagnosed and lived with this misdiagnosis for eight years. Further complications and additional tests eventually revealed that she was suffering from cirrhosis due to NAFLD/NASH with difficulties from hepatitis encephalopathy. “While caring for my mother, I gained such pride and admiration for her in all of the obstacles she was forced to endure trying to receive every treatment available to live after being wrongly diagnosed for years, allowing her NASH to progress to cirrhosis,” Nancy said.
In 2015, Nancy’s mother lost her battle against the disease. “She died needlessly from a wrong/missed diagnosis that could have possibly had a far better outcome had we known about NAFLD/NASH,” Nancy said. Since then, Nancy has dedicated her time to raising awareness around NAFLD/NASH and advocating on behalf of liver patients and caregivers, including taking part in GLI's Advanced Advocacy Academy (A3), which equips patients and caregivers to effectively advocate for liver health. More information on A3 2021 is here.
GLI Partner Highlight
Boston Scientific
GLI is pleased to welcome Boston Scientific as one of the newest members of the GLI NASH Council. Boston Scientific is dedicated to transforming lives through innovative medical solutions that improve the health of patients around the world. It collaborates with healthcare professionals to develop a broad portfolio of meaningful innovations that improve outcomes, reduce costs, increase efficiencies and—most importantly—help more people in more places around the world live longer, healthier lives.
Treatment advances for Non-Alcoholic Fatty Liver Disease (NAFLD) announced at ILC 2021
Leading hepatology researchers announced important new developments in the treatment of NAFLD and NASH at the International Liver Congress 2021 (ILC 2021) in June. This includes new data on trials involving RNAi therapeutics, antivirals, and structurally engineered fatty acids to treat NAFLD, which is now one of the fastest-growing diseases globally. Experts predict that over the next decade, NAFLD will become the leading cause of end-stage liver disease and liver transplantation. “News that Resmetiron appears to make inroads against NASH is most welcome – we are hopefully beginning to draw a line in the sand on the treatment of fatty liver disease,” said Philip Newsome, General Secretary of EASL and Professor of Experimental Hepatology and Director of the Centre for Liver Research at the University of Birmingham in the UK. Read about the five highlighted studies discussing treatment research for NAFLD and NASH.
Research & Development
Insights Into Metabolic Mechanisms and Their Application in the Treatment of NASH
NAFLD is the manifestation of metabolic dysregulation in the liver— an alteration of fatty acid supply, production, oxidation, and storage, resulting in NASH and risk for fibrotic liver injury leading to cirrhosis. In this review, experts highlight key metabolic mechanisms underlying NAFLD pathogenesis and indicate that new therapies targeting lipid metabolism and de novo lipogenesis (DNL) as having biological plausibility for the treatment of NAFLD and NASH. Read the review.
EASL to Hold Digital NAFLD Summit 2021
The European Association for the Study of Liver Disease (EASL) has announced the launch of the Digital NAFLD Summit 2021, taking place September 16-17, 2021. Attendees will learn from the latest research on NAFLD and NASH presented by top international experts. Submit an abstract or register online at the early-bird price.
Clinical Care
Food Insecurity Linked to Higher Mortality in People with NAFLD and Advanced Fibrosis
A team of researchers, led by Ani Kardashian, MD, from the University of Southern California in Los Angeles, carried out a retrospective cohort study using data from the U.S. National Health and Nutrition Examination Survey between 1999 and 2014 that followed 34,134 eligible participants. They found that food insecurity is associated with 46% higher risk of mortality among people with non-alcoholic fatty liver disease (NAFLD) and 37% higher risk for people with advanced liver fibrosis. Food insecurity, or the insubstantial or unsure access to nutritionally sufficient food, affected approximately 35 million people in the U.S. prior to COVID-19 and since the pandemic, has risen to 50 million. Read more in Hep magazine.
Non-Alcoholic Fatty Liver Disease is Associated with the Development of Obstructive Sleep Apnea
Obstructive sleep apnea (OSA), the recurrent collapse of the upper respiratory tract during sleep, leading to intermittent hypoxia, snoring, and sleep fragmentation, affects 7-20% of the general population and up to 48-70% of obese populations. This study, of 8,116,524 participants from the Korean National Health Insurance System, found that those with a high fatty liver index were also at high risk for OSA. When data were stratified, the risk of OSA was significantly higher in NAFLD patients with younger age, male sex, obesity, and abdominal obesity than in those with older age, female sex, without obesity and abdominal obesity. Understanding the association between NAFLD and OSA may have clinical implications for the risk-stratification of individuals with NAFLD. Read the article.
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