How do you plan to #StepUpforNASH? Check out our toolkit, join the conversation on social media and make sure to use #NASHday and #StepUpforNASH.
GLI & NASH Advocates Address the Importance of Real-World Impact: FDA GIDAC Rejects NASH Treatment, Sparking Concerns
On May 19, the FDA’s Gastrointestinal Drugs Advisory Committee (GIDAC) rejected Intercept Pharmaceutical’s application for obeticholic acid (OCA) as a treatment for NASH, despite meeting the FDA’s endpoint of reducing fibrosis. The FDA’s GIDAC failed to recognize the urgent need for NASH treatments and disregarded the real-world impact on patients. Patient advocates call for a reorganization of FDA committees and divisions to prioritize patient outcomes and ensure a consistent, evidence-based regulatory process. Read more.
Nonalcoholic steatohepatitis diagnosis and treatment from the perspective of patients and primary care physicians: a cross-sectional survey
An article co-authored by Global Liver Institute revealed that although primary care doctors often treat NASH patients as their first point of contact, only a small percentage do so accurately. Many primary care doctors are unaware of guidelines for NASH diagnosis and management, highlighting the need for increased awareness. However, other physicians recognize the potential role of primary care doctors in supporting patients with lifestyle changes, weight loss, and managing related conditions.
Navigating Nutrition – Innovating for Better Health in Chronic Diseases
Jeff McIntyre, GLI’s VP of Liver Health Programs, chaired a panel at the Tufts University’s Friedman School of Nutrition, Food & Nutrition Innovation Council’s recent meeting. The panel, “Can We Innovate with Human Studies to Demonstrate Health Benefits?” discussed the perspective of how those with chronic disease can be better addressed in clinical trials around nutrition. The panel featured Noah Craft MD, PhD, of People Science; Diedre Tobias, PhD, of Harvard T.H. Chan School of Public Health; Jeffrey Blumberg, PhD, Professor Emeritus, Tufts University; and Jan-Willem van Klinken, MD, PhD, Senior Vice President Medical, Scientific & Regulatory Affairs at Brightseed Bioactives.
Global Liver Institute is proud to support the Living Donor Protection Act (H.R. 2923). In the House of Representatives, the bipartisan bill is led by Congressman Jerrold Nadler (D-NY), Congressman Troy Balderson (R-OH), Congresswoman Lisa Blunt Rochester (D-DE), Congressman Jim Costa (D-CA), Congressman John Curtis (R-UT), Congresswoman Diana DeGette (D-CO), Congresswoman Mariannette Miller-Meeks (R-IA), and Congressman Gregory F. Murphy, M.D. (R-NC). In the U.S. Senate, it is led by the bipartisan leadership team of Senator Kirsten Gillibrand (D-NY) and Senator Tom Cotton (R-AR). Organ donors, kidney patients, the insurance industry, transplant professionals, nephrologists, and many other advocacy organizations and disease professionals provided input on the policies reflected in the legislation to protect living organ donors and remove barriers to greater donation.
First, the bill prohibits life, disability, and long-term care insurance companies from denying or limiting coverage and from charging higher premiums for living organ donors.
Second, it amends the Family and Medical Leave Act of 1993 to specifically include living organ donation as a serious health condition for private and civil service employees.
Third, it directs HHS to update their materials on live organ donation to reflect these new protections and encourage more individuals to consider donating an organ.
Global Liver Institute is proud to collaborate with allies in the kidney disease space to support this legislation. Click here to contact your Member of Congress through the American Association of Kidney Patients Action Center.
Global Liver Institute Frustrated by FDA GIDAC Committee Failure to Recognize Serious, Unmet Need of Liver Patients in Rejection of Obeticholic Acid for Pre-Cirrhotic Fibrosis Due to NASH
May 22, 2023, Washington, DC – On Friday, May 19, the Food and Drug Administration’s Gastrointestinal Drugs Advisory Committee (GIDAC) voted to reject Intercept Pharmaceutical’s application for obeticholic acid (OCA) as a treatment for NASH. Despite acknowledging OCA met the endpoint established by the FDA of reducing fibrosis by at least one stage, GIDAC voted against approval for treatment for NASH. After this morning’s Intercept Pharmaceuticals investor call, our hope is that Intercept and other drug developers persist in their investments in non-viral liver diseases despite this setback.
The FDA’s GIDAC has failed to recognize the serious, unmet need of liver patients with NASH. By guessing hypothetical consequences of drug-induced liver injury against real-world patient impact without therapy, GIDAC has usurped patient/provider decision-making of benefits and individual risk. GIDAC’s fear of scaling risk ignores the real risks to current patients every day.
As Intercept met the endpoint designated by the FDA, Agency staff should not argue about the validity of that endpoint during the Advisory Committee. This unfairly shifts the regulatory goal after the fact. Actual experts and the patient community were unified in testifying that medication is needed now, not in 2-3 years when the outcome trial is finished. As Donna R. Cryer, JD, President and CEO of Global Liver Institute, stated in her testimony “Unlike Pruritus and elevated LDL, death is not a manageable side effect.”
As the authority for approval, patients rely on the FDA for a consistent, evidence-based regulatory process that prioritizes the patient experience. Patients with chronic diseases need a reliable regulatory partner to address life-impacting and life-threatening conditions. To address this sufficiently, the FDA should consider reorganizing its Committees with the realignment of their Divisions to better determine helpful patient outcomes.
With this vote, NASH patients are again left without a therapy to slow their chronic, progressive disease. Drugs with a higher efficacy and better safety profile should be approved, but we are concerned that patients do not have a regulatory partner who is acting in good faith.
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About Global Liver Institute
Global Liver Institute (GLI) was built to solve the problems that matter to liver patients, equipping advocates to improve the lives of individuals and families impacted by liver disease. GLI promotes innovation, encourages collaboration, and supports the scaling of optimal approaches to help eradicate liver diseases. GLI believes liver health must take its place on the global public health agenda commensurate with the prevalence and impact of liver illness. GLI is the only patient-created, patient-driven nonprofit organization tackling liver health and all liver disease holistically, operating globally. Follow GLI on Facebook, Instagram, LinkedIn, and YouTube.
Global Liver Institute is proud to support H.R. 2544, the Securing the U.S. Organ Procurement and Transplantation Network Act. This bipartisan legislation responds to the urgent need for reforming the Organ Procurement Transplantation Network (OTPN), breaking up the national monopoly contract so that the Health Resources Services Administration (HRSA) can run an open, competitive process and choose from the best of the best in each field (e.g., health policy, IT, logistics, patient safety) to save more lives.
We urge your help by contacting Members of Congress requesting their support for the bill introduced by Reps. Larry Bucshon (R-IN) and Robin Kelly (D-IL) to fix this broken system.
Recent train derailments in East Palestine, Ohio, have made many people wonder about the safety of their environment – which was only exacerbated when the Kansas Department of Health and Environment announced that it had identified doubled rates of liver cancer in a historically Black neighborhood that faced a similar spill two decades ago. While lifestyle choices usually receive the bulk of attention around liver disease, the environment that we live in can also play an important role. Since liver health is public health, it is important to consider how our surroundings affect our livers.
Toxins: natural products found in the environment that can be harmful to humans Toxicants: man-made, artificial products introduced to the environment by human activity that can be harmful to humans
In fact, liver damage is the most common organ damage as a result of occupational and environmental chemical exposures. A third of the most common workplace chemicals are associated with hepatotoxicity. The liver is the largest solid organ in the human body and is necessary for filtering out waste, among many critical functions. The presence of toxicants like vinyl chloride in the liver promote the presence of fat, inflammation, and scarring. These effects are similar to those of alcohol-associated liver disease and nonalcoholic fatty liver disease (NAFLD). This form of liver disease, caused by exposure to occupational or environmental toxicants, is referred to as toxicant-associated fatty liver disease (TAFLD).
Beyond an accidental spill, vinyl chloride can be found in pipes, packaging, and even wires. Industrial workers, people on military bases, or people impacted by an accident like the recent train derailment are exposed to high enough levels of vinyl chloride to be concerned. It is still unknown how low-level exposure may affect those with or without preexisting liver disease.
It is important to educate the community and encourage legislators and public health officials to better regulate toxins and toxicants like vinyl chloride and similar chemicals.
There are still steps you can take to protect yourself and others:
If you notice a chemical smell, itchiness, or become disoriented make sure to seek medical attention immediately.
Ask about the contents of the air you breathe and the water you drink.
If you work in an industry with regular chemical exposure, be sure to follow safety guidelines and properly use protective equipment.
If you have any questions or concerns about your environmental exposures, be sure to consult local poison control or public health officials. TASH, along with many other liver diseases, is preventable, and sharing this information can save lives.
Monday, May 15, 2023 Washington D.C. – As the leader of the liver cancers community, Global Liver Institute proudly supports President Biden’s nomination of Monica Bertagnolli, MD, to be the Director of the National Institutes of Health (NIH). Dr. Bertagnolli was the first woman to serve as the director of the National Cancer Institute and before that, as chief of the Division of Surgical Oncology at Brigham and Women’s Hospital and Dana-Farber Cancer Institute. She has been a professor of surgery at Harvard Medical School, was recently elected to the National Academy of Medicine and served as the president of the American Society of Clinical Oncology.
“All of us here at Global Liver Institute are grateful for President Biden nominating Dr. Monica Bertagnolli to be the next NIH director,” said Donna R. Cryer, JD, founder and CEO of Global Liver Institute. “We appreciate that Dr. Bertagnolli is a cancer survivor and will continue to drive forward the Cancer Moonshot goals to end cancer as we know it. With her background, coming from rural America, she brings a unique and fundamental understanding of the challenges people face with access to health care. We share her concern that clinical trials need to be more diverse, a significant challenge for liver disease and cancers that disproportionately affect people of color. We are excited to work with Dr. Bertagnolli as she leads NIH with a focus on collaboration, as she has demonstrated in her leadership of the National Cancer Institute.”
Global Liver Institute provided comment on the Report of the Health Disparities and Health Equity Working Group of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Advisory Council supporting NIDDK’s recommendations. The comments encouraged a more robust focus on liver disease in the report’s specific research recommendations and opportunities, especially in light of the fact that liver diseases disproportionately impact communities of color.
Therefore, GLI recommended that NIDDK list liver disease as one of the most stigmatized health conditions and partner with trusted community organizations. GLI suggested incorporating research on NASH patients as NIDDK looks to determine relationships between social determinants of health (SDOH), genetics, and the effects of each on health disparities and disease heterogeneity. GLI also advised NIDDK to train its grant reviewers so that applications advancing health equity are given weight. Lastly, GLI urged NIDDK to recruit a diverse and representative membership for its Advisory Council.
With the rise of chronic liver diseases such as NAFLD and NASH, and the growing awareness of the importance of a healthy liver for overall well-being, it is more crucial than ever to not only understand the latest developments in the field but also play an active role in them. Read on to learn about how GLI and its partners are paving the way.
GLI and NASH Patients Address ICER Shortcomings in Assessment of Treatments for NASH
On April 28, the Institute for Clinical and Economic Review (ICER) held a public meeting to present the evidence on its assessment of resmetirom and obeticholic acid for the treatment of NASH. Donna R. Cryer, alongside patient advocates from the liver community, highlighted the challenges of their personal experiences. Approval of and access to effective NASH treatments is essential to our population, and it is critically important that patient voices were heard in ICER’s assessment and in surrounding decisions made on NASH treatments.
After the event, GLI released a statement summarizing the top takeaways from the ICER review – including not recognizing concerns around caregiver impact, possible endorsement of unnecessary barriers to care (e.g., step therapy, cumbersome documentation of repeated attempts at weight loss, etc), and their lowering of grades for lack of available data when ICER rushed the review before outcomes data, long-term studies, prices and other essential elements of an accurate model are available.
GLI at The Liver Forum 14, Washington, DC
GLI President & CEO, Donna R. Cryer and Vice President of Liver Health Programs, Jeff McIntyre, appeared together on a panel at the Liver Forum 14 – a closed-door, invitation-only meeting held by the Forum for Collaborative Research to advance the regulatory sciences for the treatment of NAFLD or NASH and liver fibrosis by providing an independent and neutral venue for ongoing multi-stakeholder dialogue.
Mrs. Cryer and Jörn Schattenberg from University Medical Center, Mainz, co-moderated a panel discussion on AASLD Guidance for NASH and employing NIT-based Referral Pathways for Clinical Trials.
Media feature: Weight Matters Magazine – What Are NAFLD and NASH?
Weight Matters Magazine is the Obesity Action Coalition‘s most popular educational resource, produced quarterly with the needs of people living with obesity in mind. The Spring 2023 issue features an article written by GLI’s VP of Liver Health Programs, Jeff McIntyre, discussing why liver health is extremely important and what measures patients can take if they are diagnosed with NAFLD or NASH. ‘With the rising number of people living with obesity and other cardiometabolic disorders, liver health has gained the growing attention of medical professionals, scientists and the general public as we work to better screen, diagnose, treat and prevent liver disease.’Read it here.
Fatty Liver or NASH: The Future of Noninvasive Diagnosis and Assessment
GLI supported and helped lead the convening of a forum of international experts to discuss optimal use of non-invasive diagnostics in NAFLD or NASH using a series of clinical assertion statements. These statements covered screening patients with diabetes, applicability of liver enzymes in determining fibrosis stage, use of transient elastography and blood-based tools, such as the Enhanced Liver Fibrosis test, and potential genetic testing.
The Finnish Kidney and Liver Organization, one of GLI’s partnering organizations, is organizing a series of events around Liver Week in Finland from May 8-12. Events include a public lecture on treatment methods for fatty liver, a panel discussion on the impact of liver disease on patients’ lives and sessions targeted at primary care doctors, nurses and specialists covering diagnostic tools and advancements for treating patients with liver disease. Visit the Finnish Kidney and Liver Organization for more information.
St. Louis University Inaugural Midwest Metabolic Clinical Symposium, St. Louis, MO
GLI endorsed the inaugural Midwest Metabolic Clinical Symposium hosted by St. Louis University School of Medicine. Over 120 participants attended the event, originating from multiple states in the United States as well as the United Kingdom, Austria, and Germany. The conference was attended by healthcare professionals from a variety of fields, including Geriatric, Renal, Hematology, Primary Care, Cardiology, Critical Care, Psychiatry, as well as students to learn about current and emerging best practices in managing obesity, diabetes, NAFLD, and cardiovascular disease.
International NASH Day is 1 month away! How do you plan to #StepUpforNASH? Learn more about how you can get involved today! Or, reach out to NASHday@globalliver.org.
In this month’s edition, we feature the latest initiatives that GLI has taken part in to advocate for policy changes and highlight new innovations in the rare liver disease field to allow for better research opportunities and improved patient outcomes.
GLI Endorses the European Wilson’s Disease Policy Brief
We are excited to be endorsing the newly released policy brief on Wilson’s disease created by the Wilson’s Disease Policy Network. Wilson’s disease is an inherited condition that causes liver and brain damage due to copper accumulation. The policy brief stresses the need for increased awareness, access to appropriate care, and further research to better understand its impact.
ICP Care Creates New Patient Registry and Educational Resources for Intrahepatic Cholestasis of Liver Disease
GLI’s council member, ICP Care, launches a patient registry, which aims to pave the way for better treatment and management options. The registry is open to all, and can be accessed through a simple online consent form. Participants will be able to connect directly with researchers studying ICP and become part of a community dedicated to advancing the field. Registries like these are a crucial tool in the fight against rare diseases. We look forward to seeing the positive impact this registry will have on the lives of those affected by ICP.
Home Self-Administrable Device Uses Critical Flicker Frequency to Test for Hepatic Encephalopathy and More
Beacon is a handheld device that may change the way some liver diseases are detected and can even be used at home. By sending a dynamic, flickering light beam into the patient’s eye, it measures critical flicker frequency (CFF), a key indicator of liver function. Detection of CFF abnormalities can help identify liver diseases earlier, allowing for timely medical intervention and better patient outcomes. Read the full research study here.
On March 30, 2023, a U.S. District Court held that services recommended by the U.S. Preventive Services Task Force issued since March 2010 pertaining to the ACA coverage requirements are unlawful. Coverage requirements for vaccines recommended by CDC’s Advisory Committee on Immunization Practices (ACIP), women’s health services recommended by the Health Resources & Services Administration (HRSA), and certain services for children and adolescents recommended by Bright Future are not impacted. Below is an analysis of the impact of the decision on the preventive services impacting liver disease. The most significant impact is on screening for hepatitis B and C, which are no longer required covered services. Plans may choose to cover these preventive services, but they will be permitted to impose cost-sharing.
Topic
Impacted by Decision?
Description of Impact
Other Notes:
Screening for Hepatitis B
Yes.
The current recommendation for screening for hepatitis B virus infection in adolescents and adults at increased risk for infection would be impacted.
The services for required coverage would revert back to the 2004 recommendation, which recommends against routinely screening the general asymptomatic population for chronic hepatitis B virus infection.
It is likely that plans will no longer be required to cover screening for hepatitis B with no cost-sharing. Note that plans may still choose to cover this service but may impose cost-sharing.
Screening for Hepatitis C
Yes.
The current recommendation for screening adults aged 18-79 for hepatitis C virus infection is impacted by the decision.
The services for required coverage would revert back to the 2004 recommendation, which recommends against routine screening for hepatitis C virus infection in asymptomatic adults who are not at increased risk (general population) for infection.
It is likely that plans will no longer be required to cover screening for hepatitis C with no cost-sharing. Note that plans may still choose to cover this service but may impose cost-sharing.
Screening for Diabetes Type 2
Yes.
The current recommendation for screening for prediabetes and type 2 diabetes in adults aged 35-70 who are overweight or obese is impacted.
The services for required coverage would revert back to the 2008 recommendation, which recommends screening in asymptomatic adults with sustained blood pressure (either treated or untreated) greater than 135/80mmHG.
Diet Counseling
Yes.
The current recommendations for offering or referring adults with cardiovascular disease risk factors to behavioral counseling interventions to promote a healthy diet and physical activity is impacted.
The services for required coverage would revert back the 2003 recommendation by the USPSTF. The 2003 recommendation is for intensive behavioral dietary counseling for adult patients with hyperlipidemia and other known risk factors for cardiovascular and diet-related chronic disease.
The patient population for which such counseling will be available at no cost-sharing will likely change.
Obesity Screening for Children and Adolescents
Yes.
The current recommendation is for screening children and adolescents 6 years and older for obesity and offer/refer them to comprehensive, intensive behavioral interventions to promote improvements in weight status.
The services for required coverage would revert back to the 2010 recommendation by the USPSTF. The 2017 and 2010 recommendations appear to be very similar in wording.
The decision may not have a meaningful impact. However, the USPSTF is currently updating the recommendation, which may be placed on hold until litigation is exhausted.
Weight Loss Behavioral Health Interventions to Prevent Obesity-Related Morbidity and Mortality in Adults
Yes.
The current recommendation is that clinicians offer or refer adults with a body mass index of 30 or higher to intensive, multicomponent behavioral interventions.
The services would revert back to the 2003 recommendation by the USPSTF – which is for clinicians to screen all patients for obesity and offer intensive counseling and behavioral interventions to promote sustained weight loss for obese adults (with BMI over 30).
The impact of reverting back to the 2003 recommendation may be minimal.
STI Prevention Counseling
Yes.
The recommendations for STI behavioral counseling for all sexually active adolescents and adults at increased risk for STIs would be impacted.
The services for required coverage would revert back to the 2008 recommendation by the USPSTF – which is that sexually active adolescents and adults receive behavioral counseling to prevent STIs.
The coverage requirements for annual STI counseling for all sexually active women is not impacted by the decision, as this recommendation was made by HRSA.
Immunizations for Hepatitis B and C
Not impacted
N/A
Immunizations are recommended by ACIP and are therefore not impacted by the Court’s decision.
