Prioritizing Liver Health in ARPA-H
POLICY PERSPECTIVE
Recently, Global Liver Institute (GLI) sent a letter to U.S. Representatives Diana DeGette and Fred Upton in response to their request for stakeholder input on the development of a new Advanced Research Projects Agency for Health (ARPA-H) within the National Institutes of Health (NIH). ARPA-H is part of the Biden Administration’s actions to renew America’s commitment to medical research and development by funding projects with the potential to transform entire areas of medicine and health.
GLI supports ARPA-H and believes it is crucial to furthering access to life-saving resources for patients impacted by liver disease. Although progress has been made by the National Cancer Institute (NCI), the National Institute of Diabetes and Digestive Kidney Diseases (NIDDK), and the Centers for Disease Control and Prevention (CDC), there is still much more work that needs to be done as liver diseases impact more than 120 million Americans. A problem of this scale calls for a solution of the same magnitude. Thus, GLI made several recommendations in its letter to Representatives DeGette and Upton.
These recommendations are centered around one goal: prioritizing liver health and increasing awareness by supporting ARPA-H’s bold, innovative projects that would be transformative to patient care, especially for those with rare diseases. Currently, knowledge and awareness about liver health is lacking across all segments of the general population, including patients living with the disease and many within the medical community outside of hepatology.
Liver health exemplifies areas where medical practice would be dramatically changed through the technologies and platforms that could be developed under ARPA-H. For these reasons, GLI urged Representatives DeGette and Upton to ensure that ARPA-H would focus on the hardest problems and areas where medical practice would be dramatically changed, including liver diseases. Furthermore, GLI urged the representatives to make sure ARPA-H would be authorized in a way that speeds cures in a different fashion – by focusing on specific problems that have eluded us to date. To achieve these goals, ARPA-H must adopt a culture and operational process that is distinct from NIH and is driven by patient-centered transparency and an urgency to improve patient outcomes.
GLI looks forward to working with Representatives Diana DeGette and Fred Upton to advance the authorization of this important new agency that could improve the lives of so many Americans impacted by liver disease.
Donna R. Cryer, JD
President & CEO
Global Liver Institute
OPEN ADVOCACY OPPORTUNITIES
Going Virtual: Apply for GLI’s Advanced Advocacy Academy (A3) Class of 2021
Applications for GLI’s Advanced Advocacy Academy (A3) Class of 2021 are now open! A3 2021 will take place virtually on September 21-23, 2021. That means the entire program will be presented through interactive, online discussions.
This year’s program will offer liver patients, caregivers, and professionals the opportunity to learn from experts in the fields of drug development, clinical trials, state and federal legislation, media and communications, and liver health and disease. The virtual experience will start with a thrilling two-day journey through self-guided learning and engaging live discussions with peers and faculty. It will then conclude with a virtual advocacy day that will be an opportunity to virtually share stories and engage in discussion about our shared liver health policy priorities. If you or someone you know is interested in attending, visit the A3 website to learn more and apply or email a3@globalliver.org with any questions.
Take Action on Some of the Most Critical Liver Health Policy Priorities
Ask Congress to Support the Medical Nutrition Equity Act (S. 2013 and H.R. 3783)
The MNEA would ensure access to medical products that are essential for the treatment of children and adults with rare liver diseases. Take action here.
Ask Congress to Support the Treat and Reduce Obesity Act (H.R. 1577 and S. 596)
TROA would expand Medicare coverage to include screening and treatment of obesity from a diverse range of healthcare providers who specialize in obesity care. Take action here.
GLI POLICY DEVELOPMENTS
GLI Response to Updated Organ Transplant Societies’ Statement on Vaccine Efficacy in Transplant Recipients
Global Liver Institute has consistently advocated for a more muscular and proactive set of solutions for transplant recipients, cancer patients, and individuals with compromised immune systems during the COVID-19 pandemic. Essential to this is strong leadership by our medical society partners. (See policy statement and letter to the transplant societies.)
Global Liver Institute Urges OMB to Help Reform Organ Transplantation System That Creates and Perpetuates Inequities for People of Color
(Washington, D.C., Thursday, July 15, 2021)—Global Liver Institute (GLI) urged the U.S. Office of Management and Budget (OMB) to implement fixes to the organ transplantation system rife with inequities for minorities, starting with three concrete steps.
Global Liver Institute Urges Secretary Becerra to Consider How Recent Federal Policy Changes Could Impact Plasma Supply for Patients
(Washington, D.C., Tuesday, August 3, 2021)—GLI urged Xavier Becerra, secretary of Health and Human Services, to consider recent policy changes that could adversely affect the supply of plasma in the United States that is critical to treating individuals impacted by liver disease. We respectfully requested that the department refrain from implementing changes that would block Mexican nationals with B1/B2 tourist visas from donating plasma, a move that would imperil the lives of Americans struggling with serious illness.
Rare Disease Week on Capitol Hill (July 14-July 22)
This past month GLI and GLI’s Advanced Advocacy Academy participated in the Virtual Rare Disease Week on Capitol Hill. The week brings together rare disease community members from across the country to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators.
GLI representatives met with more than 15 congressional offices and highlighted vital rare liver disease policy initiatives like the Medical Nutrition Equity Act and the STAT Act. GLI also highlighted general positive public health initiatives like the Connect for Health Act focused on telehealth.
Advocacy Letters
GLI joins more than 195 organizations on new flyer in support of the Safe Step Act S. 464/H.R. 2163
GLI signs onto advocacy community letter to the Administration urging it to invest in hepatitis programs following the release of the presidential budget request (Link goes to aligned GLI priorities; letter not posted as of 8/10/21.)
GLI signs onto advocacy community letter in support of the FAMILIES Act reintroduction (Link goes to aligned GLI priorities; letter not posted as of 8/10/21.)
GLI signs onto advocacy community letter urging Maryland to avoid policies that would potentially discriminate by relying on discriminatory metrics such as the Quality-Adjusted Life Year (QALY) that have detrimental implications for access to needed care and treatment. (Link goes to aligned GLI priorities; letter not posted as of 8/10/21.)
GLI signs onto viral hepatitis community letter addressed to Senate Appropriations Labor-H Subcommittee leadership requesting an increase above the House of Representatives' increase of $5 million (Link goes to aligned GLI priorities; letter not posted as of 8/10/21.)
COVID-19 Response Program for Liver Patients
GLI continues to research and update publicly available information about COVID-19, specifically addressing the needs and concerns of liver patients, particularly those who are immunosuppressed or immunocompromised due to cancer or transplant. Please check here for all of our COVID-19 response resources.
GLOBAL NEWS
Experts: Unified Global Approach Needed to Address NAFLD/NASH
A new report from eight professional societies highlights the dangers of nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) and issues a call to action to create a unified, international response to NAFLD and NASH.
Not only have many of these same recommendations been included within GLI’s U.S. NASH Action plan but they are also included with our request to the United States Preventive Services Task Force and the incoming NASH Care Act in the U.S. Congress. It is great to see the continued alignment and synchronization of recommendations across the field. We applaud the actions of our peers and look forward to continued collaboration.
Report highlights include:
Develop more sensitive and specific diagnostic methods. Liver biopsy is invasive and expensive, which limits its use, but several novel noninvasive tools are under development and could have the potential to provide more sensitive and specific diagnosis.
Adopt a multidisciplinary approach to NASH. Optimal care requires clinicians from a variety of specialties, such as primary care, hepatology, obesity management, and endocrinology. “When NAFLD progresses to NASH, multidisciplinary, team-based care involving these specialties is crucial,” the authors wrote.
Develop clinical care pathways. A pathway using validated and efficient noninvasive tests and calculators, as well as careful explication of each step in screening, diagnosis, and treatment, is under way. Disseminating pathways to all stakeholders will be an important part to develop a systematic approach to the management of NAFLD and NASH.
Pursue a unified, international, public health response. There is currently no single guiding strategy in the United States or Europe. In addition, not all hepatology/gastroenterology societies have clear screening, testing, or referral guidelines, and those that do have guidelines that may conflict with others. “Intersociety collaboration for harmonizing guidelines to optimize screening, diagnosis, and therapy is urgently required,” the authors wrote.
International Study Reveals Inhibiting Proteins E21F1 or E2F2 Can Protect Against NASH
An international study led by the Lipids & Liver research team at the UPV/EHU-University of the Basque Country recently published its results in the prestigious journal, Cancer Research. The findings show that inhibiting either of the proteins E2F1 or E2F2 in the liver can protect against the development of obesity-driven nonalcoholic fatty liver disease and its progression to cancer. Thus, these two proteins are powerful therapeutic targets for treating this disease, especially since nonalcoholic fatty liver disease affects 80 percent of obese patients and has no specific pharmacological treatment.
Hon. Dr. Ayesha Verall Launches National Hepatitis C Action Plan for Aotearoa, New Zealand
On July 28, 2021, World Hepatitis Day, Dr. Verall publicly announced New Zealand’s National Hep C Action Plan. This plan aims to eliminate hepatitis C as a major public health threat in New Zealand by 2030. The plan seeks to raise awareness of hep C, scale up existing prevention and harm reduction activities and transform the approach to delivering hep C services to people and places in the community at increased risk of infection. $2.2 million in initial funding has been allocated thus far.
3D Imaging in Sweden Reveals New Findings About Degeneration of Liver Tissue in Nonalcoholic Fatty Liver Disease
Researchers at Karolinska Institute in Sweden used novel 3-D imaging technology to discover that one portion of the autonomic nervous system in the liver undergoes severe degeneration in non-alcoholic fatty liver disease. The study, which is conducted in mice and human liver tissue, shows that the degeneration of nerves is correlated with the severity of liver pathology. According to the study, this technology can reveal even early, minor or hidden structural impairments of the liver. The research team now hopes that the study results will open the door for new therapeutic approaches in the treatment of steatohepatitis and portal hypertension by targeting the liver sympathetic nervous system.
U.S. FEDERAL NEWS
U.S. House of Representatives Passes Fiscal Year 2022 Labor, Health and Human Services, Education, and Related Agencies Funding Bill
On July 29, 2021, the U.S. House of Representatives voted on and passed a seven-bill appropriations package on a 219 to 208 vote. Most likely the bulk of the Senate’s work on spending measures will occur after the August recess.
The House legislation will provide $253.8 billion to fund agencies and programs in the Departments of Health and Human Services, Labor, and Education. More specifically, the bill includes a $6 billion increase for the NIH — $3.5 billion for the Advanced Research Projects Agency for Health (ARPA-H) and $3.5 billion for existing institutes and centers.
Highlights from the Appropriations Bill FY 2022:
U.S Department of Health and Human Services (HHS) gets $119.8 billion
National Institutes of Health (NIH) gets $49 billion
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) gets an increase of $106 million
Centers for Disease Control and Prevention (CDC) gets $10.6 billion
Substance Abuse and Mental Health Services (SAMHSA) gets $9.16 billion
Health Resources and Services Administration (HRSA) gets $9.1 billion
Agency for Healthcare Research and Quality (AHRQ) gets $380 million
Centers for Medicare and Medicaid Services (CMS) gets $4.3 billion
The Administration for Children and Families (ACF) gets $31.3 billion
Administration for Community Living (ACL) gets $3.1 billion
The FDA Approves the Use of Liver Transplant Drug (Prograf) for Other Organ Transplants
Prograf was originally a drug used to prevent organ rejection in patients receiving liver transplants. The FDA has now approved the usage of the drug in combination with other immunosuppressants to prevent organ rejection in adult and pediatric patients receiving lung transplants. This approval was based on real world evidence, showing that a well-designed study using real world and patient data can be beneficial and well- controlled under FDA regulations.
U.S. House of Representatives Energy and Commerce Committee Sends Letter to FDA on the Impact of COVID-19 on the Inspection of Drug Manufacturing
Energy and Commerce Committee Chairmen and Ranking Members Frank Pallone Jr., Cathy McMorris Rodgers, Anna Eshoo, Brett Guthrie, Diana DeGette, and H. Morgan Griffith sent a letter on July 22, 2021, to the U.S. Food and Drug Administration (FDA). This letter asked for additional information about the impact COVID-19 has had on inspections of drug manufacturing facilities and what actions FDA has taken, or plans to take, to mitigate experienced and potential future delays of drug approval decisions. According to a January 2021 Government Accountability Office (GAO) report, FDA inspections of both foreign and domestic manufacturing facilities in 2020 were reduced by 56 percent compared to the numbers of inspections in the previous two fiscal years. This will postpone drug applications, approvals, and patient access to care if inspections continue to be delayed. The chair people requested a response from FDA Commissioner Janet Woodcock by August 5, 2021.
U.S. NGO NEWS
Findings from Johns Hopkins Kimmel Cancer Center Indicate Curative Surgery May Be Possible in Liver Cancer Patients
Results published July 29 in the journal Nature Cancer by researchers at the Johns Hopkins Kimmel Cancer Center describe the benefits of this drug combination (a combination of the kinase-inhibitor drug cabozantinib and the immunotherapy drug nivolumab can make curative surgery possible in some patients with liver cancer who would normally not be considered surgery candidates). In the study 15 people with hepatocellular carcinoma (HCC), who could not be treated previously with surgery, took the drug. As a result, 12 of the patients were able to undergo successful surgical removal of their cancer. Five of these 12 patients had only 10 percent or less of their tumor remaining after the drug treatment.
NAMES TO KNOW
Dr. Rahul Gupta
Dr. Gupta was recently nominated by President Biden to be director of the White House Office of National Drug Control Policy. He will steer the response to the opioid epidemic amidst the COVID-19 pandemic. Previously. Dr. Gupta was Chief Medical Officer of the March of Dimes and Commissioner of the West Virginia Department of Health and Human Resources. Dr. Gupta would replace Regina LaBelle who has served in the position since January.
GRANT OPPORTUNITIES
Early-Stage Preclinical Validation of Therapeutic Leads for Diseases of Interest to the NIDDK (Clinical Trial Not Allowed). Funding available from the NIH. Application Deadline: 11/12/2021
Mechanisms of Disparities in Chronic Liver Diseases and Cancer (R21 - Clinical Trial Not Allowed) Application Deadline: 04/01/2022