Patient Focused Drug Development Requires Focused Patients

POLICY PERSPECTIVE

Patient Focused Drug Development Requires Focused Patients

Ensuring there is a robust research ecosystem that develops and approves medical products to cure and treat liver diseases is a top priority for the Global Liver Institute. A key aspect of our work is facilitating the voice of liver patients and family members to set research priorities, comment on study protocols, and work with regulators. We do this by training patients through our Advanced Advocacy Academy (A3), participating in leadership positions in collaborations like the Liver Forum, and serving as patient representatives to the U.S. Food and Drug Administration and on the Executive Committee of the Clinical Trials Transformation Initiative.

There is a unique opportunity for liver patients to make an impact on the process of evaluating and approving medical products this fall by weighing in on guidances to define and improve patient-focused drug development at FDA. This is part of a larger ongoing strategic effort at FDA to engage patients, which is described quite well in this article by former FDA staffer James Valentine.

On October 15-16, 2018 there will be a 2 day public workshop on Patient-Focused Drug Development Guidance at the FDA’s White Oak Campus and live streamed. Registration closes October 8, 2018, and you can register online now.

Now that the concept of patient engagement has been accepted and examples of the value and impact of patient insights continue to grow, there will be increasing opportunities and need for patients to participate in ever more sophisticated scientific, legal, and policy settings to ensure that innovation ultimately serves the needs of patients and families. GLI looks forward to helping more liver patients and liver health advocates confidently meet these needs.

OPEN ADVOCACY OPPORTUNITIES

The National Organization for Rare Disorders (NORD) is celebrating 35 years! To mark this anniversary, NORD invites patients and caregivers to submit videos detailing their advocacy experience in a rare disease. If you or someone you know is interested in telling an advocacy story, learn more about the initiative and submit your video by September 19.

policy-update_banner.png

POLICY DEVELOPMENTS AT GLI

GLI’s Advanced Advocacy Academy (A3) is right around the corner! Spots are still available for liver patients, caregivers, and others affected by liver disease to learn the necessary skills for better advocating for liver health. Learn more about this year’s program; applications are closing soon!

Would you like to sponsor A3 or contribute to GLI’s scholarship fund that helps to cover patient travel costs to attend A3 training? To contribute or sponsor, click here.

world.png

GLOBAL

British Liver Trust

Beginning in Spring 2020, England will adopt an opt-out organ donation system. Organ donation consent after death will be presumed unless the person has opted out of the donation system. The policy applies to all adults over 18, who have lived in the UK for over 12 months, and who have the mental capacity to understand organ donation. Learn more about England’s opt-out organ donation system and how hundreds more liver patients could receive transplants.  

In the UK, end-stage liver disease (ESLD) deaths due to hepatitis C (HCV) decreased by 11% from 2016 to 2017. Public Health England, along with public health agencies in 193 countries across the globe, is working to eliminate viral hepatitis as a public health threat by 2030. This dramatic fall in HCV-related deaths, likely due to the rising number of antiretroviral drugs on the market, is a great advance. Explore Public Health England’s report and read British Liver Trust’s press release to learn more about what England is doing to fight viral hepatitis.

.GOV NEWS

Food and Drug Administration (FDA)

FDA recently established a new pathway to encourage the development of generic drugs and to enhance market competition. This pathway may ensure that patients will be able to more quickly and easily access drugs that are cheaper, but just as safe and effective as their patented predecessors. This month, FDA approved its first generic drug using the new generic drug approval pathway. Read FDA’s news release on this exciting development and learn more about the generic drug approval process.

FDA published Draft Guidance for Industry – Use of Expansion Cohorts in First-In-Human Clinical Trials to Expedite Development of Cancer Drugs and Biologics in an effort to advance cancer clinical trial approach, design, and conduction. Effective clinical trial approach, design, and conduction are crucial to the drug approval process and to progress in cancer research. In 2017, FDA approved 16 new cancer drugs and biologics and 30 new indications for existing cancer drugs and biologics. Learn more about clinical trials and how FDA is working to advance the clinical trial process.

In August, FDA approved Eisai Co. Ltd. oral medication, Lenvima, as a first-line treatment for unresectable hepatocellular carcinoma (HCC), a common form of liver cancer. The drug was jointly developed with Merck & Co.

FDA sent a letter to the state of California voicing concerns about the labelling of coffee as carcinogenic. According Proposition 65, foods and beverages that contain suspected or confirmed carcinogens must be labeled as such. Coffee beans roasted at high temperatures contain acrylamide, which is on the list. FDA Commissioner Dr. Scott Gottlieb stated that acrylamide has been linked to cancer in animals, but never in humans. In addition, coffee consumption has been linked to a decreased risk of developing liver cancer. Read more about the coffee debate in California and learn more about the benefits of drinking coffee.

US Department of Veterans Affairs (VA)

VA and Walgreens are collaborating on an initiative to improve care collaboration for US veterans. The program will allow Walgreens pharmacists to see a VA-enrolled patient’s entire list of medications and immunizations at the click of a button. VA-enrolled liver patients will be able to visit any of the over 8,000 Walgreens pharmacies across the country to access this coordinated care. Learn more about how Walgreens and VA are working to expand and improve care to US veterans.

Centers for Disease Control and Prevention (CDC)

CDC is working with several countries to help children infected with hepatitis B (HBV). Infants who contract the disease at birth, along with other children under the age of 5, are at highest risk of developing chronic health conditions and death due to HBV infection. The HBV vaccine, if given within 24 hours of birth and two to three times throughout infancy and childhood, can protect these children. CDC is working with public health officials in the Philippines and Haiti to identify the infants and children affected by HBV and to determine HBV birth vaccine dose. Read more about how CDC and these two countries are working to ensure HBV vaccine access to all children and to eradicate the disease.

RESEARCH

Risk of Hepatocellular Cancer in Patients with Non-alcoholic Fatty Liver Disease: A retrospective cohort study of patients with NAFLD from 130 V.A. facilities were followed.

New biomarker identified for hepatocellular carcinoma

contacts.png

TERMS TO KNOW

First-Line Treatment: In cancer treatment, a first-line therapy is the approach generally accepted by oncologists for initial treatment of a given type or stage of cancer.

ICD-10 Codes: The 10th version of the International Statistical Classification of Diseases and Health Related Problems (ICD) is a list of medical classifications published by WHO. ICD-10 is the first ICD version to include nonalcoholic steatohepatitis.

Portal Hypertension: increased blood pressure in the portal veins of the liver.

Policy UpdatesDonna Cryer