Transforming the Public Consciousness with Conversations for and about the Rare Liver Disease Community
WASHINGTON, DC, FEBRUARY 1, 2022 – Global Liver Institute (GLI), a patient-led liver health nonprofit operating globally, is proud to announce today the launch of its second annual Rare Liver Diseases Month as part of its mission to improve the lives of individuals and families impacted by liver disease. The campaign, themed #RareAware, will feature an array of activities throughout February, including education, self-care, patient empowerment, and vital awareness-raising events for several rare liver diseases.
GLI President and CEO, Donna R. Cryer, JD, understands firsthand the daunting challenges of being diagnosed and living with a rare liver disease while being a 27-year transplant recipient. Mrs. Cryer said, “As someone who has lived with the ongoing challenges of an autoimmune liver disease for over 30 years, it is of personal significance to me for GLI to once again present a high-impact, month-long campaign that will continue to draw much needed attention and bring transformative change to the rare liver disease community. As the convener of the largest alliance of organizations devoted to addressing the challenges of pediatric and rare liver disease patients, GLI is deeply committed to addressing unmet needs and collaborating with our Pediatric and Rare Liver Diseases Council Members and partners to spur policy changes and design comprehensive, creative, and thoughtful solutions to issues faced on a daily basis – often for years on end – by our rare liver patients and their caregivers and support systems.”
In alignment with the International Rare Disease Day movement, the #RareAware campaign draws attention and creates resources for people living with rare liver disease. Although there are over 100 liver diseases, the majority are often overshadowed by the few most common liver diseases. Successful research and development of treatment for pediatric and rare liver diseases lags far behind that of more common liver diseases, and some medical professionals continue to lack basic knowledge to be able to effectively identify, diagnose, and treat rare liver diseases. GLI’s #RareAware campaign will highlight patient stories, support patients needs, and advocate for policy changes that will benefit the rare liver disease community, with a full month of events and conversations leading up to International Rare Disease Day on February 28.
While not part of the #RareAware campaign, GLI kicks off the month of activities with an opportunity to listen directly to patients and providers about the real world burden of a serious rare liver condition through an Externally-Led Patient Focused Drug Development Meeting (EL-PFDD) on Primary Biliary Cholangitis (PBC) on February 4. Following the PBC EL-PFDD meeting, the campaign will incorporate policy events for all those affected by rare liver disease, including leading a “Living with Rare Liver Disease in Europe ” event on February 18 and a U.S. Congressional Briefing on February 25. Weekly activities include social media takeovers by patients, introductions to self-care and wellness tools as part of GLI Living, and a spotlight on rare liver disease during GLI LIVE, the weekly virtual show hosted by Mrs. Cryer as she engages in enlightening and informative conversations with patients, physicians, and other leaders working with rare liver diseases.
To encourage extraordinary young people facing and living with rare liver disease, GLI will host weekly conversations with Evren & Kara Ayik, the co-authors of Extraordinary! A Book for Children with Rare Diseases, and the mother-son team with first-hand ultra-rare disease experience. These Extraordinary Young People events will offer the young people facing rare liver disease an opportunity to discuss identity, recognize positive impacts of their rare disease, and connect with their peers. Read more about the book here.
Nicole Wells, who is both a rare liver disease patient and GLI’s Pediatric and Rare Program Manager, personally understands the pressing need for awareness. “We welcome everyone to join the conversation around rare liver diseases. Whether rare liver disease has impacted your life first-hand or you have been inspired by the stories of patients and caregivers, I invite you to use your voice to support rare liver disease patients this February and every month. The #RareAware Advocate Toolkit on GLI’s website provides several ways to get involved,” Ms. Wells said.
International Rare Disease Day itself is officially recognized on the rarest of all days: February 29, which occurs only during leap years. However, mirroring the experience of many rare disease patients, Leap Day is represented by a surrogate day (February 28) this year and most others. The day aims to raise awareness and generate change for the 300 million people worldwide – approximately the population of the United States of America – living with rare diseases, their families, and their caretakers.
In addition to the physical, psychosocial, and financial challenges associated with any liver disease, a rare liver disease patient can face additional burdens such as a lengthy, often invasive diagnosis, formidable travel distance to see a hepatologist, and, if even available, costly treatments. These rare diseases often manifest early in life as cumbersome obstacles to normal growth and development in childhood. This is additionally complicated by the fact that many rare diseases have geographic factors, meaning each nation bears the burden of rare diseases in its own way. Despite these challenges, innovative regulatory and reimbursement models being developed as the rare disease space matures have led to increased investment, data collection/sharing, and clinical collaborations focused on rare diseases.