Thursday, February 25th is National Rare Disease Day®
Hartford, CT – (February 23, 2016) – Fibrolamellar. You have probably never heard of it, but it is a fatal cancer that strikes teens and young adults. On Thursday, February 25th, Fibrolamellar takes center stage as a focus for National Rare Disease Day, where, here in Connecticut, statewide activities will generate awareness for rare diseases, which collectively affect 1 in 10 Americans, and are typically the most costly, and deadly.
John Hopper, Executive Director of the Fibrolamellar Cancer Foundation (FCF) will be a keynote speaker on Thursday February 25 from 8:30am – 11am at the Legislative Office Building, 2nd Floor Atrium, 300 Capitol Avenue, Hartford, CT to address the state assembly, industry, medical institutions, academia, managed care organizations, as well as the general public, about the issues that patients with rare diseases face.
Hopper said, “Rare cancers represent over 25% of all cancers, and the majority of rare diseases. Fibrolamellar is a very rare and aggressive liver cancer that attacks teens and young adults who are typically very healthy, yet not diagnosed until Stage IV. Fibrolamellar survival rate is less than 5% beyond 24 months with no curative treatments and little research to-date. We are proud to be selected as an example to bring awareness to all of the rare diseases that affect so many people.”
During the event, patients, caregivers, medical professionals, industry representatives and state legislators will share their stories and support for the estimated 357,000 residents living with a rare disease in Connecticut.
The impact for patients is monumental. Additional funding for research to discover treatments can save lives. Dr. Ghassan Abou-Alfa MD, medical oncologist at Memorial Sloane Kettering Cancer Center in New York, who treats rare cancer patients, including those with fibrolamellar, said, “One of the greatest needs oncologists who directly treat rare cancer patients, is for more clinical research. Increased support from the government and private sectors needs to accelerate, as well as encouraging young research investigators to focus careers on finding rare cancer cures. This is a dire need for fibrolamellar and others that hit a small but important per cent of our population. Without such an emphasis, oncologists are limited to the treatment options that we can offer to try to save lives.”
Government officials are backing the FCF’s mission of building a collaborative community to support research, broaden education and build patient communities around rare disease such as Fibrolamellar. Many important decisions related to rare diseases are made at the state level, including newborn screening; support services to help families cope with complex medical needs; an environment that promotes innovative medical research and product development; and insurance practices that assure patient access to medically-necessary therapies. The implementation of the Affordable Care Act has highlighted the increasingly important role of state policies and programs in assuring that the health care needs of Americans are addressed.
U.S. Senator Richard Blumenthal said, “Ensuring quality health care for all includes those with rare diseases. Federal research funding should include studies that can result in more effective and less costly treatments for rare diseases. Affected families deserve no less attention than those families with more common and widespread illnesses.”
State Representative Fred Camillo added, “The day a parent is told their child has a rare disease such as fibrolamellar- with no cure- is the day their entire life is changed- forever. The emotional and financial stress impacts home, work, and community for these families. We, in Connecticut and beyond, need to collectively focus on ways to accelerate support, and leverage our assets to help organizations like FCF.”
To hear more about Rare Disease Day here in Connecticut, tune into your local news on Thursday or attend the Hartford forum at the State Capital. Details are available at: http://www.rarediseaseday.us/events/locations/connecticut-state-house-event-location/
About Fibrolamellar Cancer Foundation:
FCF, a public 501c3 nonprofit organization based in Greenwich, CT, was founded in 2009 by 27 year-old Tucker Davis, who lost his life to the disease in 2010. The foundations mission is to:
- fund and support research towards a cure
- better educate health care professionals, patients, caregivers and all stakeholders
- create and support a vibrant patient community.
To date, the foundation has funded over $4 million in research at major research institutions in the US and Internationally. 100% of donations go to research with all operating costs funded by the Charles and Marna Davis Foundation. www.fibrofoundation.org
About Rare Disease Day:
Rare Disease Day is an annual awareness day celebrated around the world dedicated to elevating public understanding of rare diseases and calling attention to the special challenges faced by patients. The goal of Rare Disease Day, organized by the National Organization for Rare Disorders (NORD)® (Danbury CT national headquarters) is to foster collaborative efforts across all stakeholders, especially government, to increase awareness, improve care support, encourage research, and advocate resources- financial and others- to address this growing population- in hopes for a healthier community.
Facts About Rare Disease:
- A rare or “orphan” disease in the United State is one affecting fewer than 200,000.
- There are approximately 7,000 rare diseases, according to the National Institutes of Health (NIH).
- Thirty million Americans or about 1 in 10 have a rare disease.
- Currently there are only about 320 rare disease conditions that have an FDA approved treatment.
- Two-thirds of people with rare disease are children and the diseases tend to be serious and lifelong.
- At least 80% of rare diseases have a genetic component
- Patients often have limited availability of information on their condition
- People often face difficulty getting a clear diagnosis
- Common challenges facing rare disease patients and families include but are not limited to:
- Obtaining an accurate diagnosis
- Limited treatment options
- Gaining access to the treatment options that do exist
- Difficulty finding physicians with experience in treating a particular rare disease.
- The financial struggles related to the cost of treatments
- Access to medical foods
- Genetic discrimination
- Reimbursement issues with insurance companies, especially related to off-label use
- The transition from pediatric to adult care
For more information about Rare Disease Day in the U.S., go to www.RareDiseaseDay.us. For information about global activities, visit www.RareDiseaseDay.org). To search for information about rare diseases, visit NORD’s website, www.RareDiseases.org.
Fibrolamellar Cancer Foundation:
20 Horseneck Lane, 2nd Floor, Greenwich, CT 06830
T: 203-862-3196; www.fibrofoundation.org