As parents, our natural instinct is to protect our children and keep them healthy, safe, and happy. We will do almost anything to accomplish that goal. Parents and family members of children with rare diseases and chronic illnesses face even more challenges caring for their loved ones. Determining how to pay for the healthcare of children or family members is usually paramount on
that list of challenges.
Our duty as citizens includes helping and supporting our neighbors, friends, and often, strangers. The good news for many families is that this help and support is often realized in healthcare safety net organizations that provide assistance. These nonprofit organizations subsidize the high cost of health insurance premiums, deductibles, and out-of-pocket expenses related to medical costs. Some of these programs help families pay for travel expenses and other incidental costs associated with their particular disease or disorder. The mission of these charitable organizations is to keep families financially whole when going through the most difficult period of their lives.
In the last fifteen years, a major revolution has occurred in the development of medical treatments for rare and chronic diseases. Congress has supported this revolution in science by making financial commitments to the medical research portfolios through the National Institutes of Health (NIH) and passing important landmark legislation such as the Twentieth Century Cures Act (Public Law 114-255).
Funding medical research is a public policy goal to provide the initial tools to scientists to develop therapies for many disorders. Of the 7,000 rare disorders identified, only 5 percent have at least one treatment. There are millions of people waiting for the day when scientists announce that an effective treatment has been discovered for the disease that impacts their family. There is hope and progress with recent treatment approvals for the cystic fibrosis (CF) and spinal muscular atrophy communities.
Another disorder in particular that has benefited from this greater investment and awareness in medical research is sickle cell disease. Sickle cell disease is an inherited rare disorder that affects the red blood cells which carry oxygen throughout the body. Red blood cells in sickle cell disease patients form a sickle, or crescent shape, and have a shorter life cycle than standard red blood cells. These sickle-shaped cells are known to also become sticky, with the result that red blood cells adhere to joints and organs in the body consequently limiting blood flow. This causes severe pain, infection, and in some cases, death from strokes. Sickle cell disease affects primarily minority populations. According to the Centers for Disease Control and Prevention (CDC), approximately 100,000 patients live with sickle cell disease in the United States. Of those 100,000 patients, 4,000 live in the Commonwealth of Virginia. These patients are our friends and neighbors.
Recently, the first treatments for sickle cell disease have been approved by the US Food and Drug Administration (FDA). These treatments will revolutionize the life of patients with this debilitating disorder. However, what good are new treatments if a patient cannot afford to pay or afford the health insurance coverage that provides the vehicle to obtain these treatments? If access to treatment is limited by the cost, then the initiative made to develop the treatment is limited in its potential success, according to the CDC.
You Can Advocate for Equitable Care
Patient Services Incorporated (PSI), a national nonprofit patient assistance organization based in Virginia, is working to ensure that every patient with sickle cell disease in Virginia has access to these new treatments and therapies. In an attempt to see that uninsured and underinsured patients access these treatments through health insurance premium assistance and out of pocket expense assistance,
PSI is requesting that the Virginia state legislature create a patient assistance program. This program will help struggling families receive the assistance they need to fully embrace their best life and ensure that their family members receive the medical treatments they need.
Currently, the Virginia Department of Health oversees the Virginia Bleeding Disorders Program. This program provides financial assistance to patients with hemophilia AandB and Von Willebrand disease. This program is over twenty years old and has assisted hundreds of patients by subsidizing health insurance premiums. Now, PSI is advocating for the creation of a similar program at the Virginia Department of Health to provide this assistance to sickle cell patients. This new program has the potential to help families avoid having to tap into public programs like Medicare and Medicaid.
Finally, advocates from all communities understand the importance of uniting to educate and support one another around the impact of healthcare in our society. You can join PSI in these advocacy efforts to establish this program by contacting your elected representatives, specifically your State Delegate and State Senator, and asking them to support this program. View your elected representatives at vpap.org. You can also help by contacting members of the House Appropriations Committee and the Senate Finance Committee to support this budget amendment. Visit virginiageneralassembly.gov to find members of these committees.
It is our duty as parents, friends, and advocates to help ease the burden of families living with rare disease and chronic illness.
photo: Leslie Banks