The urgent case for patient assistance funding
According to the National Economic Burden of Rare Disease Study, developed by the EveryLife Foundation for Rare Diseases and the rare disease community, 548 billion dollars, is spent on out-of-pocket, non-medical costs in the United States. The list of these non-medical costs is endless because each patient, each family, has a different rare disease scenario.
Like with research, rare disease patients can either be a number on a research slide or real flesh and blood. The personal stories of real rare disease patients are what makes the case for patient assistance funding outside of the research sphere. Here is but one story.
From the fifth day of his life until the seventh month of his life, Avery spent his days and nights in the hospital. Newly diagnosed with an ultra-rare gastrointestinal (GI) disease, it took a great deal of time to dial in the treatment correctly so that he could thrive, despite the tubes and battery packs attached to his little body.
Both parents were gainfully employed at the time of Avery’s birth. Dad owned a restaurant and Mom and Dad together owned a hair salon where Mom was the head stylist. There was no way that Avery was going to be attended to only by nurses and staff. One of them had to stay, wanted to and needed to stay. The choice wasn’t easy, but Mom had the insurance and Dad had at least some employees to cover for him in his absence. Just about seven months of hospital stay were before them, but they didn’t know that yet. Ultimately, Dad had to sell his portion of the business, and now they were down to one salary and had a beautiful little boy with a very rare condition.
Finally, that first long hospital stay came to a close and Avery and Dad were discharged from hospital beds, food, bright lights and noise. With a $5 a day stipend for food, a good amount of money was spent to keep Dad fed and nourished to some degree. While discharge went smoothly, despite the frightening instructions and the images of what lay ahead, the next step was to retrieve the car and leave the hospital garage. Long hospital stays require long garage stays and hospital administration does everything in its power to lower costs. So instead of $16 a day, the cost was $5 a day. Seven months equals approximately 210 days times $5 a day. Do the math! They haven’t even left the hospital yet.
Once the credit card was processed and the gate opened it was a two-hour trip home with a stop for gas and a stop at the pharmacy. Besides the insurance-paid medications, there were the suggested vitamins, the extra supply of diapers and wipes that a rare GI disease would require. None of that is covered by insurance and was totally unexpected. That was just day one.
And so, a small all-volunteer organisation was formed to help those facing the same high cost of living with rare disease. This includes what insurance doesn’t include, what insurance denies and what becomes the many out-of-pocket expenses.
When Avery’s Hope was first forming, we grappled with what type of organisation we would be. Would we be an organisation that raised money for research in the hope that we could facilitate a cure? Would we be an organisation that raised money for both research and for patient assistance or would we just focus on patient assistance and the immediate needs of those families “living rare”.
If we became a research-based organisation, we knew that we might not ever get a cure, let alone a treatment for symptoms. Did we want to leave a legacy of work to be continued beyond our existence? Avery’s Hope decided instead to become a patient assistance organisation to help with the immediate needs of out-of-pocket and insurance-denied expenses and the often surprising costs of travel to and from medical appointments, hospital stays, loss of income and the like while all the time the rent, mortgage, cost of food, and more continues to climb.
Avery was only home a short time before the next hospitalisation, the next needed test, the change in medication, the travel to yet another hospital for another opinion, which included hotel stays and food costs. Avery is not alone. With close to 8,000 rare diseases and 350 million patients worldwide, half of them children, we knew that the need was not only there but that the need was a desperate one.
The cost of living rare
The unexpected costs of “living rare”, no matter what the diagnosis, never stops. With the COVID-19 pandemic upon us as well, there are other considerations and situations. And, so, Avery’s Hope chose patient assistance. We won’t be the organisation that worked hard to find a cure or a path to a cure, but we will be that organisation that puts food on the table, pays for medications that insurance will not and will get patient families to their next appointment.
Research is incredibly important and we wish we could be a part of that world as well. Instead, we made the case for patient assistance in the hope that we could ease a rare patient family’s burden. And then another, and another.
The unfortunate truth is that patient assistance organisations, the small grassroots organisations made up entirely of volunteers, don’t often raise as much money as those that are funding research. We accept that and can only hope that donors and stakeholders will recognise the enormous patient need as well as the enormous need for research dollars and consider writing some of those big checks to this side of the rare disease story. It’s really two sides of the very same coin. One cannot truly exist without the other.
Convincing and educating the public at large about immediate financial assistance while navigating the rare, ultra-rare and undiagnosed journey is our biggest obstacle. Donating to a well-known, very large charity that funds (or at least appears to in their marketing) for research is often where people put their charity dollars. Patient assistance, especially in those small charities, is often looked down upon, as helping individuals does not help as many who could benefit from a cure. That would be true if we could be sure of a cure or a treatment in the very immediate future. For those stuck in the financial crisis of living rare, they may not have the benefit of a cure at all. However, they may benefit from transportation costs and lodging to see a team of specialists out of their immediate area. Families may benefit from a food stipend while spending time in the hospital with their child.
Helping to eliminate some of the financial burden offers both relief and Hope.