The U.S. Senate’s Special Committe on Aging is holding a hearing Thursday (October 26), and has invited public input. There’s still time (barely) to send your story about living with a rare and progressive disorder. Here’s the announcement of what they’re looking for:
Have you or a loved one been affected by a rare, progressive, or similar serious disease? Senator Braun, Ranking Member of the Senate Special Committee on Aging, will be holding a hearing on the unmet medical need experienced by people with rare, progressive, and serious diseases face, on Thursday, October 26 at 10:00AM. We want to amplify your experiences and make sure that your story is heard by Congress. You can submit your stories to the Committee for entry into the record at PatientStories@Aging.Senate.gov
There are plenty of topics that XLHers have experience with, like the need for a cure rather than just a treatment, the cost of treatment, the need for accommodations, and so on. I encourage you to find a topic and write about your experience. For greatest impact, pick just one topic and how it’s a little different to age with XLH than without a progressively debilitating disorder. Or how it’s different to age with a rare disorder than a more common one.
I chose to write about the horror of Prior Authorization, the insurance companies’ requirement that expensive treatments be reviewed every year (or more often). Here’s my letter:
Dear Senator Braun and the Senate Special Committee on Aging:
October 23 is International XLH Awareness Day, so it seems fitting that I should share my story about aging with XLH today. I have x-linked hypophosphatemia (XLH), a rare genetic disorder that causes progressive function/mobility restrictions, along with widespread fatigue and chronic pain. You can read more about it in “Whole Body, Whole Life, Whole Family: Patients’ Perspectives on X-Linked Hypophosphatemia,” published in the Journal of the Endocrine Society, and available here: https://academic.oup.com/jes/article/6/8/bvac086/6607648
I had relatively mild symptoms as a child, but once I hit young adulthood, they started to worsen and, over time, became severely disabling. By the time I was forty, I had the arthritis, spinal stenosis, and mobility restrictions of a not-very-healthy person in their eighties. By the time I was 53, I was totally disabled, even from significant, sedentary work. And the symptoms just kept getting worse until, at age 60, I started a new treatment, which couldn’t undo the underlying bone and calcification damage, at least stopped the progression, and alleviated the pain/fatigue, making me more functional than ten years earlier!
Despite finally having an effective treatment, the problem that I, and everyone else with a progressive disorder, face is the prospect of having that effective treatment yanked away from us with no warning and without any medical justification. I’m referring to “prior authorization” requirements. (Step therapy requirements are a similar problem for children with XLH, who are forced to try an old, ineffective and potentially dangerous but cheap treatment before they can start the newer, truly effective, and more expensive treatment. This is particularly outrageous given that time is of the essence for bone growth and mineralization during childhood.)
Prior authorization may make sense initially, for the insurer to confirm the diagnosis and the lack of any other effective treatment options. That’s annoying, but manageable. The problem comes when that prior authorization applies to renewal of the prescription every year (or sooner). Progressive disorders, whether rare or common, necessarily require treatment for the patient’s entire lifetime. In the XLH context, once the initial authorization is granted, the scientific consensus is clear—the treatment should continue for the patient’s lifetime (absent the development of side effects, in which case, the clinician and patient, not the insurer, would adjust the treatment).
It’s particularly obvious that a lifetime of treatment is necessary for XLH patients. We do not (yet) have a cure, and there is only one effective treatment. If a patient has the genetic variant that causes phosphate-wasting, they will never have normal phosphorus metabolism without this new treatment, and phosphorus is one of the most critical minerals in the human body, necessary for the health of bones, teeth, nerves, muscles, and energy levels.
No one would doubt that a patient, once diagnosed with osteoporosis or a heart condition would require non-stop treatment for the patient’s lifetime. It’s only when the condition is rare and poorly understood, and/or the treatment is expensive, that the insurer imposes the “prior authorization” requirement, not just for the initial approval, but to annual (or shorter) renewals. In the XLH context, and presumably many others, there cannot be any scientific/medical justification for this requirement. It is just an attempt to delay incurring the cost, while the patient’s health deteriorates. For a patient with XLH, the consequences of going off treatment (poor bone mineralization and increased soft-tissue calcification) can be permanent, with the effects lasting for the rest of the patient’s life.
The harm goes beyond the physical too. Even if the treatment is authorized before any doses are missed, the stress of uncertainty is overwhelming. Our treatment is a once-a-month injection, and many of us begin to feel an increase in pain and fatigue (signs that our phosphorus levels have dropped) a few days before the next dose is due. If we go a week beyond the due date, we experience severe fatigue and bone pain throughout the body, among other symptoms. More than a week, and I don’t even want to think about how badly I’d hurt or how physically dysfunctional I’d be.
The prospect of suddenly having XLH treatment withheld reminds me of a short story by the acclaimed mystery writer, Dorothy Sayers, in which the inimitable detective confronts the mystery of a once-vibrant woman who had become a mental and physical mess over a short period, and he eventually determines that her evil husband had been withholding her thyroid medication. (The story is “The Incredible Elopement of Lord Peter Wimsey.” See https://www.goodreads.com/en/book/show/40679677.)
That’s the horror that XLH patients (and all patients with a progressive disorder) live with when they have an effective treatment that is subject to ongoing prior authorization requirements. We are constantly aware that access to the treatment that allows us to function is subject to the whims of a potentially villainous third party, and we cannot rely on having that access beyond the current authorization period. For us, it’s not just a bit of fictional nightmare-inducing horror, but the reality of our lives, that our quality of life is totally controlled by the whims of a third party that is motivated by money, not by what’s best for patients.
I’m sure the XLH community is not the only one suffering from the abuse of prior authorization requirements that are entirely profit-inspired, with no justification whatsoever in medical science. It’s time to require insurers to follow the science, not the money. Patients with progressive disorders should not have to live in the constant and unnecessary fear that today, or next week, or next month will be the last time we’ll ever have any reasonable quality of life before the lack of treatment sends us into a rapid decline.
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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