Patients in the England are waiting for a decision later this month about whether burosumab will be available for XLH adults there, waiting to find out if they’ll have a chance at a somewhat normal life instead of a dysfunctional one. Burosumab is available to kids there now, but not adults, except for a few who received it through what’s essentially a temporary compassionate-use program.
I can’t fully understand what patients there are going through, but my own personal nightmare is that after so many years of being somewhat functional on burosumab, the treatment could get taken away from me due to forces outside my control. I’m guessing that that’s a nightmare for a lot of patients on burosumab, and while it’s not as real and imminent as the situation in England, it’s not as unlikely to come true as it should be.
One of the symptoms that burosumab mostly eradicated for me is spinal spasms, which happen in a very specific spot where I have a calcification that can poke into my spinal cord if the area is inflamed. And when it happens, it’s breathtakingly, off-the-charts, paralyzingly painful for a second that feels like hours. I used to have at least one a day in the ten years or so before burosumab, but after a few months of treatment they became more of an infrequent event, happening maybe three or four times a year, mostly when I did something foolish that irritated that spot in my spine. They’ve became even less frequent over time, and I can’t even remember the last time I had one of these spasms before this week,. But they returned about a week ago, probably from spending too much time at my desk without a break, and they didn’t get better with my usual responses (anti-inflammatories and avoiding the activities that irritate my spine).
Part of me — the more well-grounded part — knows that this is just temporary and with a few more days of anti-inflammatories and rest, the irritation will settle down and I can forget about it again for months, possibly even years. But there’s also a part of me, the part that has nightmares about losing access to burosumab, that’s wondering if it’s possible that burosumab will lose its effectiveness with long-term use. After all, I’m coming up on the eight-year anniversary of starting treatment, and that’s longer than most patients have been on it, so there’s no data to predict the future course of my symptoms. It’s hardly reassuring when all the medical journals contain a caveat along the lines of “Burosumab has worked well so far, but we don’t know the consequences of long-term treatment.” What if one of the long-term consequences is that the body grows used to the anti-FGF23 antibody, and finds a way to ignore it?
I don’t really think that’s what’s happening to me at the moment (a few patients have reportedly developed a sort of antibody to the burosumab antibody, but it’s rare and seems to have happened early on in treatment, not after several years). Still, it’s a thought that lurks in the back of my mind, along with the (more common) possibility that my insurer could suddenly stop approving the expensive treatment. Or, as I’m seeing with other treatments in other settings, there could be a shortage of burosumab, requiring rationing.
For me, these worries are relatively minor and easily pushed out of mind, except during periods like now when my body is reminding me of just how badly it can hurt. But for patients in England, it’s a very large and very real worry at the moment. The anxiety is perhaps most acute for the patients who were fortunate enough to start burosumab on a temporary basis, because they know exactly how much their lives have improved with treatment. My heart breaks (and my rage is triggered) at the thought of them losing access and knowing that they will revert fairly quickly (not overnight, but over a few weeks or months) to their previous level of pain and dysfunction, and resume their slide into even worse pain and dysfunction. They’ll know, viscerally, not just in theory, how much their pain and dysfunction isn’t just a normal and/or unfixable part of their lives, but something that has been imposed on them by a society that doesn’t recognize their suffering.
I really hope that the regulatory agency’s decision won’t be that callous, and adults will indeed have access to burosumab in England. But living with the knowledge that our access to a treatment that’s unquestionably effective, can be denied for reasons that have nothing to do with medical science, is one more burden that rare-disorder patients carry. We know that our quality of life depends on factors entirely outside our control, so even the existence of a virtual cure is not the end of our struggles. We still have to fight until every member of our chronic hypophosphatemia community can access a true cure and not be dependent on the whims of other entities.
If you’re living with anxiety over access to effective treatment, first, I’m sorry you have to go through that, and I know how you feel! Second, please spare a thought for the #XLH (and TIO) patients in England, who are particularly affected by that anxiety at the moment. Let’s hope that the government agency does the right thing, and we can rejoice that one more segment of our global community can access truly effective treatment. Then, if you’re a natural-born worrier like me, you can find another part of our community that’s being denied access and worry about them instead!
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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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