This week is the eighth anniversary of the FDA approval of burosumab (and the eighth birthday of my cats who were born the day before the announcement). Which means we’re coming up on the tenth anniversary of my starting on burosumab, and I’ll be talking about that in June before I go on my summer hiatus.
For now, the FDA anniversary has me thinking about the challenges that rare-condition patients have in getting a treatment approved. And how, if we were going through the process now, the FDA is such a mess and so resistant to both caring about rare diseases and trusting new-ish forms of treatment, it’s not entirely clear to me that burosumab (a biologic, which is somewhat new, albeit not as new as gene/cell therapy) would be approved if it had come up for consideration now.
I went back to see what I had in my blog about waiting for the approval, in order to refresh my memory, but I didn’t start writing here until several years later. The closest I have to documentation of the anxiety I felt in 2018 was this post from 2024, “Waiting For Our Lives.” https://www.ginjones.com/waiting-for-our-lives/ It’s about the anxiety over whether the British authorities would approve the use of burosumab for adults (after previously approving it only for kids).
The decision in England didn’t directly affect me, but it still triggered nightmares I still have about suddenly losing access to burosumab. At the time, I noted, “The anxiety [in England] 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.”
Fortunately, the authorities in England did approve access to burosumab for adults, but every year, this week’s anniversary reminds me that there are no guarantees for the future, for ourselves or for future generations of XLHers (and TIO patients). Until we have an actual cure (plus universal access), rather than an imperfect lifelong (and expensive) treatment, patients always live with that fear hanging over us—that someday the treatment will stop working or will be taken away from us for reasons beyond our control.
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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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