Lawyers use the term “standard of care” slightly differently from the medical profession, but in both cases, it refers to the bare minimum of care that one person owes to another to avoid liability. In the law, it’s usually based on a “reasonable person” test — what would a reasonable person do in the circumstances?
When the circumstances are health care, the bar is a little higher, and it becomes a question of what a reasonable health care provider do? For many medical conditions, there are literal standards of care adopted and promulgated by experts so, for instance, it’s clear that if a patient has blood pressure above 140/90, a reasonable clinician would take certain steps to help the patient lower the blood pressure. In fact, the American Heart Association has published a twenty-page document from the International Society of Hypertension laying out “guidelines,” which are basically the standard of care for treating high blood pressure.
Since very few rare disorders (reportedly only five percent of them) have an approved treatment, they can’t have a standard of care. At best, there’s an informal consensus among a handful of experts. That was true for XLH before 2018, when, to the extent there was an informal standard of care, it focused on pills (phosphorus and active vitamin D), plus a testing regimen (blood, urine, x-rays, kidney scans) and possibly surgery, but there was also significant variation in practice, with some clinicians adding calcium to the supplements, some adding human growth hormone, some recommending surgery early, and some recommending surgery late.
Everything changed for patients in 2018 when burosumab was approved by various governmental agencies, but there still isn’t any widely recognized standard of care. There are a couple journal articles (listed below) aiming to get at consensus, but 1) they’re limited geographically or by patient age, and 2) they rely too much, in my opinion, on the crutch of “we need more long-term data on burosumab before it becomes the default treatment” in the face of absolutely clear evidence of its superiority over the alternatives for patients of all ages.
One really noticeable omission from these initial discussions about a standard of care is the total lack of patient input. All of the articles are written by clinicians and researchers, with no representation by patients.
The XLH Alliance is working to change that. They’ve got a new survey that may help to give patients a voice in the development of a standard of care, not just for individual countries, but worldwide, which will help patients get the very best treatment, no matter where we live.
I’ve said before that our voice isn’t being heard. I’m out there, shouting into the headwinds, and I know many other advocates are shouting too, like all the representatives of the groups that make up the XLH Alliance. But we need your voice added to ours if we want to be heard. That’s why it’s so important to respond to this survey. The more responses the Alliance gets, the louder we are, the better chance that the clinicians/researchers/entities writing the standard of care for XLH will incorporate our feedback. I’d love to see in the vicinity of five hundred responses, and the last I heard, there are only about one hundred, so there’s plenty of room for your input!
Please do it now. If the link above doesn’t work, you can copy and past this address: https://xlhalliance.org/survey/ Or go to xlhalliance.org, and the survey button will pop up. It only takes a very few minutes, and the outcome will affect the quality of treatment for at least a generation of XLHers.
And finally, here are the articles I’ve found to date, which purport to be presenting a standard of care:
Clinical guidelines for burosumab in the treatment of XLH in children and adolescents: British paediatric and adolescent bone group recommendations
Consensus Recommendations for the Diagnosis and Management of X-Linked Hypophosphatemia in Belgium
X-linked hypophosphatemia and burosumab: practical clinical points from the French experience
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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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