The voices of XLH (and autosomal hypophosphatemias) patients aren’t being heard.
There’s a huge disconnect between the oft-heard claim in the medical commuity that “patients are the experts in their lived experiences” and the rejection of those experiences when they don’t fit the existing medical community’s narrative.
I believe that some of those who claim to respect the patient voice do, indeed, have good intentions. Some even walk their talk. The vast majority of those with good intentions still fail to live up to them at times, perhaps because old habits are so highly ingrained.
And then there are those who don’t actually believe the words. They view patients’ stories as either mistaken (stupid or ill-informed) or lying (drug-seeking).
I’ve been advocating for myself and other patients in the rare community long enough that I no longer care if it’s intentional or not. Either way, ignoring the patients is harming us. I don’t even care if an individual doctor believes that patients are experts. I only care that they act like they believe it, and that will require structural changes in the training (initial and continuing) of health care professionals, researchers, and media (editors and peer review boards).
First, Institutional Review Boards (IRB) that approve research protocols should include a patient from the rare disorder under investigation. A standard IRB for XLH research is likely to have an endocrinologist on it, but absolutely no guarantee that the endocrinologist has treated anyone with XLH, and it’s highly unlikely they’ve ever treated an adult with XLH.
Second, the U.S. Food & Drug Administration (and comparable entities like the European Medicines Agency) need to encourage treating patients as partners in the development process, not as afterthoughts. In particular, reviewing agencies should require that applications for a new treatment answer the question, “How many patients have reviewed the protocols and what did they have to say?” And then the agencies should make patient presence at development discussions with the agency the default, not an exception. At present, when a new treatment is proposed to the FDA, the patients CANNOT attend any discussions between the agency and the developing pharmaceutical company without the developer’s invitation, and few developers will issue that invitation. Patients are therefore excluded from the main meetings and given only a separate, tiny opportunity to speak to the FDA, with no ability to know what the developer has claimed, so we’re fumbling around in the dark. It’s a classic “separate but equal is inherently unequal” situation. And it’s a clear signal that neither pharma nor the FDA truly trusts patients or values our input.
Third, peer review for medical journal articles should include a reviewer who is a patient (or for purely pediatric conditions, a caregiver for a patient) with the condition being discussed. We may not be able to comment on the biochemistry and statistics, but we can certainly highlight harmful phraseology (like calling XLH a form of rickets, when it’s far more comprehensive than that), and we can raise questions that the researcher may not have considered due to a mistaken impression of the lived experience.
Working together, not on separate and unequal paths, can improve both the science and the patients’ lives.
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Please note that the author of this blog is not a doctor, but a well-read patient, and is not offering medical advice.
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