Kinda’ seems appropriate, in the wake of the Olympics, to talk about what it means to be a champion.
You all know how much I believe in the benefits of burosumab, so much so that, as a layperson, not a doctor, I think it should be the default treatment for both XLH and TIO (when excision of the tumor isn’t sufficient) and possibly the autosomal variants that are FGF23-mediated. I will tell anyone who asks (and even those who don’t ask) how I feel about it.
BUT …
But that’s my choice, as both a patient and a patient advocate. I’m not being paid to say it, and I wouldn’t accept payment if it were offered. And I’m most definitely not doing it so I can be a “family champion.” I don’t need the label, don’t want the label, and in fact find the label icky (for lack of a better word) when it’s being promoted by the pharmaceutical company that profits from patients on burosumab, rather than arising naturally within the community of people actually doing the work on behalf of patients/caregivers.
Despite believing in burosumab, I do NOT consider myself an advocate for it. I am an advocate for PATIENTS (and caregivers), not for a specific treatment. If something better comes along (and I do think there could be something better with gene therapy in the foreseeable future), I will definitely say so.
Which is why it really annoys me that Kyowa Kirin NA (KKNA) is trying to define “family champion” for us, to mean someone who sells their family members on burosumab.
No. Just no.
For one thing, not everyone has the skills, energy, or ability even to aspire to being a champion. Anyone with a rare disorder (or caring for someone with a rare disorder, or both) has enough on their plate without having to deal with the burden of being responsible for the wellbeing of other patients.
Even if a patient/caregiver does want to be a champion, it involves much more than simply telling people about a particular treatment. It starts with educating themselves and advocating for themselves, not just for a particular pharmaceutical intervention, but for the full range of health care services (dental, occupational therapy, physical therapy, etc.) that they need, so they can be as healthy as possible. It’s like the safety instructions on a plane — put on your own oxygen mask before you try to help others. If you’re not fully informed and healthy yourself, you can’t help anyone else. Then, and only then, if you have the time and inclination, and have the right sort of relationship with your family to offer advice, you might want to be sure any other family members have access to current information about living with XLH. Not just about one particular treatment, but about the whole range of issues that patients and caregivers experience. That means referring them to a qualified patient advocacy group, not to a pharmaceutical company.
The thing is — and I’ve said it before and will say it again — pharmaceutical companies are not your friend. They may well be doing good work (and I do think burosumab is the result of good work), but they still have interests (i.e., making as much money as possible) that do not necessarily align with your best interest. Their very existence depends on maximizing how many patients buy burosumab. Which is just the way our healthcare system works; maximizing profit is what pharmaceutical companies do. And I happen to agree that most XLH/TIO patients will in fact benefit from burosumab. But it’s still important to remember that pharma is not a disinterested source of information. They make (a lot of) money from patients going on their treatment, whether it works in a particular case or not.
And that’s why a family champion cannot be someone who refers family to an entity that has a conflict of interest. A family champion refers people they care about to a resource that is not making money from that referral, a resource that, from the moment of its formation and in its mission statement, is all about what’s best for patients/caregivers, not what’s best for a corporate bottom line.
Again, I have to qualify all of this by saying that the XLH information provided by KKNA at their websites is really good. It’s still not from the patient’s point of view as it would be if it came from a patient group like the XLH Network or the International XLH Alliance (or its local members). For health issues, always, always, ALWAYS start with an objective (and patient-focused) resource for your information (a patient group or a nonprofit like the National Organization for Rare Disorders or the National Institutes of Health), not one that is bought and paid for by a commercial entity. Once you’ve got the basics, if you want to see what additional information the pharma site has, that’s fine; they often have useful graphics and other resources that the nonprofits don’t have the money to create. But start with a solidly patient-focused and/or nonprofit source first. Then if you want to be a family champion, ask that patient-focused source what they recommend.
Ultimately, I think what galls me the most about this campaign by KKNA isn’t even a principled stance against relying on a potentially biased resource, but is more about how it is implicitly shaming patients who don’t have the time, energy, inclination, or skills to become sales reps for KKNA. The message, by implication, is: “If you were a good person — a family champion — you’d be out there, converting your friends and families to the way of burosumab.” It’s like missionary work, except for profit. And not for the profit of the person doing the work.
Look, I know it’s hard to reach patients who could benefit from burosumab, and I’m as frustrated as the next person with the challenges in doing that. I wish there were an easy solution, and that someone could comb through medical records to find the specific XLHers who have been so traumatized by the health care system, or by having been told countless times that there’s just no help for them, that they’ve given up and haven’t heard that there is, in fact, an effective treatment, one that could help them feel better, regardless of age or symptom severity. But we can’t do that, and we don’t really want a world in which pharma could swim through our medical records willy-nilly. So we’re going to have to reach those patients some other way. It will be a frustratingly slow process, but it’s up to the patient groups to reach out to patients/caregivers and provide them with encouragement to seek better medical care when they are able, and to educate clinicians in all the ways that their preconceived notions about chronic hypophosphatemia are wrong.
Some examples of what the patient community needs to be doing:
- Calling out all the mistakes in medical journals and textbooks and CME (e.g., reminding the authors that XLH is NOT a form of rickets, it’s not primarily a pediatric disorder, and it’s not possible to tell early in life whether a patient will have severe symptoms over the long term)
- Advocating for bringing phosphorus labs back to routine blood testing of toddlers for early diagnosis
- Educating pediatricians in a better response to parents’ complaints about an infant’s bowed legs
- Educating patients about the whole-body/whole-life nature of chronic hypophosphatemia
- Educating endocrinologists who treat adults about the whole-body/whole-life nature of chronic hypophosphatemia
- Providing endocrinologists who treat adults in general, but not XLH patients specifically, with comprehensive and easy-to-access resources to enable them to treat XLH patients
There is so much that patient advocates — including family champions, if they want to call themselves that — could be doing to improve the wellbeing of our friends and families in the chronic hypophosphatemia community. What we are not going to do, however, is to turn patients/caregivers into sales reps for pharma.
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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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