If you haven’t seen the news already, the governmental agency (NICE) in England has decided NOT to pay for burosumab treatment of XLH adults. You can read about it, along with a call to action, at the XLH-UK website.
One of the actions is to submit comments to NICE. You’ll find a link at the XLH-UK website. Note that you need to set up a (free) account with NICE to comment, but there don’t appear to be any restrictions as to who can comment. Comments are open until January 3rd, but do it now, so you don’t forget or get overwhelmed by the holidays.
I strongly recommend writing out your comments BEFORE you go to the website and set up your commenting account. The box for comments is small, and not easy to work inside. If you write it elsewhere, you can copy and paste it into the box. You’ll have two options, one for general comments and one for answering questions. You’ll likely want to use the general comments option, since the questions are more geared toward scientists than to patients.
If you’re not sure how to write agency comments, I’ve got some advice, from my experience as both a writer and a lawyer (but this is not legal advice!).
- Keep it focused to exactly what the agency is asking about. In this case, they’re particularly interested in three topics: 1. Daily Life Impact, 2. Fractures, and 3. The friend/family support you require. You don’t need to cover everything or give your whole life history. It’s fine to pick one (or two) topics that you have solid anecdotes or examples to describe. For instance, I won’t talk about fractures or support, because I don’t have much experience with them. (I probably should ask for more help, but I don’t.) I can, however, talk easily about “any challenges in daily life such as pain, mobility/stiffness, fatigue and impact on social life and employment opportunities.”
- Keep it polite. I know you’re frustrated that they can’t see how necessary good treatment is, how much untreated adults suffer, and how terrible the old treatment is. I am too and would like to share some choice words with them. But it’s not helpful. If you have to vent, write an angry letter in a word processing file that you’ll then delete. Yell, rant, cry, throw things (safely), or whatever else you need to do to get it out of your system BEFORE you write your comment. Otherwise, you run the risk of being viewed as hysterical and not worth listening to. Notwithstanding the above, it’s okay to let some emotion come through. If you’re scared or in pain or frustrated, it’s okay to say that. Just try not to do the verbal equivalent of a toddler’s tantrum with stomping feet and name-calling (no matter how justified it may be)!
- Tell the truth. I know you won’t make up symptoms and challenges; there are plenty to choose from already! It’s more a matter of being honest about your suffering. This can be hard, since in my experience, most XLHers downplay our challenges, either intentionally or because we don’t recognize them as challenges. They’re just everyday life for us, and we’ve never known anything else. Don’t exaggerate, but also don’t minimize your experience. If someone asks you how you’re doing, you probably say “fine,” even on bad days. For this purpose, it’s important not to be nice, not to say the expected, but to dig deep and admit to your suffering. You don’t have to be (and shouldn’t be) melodramatic, but it’s okay to say that you live at a certain level of pain all the time. It’s okay to say you have to drag yourself to and from work, and then collapse at home afterward, unable to do anything for your own enjoyment. It’s okay to say you are constantly exhausted. It’s okay to say that you’re terrified of what the future will bring, in terms of surgery and disability and pain, because of the progressive nature of XLH.
- Use concrete examples from your own (or a loved one’s) life, if possible. Stories are more persuasive than simply saying a variation on “this treatment is life-changing.” Instead, you should give an example of how your life was changed by the treatment. They want to know …., so it can be really useful and really effective to share an experience where …..
- Keep it short (if you can – I have trouble with this one myself). It’s okay to respond to just one aspect of the call for feedback. Perhaps you have experience with one specific symptom or one specific disability, and can talk about the need for burosumab in that context. That’s great! You don’t need to reach for something to say about every possible symptom or disabling aspect of XLH. Find your strongest story, and use that. Other commenters will have different strongest stories, and together, we’ll present the complete picture!
It’s optional, but I like to write out my comment in a word processing file ahead of time. That gives me a chance to check for typos and also to cool off if I’m a bit too emotional. I’ll write it one day and then I’ll copy and paste it into the agency’s feedback form on another day.
Would you like some prompts and a basic structure, in case you’re nervous and need help getting started? Here you go.
First, introduce yourself, your XLH, and if relevant, your disability status:
I am _, and I live in [country]. I am [age] years old, and have X-linked hypophosphatemia.
I have been on burosumab for _ years. Before that, I was on the old treatment of phosphorus and active vitamin D supplements. I have had __ surgeries on my legs [and/or other joints/spine].
OR: I have been on the old treatment of phosphorus and active vitamin D supplements. I take _ pills a day, and require doctors’ appointments, blood tests, and imaging _ times a year. I still experience [list of symptoms].
[Whichever is relevant}:
I am totally disabled from my XLH.
I work full-time, but am in constant pain.
I work full-time, but take more days off than my colleagues, due to my XLH symptoms.
I work only part-time due to my XLH, and would like to work full-time if the pain and fatigue didn’t stop me.
I retired early at age , because of my XLH. I had to change careers from ____ to __, because of my XLH.
Next, pick a subject (or multiple subjects) you want to address, and a personal experience that demonstrates how much adult treatment with burosumab is needed:
Finally, tell them what you want, namely access to burosumab for adults.
Alternative, shorter version: I encourage you to find a story to share, since specific examples are most persuasive, but if it feels too overwhelming, it’s still worth making a shorter comment, just to add to the number of comments, which indicates how concerned patients are about this decision. In that case, it’s perfectly okay to simply say something like, “I have XLH, I suffer from [list symptoms], and my condition is getting worse. If I cannot get [or keep getting] burosumab treatment, I worry that I will [list two or three of your worst-case scenarios, such as: lose my job, involuntarily retire early, require multiple surgeries, lose my mobility, live in constant pain, be unable to provide and care for my family, become a burden to my family.].
Do it now! Please! I already posted mine, talking about calcification and the progressive nature of symptoms. I’ll post it sometime when I won’t be creating a monster-sized post.
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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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