Help those in Scotland and Ireland access new treatment
Please allow 30 minutes to complete this survey and note that questions 21-32 may take 20 minutes.

We want to hear from people who have experience of living with inheritable hypophosphatemia’s about what it is like to live with the condition and their experience of current drug treatments and supporting care.

Your experiences will help inform our submission to the Scottish Medicines Consortium (SMC) and the National Centre for Pharmacoeconomics Ireland (NCPE) that are currently considering whether to recommend a new therapy for use in Scotland and Ireland.

The therapy currently being appraised is called burosumab. It has been developed to normalise the overactive hormone, FGF23, which is responsible for signalling phosphate wasting (hypophosphatemia). A similar call for evidence was made last year as part of the exploratory part of the appraisal in England and Wales with NICE. (NICE stands for The National Institute for Health and Care Excellence. It is an independent organisation, that decides which drugs and treatments are available on the NHS in England).

We are aware of patients receiving this therapy across England and Scotland, and therefore we are also expecting to receive responses from patients who are on conventional treatment. Instead, and just as importantly, we are asking for your experiences of living with the condition to help inform committee members appraising the treatment, who will likely have limited knowledge of this condition and its impact on a day to day basis.

If you are living in England, Wales, Scotland, Northern Ireland, Republic of Ireland, the Isle of Man or the Channel Islands, and are personally affected or a carer or friend, please complete with your experiences by November 2nd 2019.

Please feel free to share as little or much of your experience as you wish to. We would like to use some quotes in our submission, to illustrate the points we make, but will not use any names unless you indicate you would be happy for us to do this.

If anyone has any questions, please email contact@xlhuk.org
Are you completing this survey on behalf of *
What country do you live in? *
Age *
Gender *
Have you or your friend or family member been diagnosed with *
Including yourself, how many people in your family have been diagnosed with hypophosphatemia? *
Your answer
Were you treated with phosphate and/or calcitriol (active VitaminD) during childhood? *
Which symptom of your hypophosphatemia has the biggest negative impact on your life? *
Which symptom of your hypophosphatemia has the second biggest negative impact on your life? *
During childhood, were your symptoms *
During your 18-35 years, were your symptoms *
During your 36-60 years, were your symptoms *
During your 61-80 years, were your symptoms *
During your 80+ years, were your symptoms *
Since the symptoms of hypophosphatemia tend to be chronic, you may feel better or worse from day to day. With that in mind, and thinking about your worst days, what is the worst (highest) level of negative impact that hypophosphatemia has on your daily life? *
Thinking about your best days, what is the least (lowest) level of negative impact that hypophosphatemia has on your daily life? *
Thinking about your average days, what is the least (lowest) level of negative impact that hypophosphatemia has on your daily life? *
Do your symptoms of hypophosphatemia limit your ability to engage in any of the following activities as much as you'd like? (Choose all that are applicable.) *
Required
If you are currently being treated to normalise the phosphate levels in your blood, which treatment(s) are you using? (Choose as many as apply.) *
Required
In relation to hypophosphatemia, are you currently being treated for chronic pain, if so which treatment(s) are you using? (Choose as many as apply.) *
Required
In your own words, please tell us what is it like to live with an inheritable hypophosphatemia? *
Your answer
What do you think of current treatments and supporting care available on the NHS (or HSE in Ireland)? What impact has current treatment/surgery had on your symptoms and your quality of life? (please specify the treatment/surgery you received) *
Your answer
If current treatment/surgery have been unsuccessful, what has been the impact on your quality of life? *
Your answer
Are there any areas of services or treatments where your needs are unmet? *
Your answer
Do you think there are advantages/disadvantages to the new treatment? If you are taking burosumab, please also explain how it has helped or not helped you? *
Your answer
If you are taking burosumab, what kind of impact has this had on your family or carers? *
Your answer
Are there any disadvantages of taking burosumab compared to current standard treatments? *
Your answer
If you have a child with an hypophosphatemia, at the point of diagnosis, what information would have helped throughout you or your child's journey? *
Your answer
How did you arrive at your diagnosis? (the journey from first noticing any symptoms in you or your child to when the diagnosis was made) *
Your answer
What resources have been most important in the intervening years? *
Your answer
In your experience leading up to the diagnosis, what went well and what could have worked better? *
Your answer
Is there anything else you'd like to tell XLH UK, clinicians, researchers, the Scottish Medicines Consortium, and the National Centre for Pharmacoeconomics (Ireland) about living with x-linked hypophosphatemia? *
Your answer
Thank you for taking the time to share your experiences.
We would appreciate it if you could share the survey with any friends or family who are also affected by any hypophosphatemia to help us make our patient submission as effective as possible.
Submit
Never submit passwords through Google Forms.
This content is neither created nor endorsed by Google. Report Abuse - Terms of Service - Privacy Policy