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.