Experiences of treatment for NICE evidence submission
Thank you for your interest in telling us your SMA story and your child’s experiences of treatment with nusinersen (Spinraza). Your views are really important. The following is not a questionnaire where you have to provide answers to certain questions. It is about you expressing your experiences in your words. This document merely provides guidance on the process and potential areas to consider. Please read this guidance note and then write your account on a separate piece of paper/ as a separate document in your own words.
Organisations representing the SMA community in the UK (including TreatSMA, SMA Support UK, the SMA Trust and Muscular Dystrophy UK) have been invited by the National Institute for Health and Care Excellence (NICE) to submit evidence on perceptions of SMA and attitudes towards and experiences of treatment. You may have already seen and completed the recent surveys from SMA Support UK on this subject. In addition to the information collected by SMA Support UK, TreatSMA are collecting detailed accounts of experiences of SMA and treatment from the SMA community. Our hope is to ‘make these issues real’ for NICE. Representing the experiences of patients receiving treatment and their families is a vital part of the NICE evidence submission process so your views are really important. We will ensure that as much information provided by families is put before NICE as possible.
Please read the information below and sign the form if you are happy to give consent for any testimonials and images you provide to be used.
What do I need to do?
We really want to provide NICE with the stories of patients and their families in their own words, especially focussing on experiences of treatment amongst those who have received nusinersen (Spinraza). Please tell us your views and experiences by providing a written account, it can be as detailed as you like. You can type up and email your story back to us or even write it and send it if you’d rather. It would also be really helpful for you to include photos or short videos of any changes to aspects of your child’s condition or abilities since starting treatment to make the issues as ‘real’ as possible. So if you are happy to send these along with your story that would be great. Also, if your child is old enough, and would like to submit a picture or any thoughts of their own along with your written account, that would also be welcomed. When sending in your written testimonial/ account and any photos or videos please also include the consent form at the end of this document in order for us to be able to use the information you provide. You do not need to include your name or your child’s name in the written account you provide if you do not wish to. Please email any written statements, photos or videos to
If you would prefer your statement to be totally anonymous, that is also fine. In that case please post a copy of your story to Kelly address.
Please make sure you send us any information by March 1st 2018.
If you require any further information please contact
Written account
Please write your account as a separate document and send it to us. Please provide as much information as you would like in what you write. Below are some ideas of things it may be useful to include in your statement if you wish to provide context for NICE, however this is not a questionnaire. The bullet points below are simply suggestions of areas you may wish to discuss in your written account. The content of your written account is totally up to you; we want to hear your experiences and what you consider to be important in your own words.
· Your child’s age
· The extent to which your child uses ventilation (NIV or trach)
· The age at which you noticed your child’s symptoms
· The age at which your child was actually diagnosed
· What approaches were suggested for managing your child’s condition prior to them receiving treatment? How successful were these in addressing certain aspects of the condition?
· How you heard about nusinersen (Spinraza)
· How you came to the decision to try nusinersen (Spinraza) for your child
· Your child’s age when they started nusinersen/ how long they have been receiving nusinersen?
· Your child’s abilities before starting nusinersen
· Any changes to your child’s abilities and health during the time they have been receiving nusinersen (this can include changes to any aspect of your child’s condition)
· If there have been changes to your child’s abilities and health, what has this meant for your child and for your family?
· If there have been any changes to your child’s abilities and health in the time that they have been receiving treatment, what impact has this had on their care needs? Have there been any changes to their care needs since starting treatment?
· Is there anything that you would consider to an advantage of treatment with nusinersen (Spinraza) that you have not already covered?
· Is there anything that you would consider to a disadvantage of treatment with nusinersen (Spinraza) that you have not already covered?
· Anything else you think that it is important to include. Please remember we want to know what is important to you and your family
Who will see what I write and any photos or videos?
The purpose of this exercise is to collect evidence to be reviewed by NICE. Therefore any details you submit may be sent to the NICE Committee and seen by the organisations involved in the NICE evidence review, including TreatSMA and other SMA organisations, NICE members and other government officials/ government bodies. We may also use quotes from written statements in our official submission, which will be evidence in the public domain.
Consent
Please fill out the section below if you have read the information in this form and are happy to give consent for TreatSMA to include any written testimonials, images and footage that you provide in the information we send to NICE. Please note that we can only use images/ footage that have either:
- Been taken by you
- Been taken by someone who you know and you have their permission to use the images
Please only sign this consent form and provide video footage or photographs if this is the case.
CHILD/ YOUNG PERSON NAME:……………………………………………………………………………….
PARENT/ CARER NAME:……………………………………………………………………………………………
PARENT/ CARER SIGNATURE:……………………………………………………………………………………