Risdiplam is an oral drug that increases SMN protein levels by targeting the SMN2 gene manufactured by Roche. The appraisal process will decide whether or not the treatment will be made available on the NHS in England.
MDUK is an official consultee in the appraisal process and we are working in partnership with SMA UK to gather and represent the views and experiences of people affected by SMA. This is a really important aspect of the appraisal process.
In preparation for the official response to the consultation that we will submit, we have launched a joint survey to gather these views and experiences.
The survey takes about 20 minutes to complete, depending on how much detail you wish to include in your comments. You will need to complete it in one go as the survey doesn’t allow for breaks. More than one person per family can take part in the survey, but please complete separate surveys for every individual who wants to take part.
You may find it useful to read summaries about risdiplam and the clinical trials, and refer to these when you complete the survey.
Decisions about treatment availability on the NHS in Wales and Northern Ireland tend to follow the outcome of the NICE appraisal process and the Scottish Medicines Consortium (SMC) will be conducting its own appraisal of risdiplam.
Risdiplam is already available to some people across the UK living with SMA through an Early Access to Medicines Scheme (EAMS). This replaces the Compassionate Use Programme (CUP) which we provided information about on 18th August 2020.
Under the EAMS, all Type 1 and Type 2 patients who have exhausted all therapeutic options (which in practice means anyone with SMA Type 1 or 2 who is unable to receive Spinraza for clinical reasons) will be able to access risdiplam if their clinician thinks this is appropriate.
It is important to speak to your clinician if you think you may be eligible to receive Risdiplam. Please note that a clinician can only refer an eligible patient for access to risdiplam if there are clinical reasons that mean they can’t receive Spinraza. People who were receiving Spinraza but who had their treatment interrupted as a direct result of the COVID-19 pandemic may also be able to receive risdiplam.
The EAMS is run by the Medicines and Healthcare products Regulatory Agency (MHRA) and you can read their announcement and further information about the EAMS on their website.
Roche have also provided answers about the scheme in response to questions from MDUK, SMA UK and Treat SMA. Read these questions and answers here.