Last updated 16th May 2018.
Access to nusinersen in the UK is currently only possible for eligible children with SMA Type 1. In England, Wales and Northern Ireland, this is via the Expanded Access Programme As of 7th May 2018, the Scottish NHS will now fund this treatment. You can find out more about this decision here
Nusinersen’s future availability in the UK depends upon the outcome of reviews by regulatory authorities of the evidence gained from clinical trials in each subtype of SMA. These authorities also review the costs of provision and consider submissions from the patient community. The authorities include the National Institute for Health and Care Excellence (NICE), NHS England, the Scottish Medicines Consortium (SMC) and other authorities in the devolved nations.
On 18th January 2018, the National Institute for Health and Care Excellence (NICE) started its appraisal of nusinersen for the treatment of all types of SMA. It is now looking at the treatment via the Single Technology Appraisal (STA) route. SMA Support UK, MDUK, the SMA Trust and TreatSMA made Patient Group submissions advocating for access for all affected by 5qSMA who wish to, would potentially benefit and for whom it is clincially safe. Thank you to those of you who responded to our surveys which informed our submission.
An STA is designed to assess drugs for both clinical and cost-effectiveness and is usually used for more common conditions. We are concerned that this route is not set up to assess rare disease drugs, like nusinersen. However, there is some promising news. Biogen, announced that they are in discussions with NICE and NHS England to put an interim access scheme in place. This scheme, known as a Managed Access Agreement (MAA), could give others with SMA access to the treatment while further data and evidence are gathered over several years.
From what we understand, NICE’s decision about whether it will recommend nusinersen for funding by the NHS is expected to be published on 21st November 2018.
Any referral from NICE to NHS England to investigate setting up an interim MAA won’t be made until, at the earliest, after NICE’s first committee meeting (27th June) to consider all the evidence and submissions it has received. (NICE selects 'Patient Experts' to participate in this meeting. They are chosen by NICE from people nominated by the patient advocacy and support groups.)
At this stage it is impossible to say which Types of SMA and what ages might be included in such an agreement.
The work to investigate and set up an MAA is led by NHSE’s commercial and clinical teams. It involves discussions with SMA clinicians, patient groups and Biogen. This can take between 6 months – 2 years to finalise.
As there has been a lot of groundwork done already with the setting up of the SMA Type 1 Expanded Access Programme and with Centres already delivering the treatment, it would be reasonable to hope that an MAA for nusinersen could be achieved within six months from the time NHSE started its work.
Time is key and it is crucial nusinersen is not subjected to any delays during the NICE assessment process. Please help us push for the MAA to be implemented as soon as possible by writing to your MP and asking for their support. Find out more about how to do this here.
You can read more about the NICE process and what has happened thus far here.
On 7th May 2018, the Scottish Medicines Consortium announced its advice that NHS Scotland should fund the treatment for those with SMA Type 1 only. You can find out more, including next steps and how you can help here
- Wales and Northern Ireland
These countries will often follow the guidance issued by NICE but could follow Scotland.
As soon as we hear any further news on any of the above, we will let you know. You can keep up to date by signing up for our monthly E-newsletter.