Last updated 9th October 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 which will be open until 1st November 2018. Read more here. As of 7th May 2018, the Scottish NHS will now fund this treatment for those with SMA Type 1. 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.
NICE’s consultation paper was released on 14th August with the devastating news that they are not recommending it for funding by the NHS. The glimmer of hope, however, is that the paper encourages the possibility of a ‘Managed Access Agreement (MAA)’. This would be a 3 – 5-year scheme agreed between Biogen, NICE and NHS England that would enable access to treatment and the opportunity to collect further evidence of its clinical benefits and impact on quality of life. NICE is now reviewing all responses to their consultation paper and is in talks with NHS England, Biogen, clinicians and patient groups. Their next Committee meeting is on October 23rd with a final decision due to be published by 21ts November 2018. You can read about our response to NICE here and how you can help here.
On 8th October, the Scottish Government announced their new pathway for ultra-orphan treatments which gives some hope that nusinersen should be re-assessed for access by those with SMA Type 2 or 3. You can find out more 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.