Tel: 01789 267520


Last updated 19th January 2018.

Access in England, Wales and Northern Ireland – pushing for progress

On 18th January 2018, the National Institute for Health and Care Excellence (NICE) started its appraisal of Spinraza for the treatment of all types of SMA.

NICE is now looking at the treatment via the Single Technology Appraisal route. SMA Support UK, MDUK, the SMA Trust and TreatSMA have been invited to make Patient Group submissions by 15th March 2018. 

The STA route is designed to assess drugs for both clinical- and cost-effectiveness and is usually used for more common conditions. As such, we are concerned that this route is not set up to assess rare disease drugs, like nusinersen.                             

However, there is some promising news. Biogen, has 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.

Time is key and it is crucial nusinersen is not subjected to any delays during the NICE assessment process. We are working with MDUK, the SMA Trust and TreatSMA to push for the MAA to be implemented as soon as possible. Find out how you can help here.

We would expect guidance produced by NICE to be followed in Wales and Northern Ireland.

Access in Scotland - preparing for our submission

The Scottish Medicines Consortium (SMC) has announced that it will be assessing nusinersen for: "pre-symptomatic infantile-onset and later-onset 5q SMA, but not for before birth or Adult Onset SMA". This means they will be considering the case for SMA Types 1, 2, and 3 only. SMA Support UK, Muscular Dystrophy UK and the SMA Trust have each been invited to provide a Patient Group Submission by 5th February 2018.

On 18th December 2017, we invited individuals and families affected by SMA who live in Scotland to complete a survey of how SMA impacts on their lives and for their views on access to nusinersen. We are now collating the results of this to inform our submission and will share what people have said with the other charities.

The SMA Type 1 Expanded Access Programme (EAP) - voicing our concerns

There are now some 60 children receiving treatment via the UK EAP.

The interim NHS England policy (4th August 2017) will fund the hospital costs of administering this programme until NICE makes a decision on NHS funding. However it has limited which children with SMA Type 1 may have access, to those diagnosed by 7 months of age and with two SMN2 copies. We joined with clinicians and the other charities and advocacy groups to appeal these limits and with them have written to NHS England and submitted a Preliminary Policy Proposition (see NHSE letter)  about expanding the SMA Type 1 EAP criteria . 

We are very aware that it is not uncommon for a family to experience delays in diagnosis, how distressing this is and how unfair this limit on access would be for any family in this position. MDUK has a training programme on SMA for GPs. We also try to raise awareness of SMA within the health community. We need to push for more to be done so that children are diagnosed in a timely way. 

Biogen has said it will continue to review the future of the SMA Type 1 EAP and that its decision will depend on what progress there has been with NICE's appraisal of the treatment. We will continue to urge Biogen to keep the programme open for new children until such time as treatment of children with SMA Type 1 can be fully funded by the NHS. We hope the news yesterday (18th January) that there is some progress with NICE’s appraisal, will ensure they do. 

Access to new treatments in general –working to change the system

We are supporting MDUK’s efforts to highlight nusinersen in national media as a strong example of what is wrong with the system. See their Fasttrack campaign.‚Äč

We have committed to support Genetic Alliance UK, which has been challenged by the All Party Parliamentary Group (APPG) on Rare, Genetic and Undiagnosed Conditions, to work with a range of stakeholder groups to propose a method of making decisions about rare disease medicines that is ambitious, effective, transparent and fair. Read more here.