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We are working with The SMA Trust, Muscular Dystrophy UK and the family led campaign group TreatSMA in the following ways:

The SMA Type 1 Expanded Access Programme (EAP)

Biogen, who initiated this global programme for children with SMA Type 1, has said that any child enrolled on the EAP will continue to receive the drug for their lifetime. This will either be funded by Biogen or, once there is an agreement about the provision of nusinersen in the longer term, by the administering country’s health authority.

Despite the efforts of clinicians across the UK, there continue to be unacceptable delays in the roll out of the EAP, especially in England. The main reason appears to be with funding / capacity of centres.

We are focusing our efforts on doing all we can to support any individual family with an eligible child to secure access to the treatment if they wish to. We are urging Biogen, NHS England and NHS Trusts to meet urgently to address the need for the EAP to be available to all eligible children with SMA Type 1, no matter where they live in the UK. Find out how you can support this effort by clicking here.

We want to ensure that:

  • Until the decision is made as to whether the NHS will take over all funding of nusinersen, Biogen will
    • continue to offer the drug free for current and newly diagnosed children with SMA Type 1
    • continue to confer short term EAP status on any clinic in the UK that meets their criteria and has a child eligible for treatment
  • There is long term equitable access, efficiency and sustainability of treatment of children enrolled on the EAP via
    • a national consensus, based on clinical evidence, as to which children with SMA Type 1 should be eligible and have priority, finalised and made public
    • centres working together now to ensure equitable access, based on this clinical prioritisation, no matter where a child lives
    • work taking place to explore the possibility of a national system that ensures equity of access based on these criteria, not on a postcode lottery
    • national agreement on whether treatment should be offered locally or by ‘hub’ centres, based on what is in the best interests of the child, not on any other arbitrary criteria
    • NHS England/ NHS Trust funding of the ancillary costs of delivering the treatment to all children who have been enrolled on the EAP

Long term access for those with SMA Types 1, 2 or 3

On 1st June 2017, the European Commission approved marketing authorisation of nusinersen or SpinrazaTM ,as it will be called, “for the treatment of 5q spinal muscular atrophy”, which includes SMA Types 1, 2 and 3. Read more here

In the UK, the availability of the drug now depends on a recommendation by the National Institute for Health and Care Excellence (NICE), NHS England, the Scottish Medicines Consortium and other authorities in the devolved nations to decide whether to fund the drug in England, Scotland, Wales and Northern Ireland. Availability may also depend on the treatment readiness of specialist centres. 

We expect to very soon hear of a ministerial referral to NICE and what path the appraisal of the drug for provision in the UK will take. The likely path is as a Highly Specialised Technology.

The NICE Process

New Highly Specialised Technology criteria introduced on 1st April 2017 threaten the possibility of nusinersen eventually being funded by the NHS in England and the long-term potential of this new treatment being available for those affected by SMA (for more information, see ‘Drug Funding and Approvals Process - changes to HST criteria’ on this page).

We have already advised decision makers of our goals as outlined below and, at the right time, will be continuing to press for these outcomes.

We want to see:

  • A referral at the earliest possible time to NICE for a full evaluation of the drug for funding by the NHS for all those affected by 5q SMA
  • Any finally agreed provision of treatment delivered in a way that will ensure equitable access based on clinical evidence, not on a postcode lottery
  • NHS England having early discussions with Biogen, to discuss a commercial agreement that could help expedite access to the drug and ensure that the new HST criteria do not create a barrier to access. To avoid delays, this could take place alongside any NICE evaluation.
  • All individuals and families affected by 5q SMA having the opportunity to voice their views to NICE on the provision of nusinersen 

You may also wish to read our information leaflet which summarises the treatment process and what SMA Support UK is doing to help get access to nusinersen in the UK. It is available to download if you wish to share it with friends and family.

how You Can Help