Medical research

Campaign to bring Spinraza to the UK

Regarding approval

The SMA Trust, in partnership with Muscular Dystrophy UK and SMA Support UK, has written to the EMA requesting assistance in ensuring nusinersen (Spinraza) is made available at the earliest possible time for the treatment of spinal muscular atrophy in patients in Europe. We have written to ask what steps the EMA can take to expedite its review and bring forward any decision on a licence for the drug and strongly urging them to consider the totality of the clinical trial data, and to grant as broad an approval as possible. Read the full letter sent on 11th January 2017.

If you are keen to lobby your local MP, we have prepared a template letter for individuals to use.

If you live in England, you can support the campaign by writing to your MP

If you live in Scotland, you can support the campaign by writing to your MSP

If you live in Wales, you can support the campaign by writing to your AM

If you live in Northern Ireland, you can support the campaign by writing to your MLA

Read more about how The SMA Trust is working hard to bring Spinraza to the UK and details of the NICE Scoping Workshop that took place on January 13th.

Regarding the Expanded Access Programme (EAP)

With SMA Support UK and Muscular Dystrophy UK, we have written a joint letter to Jeremy Hunt MP  (Secretary of State for Health), requesting support for the Expanded Access Programme (EAP) centres from the Department of Health, as whilst the drug is being made available for free there is currently no funding to support the additional infrastructure and capacity required by centres to deliver the treatment. Find out more on the EAP page of our website.

In addition, we are liaising with TreatSMA, the family led campaign group and working with SMA Support UK and Muscular Dystrophy UK towards the following goals:

  • Biogen continues to confer short term EAP status on any clinic in the UK that has a child in need of and eligible for treatment, where that clinic meets Biogen’s criteria
  • NHS England allocates new funds to support these potential short term EAP administering hospitals / local NHS Trusts with the ancillary costs (staffing and facilities) of delivering the programme
  • Long term efficiency and sustainability of the EAP via:
    • a national agreement on the number and location of ‘hub’ centres that will offer the programme
    • a national clinical consensus as to which children with SMA Type 1 should be eligible
  • NHS England funding for the ancillary costs of delivering the EAP then transferred to hub centres when these are finally agreed
  • Children who are being treated at ‘short-term’ EAP clinics transferred for treatment to the appropriate ‘hub’ centre once the hubs are operating effectively

Please write to your local MP, requesting he/ she writes to the Secretary of State for Health to ask, as a matter of urgency, for him and his staff to work with NHS England to review and make a positive decision on the proposal currently before them of a commissioning framework to support the provision of nusinersen, the new and first treatment for Spinal Muscular Atrophy (SMA). In the short term this would be so that eligible children with SMA Type 1 can access the drug, via the Expanded Access Programme (EAP). At the same time this would establish a longer term potential framework for access to the treatment by others affected by SMA. To help, please see this template letter

We will continue to do all we can to move the process forward, keep you updated and ensure that your voices are heard.