Breaking news: NICE not recommending SMA treatment Spinraza on the NHS

Published Date
Clare Lucas

Today, the National Institute for Health and Care Excellence (NICE) published their first guidance for the spinal muscular atrophy (SMA) treatment, Spinraza. We are disappointed to share the news that they have decided not to recommend the treatment for use on the NHS.

We know this news will be upsetting for families and we will continue, along with the other charities, to fight for the only treatment for this cruel condition to be approved for people with all types of SMA.

We’d also like to remind you that we are here to support you. Please call us on our freephone helpline on 0800 652 6352, or alternatively you can call Samaritans on freephone 116123, if you would like to talk.

What did NICE say?

The appraisal consultation document (ACD) released today by NICE said that the appraisal committee recognised that Spinraza provided ‘important health benefits’ for patients with early-onset and later-onset SMA. However, having evaluated all the information and evidence submitted, they made the decision not to recommend Spinraza for use on the NHS.

Their reasons for rejecting the treatment were:

  • Spinraza has a marketing authorisation for the treatment of all types of SMA but the company only presented evidence for types 1 to 3. As such, it was felt the existing data for these types was lacking
  • the long-term benefits of the treatment were too uncertain
  • the cost of the treatment was deemed to be ‘too high for it to be considered a cost-effective use of NHS resources’.

In more encouraging news, NICE acknowledged that a managed access arrangement (MAA) was being looked at. This would mean that patients could get access to the treatment while further data was gathered on the treatment’s effectiveness. However, the committee said ‘the details of the company’s proposed MAA were vague and currently insufficient for it to assess whether it could be an option’. It was also noted that any progress towards an MAA would still require further negotiations on cost.

You can read Biogen’s community statement in response to the the decision here. 

What does this mean?

This is only the first guidance from NICE on Spinraza. The current recommendation means NICE would not make Spinraza available on the NHS. It is important to note that this decision does not have any impact on patients currently accessing the treatment via the Expanded Access Programme.

The ACD is now open to consultation for feedback from charities, clinicians and other key stakeholders, as well as to the general public on the NICE website. The consultation will close on Wednesday 5 September. Following this, the NICE committee will meet again on 23 October to review the feedback and reach a final decision.

Once published, the final guidance from NICE will apply to England. We would also expect the guidance to be followed in Wales and Northern Ireland. Scotland has a separate process, run by the Scottish Medicines Consortium (SMC), which has already concluded and the SMC recommended Spinraza for Type 1 patients. Spinraza is also set to be reconsidered for wider use in Scotland under the new ‘ultra-orphan treatment’.

You can read more on the details about Spinraza and the fight for access in our Q&A.

Two-year old Avery Ball (pictured above), from Braunston, Northamptonshire, has SMA Type 2. He was diagnosed aged 18 months, and requires 24/7 care. His dad, Alex, said:

“SMA is relentlessly cruel, and it turns your life upside down. To hear that your child will never be able to walk, or will struggle to sit independently, is heartbreaking. All your hopes, dreams and plans for the future are completely thrown off course.


”This news is such a blow to families like ours. For the first time, there is a treatment that can buy families more time to spend with their loved ones – yet we cannot access it.


“It’s agonising to see your child slowly lose their mobility. But it doesn’t have to be this way. By approving Spinraza, NICE would throw families like ours the lifeline we so desperately need.”

You can read more stories here about the people fighting for access.

What are we doing?

Commenting on the announcement from NICE, Chief Executive of Muscular Dystrophy UK, Robert Meadowcroft, said:

“Spinal muscular atrophy can be devastating and today’s news will be heartbreaking for the families of those living with the condition. Once again we are seeing families suffer due to the appraisal process being too limited to assess costly but life-changing rare disease drugs. The one glimmer of hope is for a temporary scheme that ensures access. We call on NICE, NHS England and the drug company, Biogen, to urgently come together and find a solution to provision and pricing so families can get Spinraza before more lives are lost.”

Muscular Dystrophy UK has been working hard to push for the MAA to be implemented quickly. The purpose of such a scheme is to gather additional long-term data while enabling patients to benefit from the treatment. A refusal to implement an access scheme not only prevents further data being gathered but also denies patients access to a life-changing treatment. We are calling on NICE, NHS England and Biogen to have agreed an MAA with the SMA community by NICE’s original decision date of 21 November 2018.

Today’s decision makes it clear that the appraisal process for rare disease drugs is not fit for purpose, and we will continue to fight for improvements so that people are able to access the support and treatments they need.

We will continue to campaign, along with the other charities, until the only treatment for this cruel condition is approved for people with all types of SMA. This is why:

  • our CEO, Robert Meadowcroft, has written to Health Minister, Lord O’Shaughnessy, and NHS England Chief Executive, Simon Stevens, calling for urgent action on establishing an MAA
  • we are arranging a drop-in meeting in Parliament for people to share their personal stories of life with SMA with their local MP
  • we are organising an emergency APPG meeting on access to Spinraza
  • we have contacted Health Minister Steve Brine MP’s office to request an update on the MAA and ask for him to attend the emergency APPG meeting
  • we are in touch with NHS England to secure a date for a stakeholder surgery
  • we are meeting with senior representatives at Biogen to discuss the issues raised by NICE around data and cost and to find out what steps the company are taking to progress the MAA discussions with NHS England
  • we will be responding to the consultation document to argue that NICE should review their recommendation and put an access scheme in place as soon as possible.

What can you do?

It’s vital that as many people as possible give NICE their views on what the paper says. They want comments on whether:

  1. All the relevant evidence has been taken into account
  2. The summaries of clinical and cost effectiveness are reasonable interpretations of the evidence
  3. The provisional recommendations are a sound and a suitable basis for guidance to the NHS

Muscular Dystrophy UK, SMA Support UK, the SMA Trust and Treat SMA are all working on responses. If you wish to make your own response you can do so via the NICE website.

  • You can share your personal stories and photos with us to inform our response.
  • Tweet at Lord O’Shaughnessy, NHS England and Biogen calling for them to find a solution before any lives are lost.

Here is a draft tweet you could use: We urgently need @nicecomms @NHSEngland and @biogen to come together and put an access scheme in place so people with SMA don’t miss out on accessing life-changing treatment, Spinraza #EveryDayCounts

There is more activity planned over the coming weeks to keep the pressure on. If you have any questions or want to get more involved, please contact Clare Lucas, our Campaigns and Engagement Manager, on

Want to talk to someone?

Please call us on our freephone helpline on 0800 652 6352, or alternatively you can call Samaritans on freephone 116123, if you would like to talk.

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