NICE to consult on decision not to recommend SMA treatment Risdiplam on NHS in England

Published Date
Alice Cachia
Orange box stating: Breaking news research

The National Institute for Health and Care Excellence (NICE) has today published its first draft guidance for Risdiplam, a treatment for spinal muscular atrophy (SMA).

We are disappointed to share the news that, following its appraisal, NICE is not recommending the treatment for use on the NHS in England – though this decision could be changed following a consultation that is now open.

Risdiplam is an oral drug that’s manufactured by the pharmaceutical company Roche. It can be taken at home and increases SMN protein levels by targeting the SMN2 gene.

It is important to note that the NICE recommendation will not affect people who started Risdiplam treatment on the NHS in England before today’s draft guidance was published.

Why hasn’t NICE recommended Risdiplam for use on the NHS?

In today’s announcement, NICE acknowledges that there is clinical evidence showing that Risdiplam improves motor function in SMA Types 1 to 3.

However, NICE has highlighted a lack of evidence regarding effectiveness in pre-symptomatic babies with SMA as well as its long-term benefit.

They also say that the cost-effectiveness estimates of Risdiplam are much higher than that which NICE usually considers an acceptable use of NHS resources.

What happens now?

It is not unusual for NICE to have questions about evidence and cost at this stage. MDUK knows this news will be upsetting for many families and, along with Spinal Muscular Atrophy UK (SMA UK), we will continue fighting for Risdiplam to be approved for use on NHS England.

MDUK is an official consultee in the appraisal process and we are working in partnership with SMA UK to gather and represent the views and experiences of people affected by SMA.

We therefore urge people to comment on the consultation. You can do so using the following link by 5pm on 23 June:

We hope that NICE changes its decision following the consultation, and we strongly encourage Roche and NHS England to reach a compromise on cost as soon as possible.

After the consultation, the appraisal committee will meet again to consider the evidence, the consultation documents, and comments from consultees.

The committee will then prepare the final appraisal document and, subject to any appeal by consultees following its decision, this may be used as the basis of NICE’s guidance on using Risdiplam on the NHS in England.

If Risdiplam does become available on the NHS in the future, it would – for the first time – give families and individuals living with the condition a choice of treatments alongside Spinraza (currently available through a Managed Access Agreement) and Zolgensma.

What do we think?

MDUK hopes that, with negotiation on treatment cost, NICE’s initial recommendation will change.

Robert Burley, Director of Campaigns, Care and Support, said: “Muscular Dystrophy UK is extremely disappointed at today’s announcement, especially because Risdiplam could be potentially life-changing for people living with SMA.


We therefore urge NICE, NHS England and Roche to work together as quickly as possible to find a cost-effective solution that will see the initial recommendation overturned.


Early diagnosis and treatment is vital so we appreciate this news may be concerning for families.


Muscular Dystrophy UK will therefore continue campaigning with SMA UK to represent the experiences of those affected by today’s announcement and strongly encourage as many people as possible to complete the consultation.”

What does today’s announcement mean for people in Scotland, Wales and Northern Ireland?

The manufacturer, Roche will be submitting an application to the Scottish Medicines Consortium in due course. Typically, the NHS in Wales and Northern Ireland follows recommendations made by NICE.



Anyone affected by today’s announcement who needs advice or guidance can contact our free helpline Monday – Friday between 10am and 2pm on 0800 652 6352. Alternatively you can email

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