Risdiplam Fast Track - access to treatments

Risdiplam (also known as Evrysdi) is a treatment for SMA, manufactured by Roche. On 19 November 2021, NICE recommended Risdiplam in England through a 3-year Managed Access Agreement (MAA). The agreement applies to people with 5q SMA, particularly who are 2 months and older with a clinical diagnosis of SMA types 1, 2, or 3, or with pre-symptomatic SMA and 1 to 4 SMN2 copies.

There is always a slight delay between a positive recommendation from NICE and the roll out of treatment. Therefore, following the publication of the NICE guidance, Risdiplam has been available in England since early 2022.

Risdiplam is a treatment for SMA which is shown to improve motor function in SMA types 1 to 3. There is some evidence suggesting that people with type 1 SMA who have Risdiplam  live for longer. It is an oral syrup medicine and can be taken once a day after meals. The liquid drug works by modifying the SMN2 gene to produce increased amounts of SMN protein, which is essential for the health of nerve cells that control muscle movement.

Individuals and their families should discuss appropriate treatment options with their clinical team in their specialist neuromuscular centre. The most optimal treatment for any individual will be based on a number of medical considerations, and individual patients’ choice.

It has been quite a journey to reaching this point. NICE published its first draft guidance for Risdiplam on 2 June 2021, which did not recommend the treatment for use on the NHS in England. NICE acknowledged that there was clinical evidence showing that Risdiplam improves motor function in SMA Types 1 to 3, but highlighted a lack of evidence regarding effectiveness in pre-symptomatic babies with SMA as well as its long-term benefit. They also stated that the cost-effectiveness estimates of Risdiplam were much higher than that which NICE usually considers an acceptable use of NHS resources. Since then we have been working closely with them by attending committee meetings and responding to evidence submission to demonstrate the benefit of the treatment and urging NICE to reverse its decision. 

More information on NICE’s assessment of Risdiplam can be found here.

What is the status of access to Risdiplam in Wales and Northern Ireland

Roche UK is in discussions with the relevant authorities in Wales and Northern Ireland respectively to ensure that this NICE guidance on Risdiplam can be adopted as soon as possible in these countries.

What is the status of access to Risdiplam in Scotland?

As of 7 February 2022, Risdiplam has been approved by the Scottish Medicines Consortium for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA type 1, type 2 or type 3 or with one to four SMN2 copies.

MDUK was actively involved in the SMA appraisal process. Working closely with SMA UK, we collected views from the SMA community on their experiences with Risdiplam. These views fed into our joint submission to the SMC in October 2021, in which we advocated for the SMC to approve Risdiplam for use on the NHS in Scotland. At this point, the SMA was not planning on recommending Risdiplam. After which, Roche requested a Patient and Clinician Engagement meeting which was held in January 2022. During this meeting, the SMC heard from key stakeholders including MDUK's Scottish Council Chair Sheonad Macfarlane, who shared her family's experience of living with SMA, and Dr Kate Adcock, Director of Research and Innovation at MDUK. As well as SMA UK's Liz Ryburn, Support Services Manager. 

More information on the SMC’s appraisal of Risdiplam can be found here.

If you are experiencing delays to roll out, even though you are eligible, please get in touch with us at campaigns@musculardystrophyuk.org