Access to Spinraza- an update

Spinraza is the first and only treatment for people with spinal muscular atrophy. Below is a summary of some of the recent activity we have been involved in as part of our fight to make sure all people with SMA have the option of accessing this treatment.

Scottish Medicines Consortium submission

In February we sent in our submission to the Scottish Medicines Consortium (SMC). The submission focussed on patient testimonials about the impact of SMA, the lack of available treatments and the hope presented by Spinraza. In March, we headed to Glasgow with SMA Support UK and SMA Trust to support two parents, Sheonad and Kathy, at the Patient and Clinician Engagement (PACE) meeting. Both of them gave powerful testimonials about their own experiences of having a child with SMA and why they believe Spinraza should be approved. Earlier this week, we attended the SMC Committee meeting where presentations were made about the case for Spinraza to be approved. Following the presentations and discussions we were asked to leave the room whilst the committee voted in private on the availability of Spinraza in Scotland. We expect to hear the outcome of the vote in early May and will update people as soon as we know the result.

NICE submission

In January, the National Institute of Health and Care Excellence (NICE) began its appraisal of Spinraza. NICE is looking at the treatment via the Single Technology Appraisal route. This is designed to assess drugs for both clinical- and cost-effectiveness and is usually used for more common conditions. Throughout the process we have worked with the other SMA charities to make sure the patient voice is kept at the heart of the process. We made our submission to NICE in March. As with our submission to the SMC, the focus of our submission to NICE was on testimonials from people affected by SMA

Meanwhile we are still waiting for more detail about the potential Managed Access Agreement (MAA) for Spinraza. An MAA could see more people with SMA able to access the life-changing treatment while further data and evidence are gathered over several years.

Expanded Access Programme

There was good news in March when NHS England agreed to fund all children with SMA Type 1 (diagnosed by seven months) to receive Spinraza via the Expanded Access Programme. This is really positive news for those people who have been missing out until now. Special thanks go to Professor Francesco Muntoni who has fought tirelessly for this.

Finally, we would like to thank everyone who has shared their stories with us as part of this process. We will continue to work with SMA Support UK, SMA Trust and TreatSMA to fight for access to Spinraza for all people with SMA.