On 3 June 2019 NICE released its final appraisal document (the appraisal committee’s final draft guidance about the NHS funding the treatment in England) and the draft managed access agreement (MAA) relating to Spinraza (also known as nusinersen). You can see all the documents on the NICE website. The draft MAA has caused great concern amongst the SMA community, patient groups and clinicians, and this week we have been working to seek changes to it whilst being mindful that we don’t want to jeopardise access to the treatment. We have been co-ordinating activity with SMA UK and Treat SMA and have been communicating with clinicians and with the pharmaceutical company Biogen.

What are our concerns?

Last month, NHS England described the NICE outcome and their agreement with Biogen as:

“…one of the most comprehensive deals in the world, meaning all relevant SMA patients (SMA 1, 2, 3a and 3b), including adults and siblings who are yet to show symptoms, will be able to benefit from this treatment.”

We feel that some of the current criteria in the MAA run counter to this and raise questions about who will be eligible for treatment.

Clinicians have reassured us that despite some of the starting criteria appearing inflexible they do in fact allow room for interpretation and will be subject to clinical decisions on a case-by-case basis. Additionally, we are assured by clinicians, NICE and NHS England that there will be an expert Clinical Advisory Panel that will oversee the MAA to ensure equity of access for patients and assurance for clinicians.

Our biggest concern relates to the point around ambulation of people with Type 3 SMA – under the MAA as drafted, people who were once ambulant but who are no longer so will not be able to access the treatment (although those who have never been ambulant will be able to). We believe that all people with Type 3 SMA should be able to access treatment via the MAA regardless of whether they are ambulant or non-ambulant. We have made this very clear to NICE and NHS England.

What are we doing now?

These are very difficult circumstances but our priority remains, as always, to secure access to this treatment for people with SMA as soon as possible. We have been told that an appeal will halt the process for all and add many months of delay to people receiving the treatment. We hope to avoid this and are currently exploring all options to secure some changes to the MAA. Today, we have co-signed a letter along with leading clinicians, SMA UK and Treat SMA which has gone to NICE and NHS England.

In the letter we have asked NICE and NHS England to consider the following:

Amending the MAA criteria now to include access to paediatric patients with Type 3 SMA who have lost ambulation based on the strong clinical evidence available.

As part of the MAA, to introduce 6 monthly reviews of new evidence for groups of patients currently excluded from the MAA and a commitment to amend the MAA if there is clear evidence of the clinical benefit.

Establishing an SMA REACH steering committee of clinicians and academics to ensure a uniform interpretation of the guidance and to discuss complex cases.

You can read full explanations of why we have taken this approach in the letter here.

Mary Glindon MP, Chair of the APPG for muscular dystrophy has also written to NICE to echo our concerns with the current MAA. In addition, along with SMA UK and Treat SMA we have also written to NICE to raise our frustrations with how their decisions have been communicated with the SMA community and the distress this has caused. You can read the letter here.

We know that this is a worrying time for families and adults who are waiting to hear when or if they will be able to access treatment. Rest assured that SMA UK, Treat SMA and ourselves remain committed to making sure NICE, NHS England and Biogen live up to their promise of making Spinraza available to all children and adults with SMA Types 1, 2 and 3, for whom it is clinically safe to do so. We will update the community as soon as we have anymore news.