Patient Advocacy Groups and Clinicians write to NICE & NHSE about the nusinersen MAA and the impact this has had on the SMA Community

14 June 2019

Today we have co-signed two letters to address the concerns that are being widely expressed about the nusinersen Managed Access Agreement (MAA).
Letter from SMA REACH Clinicians and Patient Advocacy Groups (PAGs)
This first letter has been led by Professor Francesco Muntoni with input from clinicians and PAGs.
We have been advised that an appeal will halt the final publication of NICE/NHSE guidance and therefore the progress of the roll out of the programme for all. This will add many months of delay to the possibility of anyone other than infants with SMA Type 1 receiving the treatment. To try to avoid this we are exploring all options to secure some changes to the MAA. In summary we have asked NICE and NHS England to consider the following:
  • Based on the strong clinical evidence available, amend the MAA criteria now to include access for paediatric patients with SMA Type 3 who have lost ambulation. 
  • As part of the MAA, introduce regular 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. 

Read the letter here

Letter from the Patient Advocacy Groups (PAGs) 
This second letter to NICE and NHS England raises our concerns about the way decisions have been communicated and the impact of this on the SMA Community. It asks for this to be taken into consideration when NICE and NHSE are making their next decisions and communicating them. We make it clear that we want to be able to work with them positively and collaboratively on the next steps of the MAA.
Read the letter here
Impact on the SMA Community
We are very aware how distressing this has been for many of you. Our Support Services Team is available by email and by phone during office hours. As we are a very small team, we are sorry we can’t provide support outside of these times, but we do know what a supportive community we have out there and that you are all there for each other.