Update from the National Multidisciplinary Team (NMDT) on the progress of the Zolgensma Treatment Programme in England

03 June 2021

This newly formed team of lead consultants from the four infusion centres is meeting very regularly. They are working on the many operational aspects of this new national programme that need to be in place to ensure that it is delivered in the best most equitable way possible.

Age bands

It has been agreed that the age bands referred to by NHS England are as follows:

(a) Children with SMA Type 1 who are age 6 months or younger. This refers to calendar months and includes children below 7 months of age (e.g. if a child is born on the 1st January 2021, they will be 7 months old on the 1st August).

(b) Children with SMA Type 1 who are age 7 - 12 months. This refers to calendar months and includes children who are 7 months old up to when they turn 13 months (e.g. if a child is born on the 1st January 2021, they are 7 months old on 1st August and turn 13 months old on the 1st February 2022). 

Referral process

The NMDT has agreed what key information about the impact of a child’s SMA and their health, needs to be covered in a referral for treatment. This will mean that the referral process is equitable. 

There have been some delays with setting up the actual electronic referral portal which have been outside the control of clinicians and NHS England, but this is being addressed as quickly as possible.

Priority Groups

As capacity is limited while the programme starts, it is important that this is carefully thought through. In line with NICE guidance, two children from group (a) who had not received any previous treatment, have now been treated with zolgensma (by two different infusion Centres).

All referring Centres are now being contacted and invited to discuss with their nearest infusion Centre any children they care for who have had no treatment (treatment naïve) who are up to 13 months of age.

Work is currently taking place to consider other priority groups and how numbers can be managed equitably.

We have now set up a page based on the above, and will be adding any further updates from the NMDT here.

Other broader England Q&As are here.