Q & A - Access to NHS Zolgensma Treatment in England

Page last updated: Monday 16th March 2021

We have asked NHS England and Novartis Gene Therapies these questions, gathered from all patient groups. Some answers are now known and are included below. We will publish other answers as soon as we have them.

Q. Does a child have to live in England to be considered for treatment?

  • If someone lives in another part of the UK: Can they move permanently or temporarily to England to access the treatment? If so – are there any time limits on how long they have to have been at the English address or any other criteria?
  • What about people from outside the UK?

Q. Which groups have been identified as potentially eligible by NHS England?

A. The NHS England agreement includes five particular groups of children living with SMA Type 1. A group of experts (see below) will now work on a policy document to outline criteria for these groups and the information that a child’s clinician will need to share with the treatment centres, and which may be discussed by the National Multidisciplinary Team (see below). This will enable clinicians to have initial discussions with each family about possible access.

  • Children with SMA Type 1 up to 6 months of age.
  • Children with SMA Type 1 over 6 months of age and below 12 months of age.
  • Children with SMA Type 1 who are over 12 months old within the scope of the drug’s European Medicines Agency (EMA) marketing authorisation.
  • Children with SMA Type 1 who have been using treatment e.g., with nusinersen (Spinraza) or Risdiplam (Evrysdi) and fall within the scope of the European Medicines Agency marketing authorisation. While NICE have said that there is not currently evidence for using onasemnogene where a child has had nusinersen, our expectation is that the multidisciplinary team will consider such cases and may recommend onasemnogene for those with SMA Type 1 if there is a clear clinical case.
  • Any pre-symptomatic SMA infants identified who have up to 3 copies of the SMN2 gene who may also have been receiving treatment e.g., with nusinersen (Spinraza) or risdiplam (Evrysdi).

Q. Can you tell us more about the Expert Group that will work on the clinical criteria?

  • Who will be on the Expert Group?
  • When will the Expert Group start meeting?
  • When will the policy be announced?

Will the policy tell us:

  • At what point the age bands and different criteria apply e.g., at the age at the time of the assessment, or at the time of the treatment etc?
  • About any maximum weight limit?

Q. When will we know where the four Centre(s) are that will offer the treatment?

Q. We know that representatives from these Centres will be on the National Multidisciplinary Team (MDT) that will ‘play an important role in allowing clinicians and families to make carefully informed decisions about whether a child will clinically benefit from Zolgensma’. When will we know who is on the MDT?

Q. The AVV anti body test is essential to see if treatment would be possible (read why here):

  • At what point would my child have an AVV test – before eligibility is discussed or later in the pathway? When and where would this be done?
  • How long does it take to get the result?
  • Do we need to isolate during this time? If so, why is this?
  • What if I am breastfeeding and the test comes back showing antibodies – how long would I have to stop breastfeeding for a further test to see if treatment is possible?

Q. When can I expect to have a full discussion with my clinician and know if my child will be eligible?

  • At what point would my child’s eligibility for treatment be confirmed – could their eligibility change at any point while they wait for treatment?
  • Do I sign an agreement for treatment that is then binding on the Centre / NHE England?
  • If my child is not considered to meet the criteria, will there be any appeal process for families?

Q. When and how will the four Centres start giving treatment?

  • When will we know where they are?
  • When will they start treatment?
  • Will they operate regionally or nationally? 
  • Will there be a priority order for treatment? If so, what will this be, and would it be national or regional?
  • How many children will the Centres be able to treat within the first months of the service being up and running?
  • Will there be any cap on overall numbers?

Q. Will my child have to wait to be treated?

  • What will the timeframe be between being assessed and being treated?
  • If my child is not on a priority list how long might I have to wait?
  • Could my child be about to have treatment and then the appointment is cancelled due to a new priority child being identified?
  • What if my child’s age during this time means they come under another eligibility category?
  • What if my child’s weight is on the boundary of being eligible?
  • What if when they are assessed they are within the weight limit but while they wait for treatment they put on weight?

Q. If my child is on another treatment at the moment and eligible to move to Zolgensma:

  • Do they need to come off the other treatment?
  • How long do they need to do this before they can have Zolgensma treatment?
  • Can my child resume the other treatment after they have had Zolgensma?
  • After having had Zolgensma, can my child have a further treatment later on?

Q. The treatment process – (once the AVV test results are back and clear)

  • Where will I need to go/ who will I need to see to discuss my child’s treatment?
  • What tests and ‘baseline’ assessments take place before the treatment
  • Why do you do them?
  • How long does it take to do them?
  • Where do we need to be for them?
  • Do we need to isolate during this time?

Q. How is the actual treatment delivered?

  • How long does it take?
  • Does it hurt?
  • What if my child is too unwell for treatment on the day, or there is a problem with staffing or treatment delivery at the Centre?

Q. Immediately after treatment what happens?

  • How long do we need to stay at the Centre, when can we go home?
  • What care do we need to put in place when we get home?
  • How long do we need to do this for?

Q. Do I have to isolate with my child?

  • While the tests are being done?
  • During treatment?
  • Immediately afterwards?

Q. What support will there be while my child accesses this treatment:

  • What help will we be given to get to the treatment Centre and with accommodation and food during all these stages?
  • What about the rest of my family – who will take care of them? Time off work / for a partner to do all care?
  • Who will arrange all this?

Q. What about longer-term aftercare

  • What special care do I need to give my child?
  • What do I need to watch for?
  • What follow up tests / assessments are there?
  • Where do these take place?
  • Do we still need to isolate?

Q. What support will be provided throughout and following treatment, including emotional and psychological?

Q. What information will be collected?

  • What data will be collected and how?
  • Clinical Data Collection
  • Patient Reported Outcome Measures (PROMs)

Q. Where will all this information be kept?

Q. Who will see the information?

Q. What will you do with it?

Q. Will England offer private access now?

  • To people living in England who don’t want to wait and have the funding?
  • To people living in other parts of the UK?
  • To people from outside the UK?
  • If yes to any of the above groups, what are the eligibility criteria?

Q. What is Zolgensma and how does it work?

SMA UK explains how it works and summarises the clinical trials. We hope to bring you more detail.

  • Explanation of the term 'gene therapy' (is this a permanent genetic change? would it be passed on to future generations?)
  • Explanation of the mechanism (Which cells and tissues in the body does it reach?)
  • What have clinical trial results and other evidence shown about the possible outcomes with zolgensma treatment?
  • Are there any ‘real world’ studies of outcomes? If so, what do they show?
  • How long since the first children have been treated, how old are they now? How are they doing? (trials and real-world studies)
  • What are the risks / any safety concerns?
  • How do the results that have been seen so far compare to nusinersen?