Answers from the Panel to Follow-Up Questions from Webinar Viewers

Q.1 Is the centre of excellence still going to be in Birmingham and what do the timelines look like? How can I get to be part of this?

A. Birmingham has been confirmed as one of the 11 Specialist Centres that will offer nusinersen treatment for adults. We don’t have the timelines at the moment. Fiona Marley is looking into this.

Q.2. If not eligible for Spinraza due to spinal rods or delay due to covid, will risdiplam be looked at through EAP?

A. See this page

Q.3. What is a mobile arm support?

A. This is a device that fits to the wheelchair / assists with lifting your arm to your face, mouth and head. If safe, suitable and appropriate, your Occupational Therapist will advise on the most suitable model / what can be supplied, but below are a couple of examples to give you an idea of what’s on the market and how they work:

Neater Arm Support – powered
(been around for a few years) (We know this is used successfully by some people who have SMA)

Armon – a range of manual and powered arm supports

Some people with SMA find them really helpful but others find them difficult to adjust and use in a practical way.

Q.4. During the time it has taken John Radcliffe Hospital to set up their adult delivery service (as far as we're aware it's still not set up?) our 23-year-old son has lost the ability to walk. We understand therefore he is no longer eligible under the MAA. Is this correct?

A. At the moment this does seem to be the case. However, Fiona Marley (Head of Highly Specialised Commissioning at NHS England) has clarified that the Clinical Panel is going to be making the case for adults who have lost walking ability since the MAA was set up on 28th July 2019 to have access in view of the delays there have been with rolling out the treatment programme.

(SMA UK comments – we continue to be very active on this issue; our representative raised it at the Managed Access Committee in March as an urgent need and subsequently forwarding information about the impact the current lack of flexibility is having on individuals. We know this still doesn’t address the wider issue of access for  children and adults who have SMA Type 3 and have lost their walking ability but, as we hope you heard from the panel, there is a huge amount of work going on to gather evidence to endeavour to demonstrate to NICE that this needs to be the case.)

Q.5. I’m 23 and have SMA type 3 and can walk short distances with crutches does this count as being ambulant to qualify for nusinersen?

A. If you live in England your access to treatment is determined by the MAA eligibility criteria. For anyone age 19 and over who has Type 3, access is currently only possible for those who can ‘walk unaided’). This is the World Health Organisation definition as follows:

‘Is able to take at least five steps independently in the upright position with the back straight. One leg moves forward while the other supports most of the body weight. There is no contact with a person or object’.

In a walking unaided assessment, you can expect to have no splints, no socks and no shoes.

If you live in Scotland, you should be eligible for treatment, though it’s important to note that as yet we understand no one there has actually started treatment.

Q.6. When risdiplam becomes available, will priority be given to older (adults) with SMA Type 2-3 to get treatment i.e. Risdiplam or will priority go to children?

A. See this page

(SMA UK comments: we will make a submission to NICE once this process starts. As part of this we will work with other patient groups and  conduct a community survey. As part of this we  will ask for views on which if any groups people consider should have priority. It is impossible to predict what NICE’s recommendation will be. For more information on access routes and regulatory processes in the UK, please go here.)

Q.7. Hi, how many centres have already started administering nusinersen to Adults?

A. Data suggests two Centres have started treatment.

Q.8 Thank you for the seminar thus far. In readiness for being on treatment, in any form, is it advisable we start measuring our own strength and what format should we use? Is it the Hammersmith scale or something of that name?

A. Probably the best way to be ready for the treatment is to stay as flexible and active as possible.

If you are someone who has been prescribed a spinal brace, wear it as often and for as long as your physio has advised. This is designed to help keep your back as flexible as possible. Your stretching routine is also very important.

Activities like yoga or Pilates can be a good way to work on flexibility as well. Some Pilates videos for people who have neuromuscular conditions have been developed by colleagues at the University College in London and Oxford University Hospital and may be helpful – check with your physio to see if these may be helpful for you - watch more here.

Q.9 I don’t have a consultant or go to a Centre for care and management of my condition, but I am interested in nusinersen treatment possibilities. What should I do?

A. Contact your GP and ask for a referral to one of the 11 specialist centres that are designated to deliver the treatment.

Q.10 There are some individuals with type 3 SMA, who have not been able to walk for more than 12 months, who were accepted onto trials for the new drugs.  They have not been on trials before.  How is this the case and why is this happening for some and not all of us?  Are some individual hospital programmes making up their own rules or are you all supposedly following the same criteria, because this is not the case and as the community are in regular contact, we are very aware of the inconsistencies.

A. I think there may be confusion between a clinical trial and managed access programme. A clinical trial is funded by a sponsor, usually a pharma company. The sponsor develops and owns the trial protocol, which includes inclusion and exclusion criteria and the protocol is approved by ethics. The sponsor then chooses trial sites and agrees a contract with the hospital. The hospital running the trial must abide by the protocol.

The managed access program is agreed between the hospital trusts and NHS England. It is not a clinical trial. NHS England set the criteria for who is eligible for treatment and the Trust signs a contract with NHS England to abide by these conditions and to deliver the drug.

Q.11 My daughter age 22, type 3 non ambulant cannot take the 5 steps, so doesn’t fit the criteria. She has deteriorated to the point she is more a type 2 in strength.  My daughter is losing upper body strength fast, is unable to raise her arms above her head.  I’m concerned about the speed of the deterioration & not being able to access any treatment.

A. I do sympathise; hopefully Risdiplam may be an alternative in the future.

(Please also see answers to Qs 4 and 6)

Q.12 Not totally treatment related, but as someone who has only just re-entered the system of attending a specialist hospital, what should I expect from attending appointments? Will treatment be looked at first appointment?

A. Management should focus on standards of care: breathing/ chest, swallowing /nutrition, musculoskeletal care etc.

Treatment options, as well as emerging therapies, should be discussed and a plan made to determine eligibility for spinraza; this may require an additional visit and further imaging for some people. Not all Trusts have signed a contract yet with NHS England for Spinraza as negotiations are still underway and treatment pathways will need to be developed, so there may be a delay before treatment can be started.

Q.13 Have any of the physicians been given specific training/advice to administer the drugs to patients?

A. Biogen have produced guidance on how to administer Spinraza. However, local policies also exist as Spinraza is given intrathecally; doctors administering this injection need to be trained and accredited to do so according to local policy.