How Scales and Measurements Will Work Now for England’s MAA for Nusinersen

04 June 2021

Why are they used?

The nusinersen Managed Access Agreement (MAA) requires data to be collected about each individual person being treated. This is so that you and your clinical team can monitor if the treatment is working for you / your child – bearing in mind that stabilisation of your / your child’s condition is a key aim.

This data is also being collected overall so that towards the end of the agreement (July 2024) NICE can review all this information and assess whether nusinersen should be recommended for continued NHS funding.

What measurements and scales might be used?

When you / your child first attend(s) clinic, a physio assesses how you / your child are doing now so that they can have ‘baseline’ information. They use the best options they can from what measures are available and do recognise that these aren’t perfect by any means.   Most often measures are in the form of a scale which captures some ‘functional measure’ of your / your child’s abilities. Which ones are used depend on age and what will provide useful information.  Many focus on ‘motor performance’ and it’s recognised that they only capture what this is like on the actual day of assessment – people can have ‘off’ days or really great days.

Scales used include:

  • The Revised Hammersmith Scale (RHS),
  • Revised Upper limb Module (RULM)
  • CHOP- Infant Test of Neuromuscular Disorders (CHOP-INTEND)
  • Hammersmith Infant Neurological Examination – motor milestones (HINE)
  • WHO Motor Milestones (WHO MM)
  • The Adult version of the CHOP-INTEND, ATEND – which is in development

Some scales record patient reported outcome measures, such as:

  • The Egan Klassifikation 2 scale (EK2).

Some give information on fatigue such as:

  • The 6MWT (6-minute walk test) - but clearly aren’t useful for many who have SMA

How are measures and scales used?

At the beginning of treatment, your physio will discuss with you which scales are most suitable for you / your child. They will be looking for scales that can show current motor functional ability as well as ones that will show where change might occur for you / your child.

Ideally you / your child will remain on one key scale for the length of the Managed Access Agreement. However, it is recognised that it is essential to capture any changes happening and it may be that a scale you / your child  are currently using is no longer helpful. For example, a child may become too strong to be measured on the CHOP -INTEND so has to transfer to the RHS; a young child may no longer be able to do the CHOP-INTEND due to their age, so starts to be assessed on the ATEND.

Additionally, though, as not all scales measure the same thing, you / your child may be measured on more than one scale so that information that is important for you / your child is captured.  So, for example, an adult who is a non-sitter may be assessed on three different scales - the ATEND (motor ability measure), the EK2 (patient reported outcomes) and the RULM (Upper limb strength / ability) - as they all capture different aspects of what’s going on for you/ your child. Your physio will discuss with you if you/your child needs to change or use more than one scale.

What if one scale shows my abilities are deteriorating – what happens?

The MAA defines for each scale how many points someone needs to drop for it to be considered that they have deteriorated. The MAA also recognises that people can have ‘off days’ so states that there need to be two consecutive measures taken – with time between them (usually over two treatments) that both show deterioration. However, if you are maintaining stability or improving on another scale that has been agreed as an important measurement for you / your child, AND in the opinion of the treating clinician you are continuing to receive clinical benefit from the treatment with nusinersen then the MAA says ‘continuation of treatment may be considered’. It goes on to say that ‘These cases should be discussed with the NHS England Clinical Panel’. We suggest you don’t worry about this until or if you get to this point, at which time we would expect you to be having quality discussions with your / your child’s team about treatment options. In the unlikely event that you don’t feel listened to you might want to get in touch with one of the Patient Groups to talk through possible options open to you.