When you first attend clinic, clinicians will assess how you are doing now so that they can have ‘standard baseline’ information. These include recording:

• nutritional status, including swallowing problems
• scoliosis
• fractures
• ventilation / respiratory events; respiratory function tests
• motor function (see below).

Our thanks to the Sheffield Team for talking this through:

Measures are often in the form of a scale which captures some ‘functional measure’ of your abilities. Which ones are used depend to some extent on age but also on what will provide useful information. More than one may need to be used. Many focus on ‘motor performance’. It is recognised that they only capture what this is like on the actual day of assessment. People can have ‘off’ days or really great days.

Measures are often in the form of a scale which captures some ‘functional measure’ of your abilities. Which ones are used depend to some extent on age but also on what will provide useful information. Many focus on ‘motor performance’. It is 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:

• WHO Motor Milestones (WHO MM)
• 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)
• The Adult version of the CHOP-INTEND, ATEND

Some scales record patient reported outcome measures, such as:

• The Egan Klassifikation 2 scale (EK2)

Another scale that is possible for some gives information on both walking ability and fatigue:

• The 6MWT (6-minute walk test)

At the beginning of treatment, the physio will discuss 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.

Ideally anyone stays on one main scale for the length of the Managed Access Agreement. However, it is recognised that it is essential to capture any changes. A scale someone is currently using may 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.

As not all scales measure the same thing, someone may be measured on more than one scale so that information that is important for 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)
  • the RULM (Upper limb strength / ability)

These all capture different aspects of what is going on. The physio will discuss if you /your child’s scales need to change or they need to use more than one scale.

NICE states

"A minimum of one data entry per patient per year is required to be captured after the initial assessment, with any two entries at least four months apart. Any missed clinic appointment for assessments should be rescheduled."

For each scale, the MAA defines how many points someone needs to drop for it to be considered that they have deteriorated. You can find more detail of this in:

Table 1. Endpoints, assessments and stopping rules on Page 7 of the Risdiplam Managed Access Agreement.

NHS England has also clarified that the equivalent measure of deterioration in the RULM scale is a deterioration of more than or equal to 2 points (>=2 points).

The MAA recognises that people can have ‘off days’ so states that there need to be two consecutive measures taken that both show deterioration.

NICE states:

"If the patient has a worsening in any motor scale score the patient’s next assessments must take place within the next six months. Any patient not complying with the assessment schedule (without good reason) may be deemed as not complying with the terms of the managed access agreement and access to treatment may cease."

However, if you are maintaining stability or improving on another scale that has been agreed as an important measurement for you, AND in the opinion of the treating clinician you are continuing to receive clinical benefit from the treatment you are receiving, then the MAA says:

"Continuation of treatment may be considered’ and 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 team about treatment options. In the unlikely event that you do not feel listened to you might want to get in touch with one of the Patient Groups to talk through possible options open to you.