Access to an NHS-funded disease modifying second treatment (nusinersen or risdiplam) is only possible if the treating clinician has made a referral to the NHS England SMA clinical panel to say that, in their opinion, Zolgensma treatment has not been successful, and the clinical panel agrees. All other eligibility criteria for the second treatment would also apply.

The definition of ‘non-success’ was outlined in Appendix A of NICE’s final guidance. In April 2023, having by then considered a number of referrals, NHS England further clarified this definition in a communication to clinicians. NHS England agreed it would be useful for the community to be aware of this and agreed to SMA UK publishing the following:

“For Zolgensma™ to be agreed as having been unsuccessful, the clinician has to give evidence that the child’s respiratory function meets the criteria outlined below and / or the motor ability criteria, also outlined below.

Respiratory Function

The majority of cases that have been submitted have been put forward on the basis that there has been a deterioration in respiratory function.

The managed access agreement states that a deterioration in respiratory function is defined as:

‘… an increasing requirement for respiratory support overnight and/or, for that patient, an uncharacteristic increase in respiratory infections requiring hospital treatment that cannot be accounted for by aspiration or intrinsic lung disease.’

Following consideration of the cases, the Panel has considered information in detail and has shared the following guidance for clinicians submitting requests:

As set out in ‘A Guide to the 2017 International Standards of Care for SMA’, the use of a cough assist device is a recommended management technique for a child who has a weak cough and is not in itself an indication of deteriorating respiratory function.

As some children have had Zolgensma at an early age and may be in their first winter/year, it is not necessarily feasible to determine if a pattern of respiratory infections and any associated acute need for ventilation is uncharacteristic.

It is not unusual for children with SMA to require ADDITIONAL respiratory support during acute infections. This transient increase in support should be distinguished from cases where there is an enduring need for increased respiratory support.

Motor ability

A treating clinician may refer a child to the clinical panel for further advice if they have:

A total worsening in scale score demonstrated by two consecutive measurements from any two of the following three scales:

• More than 2 points on horizontal kick or 1 point on other HINE scores excluding voluntary grasp.
• More than 4 points on the CHOP INTEND scale.
• More than 3 points on the Revised Hammersmith Scale

A scaled equivalent of these losses would apply if a domain (one of the areas of ability to be measures) was unmeasurable or not suitable.

These scores are derived from the minimal clinical indicators of difference. For example, if a child deteriorates on one scale (e.g. loses more than 3 points on the RHS scale) but maintains stability or demonstrates improvement on another scale that has been measured since baseline (usually at the start of treatment) – for example the Revised Upper Limb Module (RULM), the child’s treatment with onasemnogene abeparvovec would be considered to be successful. A treating clinician must refer any case to the NHS England Clinical Panel for advice on non- success in respect of deterioration in scale scores.

AND / OR

A deterioration in respiratory function, defined as an increasing requirement for respiratory support overnight and/or, for that patient, an uncharacteristic increase in respiratory infections requiring hospital treatment that cannot be accounted for by aspiration or intrinsic lung disease.”