Page last updated: 8th November 2022

These 26th January 2022 answers are taken from the NICE website here.

Q.1 What are the clinical circumstances under which it would be appropriate for an individual to continue to have treatment with risdiplam (or nusinersen) after treatment with onasemnogene abeparvovec or to be initiated on treatment with risdiplam (or nusinersen) after treatment with onasemnogene abeparvovec?

A.1. All individuals being considered for treatment with risdiplam (or nusinersen) must meet the Starting criteria and must not meet the Stopping criteria set out in the treatment’s managed access agreement. These criteria include that the individual must not have had successful treatment with onasemnogene abeparvovec.

• Starting criteria for risdiplam
• Stopping criteria for risdiplam
• Starting criteria for nusinersen
• Stopping criteria for nusinersen

The definition of non-successful treatment with onasemnogene abeparvovec is as follows:

(a) A reduction in motor ability, defined as:

Total worsening in scale score corroborated by two consecutive measurements from any two of the following three scales:

• >2 points on horizontal kick or 1 point on other HINE scores excluding voluntary grasp
• >4 points on the CHOP INTEND scale
• >3 points on the RHS scale

A scaled equivalent of these losses would apply if a domain was unmeasurable / not suitable. These scores are derived from the minimal clinical indicators of difference. For example, if a patient deteriorates on one scale (e.g. loses >3 points on the RHS scale) but maintains stability or demonstrates improvement on another scale that has been measured since baseline (e.g. RULM), the patient’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

(b) 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.

This should be a permanent respiratory deterioration, not one linked to a recurrent infection.

This guidance covers:

• Children who have had onasemnogene abeparvovec only and have not previously been treated with other disease-modifying medicines for SMA
• Children who have had onasemnogene abeparvovec and who have had previous treatment with other disease-modifying medicines for SMA

Children in these two groups can only access other medicines for SMA within the NHS if treatment with onasemnogene abeparvovec is not successful, as defined above AND following advice from the NHS England Clinical Panel. The NHS England Clinical Panel provides expert advice to treatment centres about individual patients in respect of the Starting and Stopping Criteria. Individual treatment centres take decisions about individual patients.

Q.2. If a family has self-funded treatment with onasemnogene abeparvovec for their child abroad, can they have access to risdiplam (or nusinersen), regardless of their clinical situation?

A.2. The above criteria for access to risdiplam (or nusinersen) following treatment with onasemnogene abeparvovec is based on clinical criteria. It will not be relevant who paid for treatment with onasemnogene abeparvovec but rather how the patient responded to that treatment. This is important as it ensures that patients will not be disadvantaged (or advantaged) because of their economic circumstances and how previous treatment was funded, in line with the NHS constitution.