Page last checked: 19th December 2022

Last updated: 9th February 2022

This information is from the Risdiplam MAA on NICE’s website here.

If any of the MAA’s stopping critieria apply to you / your child, your treating clinician will decide whether to continue treatment. This will be discussed with you.

The criteria are:

• the patient is diagnosed with an additional progressive life-limiting condition where treatment would not provide long- term benefit such as terminal cancer or catastrophic brain injury.
• the patient uses a different disease-modifying therapy to treat SMA.
• the patient/family/carer withdraws their consent or is unwilling to comply to the associated monitoring, clinical assessments or sharing of data for the purpose of the MAA.
• the patient is not receiving benefit from treatment, as confirmed either by the annual additional Blueteq form (an online form completed by clinicians which feeds into a web-based system (Blueteq®) that triggers automatic approval for funding if the patient meets all the relevant criteria) or by meeting any of the stopping criteria within Table 1 Endpoints, assessments and stopping rules (page 7 of the MAA on NICE’s website).

Our page on assessments, scales and measurements tells you how they are used to monitor progress and to show whether treatment should stop.