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Page last checked: 29th November 2022
Last updated: 9th February 2022

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

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 on Page 3 of the Nusinersen Managed Access Agreement Variation 2.
  • The effects of scoliosis and /or spinal fusion surgery make intrathecal administration technically possible or unsafe

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