Updates: May 2021

The National Institute for Health and Care Excellence (NICE) announced on the 4 May 2021 that more people the rare genetic disorder spinal muscular atrophy (SMA) will be able to benefit from nusinersen (also called Spinraqza, made by Biogen) following a review of collected data as part of the Managed Access Agreement (MAA). This review involved Biogen, patient groups, clinicians, SMA REACH UK and NHS England and NHS Improvement assessing whether new evidence that had become available would support a change in the MAA treatment eligibility criteria. It specifically looked at whether people with type III SMA who are unable to walk could benefit from nusinsersen and therefore should be included in the MAA.

The MAA is a special arrangement between NHS England and NHS Improvement and Biogen. It allows patients to access treatment with nusinersen while at the same time collecting data on its impact in groups where additional evidence is required to address the uncertainties identified by the independent NICE appraisal committee in its original assessment of nusinersen. On top of this, the MAA also there to address the financial risk and challenges for implementation in the NHS.