Evidence-based recommendations on nusinersen (Spinraza) for treating spinal muscular atrophy in children and adults.
Is this guidance up to date?
Next review: The guidance on this technology will be reviewed and published by the end of the managed access agreement in 5 years.
In January 2022, a second variation to the managed access agreement was agreed, broadening the treatment eligibility criteria to align with the criteria for risdiplam for treating spinal muscular atrophy and clarifying the criteria to take into account the availability of onasemnogene abeparvovec for treating spinal muscular atrophy.
In June 2021, a variation to the managed access agreement was agreed, extending it to cover treatment for people with type III spinal muscular atrophy who are unable to walk. For more information on the treatment eligibility criteria review process and a summary of the review outcome, see the evidence review process and supporting documents.
Guidance development process
The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to take this guidance fully into account, alongside the individual needs, preferences and values of their patients. The application of the recommendations in this guidance is at the discretion of health professionals and their individual patients and do not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian.
All problems (adverse events) related to a medicine or medical device used for treatment or in a procedure should be reported to the Medicines and Healthcare products Regulatory Agency using the Yellow Card Scheme.
Commissioners and/or providers have a responsibility to provide the funding required to enable the guidance to be applied when individual health professionals and their patients wish to use it, in accordance with the NHS Constitution. They should do so in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities.
Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible.