4 Implementation


The Welsh ministers have issued directions to the NHS in Wales on implementing NICE highly specialised technologies guidance. When a NICE highly specialised technologies guidance recommends the use of a drug or treatment, or other technology, the NHS in Wales must usually provide funding and resources for it within 2 months of the first publication of the final draft guidance.


When NICE recommends a treatment 'as an option', the NHS must make sure it is available within the period set out in the paragraphs above. This means that, if a baby is aged 12 months and under and has presymptomatic 5q SMA with a biallelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene and the doctor responsible for their care thinks that onasemnogene abeparvovec is the right treatment, it should be available for use, in line with NICE's recommendations.

  • National Institute for Health and Care Excellence (NICE)