Resource impact statement
NICE has recommended nusinersen as an option for treating 5q spinal muscular atrophy (SMA).
Nusinersen will be available through the NHS in line with the managed access agreement with NHS England.
The managed access agreement details various risk management strategies, including patient selection, starting and stopping rules, data collection, patient consent, exit strategy and commercial offer. Taking these into account, nusinersen is recommended for people with pre-symptomatic SMA, or SMA types 1, 2 or 3 if the conditions in the managed access agreement are followed, including the collection of more data to address the uncertainties. The guidance will be reviewed based on data collected in the managed access arrangement. The review of the guidance will be published by the end of the fifth year.
It is estimated that between 600 and 1,200 children and adults are currently living with SMA, in the UK.
When NICE recommends a treatment as an option for use within a managed access agreement, NHS England will make it available according to the conditions in the managed access agreement.
This technology is commissioned by NHS England. Providers are NHS hospital trusts.
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