1 Recommendations

1.1 Nusinersen is recommended as an option for treating 5q spinal muscular atrophy (SMA) only if:

  • people have pre-symptomatic SMA, or SMA types 1, 2 or 3 and

  • the conditions in the managed access agreement are followed.

Why the committee made these recommendations

SMA is a rare genetic condition, the most severe types of which affect babies and young children. Currently, there is an unmet need for effective treatments that could slow disease progression.

Clinical trial evidence shows that nusinersen improves a range of outcomes that are important to people with early- (type 1) and later-onset (types 2 and 3) SMA. Also, there is some evidence suggesting that nusinersen is effective for pre-symptomatic SMA. However, there is no long-term evidence, so the long-term benefits are highly uncertain. The committee considered that further data collection would help address these uncertainties.

The cost-effectiveness estimates presented are higher than what NICE usually considers a cost-effective use of NHS resources. However, these estimates are difficult to interpret because of the limited evidence base to substantiate longer-term benefits, the difficulty in clearly distinguishing between the SMA subtypes, and the difference in what can be achieved for these various patients without nusinersen.

The proposed 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. This recommendation 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.

  • National Institute for Health and Care Excellence (NICE)